B ay, Na han; Spence , Llinos Ha ; Edwa ds, Rhiannon Edwa ds
A icle
P e e ence-based measu es o heal h ela ed quali y o li e
in congeni al mobili y impai men : A sys ema ic e iew o
alidi y and esponsi eness
Heal h Economics Re iew
P o ided in Coope a ion wi h:
Sp inge Na u e
Sugges ed Ci a ion: B ay, Na han; Spence , Llinos Ha ; Edwa ds, Rhiannon Edwa ds (2020) :
P e e ence-based measu es o heal h ela ed quali y o li e in congeni al mobili y impai men :
A sys ema ic e iew o alidi y and esponsi eness, Heal h Economics Re iew, ISSN 2191-1991,
Sp inge , Heidelbe g, Vol. 10, Iss. 9, pp. 1-38,
h ps://doi.o g/10.1186/s13561-020-00270-3
This Ve sion is a ailable a :
h ps://hdl.handle.ne /10419/285161
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REVIEW Open Access
P e e ence-based measu es o heal h-
ela ed quali y o li e in congeni al mobili y
impai men : a sys ema ic e iew o alidi y
and esponsi eness
Na han B ay
1,2*
, Llinos Ha Spence
1,2
and Rhiannon Tudo Edwa ds
1,2
Abs ac
In oduc ion: Mobili y impai men is he leading cause o disabili y in he UK. Indi iduals wi h congeni al mobili y
impai men s ha e unique expe iences o heal h, quali y o li e and adap a ion. P e e ence-based ou comes
measu es a e o en used o help in o m decisions abou heal hca e unding and p io i isa ion, howe e he
applicabili y and accu acy o hese measu es in he con ex o congeni al mobili y impai men is unclea . Inaccu a e
ou come measu es could po en ially a ec he ca e p o ided o hese pa ien g oups. The aim o his sys ema ic
e iew was o examine he pe o mance o p e e ence-based ou come measu es o he measu emen o u ili y
alues in a ious o ms o congeni al mobili y impai men .
Me hods: Ten da abases we e sea ched, including Science Di ec , CINAHL and PubMed. Sc eening o e e ence lis s
and hand-sea ching we e also unde aken. Desc ip i e and na a i e syn heses we e conduc ed o combine and
analyse he a ious indings. Resul s we e g ouped by condi ion. Ou come measu e pe o mance indica o s we e
adap ed om COSMIN guidance and we e g ouped in o h ee b oad ca ego ies: alidi y, esponsi eness and
eliabili y. Sc eening, da a ex ac ion and quali y app aisal we e ca ied ou by wo independen e iewe s.
Resul s: A o al o 31 s udies we e conside ed eligible o inclusion in he sys ema ic e iew. The as majo i y o
s udies ela ed o ei he ce eb al palsy, spina bi ida o childhood hyd ocephalus. O he ele an condi ions included
muscula dys ophy, spinal muscula a ophy and congeni al club oo . The mos commonly used p e e ence-based
ou come measu e was he HUI3. Repo ing o pe o mance p ope ies p edominan ly cen ed a ound cons uc
alidi y, h ough known g oup analyses and assessmen o con e gen alidi y be ween compa able measu es and
di e en ypes o esponden s. A small numbe o s udies assessed esponsi eness, bu assessmen o eliabili y was
no epo ed. Inc eased clinical se e i y appea s o be associa ed wi h dec eased u ili y ou comes in congeni al
mobili y impai men , pa icula ly in e ms o g oss mo o unc ion in ce eb al palsy and lesion le el in spina bi ida.
Howe e , p e e ence-based measu es exhibi limi ed co ela ion wi h a ious o he condi ion-speci ic and clinically
ele an ou come measu es.
(Con inued on nex page)
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* Co espondence: [email p o ec ed]
1
School o Heal h Sciences, F on Heulog, Bango Uni e si y, Gwynedd LL57
2EF, Wales, UK
2
Cen e o Heal h Economics and Medicines E alua ion, A dudwy, Bango
Uni e si y, Gwynedd LL57 2PZ, Wales, UK
B ay e al. Heal h Economics Re iew (2020) 10:9
h ps://doi.o g/10.1186/s13561-020-00270-3
(Con inued om p e ious page)
Conclusion: P e e ence-based measu es exhibi impo an issues and disc epancies ela ing o alidi y and
esponsi eness in he con ex o congeni al mobili y impai men , hus ca e mus be aken when u ilising hese
measu es in condi ions associa ed wi h congeni al mobili y impai men s.
Keywo ds: Disabili y, Mobili y impai men , Quali y o li e, Heal h- ela ed quali y o li e, Pa ien epo ed ou comes,
P e e ence-based ou come measu es, U ili ies, QALYs
In oduc ion
Mobili y impai men and assis i e mobili y echnology
Mobili y impai men is he leading cause o disabili y in
he UK, accoun ing o 52% o epo ed disabili ies [1].
Mobili y impai men s a ise om a as a ay o di e en
disabili ies, condi ions, inju ies and illnesses. Howe e ,
hey can be classi ied b oadly as ei he congeni al (i.e.
om bi h) o acqui ed (i.e. occu ing la e in li e).
Whe he a disabili y is p esen om bi h o acqui ed
la e on in li e signi ican ly in luences indi idual adap a-
ion. Fo ins ance, indi iduals wi h congeni al disabili ies
exhibi highe deg ees o li e sa is ac ion, sel -iden i y
and sel -e icacy ( ela ed o hei disabili y) han indi id-
uals who ha e had o adap o acqui ed disabili y [2].
Adap a ion o disabili y is in luenced by sel -concep and
disabili y iden i y, which in u n a e ela ed o he onse
o disabili y [2].
Common congeni al condi ions which can impac mo-
bili y include ce eb al palsy (CP) and spina bi ida (SB).
CP e e s o a numbe o condi ions caused by damage
o he pa s o he b ain which con ol mo emen , bal-
ance and pos u e, and can be caused ei he by abno mal
b ain de elopmen o auma. CP is symp omized by
a ying deg ees o pe manen mo emen diso de , in-
cluding poo coo dina ion, muscle s i ness/weakness
and in olun a y mo emen s. SB a ec s he de elopmen
o he spine and spinal co d be o e bi h, and can esul
in leg weakness and pa alysis. The e a e h ee ypes o
SB: myelomeningocele, meningocele and SB occul a.
Bo h congeni al and acqui ed mobili y impai men s
may necessi a e he use o assis i e echnology o alle i-
a e impai men s. Assis i e echnology e e s o a wide
a ay o p oduc s and se ices which enhance unc ion-
ing, pa icipa ion and p omo e independence o people
who ha e disabili ies. The Medicines and Heal hca e
P oduc s Regula o y Agency de ines assis i e echnology
as any de ice “in ended o compensa e o o alle ia e an
inju y, handicap o illness o o eplace a physical unc-
ion”[3]. Assis i e echnology, such as wheelchai s, a e
an “essen ial componen o inclusi e sus ainable de el-
opmen ”[4], and can enhance he undamen al eedoms
and equali y o oppo uni y o people wi h mobili y im-
pai men s and o he disabili ies. The Uni ed Na ions
(UN) s a es ha access o app op ia e and a o dable as-
sis i e echnology is a basic human igh [5]; he UN
Con en ion on he Righ s o Pe sons wi h Disabili ies
has been a i ied by 175 Membe S a es, who a e obli-
ga ed o ensu e ha a o dable assis i e echnology is
a ailable o all indi iduals in need. Howe e , The Wo ld
Heal h O ganiza ion (WHO) es ima es ha only 10% o
people who need assis i e echnology ha e access o i
[6], and he e emain pe sis en challenges in he equi -
able p o ision o assis i e echnology, pa icula ly in de-
eloping coun ies. One o he keys issues is in assessing
he cos s and bene i s o di e en assis i e echnologies,
and de eloping e idence-based app oaches o p o ision
which make bes use o limi ed esou ces o maximise
he ou comes o people wi h disabili ies.
The Na ional Heal h Se ice (NHS) in he UK spends
almos £200million pe yea on wheelchai s alone [7],
hus he e is an impe a i e o ensu e ha assis i e mo-
bili y echnologies (AMTs) such as wheelchai s and
o he mobili y-enhancing in e en ions a e p o ided in
an e idence-based manne , u ilising e idence o cos -
e ec i eness o guide se ice commissioning.
Economic e alua ion and quali y-adjus ed li e yea s
Me hods o economic e alua ion a e now ou inely em-
bedded in he e alua ion o heal h echnologies, and
used o es ima e he cos o inc emen al bene i s associ-
a ed wi h new and al e na i e heal h in e en ions. Cos -
u ili y analysis, speci ically es ima ion o cos pe quali y-
adjus ed li e yea s (QALYs), has become he p edomin-
an o m o economic e alua ion o new heal h ech-
nologies in he UK, in pa due o he Na ional Ins i u e
o Heal h and Ca e Excellence’s (NICE) ad ocacy o
his app oach [8]. The QALY amewo k has become in-
c easingly in luen ial in heal h policy as a heo e ically
uni e sal and gene ic app oach o measu ing bene i s ia
a single common ou come.
In o de o calcula e QALYs, heal h s a e u ili y alues
a e needed. These alues a e mos commonly de i ed
om p e e ence-based measu es (PBMs) o heal h-
ela ed quali y o li e (HRQoL). HRQoL is a subjec i e
and mul i-dimensional cons uc de ined as he pe -
cei ed impac o heal h s a us on quali y o li e, includ-
ing physical, psychological and social unc ioning. PBMs
o HRQoL a e used o assess he social desi abili y and
u ili y alues associa ed wi h di e en s a es o heal h.
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 2 o 38
As he desc ip i e sys ems and alue se s o gene ic
PBMs a e usually de i ed om adul samples o he gen-
e al popula ion, a common c i icism is ha hei gene -
ici y limi s ele ance and sensi i i y in ce ain condi ions
[9]. Mo eo e , in heal h s a es whe e quali y o li e akes
p eceden o e quan i y o li e (e.g. ch onic illness, li e-
limi ing condi ions and disabili y), QALYs de i ed om
gene ic PBMs can de alue he e ec i eness o an in e -
en ion [10].
Use o p e e ence-based measu es in he con ex o
mobili y impai men
The accu acy o a QALY es ima e is subjec o he sensi-
i i y and applicabili y o he measu emen ool used o
gene a e he u ili y da a. PBMs ha e been ound o be in-
consis en in bo h congeni al and acqui ed mobili y im-
pai men s [11–13], u he mo e di e en PBMs p oduce
signi ican ly di e en esul s o AMT use s [14,15].
P e ious esea ch shows ha pa ien s wi h congeni al
mobili y impai men s do no necessa ily conside mobil-
i y o ha e a majo impac on hei HRQoL when sui -
able adap a ions (such as AMT) a e a ailable [16,17].
Howe e , gene al popula ion PBM alue se s hea ily im-
pac es ima ion o HRQoL when abili y o walk is a -
ec ed. As an example, using he NICE app o ed UK
alue se o he Eu oQoL i e-dimension ( h ee le el
e sion) (EQ-5D-3 L), he lowes possible mobili y le el
(‘con ined o bed’) has a disu ili y o −0.664, meaning
ha an indi idual who is unable o walk bu is o he wise
mobile using AMT can achie e a maximum u ili y alue
o 0.336 (0 = dea h; 1 = pe ec heal h), e en i hey ha e
no o he HRQoL impac s. This aises he ques ions as o
whe he exis ing PBMs a e a alid sou ce o u ili y alues
in mobili y impai ed popula ions, pa icula ly AMT
use s.
The alidi y o PBMs can be es ed by compa ing e-
sul s ac oss g oups o pa ien s and by compa ing gene ic
PBMs wi h condi ion-speci ic measu es. Fo ins ance,
he EQ-5D-3 L and he Heal h Assessmen Ques ion-
nai e (HAQ; an ou come measu e o heuma oid a h-
i is) bo h measu e heal h s a us in signi ican ly di e en
ways [18], sugges ing ha he EQ-5D-3 L is lacking con-
side a ion o impo an heal h impac s associa ed wi h
heuma oid a h i is. These issues a e pa ly due o he
insensi i i y o he EQ-5D-3 L ‘mobili y’dimension o
accu a ely assess he a ied impac s o heuma oid a h-
i is on mobili y [19]. Simila ly, he limi ed le el choices
on he EQ-5D-3 L ha e been ound o cause some indi-
iduals wi h mobili y impai men s o choose le els
which a e mo e o less se e e han hei ac ual s a e,
such as using ‘I am con ined o bed’ o subs i u e being
con ined ‘ o an elec ic wheelchai ’[20]. The upda ed
i e le el e sion o he EQ-5D (EQ-5D-5 L) is unlikely
o add ess his issue as he i e le el choices s ill ocus
on walking and do no ake accoun o al e na i e
me hods o mobili y.
E en simple gene ic measu es o heal h s a us, such as
he single ques ion sel - epo ed heal h (SRH) scale (i.e.
“in gene al, would you say you heal h is excellen , e y
good, good, ai , o poo ?”), exhibi only limi ed co el-
a ion wi h PBMs such as he Heal h U ili ies Index
(HUI) 3 and Assessmen o Quali y o Li e (AQoL) in
he con ex o SB [21] and CP [22]. Likewise, o indi id-
uals wi h spinal co d inju ies, he wo ding o he 36-
I em Sho Fo m Heal h Su ey (SF-36) ( om which he
Sho -Fo m Six-Dimension (SF-6D) PBM is calcula ed)
mus be modi ied in o de o main ain ele ance [23].
Conside ing he po en ial issues o using gene ic PBMs
in disabili y, and congeni al mobili y impai men speci -
ically, i is appa en ha he e a e a numbe o impo -
an conside a ions when using PBMs o e alua e AMT
in e en ions and o he mobili y-enhancing in e en-
ions o people wi h congeni al mobili y impai men s.
The objec i e o his sys ema ic e iew is he e o e o
examine he measu emen p ope ies o gene ic u ili y-
based PBMs in a ious o ms o congeni al mobili y im-
pai men . All e idence epo ing (o in e ing) he alid-
i y, eliabili y and/o esponsi eness o PBMs in
condi ions associa ed wi h congeni al mobili y impai -
men was colla ed and syn hesised. Compa able
condi ion-speci ic e iews o PBM pe o mance ha e
been conduc ed in mobili y impai men s such as
heuma oid a h i is [24], CP [25] and mul iple scle osis
[26], howe e PBM pe o mance has no been sys ema -
ically summa ised and colla ed ac oss a ange o con-
geni al mobili y impai men s o da e.
Me hods
This sys ema ic e iew ollowed he Uni e si y o Yo k
Cen e o Re iews and Dissemina ion (CRD) p inciples
o conduc ing sea ches and ex ac ing da a [27]. In e -
ne e e ence da abase sea ching was he main s a egy
o ga he ing e idence. Da abases included: Coch ane
Collabo a ion Regis e and Lib a y, Science Di ec ,
CINAHL, ASSIA, PsychINFO, PubMed and Web o Sci-
ence. Sc eening o e e ence lis s, hand-sea ching and
a ge ed sea ching ia he CRD da abase (which co e s
he Da abase o Abs ac s o Re iews o E ec s (DARE),
he NHS Economic E alua ion Da abase (NHS EED) and
he Heal h Technology Associa ion (HTA) da abase)
we e ca ied ou in addi ion o he p ima y da abase
sea ches. Due o limi ed ansla ion esou ces, only s ud-
ies w i en o ansla ed in o English o Welsh we e eli-
gible o inclusion. Sea ch esul s we e managed using
he online bibliog aphic managemen so wa e Re wo ks
( o s o age o i les om he sys ema ic sea ches) and
Mendeley ( o e e encing pu poses). The sys ema ic
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 3 o 38
e iew p o ocol was egis e ed on PROSPERO
(CRD42018088932).
Sea ch e ms
Fo he pu pose o his e iew, mobili y impai men was
de ined as any congeni al (i.e. p esen om o sho ly a e
bi h) condi ion, impai men , disabili y o illness which
causes signi ican es ic ions o mobili y o 12 mon hs o
longe , and which necessi a es he use o AMT, su ge y o
ehabili a ion o main ain, acili a e o subs i u e ambula-
ion, o o educe complica ions ela ed o mobili y im-
pai men . Acqui ed mobili y impai men s (i.e. no p esen
om bi h) and sho - e m inju ies, such as sp ains o
acu e muscula inju ies, we e no included unde his de -
ini ion o mobili y impai men .
Sea ch e ms included a mix u e o MeSH (Medical
Subjec Heading) and non-MeSH wo ds and ph ases, di-
ided in o wo g oups: ‘popula ion’and ‘ou comes’(see
Table 1).
In o de o iden i y s udies e e ing o in e en ions a-
he han pa ien g oups (e.g. s udies examining ‘wheel-
chai use s’mo e gene ally), he ‘popula ion’sea ch e ms
also co e ed ele an AMTs and mobili y-enhancing in-
e en ions. The ‘ou comes’sea ch e ms co e ed ele an
PBM keywo ds, including speci ic ou come measu es
(such as he a ious e sions o he EQ-5D and HUI mea-
su es). An NHS pos u e and mobili y se ice manage was
consul ed o e ine he sea ch e ms. An example o a
sea ch s ing is shown in Table 2.
S udy eligibili y
Any s udy epo ing he pe o mance o PBMs o
HRQoL in pa ien g oups wi h congeni al mobili y im-
pai men s was eligible o inclusion. This included s ud-
ies epo ing p oxy ou comes. The e was no es ic ion
on s udy ype. S udies ocussing solely on non-PBMs o
HRQoL o acqui ed mobili y impai men s we e ex-
cluded. S udies which included pa ien g oups wi h a y-
ing deg ees o disabili y/disease se e i y we e conside ed
o inclusion i he majo i y o pa ien s had a congeni al
mobili y impai men o i he da a o pa ien s wi h con-
geni al mobili y impai men s was epo ed sepa a ely
(i.e. sub-g oup analysis).
Sc eening
Two esea che s unde ook each s age o he sc eening
p ocess. Fo he ini ial sc eening p ocess, all iden i ied
s udies we e assessed o ele ance based on hei i le
and desc ip o e ms, he emaining s udies we e hen
assessed by hei abs ac . All s udies conside ed ele an
a e he ini ial sc eening p ocess we e hen ob ained in
ull. Bo h e iewe s sc eened each s udy independen ly.
A hi d esea che was consul ed when he e was dis-
ag eemen abou he inclusion o a speci ic s udy.
Quali y app aisal
All ele an s udies which me he ini ial inclusion c i-
e ia we e c i ically app aised o me hodological quali y
by wo esea che s. Quali y app aisal me hods we e
adap ed om simila sys ema ic e iews in o he clinical
a eas [28–30]. Quali y app aisal was no used o exclude
s udies, bu o illus a e he o e all quali y o esea ch
conduc ed in his opic a ea. Quali y app aisal ocussed
on six key a eas:
Whe he es s o s a is ical signi icance we e ca ied
ou
Di e ences be ween in e en ions and/o pa ien
g oups (i.e. sub-g oup analysis)
Clinical signi icance and ele ance o esul s
Repo ing o missing da a
Response and comple ion a es
Explici epo ing o inclusion/exclusion c i e ia
Da a ex ac ion
Da a ex ac ion c i e ia included:
S udy cha ac e is ics: s udy ype, coun y, numbe /
composi ion o s udy g oups, missing da a
Demog aphics: numbe o pa icipan s, age, gende ,
ype/se e i y o mobili y impai men
Measu es: Gene ic PBMs used, condi ion-speci ic
measu es used, o he clinically ele an measu es
used
Ou comes: Mean u ili y sco es, mean u ili y sco es
o ele an sub-g oups, s a is ical signi icance
be ween g oups
Pe o mance: Known g oup analyses, con e gen
alidi y (co ela ion be ween ou comes and/o
esponden ypes), esponsi eness, eliabili y,
esponse/comple ion a es
Analysis o he pe o mance o p e e ence-based
measu es
PBM pe o mance indica o s we e adap ed om COS-
MIN measu emen p ope y guidance o heal h- ela ed
pa ien - epo ed ou comes [31].
Assessmen o alidi y
In his con ex alidi y e e s o he ex en o which a
PBM can be conside ed o measu e wha i has been de-
signed o measu e (i.e. HRQoL), and whe he i does so
in a sys ema ic manne . By es ablishing whe he a spe-
ci ic PBM is su icien ly alid in a pa icula pa ien
g oup, g ea e con idence can be placed in he gene a ed
da a. We ocussed p edominan ly on cons uc alidi y
in his e iew.
Cons uc alidi y was assessed in a numbe o ways.
Fi s ly, known-g oup analyses we e used o assess
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 4 o 38

whe he speci ic PBMs we e able o de ec expec ed di -
e ences be ween di e en pa ien g oups (i.e. a iance
due o se e i y o illness). Secondly, con e gen alidi y
was de e mined by examining co ela ion be ween com-
pa able ou comes, o ins ance be ween PBMs and
condi ion-speci ic measu es wi h compa able cons uc s.
Finally, con e gen alidi y was u he examined by
looking a co ela ion be ween esponden s ypes (i.e.
sel - epo ed and p oxy u ili y ou comes). In he in e es
o uni o mi y, he s eng h o co ela ions was de ined as
absen ( < 0.20), weak ( = 0.20 o 0.35), mode a e ( =
0.35 o 0.50) and s ong ( ≥0.50) [32].
Assessmen o eliabili y
Reliabili y e e s o he eplicabili y o esul s. Reliabili y
is commonly assessed by examining es - e es esul s
and in e - a e eliabili y o PBMs in de ined unchanging
pa ien g oups.
Table 1 Sea ch e ms and ph ases
Popula ion Ou comes
Assis ed mobili y Mobili y scoo e 15D
Assis i e mobili y Mobili y echnolog* AQoL
B ain damage* Mo o dis* Assessmen o Quali y o Li e
B ain inju * Mo o ised scoo e Child heal h u ili ies
Buggy Neu odisabili y Child heal h u ili y
Calipe Neu ological dis* CHU9D
Cane Neu omo o dis* CHU-9D
Ce eb al palsy Neu omuscula dis* EQ 5D
Club oo O ho i* EQ-5D
Club oo Os eogenesis impe ec a Eu oQoL
C u ch* Pa aly* Heal h u ili ies
Diplegi* Pa aplegi* Heal h-u ili ies
Dysmelia Physical disab* HUI
Dys oph* Physical impai * HUI2
Elec ic chai Physically disab* HUI3
Elec ic powe ed indoo ou doo chai Physically impai ed P e e ence based
Elec ic powe ed indoo /ou doo chai Powe chai P e e ence-based
Elec ic scoo e Powe ed chai QALY
Elec ically powe ed indoo ou doo chai Pushchai Quali y adjus ed li e yea
Elec ically powe ed indoo /ou doo chai Quad iplegi* Quali y-adjus ed li e yea
Elec onically powe ed indoo ou doo chai Rolla o Quali y o well-being scale
Elec onically powe ed indoo /ou doo chai Scoo e QWB-SA
*encephal* Spina bi ida Sho Fo m Six Dimension
EPIOC Spinal muscula a ophy Sho F om 6 Dimension
Func ional disab* Talipes SF6D
Handicap* Te aplegi* SF-6D
Hemiplegi* Walk aid
Hyd ocephalus Walk-aid
Knee scoo e Walke
Knee walke Walking aid
Mobili y aid Walking ame
Mobili y de ice Walking s ick
Mobili y dis* Walking-aid
Mobili y equipmen Wheelchai
Mobili y impai *
*
Indica es unca ed wo ds/ph ases
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 5 o 38
Assessmen o esponsi eness
Responsi eness e e s o he ex en o which a measu e
can iden i y changes in heal h s a us [28]. A esponsi e
measu e should be able o de ec clinically signi ican
changes in heal h ou comes o e ime [29]. Responsi e-
ness was de e mined by examining he ela ionship be-
ween ou comes de i ed om PBMs and o he ele an
measu es, be o e and a e an in e en ion.
E idence syn hesis
Desc ip i e syn hesis o sea ch esul s was conduc ed
[27] and p esen ed in abula ed o m. Na a i e syn hesis
was unde aken o de elop a s uc u ed na a i e o e-
sul s; ex ac ed esul s we e g ouped by ype o mobili y
impai men and ca ego y o PBM pe o mance.
Resul s
See Fig. 1 o sea ch ou comes and he sc eening p ocess
lowcha . Sea ches we e conduc ed om Ma ch o May
2018. In o al 1489 s udy a icles we e iden i ied: 1332
om he bibliog aphic sea ches and 157 a icles om
o he sou ces (i.e. CRD da abase, sc eening o e e ence
lis s and hand-sea ching), o which 410 duplica es we e
emo ed. A e sc eening o i les and abs ac s, 66
Table 2 Example o keywo d sea ch s ing
(“Assis ed mobili y”O “Assis i e mobili y”O “B ain damage*”O “B ain
inju *”O Buggy O Calipe O Cane O “Ce eb al palsy”O “Club oo ”
O Club oo O C u ch* O Diplegi* O Dysmelia O Dys oph* O
“Elec ic chai ”O “Elec ic powe ed indoo ou doo chai ”O “Elec ic
powe ed indoo /ou doo chai ”O “Elec ic scoo e ”O “Elec ically
powe ed indoo ou doo chai ”O “Elec ically powe ed indoo /ou doo
chai ”O “Elec onically powe ed indoo ou doo chai ”O “Elec onically
powe ed indoo /ou doo chai ”O encephal* O EPIOC O “Func ional
disab*”O Handicap* O Hemiplegi* O Hyd ocephalus O “Knee
scoo e ”O “Knee walke ”O “Mobili y aid”O “Mobili y de ice”O
“Mobili y dis*”O “Mobili y equipmen ”O “Mobili y impai *”O “Mobili y
scoo e ”O “Mobili y echnolog*”O “Mo o dis*”O “Mo o ised scoo e ”
O Neu odisabili y O “Neu ological dis*”O “Neu omo o dis*”O
“Neu omuscula dis*”O O ho i* O “Os eogenesis impe ec ”O Pa aly*
O Pa aplegi* O “Physical disab*”O “Physical impai *”O “Physically
disab*”O “Physically impai ed”O “Powe chai ”O “Powe ed chai ”O
Pushchai O Quad iplegi* O Rolla o O Scoo e O “Spina bi ida”O
“Spinal muscula a ophy”O Talipes O Te aplegi* O “Walk aid”O
“Walk-aid”O Walke O “Walking aid”O “Walking ame”O “Walking
s ick”O “Walking-aid”O Wheelchai ) AND (15D OR AQoL OR
“Assessmen o Quali y o Li e”OR “Child heal h u ili ies”OR “Child
heal h u ili y”OR CHU9D OR “CHU-9D”OR EQ. 5D OR “EQ-5D”OR
Eu oQoL OR “Heal h u ili ies”OR “Heal h-u ili ies”OR HUI OR HUI2 OR
HUI3 OR “P e e ence-based”OR “P e e ence based”OR QALY OR “Quali y
adjus ed li e yea ”OR “Quali y o well-being scale”OR “Quali y-adjus ed
li e yea ”OR “QWB-SA”OR “Sho Fo m Six Dimension”OR “Sho F om 6
Dimension”OR SF6D OR “SF-6D”)
Fig. 1 PRISMA lowcha o sea ch ou comes and sc eening p ocess
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 6 o 38
s udies we e iden i ied as po en ially eligible. Following
ull e iew o ull- ex s, 35 s udies we e excluded o a
a ie y o easons (see Fig. 1). In o al 31 s udies we e
iden i ied as ele an and eligible o inclusion in he sys-
ema ic e iew. Quali y app aisal ou comes a e p esen ed
in Table 3.
S udy and pa ien cha ac e is ics
S udy and pa icipan cha ac e is ics a e p esen ed in
Table 4. Mos o he s udies (22 o 31) we e c oss-
sec ional s udies [15,21,22,34,36–38,40–45,47–52,
54,58,59], six we e p ospec i e coho s udies [33,35,
39,46,53,57], wo we e case-con ol s udies [11,56]
and one was a andomised con olled ial [55]. The se-
lec ed s udies we e conduc ed in a ange o di e en
coun ies; mos we e (n= 15) om Canada [21,22,33,
35,36,38–43,46,49,53,57]. The sample sizes o ele-
an s udy g oups anged om 13 [15] o 770 [44].
Fou een s udies included da a ela ing o CP [22,33,
35,36,39,46,48–51,53,55,57,58], six included da a
ela ing o SB [21,38,51,52,54,56] and i e included
da a ela ing o childhood hyd ocephalus [40–43,45].
Da a o a ange o o he ele an condi ions we e also
ound in ei he single s udies o om ex ac able sub-
g oups, hese included muscula dys ophy [34,44],
spinal muscula a ophy [47], Mo quio A synd ome [37],
congeni al club oo [59] and mic ocephaly [51]. Th ee
s udies ocused on mul iple condi ions whe e sub-g oup
da a could no be examined sepa a ely [11,15,51].
The as majo i y o s udies (23 o 31) included only
child/adolescen pa icipan s [11,15,33,35,36,38–44,
46–54,56,58]. O he emaining s udies, six included
child en/adolescen s and adul s [21,22,34,37,55,57]
and wo included only adul s [45,59]. PBM epo ing
was p edominan ly om p oxies (13 o 23 s udies);
ele en s udies included bo h sel - epo ed and p oxy
da a [15,21,22,36,43,48–50,53,54,57] and se en
s udies included only sel - epo ed PBM da a [11,34,37,
45,55,58,59].
In e ms o use o PBMs, 22 o he s udies used a e -
sion o he HUI [15,21,22,33,35,36,38–44,46,48,49,
51–54,56,57], eigh used a e sion o he EQ-5D [11,
15,34,37,47,50,58,59], h ee used a e sion o AQoL
ins umen [21,22,57], one used he 15D [45] and one
used he SF-6D [55].
Na a i e syn hesis
U ili y ou comes a e p esen ed in Table 5and PBM pe -
o mance ou comes a e p esen ed in Table 6. The na a-
i e syn hesis is ca ego ised by ype o mobili y
impai men and PBM pe o mance indica o . No s udies
epo ed PBM eliabili y ou comes, he e o e eliabili y
esul s ha e no been p esen ed.
Ce eb al palsy
Known-g oup analyses
Fi e s udies epo ed known-g oup analyses in CP [22,
33,51,53,58]. Pe ou and Kupek [51] es ima ed ha he
adjus ed HUI3 disu ili y o childhood CP om pe ec
heal h was −0.72 (95% con idence in e al [CI] -0.61 o
−0.85), and −0.65 (95% CI −0.54 o −0.78) om child-
hood no ms. Two s udies ound ha as CP se e i y (i.e.
g oss mo o unc ion) inc eased, a e age u ili y sco es
(measu ed using HUI3 o AQoL) dec eased in adoles-
cen s and young adul s wi h CP [22,53]; Rosenbaum
e al. [53] ound s a is ically signi ican di e ences in
mean u ili y sco es be ween mos G oss Mo o Func ion
Classi ica ion Sys em (GMFCS) le els (p < 0.01). One
s udy demons a ed ha he ision, pain and cogni ion
dimensions o he HUI3 s eadily declined as GMFCS
le el inc eased, howe e s a is ical signi icance was no
epo ed [33]. Vi ale e al. [58] ound ha adolescen s
wi h CP had signi ican ly highe a e age EQ-5D u ili y
sco es (0.92) compa ed o adolescen s wi h scoliosis
(wi h como bidi ies) (0.73; p> 0.05), al hough selec ion
o hese pa ien sub-g oups was no explici ly jus i ied
and he e sion o he EQ-5D was no epo ed.
Con e gen alidi y: compa ing measu es
Two s udies epo ed ha GMFCS le el was co ela ed
wi h wo sening u ili y sco es [22,53]. Young e al. [22]
ound ha GMFCS le el in childhood was esponsible
o be ween 45% (AQoL: β=−0.148; p< 0.001) and 53%
(HUI3: β=−0.205; p< 0.001) o a iance in u ili y
sco es. Rosenbaum e al. [53] ound a s ong nega i e
co ela ion be ween he HUI3 u ili y sco es o adoles-
cen s wi h CP and hei GMFCS le el ( =−0.81). In
e ms o indi idual PBM dimensions, esul s om ou
s udies we e a ied [33,35,39,49]; Kennes e al. [39]
ound ha he dimension mos associa ed wi h GMFCS
le el in child en was ambula ion ( au-b = 0.82; p< 0.01).
Ba le e al. [33] ound ha he HUI3 ision, pain and
cogni ion dimensions gene ally wo sened as GMFCS
le el inc eased in adolescen s; howe e , he e was no in-
dica ion ha hese dimensions we e de e minan s o
mo o capaci y decline. Two s udies [35,49] epo ed a
signi ican nega i e associa ion be ween he HUI3 pain
dimension and GMFCS le el in child en wi h CP, how-
e e bo h o hese s udies only examined he HUI3 pain
dimension and no he ull HUI3 sys em.
Th ee s udies epo ed a ious le els o co ela ion be-
ween PBMs and o he ou come measu es in CP [22,46,
53]. Young e al. [22] ound mode a e co ela ion be-
ween u ili y sco e and SRH ( = 0.41; p< 0.001 o bo h
he HUI3 and AQoL). Two s udies compa ed he Qual-
i y o Li e Ins umen o People wi h De elopmen al
Disabili ies (QOL Ins umen ) and he HUI3 [46,53];
Rosenbaum e al. [53] epo ed ha adolescen s’HUI3
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 7 o 38
u ili y sco es explained be ween 3% (belonging) and 14%
(being) o a iance in QOL Ins umen dimension
sco es, while Li ings on and Rosenbaum [46] epo ed
ha he HUI3 and QOL Ins umen sha ed up o 23%
a iance, hus he ela ionship be ween he measu es
was conside ed o be mode a e a bes .
Two s udies epo ed on he ela ionship be ween
di e en PBMs in CP [22,57]. Al hough u ili y sco es
de i ed om he HUI3 and AQoL we e s ongly co -
ela ed ( = 0.87; p< 0.001) [22], HUI3 de i ed u ili y
sco es ended o be lowe han AQoL de i ed u ili y
sco es [22,57].
Con e gen alidi y: compa ing esponden s
Fou s udies examined co ela ion be ween esponden s
in CP [22,48–50]. Mo ow e al. [48] epo ed mode a e
ag eemen be ween pa en s and doc o s o child en wi h
CP o he HUI2/3 dimensions o sensa ion (63.6%
ag eemen ; Kappa 0.41), cogni ion (70% ag eemen ;
Kappa 0.56), sel -ca e (100% ag eemen ; Kappa 1.00) and
ambula ion (63.6% ag eemen ; Kappa 0.46). Good co el-
a ion was also ound be ween child sel - epo ed HUI3
pain sco es and equi alen pa en p oxy sco es (Good-
man and K uskal’s y s a is ic = 0.57; p< 0.001) [49].
Pe ez Sousa e al. [50] epo ed a high le el o dis-
ag eemen be ween pa en s and child en on he EQ-5D
Table 3 Quali y app aisal ou comes
S udy e e ence (au ho , yea ) Tes s o s a is ical
signi icance
conduc ed
Sub-g oup
analyses
conduc ed
Clinical
implica ions
discussed
P opo ions o
missing/inco ec
da a epo ed
Response and/o
comple ion a es
epo ed
Inclusion and/o
exclusion c i e ia
explici ly s a ed
Ba le e al. (2010) [33]YYYYXY
B ay e al. (2017) [15]YYYYYY
Bu s om e al. (2014) [11]YYXYYX
Ca azza e al. 2016 [34]XXXXXX
Ch is ensen e al. (2016) [35]YYYXYX
Findlay e al. (2015) [36]YYYYYX
Hend iksz e l al (2014) [37]. YYYYXY
Ka mu and Kulka ni (2018) [38]YXYYXX
Kennes e al. (2002) [39]YYYYXY
Kulka ni e al. (2004) [40]YXYXYY
Kulka ni (2006) [41]YYXYXY
Kulka ni e al. (2008) [42]YXYYYX
Kulka ni e al. (2008) [43]YYYXYY
Land eld e al. (2016) [44]YXYXYX
Lindquis e al. (2014) [45]YYYXXY
Li ings on and Rosenbaum (2008) [46]YXYYXX
Lopez-Bas ida e al. (2017) [47]XXYYXX
Mo ow e al. (2011) [48]. YYYXYY
Penne e al. (2013) [49]YYYXYX
Pe ez Sousa e al. (2017) [50]YYYYYY
Pe ou and Kupec (2009) [51]YYYYXY
Rocque e al. (2015) [52]YYYXXY
Rosenbaum e al. (2007) [53]YYYYXY
Sims-Williams e al. (2016) [54]YYYYXY
Slaman e al. (2015) [55]YXYXXY
Til o d e al. (2005) [56]YXYXYX
Usuba e al. (2014) [57]YYYYYY
Vi ale e al. (2001) [58]YYYXXY
Wallande e al. (2009) [59]. YXYXYY
Young e al. (2010) [22]. YYYYYY
Young e al. (2013) [21]YYYXYY
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 8 o 38
Table 5 Summa y o u ili y ou comes o included s udies
S udy e e ence Condi ion(s) o in e es Responden ype PBM (s) O he ele an ou come
measu es
O e all u ili y sco es (mean ± SD) Sub-g oup u ili y sco es
(mean ± SD)
Ba le e al.
(2010) [33]
Adolescen s wi h ce eb al
palsy
P oxy (pa en ) HUI3 G oss Mo o Func ion
Measu e 66 I ems (GMFM-66)
Spinal Alignmen and Range
o Mo ion Measu e
(SAROMM)
NA
O e all u ili y sco es no epo ed,
only ision, cogni ion and pain
dimensions used - ambula ion,
hea ing, speech, dex e i y and
emo ion dimensions all excluded
due o concep ual o e lap wi h
o he measu es
NA
B ay e al. (2017) [15] Child en and adolescen s
wi h impai ed mobili y
( ele an condi ions: ce eb al
palsy, hemiplegia, muscula
dys ophy)
Sel - epo ed and
p oxy (pa en );
ma ched-pai s
HUI2, HUI3 and
EQ-5D-Y
Visual analogue scale (VAS) Child sel - epo ed
HUI2: 0.53 (±0.07)
HUI3: 0.22 (±0.09)
EQ-5D-Y: 0.24 (±0.30)
Pa en p oxy
HUI2: 0.49 (±0.09)
HUI3: 0.16 (±0.10)
EQ-5D-Y: 0.01 (±0.14)
NA
Bu s om e al.
(2014) [11]
Child en and adolescen s
wi h unc ional mo o ,
o hopaedic and medical
disabili ies ( ele an
condi ions: a og yposis
mul iple congeni al,
myelomeningocele, ce eb al
palsy, o hopaedic lowe limb
de o mi ies, achond oplasia)
Sel - epo ed EQ-5D-Y VAS
KIDSCREEN-27
KIDSCREEN-10
Sel - a ed heal h (SRH)
Li e sa is ac ion ladde (LSL)
NA
O e all u ili y sco es no epo ed,
only dimension sco es epo ed
NA
Ca azza e al.
(2016) [34]
Adolescen s and adul s wi h
Duchenne muscula
dys ophy
Sel - epo ed EQ-5D-Y VAS
Ba hel Index
Mean 0.24
Va ied by coun y, anging om
−0.71 (±0.41) in Sweden o 0.66
(±0.08) in Bulga ia
NA
Ch is ensen e al.
(2016) [35]
Child en and adolescen s
wi h ce eb al palsy
P oxy (ca egi e ) HUI3 Wong-Bake FACES Pain
Ra ing Scale
NA
O e all u ili y sco es no epo ed,
only HUI3 pain dimension
measu ed
NA
Findlay e al.
(2015) [36]
Child en wi h ce eb al palsy Sel - epo ed and
p oxy (ca egi e );
p opo ion o p oxy
da a no epo ed
HUI3 DISABKIDS Ch onic Gene ic
module (DCGM-37)
DISABKIDS Smiley measu e
(DSM)
Wong-Bake FACES Pain
Ra ing Scale
NA
O e all u ili y sco es no epo ed,
only HUI3 pain dimension
measu ed
NA
Hend iksz e al.
(2014) [37]
Child en and adul s wi h
Mo quio A synd ome
Sel - epo ed EQ-5D-5 L B ie Pain In en o y Sho
Fo m (BPI-SF)
Adolescen Pedia ic Pain
Tool (APPT)
NA
O e all u ili y sco es no epo ed
Child g oup: u ili y sco e by
wheelchai use equency
No use: 0.534
Occasional use: 0.664
Full- ime use: −0.180
Adul g oup: u ili y sco e by
wheelchai use equency
No use: 0.846
Occasional use: 0.582
Full- ime use: 0.057
Ka mu and Kulka ni
(2018) [38]
Child en and adolescen s
wi h spina bi ida
(myelomeningocele) and
P oxy (ca egi e ) HUI2 and HUI3
( e sion used o
calcula e u ili y
Hyd ocephalus Ou come
Ques ionnai e (HOQ)
0.51 (±0.28) NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 15 o 38

Table 5 Summa y o u ili y ou comes o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Responden ype PBM (s) O he ele an ou come
measu es
O e all u ili y sco es (mean ± SD) Sub-g oup u ili y sco es
(mean ± SD)
shun ed hyd ocephalus sco es no s a ed)
Kennes e al.
(2002) [39]
Child en wi h ce eb al palsy P oxy (ca egi e ) HUI3 Func ional Independence
Measu e o Child en
(WeeFIM)
S eng h and Di icul ies
Ques ionnai e (SDQ)
Wide Range Achie emen
Tes (WRAT)
NA
O e all u ili y sco es no epo ed,
only dimension sco es epo ed
NA
Kulka ni e al.
(2004) [40]
Child en wi h hyd ocephalus P oxy (pa en ) HUI2 HOQ NA
O e all u ili y sco es no epo ed
NA
Kulka ni e al.
(2006) [41]
Child en wi h hyd ocephalus P oxy (pa en ) HUI2 HOQ NA
O e all u ili y sco es no epo ed
NA
Kulka ni e al.
(2008) [42]
Child en wi h hyd ocephalus P oxy (ca egi e ) HUI3 HOQ 0.58 (±0.32) NA
Kulka ni e al.
(2008) [43]
Child en wi h hyd ocephalus Sel - epo ed and
p oxy (ca egi e );
p oxy- epo ed HUI3
and HOQ, sel -
epo ed cHOQ
HUI3 Child-comple ed e sion o
he HOQ (cHOQ)
0.71 (±0.27) NA
Land eld e al.
(2016) [44]
Child en and adolescen s
wi h Duchenne muscula
dys ophy
P oxy (ca egi e );
al hough pa ien s
included in da a
collec ion, only
p oxy u ili y da a
epo ed
HUI ( e sion no
s a ed)
Pedia ic Quali y o Li e
In en o y (PedsQL)
neu omuscula module 3.0
NA
O e all u ili y sco es no epo ed
U ili y sco e by ambula o y s a us
Ea ly ambula o y: 0.75
La e non-ambula o y: 0.15
Lindquis e al.
(2014) [45]
Adul s who expe ienced
hyd ocephalus in in ancy
Sel - epo ed 15D NA S udy g oup: 0.92 Con ol g oup: 0.95
Li ings on and
Rosenbaum
(2008) [46]
Adolescen s wi h ce eb al
palsy
P oxy (pa en ) HUI3 Quali y o Li e Ins umen o
People wi h De elopmen al
Disabili ies (QOL Ins umen )
NA
O e all u ili y sco es no epo ed
NA
Lopez-Bas ida e al.
(2017) [47]
Child en wi h spinal muscula
a ophy
P oxy (ca egi e ) EQ-5D-3 L VAS
Ba hel Index
0.16 (±0.44) U ili y sco e by spinal muscula
a ophy ype
Type II spinal muscula a ophy
sub-g oup: −0.01 (±0.35)
Sco es o Type I and III no
epo ed
Mo ow e al.
(2011) [48]
Child en wi h ch onic
condi ions ( ele an
condi ion: ce eb al palsy)
Sel - epo ed and
p oxy (pa en and
doc o ); ma ched
g oups
HUI2 and HUI3 NA NA
O e all u ili y sco es no epo ed
o ele an sub-g oup
NA
Penne e al.
(2013) [49]
Child en and adolescen s
wi h ce eb al palsy
Sel - epo ed and
p oxy (pa en and
physician); p oxy-
epo ed HUI3 pain
sco e, sel - epo ed
Wong-Bake FACES
Pain Scale
HUI3 Wong-Bake FACES Pain
Ra ing Scale
NA
O e all u ili y sco es no epo ed,
only HUI3 pain dimension
measu ed
NA
Pe ez Sousa e al. Child en and adolescen s Sel - epo ed and EQ-5D-Y VAS NA NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 16 o 38
Table 5 Summa y o u ili y ou comes o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Responden ype PBM (s) O he ele an ou come
measu es
O e all u ili y sco es (mean ± SD) Sub-g oup u ili y sco es
(mean ± SD)
(2017) [50] wi h ce eb al palsy p oxy (mo he and
a he ); ma ched
g oups
O e all u ili y sco es no epo ed,
only dimension sco es epo ed
Pe ou and Kupek
(2009) [51]
Child en wi h childhood
condi ions ( ele an
condi ions: mic ocephaly,
ce eb al palsy, spinal
muscula a ophy, muscula
dys ophy, spina bi ida)
P oxy (ca egi e ) HUI3 NA Mean u ili y by condi ion
Mic ocephaly: 0.141
Ce eb al palsy: 0.276
Muscula dys ophy o spinal
muscula a ophy: 0.386
Spina bi ida: 0.452
NA
Rocque e al.
(2015) [52]
Child en and adolescen s
wi h spina bi ida
Sel - epo ed and
p oxy (ca egi e );
p edominan ly p oxy
(11% sel - epo ed)
HUI3 NA NA
O e all u ili y sco es no epo ed
U ili y sco e by ype o spina bi ida
Myelomeningocele: 0.51
Closed spinal dys aphism: 0.77
U ili y sco e by ea men his o y
No his o y o shun o CM-II
decomp ession: 0.74
Shun bu no CM-II
decomp ession: 0.49
Shun and CM-II
decomp ession: 0.29
Rosenbaum e al.
(2007) [53]
Adolescen s wi h ce eb al
palsy
Sel - epo ed and
p oxy (pa en );
p oxy- epo ed HUI3,
34% sel - epo ed
QOL Ins umen
HUI3 QOL Ins umen 0.42 (±0.41) U ili y sco e by GMFCS le el
GMFCS I: 0.84 (±0.20)
GMFCS II: 0.50 (±0.31)
GMFCS III: 0.39 (±0.31)
GMFCS IV: 0.16 (±0.26)
GMFCS V: −0.08 (±0.23)
Sims-Williams e al.
(2017) [54]
Child en wi h spina bi ida Sel - epo ed and
p oxy (ca egi e );
ma ched pai s
HUI3 VAS Child sel - epo ed
0.58 (95% CI 0.49–0.66)
Ca egi e p oxy
0.55 (95% CI 0.47–0.63)
NA
Slaman e al.
(2015) [55]
Adolescen s and young
adul s wi h ce eb al palsy
Sel - epo ed SF-6D 36-I em Sho Fo m Su ey
(SF-36) - SF-6D u ili y ou
comes de i ed om SF-36
Baseline
Con ol: 0.74 (±0.12)
In e en ion: 0.75 (±0.10)
6 mon hs pos in e en ion
Con ol: 0.77 (±0.12)
In e en ion: 0.80 (±0.03)
NA
Til o d e al.
(2005) [56]
Child en wi h spina bi ida P oxy (ca egi e ) HUI2 Quali y o Well-Being scale
(QWB)
Case g oup: 0.55 (±0.24) U ili y by case g oup lesion le el
Sac al lesion: 0.61 (±0.26)
Lowe lumba lesion: 0.54 (±0.19)
Tho acic lesion: 0.45 (±0.25)
Gene al popula ion con ol
g oup: 0.93 (±0.11)
Usuba e al.
(2014) [57]
Adolescen s and adul s wi h
ce eb al palsy
Sel - epo ed and
p oxy (unde ined);
p edominan ly p oxy
(40% sel - epo ed)
HUI3 and AQoL SRH Baseline (bo h g oups)
HUI3: 0.29 (±0.39)
AQoL: 0.35 (±0.33)
8-yea ollow-up (bo h g oups)
HUI3: 0.29 (±0.38)
AQoL: 0.35 (±0.32)
NA
Vi ale e al.
(2001) [58]
Adolescen s wi h
o hopaedic p oblems
( ele an condi ion: ce eb al
palsy)
Sel - epo ed EQ-5D ( e sion no
s a ed)
SF-36 Ce eb al palsy sub-g oup: 0.922 NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 17 o 38
Table 5 Summa y o u ili y ou comes o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Responden ype PBM (s) O he ele an ou come
measu es
O e all u ili y sco es (mean ± SD) Sub-g oup u ili y sco es
(mean ± SD)
Wallande e al.
(2009) [59]
Adul s ea ed o CTEV in
in ancy
Sel - epo ed EQ-5D ( e sion no
s a ed)
SF-36
VAS
Ame ican Academy o
O hopaedic Su geons Foo
and Ankle Ques ionnai e
NA
O e all u ili y sco es no epo ed
NA
Young e al.
(2010) [22]
Adolescen s and young
adul s wi h ce eb al palsy
Sel - epo ed and
p oxy (ca egi e );
p edominan ly sel -
epo ed (45% p oxy)
HUI3 and AQoL SRH
Heal h Assessmen
Ques ionnai e (HAQ)
Combined age g oups
HUI3: 0.30 (±0.42)
AQoL: 0.28 (±0.33)
U ili y sco e by age g oup
(HUI3 / AQoL)
‘You h’g oup: 0.30 (±0.43) /
0.28 (±0.34)
‘Adul ’g oup: 0.31 (±0.40) /
0.28 (±0.314)
You h g oup: u ili y sco e by
GMFCS le el (HUI3 / AQoL)
GMFCS I: 0.67 (±0.32) / 0.58
(±0.31)
GMFCS II: 0.59 (±0.35) / 0.53
(±0.34)
GMFCS III: 0.43 (±0.39) / 0.31
(±0.32)
GMFCS IV: 0.08 (±0.25) / 0.06
(±0.12)
GMFCS V: −0.13 (±0.19) / 0.01
(±0.07)
Adul g oup: u ili y sco e by GMFCS
le el (HUI3 / AQoL)GMFCS I: 0.64
(±0.30) / 0.52 (±0.32)
GMFCS II: 0.50 (±0.39) / 0.33
(±0.24)
GMFCS III: 0.53 (±0.27) / 0.39
(±0.27)
GMFCS IV: 0.06 (±0.21) / 0.10
(±0.20)
GMFCS V: −0.14 (±0.20) / 0.02
(±0.06)
Young e al.
(2013) [21]
Adolescen s and young
adul s wi h spina bi ida
Sel - epo ed and
p oxy (ca egi e );
p edominan ly sel -
epo ed (15% p oxy)
HUI3 and AQoL SRHHAQ Combined age g oups
HUI3: 0.52 (±0.28)
AQoL: 0.34 (±0.24)
U ili y sco e by age g oup (HUI3 /
AQoL)
‘You h’g oup: 0.58 (±0.27) / 0.37
(±0.26)
‘Adul ’g oup: 0.36 (±0.27) / 0.25
(±0.17)
U ili y sco e by lesion le el (HUI3 /
AQoL)
Tho acic: 0.29 (±0.14) / 0.22 (±
0.14)
High-lumba : 0.44 (±0.31) / 0.28
(±0.23)
Low-lumba : 0.63 (±0.23) / 0.39 (±
0.21)
Sac al: 0.76 (±0.20) / 0.51 (±0.33)
Unknown: 0.22 (±0.02) / 0.09
(±0.04)
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 18 o 38
Table 6 Summa y o PBM pe o mance o included s udies
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
Ba le e al.
(2010) [33]
Adolescen s wi h ce eb al
palsy
HUI3 ision, cogni ion
and pain dimensions
s eadily declined as
GMFCS le el
inc eased, s a is ical
signi icance no
epo ed.
Examining co ela ion
coe icien s, he e was no
indica ion ha he HUI3
ision ( =0.01; p= 0.92),
cogni ion ( =−0.05; p=
0.59) o pain dimensions
( =0.16; p= 0.07) we e
de e minan s o mo o
capaci y, as measu ed
using he GMFM-66.
NA NA Indi iduals wi h a GMFCS
le el o V exhibi ed he
la ges mean dec eases in
HUI3 dimension le els
o e ime, anging om
−0.2 (±1.2) o he HUI3
ision dimension o −0.3
(±1.3) o he HUI3
cogni ion and pain
dimensions.
B ay e al.
(2017) [15]
Child en and adolescen s
wi h impai ed mobili y
( ele an condi ions:
ce eb al palsy,
hemiplegia, muscula
dys ophy)
NA NA La ge a iance be ween
mean u ili y sco es
de i ed om di e en
PBMs, anging om 0.24
(EQ-5D-Y) o 0.53 (HUI2)
o child sel - epo ed
u ili y, and om 0.01 (EQ-
5D-Y) o 0.49 (HUI2) o
pa en - epo ed p oxy
u ili y.
A signi ican esponden
ype e ec was ound,
wi h mean child sel -
epo ed u ili y sco es
signi ican ly (p≤0.021)
highe han equi alen
p oxies on all PBMs.
Signi ican s ong
co ela ions we e ound
be ween u ili y sco es o
child en/pa en p oxies
using all measu es: EQ-
5D-Y ( =0.67; p= 0.026),
HUI2 ( =0.73; p= 0.005)
and HUI3 ( =0.84; p<
0.001).
Using Bland-Al man plo s,
su icien ag eemen
be ween u ili y sco es o
child en/pa en p oxies
was ound o he HUI2
(CL = 0.22) and HUI3
(CL = 0.22). EQ-5D-Y
exhibi ed clinically
impo an disc epancies
be ween child and pa en
p oxy esponses
(CL = 1.04).
NA
Bu s om e al.
(2014) [11]
Child en and adolescen s
wi h unc ional mo o ,
o hopaedic and medical
disabili ies ( ele an
condi ions: a og yposis
mul iple congeni al,
myelomeningocele,
ce eb al palsy,
o hopaedic lowe limb
de o mi ies,
S a is ically signi ican
(p≤0.001) di e ences
be ween case and
con ol g oups on all
dimensions. Mean
dimension sco es no
epo ed, p opo ions
o pa ien s choosing
each dimension le el
indica e case g oup
S ong signi ican
co ela ion was ound
be ween he EQ-5D-Y
anxie y/dep ession di
mension and he
KIDSCREEN-27 psycho
logical well-being dimen
sion ( =−0.51; p= 0.001);
KIDSCREEN-27 physical
well-being dimension
NA NA NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 19 o 38
Table 6 Summa y o PBM pe o mance o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
achond oplasia) epo ed mo e
p oblems on all
dimensions han he
con ol g oup: 83% o
case g oup epo ed
some/a lo o
p oblems on any
dimension, compa ed
o 37% o con ol
g oup; likewise 21%
o case g oup
epo ed ex eme
p oblems on any
dimension, compa ed
o 2% o con ol
g oup.
( =−0.53; p= 0.001); and
li e sa is ac ion ladde
(LSL) ( =−0.54; p<
0.001). Mode a e co el
a ion also ound be ween
his dimension and he
KIDSCREEN-10 HRQoL
index sco e ( =−0.50;
p= 0.001) and sel -
epo ed heal h (SRH)
(0.42; p= 0.007).
The sel -ca e EQ-5D-Y di
mension exhibi ed mod
e a e co ela ion wi h he
KIDSCREEN-27 physical
wellbeing dimension ( =
−0.37; p= 0.028) and he
usual ac i i ies EQ-5D-Y
dimension was
mode a ely co ela ed
wi h he KIDSCREEN-27
psychological wellbeing
dimension ( =−0.35; p=
0.027). All o he
co ela ions we e
weak o absen .
Ca azza e al.
(2016) [34]
Adolescen s and adul s
wi h Duchenne muscula
dys ophy
NA NA NA NA NA
Ch is ensen e al.
(2016) [35]
Child en and adolescen s
wi h ce eb al palsy
NA Using mul i a ia e linea
eg ession, a signi ican
associa ion was ound
be ween he HUI3 pain
dimension sco e a
baseline and GMFCS le el
(b = −0.11, β=−0.15;
p< 0.036; 95% CI −0.21
o −0.01): highe pain
sco e a baseline was
associa ed wi h g ea e
imp o emen in pain
s a us in GMFCS le el I
compa ed o le el V.
NA NA Using one-way ANOVA
analysis, a signi ican as
socia ion was ound
be ween physician
p ima y pain ae iology
and change in HUI3 pain
s a us (p= 0.001): child en
wi h musculoskele al pain
a baseline showed sig
ni ican imp o emen s
(mean change 0.55; 95%
CI 0.053 o 1.05) com
pa ed o child en wi hou
pain a baseline (mean
change −0.39; 95% CI −
0.62 o −0.15) (p = 0.006).
No o he associa ions
we e epo ed. HUI3 pain
dimension sco es did no
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 20 o 38

Table 6 Summa y o PBM pe o mance o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
change signi ican ly o e
ime: median sco e o 2
(ou o 5) a bo h isi s.
Howe e , 55% o child en
changed pain s a us o e
ime: 34% wo sening,
21% imp o ing.
Findlay e al.
(2015) [36]
Child en wi h ce eb al
palsy
NA NA NA NA NA
Hend iksz e al.
(2014) [37]
Child en and adul s wi h
Mo quio A synd ome
A signi ican e ec o
wheelchai use on
u ili y ou comes was
epo ed; signi ican
di e ences epo ed
be ween: adul non-
wheelchai use s and
occasional wheelchai
use s (p= 0.0115);
adul occasional
wheelchai use s and
ull- ime wheelchai
use s (p= 0.0007);
child occasional
wheelchai use s and
ull- ime wheelchai
use s (p = 0.0018); and
child non-wheelchai
use s and ull- ime
wheelchai use s (p=
0.0018). Child en who
occasionally used a
wheelchai had a
highe mean u ili y
sco e on a e age han
non-wheelchai use s,
al hough bo h g oups
had highe a e age
u ili y sco es han ull-
ime wheelchai use s.
NA NA NA NA
Ka mu and
Kulka ni
(2018) [38]
Child en and adolescen s
wi h spina bi ida
(myelomeningocele) and
shun ed hyd ocephalus
Using mul i a ia e
eg ession analysis,
ana omical le el o
myelomeningocele
had a signi ican
e ec on u ili y sco e
(p= 0.01): lowe
ana omical le el o
myelomeningocele
NA NA NA NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 21 o 38
Table 6 Summa y o PBM pe o mance o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
was associa ed wi h a
highe u ili y sco e.
Kennes e al.
(2002) [39]
Child en wi h ce eb al
palsy
NA Using Kendall’s au-b es
o associa ion, he HUI3
dimension mos associ
a ed wi h GMFCS le el
was ambula ion ( au-b =
0.82; p< 0.01): highe
GMFCS le el was
associa ed wi h inc eased
mobili y impai men .
Mode a e co ela ions
( anging om au-b =
0.36 o 0.58; p< 0.01)
we e ound be ween
GMFCS le el and he
ision, speech and
dex e i y dimensions.
O e all pa e ns we e
simila o he ambula ion
dimension, bu
co ela ions we e weake .
Hea ing ( au-b = 0.16; p=
0.04) and cogni ion ( au-
b = 0.27; p< 0.01)
dimensions had
s a is ically signi ican bu
low associa ion wi h
GMFCS le el. The
emo ion ( au-b = 0.03;
p= 0.24) and pain
( au-b = 0.07; p= 0.37)
dimensions we e no
signi ican ly associa ed
wi h GMFCS le el.
NA NA NA
Kulka ni e al.
(2004) [40]
Child en wi h
hyd ocephalus
NA S ong co ela ion was
ound be ween u ili y
sco e and he HOQ
o e all heal h ( =0.81),
physical heal h ( =0.88),
social-emo ional ( =0.56)
and cogni i e ( =0.57)
sco es.
NA NA NA
Kulka ni e al.
(2006) [41]
Child en wi h
hyd ocephalus
NA Co ela ion be ween
u ili y sco e and HOQ
o e all heal h sco e was
high ( =0.81), a
sca e plo demons a ed
NA NA NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 22 o 38
Table 6 Summa y o PBM pe o mance o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
a s ong linea
ela ionship. Simple and
complex linea eg ession
models bo h accoun ed
o a la ge p opo ion o
HUI2 a iabili y (adjus ed
R
2
= 0.66 and 0.80
espec i ely).
Kulka ni e al.
(2008) [42]
Child en wi h
hyd ocephalus
NA Mean u ili y sco e (0.58 ±
0.63) close o cHOQ
mean sco es o o e all
heal h (0.65 ± 0.20),
Physical heal h (0.66 ±
0.25), Cogni i e heal h
(0.55 ± 0.28) and Social-
emo ional heal h (0.71 ±
0.19). S a is ical
signi icance no epo ed.
NA NA NA
Kulka ni e al.
(2008) [43]
Child en wi h
hyd ocephalus
NA Signi ican co ela ion was
ound be ween he cHOQ
o e all heal h sco e (sel -
epo ed) and u ili y sco e
(p oxy- epo ed) ( =0.60;
p< 0.001).
NA NA NA
Land eld e al.
(2016) [44]
Child en and adolescen s
wi h Duchenne muscula
dys ophy
Ambula o y class was
signi ican ly
associa ed wi h p oxy-
epo ed u ili y sco es
(p< 0.001), dec easing
om ea ly ambula o y
class (mean 0.75) o
la e non-ambula o y
class (mean 0.15).
Ca egi e assessmen s
o heal h and men al
s a us we e signi i
can ly associa ed wi h
u ili y sco e
(p< 0.001).
NA NA NA NA
Lindquis e al.
(2014) [45]
Adul s who expe ienced
hyd ocephalus in in ancy
The s udy g oup had
signi ican ly (p≤
0.004) lowe
dimension sco es
compa ed o he
con ol g oup in
ision, ea ing, usual
ac i i ies, men al
NA NA NA NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 23 o 38
Table 6 Summa y o PBM pe o mance o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
unc ion dimensions.
The mos epo ed
p oblems we e
associa ed wi h he
neu oimpai men
subg oup (mean
u ili y sco e 0.87
compa ed o 0.94 in
he no
neu oimpai men
subg oup). The
subg oup wi hou
neu oimpai men
we e no signi ican ly
di e en o he
con ol in e ms o
mean u ili y sco e
(0.94 and 0.95
espec i ely, p alue
no epo ed).
Li ings on and
Rosenbaum
(2008) [46]
Adolescen s wi h
ce eb al palsy
NA Disa enua ed co ela ion
coe icien s be ween
u ili y sco es and QOL
Ins umen sco es
demons a ed weak o
mode a e co ela ion o
he being ( =0.48);
belonging ( =0.35);
becoming ( =0.29) and
o e all quali y o li e
( =0.35) scales. The wo
measu e sha ed up o
23% a iance.
NA NA Gene alizabili y
coe icien s we e
calcula ed o assess
a iabili y o sco es o e
ime. Dimensions wi h
g ea e s abili y (i.e. la ge
G sco es) had less
a iabili y be ween
indi iduals o e ime. The
ambula ion dimension
(G = 0.94) and o e all
u ili y (G = 0.91) we e
ound o be highly s able;
while he speech (G =
0.87), ision (G = 0.87);
dex e i y (G = 0.82),
cogni ion (G = 0.81), and
hea ing (G = 0.72)
dimensions we e
mode a ely s able. The
pain (G = 0.48) and
emo ion (G = 0.24)
dimensions we e ound
o ha e low s abili y.
Lopez-Bas ida
e al. (2017) [47]
Child en wi h spinal
muscula a ophy
Child en wi h Type II
spinal muscula
a ophy we e ound
o ha e a lowe
NA NA NA NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 24 o 38
Table 6 Summa y o PBM pe o mance o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
child en wi h ce eb al
palsy (mean 0.92) and
child en wi h scoliosis
wi h como bidi ies
(mean 0.725), bu no
child en wi h
idiopa hic scoliosis
(mean 0.889).
Jus i ica ion o hese
g oupings was based
on sample size, hus
he e is no explici
explana ion as o why
u ili y di e ences
be ween hese
g oups would be
expec ed.
Wallande e al.
(2009) [59]
Adul s ea ed o CTEV in
in ancy
Male pa icipan s had
signi ican ly highe
a e age u ili y sco e
han he compa able
no m g oup (p=
0.027). Male
pa icipan s epo ed
signi ican ly less
mode a e/ex eme
p oblems wi h
anxie y/dep ession
han he compa able
no m g oup (6.3% s
21.2%; p= 0.007).
Female pa icipan s
had wo se u ili y on
a e age han a
compa able no m
g oup, bu no
signi ican ly. Female
pa icipan s epo ed
signi ican ly mo e
mode a e/ex eme
p oblems wi h
mobili y (45% s
12.2%; p< 0.001) and
usual ac i i ies (35%
s 12.5%; p= 0.006)
han a compa able
no m g oup.
NA NA NA NA
Young e al.
(2010) [22]
Adolescen s and young
adul s wi h ce eb al palsy
In bo h he ‘you h’
and ‘adul ’g oups,
Linea eg ession analysis
e ealed ha GMFCS
U ili y sco es de i ed
om HUI3 we e on
P oxy u ili y sco es we e
gene ally lowe (by 0.16)
NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 31 o 38

Table 6 Summa y o PBM pe o mance o included s udies (Con inued)
S udy e e ence Condi ion(s) o in e es Known-g oup
analyses
Cons uc alidi y:
compa ing ou comes
Cons uc alidi y:
compa ing PBMs
Cons uc alidi y:
compa ing esponden s
Responsi eness
u ili y sco es
de e io a ed s eadily
as GMFCS le el
inc eased. S a is ical
signi icance no
epo ed
le el in childhood was
he mos impo an
in luence on u ili y sco es;
esponsible o 53.2% o
a iance in HUI3 u ili y
ou comes (β=−0.205;
p< 0.001) and 45.2% o
a iance in AQoL u ili y
ou comes (β=−0.148;
p< 0.001).
The SRH was ound o be
mode a ely co ela ed
wi h u ili y sco es de i ed
om he HUI3 ( =0.41;
p< 0.001) and AQoL
( =0.41; p< 0.001).
a e age highe han
hose de i ed om AQoL
ac oss all GMFCS le els;
howe e s ong
co ela ion was ound
be ween he HUI3 and
AQoL ( =0.87; p< 0.001).
when adjus ed o
cogni ion, gene al heal h
and CP se e i y. S a is ical
signi icance no epo ed.
Young e al.
(2013) [21]
Adolescen s and young
adul s wi h spina bi ida
U ili y sco es de i ed
om bo h HUI3 and
AQoL a ied in he
same way acco ding
o lesion le el, wi h
ho acic lesions
associa ed wi h
lowes u ili y. Linea
eg ession analysis
e ealed ha he
mos impo an single
ac o con ibu ing o
u ili y ou comes was
su gical lesion le el;
esponsible o 40% o
a iance in HUI3
u ili y sco es and 18%
in AQoL u ili y sco es.
Bo h lesion le el and
age we e impo an
when combined,
accoun ing o 48%
a iance in HUI3 u ili y
sco es and 22%
a iance in AQoL
u ili y sco es.
The SRH was ound o be
mode a ely/s ongly
co ela ed wi h u ili y
sco es de i ed om he
HUI3 ( =−0.45; p<
0.001) and AQoL ( =−
0.58; p < 0.001). The HAQ
was also s ongly
co ela ed wi h u ili y
sco es de i ed om he
HUI3 ( =0.79; p< 0.001)
and AQoL ( =0.70; p<
0.001).
U ili y sco es de i ed
om he AQoL we e
highe han om he
HUI3 ac oss all lesion
le el sub-g oups; how
e e he AQoL and HUI3
exhibi ed s ong co el
a ion ( =0.73; p< 0.001).
Mean u ili y sco es we e
sligh ly highe in he sel -
epo ed g oup (HUI3
mean + 0.04; AQoL
mean + 0.03) howe e
his was based on he
compa ison o you h and
adul da a and he eg es
sion models emained
unchanged.
NA
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 32 o 38
you h e sion (EQ-5D-Y); pa en s epo ed a lowe e-
quency o p oblems on all EQ-5D-Y p oxy dimensions,
pa icula ly a he s.
Responsi eness
Fi e s udies allowed analysis o PBM esponsi eness in
CP [33,35,46,55,57]. Adolescen s wi h a GMFCS le el
o V exhibi ed he la ges dec eases in HUI3 dimension
le els o e ime, compa ed o adolescen s wi h GMFCS
le els o III and IV [33]. Ch is ensen e al. [35] epo ed
a signi ican associa ion be ween physicians’p ima y
pain ae iology and change in HUI3 pain s a us (p=
0.001). Con e sely, in wo s udies u ili y ou comes did
no change signi ican ly o e ime; HUI3 u ili y ou -
comes we e ound o be s able o e a 1 yea pe iod o
adolescen s wi h CP (G = 0.91) [46], likewise u ili y ou -
comes (de i ed om HUI3 and AQoL) did no signi i-
can ly change o e an 8 yea ollow-up pe iod [57].
Slaman e al. [55] u ilised he SF-6D in a andomised
con olled ial, bu did no ind a signi ican di e ence
be ween he con ol and in e en ion g oups a he end
o he ial (p= 0.42).
Spina bi ida
Known-g oup analyses
Th ee s udies epo ed known-g oup analyses in SB [51,
52,56], wo o which ound ha clinical ac o s had a
signi ican impac on u ili y sco es. Pe ou and Kupek
[51] es ima ed ha he adjus ed HUI3 disu ili y o child-
hood SB om pe ec heal h was −0.55 (95% CI −0.40
o −0.70), and −0.48 (95% CI −0.33 o −0.63) om
childhood no ms. A s a is ically signi ican e ec o SB
diagnosis on HUI3 u ili y sco e (p< 0.001) was epo ed
[52]; child en diagnosed wi h myelomeningocele (mean
u ili y sco e = 0.51) ended o ha e lowe u ili y sco es
compa ed o child en wi h closed dys aphism (mean
u ili y sco e = 0.77). Til o d e al. [56] epo ed ha le-
sion loca ion in childhood SB had a signi ican impac
on o e all u ili y (p< 0.01); indi iduals wi h sac al le-
sions had he highes o e all mean u ili y (0.61; ±0.26).
Two s udies epo ed co ela ion be ween clinical ac-
o s and u ili y sco es in SB [21,38]. Ana omical myelo-
meningocele le el was ound o ha e a signi ican e ec
on he HUI u ili y sco es o child en wi h myelomenin-
gocele and shun ed hyd ocephalus (mean HUI sco e =
0.03; 95% CI 0.01 o 0.05; p= 0.01) [38], wi h lowe mye-
lomeningocele le el showing associa ion wi h highe
u ili y sco es. A simila end was epo ed by Young
e al. [21], who ound ha he mos impo an single ac-
o con ibu ing o u ili y ou comes in SB was su gical
lesion le el, which was esponsible o be ween 18%
(AQoL) and 40% (HUI3) o a iance in u ili y sco es.
Con e gen alidi y: compa ing measu es
Two s udies epo ed a ious le els o co ela ion be ween
PBMs and o he ou come measu es in SB [21,54]. Child
sel - epo ed HUI3 u ili y sco es we e no ound o be
highly co ela ed wi h VAS sco es ( =0.488) [54]. Like-
wise, he SRH was only mode a ely co ela ed wi h u ili y
sco es in SB (HUI3: =−0.45; p<0.001 / AQoL: =−
0.58; p<0.001) [21]. Only he HAQ was ound o be
s ongly co ela ed wi h u ili y sco e (HUI3: =0.79; p<
0.001 / AQoL: =0.70;p<0.001)[21].
Young e al. [21] compa ed esul s om di e en
PBMs in SB, and ound ha mean u ili y sco es on he
AQoL we e lowe han mean u ili y sco es on he HUI3
o all sub-g oups, despi e s ong co ela ion be ween
hese measu es ( = 0.73; p< 0.001).
Con e gen alidi y: compa ing esponden s
Sims-Williams e al. [54] epo ed ha p oxy and sel -
epo ed HUI3 u ili y sco es we e highly co ela ed o
child en wi h SB ( = 0.85; signi icance no epo ed).
Young e al. [21] ound ha mean sel - epo ed u ili y
sco es we e sligh ly highe han equi alen p oxy sco es
(HUI3 mean + 0.04; AQoL mean + 0.03) howe e e-
sponden ype was no in luen ial in hei eg ession
analysis.
PBM esponsi eness ou comes in SB we e no ound
in he li e a u e.
Mixed pa ien g oups and mobili y impai men s
This sec ion includes esul s om s udies which did no
ocus on speci ic condi ions, and whe e sub-g oup da a
could no be examined sepa a ely. Rele an condi ions/
mobili y impai men s included muscula dys ophy,
spinal muscula a ophy, CP, SB, o hopaedic lowe limb
de o mi ies, a og yposis mul iple congeni al, achond o-
plasia and hemiplegia.
Known-g oup analyses
Two s udies epo ed known-g oup analyses in s udies o
mixed pa ien g oups [11,51]. Pe ou and Kupek [51]
ound ha child en wi h muscula dys ophy o spinal
muscula a ophy had a mean u ili y sco e o 0.39, equa -
ing o an adjus ed disu ili y om pe ec heal h o −0.62
(95% CI −0.471 o −0.761), and an adjus ed disu ili y
om childhood no ms o −0.54 (95% CI −0.400 o −
0.690). Bu s om e al. [11] epo ed ha child en wi h a
unc ional disabili y (64% congeni al; see Table 4 o pa-
ien cha ac e is ics) had signi ican ly lowe EQ-5D-Y di-
mension sco es han he gene al popula ion (p<0.001).
Con e gen alidi y: compa ing ou comes
Mode a e co ela ion was ound be ween he indi idual
dimensions o he EQ-5D-Y and he dimensions o o he
measu es (KIDSCREEN-27 and KIDSCREEN-10) and
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 33 o 38
he li e sa is ac ion ladde (LSL), when comple ed by
child en wi h unc ional disabili ies [11] . The EQ-5D-Y
anxie y/dep ession dimension exhibi ed signi ican co -
ela ion wi h he KIDSCREEN-27 psychological well-
being dimension ( =−0.51; p = 0.001), he
KIDSCREEN-27 physical well-being dimension ( =−
0.53; p= 0.001), and he LSL ( =−0.54; p< 0.001) [11].
La ge a iance was obse ed be ween u ili y sco es de-
i ed om di e en PBMs o young wheelchai use s;
mean u ili y sco es anged om 0.24 (EQ-5D-Y) o 0.53
(HUI2) o sel - epo ing child en, and om 0.01 (EQ-
5D-Y) o 0.49 (HUI2) o pa en p oxies [15].
Con e gen alidi y: compa ing esponden s
A signi ican s ong co ela ion was obse ed be ween
dyads o young wheelchai use s (84.6% child en wi h
CP, see Table 4 o pa ien cha ac e is ics) and pa en
p oxies o a numbe o u ili y measu es: EQ-5D-Y ( =
0.67; p= 0.026), HUI2 ( = 0.73; p= 0.005) and HUI3
( = 0.84; p< 0.001) [15]. Using Bland-Al man plo s [60],
su icien ag eemen was obse ed be ween dyads o he
HUI2 (CL = 0.22) and HUI3 (CL = 0.22), bu no he EQ-
5D-Y (CL = 1.04). A signi ican esponden ype e ec
was ound o all measu es, wi h child sel - epo ed u il-
i y sco es signi ican ly highe o he EQ-5D-Y (p=
0.012), HUI2 (p= 0.021) and HUI3 (p= 0.009) han
equi alen p oxy measu es [15].
PBM esponsi eness ou comes we e no ound in he
li e a u e o his pa ien g oup.
Childhood hyd ocephalus (mixed ae iology)
Known-g oup analyses
Lindquis e al. [45] ound ha adul s wi h a his o y o
hyd ocephalus (wi h o wi hou neu o-impai men ) had
signi ican ly lowe 15D dimension sco es, compa ed o a
con ol g oup, in he dimensions o ision (p= 0.001),
ea ing (p= 0.000), usual ac i i ies (p= 0.004) and men al
unc ion (p= 0.000).
Con e gen alidi y: compa ing measu es
Fou s udies examined he ela ionship be ween he
Hyd ocephalus Ou come Ques ionnai e (HOQ) and
PBMs [40–43], howe e only h ee o hese s udies pe -
o med ele an s a is ical analyses [40,41,43]. Kulka ni
[41] epo ed s ong co ela ion (0.81) and a s ong lin-
ea ela ionship be ween HUI2 u ili y sco e and HOQ
ou comes o child en wi h hyd ocephalus. Simple and
complex linea eg ession models bo h accoun ed o a
la ge p opo ion o HUI2 a iabili y (adjus ed R
2
= 0.66
and 0.80 espec i ely). Simila ly, a s ong co ela ion was
ound be ween HUI2 u ili y sco e and he HOQ sco es
o o e all heal h ( = 0.81), physical heal h ( = 0.88),
social-emo ional ( = 0.56) and cogni i e ( = 0.57) [40].
Fu he mo e, a signi ican posi i e co ela ion was
exhibi ed be ween sel - epo ed sco es on he child-
comple ed e sion o he HOQ (cHOQ) and p oxy-
epo ed u ili y sco es on he HUI3 ( = 0.60; p< 0.001)
[43].
PBM esponsi eness ou comes in childhood hyd o-
cephalus we e no ound in he li e a u e.
O he condi ions and mobili y impai men s
Only known-g oup analyses we e epo ed o he ol-
lowing condi ions associa ed wi h mobili y impai men :
Muscula dys ophy
Land eld e al. [44] epo ed ha ambula o y s a us and
age we e signi ican ly associa ed wi h HUI u ili y sco es
in muscula dys ophy (HUI e sion no s a ed; p <
0.001); young ambula o s (5–7 yea s old) had he highes
u ili y sco es on a e age (0.75), whils olde non-
ambula o s (≥16 yea s old) had he lowes u ili y sco es
on a e age (0.15).
Spinal muscula a ophy
Lopez-Bas ida e al. [47] epo ed ha child en wi h Type
II spinal muscula a ophy ended o ha e lowe mean
p oxy u ili y sco es (−0.01; ±0.35) han he combined
a e age o all o ms o spinal muscula a ophy (0.16; ±
0.44), howe e s a is ical analysis was no unde aken.
Mo quio A synd ome
One s udy ound ha o bo h adul s and child en wi h
Mo quio A synd ome, wheelchai use was signi ican ly
associa ed wi h lowe u ili y sco es [37]. Signi ican di -
e ences we e epo ed in he adul g oup be ween non-
wheelchai use s and occasional wheelchai use s (p=
0.0115) and be ween occasional wheelchai use s and
ull- ime wheelchai use s (p= 0.0007). In he child
g oup signi ican di e ences we e epo ed be ween
non-wheelchai use s and ull- ime wheelchai use s
(p= 0.0018) and occasional wheelchai use s and ull-
ime wheelchai use s (p= 0.0007).
Congeni al club oo
Wallande e al. [59] ound ha male adul pa ien s wi h
congeni al club oo (CCF) had signi ican ly be e o e all
u ili y sco es han he male no m g oup (p = 0.027). The
emale CCF g oup had a lowe a e age u ili y sco e han
he no m g oup, bu no signi ican ly (signi icance le el
no epo ed).
Mic ocephaly
Pe ou and Kupek [51] ound ha child en wi h mic o-
cephaly had a mean u ili y sco e o 0.14, equa ing o an
adjus ed disu ili y om pe ec heal h o −0.82 (95% CI
−0.67 o −0.97), and an adjus ed disu ili y om child-
hood no ms o −0.75 (95% CI −0.60 o −0.90).
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 34 o 38
Discussion
The esul s om his sys ema ic e iew demons a e ha
PBMs ha e been used in a ela i ely small numbe o
s udies ela ing o congeni al mobili y impai men s. In
condi ions such as CP and SB, inc eased clinical se e i y
appea s o be associa ed wi h dec eased u ili y. This is
pa icula ly e iden using he HUI3, which was also he
mos commonly used PBM ound in his e iew. In pa -
icula , he e appea s o be a ela ionship be ween u ili y
ou comes and GMFCS le el in CP, and clinical ac o s
such as lesion le el in SB. In case-con ol s udies, u ili y
ou comes ended o be signi ican ly lowe in he case
g oups, al hough i is wo h no ing ha in one s udy he
male case-g oup had signi ican ly highe u ili y ou -
comes compa ed o he con ol [59].
In o de o demons a e su icien applicabili y and
sensi i i y in a speci ic disease o disabili y, associa ion
be ween PBMs and alida ed clinical/condi ion-speci ic
ou comes is o key impo ance. In his espec exis ing
PBMs show weakness in a ious condi ions associa ed
wi h congeni al mobili y impai men s; exhibi ing gene -
ally limi ed co ela ion wi h measu es such as he QOL
Ins umen , VAS, SRH, KIDSCREEN-27, KIDSCREEN-
10 and LSL. Only he GMFCS and HOQ/cHOQ ap-
pea ed o be well co ela ed wi h PBMs ac oss a numbe
o s udies, al hough i is impo an o no e ha GMFCS
is a classi ica ion sys em o g oss mo o unc ion and
no an ou come measu e.
The esul s om his sys ema ic e iew highligh im-
po an conside a ions o he use o PBMs in heal h
s a es associa ed wi h congeni al mobili y impai men . I
is i s o no e ha he use o PBMs has been domina ed
by s udies in CP, ollowed by SB. This is somewha un-
su p ising gi en ha hese a e wo o he mos p e alen
congeni al disabili ies which a ec mobili y. Secondly, i
is impo an o acknowledge ha only wo s udies o-
cussed on adul s alone, and ha bo h o hese s udies
we e measu ing u ili y ou comes in adul s ollowing con-
di ions expe ienced in childhood [45,59]. This ocus on
child en and adolescen s is again unsu p ising, as many
congeni al condi ions can be li e-limi ing, hus examin-
ing heal h ou comes a a young age becomes pa icula ly
impo an . Howe e , his also shows a lack o ocus on
he heal h ou comes o adul s who ha e li e-long expe i-
ence o mobili y impai men , and he po en ial ways in
which hei heal h ou comes could change o e ime o
be imp o ed.
The e is some e idence o demons a e ha di e en
PBMs a y in hei es ima ion o u ili y ou comes in
s a es o impai ed mobili y. Fo ins ance, B ay e al. [15]
ound la ge a ia ion be ween he EQ-5D-Y and HUI2/3
u ili y sco es o wheelchai use s. Likewise, Usuba e al.
[57] and Young e al. [21,22] ound ha u ili y ou -
comes we e gene ally highe when de i ed om he
AQoL han he HUI3 o indi iduals wi h CP, bu ice
e sa o indi iduals wi h SB. This is despi e he s ong
co ela ion be ween he AQoL and HUI3 in hese popu-
la ions [21,22]. Un o una ely, s a is ical di e ences be-
ween hese measu es we e no epo ed, bu i is s ill
impo an o conside he implica ions ha hese di e -
ences could ha e on subsequen QALY ou comes. Fo
ins ance, i one PBM we e o p oduce signi ican ly
highe u ili y sco es han ano he , his would mean sig-
ni ican ly di e en es ima es o cos pe QALYs and hus
es ima es o cos -e ec i eness. A p esen he e is lim-
i ed e idence o enable heal h economis s and e-
sea che s o choose be ween hese di e en PBMs o
use in congeni al mobili y impai men s.
Despi e documen ed limi a ions [61], QALYs ha e be-
come inc easingly in luen ial in heal h policy as a means
o de e mine he cos -e ec i eness o new ea men s and
se ices, and he e o e guide heal hca e unding and p i-
o i isa ion decisions. I is he e o e impe a i e ha he
PBMs used o de elop QALYs a e accu a e. O he wise,
he cos -e ec i eness o ce ain in e en ions in ce ain
pa ien g oups could be unde es ima ed. This in u n
could impac unding and p io i isa ion decisions. In he
con ex o congeni al mobili y impai men , his could im-
pac he p o ision o AMT and o he mobili y-enhancing
in e en ions, and subsequen ly impac pa ien ou comes.
This is pa icula ly impo an conside ing he ongoing is-
sues o unme need in assis i e echnology p o ision. The
WHO P io i y Assis i e P oduc s Lis (APL) was launched
in 2016 o help ackle unme need [62]. The APL con ains
50 p io i y assis i e echnology p oduc s, and was p o-
duced h ough consul a ion wi h use s, expe s and o he
key s akeholde s. App op ia e PBM da a, and subsequen
es ima es o cos -e ec i eness, could help o in o m he
APL and ensu e ha he mos e ec i e and cos -e ec i e
AMTs a e p io i ised.
Conside ing ha he majo i y o e idence ound in his
e iew ela ed o child en, he ela ionship be ween sel -
epo ed and p oxy u ili y ou comes is pa icula ly sig-
ni ican . The esul s om a ious s udies in his e iew
demons a e ha p oxy- epo ing o u ili y is consis -
en ly di e en o ha o sel - epo e s, including signi i-
can esponden - ype e ec s and limi ed ag eemen
be ween esponden s. In e es ingly, p oxy esponden s
we e ound o bo h unde es ima e and o e es ima e u il-
i y ou comes compa ed o sel - epo e s. I is he e o e
impo an o p io i ise ou come measu emen om he
pa ien , al hough his can be challenging in popula ions
who may lack capaci y, such as young child en and indi-
iduals wi h cogni i e impai men s.
Me hodological implica ions
Due o he unde lying ade-o be ween quan i y and
quali y o li e in he calcula ion o u ili y alues and
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 35 o 38
subsequen QALYs, he e is a endency o lowe alue
o be placed on ex ending he leng h o li e o people
wi h long- e m disabili ies [10,63], as hei quali y o li e
is ou inely conside ed o be wo se han ha o an able-
bodied pe son. Thus, when using he QALY amewo k
o assess he ou comes o indi iduals wi h disabili ies, i
is di icul o achie e subs an ially highe quali y o li e
when compa ed o indi iduals wi hou disabili ies, ais-
ing conce ns abou bias [10]. To some ex en hese is-
sues could be a esul o using gene ic PBMs o alue
disabled heal h s a es.
One o he unde lying issues o using PBMs in disabil-
i y is ha he de ini ion o HRQoL di e s p o oundly be-
ween people wi h disabili ies and he gene al public
[64]. When asked o de ine HRQoL, young wheelchai
use s ocus on a numbe o concep s no explici ly mea-
su ed using gene ic PBMs, such as abili y o adap ,
achie emen and independence [17]. The expe ience o
disabili y also a ec s HRQoL pe cep ions. Mechanisms
o adap a ion, coping and adjus men can help indi id-
uals wi h disabili ies o expe ience diminishing e ec s o
hei HRQoL o e ime. These p ocesses a e also in lu-
enced by he onse o disabili y, as indi iduals wi h con-
geni al disabili ies demons a e be e adap a ion han
indi iduals wi h acqui ed disabili ies [2]. The e alua ion
o s a es o disabili y by non-disabled indi iduals may
he e o e cause such s a es o ha e an exagge a ed pe -
cei ed impac on HRQoL and heal h s a us [65], pa icu-
la ly wi h ega ds o congeni al disabili y.
When assessing he desi abili y o hypo he ical heal h
s a es, indi iduals ocus on he ansi ion om hei own
heal h s a e o he hypo he ical heal h s a e, hus gene al
public belie s abou he impac o disabili y do no al-
ways e lec he li ed expe ience [66,67]. Focus on pe -
sonal ansi ion means ha p ocesses such as adap a ion
a e no accoun ed o , causing a disc epancy in how
s a es o disabili y impac HRQoL [9].
An al e na i e app oach is o use mo e sensi i e
condi ion-speci ic measu es o model u ili y alues on
gene ic PBMs. Al hough his app oach is ad oca ed by
NICE [68] he e a e se ious conce ns abou he alidi y
o modelled u ili y alues [69], as i canno be assumed
ha modelled u ili y alues a e ep esen a i e o di ec ly
measu ed u ili y alues [70]. Using modelled u ili y da a
o guide unding and esou ce alloca ion decisions is
he e o e con o e sial. Fo ins ance, Sido a e al. [71]
mapped he 12-I em Mul iple Scle osis Walking Scale
(MSWS-12) on o he EQ-5D-3 L. While p edic ion es i-
ma es we e ela i ely p ecise o pa ien s wi h mode -
a ely impai ed mobili y, hey we e signi ican ly less
accu a e o indi iduals wi h se e e impai men s.
Neilson e al. [72] sugges supplemen ing PBMs wi h
addi ional ques ions ela ing o unc ional ac i i ies
which ha e a la ge impac on o e all quali y o li e, such
as ‘si ing’ o AMT use s. Fo ins ance, Pe sson e al.
[73] added complimen a y mobili y and social ela ion-
ship i ems o he EQ-5D-3 L o inc ease sensi i i y in
disabled popula ions. Howe e , his app oach assumes
ha supplemen al ques ions can be mapped on o he
heal h s a e p e e ence alues o exis ing measu es wi h-
ou impac ing accu acy.
Limi a ions and challenges
De ining he e m congeni al mobili y impai men was
one o he key challenges o designing his sys ema ic e-
iew. An NHS pos u e and mobili y se ice manage was
consul ed o help cons uc he sea ch e ms, and a
numbe o p elimina y sea ches we e ca ied ou o es
sea ch e ms o bo h sensi i i y and scope. A mul i ude
o condi ions and disabili ies can a ec mobili y om
bi h o ea ly in ancy, hus we a emp ed o co e a wide
a ie y o hese in ou sea ch e ms, bu accep ha
he e a e likely o be condi ions and disabili ies which
we e missed. Gi en he sea ch esul s, we a e con iden
ha we ha e cap u ed he as majo i y o ele an s ud-
ies ela ing o a leas he mos common o ms o con-
geni al mobili y impai men . One key issue was
conside ing whe he o include s udies ela ing o hyd o-
cephalus in his e iew. Al hough hyd ocephalus is no
always associa ed wi h mobili y impai men , i can cause
mo emen issues and is commonly ela ed o ele an
condi ions such as CP and SB. We he e o e chose o in-
clude s udies ela ed o childhood hyd ocephalus.
We chose speci ically o ocus on congeni al mobili y
impai men due o he signi ican di e ences in li e sa -
is ac ion, sel -iden i y and sel -e icacy expe ienced by
people wi h congeni al disabili ies compa ed o people
wi h acqui ed disabili ies [2]. Fu he esea ch could
compa e PBM pe o mance in congeni al and acqui ed
mobili y impai men s. P e ious e iews ha e epo ed
mixed esul s ega ding he alidi y and esponsi eness
o exis ing PBMs in he assessmen o heal h s a es asso-
cia ed wi h acqui ed mobili y impai men s, such as
heuma oid a h i is [24] and mul iple scle osis [26].
Conclusion
To ou knowledge, his is he i s sys ema ic e iew o
he use o PBMs in congeni al mobili y impai men . E i-
dence sugges s ha exis ing gene ic PBMs exhibi im-
po an issues ela ing o alidi y and esponsi eness,
and hus ca e mus be aken when selec ing a PBM as
an ou come measu e in his con ex . Condi ion o dis-
abili y speci ic app oaches o u ili y measu emen , such
as he mobili y and quali y o li e (MobQoL) ou come
measu e [74], could imp o e he sensi i i y and applic-
abili y o u ili y measu emen in his con ex .
B ay e al. Heal h Economics Re iew (2020) 10:9 Page 36 o 38

Abb e ia ions
AMT: Assis i e mobili y echnologies; APL: WHO P io i y Assis i e P oduc s
Lis ; APPT: Adolescen Pedia ic Pain Tool; AQoL: Assessmen o Quali y o
Li e; BPI-SF: B ie Pain In en o y Sho Fo m; CCF: Congeni al club oo ;
cHOQ: Child-comple ed e sion o he Hyd ocephalus Ou come
Ques ionnai e; CI: Con idence in e al; CP: Ce eb al palsy; CRD: Cen e o
Re iews and Dissemina ion; CTEV: Congeni al alipes equinus a us;
DARE: Da abase o Abs ac s o Re iews o E ec s; DCGM-37: DISABKIDS
Ch onic Gene ic Module; DSM: DISABKIDS Smiley Measu e; EQ-5D: Eu oQoL
Fi e-Dimension; EQ-5D-Y: Eu oQoL Fi e-Dimension - You h e sion; EQ-5D-3
L: Eu oQoL Fi e-Dimension - Th ee Le el e sion; EQ-5D-5 L: Eu oQoL Fi e-
Dimension - Fi e Le el e sion; GMFCS: G oss Mo o Func ion Classi ica ion
Sys em; GMFM-66: G oss Mo o Func ion Measu e 66 I ems; HAQ: Heal h
Assessmen Ques ionnai e; HOQ: Hyd ocephalus Ou come Ques ionnai e;
HUI: Heal h U ili ies Index; HRQoL: Heal h- ela ed quali y o li e; HTA: Heal h
Technology Assessmen ; LSL: Li e Sa is ac ion Ladde ; MeSH: Medical Subjec
Headings; MSWS-12: 12-I em Mul iple Scle osis Walking Scale; NHS: Na ional
Heal h Se ice; NHS EED: NHS Economic E alua ion Da abase; NICE: Na ional
Ins i u e o Heal h and Ca e Excellence; PBM: P e e ence-based measu e;
PedsQL: Pedia ic Quali y o Li e In en o y; QALY: Quali y-adjus ed li e yea ;
QOL Ins umen : Quali y o Li e Ins umen o People wi h De elopmen al
Disabili ies; QWB: Quali y o Well-Being scale; RR: Rela i e isk;
SAROMM: Spinal Alignmen and Range o Mo ion Measu e; SB: Spina bi ida;
SD: S anda d de ia ion; SDQ: S eng h and Di icul ies Ques ionnai e; SF-
36: 36-I em Sho Fo m Heal h Su ey; SF-6D: Sho -Fo m Six-Dimension;
SRH: Sel - epo ed heal h; UN: Uni ed Na ions; VAS: Visual analogue scale;
WeeFIM: Func ional Independence Measu e o Child en; WHO: Wo ld Heal h
O ganiza ion; WRAT: Wide Range Achie emen Tes
Acknowledgemen s
The au ho ’s would like o acknowledge he suppo o D . Ha e h Al-Janabi,
P o esso Joanna Coas , P o esso Debo ah Fi szimmons, Ms. K ys Ja is and
P o esso Ka he ine Payne o ac ing as ad iso s o he p ojec .
Au ho s’con ibu ions
All au ho s we e esponsible o designing he sys ema ic e iew, de eloping
he o iginal e iew p o ocol and edi ing he manusc ip . NB de ised he
sys ema ic e iew, p epa ed he manusc ip and syn hesised ex ac ed da a.
LHS conduc ed he e iew sea ches and led he s udy app aisal p ocess. NB
and LHS sc eened pape s o eligibili y and quali y, and ex ac ed e idence
wi h suppo om RTE. All au ho (s) ead and app o ed he inal manusc ip .
Funding
This sys ema ic e iew was unded by he Welsh Go e nmen h ough
Heal h and Ca e Resea ch Wales. The unding body had no ole in he
design, conduc o epo ing o his sys ema ic e iew.
A ailabili y o da a and ma e ials
As his is a sys ema ic e iew, he e was no p ima y da a gene a ed as pa
o his esea ch. All o he da a u ilised in his e iew is a ailable om he
ci ed li e a u e, and has been summa ised in Tables 4,5and 6.
E hics app o al and consen o pa icipa e
No applicable, his is a sys ema ic e iew.
Consen o publica ion
No applicable, his is a sys ema ic e iew.
Compe ing in e es s
The au ho s decla e ha hey ha e no compe ing in e es s.
Recei ed: 4 Decembe 2019 Accep ed: 8 Ap il 2020
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