P1252428428Tcaen

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IMPAIRED FUNCTIONAL STATUS IN CHILDREN WITH
CARDIOMYOPATHY A REPORT FROM THE PEDIATRIC
CARDIOMYOPATHY REGISTRY Sleeper LA, Towbin JA,
Colan SD, Hsu DT, Lemler M, Messere J, Clunie S,
Cuniberti L, Servedio D, Rodgers-Augustyniak L,
Lowe AM, Iadarola S, Orav EJ, Lipshultz
SL Supported by a grant from the National Heart,
Lung, and Blood Institute R01 HL53392

From New England Research Institutes Texas
Childrens Hospital, Houston TX Childrens
Hospital Boston Childrens Hospital of NY UT
Southwestern Medical Center, Dallas TX Harvard
Medical School Univ of Miami School of Medicine
ABSTRACT
RESULTS
Health status and functioning of children with
heart disease has not been studied in a large
cohort with standardized instruments. The
NHLBI-sponsored Pediatric Cardiomyopathy Registry
(PCMR) conducted a study of functional status in
children diagnosed with cardiomyopathy (CM) at
birth through age 18 years. CM was defined using
strict echocardiographic criteria or biopsy.
METHODS The PCMR has over 2700 CM cases with
demographic, echocardiographic, and clinical
findings compiled by annual chart review. In
this substudy, health status was assessed at 5
cardiac medical centers by the parents of 189
children using the Child Health Questionnaire
(CHQ) for children age ? 5 yrs and the Functional
Status II Revised (FSIIR) instrument for all
ages. Both English and Spanish instruments were
available. This report summarizes CHQ findings
and examines the association of health status and
CM severity, defined by fractional shortening
(FS) Z-score for BSA. RESULTS The CHQ sample
(? 5 yrs old) was 11.7?4.4 yr at assessment
(2.9?2.2 yr since CM diagnosis) and was 57 male.
Pure dilated and hypertrophic CM comprised 45
and 42 of cases, and 72 were idiopathic. The
echo closest to CHQ administration (n81 FSz
0.45?5.13 FSzlt -2 in 25) was between 2 and 14
months for most subjects. The CHQ Physical
Summary Score was 43.1?13.4, one standard
deviation below the U.S. norm for healthy
children (53.0?8.8 plt .001) 25 were gt2SD below
the norm. The CHQ Psychosocial Summary Score was
48.2?10.4, also lower than the U.S. norm of
51.2?9.1 (plt .001). CHQ domain scores indicated
that greatest impairment was specifically in
lower physical functioning and limitations in
everyday physical activities, child self-esteem,
impact on parental emotions, and general health
perceptions. Although FS Z-score (lt -2 vs. ? -2)
was not associated with CHQ Physical Summary
Score (43?13 vs. 46?12), there was a significant
correlation with Impact on Parental Time domain
(R .30, p .007), with lower CHQ score in
children with FSZ lt -2 (67?35 vs. 88?20, p
.010). CONCLUSIONS One-quarter of children
with CM have severely impaired physical
functioning gt 2SD below the norm, and
psychosocial functioning is also affected.
NOTE Updated analyses of Fractional Shortening
are presented in this poster which supercede this
abstract.
Fractional shortening Z-score (adjusted for BSA)
was used as an indicator of CM severity. 25 of
subjects had a Z-score lt -3 (dilated CM) and 25
had Zgt3 (hypertrophic CM). Figure 2 Table 2
demonstrate that in children with CM, fractional
shortening is significantly associated with child
mental health, self-esteem and parent burden, as
well as physical functioning.
As of 4/30/04, 189 subjects were enrolled and
were representative of the overall Registry. The
189 subjects were 8.1 5.7 years at the time of
questionnaire completion and 2.5 2.0 years past
CM diagnosis. Over half (54) were male and 39
had symptoms of CHF at diagnosis. Disease type
was dilated CM in 51, hypertrophic in 38, and
mixed type in 11. The overall FSIIR score was
88 ? 15 (median 96), significantly lower than the
healthy normative sample. The Parent Report CHQ
was completed for the 115 PCMR subjects age 5
years and over. Their disease characteristics
and CHQ results are reported below.
METHODS
The PCMR collects information by chart review on
children with CM under age 18 at diagnosis from
over 100 pediatric cardiac centers in North
America. All enrolled patients have CM according
to 1) strict echocardiographic criteria (at least
2 abnormal LV measurements or 1 pattern of CM)
or 2) Pathologic diagnosis of CM on autopsy or
endomyocardial biopsy or 3) Other clinical
evidence of CM provided by the cardiologist.
There were 14 clinical exclusion criteria
(Grenier et al., 2000), including a congenital
heart defect not associated with a malformation
syndrome, endocrine disease known to cause
myocardial damage and chronic arrhythmia. The
Functional Status Substudy began in 2001 and
enrolled non-transplanted children from 5 PCMR
centers. Parents complete annually the 50-item
Parent Report Child Health Questionnaire (CHQ) if
the child is ? age 5 yr (Landgraf Ware, 1996)
and the Functional Status II Revised instrument
(FSIIR Stein Jessop, 1990). The CHQ has 10
domains and 2 summary scores (Physical and
Psychosocial). The FSIIR has 2 versions and is
appropriate for infants and children, with a
summary score based on 14 items common to all
ages.  
CONCLUSION
Children with CM have significantly impaired
physical and psychosocial function relative to
healthy children and functional status is
associated with fractional shortening.
Children with CM are significantly impaired in
overall physical and to a lesser degree,
psychosocial functioning. Domain scores indicate
impairment in global health and parental impact
on emotions as well. Children with CM do not
differ from healthy children in behavior, mental
health, or self-esteem.