Children’s Health-Related Quality of Life After Brachial Plexus Birth Injury

Abstract

This cross-sectional study assesses overall health-related quality of life among children in North America who have brachial plexus birth injury.

Introduction

Brachial plexus birth injury (BPBI) is the most common serious birth injury, occurring in 1 to 3 per 1000 live births, with approximately one-third of affected children experiencing permanent neurologic deficits.¹ Despite advances in treatments, the musculoskeletal sequelae of BPBI remain incurable and impair many physical and nonphysical health factors. The relative contributions of these health factors to overall health-related quality of life (HRQOL) are unknown, limiting the ability to optimally allocate resources to provide impactful care. Using the International Classification of Functioning, Disability, and Health (ICF)² as a conceptual framework, we sought to (1) assess overall HRQOL in children with BPBI, (2) evaluate components of HRQOL across all ICF domains (including body structure, body function, activity and/or participation, and environment), and (3) determine the relative contributions of these components to overall HRQOL.

Methods

This multicenter cross-sectional study was approved by the institutional review boards at all study sites, and written informed consent was obtained from the guardians of all participants. The study followed the Strengthening the Reporting of Observational Studies in Epidemiology (STROBE) reporting guideline.

We prospectively recruited children aged 8 to 18 years at 4 quaternary BPBI centers in North America (Boston Children’s Hospital, Boston, Massacusetts; The Hospital for Sick Children, Toronto, Ontario, Canada; Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio; and Shriners Hospitals for Children Northern California, Sacramento). Participants underwent physical examinations guided by iPLUTO international expert consensus³ and completed pediatric patient-reported outcome measures (PROMs) informed by the ICF (eTable in Supplement 1). Participants’ PROM scores were compared with normative data where available and between upper (C5-C6/7 nerve root [shoulder and elbow affected]) and global (C5-C8/T1 nerve root [entire upper limb affected]) BPBI. All physical examination measures and PROMs were tested for correlations with Patient-Reported Outcomes Measurement Information System (PROMIS) Pediatric Global Health using Spearman rank correlation coefficients (ρ), with hypothesis tests conducted against a null correlation of 0. Factor analyses were conducted to identify statistically redundant measures and group measures into latent factors. Structural equation modeling was used to identify associations between these latent factors and PROMIS Pediatric Global Health.

Data analyses were conducted using R, version 4.1.2 (R Project for Statistical Computing). Two-sided P < .05 was considered statistically significant.

Results

The study included 207 children (mean age, 12.1 years [95% CI, 11.8-12.6 years]); 89 (43.0%) were females and 118 (57.0%) were males. The mean birth weight was 4137.0 g (95% CI, 4056.9-4217.1 g ); 156 participants (75.4%) had upper BPBI and 51 participants (24.6%) had global BPBI. The mean PROMIS Pediatric Global Health score for the study cohort (48.5 [95% CI, 47.3-49.7]) was significantly lower than that of the general population vs the PROMIS reference population (mean [SD], 50 [10]) but did not differ between children with upper BPBI (48.4 [95% CI, 47.0-49.9]) vs global BPBI (48.5 [95% CI, 46.5-50.5]). Minimal correlations existed between physical examination components and PROMIS Pediatric Global Health score. The strongest positive correlations with PROMIS Pediatric Global Health score were with measures of emotional functioning (r, 0.52; median score, 70.0 [95% CI, 65.0-70.0]) and family and/or peer relationships (r, 0.47; median score, 55.8 [95% CI, 54.7-57.7]); the strongest negative correlations were with measures of pain interference (r, −0.42; median score, 46.6 [95% CI, 44.3-49.4]), pain intensity (r, −0.37; median score, 4.4 [95% CI, 0-10.0]), and stigma (r, −0.35; median score, 46.6 [95% CI, 45.3-47.5]) (Table).

Table. Patient-Reported Outcomes of Brachial Plexus Birth Injury in Childhood.

Outcome measurea	Correlation with global healthb		Comparison with population normative data
	Correlation coefficient (r)	P value	Score, median (95% CI)	Normative value, mean (SD) or median (range)	P value
PROMIS Pediatric Global Health	NA	NA	48.5 (47.3-49.7)	50.0 (10.0)	.01
PedsQL emotional functioning	0.52	<.001	70.0 (65.0-70.0)	79.0 (18.0)	<.001
PROMIS pediatric family relationships	0.47	<.001	55.8 (54.7-57.7)	50.0 (10.0)	<.001
PROMIS pediatric pain interference	−0.42	<.001	46.6 (44.3-49.4)	50.0 (10.0)	<.001
PROMIS pediatric peer relationships	0.40	<.001	48.9 (46.9-50.5)	50.0 (10.0)	.57
PROMIS pediatric pain intensity	−0.37	<.001	4.4 (0-10.0)	1.0 (0-1.0)	<.001
NeuroQoL pediatric stigma	−0.35	<.001	46.6 (45.3-47.5)	50.0 (10.0)	<.001
BPOM self-assessment of appearance	0.29	<.001	75.0 (50.0-90.0)	NA	NA
CASP total summary	0.27	<.001	87.5 (85.0-90.0)	84.0 (31.0-100)	<.001
PROMIS pediatric upper extremity	0.17	.01	44.5 (41.9-47.1)	50.0 (10.0)	.06
CASE total summary	−0.10	.14	37.0 (35.2-38.9)	46.0 (33.0-100)	<.001

Abbreviations: BPOM, Brachial Plexus Outcome Measure; CASE, Child and Adolescent Scale of Environment; CASP, Child and Adolescent Scale of Participation; NA, not applicable; PedsQL, Pediatric Quality of Life; PROMIS, Patient-Reported Outcomes Measurement Information System.

^a Outcome measures are ordered by absolute value strength of correlation with global health.

^b As defined by the PROMIS Pediatric Global Health scale.

Latent factors identified included social and/or family relationships, self-image, sensation, motor function, and physical structure. Structural equation modeling (Figure) determined that PROMIS Pediatric Global Health score correlated directly with social and/or family factors (standardized effect size, 0.75 [95% CI, 0.32-1.18]), including measures of peer and family relationships, pain interference, and activity participation. Motor function, sensation, and physical structure factors correlated only with PROMIS Pediatric Global Health score indirectly for self-image (standardized effect size, 0.26 [95% CI, 0.03-0.48]), which in turn was associated with social and/or family factors (standardized effect size, 0.71 [95% CI, 0.40-1.02]).

Figure. Flow Diagram of Structural Equation Model.

The model estimates the associations between latent ICF-informed constructs and PROMIS Pediatric Global Health. Latent variables in ovals were derived via confirmatory factor analysis. Model fit was assessed using comparative fit index, Tucker-Lewis index, and root mean square error of approximation (<0.08). For PROMIS and Neuro-QoL scales, higher scores indicate more of the measured construct. For all physical examination measures, including Semmes-Weinstein (SW) monofilament testing, higher scores reflect better function of the measured variable. Path coefficients are standardized: positive values indicate better health-related quality of life (HRQOL) with higher construct scores; negative values indicate inverse associations. For example, a 1-unit increase in social and/or family relationships was associated with a 0.75 SD increase in HRQOL. Indirect effects are also shown. ADD indicates shoulder adduction; AG, against gravity; BPOM, Brachial Plexus Outcome Measure; CASP, Child and Adolescent Scale of Participation; ER, external rotation; GE, gravity eliminated; IR, internal rotation; PROMIS, Patient-Reported Outcomes Measurement Information System.

Discussion

This study found that BPBI was associated with negative outcomes for overall HRQOL in childhood, driven primarily by psychosocial factors and pain rather than by physical parameters. Although impacts of BPBI on psychosocial factors^4,5 and pain⁶ have been previously identified, our findings are, to our knowledge, the first to suggest that these factors are more important than physical function in driving overall patient-reported HRQOL, challenging current treatment paradigms that focus primarily on improving physical function.

The limitations of this study include a sample that was limited to patients from North America, which restricts generalization worldwide, and the cross-sectional design, which limits conclusions regarding changes over time or effects of treatments. Nonetheless, our findings highlight an opportunity to advance care for pediatric musculoskeletal conditions, including BPBI, by expanding the scope of treatment beyond physical function.

Supplement 1.

eTable. Outcome Tools Used to Measure Global HRQOL and All Relevant Domains of the WHO-ICF

Supplement 2.

Data Sharing Statement