Functional Limitations, Physical Disability and Social Competence among HCT Survivors Transplanted during Childhood or Adolescence: A Report from the Bone Marrow Transplant Survivor Study.
Authors: Ness Kirsten K. , Gurney James G. , Baker K. S. , Francisco Liton , Carter Andrea , Forman Stephen J. , Robison Leslie L. , Rosenthal Joseph , Bhatia Smita 
Published: October 2019

Abstract
HCT may result in a number of important disease- and treatment-related late effects, including limitations in functional abilities. This study estimated physical, emotional and educational limitations (i.e. functional limitations) and restrictions in ability to perform personal care or routine daily activities (i.e. physical disability), as well as marital status, employment status in adults, and ability to interact with peers, teachers, and family members (social competence) in children. Subjects included 235 persons who had received an HCT for a malignancy or a hematological disorder before age 21 years and survived at least 2 years post-transplant. For comparison purposes, 705 siblings of cancer survivors were recruited and frequency matched on age, sex and ethnicity. Medical data were abstracted, and subjects or their parents (if <18 years old at survey completion) completed a 24-page mailed questionnaire that included items on medical late effects, current functional limitations, current physical disability, marital status, employment status, educational attainment and social competence. The Brief Symptom Inventory (BSI) was included on the adult version of the questionnaire to evaluate emotional status. A modified version of the Child Behavior Checklist was included on the child version of the questionnaire to evaluate emotional status and social competence. Median age at HCT was 9.8 years, at time of survey completion was 21.8 years, and the median length of follow-up was 10.7 years. Compared with siblings, adult survivors were more likely to report functional limitations in physical (Prevalence Odds Ratio (OR)=2.2; 95% CI 1.9–8.2) and emotional domains (OR=2.9; 95% CI 1.4–5.8), and to report physical disability (OR=3.9; 95% CI 1.9–8.2). Adult survivors were also less likely than siblings to be employed (OR=25.0; 95% CI 10.0–100.0) or married/living as married (OR=2.5; 95% CI 1.7–5.0). Child survivors were more likely than siblings to have participated in special education (OR=3.0; 95% CI 1.5–6.0), to report physical disability (OR=10.8; 95% CI 2.2–53.9), and to have behaviors indicating impaired social competence (OR=2.0; 95% CI 0.9–4.2). The outcome percentages are shown in the table. This study demonstrated that long-term survivors of HCT performed during childhood or adolescence were more likely than a matched sibling comparison group to have functional limitations that impact performance in routine daily activities and interpersonal relationships. Surveillance and long-term follow-up of HCT treated individuals should include monitoring for functional loss and referral for appropriate intervention.
Limitations in Childhood HCT Survivors - Comparison with Siblings
Survivors [N(%)] Siblings [N(%)] Children (<18 at study: Survivors: n=78; Siblings: n=234) Physical Disability 7 (9.0) 2 (0.8) Impaired Social Competence 13 (16.7) 21 (9.0) Adults (18–50 at study: Survivors: n=157; Siblings: n=471 Physical disability 17 (10.8) 14 (3.0) Currently not employed 18–24 year-olds 31 (40.8) 5 (2.3) 20–50 year olds 23 (28.4) 4 (1.6) Not married or living with a partner 18–24 year olds 68 (89.5) 168 (75.7) 20–50 year olds 47 (61.8) 84 (33.7)