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Quality of life among boys with adrenoleukodystrophy following hematopoietic stem cell transplant.

Authors
  • Beckmann, Nicole B1, 2
  • Miller, Weston P3
  • Dietrich, Mary S4
  • Orchard, Paul J3
  • 1 a University of Minnesota Masonic Children's Hospital , Pediatric Blood & Marrow Transplantation , Minneapolis , MN , USA.
  • 2 b Vanderbilt University Schools of Medicine , Nashville , TN , USA.
  • 3 c University of Minnesota Medical School , Department of Pediatrics, Division of Blood & Marrow Transplantation , Minneapolis , MN , USA.
  • 4 d Vanderbilt University Schools of Medicine and Nursing , Nashville , TN , USA.
Type
Published Article
Journal
Child Neuropsychology
Publisher
Informa UK (Taylor & Francis)
Publication Date
Sep 21, 2017
Pages
1–13
Identifiers
DOI: 10.1080/09297049.2017.1380176
PMID: 28934891
Source
Medline
Keywords
License
Unknown

Abstract

Hematopoietic stem cell transplant (HSCT) is the only accepted treatment capable of halting the progression of X-linked cerebral adrenoleukodystrophy (CALD). While survival and neurological outcomes have been described, there is little information regarding the quality of life (QoL) of transplanted patients with CALD. This analysis is a cross-sectional study of QoL in 16 males diagnosed with CALD who underwent HSCT at a single institution. Each child or parent proxy completed subscales from the Neuro-QoL and the PROMIS Pediatric Profile Instrument representing physical, mental, and social health domains. Descriptive statistics summarized the demographic characteristics and QoL subscale T-scores, Spearman Rho correlations identified the relationships among the variables, and Mann-Whitney tests examined group differences between those with pre-HSCT Loes scores <10 and those with pre-HSCT Loes scores ≥10. The median age of respondents at the time of transplant was 8 years at HSCT (5-14) with a median of 5 years since HSCT (0.5-11). Scores from the selected QoL subscales were similar to healthy peers, though those with pre-HSCT Loes scores ≥10 had lower mobility, upper extremity function, peer interaction, and higher scores for anxiety. Although HSCT has the capability of halting progression of CALD, those with pre-HSCT Loes scores ≥10 after HSCT are at-risk for poor QoL. Longitudinal monitoring is necessary to further appreciate the factors affecting QoL among boys with CALD after HSCT, and how this may be improved.

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