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

Nicole B. Beckmann, Weston P. Miller, Mary S. Dietrich, Paul J. Orchard

Research output: Contribution to journalArticlepeer-review

9 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)986-998
Number of pages13
JournalChild Neuropsychology
Issue number7
StatePublished - Oct 3 2018

Bibliographical note

Funding Information:
This work was supported by the National association of pediatric nurse practitioners Minnesota chapter [Research Scholar Grant].

Publisher Copyright:
© 2017, © 2017 Informa UK Limited, trading as Taylor & Francis Group.


  • Hematopoietic stem cell transplant
  • bone marrow transplant
  • cerebral adrenoleukodystrophy quality of life
  • childhood adrenoleukodystrophy


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