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

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.

KEYWORDS:

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

Disclosure statement

No potential conflict of interest was reported by the authors. Research was conducted at the University of Minnesota Masonic Children’s Hospital.

Additional information

Funding

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