Time to Transplant in X-Linked Adrenoleukodystrophy

Author:

Bonkowsky Joshua L.1ORCID,Wilkes Jacob2

Affiliation:

1. University of Utah School of Medicine; Primary Children’s Hospital, Intermountain Healthcare, Salt Lake City, Utah, USA

2. Intermountain Healthcare, Salt Lake City, Utah, USA

Abstract

Objectives Cerebral X-linked adrenoleukodystrophy (cALD) is an inflammatory demyelination of the brain that can lead to death unless treated by hematopoietic stem cell transplantation. Survival and improved outcomes for cerebral adrenoleukodystrophy are associated with hematopoietic stem cell transplantation at earliest evidence of disease on magnetic resonance imaging (MRI). Our goal was to determine average duration between diagnosis of cALD and hematopoietic stem cell transplantation. Methods This was a retrospective review of data of patients aged 18 years or younger, using a nationwide administrative health care database (Pediatric Health Information System), with an International Classification of Diseases, Tenth Revision ( ICD-10) diagnosis of adrenoleukodystrophy. Time range was October 1, 2015, through June 30, 2021. We determined time to hematopoietic stem cell transplantation by duration between index brain MRI and a code for hematopoietic stem cell transplantation. Results We identified 27 patients with cerebral adrenoleukodystrophy. Total charges for the cohort was $53 million. Time to transplant averaged 97 days. For Hispanic patients, time to transplant was 117 days, compared with 80 days for White, non-Hispanic patients. Comparison of different hospitals showed significant variability in time to hematopoietic stem cell transplantation. Discussion We found that time to hematopoietic stem cell transplantation was >3 months for patients with cerebral adrenoleukodystrophy in the hospitals we evaluated. We noted differences in average time by race/ethnicity and by hospital. Our findings suggest opportunity to reduce time to transplant in cerebral adrenoleukodystrophy.

Funder

National Institutes of Health

Publisher

SAGE Publications

Subject

Neurology (clinical),Pediatrics, Perinatology and Child Health

Reference10 articles.

1. Raymond GV, Moser AB, Fatemi A. X-Linked adrenoleukodystrophy. In: Adam MP, Ardinger HH, Pagon RA, et al., eds. GeneReviews. University of Washington; 1993.

2. Survival analysis of haematopoietic cell transplantation for childhood cerebral X-linked adrenoleukodystrophy: a comparison study

3. Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999

4. Geographic and Specialty Access Disparities in US Pediatric Leukodystrophy Diagnosis

5. Pediatric Health Information System (PHIS) Database web page. Accessed January 6, 2022. https://www.childrenshospitals.org/phis

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