Time to Transplant in X-Linked Adrenoleukodystrophy

Area of Science:

• Neurology
• Hematology
• Pediatric Medicine

Background:

• Cerebral X-linked adrenoleukodystrophy (cALD) is a fatal inflammatory brain demyelination.
• Hematopoietic stem cell transplantation (HSCT) offers improved survival for cALD patients.
• Early HSCT, guided by MRI, is critical for better outcomes.

Purpose of the Study:

• To determine the average time from diagnosis of cALD to HSCT.
• To identify factors influencing the time to HSCT for cALD.

Main Methods:

• Retrospective review of pediatric patients (≤18 years) diagnosed with adrenoleukodystrophy.
• Utilized a nationwide administrative healthcare database (2015-2021).
• Calculated time from index brain MRI to HSCT code.

Main Results:

• Identified 27 cALD patients with total charges of $53 million.
• Average time to HSCT was 97 days.
• Hispanic patients (117 days) had longer wait times than White, non-Hispanic patients (80 days).
• Significant hospital-based variability in HSCT wait times was observed.

Conclusions:

• Time to HSCT for cALD exceeded 3 months in evaluated hospitals.
• Disparities in HSCT wait times exist based on race/ethnicity and hospital.
• Opportunities to reduce HSCT wait times for cALD patients are suggested.