Outcomes after allogeneic hematopoietic cell transplantation for childhood cerebral adrenoleukodystrophy: The largest single-institution cohort report

Weston P. Miller, Steven M. Rothman, David Nascene, Teresa Kivisto, Todd E. Defor, Richard S. Ziegler, Julie Eisengart, Kara Leiser, Gerald Raymond, Troy C. Lund, Jakub Tolar, Paul J. Orchard

Research output: Contribution to journalArticlepeer-review

145 Scopus citations

Abstract

Cerebral adrenoleukodystrophy (cALD) remains a devastating neurodegenerative disease; only allogeneic hematopoietic cell transplantation (HCT) has been shown to provide long-term disease stabilization and survival. Sixty boys undergoing HCT for cALD from 2000 to 2009 were analyzed. The median age at HCT was 8.7 years; conditioning regimens and allograft sources varied. At HCT, 50% demonstrated a Loes radiographic severity score ≥ 10, and 62% showed clinical evidence of neurologic dysfunction. A total of 78% (n = 47) are alive at a median 3.7 years after HCT. The estimate of 5-year survival for boys with Loes score < 10 at HCT was 89%, whereas that for boys with Loes score ≥ 10 was 60% (P = .03). The 5-year survival estimate for boys absent of clinical cerebral disease at HCT was 91%, whereas that for boys with neurologic dysfunction was 66% (P = .08). The cumulative incidence of transplantation- related mortality at day 100 was 8%. Post-transplantation progression of neurologic dysfunction depended significantly on the pre-HCT Loes score and clinical neurologic status. We describe the largest single-institution analysis of survival and neurologic function outcomes after HCT in cALD. These trials were registered at www.clinicaltrials.gov as #NCT00176904, #NCT00668564, and #NCT00383448.

Original languageEnglish (US)
Pages (from-to)1971-1978
Number of pages8
JournalBlood
Volume118
Issue number7
DOIs
StatePublished - Aug 18 2011

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