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

Peters, Charles; Charnas, Lawrence; Tan, Ye; Ziegler, Richard; Shapiro, Elsa; E. DeFor, Todd; S. Grewal, Satkiran; J. Orchard, Paul; L. Abel, Susan; I. Goldman, Anne; K.C. Ramsay, Norma; E. Dusenbery, Kathryn; J. Loes, Daniel; A. Lockman, Lawrence; Kato, Shunichi; Aubourg, Patrick; W. Moser, Hugo; Krivit, William

doi:10.1182/blood-2003-10-3402

Public

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

Published in Blood • Apr 12, 2004

Authors:

Charles Peters

Lawrence Charnas

Ye Tan

Abstract

Cerebral X-linked adrenoleukodystrophy (X-ALD) is a disorder of very-long-chain fatty acid metabolism, adrenal insufficiency, and cerebral demyelination. Death occurs within 2 to 5 years of clinical onset without hematopoietic cell transplantation (HCT). One hundred twenty-six boys with X-ALD receiv...

Subject

Adrenoleukodystrophy

Medicine

Hematopoietic stem cell transplantation

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