Treatment of X-linked childhood cerebral adrenoleukodystrophy by the use of an allogeneic stem cell transplantation with reduced intensity conditioning regimen

Igor B. Resnick, Ali Abdul Hai, Michael Y. Shapira, Menachem Bitan, Eli Hershkovitz, Arie Schwartz, Miriam Ben-Harush, Reuven Or, Shimon Slavin, Joseph Kapelushnik

Research output: Contribution to journalArticlepeer-review

17 Scopus citations

Abstract

Childhood cerebral form of X-linked adrenoleukodystrophy (X-ALD) is a rapidly progressive demyelinating condition affecting the cerebral white matter, which rapidly leads to total disability and death. The only known curative treatment for this condition is allogeneic hematopoietic stem cell transplantation (HSCT). Procedure-related toxicity is assumed to be the cause of death of patients with X-ALD. Three cases of ALD successfully transplanted with the use of non-myeloablative fludarabine based conditioning are described. Patients showed smooth peri-bone marrow transplantation course with fast and stable engraftment. In the 3- to 5 yr follow-up period, patients showed no deterioration in their clinical and neurological condition. Levels of very long chain fatty acids were very variable and had a tendency to decrease in at least one of the three patients. In another patient, an improvement of magnetic resonance imaging changes was found. Non-myeloablative HSCT should be considered as an early treatment for X-ALD.

Original languageEnglish
Pages (from-to)840-847
Number of pages8
JournalClinical Transplantation
Volume19
Issue number6
DOIs
StatePublished - 1 Dec 2005
Externally publishedYes

Keywords

  • Allogeneic stem cell transplantation
  • Hematopoietic stem cell transplantation
  • Non-myeloablative
  • X-linked childhood cerebral adrenoleukodystrophy

Fingerprint

Dive into the research topics of 'Treatment of X-linked childhood cerebral adrenoleukodystrophy by the use of an allogeneic stem cell transplantation with reduced intensity conditioning regimen'. Together they form a unique fingerprint.

Cite this