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Treatment of X‐linked childhood cerebral adrenoleukodystrophy by the use of an allogeneic stem cell transplantation with reduced intensity conditioning regimen
Author(s) -
Resnick Igor B,
Abdul Hai Ali,
Shapira Michael Y,
Bitan Menachem,
Hershkovitz Eli,
Schwartz Arie,
BenHarush Miriam,
Or Reuven,
Slavin Shimon,
Kapelushnik Joseph
Publication year - 2005
Publication title -
clinical transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.918
H-Index - 76
eISSN - 1399-0012
pISSN - 0902-0063
DOI - 10.1111/j.1399-0012.2005.00411.x
Subject(s) - adrenoleukodystrophy , medicine , transplantation , total body irradiation , hematopoietic stem cell transplantation , fludarabine , stem cell , white matter , magnetic resonance imaging , gastroenterology , surgery , chemotherapy , radiology , cyclophosphamide , peroxisome , receptor , biology , genetics
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.