Treatment of X-linked childhood cerebral adrenoleukodystrophy by the use of an allogeneic stem cell transplantation with reduced intensity conditioning regimen.
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.
Resnick
Igor B
IB
Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah University Hospital, Hebrew University - Hadassah Medical School, Jerusalem, Israel. gashka@hadassah.org.il
Abdul Hai
Ali
A
Shapira
Michael Y
MY
Bitan
Menachem
M
Hershkovitz
Eli
E
Schwartz
Arie
A
Ben-Harush
Miriam
M
Or
Reuven
R
Slavin
Shimon
S
Kapelushnik
Joseph
J
D000326:Adrenoleukodystrophy / Q000601:surgery*; D002066:Busulfan / Q000008:administration & dosage / Q000627:therapeutic use; D002648:Child; D004359:Drug Therapy, Combination; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D019653:Myeloablative Agonists / Q000008:administration & dosage / Q000627:therapeutic use; D019172:Transplantation Conditioning / Q000379:methods*; D014740:Vidarabine / Q000008:administration & dosage / Q000031:analogs & derivatives / Q000627:therapeutic use
D019653:Myeloablative Agonists; D014740:Vidarabine; D002066:Busulfan; C024352:fludarabine