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Unrelated bone marrow transplantation with a reduced toxicity myeloablative conditioning regimen in Wiskott-Aldrich syndrome
Authors:Kang Hyoung Jin  Shin Hee Young  Ko Sun Hie  Park Jeong Ah  Kim Eun Kyung  Rhim Jung Woo  Kim Joong Gon  Ahn Hyo Seop
Affiliation:Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.
Abstract:
Wiskott-Aldrich syndrome (WAS) is an X-linked congenital immune-deficiency syndrome, and bone marrow transplantation (BMT) has become a curative modality. However, the transplant with the alternative donor needed more intensive conditioning with increased treatment-related toxicities. Recently, fludarabine-based reduced toxicity myeloablative conditioning regimens have been developed for adult myeloid malignancies with promising results of good engraftment and low treatment-related toxicities. To increase the engraftment potential without serious complications, a boy with WAS received successful unrelated BMT with a reduced toxicity myeloablative conditioning regimen composed of fludarabine (40 mg/m2) on days -8, -7, -6, -5, -4, -3), busulfan (0.8 mg/kg i. v. q 6 hr on days -6, -5, -4, -3), and thymoglobulin (2.5 mg/kg on days -4, -3, -2). This novel conditioning regimen could improve the outcome of allogeneic transplantation for other non-malignant diseases such as congenital immune-deficiency syndromes or metabolic storage diseases.
Keywords:Wiskott-Aldrich Syndrome   Bone Marrow Transplantation   Busulfan   Fludarabine
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