Unrelated bone marrow transplantation with a reduced toxicity myeloablative conditioning regimen in Wiskott-Aldrich syndrome

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, flud...

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Bibliographic Details
Published in:Journal of Korean medical science 2008, 23(1), , pp.146-148
Main 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
Format: Article
Language:English
Subjects:
Bone Marrow Transplantation - adverse effects
Case Report
Child, Preschool
Graft vs Host Disease - etiology
Humans
Male
Transplantation Conditioning
Wiskott-Aldrich Syndrome - surgery
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Summary: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.
ISSN:1011-8934
1598-6357
DOI:10.3346/jkms.2008.23.1.146