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

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.

• Authors: Hyoung Jin Kang, Hee Young Shin, Sun Hie Ko, Jeong Ah Park, Eun Kyung Kim, Jung Woo Rhim, Joong Gon Kim, Hyo Seop Ahn
• Journal: Journal of Korean medical science (J Korean Med Sci) Vol. 23 Issue 1 Pg. 146-8 (Feb 2008) ISSN: 1011-8934 [Print] Korea (South)
• PMID: 18303217 (Publication Type: Case Reports, Journal Article)

Topics

• Bone Marrow Transplantation (adverse effects)
• Child, Preschool
• Graft vs Host Disease (etiology)
• Humans
• Male
• Transplantation Conditioning
• Wiskott-Aldrich Syndrome (surgery)