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, 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.
Kang
Hyoung Jin
HJ
Department of Pediatrics, Cancer Research Institute, Seoul National University College of Medicine, Seoul, Korea.
Shin
Hee Young
HY
Ko
Sun Hie
SH
Park
Jeong Ah
JA
Kim
Eun Kyung
EK
Rhim
Jung Woo
JW
Kim
Joong Gon
JG
Ahn
Hyo Seop
HS
D016026:Bone Marrow Transplantation / Q000009:adverse effects; D002675:Child, Preschool; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D008297:Male; D019172:Transplantation Conditioning; D014923:Wiskott-Aldrich Syndrome / Q000601:surgery*