Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS)
Status:
Completed
Trial end date:
2012-03-01
Target enrollment:
Participant gender:
Summary
Wiskott-Aldrich syndrome (WAS) is a rare X-linked congenital immune-deficiency syndrome and
hematopoietic stem cell transplantation (HSCT) has become a curative modality. But the
transplant with the conventional conditioning resulted in high incidence of treatment related
toxicities and non-myeloablative conditioning resulted in high incidence of engraftment
failure. Recently, fludarabine based reduced toxicity myeloablative conditioning regimen was
developed for adult myeloid malignancies with promising result of good engraftment and low
treatment related toxicities. To increase the engraftment potential without serious
complication, reduced toxicity myeloablative conditioning regimen composed of fludarabine,
busulfan, and thymoglobulin is designed for Wiskott-Aldrich syndrome.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
The Korean Society of Pediatric Hematology Oncology