BJH - volume 3, issue 2, june 2012
S. Huybrechts MD, Y. Beguin MD, PhD, V. Bordon MD, PhD, MF. Dresse , S. Dupont MD, A. Ferster MD, PhD, G. Laureys MD, PhD, I. Meyts , M. Renard , C. Vermylen
Busulfan is commonly used in preparative conditioning regimens prior to haematopoietic stem cell transplantation in children and young adults for malignant and non-malignant disorders. For many years busulfan was only available in oral form, resulting in large inter- and intra-patients variability in plasma exposure, associated with higher graft failure rate as well as higher toxicity such as veno-occlusive disease. With the development of an intravenous formulation of busulfan, a more accurate control of both the inter- and intra-patient variability has been provided. The goal of this study was to evaluate the use and efficacy of intravenous busulfan in comparison with the oral formulation in children undergoing an autologous transplantation after conditioning with busulfan. Despite the small number of patients, this study confirmed the apparent benefit of intravenous busulfan in children undergoing an autologous HSCT. The use of a five-level dose schedule defined by body weight resulted in an efficient engrafitment with marked reduction in the incidence of veno-occlusive disease compared with oral busulfan. In terms of disease-free outcome, survival and event-free survival, similar results have been obtained in both groups. The choice of this formulation of busulfan should therefore be considered.
(BELG J HEMATOL 2012;3:34–40)
Read moreBJH - volume 3, issue 2, june 2012
A. Vandamme MSc, R. Schots MD, PhD, Y. Beguin MD, PhD
The Transplantation Committee of the Belgian Hematological Society (BHS) is supported by all university centres and nonuniversity centres with significant transplant activity. The committee is involved in the development of transplant guidelines and recommendations, the transplant peer review process, contacts with regulatory authorities, the introduction of expanded access and medical need programs and the initiation of academic studies addressing important questions in the transplant field. Since 2008, eight clinical trials have been initiated after approval by the Ethics Committees and the National Competent Authority (AFMPS/FAGG). So far, one of them has been completed and is being prepared for publication. In this paper, we briefly describe the rationale, objectives, treatment arms, major inclusion criteria and current status of these different trials. In addition and for each trial a link is provided to the BHS website to obtain more details regarding inclusion criteria, participating centres and administrative/contact information.
(BELG J HEMATOL 2012;3:62–67)
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