Articles

News in sickle cell disease

BJH - volume 4, issue 3, september 2013

A. Ferster MD, PhD, B. Gulbis MD, PhD

Summary

During the twelfth meeting dedicated to sickle cell disease and hereditary red cell disorders held in Brussels on 21 February 2013, several topics related to major aspects for the management of sickle cell disease were presented. Six of them are summarised.

(BELG J HEMATOL 2013;4(3): 115–116)

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P.31 Study of the miRNome of childhood myelodysplastic syndrome

BJH - 2013, issue BHS Abstractbook, january 2013

H.H. Helsmoortel , T. Lammens PhD, N. Van Roy PhD, A. Uyttebroeck MD, PhD, A. Ferster MD, PhD, Y Benoit MD, PhD, F. Speleman PhD, B. De Moerloose MD, PhD

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P.51 Relationship between thrombin generation and blood flow velocity in large cerebral arteries of children with sickle cell disease

BJH - 2013, issue BHS Abstractbook, january 2013

D. Noubouossie MD, F. Ziereisen , P.-Q. Le , D. Willems , B. Mahadeb , L. Rozen PharmD, M. Ngalula Mujinga , A. Demulder MD, PhD, A. Ferster MD, PhD

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P.52 Newborn screening for Sickle Cell Disease in Brussels, a program with an ongoing clinical outcome improvement

BJH - 2013, issue BHS Abstractbook, january 2013

P.-Q. Le , B. Gulbis MD, PhD, L. Dedeken , C. Heijmans , A. Vanderfaeillie MD, C. Vermylen , S. Huybrechts MD, C. Devalck , F. Cotton , B.C. Nguyen , F. Vertongen , M. Ngalula , A. Ferster MD, PhD

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Less veno-occlusive disease after intravenous versus oral busulfan for autologous haematopoietic stem cell transplantation: the Belgian paediatric experience

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

Summary

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)

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