BJH - volume 9, issue 5, september 2018
A. Van de Velde MD, B. Willekens , L. Vanopdenbosch MD, O. Deryck , D. Selleslag MD, M. D’Haeseleer , A. De Becker MD, B. Dubois MD, PhD, D. Dierickx MD, PhD, G. Perrotta , V. De Wilde MD, PhD, V. Van Pesch MD, PhD, N. Straetmans MD, PhD, D. Dive MD, Y. Beguin MD, PhD, B. Van Wijmeersch MD, PhD, K. Theunissen , T. Kerre MD, PhD, G. Laureys MD, PhD
Multiple sclerosis is considered to be an immune mediated inflammatory disorder of the central nervous system. It mainly affects young, socioeconomic active patients. Although our armamentarium for this disease has significantly evolved in recent years some patients remain refractory to conventional therapies. In these cases, autologous haematopoietic stem cell transplantation can be considered as a therapeutic option. Decreasing morbidity, mortality and increasing patient awareness have led to rising inquiry by our patients about this treatment option. With the aim of a standardised protocol and data registration, a Belgian working party on stem cell therapy in multiple sclerosis was established. In this paper, we report the consensus protocol of this working party on autologous haematopoietic stem cell transplantation in multiple sclerosis.
(BELG J HEMATOL 2018;9(5):167–74)
Read moreBJH - volume 9, issue 5, september 2018
V. Liberton MD, R. Colman , G. Laureys MD, PhD, V. Bordon MD, PhD, C. Dhooge MD, PhD
Adult patients with high serum ferritin have an increased risk of organ toxicity and iron chelation before stem cell transplant might be an option. We report our experience in 58 paediatric patients (excluding patients with haemoglobinopathy and hyper-transfused patients) undergoing allogeneic stem cell transplant between 2007 and 2012. Serum ferritin pre-transplant was highly variable (mean: 932 µg/L) and related to a number of PRC transfusions. Eighteen of 58 patients had ferritin level >1000 µg/L before transplant. Eight patients suffered from transplant-related mortality. We found no correlation between transplant-related mortality and pre-transplant serum ferritin (p=0.67). Seven patients developed veno-occlusive disease, reversible in all cases. We did not find a correlation between serum ferritin and veno-occlusive disease, graft-versus-host disease or relapse. The evolution of ferritin post-transplant shows a spontaneous lowering of ferritin in the first two years after haematopoietic stem cell transplantation to normal range. An association between serum ferritin and elevated AST/ALT at 12 and 24 months was noted and follow-up concerning possible liver damage in patients with a persistently high serum ferritin is recommended. This study concludes that high serum ferritin has no influence on transplant-related mortality in children, chronically poly-transfused patients excluded. Although chelation is already used in paediatric HSCT, there is insufficient, current evidence to do so.
(BELG J HEMATOL 2018;9(5):182–7)
Read moreBJH - volume 7, issue Abstract Book BHS, january 2016
V. Bordon MD, PhD, C. Dhooge MD, PhD, E. Claerebout , S. Casaert , G. Laureys MD, PhD
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)
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