BJH - volume 9, issue 5, september 2018
A. Van De Velde MD, PhD, B. Willekens , L. Vanopdenbosch MD, O. Deryck , D. Selleslag MD, M. D’Haeseleer , A. De Becker MD, PhD, 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 MD, 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
D. van den Bossche MSc, M. Jacquemin MD, PhD, K. Peerlinck MD, PhD
In the past few years, several new treatment options for haemophilia A and B have emerged. Formerly, replacement therapy comprised plasma-derived and recombinant factor VIII and IX concentrates containing human- and animal-derived components associated with a potential risk of contamination with infectious agents. Further optimisation of the manufacturing procedures virtually eliminated these hazards. Nowadays, the major drawbacks of the standard plasma-derived and recombinant factor VIII and IX products are their relatively short half-life. To overcome these limitations, different therapeutic approaches were developed. Novel extended half-life rFVIII and rFIX concentrates allow a reduction of the injection frequency and improve the efficacy of therapy and the quality of life of haemophilia patients. Besides the prophylactic treatment options, important progress has been made in gene therapy. Currently, the major complication of the treatment with FVIII or FIX concentrates is the development of inhibitor antibodies. In these cases, bypassing agents allow treating or preventing bleedings. However, the currently available bypassing agents have a short half-life, which limit their use for prophylactic treatment. Accordingly, several new therapies are now being developed to treat patients with inhibitors, including rFVIIa with extended half-life, recombinant porcine FVIII and bispecific antibodies bridging FVIII and FIX.
(BELG J HEMATOL 2018;9(5):175–81)
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 9, issue 3, june 2018
Y. Serroukh MD, PhD, H. Claerhout MD, A. Janssens MD, PhD, T. Tousseyn MD, PhD, N. Boeckx MD, PhD, J. Maertens MD, PhD, T. Devos MD, PhD
Aplastic anaemia is a rare condition characterised by pancytopenia and bone marrow hypocellularity and caused by the immune-mediated destruction of the haematopoietic precursors. The early complications are related to cytopaenias with infections being the major cause of morbi-mortality. The main long-term issue is clonal evolution to myelodysplastic syndrome or acute leukaemia. The diagnosis relies on exclusion of other causes of pancytopenia and characteristic pathologic findings. Severity is stratified according to peripheral blood counts. Nowadays, the survival of treated patients reaches 80–90%. The treatment of the severe form of aplastic anaemia consists on haematopoietic stem cell transplantation in eligible patients and immunosuppressive therapy in non-transplant candidates. Supportive therapy is an option in frail and/or elderly patients. Here, we define and briefly review the pathogenesis of aplastic anaemia. We propose a diagnostic and therapeutic strategy based on existing literature and experts’ recommendations. We finally report three cases illustrating particular clinical associations with pregnancy, hepatitis and paroxysmal nocturnal haemoglobinuria.
(BELG J HEMATOL 2018;9(3):76–85)
Read moreBJH - volume 9, issue 2, march 2018
S. Meers MD
The field of myelodysplastic syndromes has entered the molecular era. New diagnostic tools such as next-generation sequencing are rapidly entering clinical practice and will change the way we diagnose and manage patients with a myelodysplastic syndrome, especially patients considered lower risk by standard diagnostic tools. The treatment of lower risk patients has not changed much since the publication of the BHS recommendations in 2013. However, important trials in lower risk patients have recently been published and will be reviewed here. Finally, the recommendations from an international expert panel for allogeneic transplantation have been published. The key points of this paper will also be discussed as well as results of recently published trials.
(BELG J HEMATOL 2018;9(2):48–56)
Read moreBJH - volume 9, issue 2, march 2018
B. De Moerloose MD, PhD, E. Nauwynck MD, K. Arts MD, L. Willems MD, PhD, V. Labarque MD, PhD, T. Lammens PhD, A. Uyttebroeck MD, PhD
Infant leukaemia is a rare disease but the 3rd most frequent malignancy in this age group. Both acute lymphoblastic leukaemia and acute myeloid leukaemia in the first year of life have particular clinical and biological characteristics such as B-cell phenotype with co-expression of myeloid markers in acute lymphoblastic leukaemia, FAB M5 or M7 in acute myeloid leukaemia, the presence of extramedullary symptoms and a high frequency of KMT2A rearrangements. Survival rates for infant acute leukaemia are worse than for older children. In this study, the characteristics and outcome of 50 infants with acute lymphoblastic leukaemia and acute myeloid leukaemia treated at the University Hospitals of Ghent and Leuven between 1989 and 2015 were studied and correlated with literature data. With event-free survival and overall survival rates of 44% and 52% for the entire cohort, the outcome of these patients was comparable to those in published clinical trials. In general, the event-free survival and overall survival was superior in acute myeloid leukaemia compared to acute lymphoblastic leukaemia infants and not influenced by age (< or ≥6 months), white blood cell count at diagnosis or presence of a KMT2A rearrangement. For future trials in infant leukaemia, the high number of early deaths, toxic deaths and relapses remain the most challenging problems.
(BELG J HEMATOL 2018;9(2):57–63.)
Read moreBJH - volume 8, issue 7, december 2017
K.P.M. van Galen MD, MSc, E.P. Mauser-Bunschoten MD, PhD
Von Willebrand disease is the most common congenital bleeding disorder characterised by mucocutaneous bleeding. However, joint bleeds also occur in a significant proportion of patients with severe Von Willebrand disease. Until recently, joint bleeding did not get much attention in clinical research on Von Willebrand disease, despite the fact that recurrent joint bleeds may lead to arthropathy. Arthropathy in Von Willebrand disease has a negative impact on joint function, participation and quality-of-life. Risk factors are a low FVIII level and a history of more than five joint bleeds. Arthropathy in Von Willebrand disease can be measured using the Haemophilia Joint Health Score and the joint X-ray Pettersson score. The value of magnetic resonance imaging or ultrasound to detect early arthropathy in Von Willebrand disease remains to be determined. The Haemophilia Activities List questionnaire is feasible to quantify functional abilities in Von Willebrand disease. The most important measure to prevent arthropathy is to prevent joint bleeding. Clotting factor prophylaxis has proven very effective to do so. Since there is no general consensus to guide the use of prophylaxis in Von Willebrand disease, the decision on when and how to start prophylaxis is based on individual patient’s assessments by a haemophilia treatment centre. Future studies in Von Willebrand disease arthropathy could address the optimal timing and schedule of prophylaxis, optimal treatment of an acute joint bleed to prevent arthropathy, effectiveness of rehabilitation programs, orthopaedic surgery and the clinical use of measurement instruments. Prospective joint assessments and registration of joint bleeds in future population studies on severe Von Willebrand disease will be a good starting point.
(BELG J HEMATOL 2017;8(7):253–8)
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