BJH - volume 7, issue 2, april 2016
A. Rogiers MD, E. Porcher , D. Dierickx MD, PhD
Thrombotic thrombocytopenic purpura is a non-malignant but life-threatening haematological disorder caused by deficiency of ADAMTS13, a metalloproteinase cleaving ultra-large von Willebrand factor multimer. Urgent initiation of therapeutic plasma exchange, in most cases associated with corticosteroids, has dramatically increased outcome of patients presenting with thrombotic thrombocytopenic purpura. Recently a phase II trial with caplacizumab, a humanised nanobody binding to the A1 domain of von Willebrand factor, has shown promising results by reducing the number of plasma exchanges with an acceptable safety profile.
(BELG J HEMATOL 2016;7(2):79–81)
Read moreBJH - volume 6, issue 5, december 2015
A. Janssens MD, PhD
The first-in-class Bruton’s tyrosine kinase and phosphatidylinositol-3-kinase delta inhibitors have demonstrated impressive clinical activity and tolerability in several B-cell malignancies, both as single agent or in combination with rituximab. As reimbursement of ibrutinib and idelalisib by the Belgian national public health insurance has been granted, this review describes mechanism of action, dosage and administration, efficacy and tolerability. Although both molecules show a very favourable toxicity profile, treating physicians and patients must be aware of some medical events of interest.
(BELG J HEMATOL 2015;6(5):216–24)
Read moreBJH - volume 6, issue 5, december 2015
K. Fostier MD, R. Schots MD, PhD
Monoclonal antibodies have a profound impact on the prognosis and survival of patients with haematological malignancies. In the treatment of multiple myeloma, until recently, results of monoclonal antibodies have been disappointing. The introduction of two novel classes of monoclonal antibodies holds great promise to change this. Daratumumab (and related antibodies) is a monoclonal antibody directed to CD38, an intriguing multifunctional surface protein abundantly expressed on malignant plasma cells and their precursors. Daratumumab displays impressive single agent activity in heavily pretreated multiple myeloma patients and due to its favourable safety profile, this molecule seems to be an excellent accessory companion to known anti-multiple myeloma regimens and also in monotherapy as a maintenance agent. Elotuzumab, to the contrary, is an anti-CS1 monoclonal antibody, which does not show any clinically relevant single agent activity, but when combined with other anti-multiple myeloma drugs appears to greatly enhance their efficacy and can even revert the refractory state to the agents. When these promising results are confirmed in phase III trials, immunotherapy can finally be incorporated in the treatment schedule of newly diagnosed and relapsed/refractory multiple myeloma patients.
(BELG J HEMATOL 2015;6(5):209–15)
Read moreBJH - volume 6, issue 4, october 2015
D. Bron MD, PhD, M. Maerevoet MD
Phosphoinositide 3-kinase inhibitors represent a new group of promising targeted therapies for malignant hemopathies and primarily lymphoproliferative disorders. This short report summarises recent knowledge on the mechanism of action, the rationale to use it in humans bearing malignant hemopathies and preliminary clinical trials’ data that led to the Food and Drug Administration approval of one of these compounds (idelalisib).
(BELG J HEMATOL 2015;6(4): 152–5)
Read moreBJH - volume 6, issue 2, may 2015
J. Van Droogenbroeck MD
More than ten years ago the dipeptidyl boronic acid derivative proteasome inhibitor bortezomib changed the therapeutic field of multiple myeloma dramatically. This introduction of a whole new class of chemotherapy lead to a significant extension of disease-free and overall survival for most myeloma patients. But as with most first-in-class molecules, efficacy can probably be improved — primary and secondary bortezomib resistance being common – and treatment is all too often limited by dose-limiting side effects.2 Carfilzomib is without doubt a very promising next generation proteasome inhibitor.
(BELG J HEMATOL 2015;6(2):71–3)
Read moreBJH - volume 5, issue 4, december 2014
C. Doyen MD
Relapsed and refractory multiple myeloma patients have a particularly poor prognosis and the development of new drugs is urgently needed. Pomalidomide, a third-generation immunomodulatory drug with a pleiotropic activity, was approved in 2013 by the Food and Drug Administration and the European Medicines Agency, in association with low dose dexamethasone in relapsed and refractory multiple myeloma, in patients who received at least two prior therapies, including bortezomib and lenalidomide and demonstrated progression on the last therapy. In the phase III MM-003 study, pomalidomide associated with low dose dexamethasone was superior to high dose dexamethasone in these patients, with a manageable safety and tolerability profile. This paper will review the available data concerning the mechanisms of action, the efficacy in clinical studies and the safety of this very promising new drug.
(BELG J HEMATOL 2014;5(4):137–42)
Read moreBJH - volume 5, issue 3, september 2014
A. Janssens MD, PhD
Bendamustine is an efficacious treatment for several lymphoma subtypes with a mild toxicity profile. This review will describe practical recommendations concerning administration and adverse events to guide the clinician in the optimal use of this compound.
(BELG J HEMATOL 2014;5(3):97–103)
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