BJH - volume 16, issue 4, july 2025
M. Zivkovic PhD, Q. Van Thillo MD, PhD, P. Verhamme MD, PhD, R.T. Urbanus PhD, R.E.G. Schutgens MD, PhD
Inherited rare bleeding disorders such as Glanzmann thrombasthenia currently lack preventive treatment options. Consequently, severe bleeding episodes are typically managed through platelet transfusions or repeated administrations of recombinant activated factor VII (rFVIIa). This article introduces HMB-001, a bispecific antibody engineered to bind endogenous FVIIa and direct it to sites of vascular injury by targeting TLT-1, a specific platelet receptor, which becomes active on the platelet surface upon injury. In preclinical studies involving non-human primates, HMB-001 was shown to extend the half-life of FVIIa, resulting in elevated plasma levels. In murine models, HMB-001 enhances the haemostatic efficacy of FVIIa by binding to the TLT-1 receptor on activated platelets. Additionally, ex vivo experiments with platelets from individuals with Glanzmann thrombasthenia demonstrate that HMB-001 facilitates the binding of FVIIa to activated platelets, thereby promoting clot formation. Collectively, these findings suggest that HMB-001 represents a promising new approach for the preventive management of bleeding in individuals with Glanzmann thrombasthenia and related disorders.
(BELG J HEMATOL 2025;16(4):169–75)
Read moreBJH - volume 16, issue 2, april 2025
B. Heyrman MD, S. Meers MD, S. Sid MD, K. Van Eygen MD, N. De Beule MD, PhD, M. Clauwaert MD, H. Maes MD, A. Salembier MD, J. Lemmens MD, A. Van De Velde MD, PhD, D. Selleslag MD, J. Bouziotis Msc, N. Put MD, A. De Becker MD, PhD
This report on the real life use of luspatercept in Belgium, describes data of 77 patients. In the response analysis, 65.8% showed a response to treatment, including 35.4% that reached transfusion independency for a minimum of eight weeks. In the responding group, the duration of treatment was at least 38 weeks in 75% of patients. Reasons to stop treatment were adverse event (16,3%), death (23,3%), no response (34,9%) and disease progression (25,6%). One patient stopped treatment due to pronounced fatigue although having an erythroid response. These data confirm the efficacy of luspatercept and the need for real-life data on quality of life.
(BELG J HEMATOL 2025;16(2):65–9)
Read moreBJH - volume 15, issue 8, december 2024
G. Vermeersch MD, T. Devos MD, PhD
Paroxysmal nocturnal haemoglobinuria (PNH) is characterised by the deficiency of glycosylphosphatidylinositol (GPI) anchored proteins, such as CD55 and CD59, on hematopoietic cells resulting in uncontrolled complement activation and haemolysis. Ravulizumab is a new monoclonal antibody with high affinity for complement factor C5 and hereby inhibits complement activation. Due to its significant longer half-life time compared to eculizumab, another C5 targeting complement inhibitor, ravulizumab, is administered intravenously once every eight weeks. Since November 2023, ravulizumab is reimbursed in Belgium for patients with haemolysis and showing clinical symptoms of high disease burden, as well as for clinically stable patients who are treated with eculizumab for at least six months.
(BELG J HEMATOL 2024;15(8):318–24)
Read moreBJH - volume 15, issue 7, november 2024
J. Neefs PharmD, T. Van Nieuwenhuyse PharmD, A. Janssens MD, PhD
Bruton’s tyrosine kinase inhibitors (BTKis) have demonstrated impressive clinical activity and tolerability in several B-cell malignancies, both as single agents and in combination with anti-CD20 monoclonal antibodies. The reimbursement for zanubrutinib, a next-generation BTKi, has recently been extended by the Belgian national public health insurance from Waldenström macroglobulinaemia (WM) to chronic lymphocytic leukaemia (CLL) and marginal zone lymphoma (MZL). This review describes the mechanism of action, dosage and administration, efficacy, and tolerability of zanubrutinib.
(BELG J HEMATOL 2024;15(7):269–80)
Read moreBJH - volume 15, issue 4, june 2024
N. Granacher MD
Myelofibrosis (MF) is a rare Philadelphia chromosome negative myeloproliferative bone marrow disorder characterised by a high symptom burden due to splenomegaly, inflammatory status and cytopenias. Until recently, the only approved and reimbursed pharmacotherapy for non-haematopoietic stem cell transplant candidates in Belgium was ruxolitinib. However, as of September 1st, 2023, the novel JAK (janus kinase) 1/2 inhibitor (JAKi) fedratinib has obtained its reimbursement in second line. The other FDA approved JAKi, momelotinib and pacritinib, play an important role in patient-tailored JAKi treatment but are not yet reimbursed in Belgium.
(BELG J HEMATOL 2024;15(4):165–71)
Read moreBJH - volume 14, issue 6, october 2023
A. De Voeght MD
Magrolimab is a first-in-class monoclonal antibody that targets and inhibits CD47, a “don’t eat me” signal overexpressed on the surface of cancer cells that allows them to evade the innate immune response. Blocking CD47 leads to increased recognition and phagocytosis of cancer cells. Since its initial description and promising data in pre-clinical and clinical studies, three phase III studies investigating magrolimab in AML patients are ongoing in Belgium and worldwide.
(BELG J HEMATOL 2023;14(6):250–4)
Read moreBJH - volume 14, issue 2, march 2023
J. Brijs MD, J. Neefs PharmD, A. Janssens MD, PhD
Lymphomas are the most common haematological malignancy and represent a heterogenous group of lymphoproliferative diseases with a variable prognosis. Chemotherapy and radiotherapy, and anti-CD20 immunotherapy for B-cell lymphomas, currently form the basis of lymphoma treatment. New agents, especially new forms of cancer immunotherapy, such as bispecific antibodies (bsAbs), have expanded therapeutic approaches in the last years. bsAbs have two different antigen binding sites, which enables them to simultaneously target tumour cells and immune effector cells (T-cells). By binding and activating T-cells in the proximity of tumour cells, an effective T-cell mediated anti-tumour response can be achieved. Target antigens in lymphomas are mostly CD19 or CD20 on the malignant B-cell and CD3 on the T-cell. This article will briefly review the basic principles and mechanisms of action of bsAbs, discuss the molecules approved or in advanced clinical development for lymphomas with their most relevant (dose-escalation/dose-expansion) trials, and pay attention to possible adverse events and future perspectives of bsAbs.
(BELG J HEMATOL 2023;14(2):67–72)
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