BJH - volume 15, issue 8, december 2024
N. Elbert PhD
Following the lead of the United States (US) Food and Drug Administration (FDA), the European Medicines Agency (EMA) approved gene-editing therapy with exagamglogene autotemcel in early 2024 for the treatment of patients with transfusion-dependent β-thalassemia or severe sickle cell disease. A year earlier, the FDA granted marketing authorisation for gene therapy with lovotibeglogene autotemcel in patients with severe sickle cell disease. This article describes the results of the clinical trials on the basis of which these new treatments were authorised for marketing.
(BELG J HEMATOL 2024;15(8):313–4)
Read moreBJH - volume 15, issue 8, december 2024
R. van der Voort PhD
Teclistamab is registered as a monotherapy in patients with relapsed or refractory multiple myeloma after previous ‘triple-class’ therapy. Recently, results from the MajesTEC-1 trial showed that teclistamab was safe and effective not only in patients who received ‘triple-class’ therapy in the past, but also for those who received both ‘triple-class’ therapy and BCMA-targeted therapy.
(BELG J HEMATOL 2024;15(8):315–7)
Read moreBJH - volume 15, issue 7, november 2024
A. Reekmans MD, U. Ilan MD, J.M. Boer PhD, T. Lammens PhD, S. Goossens PhD, C.M. Zwaan MD, PhD, M.L. den Boer PhD, B. De Moerloose MD, PhD
Despite improvements in the outcome of newly diagnosed paediatric haematological malignancies, the prognosis of refractory and high-risk relapsed patients remains poor, and their treatment is challenging. The increased use of molecular and functional profiling technologies and the growing number of targeted therapies provide opportunities to alter the treatment landscape of these patients and offer the potential to improve patient outcomes. However, this approach comes with many challenges. First, there is a need for accessibility to the analysis and interpretation of sequencing technologies. Second, the prioritisation of targetable events and matching treatments needs to be established. Third, there is a need to facilitate access to early-phase clinical trials. Recently, the international Leukaemia/Lymphoma Target Board (iLTB, NCT05270096, ITCC107) was initiated by the Prinses Máxima Centre and developed in collaboration with the international community to address these barriers. The iLTB provides a forum for an international panel of experts to advise the treating physicians on the best possible treatment options for their patients diagnosed with relapsed or refractory haematological malignancies, for which there is no standard treatment option. In addition, the iLTB aims to facilitate enrolment in clinical trials and collect structured real-world data on patients treated with compassionate use programs. The purpose of this article is to discuss the current challenges in relapsed and refractory haematological malignancies and to introduce the use of international tumour boards to help physicians when confronted with difficult cases.
(BELG J HEMATOL 2024;15(7):263–8)
Read moreBJH - volume 15, issue 6, october 2024
P. Sriskandarajah MD, MRCP, FRCPath, PhD, D.H. Radia MD, MRCP, FRCPath
Systemic Mastocytosis (SM) is a rare, clinically heterogeneous haematological disorder. Over 90% of patients carry a mutation in the receptor tyrosine kinase KIT (i.e. KIT D816V) resulting in the clonal expansion of neoplastic mast cells (MCs) impacting multiple organs and leading to variable clinical presentations ranging from skin-limited disease to more aggressive variants associated with multi-organ dysfunction and reduced survival. Furthermore, the quality of life can be impaired by excess mediator release from neoplastic mast cells resulting in a significant symptom burden. Based on the above, there have been increasing efforts to develop novel, targeted therapies in order to improve quality of life while also affecting survival outcomes. This review will give an overview of the diagnostic work-up for SM patients as well as therapeutic management, including updates from recent clinical trials.
(BELG J HEMATOL 2024;15(6):216–24)
Read moreBJH - volume 15, issue 4, june 2024
S. Le Roy MD, R. de Putter MD, L. Vandepitte PharmD, K. Vandepoele PhD, K. Claes BM, PhD, T. Kerre MD, PhD, I. Moors MD
The awareness of potential germline predisposition in patients with acute myeloid leukaemia (AML) has increased in the years since NGS testing with large gene panels became standard of care. Yet, it must be noted that still little is known regarding incidences in the Belgian population and specific guidelines for clinical practice are lacking. This narrative review attempts to provide an overview of the most common germline variants in the context of AML, optimal diagnostic approaches, and the impact on the patient and family. In a retrospective study of a cohort of 241 AML-patients, we describe the current situation at Ghent University Hospital. Using the available NGS panels, we identified twelve patients with germline pathogenic variants: 5.0% of the total cohort, 34.3% of the patients that were referred for germline testing. It must be realized that the NGS panels expanded during the study period, and probably will expand further in the future: the amount of germline pathogenic variants will likely be higher. This demonstrates the importance of awareness for underlying germline predisposition, and the implications for the patient, their family, as well as during donor search in case of allogenic stem cell transplantation.
(BELG J HEMATOL 2024;15(4):135–46)
Read moreBJH - volume 15, issue 3, may 2024
A. Janssens MD, PhD, C. Lambert MD, PhD
Chemotherapy-induced thrombocytopenia (CIT) is a common complication of cancer treatment that poses a severe clinical burden to patients with solid or haematologic malignancies. As this thrombocytopenia can present a barrier to continue chemotherapy at full dose and on schedule, it can hamper the patient’s long-term oncologic outcomes. Despite the clinical challenges related to CIT, there are currently no available agents approved by the FDA or EMA for the treatment or prevention of CIT. However, treatment with thrombopoietin receptor agonists (TPO-RAs) may increase platelet counts and benefit the safe administration of full-dose chemotherapy without dose delays. This not only reduces the patient’s bleeding risks, but also benefits the long-term oncologic outcomes. To date, most evidence for the use of TPO-RAs in the setting of CIT come from trials with romiplostim.
(BELG J HEMATOL 2024;15(3):94–102)
Read moreBJH - volume 15, issue 2, march 2024
A.G.H. Niezink MD, R.W.M. van der Maazen MD, PhD, B.D.P. Ta MSc, H. Vos-Westerman MD, C.P.M. Janus MD, L.A. Daniels MD, PhD
Chimeric antigen receptor T-cell (CAR T-cell) therapy is available in the Netherlands as a third-line treatment option in patients with large B-cell lymphoma. Bridging is often required during the waiting period between leukapheresis and CAR T-cell infusion. Radiotherapy is an increasingly applied bridging strategy, which is well tolerated by patients and also safe and effective. The most commonly used radiation schedule is 20 Gy in five fractions. In addition to bridging, radiotherapy can also be used as consolidation or salvage treatment after CAR T-cell therapy. Involving a radiation oncologist in a timely manner for patients who are going to be treated with CAR T-cell therapy is of benefit.
(BELG J HEMATOL 2024;15(2):37–42)
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