BJH - volume 14, issue 6, october 2023
M. Piccart MD, PhD, J. Vansteenkiste MD, PhD, H. Prenen MD, PhD, F. Duhoux MD, PhD, A. Janssens MD, PhD, M. Delforge MD, PhD, A. Awada MD, PhD, P. Neven MD, PhD, A. Sibille MD, B. Neyns MD, PhD
The oncological treatment landscape is evolving at a very rapid pace with a continuous stream of novel treatment options. To fully leverage these therapeutic advances in clinical practice it is important to facilitate a rapid access to innovative anticancer drugs for patients. Specifically for Belgium, the delay from EMA approval to reimbursement for anticancer drugs is very long, with an average of almost 600 days, and a substantial proportion of innovative drugs never make it to the patient. The stringent focus of the Belgian Commission for Reimbursement of Medicines (CRM) on overall survival (OS) data in reimbursement decisions is believed to be an important contributor to this situation. However, the ever-increasing pace at which new anticancer therapies are being developed in combination with an earlier detection of cancer will make it increasingly difficult to present mature OS data at the time of regulatory approval in the years to come. As such, there is an urgent need for a debate with the regulators to consider more readily available endpoints in their reimbursement assessments. In fact, when a strong treatment effect is seen on an intermediate endpoint such as disease-free or progression-free survival, a benefit in OS is quite likely. As such, our reimbursement system needs to be adapted to better align with the scientific progress in oncology. In this, a temporary reimbursement decision based on intermediate endpoints could give Belgian patients earlier access to promising lifesaving medicines. In this, we as oncologists, including specialists in haematology, respiratory oncology, and gastrointestinal cancer, strongly encourage the CRM to use the grading provided by the ESMO magnitude of clinical benefit scale to evaluate the clinical added value of future cancer treatments. This will not only facilitate a faster patient access to innovative therapies, but will also help policy-makers in advancing ‘accountability for reasonableness’ in their resource allocation deliberations.
(BELG J HEMATOL 2023;14(6):245–9)
Read moreBJH - volume 14, issue 5, september 2023
M. Delforge MD, PhD
The treatment of relapsed and refractory multiple myeloma (MM) currently undergoes a new (r-)evolution. Real-world data like those from the LocoMMotion study have confirmed the grim outcome of MM patients who have received ≥3 treatment lines or who are refractory to the current backbone treatments that include a proteasome inhibitor, an immunomodulatory drug (IMiD) and an anti-CD38 monoclonal antibody.1 The median progression-free survival (PFS) and overall survival (OS) of this heavily pre-treated patient population is around four months and their overall survival barely exceeds one year. Until recently, the treatment options for this patient population were very limited and consisted mostly of retreatment with the drugs that had already been given in earlier treatment lines.2 Currently, the recently introduced T-cell redirecting therapies are completely reshaping the treatment paradigm of relapsed and refractory MM (triple class exposed and mostly triple class refractory). These therapies include CAR-T cells, bispecific antibodies and to a lesser extent antibody drug conjugates. Whereas B-Cell Maturation Antigen (or BCMA) is the most frequently targeted surface antigen, other targets include G-Protein Coupled Receptor family C group 5 member D (or GPRCD). It was therefore no surprise that the oral sessions on treatment of MM were nearly completely dedicated to T-cell redirecting therapies. In the following article, the most recent insights presented at the EHA congress in Frankfurt are discussed.
(BELG J HEMATOL 2023;14(5):216–8)
Read moreBJH - volume 13, issue 7, november 2022
N. Kint MD, PhD, M. Delforge MD, PhD
Plasma cell leukaemia (PCL) is a rare and aggressive plasma cell malignancy and is generally considered to be the final stage of multiple myeloma (MM). Although treatment modalities for MM have significantly evolved in the past decades, PCL unfortunately still retains an overall dismal prognosis, with most patients presenting with a highly symptomatic and aggressive disease course. We present a case of a transplant-ineligible patient diagnosed with a primary PCL who had an indolent presentation and achieved a durable complete remission after treatment via bortezomib-lenalidomide-dexamethasone (VRD). The present case illustrates the clinical heterogeneity of plasma cell disorders, and highlights the necessity of careful cytomorphological and flow cytometric analysis of aberrant lymphoid cells, even in the absence of stigmata of MM.
(BELG J HEMATOL 2022;13(7):277–80)
Read moreBJH - volume 12, issue 8, december 2021
N. Meuleman MD, PhD, J. Caers MD, PhD, K. Fostier MD, P. Vlummens MD, S. Pans MD, L. Goethals MD, PhD, J. Alexiou MD, M. Cliquennois MD, C. Doyen MD, PhD, A. de Weweire MD, R. Schots MD, PhD, M. Delforge MD, PhD, M-C. Vekemans MD
Despite major improvements in the diagnosis and treatment of multiple myeloma (MM), bone damage remains a major feature of this disease. With the development of new diagnostic tools, conventional skeletal studies have been progressively replaced by novel imaging techniques. Today, imaging plays a crucial role in defining symptomatic multiple myeloma, measurement of the extent of skeletal involvement and assessing therapeutic response including minimal residual disease (MRD). Based on an extensive review of the recent literature, we propose an array of Belgian recommendations for myeloma imaging to be used as a reference by haematologists in their daily practice.
(BELG J HEMATOL 2021;12(8):338–43)
Read moreBJH - volume 12, issue 6, october 2021
A. Kentos MD, PhD, N. Mavroudakis MD, PhD, M. Delforge MD, PhD
Monoclonal gammopathy of undetermined significance (MGUS) is quite frequent in the general population. The association between MGUS and peripheral neuropathy (PN) was described in various studies demonstrating a higher than expected prevalence of PN in patients with MGUS. The demonstration of causality remains a diagnostic challenge as a coincidental association may also occur. Specific diagnostic criteria are available for only a few disorders: DADS, POEMS, amyloidosis, cryoglobulinemia. Data to guide management are quite limited. We present a short review of the literature and emphasise the need of a close collaboration between haematologists and neurologists for an optimal management.
(BELG J HEMATOL 2021;12(6):251-7)
Read moreBJH - volume 12, issue 3, may 2021
Y. Serroukh MD, PhD, M. Delforge MD, PhD
Despite significant progress in management of multiple myeloma (MM), prognosis of patients who fail standard treatment options is dismal. Therefore, refractory MM remains an unmet medical need. CAR-T cells are a form of cellular immunotherapy redirecting autologous T cells against tumour antigens after in vitro manufacturing. B-cell maturation antigen (BCMA) is the most promising target antigen for the development of CAR-T cell therapy for MM. In this review, we briefly go through the basics of CAR-T cell therapy applied to MM. We present the results on efficacy and safety of two recently developed CAR-T cell products: idecabtagene vicleucel (ide-cel or bb2121) and ciltacabtagene autoleucel (cilta-cel or JNJ-4528) and put them in perspective with what is published for approved CD19-CAR-T cells.
(BELG J HEMATOL 2020;12(3):128-31)
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BJH - volume 11, issue 8, december 2020
M-C. Vekemans MD, C. Doyen MD, PhD, K.L. Wu MD, PhD, A. Kentos MD, PhD, P. Mineur MD, L. Michaux MD, PhD, J. Caers MD, PhD, N. Meuleman MD, PhD, M. Delforge MD, PhD, On behalf of the BHS Myeloma Subgroup
With the introduction of immunomodulatory drugs, proteasome inhibitors and anti-CD38 monoclonal anti-bodies, major improvements have been achieved in the treatment and outcome of multiple myeloma (MM). Different treatment combinations are now in use and other therapies are being developed. This rapidly changing therapeutic landscape urges for an update on practical guidelines. Based on an extensive review of the recent literature, we propose recommendations on myeloma management, to be used by haematologists as a reference for daily practice.
(BELG J HEMATOL 2020;11(8):357-75)
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