J. Blokken PhD, PharmD

Highlights in acute myeloid leukaemia and myelodysplastic syndromes

SUMMARY

EHA 2023 again featured a plethora of interesting oral abstracts and poster presentations in the field of acute myeloid leukaemia (AML) and myelodysplastic syndromes (MDS). In this article, we will highlight some of the most promising data with regard to AML clinical studies, novel combinations in the treatment of AML and risk stratification. Furthermore, we will discuss some important clinical updates in the field of MDS.

(BELG J HEMATOL 2023;14(5):221–5)

Navigating secondary acute myeloid leukaemia in 2023

SUMMARY

In 2022, both the World Health Organisation (WHO) and International Consensus Classification (ICC) have updated the classification of acute myeloid leukaemia (AML) in order to reflect recent advances in disease understanding. In terms of secondary AML, there are now new definitions of therapy-related AML and AML with myelodysplasia-related changes. Furthermore, it is becoming more and more clear that also older patients benefit from intensive chemotherapy, followed by haematopoietic stem cell transplantation. With regard to Vyxeos Liposomal, a multitude of real-world studies could confirm the safety and efficacy that was observed in the randomised, phase III Study 301.

Long-term outcomes of isatuximab in high-risk multiple myeloma

SUMMARY

Patients with high-risk multiple myeloma (HRMM) represent a subgroup of 15–20% of MM patients and have reduced survival rates.^1,2 The current classification of HRMM includes the presence of at least one mutation associated with poor prognosis, such as del(17p), t(4;14), or t(14;16), and was extended to include 1q21+ in the second revision of the Revised International Staging System.^3,4 In recent years, new diagnostic and prognostic factors have emerged, making the HRMM disease profile even more complex.^5,6 As the prevalence of MM continues to increase globally, and the disease is still incurable, extensive research is being conducted to improve current treatment strategies.⁷ One potential improvement is the addition of anti-CD38 antibodies, such as isatuximab or daratumumab, to existing treatment regimens. At EHA 2023, Prof. Dr. Thierry Facon (Lille University Hospital, France) presented the long-term results of isatuximab in combination with carfilzomib-dexamethasone (Isa-Kd) in patients with 1q21+ status.⁸ Prof. Dr. Philippe Moreau (University Hospital of Nantes, France) discussed the results of Isa-Kd and isatuximab in combination with pomalidomide-dexamethasone (Isa-Pd) in MM patients with an ultra-high-risk profile.⁹ In addition, new data demonstrated that isatuximab with weekly Kd can be used as an appropriate and more convenient salvage regimen in patients with relapsed and/or refractory multiple myeloma (RRMM).¹⁰

Journal scan

SUMMARY

In this section of the Belgian Journal of Hematology (BJH), we aim to provide a snapshot of pivotal studies published in recent issues of the most important international journals focusing on haematology. Importantly, the selection of the studies discussed here is the sole responsibility of the publisher and was not influenced by third parties. Do you miss an important study, or did you read a hidden jewel that deserves to be shared with your colleagues? Please, let us know (editor@bjh.be) and we will make sure to include it in the journal scan section of the next BJH issue.

(BELG J HEMATOL 2023;14(4):192–5)

Journal Scan

SUMMARY

In this section of the Belgian Journal of Hematology (BJH), we aim to provide a snapshot of pivotal studies published in recent issues of the most important international journals focusing on haematology. Importantly, the selection of the studies discussed here is the sole responsibility of the publisher and was not influenced by third parties. Do you miss an important study, or did you read a hidden jewel that deserves to be shared with your colleagues? Please, let us know (editor@bjh.be) and we will make sure to include it in the journal scan section of the next BJH issue.

(BELG J HEMATOL 2023;14(3):145–8)

Bispecific monoclonal antibodies for the treatment of lymphoma and multiple myeloma

Bispecific antibodies (BsAbs) represent a promising new treatment modality for many patients. Several clinical trials with BsAbs have emerged over the past years, either evaluating these agents monotherapy or in combination with other drugs. BsAbs offer an off-the shelf alternative for CAR-T cell therapy and have already been used successfully as salvage therapy after CAR-T cell failure. First, Prof. Guillaume Cartron (CHU Montpellier, University of Montpellier) gave an update of the use of BsAbs in non-Hodgkin lymphoma. Thereafter, Prof. Nathalie Meuleman (Hôpital Erasme, ULB) shared her expertise and gave an overview on the use of bispecifics in multiple myeloma.

Ethics & Economy – How to get a drug reimbursed in Belgium?

The journey a drug makes from the laboratory to the patient’s bedside is very long. After years of clinical research and development, a lengthy administrative procedure needs to be completed. It starts with the granting of a marketing authorisation by the EMA and is followed in Belgium by a reimbursement decision. In order to get a better view on how a drug gets reimbursed in Belgium, Diane Kleinermans (RIZIV-INAMI, President CRM/CTG, Brussels), Marjan Willaert (Policy Advisor – Market Access, Pharma.be, Brussels), and Hanne Wouters (Market Access Advisor, Pharma.be, Brussels) gave an overview on the evaluation and review by the commission for reimbursement of medicines (CRM).