Articles

Understanding hairy cell leukaemia after analysing the University Hospitals Leuven database retrospectively

BJH - volume 15, issue 6, october 2024

H. Lismont MD, Ir J. Van Ham , A. Janssens MD, PhD

SUMMARY

Hairy cell leukaemia (HCL) is a rare hematologic malignancy with high response rates after purine-analogue based therapy and an excellent long-term prognosis. We reviewed 104 HCL patients diagnosed between 1980 and 2022 at the University Hospitals Leuven to analyse long-term outcomes and complications. Median follow-up was twelve years. In total, 96 patients (92%) received a first-line treatment consisting of splenectomy (n=13), interferon-α (n=13) and cladribine (n=70). This last therapy resulted in the best response rates (overall response (OR) 99%, complete response (CR) 71%) and a long-lasting progression-free-survival (median PFS ten years). Forty-three percent of patients received multiple therapies for subsequent relapses. The median number of treatment lines was one. The median overall survival (OS) was 30.8 years with a 5-year and 10-year OS of approximately 98% and 91%. Although the prognosis of HCL patients is very good, infections and second malignancies are frequently observed. In this cohort, 55% of the patients had a major infection with an infection-related mortality of 3%. After diagnosis, 22% of the HCL patients developed one or more second malignancies, ranging from 1–3 per patient, with a 10-year cumulative risk of 14.5%.

(BELG J HEMATOL 2024;15(6):249–56)

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INTRODUCTION

BJH - volume 15, issue 6, october 2024

A. Janssens MD, PhD

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Highlights in chronic lymphocytic leukaemia

BJH - volume 15, issue 5, september 2024

A. Janssens MD, PhD

SUMMARY

At EHA 2024 new information on TP53 mutations in chronic lymphocytic leukemia (CLL) were presented together with preliminary or more mature data on doublets and triplets of novel agents. In the session on novel therapies in relapsed/refractory (R/R) CLL we were informed on the preliminary efficacy and safety of two BTK degraders (BGB-16673,NX-5948) and on the results of the bcl-2 inhibitor sonrotoclax in combination with zanubrutinib. Preliminary results on characteristics associated with response to lisocabtagene maraleucel (Liso-cel) in R/R CLL were also communicated.

(BELG J HEMATOL 2024;15(5):184–89)

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The role of thrombopoietin receptor agonists in the management of chemotherapy-induced thrombocytopenia

BJH - volume 15, issue 3, may 2024

A. Janssens MD, PhD, C. Lambert MD, PhD

SUMMARY

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)

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BHS guidelines for the management of small lymphocytic lymphoma and chronic lymphocytic leukaemia, anno 2024

BJH - volume 14, issue 7, november 2023

A. Janssens MD, PhD, On behalf of the BHS Lymphoproliferative Disease Committee 2023: Marc André , Christophe Bonnet , Sarah Bailly , Veerle Beckers , Dominique Bron , Rutgers Callens , Charlotte Caron , Maxim Clauwaert , Sarah Debussche , Ciel De Vriendt , Virginie De Wilde , Vanessa Delrieu , Daan Dierickx , Pierre Heimann , Jan Lemmens , Marie Maerevoet , Claire Maquet , Fulvio Massaro , Caressa Meert , Marie-Christine Ngiribacu , Fritz Offner , Juliette Rademaekers , Ornella Rizzo , Kirsten Saevels , Liesbeth Schauvliege , Sylvia Snauwaert , Joan Somja , Cécile Springael , Thomas Tousseyn , Eric Van Den Neste , Sam Vandermeeren , Sam Van Hecke , Vanessa Van Hende , Marie Vercruyssen , Vibeke Vergote , Inge Vrelust , Alice Wolfromm , Ka Lung Wu.

SUMMARY

The Belgian Haematological Society (BHS) Lymphoproliferative disease (LPD) committee updated the existing recommendations on diagnosis, prognostic scores, best strategies for front-line and subsequent-line treatment of small lymphocytic lymphoma (SLL)/chronic lymphocytic leukaemia (CLL), according to robust new data.

(BELG J HEMATOL 2023;14(7):278–303)

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Rethinking the reimbursement of innovative medicines in oncology: Looking beyond overall survival

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

SUMMARY

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)

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Highlights in chronic lymphocytic leukaemia

BJH - volume 14, issue 5, september 2023

A. Janssens MD, PhD

SUMMARY

Most of the selected abstracts deal with longer-term results of clinical trials already presented previously and urgent medical needs still existing in chronic lymphocytic leukaemia (CLL).

(BELG J HEMATOL 2022;14(5):201–6)

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