C. Hermans MD, PhD

Highlights in haemostasis and thrombosis

SUMMARY

The 2024 ASH Congress once again highlighted ground-breaking advancements in the field of haemostasis and thrombosis, with four studies standing out for their clinical and scientific significance. First, a study demonstrated that low-dose direct oral anticoagulants (DOACs) are not inferior to standard doses for long-term anticoagulation in patients with venous thromboembolism (VTED), offering a promising alternative therapeutic approach. Furthermore, VMX-C001 shows potential as a significant advancement in managing emergencies in patients on factor Xa inhibitors (FXa-DOACs), enabling rapid and effective anticoagulation reversal; however, its efficacy and safety still require validation in phase III clinical trials. Additionally, gene therapy for haemophilia A using giroctocogene fitelparvovec achieved sustained factor VIII expression, leading to a substantial reduction in bleeding episodes among adults with moderately severe to severe haemophilia A. Lastly, autologous transplantation of CD34+ hematopoietic stem cells transduced with a lentiviral vector carrying a novel F8 transgene (ET3) resulted in stable expression of factor VIII for up to two years, representing a transformative breakthrough in the treatment of haemophilia A. Together, these findings underscore the remarkable progress achieved this year in addressing these critical medical challenges.

(BELG J HEMATOL 2025;16(1):34–40)

Highlights in thrombosis and Haemostasis

SUMMARY

New therapeutic developments in the field of haemophilia were highlighted at the ASH 2021 congress. These included the results of inhibition of antithrombin production via fitusiran for the prevention of bleeding events in haemophilia A and B patients with or without inhibitors, the success of emicizumab, a bispecific antibody that mimics the action of FVIII, in patients with moderate or mild haemophilia A, and finally the promising results of FVIII fused to the Fc fragment of the immunoglobulins as an agent for FVIII tolerance induction in patients with inhibitors. In the field of antithrombotics, a meta-analysis confirms the place of direct oral anticoagulants in cancer patients with thrombosis. Xarelto has demonstrated its value in patients with visceral venous thrombosis and a large study does not support the thrombogenicity of mRNA-based COVID vaccines.

(BELG J HEMATOL 2022;13(1):37-42)

Effectiveness of a recombinant coagulation Factor IX–albumin fusion protein, rIX-FP, in haemophilia B patients: Results of a non-interventional study in Belgium

SUMMARY

The aim of this non-interventional, observational study conducted in haemophilia B patients in Belgium was to collect real-world data to confirm the efficacy and safety of rIX-FP established in pivotal clinical trials.

(BELG J HEMATOL 2021;12(5):203-6)

Ibrutinib and atrial fibrillation: A Belgian expert consensus paper

SUMMARY

Over the last decade, the oral Bruton’s tyrosine kinase (BTK) inhibitor ibrutinib induced a paradigm shift in the treatment of patients with chronic lymphocytic leukaemia (CLL), mantle cell lymphoma (MCL), and Waldenströms macroglobulinemia (WM). In clinical trials and in real-world studies, ibrutinib proved to be an effective agent with an overall favourable safety and tolerability profile. However, compared with standard chemo-immunotherapy (CIT), ibrutinib was associated with a higher incidence of atrial fibrillation (AF). The patho-physiological mechanisms underlying this increased AF incidence are not completely understood, but it is thought to be related to off-target inhibitory effects of ibrutinib on the Tec protein tyrosine kinase (TEC) in cardiac cells. The prevalence of AF in patients treated with ibrutinib is highest during the first three months of therapy, which warrants an increased vigilance during this treatment phase. However, AF in patients treated with ibrutinib is generally well manageable without ibrutinib discontinuation. Prior to the start of ibrutinib treatment, identification and addressing modifiable risk factors for AF is a first important step. The threshold for haematologists to consult a cardiologist or a cardio-oncologist should be low and a close collaboration between both specialties is warranted. Unnecessary ibrutinib interruptions should be avoided, and uncomplicated AF is not a valid reason to discontinue or interrupt ibrutinib. If anticoagulation is required, direct oral anticoagulants are preferred. In this paper, a panel of haematology and cardiology specialists have provided practical guidance on how to evaluate patients prior to ibrutinib treatment and monitor during ibrutinib therapy. Furthermore, they have provided practical guidance on how to manage AF in ibrutinib-treated patients.

(BELG J HEMATOL 2021;12(4):155-64)

Ibrutinib and bleeding management: a Belgian expert consensus

SUMMARY

In recent years ibrutinib emerged as a paradigm shifting agent in the treatment of chronic lymphocytic leukaemia (CLL), mantle cell lymphoma (MCL) and Waldenström’s macroglobulinemia (WM). In clinical trials and in real-world studies ibrutinib proved to be an effective agent with an overall favourable tolerability profile. However, compared with standard chemo-immunotherapy (CIT), ibrutinib was associated with a higher incidence of clinically significant bleeding. This has been hypothesized to be linked to the platelet-specific effects of inhibiting Bruton’s tyrosine kinase (BTK). Most bleeding events under ibrutinib are low-grade with a decreasing incidence over time. However, bleeding can have a significant impact on patients and interfere with persistence and compliance of ibrutinib treatment. Currently, no clear consensus exists on the use of ibrutinib in patients with an increased bleeding risk, on the management of ibrutinib-induced bleeding and on the use of ibrutinib around surgery or invasive procedures. In this paper, a panel of Belgian haematology and haemostasis specialists formulated practical advice on bleeding prevention and management in ibrutinib-treated patients.

(BELG J HEMATOL 2020;11(4):174–84)

O.5 Confirmation of the very low rate of subclinical venous thrombosis in people with hemophilia undergoing major orthopedic surgery in the absence of pharmacological thromboprophylaxis

A-138 DEVELOPMENT OF APPROPRIATE AND CULTURALLY ADAPTED EDUCATIONAL TOOLS ON HAEMOPHILIA FOR THE PATIENTS FROM IVORY COAST AND THEIR FAMILIES