T. Feys MBA, MSc

New advances in the treatment of haemophilia B

Summary

Haemophilia B is a genetic condition that is caused by a shortage in clotting factor IX (FIX). The severity of the haemophilia is determined by the clotting activity of FIX in the blood. The standard of care for patients with haemophilia B consists of substitution therapy with FIX. In this procedure, FIX, either prepared from human plasma or a recombinant form, is administered through an intravenous (IV) infusion with a frequency that depends on the severity of the haemophilia. These infusions are given once or twice a week and lay a heavy burden on patients. In addition to this, the presence of inhibitory antibodies in haemophilia patients can hamper the treatment. In these cases, FIX is no longer active, or is sometimes even eradicated. More recently, important advances have been made in the development of gene therapy for haemophilia B patients. With this technique, a non-mutated FIX gene is introduced into the DNA of patients using a viral vector that targets the hepatocytes of patients. As such, the missing clotting factor is reintroduced in patients allowing them to produce sufficient amounts of the clotting factor. During the 2016 annual EHA meeting, several haemophilia studies addressed this technique.

(BELG J HEMATOL 2016;7(4):174–5)

New haematology reimbursements in Belgium

Summary

Overview of Belgian reimbursement news

(BELG J HEMATOL 2016;7(2):93)

Highlights in chronic myeloid leukemia and myeloproliferative neoplasms

Summary

Major therapeutic progress has been accomplished in chronic myeloid leukemia (CML) and myeloproliferative neoplasms (MPN) over the past 40 years. Therapeutic progress happened through better understanding of disease pathophysiologiy and rational development of targeted agents, like imatinib mesylate in CML. This report will discuss the key studies related to CML or MDS, presented during the 2015 annual ASH meeting of the American Society of Hematology (ASH).

(BELG J HEMATOL 2016; 7(1):9–13)

Novelties in the treatment of sickle cell disease

Summary

At the 2015 annual meeting of the American Society of Hemtology (ASH), potentially practice-changing novelties were published, which reform our way of thinking about sickle cell disease. Three highlighted studies were mentioned, two of these present promising new insights for managing the disease in children, the third demonstrates a new oral therapy.

(BELG J HEMATOL 2016; 7(1): 53–4)

Clinical highlights in hemophilia

(BELG J HEMATOL; 7(1):55–8)

New hematology reimbursements in Belgium

Summary

Overview of Belgian reimbursement news

(BELG J HEMATOL 2016; 7(1):59)

New haematology reimbursements in Belgium

Overview of Belgian reimbursement news

(Belg J Hematol 2015;6(5):225)