A new article accepted for publication! Lambert et al. 2022

06/02/2023

Congratulations to Pr. Catherine Lambert and her colleagues for the publication of their article on hemophilia treatment in low-income countries by the Research and Practice in Thrombosis and Haemostasis journal. We are glad to have been able to participate to the success of this article!

Hemophilia is a genetic disorder in which the blood cannot clot. There are two types of hemophilia: hemophilia A, characterized by a deficiency of coagulation factor VIII, and hemophilia B, which is related to a deficiency of coagulation factor IX. Treatment options have greatly improved during the last decades. However, even though they are available in developed countries, they could easily be implemented in low-income countries, where patients are either not or inadequately treated with clotting factor concentrates.

Data on implementation and prophylaxis results with emicizumab administered subcutaneously, a bispecific monoclonal antibody mimicking the factor VIII activity, have been prospectively reported. The sample was composed of 33 boys (aged 2 to 13 years old) suffering from a severe hemophilia A and living in Ivory Coast. A 99% reduction in bleeding rates was observed after 12 months after treatment initiation. Patient’s quality of life significantly improved and the treatment was positively rated by the children and their parents. Acceptance, tolerance, and adherence to emicizumab were excellent.

The results from this article show that new non-replacement therapies could be implemented in low-income countries and improve the clinical management of patients with hemophilia living in these countries.

The pre-proof of the article is already available on the journal’s website! https://www.sciencedirect.com/science/article/pii/S2475037922022865