Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B

Abstract

This guideline was compiled according to the British Society for Haematology (BSH) process at https://b-s-h.org.uk/med ia/16732/bsh-guidance-development-process-dec-5-18.pdf. The Grading of Recommendations Assessment, Development and Evaluation (GRADE) nomenclature was used to evaluate levels of evidence and to assess the strength of recommendations. The GRADE criteria can be found at http:// www.gradeworkinggroup.org. The references to support the recommendations are listed in the preceding discussion. Literature review The information contained in this review was gathered from several sources. These included references from a search of MEDLINE, EMBASE and the Cochrane databases to identify studies and reviews relevant to prophylaxis in patients with haemophilia, abstracts from international meetings and references known to the authors (Appendix S1). Review of the manuscript The writing group produced the draft guideline, which was subsequently revised by consensus. Review of the manuscript was performed by the BSH Guidelines Committee Haemostasis and Thrombosis Taskforce, the BSH Guidelines Committee and the Haemostasis and Thrombosis sounding board of the BSH. It was also on the members section of the BSH website for comment. It has also been reviewed by members of the United Kingdom Haemophilia Centre Doctors' Organisation (UKHCDO) Advisory Board, Haemophilia Nurses Association (HNA), Haemophilia Chartered Physiotherapists Association (HCPA); these organisations do not necessarily approve or endorse the contents.

Clinical phenotype of severe and moderate haemophilia: Who should receive prophylaxis and what is the target trough level?

“…Prophylaxis is recommended to continue life-long. [1][2][3][4] In older people with haemophilia (PwH), secondary or tertiary prophylaxis is used to reduce bleeding episodes, limit progression of arthropathy and improve mobility. [5][6][7] Long-term studies support the efficacy of primary prophylaxis for preventing arthropathy in the large majority of children with severe haemophilia.…”

Emicizumab (Hemlibra®) in hemophilia A patients with inhibitors against factor VIII — guidelines of the Group for Haemostasis of the Polish Society of Haematology and Transfusion Medicine

“…It is worth mentioning that one severe haemophilia A patient with inhibitor, who received recombinant activated factor VII (rFVIIa) during emicizumab prophylaxis, experienced myocardial infarction without ST-segment elevation and pulmonary embolism [25]. Up to date however, no thromboembolic complications or TMA cases have been reported for hemophilia A patients with inhibitor during concomitant administration of emicizumab and factor VIII concentrates [26,27]. Subcutaneous Hemlibra ® injections are well tolerated, although 22% of study patients reported mild or moderate skin irritation at injection site [2,5].…”

Resolution of the Expert сouncil on сurrent issues in the treatment of patients with hemophilia A with clotting factor VIII with prolonged half-life (INN – Efmoroctocog alfa)

“…Отдельно был рассмотрен вопрос о национальных клинических рекомендациях по лечению гемофилии в различных странах. Так, в рекомендациях Великобритании указаны критерии препарата с пролонгированным периодом полувыведения: увеличение периода полувыведения в 1,3 раза и более и снижение частоты инъекций по сравнению со стандартными факторами VIII [8]. В рекомендациях Всемирной федерации гемофилии показаниями к назначению препаратов с пролонгированным периодом полувыведения являются: неэффективность профилактики препаратами со стандартным периодом полувыведения (сохранение спонтанных кровотечений  1) и выявление поражения суставов у молодых пациентов.…”