Prophylaxis for hemophilia A without inhibitors: treatment options and considerations.
Review published in Expert review of hematology (2020)
Abstract
INTRODUCTION: Hemophilia A is a bleeding disorder traditionally managed with standard half-life (SHL) factor (F) VIII concentrates. Extended half-life (EHL) FVIII products and emicizumab-kywh, a nonfactor therapy, are newer treatment options. Additional nonfactor agents and gene therapy are expected to reach the market in the near future. AREAS COVERED: A PubMed (MEDLINE) search from 1962 to April 2020 related to hemophilia A, its management, and the products currently available for prophylaxis was performed to comprehensively review these topics and analyze the benefits and drawbacks of each therapeutic. EXPERT OPINION: Prophylaxis with SHL FVIII concentrates remains the standard of care for patients with severe hemophilia A and may also be considered for selected individuals with moderate disease. Several years of real-world experience with EHL FVIII, emicizumab-kywh, and other agents in development will be necessary to determine their ultimate roles in the prevention of bleeding and its complications. Gene therapy may not provide a permanent cure for hemophilia A.
Abstract sourced from PubMed (NCBI) for the cited record. See the original publication for the authoritative version.
Summary
Hemophilia A is a bleeding disorder traditionally managed with standard half-life (SHL) factor (F) VIII concentrates. Extended half-life (EHL) FVIII products and emicizumab-kywh, a nonfactor therapy, are newer treatment options. Additional nonfactor agents and gene therapy are expected to reach the market in the near future.
Why This Matters for Hirudotherapy
This review examined prophylactic treatment options for hemophilia A, covering standard half-life and extended half-life factor VIII concentrates, emicizumab-kywh, and emerging nonfactor agents and gene therapy, analyzing the benefits and drawbacks of each. For the ASH and hirudotherapy community, the article provides relevant background on bleeding disorder management, a field conceptually adjacent to the anticoagulation mechanisms central to leech therapy. Understanding hemophilia prophylaxis informs broader knowledge of hemostatic balance, which is relevant context when considering the clinical use of leech-derived anticoagulants such as hirudin. However, the review does not discuss leech-derived therapeutics or anticoagulants directly; its focus is exclusively on factor replacement and nonfactor therapies for hemophilia A, so the connection to hirudotherapy is indirect.
Citation
Prophylaxis for hemophilia A without inhibitors: treatment options and considerations.
Valentino LA et al. · Expert review of hematology, 2020
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