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 surveys prophylaxis options for hemophilia A without inhibitors, describing standard- and extended-half-life factor VIII concentrates, the nonfactor agent emicizumab-kywh, and emerging gene therapy, and concluding that standard-half-life FVIII prophylaxis remains the standard of care while real-world experience is still needed to define the newer agents' roles. Its relevance to ASH is as a counterpoint within hemostasis science: hemophilia is the disorder of too little clotting, the mirror image of the pro-thrombotic conditions that leech-secretome anticoagulants target, and the review usefully illustrates how the coagulation cascade is modulated therapeutically from both directions. Honest caveat: this paper concerns boosting clotting factors in a bleeding disorder and has no bearing on hirudotherapy or leech-derived compounds; it is a review (no primary data), and ASH includes it only to round out the coagulation-biology context, not as evidence for leech therapy.
Citation
Prophylaxis for hemophilia A without inhibitors: treatment options and considerations.
Valentino LA et al. · Expert review of hematology, 2020
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