An expert panel failed to reach consensus on how to treat some patients with severe hemophilia A, suggesting that further study is needed where the optimal treatment remains equivocal, according to research published in the Journal of Blood Medicine.
Hemophilia A is linked with a reduced quality of life (QoL) because of the risk of severe bleeding. While factor VIII (FVIII) replacement treatment has been repeatedly shown to improve symptoms in this patient population, about 30% of patients develop inhibitors, which have a significant and negative impact on patient QoL.
The high rate of inhibitor development has spurred the creation of novel treatments that aim to improve QoL among patients with hemophilia A. While these new options create a greater possibility of targeted therapy for specific patients, there is often insufficient evidence for choosing 1 regimen over another.
The ability to target treatment is, however, impaired by particular patient qualities as determined in the real-world setting. There is, furthermore, a lack of the robust treatment guidelines necessary for successfully developing targeted treatment plans. For this study, researchers used a Delphi method to develop consensus in areas of difficult-to-treat hemophilia A care where there is no gold standard treatment recommendation.
The study used a 3-round Delphi method; 13 experts in the United States were consulted. The majority of experts were male sex (69.2%) and White (76.9%); more than half (53.8%) had been caring for patients with hemophilia A for 10 to 19 years.
Of 25 scenarios about which experts were asked, consensus was not reached in 11 cases. The cases where no consensus was reached included the use of direct oral anticoagulants among patients with hemophilia A and stroke and the correct treatment among patients who expressed an interest in increased physical activity.
Among challenging cases, experts tended to support the use of FVIII therapy over emicizumab.
“A majority of panelists preferred FVIII replacement therapy rather than emicizumab prophylaxis due to established evidence on safety, efficacy, and level of bleed protection for FVIII treatment,” the authors wrote. “Further studies are needed to identify appropriate therapeutic approaches in those clinical cases for which consensus was not reached.”
Disclosure: The study author(s) declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Veeranki SP, Pednekar P, Graf M, Tuly R, Recht M, Batt K. A Delphi consensus approach for difficult-to-treat patients with severe hemophilia A without inhibitors. J Blood Med. 2021;12:913-928. doi:10.2147/JBM.S334852