Episodic treatment for hemophilia A may be associated with higher risk of bleeding compared with prophylactic treatment, according to research published in Haemophilia.
This multicenter, prospective, noninterventional study was the first to assess bleeding and safety outcomes in patients with hemophilia A and no prior history of inhibitors in a real-world setting. Researchers enrolled 94 patients with a median age of 34.0 years, with 45 patients receiving episodic factor VIII (FVIII) treatment and 49 patients receiving prophylactic treatment.
The primary endpoint was the annualized bleeding rate (ABR), which was calculated using number of treated bleeds over time and multiplying that rate by 365.25 days. Number, type, location, and cause of all treated and untreated bleeds were also measured, as were adverse events.
Patients receiving episodic treatment experienced 1066 bleeds overall, 872 of which were treated. In this group, ABR for treated bleeds and all bleeds was 36.1 and 43.1, respectively. All patients had at least 1 bleed and individual ABR of greater than 10.
Patients receiving prophylactic treatment experienced 189 bleeds overall, 151 of which were treated. At least 1 bleed was reported by 63.3% of patients. In this group, ABR for treated bleeds was 5.0, and ABR for all bleeds was 6.2. Individual ABR was greater than 10 for 22.4% of patients.
There were 51 adverse events reported by 33 patients, with the most common being viral upper respiratory tract infection. Protocol adherence for at least 80% of study weeks was observed in 66.7% of patients; overall, patients adhered to prophylaxis for a median of 86% of study weeks.
The authors noted that their findings were comparable to data from other studies showing that prophylactic treatment of hemophilia may lead to satisfactory bleed control in patients with hemophilia A without inhibitors. However, patients still experienced “a considerable rate of breakthrough bleeding,” thus revealing “an unmet need for more effective therapies that are easier to adhere to.”
Disclosures: This study was funded by F. Hoffman-La Roche Ltd. For a full list of author disclosures, please refer to the original study.
1. Kruse-Jarres R, Oldenburg J, Santagostino E, et al. Bleeding and safety outcomes in persons with haemophilia A without inhibitors: results from a prospective non-interventional study in a real-world setting [published online February 6, 2019]. Haemophilia. doi: 10.1111/hae.13655