Changing Trends in Hemophilia Treatment

The management of hemophilia, a rare bleeding disorder, relies heavily on the expertise of healthcare specialists. However, specific dosing regimens for treatments in this field have not been extensively studied. A recent study conducted in the United States (US) described the evolving trends in clinician prescribing practices for hemophilia management.

The study aimed to assess the prescribing practices of clinicians who specialize in the treatment of hemophilia, with a focus on factor dosing, inhibitor therapy, and gene therapy.

“With other novel therapies (including gene therapy) entering the US market, it is important to document the variability in existing prescribing practices among broad-based cohorts of clinicians,” explain study authors.

The research methodology involved administering surveys to members of the Hemostasis and Thrombosis Research Society. Surveys were conducted at the society's in-person annual symposia in 1999 and 2015, and an online survey was conducted in 2021. These surveys were designed to collect data on the treatment approaches used for hemophilia patients.

The results of the study revealed several significant trends in the prescribing practices of clinicians over the span of 22 years. The percentage of clinicians who dedicated over 50% of their practice to treating hemophilia patients increased from 37.5% in 1999 to 46.3% in 2021, indicating a growing specialization in this area of medicine.

One notable trend was the preference for higher doses of factor concentrates for routine bleeding events. In 1999, none of the clinicians prescribed more than 40 units per kilogram for such events in hemophilia A patients. However, in 2021, this percentage had risen to 29.3%. In the case of hemophilia B, clinicians prescribing factor concentrates at over 40 units per kilogram increased from 22.5% in 1999 to a substantial 87.8% in 2021. This shift in dosing practices may reflect advancements in treatment and a desire to improve patient outcomes.

Another significant change was the increasing use of emicizumab, a medication used to treat hemophilia A patients, both pediatric and adult, with or without inhibitors. In 2021, more than 89.5% of pediatric patients and 85.7% of adult patients received emicizumab at least some of the time. This suggests that emicizumab is becoming a preferred prophylactic treatment for hemophilia A patients.

Additionally, clinicians expressed optimism about the potential of gene therapy as a valuable treatment option for hemophilia patients. About 78.0% of respondents reported that they expected to recommend gene therapy to their patients at least some of the time. This indicates the evolving landscape of treatment options for hemophilia, with clinicians embracing innovative approaches to improve the quality of care.

In conclusion, the study highlights notable changes in prescribing practices among hemophilia specialists in the United States over the past two decades. These changes include an increased preference for higher doses of factor concentrates, the growing use of emicizumab, and a positive outlook on gene therapy as a future treatment option. These evolving trends reflect the dynamic nature of hemophilia care and the ongoing efforts to improve the management of this rare bleeding disorder.

“These data from a sample of US haemophilia clinicians provide a longitudinal view of changes in prescribing practices during a 22-year time period. Dosing practices have trended upward, particularly for HB, and especially for patients who experience major life-threatening bleeds.”

“Further studies should compare clinician prescribing with patient use patterns for these new treatment options, with particular attention given to outcome evaluation,” concluded authors.

Reference:

Curtis R, Roberts JC, Crook N, et al. Trends in prescribing practices for management of haemophilia: 1999-2021. Haemophilia. 2023;29(3):761-769. doi:10.1111/hae.14769