Initiating Prophylaxis at Younger Age May Preserve Joint Status in Severe Hemophilia A

Starting tailored prophylaxis before age 2 in patients with severe hemophilia A may help reduce joint involvement, according to a study published online in Research and Practice in Thrombosis and Haemostasis.

“The advent of new nonsubstitutive therapies, such as emicizumab, is undoubtedly transforming the current and future landscape of prophylaxis,” researchers explained in the study background.  “Given the proposed definition of prophylaxis recently published in the World Federation of Hemophilia guidelines, which prioritizes outcomes over product regimens, there is a need for reliable, homogeneous data on the achievements of classical primary prophylaxis regimens with factor VIII and their long-term outcomes to establish the goals for treating these patients.”

The retrospective analysis included 60 patients with severe hemophilia A who did not develop early inhibitors at the start of primary prophylaxis. Participants were consecutively diagnosed between January 2000 and December 2019 and were under follow-up at La Paz University Hospital in Madrid, Spain.

Over a median 10 years of follow-up after starting prophylaxis, 76.7% of patients had no joint involvement. Researchers defined joint involvement as Hemophilia Joint Health Score or Hemophilia Early Arthropathy Detection with an ultrasound score of 1 or higher.

According to the study, patients with no joint involvement initiated prophylaxis at a median age of 1 year compared with 3 years for the 23.3% of patients with joint involvement at the end of follow-up. Patients without joint involvement also had a lower annual rate of joint bleeds, were more likely to be physically active, and had lower factor VIII trough levels.

“While we await the results of long-term prophylaxis using the new nonreplacement therapies,” researchers wrote, “our data illustrate the importance of starting treatment early and serve as a basis for establishing a solid long-term comparison of the specific variables that influence the treatment of these patients.”

Reference:

Meijón Ortigueira MDM, Álvarez-Román MT, De La Corte Rodríguez H, Butta Coll N, Jiménez-Yuste V. Long-term impact of primary prophylaxis on joint status in patients with severe hemophilia A. Res Pract Thromb Haemost. 2023;7(1):100005. doi:10.1016/j.rpth.2022.100005