Assessing Reasons for Treatment and Treatment Type in Females with Bleeding Disorders

A majority of female patients with inherited bleeding disorders, including von Willebrand Disease (VWD), hemophilia A, and hemophilia B, experienced bleeding symptoms that may call for the initiation of prophylaxis with factor and non-factor replacement therapy, particularly with more than one or two bleeding events per year, according to a retrospective single-center database analysis.

Additionally, during a presentation at the 65th American Society of Hematology Annual Meeting & Exhibition in San Diego, California, lead author, Daniel Barnett, MD, from the Banner University Medical Center in Phoenix, Arizona, suggested that “heavy menstrual bleeding, joint bleeding, joint pain, [and] gastrointestinal and genitourinary bleeding are reported in this patient population as reasons to start prophylaxis with hemostatic agents.”

The analysis reviewed 101 female patients from the Banner MD Anderson Cancer bleeding disorder database, of whom 64 had a diagnosis of hemophilia A, hemophilia B, or VWD. Researchers reviewed the type of treatment, reason for treatment initiation, and type of administration.

Overall, 13 of 17 (76%) patients with hemophilia A were receiving prophylaxis with factor replacement or emicizumab. The most common reasons for initiating prophylaxis were joint pain and bleeding in six patients and menorrhagia in three patients. Among the four patients with hemophilia B, two (50%) were receiving factor replacement, with rectal bleeding and menorrhagia recorded as reasons for starting prophylaxis.

In the VWD group, 24 of 43 (56%) patients were receiving prophylaxis with one or more of the following: factor replacement, desmopressin, or antifibrinolytic replacement. The most common reason for initiating treatment was menorrhagia in 13 patients followed by joint bleeding in four patients. Additional reasons including trauma, mucocutaneous, urinary tract, and pregnancy bleeding.

Notably, patients with VWD were much more likely to require central intravenous treatment administration via a chest portacath (n=14; 33%) and were more likely to have received their diagnosis prior to age 18 years (n=16; 37%).

In closing, Dr. Barnett noted, “Once factor replacement is started, patients are pleased with the change in their health status after initiation of treatment and continue to be compliant with treatment recommendations although they may have difficulty in administering those treatments.”

Reference

Barnett D, Nance D. a retrospective analysis and characterization of female patients with bleeding disorders hemophilia B, hemophilia B and von Willebrand’s disease and their treatment type at Banner MD Anderson Cancer Center. Abstract #1254. Presented at the 65th ASH Annual Meeting & Exposition; December 9-12, 2023; San Diego, California.