A large US insurance claims database suggests a delay in diagnosis and therapeutic intervention in Women and Girls with Hemophilia (WGH)

The median age of WGH diagnosis and factor therapy initiation was 35 and 37 years old

NHF 2017 (Chicago, IL August 24-26):Hemophilia A is an X-linked, bleeding disorder that results from deficiency in coagulation factor VIII (FVIII). While inheritance of the disease is more common in males, women and girls commonly present as carriers and may be asymptomatic or symptomatic. Some women may have sufficiently low levels (<40%) to meet the diagnosis of hemophilia. Although men and symptomatic women may have similar symptoms, such as bleeds into joints and tissues, women also experience complications during menstruation, pregnancy, labor and delivery. Healthcare providers may not be as familiar with bleeding disorders in women, leading to inadequate diagnosis and management.

In the poster,   Characterization of Women and Girls with Hemophilia Treated in the US from A Claims Database, presented at the National Hemophilia Foundation (NHF) annual meeting researchers aimed to provide real-world evidence on women and girls with hemophilia (WGH) treated in the US. Between January 2011 and November 2016, WGH were identified from a large US commercial insurance claims database.   Eligible patients were women or girls who either had hemophilia A diagnosis (International Classification of Disease or ICD version 9 code 286.0 or newer version 10 code D66) or received factor VIII treatment, including plasma-derived, conventional, and extended half-life (EHL) recombinant factors. WGH with coded Von Willebrand disease (VWD) diagnosis were excluded.

The earliest date of factor use was defined as the index date, and patients were followed till the end of continuous enrollment or end of data, whichever happened earlier. Patient characteristics such as age, region, and insurance type and treatment patterns were obtained. An assessment based on proportion of days covered by factor treatment (PDC) during the follow-up period was used to classify factor use as routine prophylaxis (PDC of 70% or more) or on-demand treatment of bleeds (PDC less than 70%)

A total of 228 female patients used factor VIII treatment out of 557,391 patients with any coagulation disorders in the database. Forty-four of these were diagnosed with WGH.  The median age of WGH diagnosis and factor therapy initiation was 35 and 37 years old, respectively; the majority (more than 70%) of patients initiated factor therapy in adulthood. All WGH treated were covered by managed care plans. About 1 in 10 (9%)  used plasma-derived FVIII, over 8 in 10 (84%) used standard  recombinant FVIII, and about 1 in 15 (7%)  used EHL recombinant FVIII. Based on the assessment on proportion of days covered by factor treatment, the majority (4 of 5 ~80%) of WGH used factor therapy as on-demand treatment, and only 1 in 5 (20%) of them used it as prophylaxis.

Using this large US insurance claims database, more than half of the WGH patients received factor therapy in their mid-thirties, suggesting a delay in diagnosis and therapeutic intervention. The data support the unmet need that currently exists in management of this affected population, including appropriate and timely diagnosis aided by recognition of bleeding symptoms and improved dialogue with healthcare professionals.