Evaluation of clinical characteristics, health care resource utilization, and cost outcomes of hemophilia A carriers and noncarriers in the United States: A real-world comparative analysis.

Hemophilia A is often viewed as a male disease; females are usually considered asymptomatic hemophilia A carriers. However, hemophilia A carriers may experience mild-to-severe bleeding events. To compare clinical characteristics, health care resource utilization, and costs incurred by hemophilia A carriers compared with a non-hemophilia A carrier female control population in the United States. This retrospective observational cohort study used data from IBM MarketScan Commercial Claims and Encounters and Multi-State Medicaid Databases from January 1, 2016, to September 30, 2019. Patients with a hemophilia A carrier diagnosis were matched to a non-hemophilia A carrier female control group in a 1:2 ratio based on sociodemographic characteristics, pregnancy status, and insurance type. Billed annualized bleed rates, health care resource utilization, and annualized costs were evaluated. Generalized linear models compared annualized total costs in the hemophilia A carrier and control groups. After matching, the hemophilia A carrier group included 121 (Commercial) and 55 (Medicaid) patients, matched 1:2 in the control group. Patients in the hemophilia A carrier group (compared with the control group) had numerically higher joint-related health issues (Commercial: 11.6% vs 7.9%; Medicaid: 7.3% vs 4.5%) and lower soft-tissue disorders (Commercial: 13.2% vs 17.4%; Medicaid: 12.7% vs 14.5%). Musculoskeletal pain was higher (33.1% vs 31.0%) and lower (21.8% vs 25.5%) in the Commercial and Medicaid databases, respectively. Billed annualized bleed rates were higher in the hemophilia A carrier group (Commercial: 0.49 vs 0.33; Medicaid: 0.50 vs 0.29). Significantly more patients in the hemophilia A carrier group had minor bleeds (Commercial: 34.7% vs 22.3% [ = 0.001]; Medicaid: 43.6% vs 20.0% [ < 0.001]) and spontaneous bleeds (Commercial: 35.5% vs 21.5%; Medicaid: 47.3% vs 23.6% [ < 0.001 for both]). Outpatient visits represented the majority of health care resource utilization and were higher in the hemophilia A carrier group for all-cause and bleed-related claims; although less frequent, emergency department and inpatient visits followed a similar trend. In the Commercial and Medicaid databases, hemophilia A carriers incurred approximately 2 times higher mean (SD) all-cause health care total costs than patients in the control group (Commercial: $15,345 [21,871] vs $8,358 [11,939] per patient per year [PPPY]; Medicaid: $9,022 [19,461] vs $4,533 [9,532] PPPY). Hemophilia A carriers experienced more complications and incurred higher costs (resulting from more outpatient, emergency department, and inpatient visits) compared with patients in the control group. These data suggest that hemophilia A carriers have a high disease and economic burden and may benefit from early diagnosis and management to prevent long-term complications. Dr Xing, Dr Bullano, Dr Caicedo, and Mr Farahbakhshian are employees of Takeda Pharmaceuticals U.S.A., Inc., hold Takeda stocks, and have been granted restricted stock shares; Drs Xing and Caicedo received support from Takeda Pharmaceuticals U.S.A., Inc., for travel to THSNA 2022, where the data included in this manuscript were presented. Dr Batt received consulting fees from Complete HEOR Solutions (CHEORS) LLC for the protocol development, data analysis, and interpretation of this study; she also holds stocks from Merck and Sanofi. Ms Kuharic is an employee of the University of Illinois at Chicago and has been supported by a Takeda fellowship during the execution of the study. Ms Chakladar and Ms Markan were employees of CHEORS LLC at the time of the study. CHEORS has received funding from Takeda Pharmaceuticals U.S.A., Inc., for conducting the analysis of this study. This study was funded by Takeda Pharmaceuticals U.S.A., Inc. The sponsor was involved in the study design; collection, analysis, and interpretation of data; development and review of the manuscript; and decision to submit manuscript to publication.