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.

BACKGROUND: 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. OBJECTIVE: 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. METHODS: 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. RESULTS: 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% [P = 0.001]; Medicaid: 43.6% vs 20.0% [P < 0.001]) and spontaneous bleeds (Commercial: 35.5% vs 21.5%; Medicaid: 47.3% vs 23.6% [P < 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). CONCLUSIONS: 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. DISCLOSURES: 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.

Real-world analysis of patients with haemophilia A and haemophilia A carriers in the United States: Demographics, clinical characteristics and costs.

INTRODUCTION: Females with haemophilia A (HA [FHAs]) and HA carriers (HACs) have an increased risk of bleeding and complications compared to the general population. AIM: To examine the characteristics, billed annualised bleed rates (ABRb ), costs and healthcare resource utilisation for males with HA (MHAs), FHAs and HACs in the United States. METHODS: Data were extracted from the IBM® MarketScan® Research Databases (Commercial and Medicaid) for claims during the index period (July 2016 to September 2018) and analysed across MHAs, FHAs and HACs. RESULTS: Dual diagnosis females (DDFs; both HA and HAC claims) were grouped as a separate cohort. MHAs were generally younger than females (all cohorts) by up to 19 years (Commercial) and 23 years (Medicaid). ABRb  >0 was more frequent in females. Factor VIII claims were higher for MHAs versus female cohorts. Joint-related health issues were reported for 24.4 and 25.6% (Commercial) and 29.3 and 26.6% (Medicaid) of MHAs and FHAs, respectively; lower rates were reported in the other two cohorts. Heavy menstrual bleeding claims occurred for approximately a fifth (Commercial) to a quarter (Medicaid) of female cohorts. All-cause emergency department and inpatient visits in FHAs and DDFs were similar to, or more frequent than, those in MHAs; bleed-related inpatient visits were infrequent. In MHAs (Commercial), mean all-cause total costs ($214,083) were higher than in FHAs ($40,388), HACs ($15,647) and DDFs ($28,320) with similar trends for Medicaid patients. CONCLUSIONS: FHAs and HACs may be undermanaged and undertreated. Further research is needed to fully understand these cohorts' bleeding rates, long-term complications and costs.

Surgical and Pharmacological Treatment Patterns in Women with Endometriosis: A Descriptive Analysis of Insurance Claims.

Background: Many women with endometriosis experience chronic abdominal pain. Clinical guidelines recommend treatment with analgesics, contraceptive hormones, gonadotropin-releasing hormone analogs, and surgery. Treatment patterns in women with endometriosis are not well characterized. Methods: Data from the IBM® MarketScan® Commercial Database were accessed from 2009 to 2017. One-year baseline and follow-up periods were defined around the date of the first claim with a diagnosis of endometriosis (the index date). Women 18-49 years of age on the index date with a diagnosis of endometriosis, continuous enrollment during baseline and follow-up, and pharmacy benefits were included. The following outcomes were analyzed descriptively: baseline comorbidities; medication use and surgeries; and sequence of treatment utilization in the baseline and the follow-up period. Results: A total of 190,921 women were included. The mean ± (standard deviation) age was 39.0 ± (7.3), and abdominal/pelvic pain (36.0%) and excessive or frequent menstruation (32.0%) were the most prevalent comorbidities. In the baseline period, the utilization of pharmacological treatment was: estrogen/progestin 42.5%, opioids 41.5%, and nonsteroidal anti-inflammatory drugs (NSAIDs) 37.5%. In the follow-up period, utilization of opioids and NSAIDs increased to 68.9% and 51.1%, respectively, whereas the use of estrogen/progestin dropped to 23.8%. Surgeries were infrequent in the baseline period (6.3%). However, in the follow-up period, 27.9% of women underwent laparoscopy and 29.7% had a hysterectomy, with a total of 68.1% of the study population undergoing surgical treatment. Conclusions: A diagnosis of endometriosis is accompanied by an increase in the use of analgesics and surgical procedures. The diversity of treatments suggests a lack of clarity in management guidelines.

Treatment patterns and clinical outcomes among patients <65 years with a worsening heart failure event.

AIMS: Data regarding patients with chronic heart failure (HF) and reduced ejection fraction (HFrEF) following a worsening HF event (WHFE) are largely driven by findings from elderly patients. Younger patients are not well studied. The aim of this study was to evaluate treatment patterns and clinical outcomes in commercially insured chronic HFrEF patients <65 years old during 1-year periods before and after a WHFE. METHODS AND RESULTS: A retrospective claims analysis was performed using the IBM® MarketScan® Commercial Database on HFrEF patients aged <65 years during the year before and after a WHFE, defined as HF hospitalization or outpatient intravenous diuretic use. Treatment patterns, rehospitalizations, health care resource utilization, and costs were assessed. A total of 4460 HFrEF patients with WHFE were included. Guideline-recommended HF therapy was initially underutilized, increased pre-WHFE, and peaked 0-3 months post-WHFE. The proportions of patients using dual and triple therapy were 31.5% and 9.8% pre-WHFE, 41.5% and 17.4% 0-3 months post-WHFE, and 34.6% and 13.9% 10-12 months post-WHFE, respectively. Within 30 and 90 days after a WHFE, 12% and 23% of patients had HF-related and 16% and 30% had all-cause rehospitalizations, respectively. HF-related and all-cause hospitalizations and outpatient visits peaked 0-3 months post-WHFE, whereas emergency department visits peaked 0-3 months pre-WHFE. CONCLUSIONS: Use of HF medications increased pre-WHFE but decreased post-WHFE, despite recurrent hospitalizations. These findings suggest that age and insurance status may not totally explain the suboptimal treatment of HFrEF patients before and after a WHFE. Reasons for these trends need further study.

Clinical and Economic Burden of Chronic Heart Failure and Reduced Ejection Fraction Following a Worsening Heart Failure Event.

INTRODUCTION: A worsening heart failure event (WHFE) is defined as progressively escalating heart failure signs/symptoms requiring intravenous diuretic treatment or hospitalization. No studies have compared the burden of chronic heart failure with reduced ejection fraction (HFrEF) following a WHFE versus stable disease to inform healthcare decision makers. METHODS: A retrospective study using the IBM® MarketScan® Commercial Database included patients younger than 65 years of age with HFrEF (one inpatient or two outpatient claims of systolic HF or one outpatient claim of systolic HF plus one outpatient claim of any HF). The first claim for HFrEF during 2016 was the index date. Patients were followed for the first 12 months after the index date (the worsening assessment period) to identify a WHFE, and for an additional 12 months or until the end of continuous enrollment (the post-worsening assessment period). Mean per patient per month (PPPM) health care resource use (HCRU) and costs were compared between patients following a WHFE and stable patients during the two periods using generalized linear models adjusting for patient characteristics. RESULTS: Of 16,646 patients with chronic HFrEF, 26.8% developed a WHFE. Adjusted all-cause hospitalizations (0.16 vs. 0.02 PPPM, P < 0.0001), outpatient visits (3.54 vs. 2.73 PPPM, P < 0.0001), and emergency department visits (0.25 vs. 0.06 PPPM, P < 0.0001) were higher in patients following a WHFE than stable patients during the worsening assessment period. Similar differences in HCRU were observed between the two cohorts during the post-worsening assessment period. Mean total adjusted cost of care PPPM was $8657 in patients with HFrEF following a WHFE versus $2195 in stable patients during the worsening assessment period, and $6809 versus $2849, respectively, during the post-worsening assessment period. CONCLUSION: HCRU and costs were significantly greater in patients with chronic HFrEF following a WHFE compared to those who remained stable, suggesting an unmet need to improve clinical and economic outcomes among these patients.