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.

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.

Incremental healthcare resource utilization and costs for patients with cervical, vaginal, vulvar, anal, and oropharyngeal cancer in the United States.

INTRODUCTION: Human papillomavirus (HPV) cause cancers in a variety of anatomic sites presenting at various stages of disease. Current economic assessments rely on HPV-related cancer cost estimates from data prior to the launch of the nonavalent HPV vaccine (2014). The goal of the present study was to assess and describe the current direct medical care burden of HPV-related cancers in the US. METHODS: Using Clinformatics Data Mart, patients in the US who were newly diagnosed with cervical, vulvar, vaginal, anal, and oropharyngeal cancers between 2012 and 2015 were compared to non-cancer matched (propensity score) controls. Health care resource utilization and direct medical cost (2020 USD) were assessed over a 2-year follow-up period following index diagnosis from a payer perspective. The cost for censored time was estimated using generalized linear model while adjusting for survival probability using cox-proportional hazard model. Confidence intervals were calculated with bootstrapping technique. RESULTS: The analyses included 4128 cervical, 1580 vulvar, 538 vaginal, 1827 anal, and 6106 oropharyngeal cancers and matched controls. Cases and controls had similar baseline clinical characteristics and length of follow-up. The 2-year incremental direct medical costs were $93,272, $81,676, $141,096, $129,366, and $134,045 for cervical, vulvar, vaginal, anal, and oropharyngeal cancers respectively. Outpatient care costs was the biggest driver of the total incremental medical costs. Most cancer costs were incurred during the first 6 months of follow-up and then stabilized during follow-up. CONCLUSION: HPV-related cancers are responsible for substantial health care expenditure each year.

Level of glycemic control among US type 2 diabetes mellitus patients on dual therapy of metformin and sodium-glucose cotransporter 2 inhibitor: a retrospective database study.

OBJECTIVE: To assess the level of glycemic control among type 2 diabetes patients on sodium-glucose cotransporter 2 inhibitor (SGLT2i) and metformin dual therapy. METHODS: Observational, retrospective database study in adult type 2 diabetes mellitus patients from the IQVIA Electronic Medical Record (EMR) database was conducted. The observation period was June 2015 to June 2018. Patient's earliest encounter in the observation period while on SGLT2i and metformin dual therapy served as the index date. Patients were required to have at least one HbA1c measure in the 12 months prior to the index date and be on SGLT2i and metformin dual therapy and no other antihyperglycemic treatment as of the HbA1c measurement date or any time during the 90 days prior. The associations between sociodemographic factors and clinical burden on achievement of HbA1c <8% were assessed using multivariable logistic regression with backward stepwise selection. RESULTS: Of 3491 patients, 2176 (62.3%) achieved HbA1c <8%, with a median distance to goal of 1.1% (IQR 0.5-2.3%) for those not at glycemic target. Mean age was 56.5 years and 52.6% were male. At baseline, 28.3% of patients had established cardiovascular disease/chronic kidney disease, and of those 63.8% had HbA1c <8%. African American patients had lower odds of attaining HbA1c <8% when compared with white patients [OR 0.69], while older patients had marginally higher odds [OR 1.01]. CONCLUSION: Approximately 3 out of 5 patients on metformin and SGLT2i dual therapy achieved HbA1c <8%, with African Americans having a lower likelihood of achieving this glycemic goal.