Introduction

Hemophilia imposes significant clinical and societal burdens. Its management requires frequent medication administration, physician visits, hospitalizations, medical and surgical procedures, and laboratory assessments. Healthcare resource utilization (HCRU) may be particularly relevant for people with hemophilia (PwH) (moderate to severe), as prophylactic and the management of bleeding episodes, which may require hospitalization. Various factors - patient demographics, geographical region, treatment regimens, and a healthcare provider involvement can impact HCRU. This analysis describes the hemophilia treatment landscape and the HCRU burden in PwH.

Methods

Real-world data were drawn from Adelphi Hemophilia Wave III Disease Specific Programme™ database (July 2023 to March 2024) in US, France, Germany, Italy, Spain, UK, China, and Japan. Data sources were physician surveys, physician-completed patient record forms, and patients' and caregivers' self-completion forms. The study analyzed descriptively male patients ≥12 years old with moderate or severe hemophilia A (HA) and B (HB), with or without inhibitors.

Results

The study included 1,418 patients, with a mean (SD) age of 30.7 (13.9) years, mostly Caucasian (84%), with 80% HA and 20% HB. Most patients (94%) had no current inhibitors; 6% had current inhibitors. At the time of survey, 13% HA Non-factor Therapy (NFT) patients had inhibitors. Physicians perceived 35% of their patients as moderate, 23% moderately severe, and 42% severe.

Overall, 82% of patients were on prophylactic therapy. HA patients' current treatments were Standard Half-Life (SHL) (43%), Extended Half-Life (EHL) (26%), NFT (25%), and bypassing agents (BPA) (1%); 56% HB patients were prescribed EHL, 34% SHL, and 1% BPA.

Physicians reported that 71.8% of patients were diagnosed by a hematologist, and 16.9% were diagnosed by a hematologist-oncologist, including their pediatric equivalents.

In the 12 months prior to survey, patients on prophylactic therapy used a mean (SD) of 18.8 (15.3) tests (EHL 17.9 [14.0], SHL 18.9 [15.6], and NFT 18.2 [15.1]). Physicians reported that patients in US had a mean (SD) of 16.1 (20.0) tests and Spain had 25.1 (16.0) tests.

Common diagnostic and monitoring tests were complete blood count (86% and 83%), activated partial thromboplastin time (aPTT) (82% and 76%), prothrombin time (PT) (59% and 50%), and 1-stage assay (59% and 43%). Physicians reported using PT and aPTT to monitor 63% and 92% of patients in Japan, while in US, this was 53% and 36% of patients. Physicians reported a proportion of their adolescent patients were monitored using PT (57%) or aPTT (84%). For adult patients, 49% were monitored using PT and 75% aPTT.

In the 12 months prior to survey, proportion of patients hospitalized due to hemophilia was 9% HA NFT, 6% HA EHL, 10% HA SHL, 3% HB EHL, and 17% HB SHL. Mean (SD) hospitalizations for HA patients were 1.6 (1.2) (EHL), 1.8 (1.2) (SHL), and 1.3 (1.0) (NFT); for HB patients, 1.0 (0.0) (EHL) and 1.5 (0.6) (SHL). Common reasons for hospitalization across hemophilia types and treatment classes were to treat uncontrolled bleeding (46%) and hemophilia-related complication (34%).

In the 12 months prior to survey, patients visited a healthcare professional (HCP) a mean (SD) of 6.7 (14.0) times. US patients consulted a mean (SD) of 3.9 (2.8) times and Spanish patients consulted a mean (SD) of 16.7 (39.3) times. In HA patients, 15% of SHL, 10% EHL, and 5% NFT had an initial consultation with an emergency room physician. A proportion of patients had an initial consultation with a physiotherapist (HA, NFT: 61%, EHL: 56%, SHL: 54% and HB, EHL: 55%, SHL: 52%). Of adult patients, 57% involved a physiotherapist in the 12 months prior to survey collection vs. 23% of pediatric patients.

Patient self-reported data was provided by 310 patients. In HA patients (81%), 34% and 42% NFT, 25% and 36% SHL, and 18% and 54% EHL aimed to reduce hospitalizations/urgent care visits and protect joints, respectively. For HB patients (19%), 21% and 50% EHL, and 19% and 35% SHL aimed to reduce hospitalizations/urgent care visits and protect joints, respectively.

Conclusions

HCRU was high among PwH, in terms of hospitalizations, HCP visits and use of monitoring tests. PwH aimed to protect joints and reduce hospitalizations/urgent care visits. With this understanding, there is a need for a novel treatment with optimized treatment strategies to reduce clinical burden in PwH.

Disclosures

Gomez:Bayer: Consultancy; Global Blood Therapeutics: Consultancy; Genentech: Consultancy; CSL Behring: Consultancy; Sanofi: Consultancy. Salehi:Adelphi Real World: Current Employment. Morton:Adelphi Real World: Current Employment. Afonso:Sanofi: Current Employment, Other: hold stocks and/or stock options in the company.

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2024
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