Research Supports Need for Diversity in Hemophilia Trials and Reducing Resource Strain

A pair of recent studies featured at the 2024 American Society of Hematology (ASH) Annual Meeting & Exposition shed light on advancements in clinical trial diversity and the health care resource utilization (HCRU) burden in managing the rare blood disorder.^1,2

High health care burden for patients with hemophilia highlights the need for innovative treatments.

Image Credit: jarun011-stock.adobe.com

Enhancing Diversity in Hemophilia Clinical Trials

Historically, Black, African American (AA), and Hispanic patients with hemophilia have faced higher risks of severe complications, such as intracranial hemorrhages, and have been underrepresented in clinical trials.¹ Addressing this, a recent analysis evaluated operational strategies to enhance racial and ethnic diversity in hemophilia trials (NCT03974113, NCT04759131, and NCT05662319).

Between January 2020 and July 2024, trials established diversity and inclusion (D&I) goals based on real-world epidemiological data. Recruitment leveraged the Diversity & Inclusion Metrics Overview (DIMO), a proprietary tracking software, to monitor enrollment progress. Key strategies included prioritizing trial sites with access to diverse populations, offering participant support such as transportation reimbursements, and translating recruitment materials into multiple languages.

The approach taken in this study successfully achieved diversity enrollment targets across all minority groups assessed. Specifically, Black or African American participants comprised 31.1% of the total enrollment, surpassing the target of 13%. Asian participants accounted for 13.1%, exceeding the target of 5%, while Hispanic or Latino participants comprised 24.6%, well above the target of 13%. These results underscore the effectiveness of end-to-end, technology-enabled strategies in fostering diverse participation in trials. By broadening inclusivity, the results demonstrate not only an enhancement in the validity of clinical trials but also the potential for improved treatment outcomes for underserved populations.

The Burden of Hemophilia Management on Health Care Resources

A separate study examined HCRU among people with hemophilia (PwH) using real-world data from 8 countries, including the US and Japan.² The analysis included 1418 male patients aged 12 years and older with moderate to severe hemophilia A (HA) and B (HB).

The majority of patients (82%) were on prophylactic therapy. Patients with HA primarily received standard half-life (SHL, 43%), extended half-life (EHL, 26%), or non-factor therapies (NFT, 25%). For patients with HB, EHL (56%) was the most common treatment. Most patients (94%) did not have current inhibitors.

The study revealed significant findings demonstrating that, on average, these individuals underwent 18.8 monitoring tests annually, with common tests including complete blood counts (86%), activated partial thromboplastin time (82%), and prothrombin time (59%). Hospitalization rates varied between 3% and 17%, influenced by hemophilia type and treatment regimen. Uncontrolled bleeding accounted for 46% of hospitalizations, while complications represented 34%. Furthermore, PwH consulted health care providers an average of 6.7 times per year, with notable variability observed across different countries. Physiotherapy played a role in the care of 57% of adult patients, compared with 23% of pediatric patients.

Patients reported aiming to reduce hospitalizations and protect joint health. However, the high HCRU and frequency of hospitalizations underscore the need for novel treatment strategies to alleviate the clinical and societal burdens of hemophilia.

Implications for Future Research and Care

The studies underscore critical aspects of hemophilia research and management, particularly the importance of diversity in clinical trials. Achieving inclusive trial participation is essential for ensuring findings are generalizable and that treatment advancements are equitable. Additionally, the high HCRU associated with hemophilia highlights the necessity for optimized treatment strategies to minimize hospitalizations, enhance the quality of life for patients, and reduce the financial burden on health care systems.

Collectively, these insights suggest a future where improved representation in clinical trials and the development of innovative therapies could significantly transform outcomes for individuals with hemophilia on a global scale.

References

1. Boynton K, Adams M, Knobe K, Farfan S, Darkwah I. Strategies for increasing racial and ethnic diversity in hemophilia clinical trials and their outcomes. Blood. 2024;144(Supplement 1):3969.

2. Gomez E, Salehi H, Morton E, Afonso M. Real-world analysis of healthcare resource utilization in patients with hemophilia A and B with or without inhibitors using data from Disease Specific Programmes. Blood. 2024;144(Supplement 1):2262.