ASH: Data on warm autoimmune hemolytic anemia burden

These findings underscore the significant disease burden for the estimated one in 8000 people living with wAIHA, including the high unmet need for targeted therapies with proven safety and efficacy profiles and the associated impact the disease has on healthcare utilization.

“Approximately 50,000 people in the U.S. are living with wAIHA and grappling with devastating physical symptoms such as debilitating fatigue, dizziness, shortness of breath, and jaundice and in severe cases, chest pain or loss of consciousness, often with profound implications to their mental health,” said Karen Jones, Executive Director of the patient advocacy group wAIHA Warriors and an abstract co-author.* “Our findings demonstrate the continued need for research into new treatment approaches for people living with this condition with the potential to maintain immune–function and reduce the need for invasive surgeries and repeat blood transfusions.”

Johnson & Johnson is collaborating with patient advocates and the scientific community to generate real-world evidence and harness the patient perspective to inform their clinical development process. Members of a global patient council shared their diagnosis and treatment journeys, the emotional and financial impacts of the disease, and their views about opportunities for improved management. These patients expressed high levels of anxiety related to the cyclical nature of the condition and lack of sustained disease control as well as dissatisfaction with current treatment management options.  All council members experienced side effects from at least one treatment prescribed to them, none of which are indicated specifically for the treatment of wAIHA.3 This was also demonstrated in a sentiment analysis, which looked at over 22,000 conversations among adults who self-identified as having wAIHA, revealing that the most common negative sentiment themes for rituximab, a common treatment approach for the condition, were lack of efficacy and side effects.

Two of the accepted posters established that wAIHA is associated with significant long-term healthcare resource utilization – including ongoing need for emergency and inpatient care – as demonstrated through observational studies on the healthcare utilization of individuals living with wAIHA in the United States and Sweden. These findings highlight the need to achieve greater disease control after initial wAIHA diagnosis.

ABOUT WARM AUTOIMMUNE HEMOLYTIC ANEMIA (wAIHA); Warm autoimmune hemolytic anemia (wAIHA) is a rare, life-threatening condition where autoantibodies lead to the premature destruction of red blood cells (RBCs), resulting in anemia. Approximately 1-3 new people per 100,000 are affected by wAIHA per year, and about 1 in 8,000 individuals are living with the condition. This condition affects both women and men, and can affect people at any age with incidence increasing over the age of 50. Additionally, people with wAIHA are at increased risk of other serious complications such as venous thrombotic events, acute renal failure, and infection.

There are no Food and Drug Administration (FDA)-approved drugs indicated for wAIHA, and treatment typically consists of corticosteroids, broad immunosuppressants and B-cell directed therapies.6 With an unmet need for treatment in wAIHA, continued research for evidence-based potential therapies is critical.

ABOUT NIPOCALIMAB; Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Prevalent Rheumatology. Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.

Johnson & Johnson is evaluating nipocalimab for the potential treatment of wAIHA in the Phase II/III ENERGY study, which is expected to read out in 2025.

Citation:A Retrospective Database Analysis of Healthcare Resource Utilization in Patients with Warm Autoimmune Hemolytic Anemia in the United States.  ASH Program: Oral and Poster Abstracts .Session: 905. Outcomes Research: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster I Hematology Disease Topics & Pathways: Clinical Practice (Health Services and Quality), Treatment Considerations. Saturday, December 7, 2024, 5:30 PM-7:30 PM Louis A Jackson III, PharmD. Jacqueline Pesa, MSEd, PhD, MPH, Zia Choudhry, MD, PhD, MBA, Shannon Ferrante, Alicia K Campbell, PharmD and Caroline Piatek, MD.

Citation:Health Resource Utilization Among Patients with Warm Autoimmune Hemolytic Anemia in Sweden: A Retrospective Registry-Based Study; ASH Program: Oral and Poster Abstracts. Session: 901. Health Services and Quality Improvement: Non-Malignant Conditions Excluding Hemoglobinopathies: Poster I Hematology Disease Topics & Pathways: Clinical Practice (Health Services and Quality). Saturday, December 7, 2024, 5:30 PM-7:30 PM Christian Kjellander, MD, PhD^*, Concetta Crivera, PharmD, MPH, Ann Leon, PharmD, Qian Cai, MPH, MSc, Tina Jacob, PhD, Erwei Zeng, PhD, Christina V Jones, PhD, Amy Leval, PhD, Marie Fitzgibbon, MPharm, PhD, MBA, Wim Noel, PhD, Cathye Shu, MD, PhD^* and Gunnar Larfors, MD, PhD^*