EXPERT-ESTIMATED TREATMENT OUTCOMES AND RESIDUAL DISEASE IN HEMOPHILIA CARE IN BRAZIL: FINDINGS FROM A MODIFIED DELPHI CONSENSUS
Author(s)
Ieda S. Pinto, MD1, Franciele Iachecen, MSc2, Bruna Webler, MS2, JULIA LIMA, MSc2, Mariana Alves, PhD3, Ana Karolina Nunes, PhD3, Ana Dantas, MD3, Rodrigo F. Alexandre, PharmD, MSc3, Daniela Pachito, MD, PhD3, Margareth C. Ozelo, MD, PhD4;
1Federal University of Para, Belem, Brazil, 2IQVIA Solutions, Sao Paulo, Brazil, 3Pfizer Brazil, Sao Paulo, Brazil, 4University of Campinas, Hemocentro UNICAMP, Campinas, Brazil
1Federal University of Para, Belem, Brazil, 2IQVIA Solutions, Sao Paulo, Brazil, 3Pfizer Brazil, Sao Paulo, Brazil, 4University of Campinas, Hemocentro UNICAMP, Campinas, Brazil
OBJECTIVES: To describe expert-based estimates related to treatment outcomes, adherence, and residual disease in hemophilia A and B, derived from a modified Delphi consensus conducted among Brazilian hematologists.
METHODS: As part of a comprehensive Delphi study on hemophilia care in Brazil, a specific block addressed treatment effectiveness, adherence, and residual disease. Ten hematologists with extensive experience in the public health system provided estimates grounded in clinical practice on the number of annual bleeding episodes, proportion of joint complications, and adherence rates. Responses were summarized using descriptive statistics (mean, standard deviation [SD], and range).
RESULTS: Experts estimated that patients with hemophilia A without inhibitors experience a mean of 3.3 bleeding episodes per year while on continuous replacement prophylaxis (SD: 2.0; range: 1-8), increasing to 5.6 episodes among those with inhibitors (SD: 1.8; range: 3-10). Emicizumab was perceived to be associated with lower number of bleeding episodes (mean 0.9 per year; SD: 0.9; range: 0-2). For hemophilia B, estimated mean annual bleeding rates were 2.3 episodes in patients without inhibitors (SD: 1.6; range: 0-6) and 5.2 episodes in those with inhibitors (SD: 2.4; range: 2-10). Reduced adherence to factor replacement therapy was estimated to increase bleeding frequency by a mean of 6.1 episodes per year (SD: 2.2; range: 4-10). Arthropathy was estimated to affect 27.0% of patients receiving primary prophylaxis (SD: 15.3; range: 5-50) and 42.5% of those on secondary prophylaxis (SD: 18.4; range: 10-70). Surgical intervention was required in an estimated 10.4% (SD: 9.0; range: 0-25) and 20.1% (SD: 14.2; range: 0-40), respectively. Compromised adherence to prophylaxis was estimated in 33.0% of patients (SD: 25.7; range: 10-80).
CONCLUSIONS: Consensus-based estimates underscore residual bleeding risk, joint complications, and adherence challenges in hemophilia care in Brazil, reinforcing the need for strategies to improve treatment adherence and outcomes.
METHODS: As part of a comprehensive Delphi study on hemophilia care in Brazil, a specific block addressed treatment effectiveness, adherence, and residual disease. Ten hematologists with extensive experience in the public health system provided estimates grounded in clinical practice on the number of annual bleeding episodes, proportion of joint complications, and adherence rates. Responses were summarized using descriptive statistics (mean, standard deviation [SD], and range).
RESULTS: Experts estimated that patients with hemophilia A without inhibitors experience a mean of 3.3 bleeding episodes per year while on continuous replacement prophylaxis (SD: 2.0; range: 1-8), increasing to 5.6 episodes among those with inhibitors (SD: 1.8; range: 3-10). Emicizumab was perceived to be associated with lower number of bleeding episodes (mean 0.9 per year; SD: 0.9; range: 0-2). For hemophilia B, estimated mean annual bleeding rates were 2.3 episodes in patients without inhibitors (SD: 1.6; range: 0-6) and 5.2 episodes in those with inhibitors (SD: 2.4; range: 2-10). Reduced adherence to factor replacement therapy was estimated to increase bleeding frequency by a mean of 6.1 episodes per year (SD: 2.2; range: 4-10). Arthropathy was estimated to affect 27.0% of patients receiving primary prophylaxis (SD: 15.3; range: 5-50) and 42.5% of those on secondary prophylaxis (SD: 18.4; range: 10-70). Surgical intervention was required in an estimated 10.4% (SD: 9.0; range: 0-25) and 20.1% (SD: 14.2; range: 0-40), respectively. Compromised adherence to prophylaxis was estimated in 33.0% of patients (SD: 25.7; range: 10-80).
CONCLUSIONS: Consensus-based estimates underscore residual bleeding risk, joint complications, and adherence challenges in hemophilia care in Brazil, reinforcing the need for strategies to improve treatment adherence and outcomes.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HSD65
Topic
Health Service Delivery & Process of Care
Disease
SDC: Rare & Orphan Diseases, SDC: Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)