Research Article 2026-04-21 posted v1

Expert Consensus on Unmet Needs and Barriers to Optimal Hemophilia Care in Brazil: Results from a Modified Delphi Study

M
Margareth C. Ozelo Hemocentro UNICAMP, University of Campinas, Campinas, SP, Brazil
B
Bruna Webler IQVIA Solutions, São Paulo, SP, Brazil
J
Julia Lima IQVIA Solutions, São Paulo, SP, Brazil
M
Mariana Alves Pfizer, São Paulo, SP, Brazil
A
Ana Karolina de Nunes Pfizer, São Paulo, SP, Brazil
A
Ana Dantas Pfizer, São Paulo, SP, Brazil
R
Rodrigo F. Alexandre Pfizer, São Paulo, SP, Brazil
D
Daniela V. Pachito Pfizer, São Paulo, SP, Brazil
I
Ieda Solange de Souza Pinto Fundação HEMOPA, Belém, PA, Brazil

Abstract

Background: Despite therapeutic advances, important gaps persist in hemophilia care within public healthcare systems in middle-income countries. In Brazil, universal access to prophylaxis is ensured through a centralized public system, however, the available treatments remain limited compared to international standards. This study aimed to identify unmet needs, barriers to optimal care, and priority attributes for emerging therapies in hemophilia A and hemophilia B within the Brazilian Unified Health System (SUS), using expert consensus.Results: A modified Delphi study was conducted with twelve hematologists experienced in hemophilia care from all Brazilian regions. Consensus indicated persistent unmet needs among individuals with moderate to severe hemophilia A without inhibitors (100%) and with inhibitors (80.0%), as well as hemophilia B without inhibitors (100%) and with inhibitors (91.7%). Consolidation of national clinical protocols into a single guideline was considered important to standardize care (83.3%). Key extrinsic barriers included limited access to treatment centers for patients in remote areas (83.3%), social vulnerability (83.3%), restricted access to arthropathy management and rehabilitation services (91.7%), and limited access to specialized care for acute bleeding events (83.3%). Additional barriers included shortages of trained professionals outside specialized centers (91.7%), delayed incorporation of new technologies (91.7%), insufficient integration across levels of care (83.3%), an inadequate number of specialized centers (90.0%), failures in referral and counter-referral systems (90.0%), and lack of specialized technical support in emergency settings (100%). Intrinsic barriers to adherence included difficult venous access in children aged 0 to 6 years (100%), reduced adherence during transition to self-infusion in adolescents (91.7%), lack of adequate self-infusion training (83.3%), and venous access difficulties with functional limitations in older adults (83.3%). Panelists also estimated that, among individuals receiving prophylaxis with factor replacement therapy, the mean proportion with compromised adherence was 33% (SD: 2.0). Regarding emerging therapies, consensus supported less invasive subcutaneous administration (100%), reduced dosing frequency (100%), and therapies not requiring routine laboratory monitoring (83.3%) as facilitators of adherence.Conclusions: This expert consensus highlights persistent unmet needs and structural and individual barriers to optimal hemophilia care in Brazil. The findings support health system reorganization and the incorporation of innovative therapies to improve adherence and clinical outcomes in the SUS.

Citation Information

@article{margarethcozelo2026,
  title={Expert Consensus on Unmet Needs and Barriers to Optimal Hemophilia Care in Brazil: Results from a Modified Delphi Study},
  author={Margareth C. Ozelo and Bruna Webler and Julia Lima and Mariana Alves and Ana Karolina de Nunes and Ana Dantas and Rodrigo F. Alexandre and Daniela V. Pachito and Ieda Solange de Souza Pinto},
  journal={Research Square},
  year={2026},
  doi={https://doi.org/10.21203/rs.3.rs-9471111/v1}
}
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