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Thank you for attending THSNA 2026. The virtual meeting is now closed.
Presentation Details
| Understanding Information Access, Treatment Decision-Making and Treatment Satisfaction Among Caregivers of Children with ≤17 years old with Hemophilia A or B: Preliminary Findings From a Survey Study Poojita Dasika1, Jennifer Davila2, . 1Albert Einstein College of Medicine, Bronx, NY, USA.2Albert Einstein College of Medicine, Department of Pediatrics, Bronx, NY, USA |
Abstract
Background Hemophilia A (HA) and B (HB) are inherited bleeding disorders that may lead to chronic complications without early and effective management. Replacement with standard half-life factor has long been the standard of care; however, the past decade has introduced multiple new therapies. These include extended half-life products, non-factor rebalancing agents, and gene therapy, adding complexity to treatment decisions. While prior studies in adults have identified key factors influencing treatment choice, little is known about how caregivers of children with hemophilia navigate these decisions. Understanding parental information sources and decision-making factors is essential to support effective shared decision-making in pediatric hemophilia care. Objectives To assess how parents/guardians of children with HA or HB obtain information and make decisions about available treatment options. Methods This multi-site survey-based study was conducted at hemophilia treatment centers at Montefiore Medical Center and the University of Miami starting in June 2025 and is ongoing. Parents/guardians of children ≤17 years with HA or HB were recruited during clinic visits and provided in-person informed consent. Participants completed the survey either on paper or REDCap accessed via QR code. The survey collected sociodemographic information, learning preferences, information sources, and factors influencing treatment decision-making. Data were analyzed using descriptive statistics, including ranking of decision-making factors and preferred information sources. This survey study is onboarding 2 additional sites with the goal of administering the survey to 100 caregivers. Results Nineteen caregivers completed the survey for this preliminary analysis. Among respondents, 84% reported caring for a child with HA and 16% for a child with HB. Reported disease severity included 5% mild, 47% moderate, and 47% severe. Reported ages of children ranged from 2 months to 15 years. All 19 caregivers stated their child was on prophylaxis, with 16(82%) reporting using a subcutaneous non-factor product and 3(17%) using a factor product. Fourteen(73.7%) caregivers stated their child had never received factor to treat a bleed. Sixteen caregivers stated they were either “very” (58%) or “extremely” (35%) satisfied with their child’s current prophylactic regimen. Caregivers most often preferred to receive information from their hemophilia treatment team (84.2%), followed by independent research such as reading or attending presentations (36.8%) and, less commonly, other parents or caregivers (15.8%) (Table 1). When asked which sources they use, most relied on the treatment team (89.5%), with additional use of support groups, national organization websites, and medical literature. Decision-making responses indicated that route of administration (63.2%) and peak factor levels (31.6%) were the most influential factors when choosing a treatment (Figure 1). Conclusion
Preliminary findings indicate that caregivers of children with hemophilia primarily rely on their treatment team for information and decision-making, with limited use of independent research or peer input. Satisfaction with current prophylactic regimens was high, particularly among those using subcutaneous non-factor products. Route of administration and peak factor levels emerged as the most influential factors guiding treatment choices. These preliminary findings emphasize the importance of provider-led education and personalized counseling to support informed decisions as therapeutic options continue to expand in pediatric hemophilia care.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.
Preliminary findings indicate that caregivers of children with hemophilia primarily rely on their treatment team for information and decision-making, with limited use of independent research or peer input. Satisfaction with current prophylactic regimens was high, particularly among those using subcutaneous non-factor products. Route of administration and peak factor levels emerged as the most influential factors guiding treatment choices. These preliminary findings emphasize the importance of provider-led education and personalized counseling to support informed decisions as therapeutic options continue to expand in pediatric hemophilia care.
No part of this publication may be reproduced, distributed, or transmitted in any form or by any means, including photocopying, recording, or other electronic or mechanical methods, without the prior written permission of the author.