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denotes that this is a ticketed session.Type: Oral
Session: 904. Outcomes Research: Hemoglobinopathies: Non-Malignant Conditions: Transforming Care: Insights into Healthcare Utilization, Outcome Measurement, and Treatment Impact in Sickle Cell Disease
Hematology Disease Topics & Pathways:
Research, Sickle Cell Disease, Clinical Research, Health outcomes research, Hemoglobinopathies, Patient-reported outcomes, Diseases, Young adult , Study Population, Human
Methods: This randomized, multicenter, controlled trial, enrolled individuals with SCD aged >=17 years from five U.S. children’s hospitals. Participants were randomized 1:1:1 to receive a six-month intervention of CHW support, iManage SCD, or usual care. Both the interventions included goal setting, self-management skill development, symptom tracking, and transition support. The CHW intervention provided weekly synchronous support to participants primarily via phone calls, while iManage SCD offered virtual peer support via interaction with discussion boards. The usual care group received standard care with a transition checklist for consistency across sites. The primary outcome was quality of life, assessed using the PedsQL for SCD. Secondary outcomes included SCD knowledge, transition readiness, and social support. Mixed effects models were used to predict baseline and follow-up scores within groups and differences in changes between groups.
Results: A total of 405 participants with SCD were enrolled in the study. Baseline demographic data, clinical characteristics and markers of disease severity were comparable across study arms. At 6 months, the intervention groups (CHW and iManage SCD), combined for analyses, showed modest improvements in quality of life compared to usual care (PedsQL score : 3.4 [95% CI -0.9, 7.6], P=0.005). The largest change was seen among the CHW group, which showed an increase of 4.7 points at 180 days compared to controls (95% CI: -0.3, 9.7, p = 0.002). The iManage SCD group improved by 2.2 points compared to controls (95% CI: -2.7, 7.1, p = 0.048). Among the components of PedsQL, the largest change was observed in the communication II subscale. No significant differences were found in SCD knowledge, transition readiness, or social support.
Conclusion(s): Both community health worker support and mobile health programs hold promise for enhancing the quality of life for young adults with SCD transitioning to adult care, with the CHW intervention demonstrating a more pronounced effect. These findings underscore the importance of integrating such programs into routine care and can provide guidance to clinic directors in their adoption of these tools to improve transition outcomes for this vulnerable population.
Disclosures: Jan: Merck: Current holder of stock options in a privately-held company; Vertex: Current equity holder in publicly-traded company. Niss: Pfizer: Consultancy. Crosby: Novartis: Honoraria; Sanofi Genzyme: Membership on an entity's Board of Directors or advisory committees; Novo Nordisk (Forma Therapeutics): Membership on an entity's Board of Directors or advisory committees; Sobi: Membership on an entity's Board of Directors or advisory committees; Takeda: Membership on an entity's Board of Directors or advisory committees; Professional Resource Exchange: Patents & Royalties. Andemariam: Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; American Society of Hematology: Research Funding; bluebird bio: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Research Funding; Agios: Consultancy, Membership on an entity's Board of Directors or advisory committees; Global Blood Therapeutics: Consultancy, Membership on an entity's Board of Directors or advisory committees; Hemanext: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Fulcrum Therapeutics: Other: Data Safety Monitoring Board / Adjudication Committee; Editas: Other: Data Safety Monitoring Board / Adjudication Committee; Novo Nordisk: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Connecticut Department of Public Health: Research Funding; Health Resources and Services Administration: Research Funding; Roche: Consultancy, Membership on an entity's Board of Directors or advisory committees; Sanofi Genzyme: Consultancy, Membership on an entity's Board of Directors or advisory committees; Vertex: Consultancy, Membership on an entity's Board of Directors or advisory committees; Afimmune: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Accordant: Consultancy, Membership on an entity's Board of Directors or advisory committees. Smith-Whitley: Pfizer: Current Employment.