Highlight
– Community health worker (CHW) support produced small but statistically significant, durable improvements in health-related quality of life (HRQOL) over 18 months among young adults with sickle cell disease (SCD) transitioning to adult care.
– A mobile health (mHealth) application with virtual peer discussion produced a short-term HRQOL improvement at 6 months but did not sustain effects through 12 and 18 months.
– Neither intervention reached the prespecified 10-point threshold for a large clinical effect on the PedsQL SCD module, but CHW support halted the decline observed with enhanced usual care (EUC).
Background
Sickle cell disease (SCD) is a chronic, inherited hemoglobinopathy that confers substantial morbidity and premature mortality. Advances in pediatric care have improved survival into young adulthood, but the transition from pediatric to adult care is a recognized high-risk period characterized by interruptions in care, increased acute events, and higher mortality. Young adults with SCD also face psychosocial stressors, health literacy gaps, and social determinants of health that influence outcomes. Interventions to support the transition process—ranging from structured transition clinics to peer support and digital tools—aim to preserve engagement with care and improve patient-centered outcomes such as health-related quality of life (HRQOL).
Study design
The Community Health Workers and Mobile Health for Emerging Adults Transitioning Sickle Cell Disease Care study (NCT03648710) was an observer-blinded, multicenter randomized clinical trial conducted at five U.S. children’s hospitals between January 15, 2019, and December 31, 2022, with analyses completed through mid-2025. Eligible participants were aged ≥17 years with SCD and were randomized 1:1:1 to one of three 6-month interventions:
- Enhanced usual care (EUC): standard transition checklist provided across sites.
- CHW plus EUC: weekly synchronous, primarily telephone-based support from community health workers focused on goal setting, self-management, skill development, symptom tracking, and transition planning.
- mHealth plus EUC: an app-based intervention with self-management features and virtual peer support via discussion boards, plus the EUC checklist.
The primary outcome was HRQOL measured with the Pediatric Quality of Life Inventory (PedsQL) SCD module at baseline and 6, 12, and 18 months. A prespecified 10-point change on PedsQL was considered a large clinical effect. Secondary outcomes included SCD knowledge, transition readiness, and social support.
Key findings
Of 700 eligible patients, 405 were enrolled and 375 randomized (mean age 18.9 years; 51.5% women). Baseline characteristics and disease severity markers were similar across arms.
Primary outcome: HRQOL (PedsQL SCD module)
- At 6 months, compared with EUC, CHW plus EUC showed modest improvement in HRQOL: adjusted difference 5.25 points (95% CI, 2.05–8.45).
- mHealth plus EUC improved HRQOL at 6 months compared with EUC by 3.31 points (95% CI, 0.27–6.35).
- The EUC group experienced a decline in PedsQL scores over time (−2.58 points at 6 months; 95% CI, −4.67 to −0.49), suggesting deterioration in HRQOL during transition when only checklist-based support was provided.
- CHW effects were durable: CHW plus EUC retained statistically significant improvements versus EUC at 12 months (5.56 points; 95% CI, 1.52–9.61) and 18 months (6.14 points; 95% CI, 1.75–10.54).
- Although statistically significant, neither intervention reached the prespecified 10-point threshold for a large clinical effect.
Secondary outcomes
No significant differences were observed between groups for secondary outcomes including SCD knowledge, transition readiness measures, and social support across the follow-up visits.
Interpretation of effect sizes
The observed 5–6 point improvements with CHW support fall below the 10-point large-effect threshold but are clinically meaningful in several ways. First, they represent a reversal or prevention of the decline seen in EUC, suggesting that CHW support stabilizes HRQOL during a vulnerable period. Second, the persistence of benefit at 12 and 18 months indicates durable behavioral or engagement changes beyond the 6-month intervention window. Third, in complex chronic conditions where multiple social and health-system barriers exist, smaller increments that are sustained can translate into meaningful downstream benefits—potentially reducing acute care use or improving medication adherence—though those outcomes were not reported as primary endpoints in this trial.
Expert commentary and clinical implications
Mechanisms: Community health workers commonly address social determinants, provide individualized coaching, improve care navigation, and reinforce self-management—functions that appear to translate into patient-reported improvements in HRQOL for young adults with SCD. The CHW model’s synchronous, personalized contact likely explains both larger and more durable effects relative to the largely asynchronous, peer-based mHealth discussion boards.
Why did mHealth show transient benefit?
Digital tools can extend reach but depend heavily on user engagement, content interactivity, and integration with personalized coaching. In this trial, mHealth produced a modest short-term HRQOL gain at 6 months that was not sustained, suggesting that peer discussion and self-monitoring alone may be insufficient for long-term behavior change in this population without stronger facilitation, tailored feedback, or integration with human coaching.
Generalizability and equity considerations
The trial enrolled participants from five U.S. children’s hospitals; results therefore best apply to similar clinical settings and to emerging adults already connected to pediatric systems. Real-world populations may include individuals with less access to technology or intermittent care engagement, where CHW models may be even more impactful. Implementation must consider workforce training, cultural congruence, language needs, and sustainable financing, especially because community health worker programs are variably reimbursed across health systems and payers.
Limitations
- The trial’s primary outcome was patient-reported HRQOL; objective health-service outcomes (e.g., hospitalizations, ED visits) were not emphasized in the summary and may be reported elsewhere.
- Participants and intervention deliverers could not be blinded, which can introduce expectation bias toward interventions with more human contact.
- Engagement metrics (frequency of contacts, app usage) and dose–response relationships were not presented in the summary; such data are important to understand who benefits most.
- Cost-effectiveness and scalability were not addressed in the primary report; these are key for health-system adoption.
Implications for practice and policy
For clinicians and health systems caring for adolescents and young adults with SCD, this trial supports integrating CHW support into transition programming as a strategy to preserve HRQOL during transfer to adult care. Practical steps include:
- Embedding CHWs within multidisciplinary transition teams to provide ongoing outreach, care navigation, and linkage to social services.
- Combining CHW support with tailored digital tools to maximize both personalization and scalability—e.g., CHW-coached app use, automated symptom tracking with human follow-up for alerts.
- Advocating for sustainable reimbursement models for CHWs, including through value-based payment arrangements or targeted grants, given evidence of patient-centered benefit.
Research gaps and future directions
Future work should include:
- Cost-effectiveness and implementation studies assessing scalability, workforce training needs, and payer perspectives.
- Hybrid interventions that integrate CHWs with more sophisticated, interactive mHealth features (e.g., tailored feedback, clinician dashboards) to combine the strengths of both approaches.
- Subgroup analyses to identify patients most likely to benefit (e.g., those with social instability, poor baseline engagement, or higher symptom burden).
- Longer-term follow-up focused on clinical outcomes such as acute care utilization, adherence to disease-modifying therapies, and transition to stable adult care.
Conclusion
This multicenter randomized trial demonstrates that community health worker support yields modest but statistically significant and durable improvements in health-related quality of life for young adults with sickle cell disease transitioning to adult care, and importantly it halts the HRQOL decline seen with checklist-based enhanced usual care. A mobile health peer-support intervention delivered transient benefit. Although effects did not meet the prespecified threshold for a large clinical change, the findings are clinically relevant: CHW programs provide practical, patient-centered support during a known high-risk period and merit integration into transition services alongside evaluation of cost, implementation strategies, and combination with digital tools.
Funding and clinicaltrials.gov
Trial registration: ClinicalTrials.gov Identifier: NCT03648710. Funding details are reported in the original publication (Jan S et al., JAMA Netw Open. 2025).
References
Jan S, Steinway C, Belton T, et al. Community Health Worker and Mobile Health Interventions for Quality of Life Among Young Adults With Sickle Cell Disease: A Randomized Clinical Trial. JAMA Netw Open. 2025;8(11):e2543571. doi:10.1001/jamanetworkopen.2025.43571
National Heart, Lung, and Blood Institute. Evidence-Based Management of Sickle Cell Disease: Expert Panel Report (2014). Available at: https://www.nhlbi.nih.gov/ (access clinical guideline resource).
Quinn CT, Rogers ZR, McCavit TL, Buchanan GR. Improved survival of children and adolescents with sickle cell disease. Blood. 2010;115(17):3447-3452. doi:10.1182/blood-2009-07-233700
For the full methodological details, engagement metrics, and secondary analyses, consult the published JAMA Network Open report.
