Background: Sickle cell disease (SCD) is the most common inherited hemoglobinopathy globally, with the greatest burden in low- and middle-income countries (LMICs). Advances in medical care and disease-modifying therapies have improved survival into adulthood, making structured healthcare transition (HCT) strategies critical. In the absence of such strategies, adolescents and young adults (AYAs) with SCD are at an increased risk for poor health outcomes, such as interruptions in care, disease complications, and early mortality.

In Brazil, despite universal newborn screening and specialized care, the SCD burden remains high, with an estimated prevalence of 100,000 individuals. Although survival to age 18 is approaching 80%, mortality peaks between ages 18-30, coinciding with the transition to adult care, a period often marked by worsening disease severity and increase healthcare use. No formalized SCD HCT program exist in Brazil.

The 21-item Self-Management Skills Checklist (SMSC) assesses self-management skills as an indicator of HCT readiness in AYAs, specifically with SCD, capturing patient and caregiver perspectives across disease knowledge and self-management skills domains. The SMSC assesses domains using Likert-type scales, where higher scores indicate greater HCT readiness and associated with increased treatment adherence and decreased mortality. While tools like the SMSC have been implemented and studied in high-income countries (HICs) like the United States (US), research on HCT readiness for SCD patients in LMICs like Brazil or how it compares to HICs is limited.

Objectives: (i) To identify key factors associated with transition readiness among AYAs with SCD and caregivers in Brazil and the US.

(ii) To compare SMSC scores across domains and across country-specific factors to assess similarities in HCT preparedness.

Methods: In Brazil, the SMSC was administered at two leading SCD centers in the nation's largest cities: UNIFESP in São Paulo and HEMORIO in Rio de Janeiro. In the US, the SMSC was administered at St. Jude Children's Research Hospital, which operates a SCD dedicated HCT program. AYAs with SCD aged 12-21 were recruited to complete the SMSC during routine clinic visits between October 2023 to April 2024 (Brazil) and November 2013 to April 2024 (US). Caregiver proxies completed the SMSC based on perception of the child's self-management skills. Descriptive statistics, Chi-square tests and t-tests compared demographic and clinical variables across countries. Multivariable linear regression assessed associations between SMSC scores and participants characteristics. Analyses were conducted using SAS 9.4 with a significance level of 0.05.

Results: A total of 199 AYAs (mean age:16.70±2.78) and 135 caregivers from Brazil, and 220 AYAs (mean age:16.03±0.93) and 187 caregivers from the US participated. Across all AYAs, increasing age was significantly associated with higher HCT readiness scores (p<0.01). Disease-modifying therapies including chronic transfusion, was positively associated with higher overall (β=2.99,p=0.01) and skill scores (β=1.86,p=0.04), with an even stronger effect observed among Brazilian participants (overall:β=3.37;skills:β=2.96). Hydroxyurea use was similarly associated with an increase in both the total sample (β=2.20) and Brazilian cohort (β=2.54). Brazilian AYAs scored significantly lower than US peers in overall (-3.06 points), skill (-1.73 points), and knowledge domains (-1.88 points;p<0.01). Among Brazilian caregivers, separated/divorced status correlated with a 2.20-point lower knowledge score, and each additional child was associated with a 0.31-point increase.

Conclusion: Age, chronic transfusion, and hydroxyurea use were significant predictors of transition readiness, with stronger effects observed in the Brazilian cohort. While caregiver characteristics showed moderate influence, lower readiness scores among Brazilian AYAs compared to US highlight disparities in healthcare access, education, and HCT preparation. These findings suggest that disease-modifying therapies may promote care engagement and self-management through increased provider visits, key elements to successful HCT. Implementing a formalized HCT program in Brazil with routine readiness assessments and education support may improve outcomes for AYAs with SCD. Bridging the pediatric-to-adult care gap is essential in low-resource settings to advance equitable and continuous care.

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2025
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