Sickle Cell Disease Insights

Cell and gene therapies are transforming rare disease treatment, offering new hope where options were once limited. But how do they work, and what choices exist for your community?

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The study by Prince et al. (2025) evaluated the effectiveness of a young adult clinic (YAC) designed for individuals with sickle cell disease aged 18-30. The study found that a high percentage (89%) of referred patients attended their appointments at the YAC. Notably, those referred from pediatric care had the highest attendance rates. The average number of scheduling attempts before a patient attended was approximately three, indicating some persistence is required to ensure attendance. Over the 55-month study period, there were five deaths (3%) and seven successful haematopoietic stem cell transplants (4%) within the cohort.

These findings suggest that healthcare systems tailored to the needs of young adults with sickle cell disease can significantly improve their engagement in adult care. The success of the YAC underscores the importance of developing dedicated resources and care models to facilitate the transition from pediatric to adult healthcare services. Prince et al. highlight the need for further research on the best practices for integrating young adults into adult sickle cell disease care.