Long-Term Effects on Heart, Lung, and Kidney Health After Curative Therapies Versus Standard Treatment in Children and Adults with Sickle Cell Disease

What this Trial Is About

Researchers are evaluating the long-term effects of curative therapies for children and adults with sickle cell disease SCD, focusing on heart, lung, and kidney health. This observational study aims to compare outcomes of those receiving curative therapies like hematopoietic stem cell transplants HSCT to those receiving standard disease-modifying treatments. The study also explores genetic factors that might influence results after curative therapy. Participants include children aged 4 to 17 who receive myeloablative allo-HSCT or standard therapy, and adults aged 18 to 65 who receive nonmyeloablative allo-HSCT or standard therapy. The study observes lung function through measures like FEV1 and FVC, heart function using tricuspid regurgitant jet velocity TRJV, and kidney function via estimated glomerular filtration rate eGFR and albuminuria levels over an average of four years. Throughout the study, participants provide pre- and post-treatment health data including lung and kidney function tests and heart assessments. Researchers monitor changes over time to understand potential benefits or risks of curative therapies compared to standard treatment. The study also gathers information from patient records and family reports to assess data accuracy. Participation lasts about four years with ongoing evaluations of heart, lung, and kidney health.

Cooperative Assessment of Late Effects for SCD Curative Therapies