Background There are sparse data on long-term and late effects of hematopoietic cell transplantation (HCT) for sickle cell disease (SCD) Objectives To establish an international registry of long-term outcomes post-HCT for SCD and demonstrate the feasibility of recruitment at a single site in the US. Methods The STELLAR registry is designed to enroll SCD patients ≥ 1-year post-HCT, their siblings without SCD, and non-transplanted SCD controls to collect participant self-report of health status and practices using the BMT survivor study surveys, HRQOL using PROMIS 25 or 29, cGVHD using the symptom scale survey, daily pain using an electronic pain diary, economic impact of HCT using the financial hardship survey, and sexual function using PROMIS SexFSv2.0. We also piloted retrieval of clinical data previously submitted to CIBMTR, recorded demographics, height, weight, BP, hip and waist circumference, timed-up-and-go, and handgrip test, and obtained blood for metabolic screening, gonadal function, fertility potential, and biorepository of plasma, serum, RNA, and DNA. Results Among 100 eligible post-HCT patients, we enrolled 72 participants 9-38 (median 17) years age. We also enrolled 19 siblings 5-32 (median10)years age and 28 non-transplanted SCD controls 4-46 (median 22) years age. Of 119 participants, 73 completed 85 sets of surveys and 41 contributed samples to the biorepository. We successfully piloted retrieval of data submitted to CIBMTR and expanded recruitment to seven US, Canada, UK, and Nigeria sites. Conclusions It is feasible to recruit subjects and conduct study procedures for the STELLAR registry of long-term and late effects of HCT for SCD.

Background: Sickle cell disease (SCD), the commonest monogenic disorder, affects more than 300,000 births annually, with 44,000 in India. While the clinical phenotype of SCD is considered to be milder in aboriginal populations in India, there is a paucity of data on outcomes. To determine the severity of SCD in this population, we studied mortality rates and causes of mortality in a longitudinal cohort of patients with SCD in a remote aboriginal community in India receiving community-based comprehensive care. Procedures: Causes of death in this cohort from January 2008 to December 2018 were analyzed. Details were collected from hospital records and in case of deaths at home, by utilizing the WHO verbal autopsy questionnaire. Results: The cohort consisted of 157 patients belonging to the Paniya, Betta Kurumba, Kattunyakan, and Mullu Kurumba tribes. During the study period, there were 22 deaths, all from the Paniya tribe. Twelve deaths (54.5%) occurred in the hospital and the remaining at home (45.5%) reflecting a crude mortality rate of 140 per 1000 population. 25% of deaths occurred in the 6-18 age group. There were no deaths in the 0-5 age group. The median age of death was 25 years, which was 20 years less than in the non-SCD aboriginal population. The leading causes of death were acute chest syndrome, anemia, and sepsis among the SCD patients and stroke and suicides in the non-SCD aboriginal population Conclusion: SCD is a severe disease among the Gudalur Valley’s aboriginal population with a significant risk of premature mortality.