Design and Implementation of the Sickle Cell Disease Hematopoietic Cell
Transplantation Evaluating Long Term and Late Outcomes Registry
(STELLAR) to Compare Long Term Outcomes after Hematopoietic Cell
Transplantation to that in Siblings without Sickle Cell Disease and in
Non-Transplanted Individuals with Sickle Cell Disease
Abstract
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.