The ASH Research Collaborative (ASH RC) funded three research projects which will leverage the Sickle Cell Disease (SCD) Research Network to improve quality of care, quality of life, and access to effective treatments for people living with SCD.

This research leverages the unique strengths and resources of the Network and collectively addresses high-priority questions that are a part of the ASH RC’s research agenda and ASH research priorities, including the assessment, prevention, and treatment of end-organ damage; optimization of existing therapies; strengthening of curative strategies; and the dissemination and implementation of evidence-based care through collaboration.

The awardees include:

Allison King, MD, Washington University School of Medicine, Longitudinal Kidney Function Trajectories During Pregnancy and Postpartum in Women with Sickle Cell Disease: A Multicenter Prospective Feasibility Study

This feasibility study will map kidney function during pregnancy and the early postpartum period in women with SCD in integrated obstetrics-hematology clinics. This study will evaluate the practicality of renal phenotyping and data collection to inform the structure, endpoints, and timing of future interventional trials targeting renal risk in this population.

Julia Xu, MD, University of Pittsburgh, STELLAR-SCD: STimulating Erythropoiesis for Long-Lasting Anemia Relief in Sickle Cell Disease

This trial will evaluate a potential treatment for adults on hydroxyurea with and without chronic kidney disease. This study will evaluate changes in hemoglobin concentration and transfusion requirements alongside function and patient-reported outcomes. The data generated from this study will guide dosing, safety monitoring, and patient selection for future larger-scale studies of this treatment.

Zachary Crees, MD, Washington University School of Medicine, A Phase II, Pivotal Multi-Center, Randomized Controlled Trial of Plerixafor versus Motixafortideto Mobilize Hematopoietic Stem and Progenitor Cells (HSPCs) Prior to Gene Therapy in Adults and Pediatric Patients with Sickle Cell Disease (SCD)

This phase II study will compare two strategies to optimize cell collection in adolescents and adults with sickle cell disease undergoing gene therapy or gene editing. The trial evaluates efficiency and safety, including pain events. Findings are intended to define evidence-based mobilization parameters that can be generalized across platforms and age groups.