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Updated: Feb 28, 2026

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Emerging Considerations in Transfusion Medicine: Hematopoietic Stem and Progenitor Cell Collection for Gene Therapy
Nora M Gibson1, Janet L Kwiatkowski2, Stella T Chou3
1Division of Oncology, Department of Pediatrics, Children's Hospital of Philadelphia, Colket Translational Research Building, Room 4020, 3501 Civic Center Boulevard, Philadelphia, PA 19104, USA.
Abstract:
Gene-modified hematopoietic stem cell transplant (GM-HSCT) is an innovative curative therapy for transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD), which has demonstrated excellent efficacy in clinical trials. Two commercial products are currently available, with several additional products in clinical trials. GM-HSCT requires the mobilization and subsequent collection of hematopoietic stem and progenitor cells (HSPCs) by apheresis. Current protocols consistently demonstrate effectiveness in collecting HSPCs in patients with TDT. However, disease-specific characteristics of SCD create challenges in collecting HSPCs. Further research remains in optimizing collection procedures in this population.
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