In vivo measurement of RBC survival in patients with sickle cell disease before or after hematopoietic stem cell transplantation

•Biotin labeling of RBCs is a safe and feasible methodology to evaluate RBC survival in patients with SCD before and after HSCT.•Understanding differences in RBC survival may inform hemoglobin composition thresholds required to reverse the phenotype after gene therapy. [Display omitted] Stable, mixe...

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Published in:Blood advances 2024-04, Vol.8 (7), p.1806-1816
Main Authors: Leonard, Alexis K., Furstenau, Dana, Inam, Zaina, Luckett, Christina, Chu, Rebecca, Demirci, Selami, Essawi, Khaled, Gudmundsdottir, Bjorg, Hinds, Malikiya, DiNicola, Julia, Li, Quan, Eaton, William A., Cellmer, Troy, Wang, Xunde, Thein, Swee Lay, Macari, Elizabeth R., VanNest, Sara, Hsieh, Matthew M., Bonner, Melissa, Pierciey, Francis J., Tisdale, John F.
Format: Article
Language:English
Subjects:
Anemia, Sickle Cell - pathology
Biotin
Clinical Trials and Observations
Erythrocytes - pathology
Hematopoietic Stem Cell Transplantation - adverse effects
Hematopoietic Stem Cell Transplantation - methods
Hemoglobins
Humans
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Summary:•Biotin labeling of RBCs is a safe and feasible methodology to evaluate RBC survival in patients with SCD before and after HSCT.•Understanding differences in RBC survival may inform hemoglobin composition thresholds required to reverse the phenotype after gene therapy. [Display omitted] Stable, mixed-donor–recipient chimerism after allogeneic hematopoietic stem cell transplantation (HSCT) for patients with sickle cell disease (SCD) is sufficient for phenotypic disease reversal, and results from differences in donor/recipient–red blood cell (RBC) survival. Understanding variability and predictors of RBC survival among patients with SCD before and after HSCT is critical for gene therapy research which seeks to generate sufficient corrected hemoglobin to reduce polymerization thereby overcoming the red cell pathology of SCD. This study used biotin labeling of RBCs to determine the lifespan of RBCs in patients with SCD compared with patients who have successfully undergone curative HSCT, participants with sickle cell trait (HbAS), and healthy (HbAA) donors. Twenty participants were included in the analysis (SCD pre-HSCT: N = 6, SCD post-HSCT: N = 5, HbAS: N = 6, and HbAA: N = 3). The average RBC lifespan was significantly shorter for participants with SCD pre-HSCT (64.1 days; range, 35-91) compared with those with SCD post-HSCT (113.4 days; range, 105-119), HbAS (126.0 days; range, 119-147), and HbAA (123.7 days; range, 91-147) (P
ISSN:2473-9529
2473-9537
2473-9537
DOI:10.1182/bloodadvances.2023011397