How do I overcome apheresis collection challenges for sickle cell disease gene therapy

Patients must undergo automated red blood cell exchange (RCE) to lower hemoglobin S in a relatively short window of time before apheresis collection of HSCs. The risks and benefits of transfusion as well as the logistics of providing adequate and appropriate blood products must be considered. Limited agents are available for mobilization of HSCs into the peripheral blood for collection and the timing of administration is short. Each collection cycle consists of RCE followed by at least three mobilization and apheresis collection events in addition to collecting backup cells. More than one cycle is typically needed to collect enough cells for GT drug product manufacturing. Optimization of patient factors and apheresis instrument parameters leads to better collection yields. Early and frequent ongoing coordination with all stakeholders involved is critical to developing personalized treatment plans for patients.

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Publication Details

Journal
Transfusion
Published
2025-10-19
DOI
https://doi.org/10.1111/trf.18461
Primary Topic
Hemoglobinopathies and Related Disorders
Type
article
Field-Weighted Citation Impact
0.00
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article

How do I overcome apheresis collection challenges for sickle cell disease gene therapy

Yvette C. Tanhehco, Nicole A. Aqui, Jan Hofmann, Abba C. Zubair et al.
Transfusion
Hemoglobinopathies and Related Disorders
article

How do I overcome apheresis collection challenges for sickle cell disease gene therapy

Yvette C. Tanhehco, Nicole A. Aqui, Jan Hofmann, Abba C. Zubair, Patricia A. Shi, Suzanne R. Thibodeaux, Hien D. Liu, Jennifer Schneiderman, Elizabeth A. Godbey, Gaurav K. Gupta, Yanyun Wu, Nirupama Singh
article en

Abstract

Patients must undergo automated red blood cell exchange (RCE) to lower hemoglobin S in a relatively short window of time before apheresis collection of HSCs. The risks and benefits of transfusion as well as the logistics of providing adequate and appropriate blood products must be considered. Limited agents are available for mobilization of HSCs into the peripheral blood for collection and the timing of administration is short. Each collection cycle consists of RCE followed by at least three mobilization and apheresis collection events in addition to collecting backup cells. More than one cycle is typically needed to collect enough cells for GT drug product manufacturing. Optimization of patient factors and apheresis instrument parameters leads to better collection yields. Early and frequent ongoing coordination with all stakeholders involved is critical to developing personalized treatment plans for patients.

TransfusionVol. 65(12)
Northwestern University (US), Lurie Children's Hospital (US), Memorial Sloan Kettering Cancer Center (US), New York Blood Center (US), University of Miami (US), University of California, San Francisco (US), Washington University in St. Louis (US), Columbia University Irving Medical Center (US), Hospital of the University of Pennsylvania (US), Jacksonville College (US), Moffitt Cancer Center (US), University of Alabama at Birmingham (US), Mayo Clinic in Florida (US), Saint Louis University (US)
Openalex Percentile: Top 59%
Hemoglobinopathies and Related Disorders
DOISourceOpenAlex
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