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Mean Corpuscular Hemoglobin Modulates HbF /F‐Cell and Clinical Response to Gene Therapy and Hydroxyurea in Sickle Cell Disease

28 May 2025

Abstract excerpt

We hypothesized that mean corpuscular hemoglobin (MCH) modulates the distribution of fetal hemoglobin (HbF) among F-cells in sickle cell disease, and mathematically modeled a hypothetical HbF distribution among F-cells following gene therapy or during highly successful hydroxyurea (HU) treatment. HbF, the prime genetic modulator of the phenotype of sickle cell disease, is largely excluded from the sickle...

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PMID: 40434205
DOI: 10.1002/ajh.27729

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