Publications by authors named "H Frangoul"
CRISPR-based gene therapy for the induction of fetal hemoglobin in sickle cell disease.
Expert Rev Hematol
December 2024
Introduction: Sickle cell disease is ameliorated and perhaps can be 'cured' if enough fetal hemoglobin is present in most erythrocytes. Hydroxyurea, which increases fetal hemoglobin levels, is widely available and effective, especially in children. Nevertheless, only cell-based gene therapy can achieve a 'curative' fetal hemoglobin threshold.
View Article and Find Full Text PDFImpact of Race and Ethnicity on Outcomes After Umbilical Cord Blood Transplantation.
Transplant Cell Ther
October 2024
Article Synopsis
- - The study investigates the survival outcomes of patients receiving umbilical cord blood transplants (UCBT) across different racial and ethnic groups, focusing on Black, Latinx, White, and Asian patients, as previous research indicated disparities in survival rates.
- - A retrospective analysis of data from the Center for International Blood and Marrow Transplant Research (CIBMTR) included 983 single and 1529 double UCBT recipients, measuring outcomes like overall survival (OS), disease-free survival, and transplant-related mortality over two years.
- - Results showed that while overall survival rates varied by race/ethnicity, with Latinx patients having significantly lower OS compared to Blacks, no significant differences were observed in child patients,
Article Synopsis
- Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy utilizing CRISPR-Cas9 gene editing to increase fetal hemoglobin production in patients with sickle cell disease.
- A phase 3 study involved 44 patients aged 12 to 35 with a history of severe vaso-occlusive crises; patients received edited stem cells after myeloablative conditioning.
- Results showed 97% of patients were free from vaso-occlusive crises and 100% avoided hospitalization for over 12 months, with a safety profile similar to standard treatments.
Article Synopsis
- Exagamglogene autotemcel (exa-cel) is a novel cell therapy using CRISPR-Cas9 gene editing to boost fetal hemoglobin production in patients with transfusion-dependent β-thalassemia.
- In a phase 3 study, 52 patients aged 12 to 35 underwent treatment with exa-cel after myeloablative conditioning, and 91% achieved transfusion independence for at least 12 months.
- The therapy showed promising results with high mean total and fetal hemoglobin levels, a favorable safety profile, and no serious adverse events like deaths or cancers reported during the follow-up period.