ArticleHaematologica2020
Extensive multilineage analysis in patients with mixed chimerism after allogeneic transplantation for sickle cell disease: insight into hematopoiesis and engraftment thresholds for gene therapy.
Article in Haematologica, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed, 35 citations in OpenAlex.
- Nonmyeloablative HLA-identical sibling transplant for sickle cell disease in children: a multicenter prospective study.Blood advances · 2026Trial
- Trial
- Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in theMolecular therapy. Nucleic acids · 2026Article
- Case Report: Recurrence of recipient-derived erythropoiesis in hematopoietic stem cell transplant for sickle-cell anemia: multi-lineage chimerism analysis as a tool to distinguish atypical pure red cell aplasia from poor erythroid engraftment.Frontiers in immunology · 2026Article
- Article
- Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease.Cell reports. Medicine · 2025Article
- Two Nonmyeloablative HLA-Matched Related Donor Allogeneic Hematopoietic Cell Transplantation Regimens in Patients with Severe Sickle Cell Disease.Transplantation and cellular therapy · 2025Article
- Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses.Molecular therapy : the journal of the American Society of Gene Therapy · 2024Article
- Late effects of hemopoietic stem cell transplant for sickle cell disease: monitoring and management.Expert review of hematology · 2024Review
- Non-viral DNA delivery and TALEN editing correct the sickle cell mutation in hematopoietic stem cells.Nature communications · 2024Article
- Gene correction for sickle cell disease hits its prime.Nature biomedical engineering · 2023Article
- Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression.Nature communications · 2022Article
- Across the Myeloablative Spectrum: Hematopoietic Cell Transplant Conditioning Regimens for Pediatric Patients with Sickle Cell Disease.Journal of clinical medicine · 2022Review
- Unresolved issues in allogeneic hematopoietic cell transplantation for non-malignant diseases.International journal of hematology · 2022Review
- Mobilization-based chemotherapy-free engraftment of gene-edited human hematopoietic stem cells.Cell · 2022Article
- Matched related hematopoietic cell transplant for sickle cell disease with alemtuzumab: the Texas Children's Hospital experience.Bone marrow transplantation · 2021Article
- The Mandible Ameliorates Facial Allograft Rejection and Is Associated with the Development of Regulatory T Cells and Mixed Chimerism.International journal of molecular sciences · 2021Article
- Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.Science translational medicine · 2021Article
- Parameters affecting successful stem cell collections for genetic therapies in sickle cell disease.Transfusion and apheresis science : official journal of the World Apheresis Association : official journal of the European Society for Haemapheresis · 2021Review
- Cas9-AAV6 gene correction of beta-globin in autologous HSCs improves sickle cell disease erythropoiesis in mice.Nature communications · 2021Article
Corrections and comments
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Authors and funding
20 authors at 12 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Although studies of mixed chimerism following hematopoietic stem cell transplantation in patients with sickle cell disease (SCD) may provide insights into the engraftment needed to correct the disease and into immunological reconstitution, an extensive multilineage analysis is lacking. We analyzed chimerism simultaneously in peripheral erythroid and granulomonocytic precursors/progenitors, highly purified B and T lymphocytes, monocytes, granulocytes and red blood cells (RBC). Thirty-four patients with mixed chimerism and ≥12 months of follow-up were included. A selective advantage of donor RBC and their progenitors/precursors led to full chimerism in mature RBC (despite partial engraftment of other lineages), and resulted in the clinical control of the disease. Six patients with donor chimerism <50% had hemolysis (reticulocytosis) and higher HbS than their donor. Four of them had donor chimerism <30%, including a patient with AA donor (hemoglobin >10 g/dL) and three with AS donors (hemoglobin <10 g/dL). However, only one vaso-occlusive crisis occurred with 68.7% HbS. Except in the patients with the lowest chimerism, the donor engraftment was lower for T cells than for the other lineages. In a context of mixed chimerism after hematopoietic stem cell transplantation for SCD, myeloid (rather than T cell) engraftment was the key efficacy criterion. Results show that myeloid chimerism as low as 30% was sufficient to prevent a vaso-occlusive crisis in transplants from an AA donor but not constantly from an AS donor. However, the correction of hemolysis requires higher donor chimerism levels (
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