Trial reportHaematologica2018
Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.
Trial report in Haematologica, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02212535 (Open Study of Phase I / II Evaluating Tolerance and Efficacy of Mobilization and Collection of Peripheral Hematopoietic Stem Cells Device After a Single Injection of 0.24mg/kg of Plerixafor in Sickle Cell Patients), which is not on this map. Cited by 55 papers.
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Open Study of Phase I / II Evaluating Tolerance and Efficacy of Mobilization and Collection of Peripheral Hematopoietic Stem Cells Device After a Single Injection of 0.24mg/kg of Plerixafor in Sickle Cell Patients
Who cites it
55 citing papers in PubMed, 108 citations in OpenAlex.
- Clinical outcomes of lentiviral vector gene therapy for sickle cell disease.Blood advances · 2026Trial
- Severe inflammation and lineage skewing are associated with poor engraftment of engineered hematopoietic stem cells in patients with sickle cell disease.Nature communications · 2025Trial
- Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy.Nature communications · 2023Trial
- Motixafortide and G-CSF to mobilize hematopoietic stem cells for autologous transplantation in multiple myeloma: a randomized phase 3 trial.Nature medicine · 2023Trial
- Trial
- Blood Transfusion Practices in Rural Hospitals: Indications, Challenges, and Practical Solutions.Cureus · 2026Review
- Emerging Considerations in Transfusion Medicine: Hematopoietic Stem and Progenitor Cell Collection for Gene Therapy for Sickle Cell Disease and Transfusion-Dependent Thalassemia.Clinics in laboratory medicine · 2026Review
- Challenges and limitations of mobilization and stem cell collection for gene therapy of sickle cell disease.Blood advances · 2025Review
- IFN-I-Mediated Transcriptional Reprogramming Drives Myeloid-skewed Hematopoiesis in Sickle Cell Anemia.Research square · 2025Article
- GroβT rapidly and reliably mobilizes primitive hematopoietic stem and progenitor cells in nonhuman primates.Molecular therapy. Methods & clinical development · 2025Article
- HCK regulates NLRP12-mediated PANoptosis.Proceedings of the National Academy of Sciences of the United States of America · 2025Article
- Viral-based gene therapy clinical trials for immune deficiencies and blood disorders from 2013 until 2023 - an overview.Regenerative therapy · 2025Review
- Emerging Gene Therapies in Sickle Cell Disease: A Comparative Review of Efficacy and Safety Against Standard Treatments.Journal of blood medicine · 2025Review
- Article
- NLRC5 senses NADCell · 2024Article
- Gene therapy for sickle cell disease.Hematology. American Society of Hematology. Education Program · 2023Article
- Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.The Lancet. Haematology · 2023Review
- Article
- Prime editing in hematopoietic stem cells-FromFrontiers in genome editing · 2023Review
- Development of tolerance to chemokine receptor antagonists: current paradigms and the need for further investigation.Frontiers in immunology · 2023Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
21 authors at 6 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sickle cell disease is characterized by chronic anemia and vaso-occlusive crises, which eventually lead to multi-organ damage and premature death. Hematopoietic stem cell transplantation is the only curative treatment but it is limited by toxicity and poor availability of HLA-compatible donors. A gene therapy approach based on the autologous transplantation of lentiviral-corrected hematopoietic stem and progenitor cells was shown to be efficacious in one patient. However, alterations of the bone marrow environment and properties of the red blood cells hamper the harvesting and immunoselection of patients' stem cells from bone marrow. The use of Filgrastim to mobilize large numbers of hematopoietic stem and progenitor cells into the circulation has been associated with severe adverse events in sickle cell patients. Thus, broader application of the gene therapy approach requires the development of alternative mobilization methods. We set up a phase I/II clinical trial whose primary objective was to assess the safety of a single injection of Plerixafor in sickle cell patients undergoing red blood cell exchange to decrease the hemoglobin S level to below 30%. The secondary objective was to measure the efficiency of mobilization and isolation of hematopoietic stem and progenitor cells. No adverse events were observed. Large numbers of CD34
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What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.