ReviewBiology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation2016
Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease.
Review in Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 56 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.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
56 citing papers in PubMed, 123 citations in OpenAlex.
- Clinical outcomes of lentiviral vector gene therapy for sickle cell disease.Blood advances · 2026Trial
- Improvements in health-related quality of life in patients with severe sickle cell disease after exagamglogene autotemcel.Blood advances · 2025Trial
- Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.American journal of hematology · 2019Trial
- Pain and opioid use after reversal of sickle cell disease following HLA-matched sibling haematopoietic stem cell transplant.British journal of haematology · 2019Trial
- Plerixafor enables safe, rapid, efficient mobilization of hematopoietic stem cells in sickle cell disease patients after exchange transfusion.Haematologica · 2018Trial
- Systemic delivery of phagocytosis-shielded retroviral vectors enables in vivo HSC gene therapy for sickle cell disease.Cell stem cell · 2026Article
- MeMAGEN: A Phase IIa/IIb open-label trial of memantine testing safety and tolerability in sickle cell patients.HemaSphere · 2026Article
- Sickle Cell Disease: Treatment Options of the Past, Present, and Future.The American journal of nursing · 2026Review
- Review
- Article
- Clinical Outcomes Among Patients With Sickle Cell Disease and Transfusion-Dependent Beta-Thalassemia Treated With Allogeneic Hematopoietic Stem Cell Transplantation: A Systematic Literature Review.Journal of blood medicine · 2025Review
- Late effects of hemopoietic stem cell transplant for sickle cell disease: monitoring and management.Expert review of hematology · 2024Review
- The Current Role of Hydroxyurea in the Treatment of Sickle Cell Anemia.Journal of clinical medicine · 2024Review
- Exploring non-viral methods for the delivery of CRISPR-Cas ribonucleoprotein to hematopoietic stem cells.Stem cell research & therapy · 2024Review
- Improvement in Cardiac Morphology Demonstrated by Cardiac Magnetic Resonance Imaging and Echocardiography after Haploidentical Hematopoietic Cell Transplantation in Adults with Sickle Cell Disease.Transplantation and cellular therapy · 2024Article
- LentiGlobin Administration to Sickle Cell Disease Patients: Effect on Serum Markers and Vaso-Occlusive Crisis.Cureus · 2024Review
- Preferences for potential benefits and risks for gene therapy in the treatment of sickle cell disease.Blood advances · 2023Article
- Hope on the Horizon: New and Future Therapies for Sickle Cell Disease.Journal of clinical medicine · 2023Review
- Enrollment Lessons from a Biological Assignment Study of Marrow Transplantation versus Standard Care for Adolescents and Young Adults with Sickle Cell Disease: Considerations for Future Gene and Cellular Therapy Trials.Transplantation and cellular therapy · 2023Article
- Lovo-cel gene therapy for sickle cell disease: Treatment process evolution and outcomes in the initial groups of the HGB-206 study.American journal of hematology · 2023Article
Corrections and comments
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Authors and funding
11 authors at 11 institutions in 2 countries.
Funding
Abstract
Although a number of published trials exist of HLA-identical sibling hematopoietic cell transplantation (HCT) for sickle cell disease (SCD) that span 2 decades, when and for whom this therapy should be pursued is a subject of debate. Assessments of the risks of transplant-related complications that include infertility and debilitating graft-versus-host disease and long-term quality of life after successful HCT are difficult to perform without prospective trials in transplant and nontransplant cohorts. However, it is possible to assess the risk of mortality and to compare published rates of survival in individuals with SCD treated and not treated by HCT. In this brief review, projections about mortality risk based on recent published reports are reviewed and summarized. The published data show overall survival and event-free survival rates of 95% and 92%, respectively, in children treated by HLA-identical sibling HCT. The overall survival rates in the Center for International Blood and Marrow Transplant Research (N = 412) and European Blood and Marrow Transplant (N = 487) registries were 91% and 95%, respectively. These results provide broad support for the therapeutic value of HLA-identical sibling HCT for children with SCD and serve as the basis for a strong recommendation in favor of the option of HCT when a suitable donor is available. The experience of HLA-identical sibling HCT in adults with SCD is limited but appears to be similar to results in children. These preliminary observations, however, warrant further investigation.
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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.