ArticleBlood advances2017
Cyclophosphamide improves engraftment in patients with SCD and severe organ damage who undergo haploidentical PBSCT.
Article in Blood advances, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT00977691 (Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-Thalassemia), which is not on this map. Cited by 56 papers, 1 of them a synthesis that pooled it.
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.
Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Severe Congenital Anemias Including Sickle Cell Disease and Beta-Thalassemia
Who cites it
56 citing papers in PubMed, 1 synthesis or guideline pooled it, 104 citations in OpenAlex.
- American Society of Hematology 2021 guidelines for sickle cell disease: stem cell transplantation.Blood advances · 2021Guideline
- Trial
- Trial
- Effect of increased dose of total body irradiation on graft failure associated with HLA-haploidentical transplantation in patients with severe haemoglobinopathies: a prospective clinical trial.The Lancet. Haematology · 2019Trial
- Bone marrow transplantation for adolescents and young adults with sickle cell disease: Results of a prospective multicenter pilot study.American journal of hematology · 2019Trial
- Clonal hematopoiesis in the setting of sickle cell disease and its relevance to curative therapies.Seminars in hematology · 2026Article
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- Comparative analysis of CRISPR-Cas9, lentiviral transduction, and base editing for sickle cell disease in a murine model.Blood advances · 2026Article
- Nonmyeloablative Pentostatin-Cyclophosphamide Preconditioning Improves Rates of Engraftment in Adults Undergoing Haploidentical HCT for Sickle Cell Disease.medRxiv : the preprint server for health sciences · 2025Article
- Current and future treatments for sickle cell disease: From hematopoietic stem cell transplantation to in vivo gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Lipid Levels Increase to the Normal Range After Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease.Transplantation and cellular therapy · 2025Article
- Late effects of hemopoietic stem cell transplant for sickle cell disease: monitoring and management.Expert review of hematology · 2024Review
- Low dose post-transplant cyclophosphamide and sirolimus induce mixed chimerism with CTLA4-Ig or lymphocyte depletion in an MHC-mismatched murine allotransplantation model.Bone marrow transplantation · 2024Article
- Galectin-1 is associated with hematopoietic cell engraftment in murine MHC-mismatched allotransplantation.Frontiers in immunology · 2024Article
- Post-transcriptional (re)programming of B lymphocyte development: From bench to bedside?Advances in immunology · 2024Review
- The range of haploidentical transplant protocols in sickle cell disease: all haplos are not created equally.Hematology. American Society of Hematology. Education Program · 2023Article
- Excellent outcome of stem cell transplantation for sickle cell disease.Annals of hematology · 2023Article
- Defining global strategies to improve outcomes in sickle cell disease: a Lancet Haematology Commission.The Lancet. Haematology · 2023Review
- Incidence, risk factors, and impact of early cardiac toxicity after allogeneic hematopoietic cell transplant.Blood advances · 2023Article
- Outcomes following posttransplant virus-specific T-cell therapy in patients with sickle cell disease.Blood advances · 2023Article
Corrections and comments
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Authors and funding
12 authors at 5 institutions in 1 country.
Funding
Abstract
Peripheral blood stem cell transplantation (PBSCT) offers a curative option for sickle cell disease (SCD). Although HLA-matched sibling transplantation is promising, the vast majority of patients lack such a donor. We sought to develop a novel nonmyeloablative HLA-haploidentical PBSCT approach that could safely be used for patients with severe organ damage. Based on findings in our preclinical model, we developed a phase 1/2 trial using alemtuzumab, 400 cGy total body irradiation, and escalating doses of posttransplant cyclophosphamide (PT-Cy): 0 mg/kg in cohort 1, 50 mg/kg in cohort 2, and 100 mg/kg in cohort 3. A total of 21 patients with SCD and 2 with β-thalassemia received a transplant. The mean hematopoietic cell transplant-specific comorbidity index of 6 reflected patients with cirrhosis, heart failure, and end-stage renal disease. The engraftment rate improved from 1 (33%) of 3 in cohort 1 to 5 (63%) of 8 in cohort 2 and 10 (83%) of 12 in cohort 3. Percentage of donor myeloid and CD3 chimerism also improved with subsequent cohorts. There was no transplant-related mortality, and overall survival was 87%. At present, 0% in cohort 1, 25% in cohort 2, and 50% in cohort 3 remain free of their disease. There was no grade 2 to 4 acute or extensive chronic graft-versus-host disease (GVHD). Therefore, PT-Cy improves engraftment and successfully prevents severe GVHD after nonmyeloablative conditioning in patients with SCD who are at high risk for early mortality. Additional strategies are necessary to decrease the graft rejection rate and achieve a widely available cure for all patients with SCD. This trial was registered at www.clinicaltrials.gov as #NCT00977691.
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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.