ArticleBlood2020
Risk score to predict event-free survival after hematopoietic cell transplant for sickle cell disease.
Article in Blood, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 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.
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Who cites it
26 citing papers in PubMed, 48 citations in OpenAlex.
- Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease.Blood advances · 2025Trial
- Hematopoietic Cell Transplantation for Sickle Cell Disease.Children (Basel, Switzerland) · 2026Review
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- Rules, risks, and reality: rethinking gene therapy eligibility.Blood advances · 2026Article
- Article
- Article
- Hematopoietic Stem Cell Transplantation in Severe Pediatric Sickle Cell Disease: Outcome and long-term complications, Saudi experience at King Faisal Specialist Hospital, Riyadh, Saudi Arabia.Mediterranean journal of hematology and infectious diseases · 2025Article
- Article
- CRISPR/Cas9 in the treatment of sickle cell disease (SCD) and its comparison with traditional treatment approaches: a review.Annals of medicine and surgery (2012) · 2024Review
- The effectiveness of hematopoietic stem cell transplantation in treating pediatric sickle cell disease: Systematic review and meta-analysis.Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society · 2024Review
- Long-term outcomes after unrelated donor transplantation for severe sickle cell disease on the BMT CTN 0601 trial.American journal of hematology · 2024Article
- Gene therapies for sickle cell disease: Effectiveness and value.Journal of managed care & specialty pharmacy · 2023Article
- Genetic reversal of the globin switch concurrently modulates both fetal and sickle hemoglobin and reduces red cell sickling.Nature communications · 2023Article
- 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
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- Allogeneic Transplant and Gene Therapy: Evolving Toward a Cure.Hematology/oncology clinics of North America · 2022Review
- Is Severity Score Associated With Indication for Hematopoietic Stem Cell Transplantation in Individuals With Sickle Cell Anemia?Transplantation and cellular therapy · 2022Observational
- Evidence-Based Minireview: In young children with severe sickle cell disease, do the benefits of HLA-identical sibling donor HCT outweigh the risks?Hematology. American Society of Hematology. Education Program · 2021Review
- Hematopoietic cell transplantation for sickle cell disease: updates and future directions.Hematology. American Society of Hematology. Education Program · 2021Article
- Matched related hematopoietic cell transplant for sickle cell disease with alemtuzumab: the Texas Children's Hospital experience.Bone marrow transplantation · 2021Article
Corrections and comments
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Authors and funding
17 authors at 13 institutions in 5 countries.
Funding
Abstract
We developed a risk score to predict event-free survival (EFS) after allogeneic hematopoietic cell transplantation for sickle cell disease. The study population (n = 1425) was randomly split into training (n = 1070) and validation (n = 355) cohorts. Risk factors were identified and validated via Cox regression models. Two risk factors of 9 evaluated were predictive for EFS: age at transplantation and donor type. On the basis of the training cohort, patients age 12 years or younger with an HLA-matched sibling donor were at the lowest risk with a 3-year EFS of 92% (score, 0). Patients age 13 years or older with an HLA-matched sibling donor or age 12 years or younger with an HLA-matched unrelated donor were at intermediate risk (3-year EFS, 87%; score, 1). All other groups, including patients of any age with a haploidentical relative or HLA-mismatched unrelated donor and patients age 13 years or older with an HLA-matched unrelated donor were high risk (3-year EFS, 57%; score, 2 or 3). These findings were confirmed in the validation cohort. This simple risk score may guide patients with sickle cell disease and hematologists who are considering allogeneic transplantation as a curative treatment relative to other available contemporary treatments.
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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.