ArticleBlood2024
How I treat sickle cell disease with gene therapy.
Article in Blood, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
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
18 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy.Transplantation and cellular therapy · 2026Guideline
- Blood rheology after allogeneic hematopoietic stem cell transplantation or gene therapy in sickle cell disease.Blood advances · 2026Article
- Alternative conditioning regimens for hemoglobinopathy gene therapy: balancing efficacy, toxicity, and the next frontier.Blood advances · 2026Review
- Article
- Premature senescence impairs hematopoietic stem cell function during sickle cell disease in mice and humans.Science translational medicine · 2026Article
- Article
- Isovolemic hemodilution red cell exchange and blood use in children and young adults with sickle cell disease.Blood. Red cells & iron · 2026Article
- G-CSF for Mobilizing CD34American journal of hematology · 2026Article
- The Journey of Gene Therapy in Sickle Cell Disease: How Molecular Advances Meet Clinical Care.Cells · 2026Review
- Discontinued therapies for sickle cell disease: status and future directions.Expert opinion on investigational drugs · 2026Review
- Transformative therapies for sickle cell disease.CMAJ : Canadian Medical Association journal = journal de l'Association medicale canadienne · 2026Article
- The right patient, the right treatment: tailoring sickle cell disease care.Hematology. American Society of Hematology. Education Program · 2025Review
- Strategies to mitigate transfusion-associated red blood cell alloimmunization in sickle cell disease: A retrospective analysis.Transfusion · 2025Article
- Gene Therapy for Sickle Cell Disease: No Donor, No Problem.Clinical journal of oncology nursing · 2025Article
- To pursue gene therapy or not? Is it feasible after graft failure in allogeneic hematopoietic cell transplant recipients.Blood advances · 2025Review
- The wider perspective: Barriers and recommendations for transfusion support for patients with sickle cell disease in low- and middle-income countries.British journal of haematology · 2025Review
- Beyond the traditional oncology patient: the role of palliative care in patients with sickle cell disease receiving stem cell transplantation or gene therapy.Frontiers in oncology · 2025Article
- The Current Role of Hydroxyurea in the Treatment of Sickle Cell Anemia.Journal of clinical medicine · 2024Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
1 author.
Funding
Abstract
abstractIn 2023, 2 different gene therapies were approved for individuals with severe sickle cell disease (SCD). The small number of patients treated on the pivotal clinical trials that led to these approvals have experienced dramatic short-term reductions in the occurrence of painful vaso-occlusive crises, but the long-term safety and efficacy of these genetic therapies are yet to be ascertained. Several challenges and treatment-related concerns have emerged in regard to administering these therapies in clinical practice. This article discusses the selection and preparation of individuals with SCD who wish to receive autologous gene therapy, as well as the salient features of the care needed to support them through a long and arduous treatment process. I specifically focus on postinfusion care, as it relates to immune monitoring and infection prevention. Compared with allogeneic hematopoietic cell transplantation, delivering autologous gene therapy to an individual with SCD has distinct nuances that require awareness and special interventions. Using clinical vignettes derived from real-life patients, I provide perspectives on the complex decision-making process for gene therapy for SCD based on currently available data and make recommendations for evaluating and supporting these patients.
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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.