Written from the studies on PubMed, searched on 2026. nov. 11.. Every number below comes from a study listed under Sources.
What people read
Posts online say that stem cell treatment helps with thalassaemia.
What has been studied
- 11In people2 randomised trials
- 3In animals
- 1Only in the lab
What the studies found
Stem cell treatment has been tested in randomised trials in people with thalassaemia.
We found 153 published papers on stem cell treatment and thalassaemia. They were published between 2001 and 2026.
11 of them are studies in people (2 randomised trials), 33 are reviews, 3 are studies in animals and 1 are studies only in the lab.
The most important ones are listed under Sources.
What this means for you: A study in people is not proof that it helps you. Do not stop or change your medicines because of it. Ask your doctor before you start taking it.
Sources
- Comparing two different doses of anti-thymocyte globulin in allogeneic hematopoietic stem cell transplantation for transfusion-dependent thalassemia: an open-label, randomized clinical trial.2026 · Ther Adv Hematol · Randomised trial
- Late-onset hemorrhagic cystitis in children after hematopoietic stem cell transplantation for thalassemia and sickle cell anemia: a prospective evaluation of polyoma (BK) virus infection and treatment with cidofovir.2010 · Biol Blood Marrow Transplant · Randomised trial
- Haploidentical haematopoietic stem cell transplantation for thalassaemia major: A phase IV, open-label, single-centre trial.2026 · Br J Haematol · Study in people
- Impact of splenectomy on outcomes of hematopoietic stem cell transplantation in pediatric patients with transfusion-dependent thalassemia.2020 · Pediatr Blood Cancer · Study in people
- Haploidentical haematopoietic stem cell transplantation for thalassaemia major based on an FBCA conditioning regimen.2018 · Br J Haematol · Study in people
- Influence of glutathione S-transferase gene polymorphisms on busulfan pharmacokinetics and outcome of hematopoietic stem-cell transplantation in thalassemia pediatric patients.2016 · Bone Marrow Transplant · Study in people
- French multicenter 22-year experience in stem cell transplantation for beta-thalassemia major: lessons and future directions.2013 · Biol Blood Marrow Transplant · Study in people
- Quality of life assessment in hematopoietic stem cell transplantation performed on thalassemia major patients.2012 · Pediatr Hematol Oncol · Study in people
- Hematopoietic stem cell mobilization for gene therapy of adult patients with severe β-thalassemia: results of clinical trials using G-CSF or plerixafor in splenectomized and nonsplenectomized subjects.2012 · Mol Ther · Study in people
- Novel pharmacokinetic behavior of intravenous busulfan in children with thalassemia undergoing hematopoietic stem cell transplantation: a prospective evaluation of pharmacokinetic and pharmacodynamic profile with therapeutic drug monitoring.2010 · Blood · Study in people
- Long-term survival rates of thalassemia patients following hematopoietic stem cell transplantation: a systematic review and meta-analysis.2026 · Hematol Transfus Cell Ther · Review
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.2026 · J Clin Med · Review
- Haploidentical Hematopoietic Stem Cell Transplantation in Pediatric Transfusion-Dependent Thalassemia: A Systematic Review and Meta-Analysis.2025 · Transplant Cell Ther · Review
- Immediate hypersensitivity reactions to parenteral cyclosporine in patients with thalassemia major undergoing hematopoietic stem cell transplantation: a case report and review of the literature.2024 · J Med Case Rep · Review
- Systematic review of hematopoietic stem cell gene therapy approach in thalassemia: Comparative analysis in animal models.2024 · Eur J Haematol · Review
- Bone Marrow Quality Index: A Predictor of Acute Graft-versus-Host Disease in Hematopoietic Stem Cell Transplantation for Thalassemia.2023 · Transplant Cell Ther · Review
- CD117 epitope-shielded hematopoietic stem cell transplantation with toxin-free conditioning and in vivo selection ameliorates a β-thalassemia model.2026 · bioRxiv · In animals
- Therapeutic Effects of Hematopoietic Stem Cell Derived From Gene-Edited Mice on β654-Thalassemia.2024 · Stem Cells · In animals
- Curative in vivo hematopoietic stem cell gene therapy of murine thalassemia using large regulatory elements.2020 · JCI Insight · In animals
- Factor-induced Reprogramming and Zinc Finger Nuclease-aided Gene Targeting Cause Different Genome Instability in β-Thalassemia Induced Pluripotent Stem Cells (iPSCs).2015 · J Biol Chem · In the lab
- Child With β-Thalassemia Major Born Following Hematopoietic Stem Cell Transplantation in the Mother: A Case Supporting the Expansion of Genetic Counseling Services in Lebanon and the Middle East.2026 · J Genet Couns · Other paper
- Successful Management of Severe Hepatic Acute Graft-Versus-Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation in a Child With β-Thalassemia Major: Clinical Lessons From Early Therapeutic Escalation.2026 · Clin Case Rep · Other paper
- BK virus-associated haemorrhagic cystitis following allogeneic haematopoietic stem cell transplantation in a paediatric patient with β-thalassaemia major: A case report.2026 · Indian J Med Microbiol · Other paper
- Hematopoietic Stem Cell Transplantation in Children With Transfusion Dependent β-Thalassemia: A Two-Decade Single-Center Experience.2026 · Am J Hematol · Other paper
