ArticleBone marrow transplantation2022
Survival and late effects of hematopoietic cell transplantation in patients with thalassemia major.
Article in Bone marrow transplantation, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.
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Who cites it
13 citing papers in PubMed.
- Long-term survival rates of thalassemia patients following hematopoietic stem cell transplantation: A systematic review and meta-analysis.Hematology, transfusion and cell therapy · 2026Article
- Outcomes of hematopoietic stem cell transplantation for pediatric patients with transfusion-dependent thalassemia in Thailand.Clinical and experimental pediatrics · 2026Article
- Curative Approach to the Treatment of Beta-Thalassemia and Sickle Cell Disease with Hematopoietic Stem Cell Transplantation.Journal of clinical medicine · 2026Review
- Late renal, liver, endocrine, and cardiac outcomes of bone marrow transplantation in Kurdish children with β-thalassemia major.International journal of hematology · 2026Article
- Fertility and endocrinopathies among adults with β-thalassemia major treated at Dubai thalassemia center.Frontiers in endocrinology · 2026Observational
- Gene Therapy of Beta Hemoglobinopathies.Biomedicines · 2025Review
- Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-versus-Host Disease Survivorship after Hematopoietic Cell Transplantation: Part II. Organ Dysfunction and Immune Reconstitution Considerations for Children with Chronic Graft-versus-Host Disease after Hematopoietic Cell Transplantation.Transplantation and cellular therapy · 2025Article
- Cardiac injury caused by iron overload in thalassemia.Frontiers in pediatrics · 2025Review
- CRISPR/Cas-edited iPSCs and mesenchymal stem cells: a concise review of their potential in thalassemia therapy.Frontiers in cell and developmental biology · 2025Review
- Safety and effectiveness of controlled ovarian stimulation and oocyte retrieval during prepubertal and peripubertal period.Journal of assisted reproduction and genetics · 2024Article
- Article
- Exploring the bone marrow micro environment in thalassemia patients: potential therapeutic alternatives.Frontiers in immunology · 2024Review
- Case report: Curing a rare, unstable hemoglobin variant Hb Bristol-Alesha using haploidentical hematopoietic stem cell transplantation.Frontiers in immunology · 2023Article
Corrections and comments
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In this retrospective study, we evaluated long-term survival and late effects in 137 patients affected by thalassemia major (TM) who received an allogeneic hematopoietic cell transplantation (HCT). Median age at HCT was 10.1 years. After a median follow-up of 30 years, 114 (83.2%) patients are living and 108 (78.8%) are cured. The cumulative incidence of nonrelapse mortality and thalassemia recurrence was 9.5% at 1 year and 10.2% at 39 years respectively. The 39-years cumulative incidence of overall survival and disease-free survival were 81.4% and 74.5%. One hundred twenty-three patients who survived more than 2 years after HCT were evaluated for late effects concerning hematological disorders, iron burden, growth, obesity, diabetes mellitus, thyroid and gonadal function, eye, heart, liver, lung, kidney, gastrointestinal, neurologic and psychiatric system, osteoarticular system, secondary solid cancer (SSC), performance status, and Covid-19 infection. Fertility was preserved in 21 males whose partners delivered 34 neonates and 25 females who delivered 26 neonates. Fifteen cases of SSC were diagnosed for a 39-year cumulative incidence of 16.4%. HCT represents a definitive cure for the majority of TM patients at the price, however, of a non-negligible early and late mortality which in the long run affects survival and disease-free survival.
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