ArticleHaematologica2014
First-line treatment for severe aplastic anemia in children: bone marrow transplantation from a matched family donor versus immunosuppressive therapy.
Article in Haematologica, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 71 papers, 4 of them syntheses that pooled it.
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Who cites it
71 citing papers in PubMed, 4 syntheses or guidelines pooled it, 128 citations in OpenAlex.
- American Society of Hematology 2026 guidelines for the diagnosis and management of severe and very severe acquired aplastic anemia.Blood advances · 2026Guideline
- Long-Term Results of Hematopoietic Cell Transplantation (HCT) for Adults with Blood Disorders- A Systematic Review.Stem cell reviews and reports · 2025Pooled it
- Efficacy of Eltrombopag with Immunosuppressive Therapy Versus Immunosuppressive Therapy Alone on Severe Aplastic Anaemia: A Systematic Review and Meta-analysis.Clinical drug investigation · 2023Pooled it
- Allo-HSCT compared with immunosuppressive therapy for acquired aplastic anemia: a system review and meta-analysis.BMC immunology · 2020Pooled it
- Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: phase 2 ESCALATE trial.Blood advances · 2025Trial
- Efficacy of combined immunosuppression with or without eltrombopag in children with newly diagnosed aplastic anemia.Blood advances · 2023Trial
- Eltrombopag added to immunosuppression for children with treatment-naïve severe aplastic anaemia.British journal of haematology · 2021Trial
- Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study.Haematologica · 2019Trial
- Haploidentical transplantation for pediatric patients with acquired severe aplastic anemia.Bone marrow transplantation · 2017Trial
- Immunosuppressive therapy versus alternative donor hematopoietic stem cell transplantation for children with severe aplastic anemia who lack an HLA-matched familial donor.Bone marrow transplantation · 2017Trial
- Reconceptualizing Aplastic Anemia-Seed, Worm, Soil.Journal of clinical laboratory analysis · 2026Review
- Cost-effectiveness of frontline eltrombopag with immunosuppressive therapy for severe aplastic anemia.Blood red cells & iron · 2026Article
- Comparable outcomes of HSCT up-front and after failure of IST in pediatric aplastic anemia in recent years.Blood advances · 2026Article
- Machine learning mortality prediction model for cyclosporine therapy in pediatric aplastic anemia.Annals of hematology · 2026Article
- Successful Haploidentical Hematopoietic Stem Cell Transplantation for Chemotherapy-Induced Aplastic Anemia in a Breast Cancer Survivor: A Case Report and Literature Review.Case reports in hematology · 2026Article
- Real-world retrospective analysis of clinical outcomes in pediatric acquired aplastic anemia: a single-center 10-year cohort study.Frontiers in pediatrics · 2026Article
- Future directions in transplantation for aplastic anemia.Hematology. American Society of Hematology. Education Program · 2025Review
- Vaginoscopy approach with Mechanical Tissue Removal System for AUB inPediatric Aplastic Anemia: a case report.BMC surgery · 2025Article
- Eltrombopag combined with immunosuppressive therapy for pediatric severe aplastic anemia.Pediatric research · 2025Article
- Adjunctive effects of eltrombopag on immunosuppressive therapy for childhood aplastic anemia.International journal of hematology · 2025Article
11 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
15 authors at 11 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
The current treatment approach for severe aplastic anemia in children is based on studies performed in the 1980s, and updated evidence is required. We retrospectively compared the outcomes of children with acquired severe aplastic anemia who received immunosuppressive therapy within prospective trials conducted by the Japanese Childhood Aplastic Anemia Study Group or who underwent bone marrow transplantation from an HLA-matched family donor registered in the Japanese Society for Hematopoietic Cell Transplantation Registry. Between 1992 and 2009, 599 children (younger than 17 years) with severe aplastic anemia received a bone marrow transplant from an HLA-matched family donor (n=213) or immunosuppressive therapy (n=386) as first-line treatment. While the overall survival did not differ between patients treated with immunosuppressive therapy or bone marrow transplantation [88% (95% confidence interval: 86-90) versus 92% (90-94)], failure-free survival was significantly inferior in patients receiving immunosuppressive therapy than in those undergoing bone marrow transplantation [56% (54-59) versus 87% (85-90); P<0.0001]. There was no significant improvement in outcomes over the two time periods (1992-1999 versus 2000-2009). In multivariate analysis, age <10 years was identified as a favorable factor for overall survival (P=0.007), and choice of first-line immunosuppressive therapy was the only unfavorable factor for failure-free survival (P<0.0001). These support the current algorithm for treatment decisions, which recommends bone marrow transplantation when an HLA-matched family donor is available in pediatric severe aplastic anemia.
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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.