ArticleJournal of clinical oncology : official journal of the American Society of Clinical Oncology2023
Clinical and Molecular Determinants of Clonal Evolution in Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria.
Article in Journal of clinical oncology : official journal of the American Society of Clinical Oncology, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 papers, 1 of them a synthesis that pooled it.
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Who cites it
52 citing papers in PubMed, 1 synthesis or guideline pooled it, 75 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
- Efficacy and safety of immunosuppressive therapy versus cyclosporine combined with avatrombopag in older adults with severe aplastic anemia: a multicenter prospective study.Blood cancer journal · 2025Trial
- Article
- Bone marrow failure syndromes across the age spectrum: diagnostic and therapeutic principles.EClinicalMedicine · 2026Review
- 2026 Korean expert recommendations for the use of thrombopoietin receptor agonists in severe aplastic anemia.Blood research · 2026Review
- High-resolution single-cell mapping of clonal hematopoiesis and structural variation in aplastic anemia.Nature genetics · 2026Article
- Post-Transplant Cyclophosphamide Allows Allogeneic Hematopoietic Stem-Cell Transplantation Across Donor Types for Nonmalignant Hematologic Diseases.Journal of hematology · 2026Article
- Redefining Curative Intent in Severe Aplastic Anemia-When Access, Durability, and Biology Finally Align.Transplantation and cellular therapy · 2026Article
- Role of Interleukin-10 Overexpressing Mesenchymal Stem Cells in Promoting Bone Marrow Functional Recovery in Aplastic Anemia Mice.Stem cells international · 2026Article
- Hematopoietic stem cell transplantation is effective in achieving long-term survival for post-aplastic anemia myeloid neoplasms: the EBMT Severe Aplastic Anemia Report.Haematologica · 2026Article
- The Dual Role of RUNX1 in Inflammation-Driven Age-Related Diseases: From Molecular Mechanisms to Clinical Translation.Biomedicines · 2025Review
- Future directions in transplantation for aplastic anemia.Hematology. American Society of Hematology. Education Program · 2025Review
- Insights in bone marrow failure syndromes: take home messages from the 3rd ESH-EBMT-EHA-IPIG translational research conference.Bone marrow transplantation · 2025Article
- Predicting secondary myeloid neoplasms in acquired aplastic anemia using machine learning models.Blood neoplasia · 2025Article
- Up-front alternative donor HCT in severe aplastic anemia: gaps and opportunities to translate evidence into practice.Blood advances · 2025Review
- PNH clones prevalence study in ph-negative myeloproliferative neoplasms: a multicenter Italian study.Annals of hematology · 2025Observational
- Towards graft-Haematologica · 2025Review
- Somatic mutations and the efficacy of immunosuppressive therapy in severe/very severe aplastic anemia and transfusion-dependent nonsevere aplastic anemia.Annals of hematology · 2025Article
- Origins of T-cell-mediated autoimmunity in acquired aplastic anaemia.British journal of haematology · 2025Review
- Hematopoietic stem cell transplantation and immunosuppressive therapy: implications of clonal haematopoiesis.Annals of hematology · 2025Review
Corrections and comments
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Authors and funding
24 authors at 8 institutions in 4 countries.
Funding
Abstract
purposeSecondary myeloid neoplasms (sMNs) remain the most serious long-term complications in patients with aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH). However, sMNs lack specific predictors, dedicated surveillance measures, and early therapeutic interventions. PATIENTS AND
methodsWe studied a multicenter, retrospective cohort of 1,008 patients (median follow-up 8.6 years) with AA and PNH to assess clinical and molecular determinants of clonal evolution.
resultsAlthough none of the patients transplanted upfront (n = 117) developed clonal complications (either sMN or secondary PNH), the 10-year cumulative incidence of sMN in nontransplanted cases was 11.6%. In severe AA, older age at presentation and lack of response to immunosuppressive therapy were independently associated with increased risk of sMN, whereas untreated patients had the highest risk among nonsevere cases. The elapsed time from AA to sMN was 4.5 years. sMN developed in 94 patients. The 5-year overall survival reached 40% and was independently associated with bone marrow blasts at sMN onset. Myelodysplastic syndrome with high-risk phenotypes, del7/7q, and
conclusionThe risk of sMN in AA is associated with disease severity, lack of response to treatment, and patients' age. sMNs display high-risk morphological, karyotypic, and molecular features. The landscape of acquired somatic mutations is complex and incompletely understood and should be considered with caution in medical management.
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