ArticleLeukemia2021
Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia.
Article in Leukemia, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 46 papers, 1 of them a synthesis that pooled it.
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Who cites it
46 citing papers in PubMed, 1 synthesis or guideline pooled it, 67 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
- Phase II study of the triple combination of rabbit ATG, ciclosporin and eltrombopag in patients with transfusion-dependent aplastic anaemia: West Japan Hematology Study Group (W-JHS) AA02 trial.British journal of haematology · 2026Trial
- Romiplostim with ciclosporin A in patients with aplastic anaemia naïve to immunosuppressive therapy: A phase 2/3 study.British journal of haematology · 2025Trial
- Allogeneic hematopoietic cell transplantation for myelodysplastic syndromes harboring a paroxysmal nocturnal hemoglobinuria clone-a study on behalf of the Chronic Malignancies and Severe Aplastic Anemia Working Parties of the EBMT.Bone marrow transplantation · 2026Article
- Imputing causality and clonal dynamics from single-cell transcriptomics in paroxysmal nocturnal hemoglobinuria.Leukemia · 2026Article
- Immunological links between aplastic anemia and paroxysmal nocturnal hemoglobinuria.International journal of hematology · 2026Review
- Prognostic significance of PNH clones in aplastic anemia treated with immunosuppression or allogeneic HSCT: a 20-year single-center experience.Blood research · 2026Article
- The PNH French Working Group Experience: Building a Strong Network of Cytometrists.International journal of laboratory hematology · 2026Review
- Aplastic anaemia with small paroxysmal nocturnal haemoglobinuria clones developing during osimertinib therapy for non-small cell lung cancer.Leukemia research reports · 2026Article
- Systemic lupus erythematosus associated with paroxysmal nocturnal hemoglobinuria: a case report and literature review highlighting the clinical significance of small PNH clones.Frontiers in immunology · 2026Review
- Unprovoked Venous Thromboembolism in a Patient with a Small PNH Clone: A Therapeutic Dilemma.Journal of Brown hospital medicine · 2026Article
- Recursive Partitioning to Differentiate Acquired From Inherited Bone Marrow Failure Syndromes.American journal of hematology · 2025Article
- Diffuse Large B-Cell Lymphoma Combined With Paroxysmal Nocturnal Hemoglobinuria: A Rare Case Report.Clinical case reports · 2025Article
- Correlation of clone size with clinical and lab presentation in Paroxysmal nocturnal hemoglobinuria (PNH): A single center experience.Pakistan journal of medical sciences · 2025Article
- Comparison of severe aplastic anaemia and lower risk hypoplastic myelodysplastic neoplasms: Critical role of megakaryocyte count in distinguishing aplastic anaemia from myelodysplastic neoplasms.British journal of haematology · 2025Article
- Molecular classification and outcomes in pediatric aplastic anemia with myeloid neoplasm-associated gene variants.Frontiers in pediatrics · 2025Article
- Monitoring and Treatment of Paroxysmal Nocturnal Hemoglobinuria in Patients with Aplastic Anemia in Asia: An Expert Consensus.International journal of molecular sciences · 2024Review
- Modified Delphi panel consensus recommendations for management of severe aplastic anemia.Blood advances · 2024Article
- Deciphering Paroxysmal Nocturnal Hemoglobinuria: An Unusual Paradigm of Hemolytic Anemia.Cureus · 2024Article
- Diagnostic landscape of first-time cytometric screening for paroxysmal nocturnal hemoglobinuria in Poland in 2013-2022.Orphanet journal of rare diseases · 2024Article
Corrections and comments
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Authors and funding
15 authors at 4 institutions in 2 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
In this large single-centre study, we report high prevalence (25%) of, small (<10%) and very small (<1%), paroxysmal nocturnal hemoglobinuria (PNH) clones by high-sensitive cytometry among 3085 patients tested. Given PNH association with bone marrow failures, we analyzed 869 myelodysplastic syndromes (MDS) and 531 aplastic anemia (AA) within the cohort. PNH clones were more frequent and larger in AA vs. MDS (p = 0.04). PNH clone, irrespective of size, was a good predictor of response to immunosuppressive therapy (IST) and to stem cell transplant (HSCT) (in MDS: 84% if PNH+ vs. 44.7% if PNH-, p = 0.01 for IST, and 71% if PNH+ vs. 56.6% if PNH- for HSCT; in AA: 78 vs. 50% for IST, p < 0.0001, and 97 vs. 77%, p = 0.01 for HSCT). PNH positivity had a favorable impact on disease progression (0.6% vs. 4.9% IPSS-progression in MDS, p < 0.005; and 2.1 vs. 6.9% progression to MDS in AA, p = 0.01), leukemic evolution (6.8 vs. 12.7%, p = 0.01 in MDS), and overall survival [73% (95% CI 68-77) vs. 51% (48-54), p < 0.0001], with a relative HR for mortality of 2.37 (95% CI 1.8-3.1; p < 0.0001) in PNH negative cases, both in univariate and multivariable analysis. Our data suggest systematic PNH testing in AA/MDS, as it might allow better prediction/prognostication and consequent clinical/laboratory follow-up timing.
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