Clinical and prognostic significance of small paroxysmal nocturnal hemoglobinuria clones in myelodysplastic syndrome and aplastic anemia
Open Access
- 4 March 2021
- journal article
- research article
- Published by Springer Science and Business Media LLC in Leukemia
- Vol. 35 (11), 3223-3231
- https://doi.org/10.1038/s41375-021-01190-9
Abstract
In this large single-centre study, we report high prevalence (25%) of, small (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.Keywords
This publication has 22 references indexed in Scilit:
- Multiparameter FLAER-based flow cytometry for screening of paroxysmal nocturnal hemoglobinuria enhances detection rates in patients with aplastic anemiaAnnals of Hematology, 2014
- Prognostic value of paroxysmal nocturnal haemoglobinuria clone presence in aplastic anaemia patients treated with combined immunosuppression: results of two‐centre prospective studyBritish Journal of Haematology, 2013
- A prospective multicenter study of paroxysmal nocturnal hemoglobinuria cells in patients with bone marrow failureCytometry Part B: Clinical Cytometry, 2013
- A Prospective Multicenter Study of Paroxysmal Nocturnal Hemoglobinuria Cells in Patients with Bone Marrow FailureCytometry Part B: Clinical Cytometry, 2013
- Guidelines for the diagnosis and monitoring of paroxysmal nocturnal hemoglobinuria and related disorders by flow cytometryCytometry Part B: Clinical Cytometry, 2010
- Origin and fate of blood cells deficient in glycosylphosphatidylinositol‐anchored protein among patients with bone marrow failureBritish Journal of Haematology, 2009
- Predicting response to immunosuppressive therapy and survival in severe aplastic anaemiaBritish Journal of Haematology, 2008
- A cohort study of the nature of paroxysmal nocturnal hemoglobinuria clones and PIG‐A mutations in patients with aplastic anemiaEuropean Journal of Haematology, 2006
- Minor population of CD55-CD59- blood cells predicts response to immunosuppressive therapy and prognosis in patients with aplastic anemiaBlood, 2006
- Polyclonal hematopoiesis maintained in patients with bone marrow failure harboring a minor population of paroxysmal nocturnal hemoglobinuria–type cellsBlood, 2003