IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria.

Letter to Blood| June 22, 2023 IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria Carmelo Gurnari, Carmelo Gurnari 1Translational Hematology and Oncology Research Department of Cleveland Clinic, Cleveland, OH2Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy https://orcid.org/0000-0001-6829-5544 Search for other works by this author on: This Site PubMed Google Scholar Pedro Henrique Prata, Pedro Henrique Prata 3University of Paris, Paris, France4INSERM U944/CNRS UMR7212, Institut de Recherche Saint-Louis, Paris, France5Department of Medical Imaging, Hematology and Oncology, University of São Paulo, Riberão Preto, Brazil6Hematology and Transplantation Unit, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France https://orcid.org/0000-0003-0235-7890 Search for other works by this author on: This Site PubMed Google Scholar Luiz Fernando B. Catto, Luiz Fernando B. Catto 5Department of Medical Imaging, Hematology and Oncology, University of São Paulo, Riberão Preto, Brazil https://orcid.org/0000-0002-4019-3850 Search for other works by this author on: This Site PubMed Google Scholar Arda Durmaz, Arda Durmaz 1Translational Hematology and Oncology Research Department of Cleveland Clinic, Cleveland, OH https://orcid.org/0000-0001-8394-600X Search for other works by this author on: This Site PubMed Google Scholar Lise Larcher, Lise Larcher 3University of Paris, Paris, France4INSERM U944/CNRS UMR7212, Institut de Recherche Saint-Louis, Paris, France https://orcid.org/0000-0002-9461-0728 Search for other works by this author on: This Site PubMed Google Scholar Marie Sebert, Marie Sebert 4INSERM U944/CNRS UMR7212, Institut de Recherche Saint-Louis, Paris, France7Hematology Seniors, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Vincent Allain, Vincent Allain 3University of Paris, Paris, France8Immunology Laboratory, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Tariq Kewan, Tariq Kewan 1Translational Hematology and Oncology Research Department of Cleveland Clinic, Cleveland, OH Search for other works by this author on: This Site PubMed Google Scholar Simona Pagliuca, Simona Pagliuca 1Translational Hematology and Oncology Research Department of Cleveland Clinic, Cleveland, OH9Department of Clinical Hematology, CHRU Nancy, Nancy, France https://orcid.org/0000-0003-4688-2478 Search for other works by this author on: This Site PubMed Google Scholar Andre L. Pinto, Andre L. Pinto 5Department of Medical Imaging, Hematology and Oncology, University of São Paulo, Riberão Preto, Brazil https://orcid.org/0000-0002-0900-0504 Search for other works by this author on: This Site PubMed Google Scholar Mariana C. B. Inacio, Mariana C. B. Inacio 5Department of Medical Imaging, Hematology and Oncology, University of São Paulo, Riberão Preto, Brazil Search for other works by this author on: This Site PubMed Google Scholar Lucie Hernandez, Lucie Hernandez 3University of Paris, Paris, France4INSERM U944/CNRS UMR7212, Institut de Recherche Saint-Louis, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Nathalie Dhedin, Nathalie Dhedin 10Hematology Adolescents and Young Adults, Hôpital Saint Louis, Assistance Publique-Hôpitaux de Paris, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Sophie Caillat-Zucman, Sophie Caillat-Zucman 3University of Paris, Paris, France8Immunology Laboratory, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France https://orcid.org/0000-0002-4535-3550 Search for other works by this author on: This Site PubMed Google Scholar Emmanuelle Clappier, Emmanuelle Clappier 4INSERM U944/CNRS UMR7212, Institut de Recherche Saint-Louis, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Flore Sicre de Fontbrune, Flore Sicre de Fontbrune 6Hematology and Transplantation Unit, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France11French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France https://orcid.org/0000-0003-2000-1556 Search for other works by this author on: This Site PubMed Google Scholar Maria Teresa Voso, Maria Teresa Voso 2Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy https://orcid.org/0000-0002-6164-4761 Search for other works by this author on: This Site PubMed Google Scholar Valeria Visconte, Valeria Visconte 1Translational Hematology and Oncology Research Department of Cleveland Clinic, Cleveland, OH Search for other works by this author on: This Site PubMed Google Scholar Régis Peffault de Latour, Régis Peffault de Latour 3University of Paris, Paris, France6Hematology and Transplantation Unit, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France11French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Jean Soulier, Jean Soulier 3University of Paris, Paris, France4INSERM U944/CNRS UMR7212, Institut de Recherche Saint-Louis, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Gérard Socié, Gérard Socié 3University of Paris, Paris, France6Hematology and Transplantation Unit, Hôpital Saint-Louis, Assistance Publique-Hôpitaux de Paris, Paris, France11French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France12INSERM UMR 976, Institut de Recherche Saint-Louis, Paris, France Search for other works by this author on: This Site PubMed Google Scholar Rodrigo T. Calado, Rodrigo T. Calado 5Department of Medical Imaging, Hematology and Oncology, University of São Paulo, Riberão Preto, Brazil https://orcid.org/0000-0002-7966-6029 Search for other works by this author on: This Site PubMed Google Scholar Jaroslaw P. Maciejewski Jaroslaw P. Maciejewski 1Translational Hematology and Oncology Research Department of Cleveland Clinic, Cleveland, OH Search for other works by this author on: This Site PubMed Google Scholar Blood (2023) 141 (25): 3122–3125. https://doi.org/10.1182/blood.2023020108 Article history Submitted: February 10, 2023 Accepted: March 30, 2023 First Edition: April 13, 2023 Share Icon Share Facebook Twitter LinkedIn Email Tools Icon Tools Request Permissions Cite Icon Cite Search Site Citation Carmelo Gurnari, Pedro Henrique Prata, Luiz Fernando B. Catto, Arda Durmaz, Lise Larcher, Marie Sebert, Vincent Allain, Tariq Kewan, Simona Pagliuca, Andre L. Pinto, Mariana C. B. Inacio, Lucie Hernandez, Nathalie Dhedin, Sophie Caillat-Zucman, Emmanuelle Clappier, Flore Sicre de Fontbrune, Maria Teresa Voso, Valeria Visconte, Régis Peffault de Latour, Jean Soulier, Gérard Socié, Rodrigo T. Calado, Jaroslaw P. Maciejewski; IPSS-M in myelodysplastic neoplasms arising from aplastic anemia and paroxysmal nocturnal hemoglobinuria. Blood 2023; 141 (25): 3122–3125. doi: https://doi.org/10.1182/blood.2023020108 Download citation file: Ris (Zotero) Reference Manager EasyBib Bookends Mendeley Papers EndNote RefWorks BibTex toolbar search Search Dropdown Menu toolbar search search input Search input auto suggest filter your search All ContentAll JournalsBlood Search Subjects: Myeloid Neoplasia TO THE EDITOR: Clonal evolution to myelodysplastic neoplasms/syndromes (MDSs) or acute myeloid leukemia represents the most serious late complication among patients with aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH).1,2 Most post-AA/PNH MDS cases present high-risk features, with chromosome 7 abnormalities and mutations in ASXL1, RUNX1, SETBP1, and RAS genes as the most frequent molecular lesions.3 However, other cases have more favorable outcomes and are mainly driven by low-risk cytogenetic alterations (eg, del[13q] and del[20q]).4 We previously showed that MDS arising after AA and PNH constitute a genetically distinct MDS subentity with cytogenetic and molecular features that are typical of myeloid evolution from a previous acquired bone marrow (BM) failure syndrome.1 Indeed, monosomy 7 as well as ASXL1 and RUNX1 mutations were significantly enriched in secondary AA and PNH cases when compared with a cohort... References 1.Gurnari C, Pagliuca S, Prata PH, et al. Clinical and molecular determinants of clonal evolution in aplastic anemia and paroxysmal nocturnal hemoglobinuria. J Clin Oncol. 2023;41(1):132-142.Google ScholarCrossrefSearch ADS 2.Sun L, Babushok DV. Secondary myelodysplastic syndrome and leukemia in acquired aplastic anemia and paroxysmal nocturnal hemoglobinuria. Blood. 2020;136(1):36-49.Google ScholarCrossrefSearch ADS PubMed 3.Negoro E, Nagata Y, Clemente MJ, et al. Origins of myelodysplastic syndromes after aplastic anemia. Blood. 2017;130(17):1953-1957.Google ScholarCrossrefSearch ADS PubMed 4.Groarke EM, Patel BA, Shalhoub R, et al. Predictors of clonal evolution and myeloid neoplasia following immunosuppressive therapy in severe aplastic anemia. Leukemia. 2022;36(9):2328-2337.Google ScholarCrossrefSearch ADS PubMed 5.Gurnari C, Fabiani E, Falconi G, et al. From clonal hematopoiesis to therapy-related myeloid neoplasms: the silent way of cancer progression. Biology (Basel). 2021;10(2):128.Google ScholarPubMed 6.Voso MT, Falconi G, Fabiani E. What's new in the pathogenesis and treatment of therapy-related myeloid neoplasms. Blood. 2021;138(9):749-757.Google ScholarCrossrefSearch ADS PubMed 7.Awada H, Gurnari C, Durmaz A, Awada H, Pagliuca S, Visconte V. Personalized risk schemes and machine learning to empower genomic prognostication models in myelodysplastic syndromes. Int J Mol Sci. 2022;23(5):2802.Google ScholarCrossrefSearch ADS PubMed 8.Bernard E, Tuechler H, Greenberg PL, et al. Molecular international prognostic scoring system for myelodysplastic syndromes. NEJM Evidence. 2022;1(7):EVIDoa2200008.Google ScholarCrossrefSearch ADS 9.Kewan T, Bahaj W, Durmaz A, et al. Validation of the molecular international prognostic scoring system in patients with myelodysplastic syndromes. Blood. 2023;141(14):1768-1772.Google ScholarCrossrefSearch ADS PubMed 10.Gurnari C, Gagelmann N, Badbaran A, et al. Outcome prediction in myelodysplastic neoplasm undergoing hematopoietic cell transplant in the molecular era of IPSS-M. Leukemia. 2023;37(3):717-719.Google ScholarCrossrefSearch ADS PubMed 11.Baer C, Huber S, Hutter S, et al. Risk prediction in MDS: independent validation of the IPSS-M-ready for routine?. Leukemia. 2023;37(4):938-941.Google ScholarCrossrefSearch ADS PubMed 12.Arber DA, Orazi A, Hasserjian R, et al. The 2016 revision to the World Health Organization classification of myeloid neoplasms and acute leukemia. Blood. 2016;127(20):2391-2405.Google ScholarCrossrefSearch ADS PubMed 13.Kaplan EL, Meier P. Nonparametric estimation from incomplete observations. J Am Stat Assoc. 1958;53(282):457-481.Google ScholarCrossrefSearch ADS 14.Pfeilstöcker M, Tuechler H, Sanz G, et al. Time-dependent changes in mortality and transformation risk in MDS. Blood. 2016;128(7):902-910.Google ScholarCrossrefSearch ADS PubMed 15.Pagliuca S, Gurnari C, Hercus C, et al. Molecular landscape of immune pressure and escape in aplastic anemia. Leukemia. 2023;37(1):202-211.Google ScholarCrossrefSearch ADS PubMed 16.Gurnari C, Visconte V. 'We cannot paint them all with the same brush': the need for a better definition of patients with myelodysplastic syndromes for clinical trial design. Br J Haematol. 2022;196(2):268-269.Google ScholarCrossrefSearch ADS PubMed © 2023 by The American Society of Hematology2023 © 2023 by The American Society of Hematology2023 You do not currently have access to this content. Sign in via your Institution