Clinical spectrum, outcome and management of immune thrombocytopenia associated with myelodysplastic syndromes and chronic myelomonocytic leukemia
Vincent Jachiet,
Guillaume Moulis,
Jérome Hadjadj,
Julie Seguier,
Kamel Laribi,
Nicolas Schleinitz,
Norbert Vey,
Karim Sacre,
Bertrand Godeau,
Odile Beyne-Rauzy,
Romain Bouvet,
Jonathan Broner,
Natacha Brun,
Thibault Comont,
Clément Gaudin,
Olivier Lambotte,
Lenaïg Le Clech,
Pierre Peterlin,
Frédérique Roy-Peaud,
Clémentine Salvado,
Mathilde Versini,
Françoise Isnard,
Jean Emmanuel Kahn,
Delphine Gobert,
Lionel Adès,
Pierre Fenaux,
Olivier Fain,
Arsène Mekinian
Affiliations
Vincent Jachiet
Sorbonne Université, AP-HP, Hôpital Saint-Antoine, Service de Médecine Interne and Inflammation-Immunopathology-Biotherapy Department (DMU 3iD), F-75012, Paris
Guillaume Moulis
Service de médecine interne, CHU de Toulouse, France; CIC 1436, CHU de Toulouse, France; UMR 1027 Inserm-Université de Toulouse
Jérome Hadjadj
Imagine Institute, laboratory of Immunogenetics of Pediatric Autoimmune Diseases, INSERM UMR 1163, Université de Paris, F-75015, Paris ; Department of Internal Medicine, National Referral Center for Rare Systemic Autoimmune Diseases, Assistance Publique Hôpitaux de Paris-Centre (APHP-CUP), Université de Paris, F-75014
Julie Seguier
Département de médecine interne, Hôpital de la Timone, AP-HM, Aix Marseille Université, Marseille
Kamel Laribi
Department of Hematology, Centre hospitalier Le Mans, Le Mans
Nicolas Schleinitz
Département de médecine interne, Hôpital de la Timone, AP-HM, Aix Marseille Université, Marseille
Departement de Médecine Interne, Hôpital Bichat, APHP, Université de Paris, INSERM U1149, Paris
Bertrand Godeau
Hôpitaux de Paris, Hôpital Henri Mondor, Médecine Interne, Centre de Référence des Cytopénies Autoimmunes de L'Adulte, Université Paris-Est Créteil, F-94010, Créteil
Odile Beyne-Rauzy
Department of internal medicine, Toulouse University Hospital, Institut universitaire du cancer de Toulouse, and University of Toulouse, F-31059, Toulouse
Romain Bouvet
Médecine interne et maladies systémiques, CHU Dijon Bourgogne, 21000 Dijon
Jonathan Broner
Internal Médicine Department, Nîmes University Hospital, University of Montpellier, Nîmes
Natacha Brun
Service de Médecine Interne, Centre Hospitalier de Rodez, Rodez
Thibault Comont
Department of internal medicine, Toulouse University Hospital, Institut universitaire du cancer de Toulouse, and University of Toulouse, F-31059, Toulouse
Clément Gaudin
Department of oncogeriatric medicine, University Hospital Purpan, Toulouse
Olivier Lambotte
Hôpitaux de Paris, Hôpital Bicêtre, Médecine Interne et Immunologie Clinique, F-94275, Le Kremlin-Bicêtre, France; INSERM U1184, Immunology of Viral Infections and Autoimmune Diseases, F-94276, Le Kremlin-Bicêtre, France; Université Paris Sud, UMR 1184, F-94276, Le Kremlin-Bicêtre, France; CEA, DSV/iMETI, IDMIT, F-92265, Fontenay-aux-Roses
Lenaïg Le Clech
Department of Internal Medicine, Infectious Diseases and Haematology, Cornouaille Hospital Quimper
Pierre Peterlin
Service d'hématologie clinique, CHU de Nantes
Frédérique Roy-Peaud
Service de médecine interne, maladies infectieuses et tropicales, CHU de Poitiers, Poitiers
Clémentine Salvado
CH de Dax, Service de Maladies Infectieuses, Dax
Mathilde Versini
Institut Arnault Tzanck, Saint Laurent du Var
Françoise Isnard
Department of Clinical Hematology, Saint-Antoine Hospital, AP-HP, Paris
Jean Emmanuel Kahn
AP-HP, Hôpital Ambroise Paré, Service de Médecine Interne, Paris
Delphine Gobert
Sorbonne Université, AP-HP, Hôpital Saint-Antoine, Service de Médecine Interne and Inflammation-Immunopathology-Biotherapy Department (DMU 3iD), F-75012, Paris
Lionel Adès
Hopital Saint-Louis (APHP) and Paris University and INSERM U944, Paris
Pierre Fenaux
Hopital Saint-Louis (APHP) and Paris University and INSERM U944, Paris
Olivier Fain
Sorbonne Université, AP-HP, Hôpital Saint-Antoine, Service de Médecine Interne and Inflammation-Immunopathology-Biotherapy Department (DMU 3iD), F-75012, Paris
Arsène Mekinian
Sorbonne Université, AP-HP, Hôpital Saint-Antoine, Service de Médecine Interne and Inflammation-Immunopathology-Biotherapy Department (DMU 3iD), F-75012, Paris
Myelodysplastic syndromes (MDS) and chronic myelomonocytic leukemia (CMML) are associated with systemic inflammatory or autoimmune diseases in 10-20 % of cases. Among them, immune thrombocytopenia (ITP) has been reported but large studies assessing this association are missing. Whether such patients have a particular phenotype and require particular management is unclear. This study analyzes the clinical spectrum, outcome and therapeutic management of patients with ITP associated with MDS or CMML, in comparison (i) to patients with primary ITP without MDS/CMML and (ii) to patients with MDS/CMML without ITP. Forty-one MDS/CMML-associated ITP patients were included, with chronic ITP in 26 (63%) patients, low-risk myelodysplasia in 30 (73%) patients and CMML in 24 (59%) patients. An associated autoimmune disease was noted in 10 (24%) patients. In comparison to primary ITP patients, MDS/CMML-associated ITP patients had a higher occurrence of severe bleeding despite similar platelet counts at diagnosis. First-line treatment consisted of glucocorticoids (98%) and intravenous immunoglobulin (IVIg) (56%). Response achievement with IVIg was more frequent in primary ITP than in MDS/CMML-associated ITP patients. Response rates to second-line therapies were not statistically different between primary ITP and MDS/CMMLassociated ITP patients. Ten percent (n=4) of patients with MDS/CMML-associated ITP had multirefractory ITP versus none in primary ITP controls. After a median follow-up of 60 months, there was no difference in overall survival between MDS/CMML-associated ITP and primary ITP patients. Leukemia-free-survival was significantly better in MDS/CMMLassociated ITP patients than in MDS/CMML without ITP MDS/CMML-associated ITP have a particular outcome with more severe bleeding and multirefractory profile than primary ITP, similar response profile to primary ITP therapy except for IVIg, and less progression toward acute myeloid leukemia than MDS/CMML without ITP.
