Eltrombopag for myelodysplastic syndromes or chronic myelomonocytic leukaemia with no excess blasts and thrombocytopenia: a French multicentre retrospective real-life study.
Br J Haematol - 2021 - Comont - Eltrombopag for myelodysplastic syndromes or chronic myelomonocytic leukaemia with no excess blasts and thrombocytopenia.pdf
[en] Despite a moderate prevalence in low-risk myelodysplastic syndromes (MDS) and chronic myelomonocytic leukaemia (CMML), thrombocytopenia remains a risk of severe bleeding and therapeutic options are still limited. There are only a few studies with eltrombopag (ELT), a thrombopoietin receptor agonist, in those patients. In this retrospective multicentre study, ELT was used in 50 patients with MDS and 11 with CMML, with no excess of marrow blasts and platelet counts of <50 × 109 /l in a 'real-life' situation. Platelet response occurred in 47 (77%) patients. The median (range) duration of response was 8 (0-69) months. None of the eight still responders who discontinued ELT had relapsed, at a median (range) of 16 (6-23) months after ELT discontinuation. Although 36% of the patients were anti-coagulated or anti-aggregated only 10% of patients had Grade ≥3 bleeding events. Thrombotic events were observed in six (10%) patients, who all but one had a medical history of arterial or venous thrombosis. Progression to acute myeloid leukaemia occurred in four (7%) patients. In this first 'real-life' study, ELT was effective and generally well tolerated in patients with MDS/CMML without excess blasts.
Disciplines :
Hematology
Author, co-author :
Comont, Thibault; Service de Médecine Interne, IUCT Oncopole, Centre Hospitalier Universitaire de Toulouse, Toulouse, France
Meunier, Mathieu; CHU Grenoble Alpes, Université Grenoble Alpes, Institute for Advanced Biosciences, INSERM U1209, CNRS, UMR 5309, Grenoble, France
Cherait, Amina; Service d'Hématologie Sénior, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris (APHP), Université de Paris, Paris, France
Santana, Clemence; Service d'Hématologie, Centre Léon Bérard, Lyon, France
Cluzeau, Thomas; Service d'Hématologie Clinique, CHU de Nice, Nice, France
Slama, Bohrane; Service d'onco-hématologie, Centre Hospitalier Général d'Avignon, Avignon, France
Laribi, Kamel ; Service d'Hématologie, Centre Hospitalier Le Mans, Le Mans, France
Giraud, Jean-Thomas; Service de Médecine Interne, Centre Hospitalier de Tarbes, Tarbes, France
Dimicoli, Sophie; Service d'Hématologie, Centre Hospitalier Universitaire de Bordeaux, Bordeaux, France
Berceanu, Ana; Service d'Hématologie, Centre Hospitalier Universitaire de Besançon, Besançon, France
Le Clech, Lenaïg; Service d'Hématologie, Centre Hospitalier de Quimper, Quimper, France
Cony-Makhoul, Pascale ; Service d'Hématologie, Centre Hospitalier d'Annecy Genevois, Pringy, France
Gruson, Berangere; Service d'Hématologie, Centre Hospitalier Universitaire d'Amiens, Amiens, France
Torregrosa, Jose; Service d'Hématologie et Thérapie Cellulaire, Centre Hospitalier Universitaire de Poitiers, Poitiers, France
Sanhes, Laurence; Service d'Hématologie, Centre Hospitalier de Perpignan, Perpignan, France
Jachiet, Vincent ; Service de Médecine Interne, Hôpital Saint-Antoine, AP-HP, Paris, France
Azerad, Marie-Agnès ; Centre Hospitalier Universitaire de Liège - CHU > > Service d'hématologie clinique
Al Jijakli, Ahmad; Service d'Hématologie, Centre Hospitalier d'Argenteuil, Argenteuil, France
Gyan, Emmanuel; Service d'Hématologie et Thérapie Cellulaire, Centre Hospitalier Universitaire de Tours, Tours, France
Gaudin, Clement; Service de Médecine Interne-Oncogériatrie, Hôpital Purpan, Centre Hospitalier Universitaire de Toulouse, Toulouse, France
Broner, Jonathan; Service de Médecine Interne, Centre Hospitalier Universitaire de Nîmes, Nîmes, France
Guerveno, Claire; Service de Médecine Interne, Centre Hospitalier d'Albi, Albi, France
Guillaume, Thierry ; Service d'Hématologie, Centre Hospitalier Universitaire de Nantes, Nantes, France
Ades, Pr Lionel; Service d'Hématologie Sénior, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris (APHP), Université de Paris, Paris, France
Beyne-Rauzy, Odile; Service de Médecine Interne, IUCT Oncopole, Centre Hospitalier Universitaire de Toulouse, Toulouse, France
Fenaux, Pierre ; Service d'Hématologie Sénior, Hôpital Saint-Louis, Assistance Publique Hôpitaux de Paris (APHP), Université de Paris, Paris, France
Eltrombopag for myelodysplastic syndromes or chronic myelomonocytic leukaemia with no excess blasts and thrombocytopenia: a French multicentre retrospective real-life study.
Thibault Comont received honoraria and/or research or educational support from AbbVie, AstraZeneca, Bristol Myers Squibb (Celgene), Novartis and Takeda. Emmanuel Gyan received research support from Novartis; Thomas Cluzeau received honoraria and/or research or educational support from Abbvie, Agios, Alexion, Amgen, Aprea, Arog, Bristol Myers Squibb (Celgene) Jazz, Kartos, Novartis, Sanofi. Kamel Laribi has received in the last 24 months personal fees for participating to advisory boards or consulting from: Abbvie, Jansen, Beigene, BMS/Celgene, Astellas, Sandoz, Iqone, Novartis. Jonathan Broner and Laurence Sanhes received educational support from Novartis Odile Beyne‐Rauzy received research support from Novartis and educational grant from Bristol Myers Squibb (Celgene). Pierre Fenaux has received honoraria and/or research support from Celgene, Janssen, AbbVie, Jazz, Novartis, Roche and Aprea. All other authors have no conflicts of interest to declare.
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