- Myelodysplastic Syndromes (MDS)
The GFM group gathers specialists who focus on myelodysplastic syndromes (MDS) and their associated symptoms.
MDS is one of the most common hematological diseases. In recent years, MDS has received significant attention thanks to new diagnostic methods, discoveries in pathophysiology and, above all, new therapeutic options. Due to the diversity of MDS, it is important to precisely characterize the disease biologically, to predict the course of the disease as accurately as possible, and to determine the most appropriate therapies for the patient.
The objective of our group is:
To optimize diagnostic procedures, to perfect and to adapt them to the requirements of therapeutic innovations. Other objectives are to participate in the establishment, implementation and validation of quality assurance measures.
To create a central, independent, organized and active platform for translational research
- Targeted therapy
- Cell therapy
- Immunotherapy
- Chemotherapy
Scientific
- Contribute to biological research (genetics, cell biology, ...) to advance the pathophysiology and treatment of MDS. This collaboration is through a reconciliation between basic and clinical teams, a supply of biological material according to good practice and the scientific work at meetings of the group
- Establish epidemiological studies and data collection tools to determine the environmental or occupational factors that could be responsible for this disease. It also participates very actively to a European register SMD low risk only
- Collaborate on international research in MDS. This includes in particular:
- A contribution to the study of MDS International Foundation: the GFM, through its registry, has greatly contributed to the study performed to establish the new international prognostic score (IPSS-revised)
- Active involvement in the work of the European Leukemia Net, especially the European register of low-risk MDS
- The cooperation with beginner groups German, Italian and Spanish SMD to establish common therapeutic protocols
Medical
- Participation of GFM with data from “IDIOME study” in FDA approval of Ivosidenib in R/R MDS with IDH1
- Collaboration on revision of the International Prognostic Scoring System (IPSS)
- MDS/CMML from resource-limited region: Characteristics and comparison to tertiary reference European center. Sevoyan A, Mekinian A, Chermat F, Adès L, Ivanyan A, Fenaux P, Hakobyan Y.Eur J Haematol. 2023 Oct 11. doi: 10.1111/ejh.14115. Online ahead of print.PMID: 37822035
- Prognostic value of monocyte subset distribution in chronic myelomonocytic leukemia: results of a multicenter study. Jestin M, Tarfi S, Duchmann M, Badaoui B, Freynet N, Tran Quang V, Sloma I, Droin N, Morabito M, Leclerc M, Maury S, Fenaux P, Solary E, Selimoglu-Buet D, Wagner-Ballon O; Groupe Francophone des Myélodysplasies (GFM).Leukemia. 2021 Mar;35(3):893-896. doi: 10.1038/s41375-020-0955-1. Epub 2020 Jul 20.PMID: 32684630
- Development of luspatercept to treat ineffective erythropoiesis. Kubasch AS, Fenaux P, Platzbecker U.Blood Adv. 2021 Mar 9;5(5):1565-1575. doi: 10.1182/bloodadvances.2020002177.PMID: 33687432
- Myelodysplastic syndromes: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Fenaux P, Haase D, Santini V, Sanz GF, Platzbecker U, Mey U; ESMO Guidelines Committee. Electronic address: clinicalguidelines@esmo.org.Ann Oncol. 2021 Feb;32(2):142-156. doi: 10.1016/j.annonc.2020.11.002. Epub 2020 Nov 19.PMID: 33221366
- Chronic myelomonocytic leukemia diagnosis and management. Chan O, Renneville A, Padron E.Leukemia. 2021 Jun;35(6):1552-1562. doi: 10.1038/s41375-021-01207-3. Epub 2021 Mar 13.PMID: 33714974 Review.
- Characteristics and outcome of patients with low-/intermediate-risk acute promyelocytic leukemia treated with arsenic trioxide: an international collaborative study. Kayser S, Schlenk RF, Lebon D, Carre M, Götze KS, Stölzel F, Berceanu A, Schäfer-Eckart K, Peterlin P, Hicheri Y, Rahme R, Raffoux E, Chermat F, Krause SW, Aulitzky WE, Rigaudeau S, Noppeney R, Berthon C, Görner M, Jost E, Carassou P, Keller U, Orvain C, Braun T, Saillard C, Arar A, Kunzmann V, Wemeau M, De Wit M, Niemann D, Bonmati C, Schwänen C, Abraham J, Aljijakli A, Haiat S, Krämer A, Reichle A, Gnadler M, Willekens C, Spiekermann K, Hiddemann W, Müller-Tidow C, Thiede C, Röllig C, Serve H, Bornhäuser M, Baldus CD, Lengfelder E, Fenaux P, Platzbecker U, Adès L.Haematologica. 2021 Dec 1;106(12):3100-3106. doi: 10.3324/haematol.2021.278722.PMID: 34047178
- Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists. Stojkov I, Conrads-Frank A, Rochau U, Koinig KA, Arvandi M, Puntscher S, van Marrewijk C, Fenaux P, Symeonidis A, Chermat F, Garelius H, Bowen D, Mittelman M, Mora E, de Witte T, Efficace F, Siebert U, Stauder R.Blood Adv. 2022 Jan 11;6(1):1-12. doi: 10.1182/bloodadvances.2021004568.PMID: 34492684
- Eltrombopag for myelodysplastic syndromes or chronic myelomonocytic leukaemia with no excess blasts and thrombocytopenia: a French multicentre retrospective real-life study. Comont T, Meunier M, Cherait A, Santana C, Cluzeau T, Slama B, Laribi K, Giraud JT, Dimicoli S, Berceanu A, Le Clech L, Cony-Makhoul P, Gruson B, Torregrosa J, Sanhes L, Jachiet V, Azerad MA, Al Jijakli A, Gyan E, Gaudin C, Broner J, Guerveno C, Guillaume T, Ades PL, Beyne-Rauzy O, Fenaux P; Groupe Francophone des Myélodysplasies (GFM).Br J Haematol. 2021 Jul;194(2):336-343. doi: 10.1111/bjh.17539. Epub 2021 Jun 20.PMID: 34151423
- Further characterization of clinical and laboratory features in VEXAS syndrome: large-scale analysis of a multicentre case series of 116 French patients. Georgin-Lavialle S, Terrier B, Guedon AF, Heiblig M, Comont T, Lazaro E, Lacombe V, Terriou L, Ardois S, Bouaziz JD, Mathian A, Le Guenno G, Aouba A, Outh R, Meyer A, Roux-Sauvat M, Ebbo M, Zhao LP, Bigot A, Jamilloux Y, Guillotin V, Flamarion E, Henneton P, Vial G, Jachiet V, Rossignol J, Vinzio S, Weitten T, Vinit J, Deligny C, Humbert S, Samson M, Magy-Bertrand N, Moulinet T, Bourguiba R, Hanslik T, Bachmeyer C, Sebert M, Kostine M, Bienvenu B, Biscay P, Liozon E, Sailler L, Chasset F, Audemard-Verger A, Duroyon E, Sarrabay G, Borlot F, Dieval C, Cluzeau T, Marianetti P, Lobbes H, Boursier G, Gerfaud-Valentin M, Jeannel J, Servettaz A, Audia S, Larue M, Henriot B, Faucher B, Graveleau J, de Sainte Marie B, Galland J, Bouillet L, Arnaud C, Ades L, Carrat F, Hirsch P, Fenaux P, Fain O, Sujobert P, Kosmider O, Mekinian A; French VEXAS group; GFEV, GFM, CEREMAIA, MINHEMON.Br J Dermatol. 2022 Mar;186(3):564-574. doi: 10.1111/bjd.20805. Epub 2021 Nov 28.PMID: 34632574
- Azacitidine for patients with Vacuoles, E1 Enzyme, X-linked, Autoinflammatory, Somatic syndrome (VEXAS) and myelodysplastic syndrome: data from the French VEXAS registry. Comont T, Heiblig M, Rivière E, Terriou L, Rossignol J, Bouscary D, Rieu V, Le Guenno G, Mathian A, Aouba A, Vinit J, Dion J, Kosmider O, Terrier B, Georgin-Lavialle S, Fenaux P, Mekinian A; French VEXAS study group, Groupe Francophone des Myélodysplasies (GFM) and MedecineINterne, HEmato et ONco (MINHEMON) group.Br J Haematol. 2022 Feb;196(4):969-974. doi: 10.1111/bjh.17893. Epub 2021 Oct 14.PMID: 34651299
- Eprenetapopt Plus Azacitidine in TP53-Mutated Myelodysplastic Syndromes and Acute Myeloid Leukemia: A Phase II Study by the Groupe Francophone des Myélodysplasies (GFM). Cluzeau T, Sebert M, Rahmé R, Cuzzubbo S, Lehmann-Che J, Madelaine I, Peterlin P, Bève B, Attalah H, Chermat F, Miekoutima E, Rauzy OB, Recher C, Stamatoullas A, Willems L, Raffoux E, Berthon C, Quesnel B, Loschi M, Carpentier AF, Sallman DA, Komrokji R, Walter-Petrich A, Chevret S, Ades L, Fenaux P.J Clin Oncol. 2021 May 10;39(14):1575-1583. doi: 10.1200/JCO.20.02342. Epub 2021 Feb 18.PMID: 33600210
- Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms - results of the EUROPE trial by EMSCO. Kubasch AS, Giagounidis A, Metzgeroth G, Jonasova A, Herbst R, Diaz JMT, De Renzis B, Götze KS, Huetter-Kroenke ML, Gourin MP, Slama B, Dimicoli-Salazar S, Cony-Makhoul P, Laribi K, Park S, Jersemann K, Schipp D, Metzeler KH, Tiebel O, Sockel K, Gloaguen S, Mies A, Chermat F, Thiede C, Sapena R, Schlenk RF, Fenaux P, Platzbecker U, Adès L.Leukemia. 2022 Oct;36(10):2519-2527. doi: 10.1038/s41375-022-01669-z. Epub 2022 Sep 7.PMID: 36071100
- Multicenter validation of the flow measurement of classical monocyte fraction for chronic myelomonocytic leukemia diagnosis. Tarfi S, Harrivel V, Dumezy F, Guy J, Roussel M, Mimoun A, Fenaux P, Chapuis N, Solary E, Selimoglu-Buet D, Wagner-Ballon O; Groupe Francophone des Myélodysplasies (GFM).Blood Cancer J. 2018 Nov 14;8(11):114. doi: 10.1038/s41408-018-0146-8.PMID: 30429467
- Venous thromboembolism during systemic inflammatory and autoimmune diseases associated with myelodysplastic syndromes, chronic myelomonocytic leukaemia and myelodysplastic/myeloproliferative neoplasms: a French multicentre retrospective case-control study. Péan de Ponfilly-Sotier M, Jachiet V, Benhamou Y, Lahuna C, De Renzis B, Kottler D, Voillat L, Dimicoli-Salazar S, Banos A, Chauveheid MP, Alexandra JF, Grignano E, Liferman F, Laborde M, Broner J, Michel M, Lambotte O, Laribi K, Venon MD, Dussol B, Martis N, Thepot S, Park S, Couret D, Roux-Sauvat M, Terriou L, Hachulla E, Bally C, Galland J, Allain JS, Parcelier A, Peterlin P, Cohen-Bittan J, Regent A, Ackermann F, Le Guen J, Algrin C, Charles P, Daguindau E, Puechal X, Dunogue B, Blanchard-Delaunay C, Beyne-Rauzy O, Grobost V, Schmidt J, Le Gallou T, Dubos-Lascu G, Sonet A, Denis G, Roy-Peaud F, Fenaux P, Adès L, Fain O, Mekinian A; MINHEMON (French Network of Dysimmune Disorders Associated wit Hemopathies) and GFM.Clin Exp Rheumatol. 2022 Jul;40(7):1336-1342. doi: 10.55563/clinexprheumatol/nbn38d. Epub 2022 May 16.PMID: 35579092
- A randomised phase II study of azacitidine (AZA) alone or with Lenalidomide (LEN), Valproic acid (VPA) or Idarubicin (IDA) in higher-Risk MDS or low blast AML: GFM's "pick a winner" trial, with the impact of somatic mutations. Adès L, Duployez N, Guerci-Bresler A, Laribi K, Peterlin P, Vey N, Thepot S, Wickenhauser S, Zerazhi H, Stamatoullas A, Wattel E, Recher C, Toma A, Dimicoli-Salazar S, Braun T, Beyne-Rauzy O, Marolleau JP, Cheze S, Park S, Cluzeau T, Nimubona S, Bordessoule D, Benramdane R, Quesnel B, Amé S, de Botton S, Chermat F, Preudhomme C, Chevret S, Fenaux P.Br J Haematol. 2022 Aug;198(3):535-544. doi: 10.1111/bjh.18193. Epub 2022 Apr 19.PMID: 35438802 Clinical Trial.
- Decitabine Versus Hydroxyurea for Advanced Proliferative Chronic Myelomonocytic Leukemia: Results of a Randomized Phase III Trial Within the EMSCO Network. Itzykson R, Santini V, Thepot S, Ades L, Chaffaut C, Giagounidis A, Morabito M, Droin N, Lübbert M, Sapena R, Nimubona S, Goasguen J, Wattel E, Zini G, Torregrosa Diaz JM, Germing U, Pelizzari AM, Park S, Jaekel N, Metzgeroth G, Onida F, Navarro R, Patriarca A, Stamatoullas A, Götze K, Puttrich M, Mossuto S, Solary E, Gloaguen S, Chevret S, Chermat F, Platzbecker U, Fenaux P.J Clin Oncol. 2023 Apr 1;41(10):1888-1897. doi: 10.1200/JCO.22.00437. Epub 2022 Dec 1.PMID: 36455187 Clinical Trial.
- Red blood cell transfusion burden in myelodysplastic syndromes (MDS) with ring Sideroblasts (RS): A retrospective multicenter study by the Groupe Francophone des Myélodysplasies (GFM). Jouzier C, Cherait A, Cony-Makhoul P, Hamel JF, Veloso M, Thepot S, Cluzeau T, Stamatoullas A, Garnier A, Guerci-Bresler A, Dimicoli-Salazar S, Pica GM, Cheze S, Santana C, Chermat F, Fenaux P, Park S.Transfusion. 2022 May;62(5):961-973. doi: 10.1111/trf.16884. Epub 2022 Apr 22.PMID: 35452143
- Efficacy and safety of luspatercept versus epoetin alfa in erythropoiesis-stimulating agent-naive, transfusion-dependent, lower-risk myelodysplastic syndromes (COMMANDS): interim analysis of a phase 3, open-label, randomised controlled trial. Platzbecker U, Della Porta MG, Santini V, Zeidan AM, Komrokji RS, Shortt J, Valcarcel D, Jonasova A, Dimicoli-Salazar S, Tiong IS, Lin CC, Li J, Zhang J, Giuseppi AC, Kreitz S, Pozharskaya V, Keeperman KL, Rose S, Shetty JK, Hayati S, Vodala S, Prebet T, Degulys A, Paolini S, Cluzeau T, Fenaux P, Garcia-Manero G.Lancet. 2023 Jul 29;402(10399):373-385. doi: 10.1016/S0140-6736(23)00874-7. Epub 2023 Jun 10.PMID: 37311468 Clinical Trial.
- CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 study. Peterlin P, Le Bris Y, Turlure P, Chevallier P, Ménard A, Gourin MP, Dumas PY, Thepot S, Berceanu A, Park S, Hospital MA, Cluzeau T, Bouzy S, Torregrosa-Diaz JM, Drevon L, Sapena R, Chermat F, Ades L, Dimicoli-Salazar S, Chevret S, Béné MC, Fenaux P.Lancet Haematol. 2023 Jul;10(7):e521-e529. doi: 10.1016/S2352-3026(23)00090-X. Epub 2023 May 25.PMID: 37245522 Clinical Trial.