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129 results on '"Chermat A"'

1. Eltrombopag in chronic myelomonocytic leukemia with severe thrombocytopenia. A Groupe Francophone des Myélodysplasies (GFM) study

Author

Rabian, Florence, Chevret, Sylvie, Gruson, Bérengère, Thépot, Sylvain, Walter-Petrich, Anouk, Braun, Thorsten, Vey, Norbert, Torregrosa-Diaz, José Miguel, Peterlin, Pierre, Toma, Andrea, D’Aveni, Maud, Delaunay, Jacques, Legros, Laurence, Droin, Nathalie, Chermat, Fatiha, Lusina, Daniel, Adès, Lionel, Sapena, Rosa, Solary, Eric, Fenaux, Pierre, and Itzykson, Raphael

Published
2024
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2. Clinical impact of genetic alterations including germline DDX41 mutations in MDS/low-blast count AML patients treated with azacitidine-based regimens

Author

Sébert, Marie, Freiman, Lucie, Chaffaut, Cendrine, Guerci, Agnès, Peterlin, Pierre, Thépot, Sylvain, Beyne-Rauzy, Odile, Park, Sophie, Cluzeau, Thomas, Chermat, Fatiha, Fenaux, Pierre, Preudhomme, Claude, Clappier, Emmanuelle, Chevret, Sylvie, Adès, Lionel, Duployez, Nicolas, and Duchmann, Matthieu

Published
2024
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3. Bi-valve braces for treatment of pectus carinatum in teenagers: impact on patients quality of life

Author

Marion Mauduit, Anaëlle Chermat, Dorian Rojas, Simon Rouzé, Bertrand Delatour, and Jean-Philippe Verhoye

Subjects
Pectus carinatum, Bracing therapy, Quality of life, Compliance with orthotic treatment, Surgery, RD1-811, Diseases of the circulatory (Cardiovascular) system, RC666-701
Abstract

Abstract Background Pectus carinatum may be a major issue for adolescent patients, as chest-wall malformations can have a negative impact on body image and induce self-esteem disorders. Bracing has become the first line treatment for adolescent patients whose anterior chest wall is still flexible. The main obstacle is the tolerability of the bracing device along with the patient’s compliance to the device. The aim of this study was to examine the quality of life of adolescent patients treated with bracing to correct pectus carinatum in our institution. From November 2021 to July 2023, 28 patients with pectus carinatum were treated with bracing therapy in our chest wall deformities unit. We used a bivalve brace, manufactured with computer-aided design. All patients were asked to answer a multiple-choice questionnaire designed to assess how bracing therapy impacts their daily life, treatment compliance, and overall satisfaction. Patients who wore the brace for at least 3 months, and at least 5 days per week and 10 h per day, are defined as compliant with the bracing protocol. Results The mean age of the patients was 14 ± 1.36 years. Patients wore the brace for an average of 10.1 ± 6.9 months in the compliance group (n = 18), and 2.2 ± 2.5 months in the non-compliance group (n = 10). Our results show that compliant patients are fairly satisfied of the comfort of the brace, with a limited impact of the device on their daily activities and social life. However, non-compliant patients report the following dissatisfactions regarding the brace: higher pain scores, difficulty in breathing, and issues sleeping with the brace. The compliant patients noted a significant improvement in the appearance of their chest, and were satisfied with the appearance of their chest. The non-compliant patients did not notice any changes. Conclusions Compliant patients treated with a bi-valve brace for pectus carinatum seem to have a good quality of life with a limited impact on their daily life and social activities. The bi-valve brace also seems to be an effective therapy in compliant patients.

Published
2024
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5. FLT3 ligand kinetic profile predicts response to treatment in patients with high-risk myelodysplastic syndrome / chronic myelomonocytic leukemia receiving CPX-351: a study from the Groupe Francophone des Myélodysplasies

Author

Pierre Peterlin, Joëlle Gaschet, Pascal Turlure, Marie-Pierre Gourin, Pierre-Yves Dumas, Sylvain Thepot, Ana Berceanu, Sophie Park, Marie-Anne Hospital, Thomas Cluzeau, Jose-Miguel Torregrosa-Diaz, Louis Drevon, Rosa Sapena, Fatiha Chermat, Lionel Ades, Sophie Dimicoli-Salazar, Maxime Jullien, Pierre Fenaux, and Patrice Chevallier.

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Abstract

Not available.

Published
2024
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6. Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias – pilot study protocol

Author

Niels Vejlstrup, Pierre Fenaux, Jesper Petersen, Eduard J van Beers, Andreas Glenthøj, Kevin H M Kuo, Richard van Wijk, Minke A E Rab, Evelyn Groot, Nina Toft, Selma Kofoed Bendtsen, Jens Helby, and Fatiha Chermat

Subjects
Medicine
Abstract

Introduction Membranopathies encompass haemolytic disorders arising from genetic variants in erythrocyte membrane proteins, including hereditary spherocytosis and stomatocytosis. Congenital dyserythropoietic anaemia type II (CDA II) is associated with the SEC23B gene and can exhibit phenotypic similarities to membranopathies. Current treatment options for these conditions, apart from splenectomy, are primarily supportive. Mitapivat, a novel pyruvate kinase (PK) activator, has demonstrated efficacy in increasing haemoglobin levels and reducing haemolysis in patients with PK deficiency, thalassemia, sickle cell disease and a mouse model of hereditary spherocytosis.Methods and analyses Safety and efficacy of mitapivat sulfate in adult patients with erythrocyte membranopathies (SATISFY) is a prospective, multicentre, single-arm phase two trial involving approximately 25 adult patients (≥18 years) diagnosed with a membranopathy or CDA II. During the 8-week dose escalation period, subjects will receive an initial dose of 50 mg mitapivat two times per day and may increase to 100 mg two times per day at week 4 based on the safety and changes in haemoglobin levels. Patients tolerating mitapivat well may be eligible to continue in two consecutive 24-week fixed dose periods.The primary objective of this study is to evaluate the safety of mitapivat, assessed through the occurrence of treatment-emergent adverse events. Secondary objectives include assessing the effects of mitapivat on haemoglobin levels, haemolysis, erythropoiesis, patient-reported outcome measures and spleen size.SATISFY aims to assess the safety and efficacy of mitapivat in adult patients with red blood cell membranopathies and CDA II, with the aim of establishing proof-of-concept in patients living with these rare conditions.Ethics and dissemination NCT05935202/CTIS:2023-503271-24-01. Findings will be published in peer-reviewed journals.Trial registration number Clinicaltrials.gov, NCT05935202. CTIS:2023-503271-24-01. Registered 07-July-2023. Protocol number: 2.1. https://clinicaltrials.gov/study/NCT05935202.

Published
2024
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7. Efficacy and safety of bemcentinib in patients with advanced myelodysplastic neoplasms or acute myeloid leukemia failing hypomethylating agents- the EMSCO phase II BERGAMO trial

Author

Kubasch, A. S., Peterlin, P., Cluzeau, T., Götze, K. S., Sockel, K., Teipel, R., Jentzsch, M., Attalah, H., Sebert, M., Chermat, F., Gloaguen, S., Puttrich, M., Cross, M., Schneider, M., Kayser, S., Schipp, D., Giagounidis, A., Tirado-Gonzalez, I., Descot, A., van de Loosdrecht, A., Weigert, A., Metzeler, K. H., Fenaux, P., Medyouf, H., Platzbecker, U., and Ades, L.

Published
2023
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8. CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 study

Author

Peterlin, Pierre, Le Bris, Yannick, Turlure, Pascal, Chevallier, Patrice, Ménard, Audrey, Gourin, Marie-Pierre, Dumas, Pierre-Yves, Thepot, Sylvain, Berceanu, Ana, Park, Sophie, Hospital, Marie-Anne, Cluzeau, Thomas, Bouzy, Simon, Torregrosa-Diaz, Jose-Miguel, Drevon, Louis, Sapena, Rosa, Chermat, Fatiha, Ades, Lionel, Dimicoli-Salazar, Sophie, Chevret, Sylvie, Béné, Marie-Christine, and Fenaux, Pierre

Published
2023
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9. A Phase II prospective trial of azacitidine in steroid-dependent or refractory systemic autoimmune/inflammatory disorders and VEXAS syndrome associated with MDS and CMML

Author

Mekinian, Arsene, Zhao, Lin Pierre, Chevret, Sylvie, Desseaux, Kristell, Pascal, Laurent, Comont, Thibaut, Maria, Alexandre, Peterlin, Pierre, Terriou, Louis, D’Aveni Piney, Maud, Gourin, Marie-Pierre, Vey, Norbert, Rauzy, Odile Beyne, Grobost, Vincent, Bezanahary, Holy, Dimicoli-Salazar, Sophie, Banos, Anne, Wickenhauser, Stefan, De Renzis, Benoit, Durot, Eric, Natarajan-Amé, Shanti, Voillat, Laurent, Chermat, Fatiha, Lemaire, Karine, Jachiet, Vincent, Himberlin, Chantal, Thépot, Sylvain, Diaz, Jose Miguel Torregrosa, Frenzel, Laurent, Gyan, Emmanuel, Denis, Guillaume, Hirsch, Pierre, Kosmider, Olivier, Ades, Lionel, Fain, Olivier, and Fenaux, Pierre

Published
2022
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10. Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms – results of the EUROPE trial by EMSCO

Author

Kubasch, Anne Sophie, Giagounidis, Aristoteles, Metzgeroth, Georgia, Jonasova, Anna, Herbst, Regina, Diaz, Jose Miguel Torregrosa, De Renzis, Benoit, Götze, Katharina S., Huetter-Kroenke, Marie-Luise, Gourin, Marie-Pierre, Slama, Borhane, Dimicoli-Salazar, Sophie, Cony-Makhoul, Pascale, Laribi, Kamel, Park, Sophie, Jersemann, Katja, Schipp, Dorothea, Metzeler, Klaus H., Tiebel, Oliver, Sockel, Katja, Gloaguen, Silke, Mies, Anna, Chermat, Fatiha, Thiede, Christian, Sapena, Rosa, Schlenk, Richard F., Fenaux, Pierre, Platzbecker, Uwe, and Adès, Lionel

Published
2022
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11. Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists

Author

Stojkov, Igor, Conrads-Frank, Annette, Rochau, Ursula, Koinig, Karin A., Arvandi, Marjan, Puntscher, Sibylle, van Marrewijk, Corine, Fenaux, Pierre, Symeonidis, Argiris, Chermat, Fatiha, Garelius, Hege, Bowen, David, Mittelman, Moshe, Mora, Elvira, de Witte, Theo, Efficace, Fabio, Siebert, Uwe, and Stauder, Reinhard

Published
2022
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12. Giant thoracic calcinosis

Author

Chermat, Anaëlle, primary, Begueret, Hugues, additional, Tricard, Jeremy, additional, Jougon, Jacques, additional, Belaroussi, Yaniss, additional, and Thumerel, Matthieu, additional

Published
2024
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13. Designing a single-arm phase 2 clinical trial of mitapivat for adult patients with erythrocyte membranopathies (SATISFY): a framework for interventional trials in rare anaemias - pilot study protocol

Author

Poli Van Creveldkliniek Medisch, Child Health, CDL Cluster Speciële Diagnostiek, Circulatory Health, CDL Rode cel, Glenthøj, Andreas, van Beers, Eduard J., van Wijk, Richard, Rab, Minke A.E., Groot, Evelyn, Vejlstrup, Niels, Toft, Nina, Bendtsen, Selma Kofoed, Petersen, Jesper, Helby, Jens, Chermat, Fatiha, Fenaux, Pierre, Kuo, Kevin H.M., Poli Van Creveldkliniek Medisch, Child Health, CDL Cluster Speciële Diagnostiek, Circulatory Health, CDL Rode cel, Glenthøj, Andreas, van Beers, Eduard J., van Wijk, Richard, Rab, Minke A.E., Groot, Evelyn, Vejlstrup, Niels, Toft, Nina, Bendtsen, Selma Kofoed, Petersen, Jesper, Helby, Jens, Chermat, Fatiha, Fenaux, Pierre, and Kuo, Kevin H.M.

Published
2024

14. Bi-valve braces for treatment of pectus carinatum in teenagers: impact on patients quality of life.

Author

Mauduit, Marion, Chermat, Anaëlle, Rojas, Dorian, Rouzé, Simon, Delatour, Bertrand, and Verhoye, Jean-Philippe

Subjects
*ORTHOPEDIC braces, *PECTUS excavatum, *QUALITY of life, *PATIENT compliance, *TEENAGERS, *CAD/CAM systems, *BODY image
Abstract

Background: Pectus carinatum may be a major issue for adolescent patients, as chest-wall malformations can have a negative impact on body image and induce self-esteem disorders. Bracing has become the first line treatment for adolescent patients whose anterior chest wall is still flexible. The main obstacle is the tolerability of the bracing device along with the patient's compliance to the device. The aim of this study was to examine the quality of life of adolescent patients treated with bracing to correct pectus carinatum in our institution. From November 2021 to July 2023, 28 patients with pectus carinatum were treated with bracing therapy in our chest wall deformities unit. We used a bivalve brace, manufactured with computer-aided design. All patients were asked to answer a multiple-choice questionnaire designed to assess how bracing therapy impacts their daily life, treatment compliance, and overall satisfaction. Patients who wore the brace for at least 3 months, and at least 5 days per week and 10 h per day, are defined as compliant with the bracing protocol. Results: The mean age of the patients was 14 ± 1.36 years. Patients wore the brace for an average of 10.1 ± 6.9 months in the compliance group (n = 18), and 2.2 ± 2.5 months in the non-compliance group (n = 10). Our results show that compliant patients are fairly satisfied of the comfort of the brace, with a limited impact of the device on their daily activities and social life. However, non-compliant patients report the following dissatisfactions regarding the brace: higher pain scores, difficulty in breathing, and issues sleeping with the brace. The compliant patients noted a significant improvement in the appearance of their chest, and were satisfied with the appearance of their chest. The non-compliant patients did not notice any changes. Conclusions: Compliant patients treated with a bi-valve brace for pectus carinatum seem to have a good quality of life with a limited impact on their daily life and social activities. The bi-valve brace also seems to be an effective therapy in compliant patients. [ABSTRACT FROM AUTHOR]

Published
2024
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16. Predictive Model Based on K-Nearest Neighbor Coupled with the Gray Wolf Optimizer Algorithm (KNN_GWO) for Estimating the Amount of Phenol Adsorption on Powdered Activated Carbon

Author

Meriem Zamouche, Mouchira Chermat, Zohra Kermiche, Hichem Tahraoui, Mohamed Kebir, Jean-Claude Bollinger, Abdeltif Amrane, and Lotfi Mouni

Subjects
adsorption, phenol, activated carbon, modeling, k-nearest neighbor, gray wolf optimizer, Hydraulic engineering, TC1-978, Water supply for domestic and industrial purposes, TD201-500
Abstract

In this work, the adsorption mechanism of phenol on activated carbon from aqueous solutions was investigated. Batch experiments were performed as a function of adsorbent rate, solution temperature, phenol initial concentration, stirring speed, and pH. The optimal operating condition of phenol adsorption were: mass/volume ratio of 0.6 g.L−1, temperature of 20 °C and stirring speed of 300 rpm. The equilibrium data for the adsorption of phenol were analyzed by Langmuir, Freundlich, and Temkin isotherm models. It was found that the Freundlich and Temkin isotherm models fitted well the phenol adsorption on the activated carbon and that the adsorption process is favorable. The Langmuir equilibrium isotherm provides a maximum adsorption of 156.26 mg.g−1 at 20 °C. The pseudo-first-order, pseudo-second-order, intraparticle diffusion, and Boyd models were used to fit the kinetic data. The adsorption kinetics data were well described by the pseudo-second-order model. The kinetic was controlled by the external diffusion by macropore and mesopore, as well as by the micropore diffusion. The thermodynamic study revealed the exothermic and spontaneous nature of phenol adsorption on activated carbon with increased randomness at the solid-solution interface. On the other hand, a very large model based on the optimization parameters of phenol adsorption using k-nearest neighbor coupled with the gray wolf optimizer algorithm was launched to predict the amount of phenol adsorption. The KNN_GWO model showed an advantage in giving more precise values related to very high statistical coefficients (R = 0.9999, R2 = 0.9998 and R2adj = 0.9998) and very low statistical errors (RMSE = 0, 0070, MSE = 0.2347 and MAE = 0.2763). These advantages show the efficiency and performance of the model used.

Published
2023
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17. S169: CLINICAL AND MOLECULAR MARKERS FOR PREDICTING RESPONSE TO ROMIPLOSTIM TREATMENT IN LOWER-RISK MYELODYSPLASTIC SYNDROMES

Author

A. S. Kubasch, A. Giagounidis, G. Metzgeroth, A. Jonasova, R. Herbst, J. M. T. Diaz, B. De Renzis, K. S. Götze, M.-L. Huetter-Kroenke, M.-P. Gourin, B. Slama, S. Dimicoli-Salazar, P. Cony-Makhoul, K. Laribi, S. Park, K. Jersemann, D. Schipp, K. H. Metzeler, O. Tiebel, K. Sockel, S. Gloaguen, A. Mies, F. Chermat, C. Thiede, R. Sapena, R. F. Schlenk, P. Fenaux, U. Platzbecker, and L. Ades

Subjects
Diseases of the blood and blood-forming organs, RC633-647.5
Published
2022
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18. Impact of Arsenic Trioxide in the Treatment of Higher Risk Acute Promyelocytic Leukemia

Author

Da Rocha, Audrey, primary, Cassinat, Bruno, additional, Heiblig, Mael, additional, Recher, Christian, additional, Bertoli, Sarah, additional, Bonmati, Caroline, additional, Roth-Guepin, Gabrielle, additional, Zilliox, Anne, additional, Laloi, Marie, additional, Hunault, Mathilde, additional, Berthon, Céline, additional, Duployez, Nicolas, additional, Willekens, Christophe, additional, Gallego Hernanz, Maria Pilar, additional, Ochmann, Marlene, additional, Cluzeau, Thomas, additional, Chermat, Fatiha, additional, Caulier, Alexis, additional, Raffoux, Emmanuel, additional, Fenaux, Pierre, additional, Marolleau, Jean Pierre, additional, Ades, Lionel, additional, and Lebon, Delphine, additional

Published
2023
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21. 132 Hypoxia on a Chip

Author

Refet-Mollof, Elena, primary, Chermat, Rodin, additional, Gervais, Thomas, additional, and Wong, Philip, additional

Published
2023
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23. Decitabine Versus Hydroxyurea for Advanced Proliferative Chronic Myelomonocytic Leukemia: Results of a Randomized Phase III Trial Within the EMSCO Network

Author

Raphael Itzykson, Valeria Santini, Sylvain Thepot, Lionel Ades, Cendrine Chaffaut, Aristoteles Giagounidis, Margot Morabito, Nathalie Droin, Michael Lübbert, Rosa Sapena, Stanislas Nimubona, Jean Goasguen, Eric Wattel, Gina Zini, Jose Miguel Torregrosa Diaz, Ulrich Germing, Anna Maria Pelizzari, Sophie Park, Nadja Jaekel, Georgia Metzgeroth, Francesco Onida, Robert Navarro, Andrea Patriarca, Aspasia Stamatoullas, Katharina Götze, Martin Puttrich, Sandra Mossuto, Eric Solary, Silke Gloaguen, Sylvie Chevret, Fatiha Chermat, Uwe Platzbecker, and Pierre Fenaux

Subjects
Cancer Research, Oncology
Abstract

PURPOSE Hydroxyurea (HY) is a reference treatment of advanced myeloproliferative neoplasms. We conducted a randomized phase III trial comparing decitabine (DAC) and HY in advanced myeloproliferative chronic myelomonocytic leukemias (CMML). PATIENTS AND METHODS Newly diagnosed myeloproliferative CMML patients with advanced disease were randomly assigned 1:1 to intravenous DAC (20 mg/m2/d days 1-5) or HY (1-4 g/d) in 28-day cycles. The primary end point was event-free survival (EFS), events being death and acute myelomonocytic leukemia (AML) transformation or progression. RESULTS One-hundred seventy patients received DAC (n = 84) or HY (n = 86). Median age was 72 and 74 years, and median WBC count 32.5 × 109/L and 31.2 × 109/L in the DAC and HY arms, respectively. Thirty-three percent of DAC and 31% of HY patients had CMML-2. Patients received a median of five DAC and six HY cycles. With a median follow-up of 17.5 months, median EFS was 12.1 months in the DAC arm and 10.3 months in the HY arm (hazard ratio [HR], 0.83; 95% CI, 0.59 to 1.16; P = .27). There was no significant interaction between treatment effect and blast or platelet count, anemia, CMML Prognostic Scoring System, Groupe Francophone des Myelodysplasies, or CMML Prognostic Scoring System–mol risk. Fifty-three (63%) DAC patients achieved a response compared with 30 (35%) HY patients ( P = .0004). Median duration of response was similar in both arms (DAC, 16.3 months; HY, 17.4 months; P = .90). Median overall survival was 18.4 months in the DAC arm and 21.9 months in the HY arm ( P = .67). Compared with HY, DAC significantly reduced the risk of CMML progression or transformation to acute myelomonocytic leukemia (cause-specific HR, 0.62; 95% CI, 0.41 to 0.94; P = .005) at the expense of death without progression or transformation (cause-specific HR, 1.55; 95% CI, 0.82 to 2.9; P = .04). CONCLUSION Compared with HY, frontline treatment with DAC did not improve EFS in patients with advanced myeloproliferative CMML (ClinicalTrials.gov identifier: NCT02214407 ).

Published
2023

24. Clinical impact of genetic alterations including germline DDX41mutations in MDS/low-blast count AML patients treated with azacitidine-based regimens

Author

Sébert, Marie, Freiman, Lucie, Chaffaut, Cendrine, Guerci, Agnès, Peterlin, Pierre, Thépot, Sylvain, Beyne-Rauzy, Odile, Park, Sophie, Cluzeau, Thomas, Chermat, Fatiha, Fenaux, Pierre, Preudhomme, Claude, Clappier, Emmanuelle, Chevret, Sylvie, Adès, Lionel, Duployez, Nicolas, and Duchmann, Matthieu

Published
2024
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25. A Phase II prospective trial of azacitidine in steroid-dependent or refractory systemic autoimmune/inflammatory disorders and VEXAS syndrome associated with MDS and CMML

Author

Arsene Mekinian, Lin Pierre Zhao, Sylvie Chevret, Kristell Desseaux, Laurent Pascal, Thibaut Comont, Alexandre Maria, Pierre Peterlin, Louis Terriou, Maud D’Aveni Piney, Marie-Pierre Gourin, Norbert Vey, Odile Beyne Rauzy, Vincent Grobost, Holy Bezanahary, Sophie Dimicoli-Salazar, Anne Banos, Stefan Wickenhauser, Benoit De Renzis, Eric Durot, Shanti Natarajan-Amé, Laurent Voillat, Fatiha Chermat, Karine Lemaire, Vincent Jachiet, Chantal Himberlin, Sylvain Thépot, Jose Miguel Torregrosa Diaz, Laurent Frenzel, Emmanuel Gyan, Guillaume Denis, Pierre Hirsch, Olivier Kosmider, Lionel Ades, Olivier Fain, and Pierre Fenaux

Subjects
Cancer Research, Oncology, Hematology
Published
2022

26. MDS/CMML from resource‐limited region: Characteristics and comparison to tertiary reference European center.

Author

Sevoyan, Anna, Mekinian, Arsène, Chermat, Fatiha, Adès, Lionel, Ivanyan, A., Fenaux, Pierre, and Hakobyan, Yervand

Subjects
RESOURCE-limited settings, ACUTE myeloid leukemia, CHRONIC leukemia, MYELODYSPLASTIC syndromes, BONE marrow, PRELEUKEMIA
Abstract

Introduction: Myelodysplastic Syndromes (MDS) and Chronic Myelomonocytic Leukemia (CMML) are clonal myeloid malignancies, characterized by bone marrow failure leading to cytopenias (and possible myeloproliferation for CMML) and a high propensity to evolve to Acute Myeloid Leukemia (AML). Objective and Methods: The aim of our retrospective study was to evaluate the clinical and hematological features; the prevalence of MDS subtypes, R‐IPSS, and the outcome of 106 Armenian MDS/CMML patients diagnosed over the 2008–2020 period in a single Armenian Hematology center and compare them to French MDS patients included in the GFM registry. Results: Median age in the Armenian cohort was 64 years (range 19–84) and 55% were males. The main MDS subtypes were MDS‐MLD (29.2%) and MDS‐SLD (27.3%), the least frequent was del 5q (0.9%). By comparison, a higher prevalence of MDS‐MLD, MDS‐EB2, and MDS‐RS was found in the French cohort. Armenian patients' cohort generally had poor access to standard MDS treatment and 42.3% of the patients were transfusion dependent. Overall survival, however, did not significantly differ between Armenian and French cohorts. Conclusion: Our study stresses issues regarding epidemiology, access to diagnosis, difficulties of risk stratification, and access to treatment. [ABSTRACT FROM AUTHOR]

Published
2024
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27. Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: ACCELERATED FAILURE TIME MODELS AS A CLINICALLY PLAUSIBLE WAY TO DESCRIBE SURVIVAL IN MDS – AN ANALYSIS OF THE IWG-PM DATABASE

Author

Pfeilstöcker, M., primary, Tuechler, H., additional, Ades, L., additional, Cermak, J., additional, Chermat, F., additional, Della Porta, M., additional, Fenaux, P., additional, Garcia-Manero, G., additional, Germing, U., additional, Haase, D., additional, Kündgen, A., additional, Lübbert, M., additional, Magalhaes, S., additional, Malcovati, L., additional, Miyazaki, Y., additional, Sanz, G., additional, Santini, V., additional, Sekeres, M., additional, Walter, M., additional, Valent, P., additional, and Greenberg, P., additional

Published
2023
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28. CPX-351 in higher risk myelodysplastic syndrome and chronic myelomonocytic leukaemia: a multicentre, single-arm, phase 2 study

Author

Pierre Peterlin, Yannick Le Bris, Pascal Turlure, Patrice Chevallier, Audrey Ménard, Marie-Pierre Gourin, Pierre-Yves Dumas, Sylvain Thepot, Ana Berceanu, Sophie Park, Marie-Anne Hospital, Thomas Cluzeau, Simon Bouzy, Jose-Miguel Torregrosa-Diaz, Louis Drevon, Rosa Sapena, Fatiha Chermat, Lionel Ades, Sophie Dimicoli-Salazar, Sylvie Chevret, Marie-Christine Béné, and Pierre Fenaux

Subjects
Hematology
Published
2023

29. Molecular Response Analysis By High Throughput Sequencing in Higher Risk Myelodysplastic Syndrome (HR-MDS) Treated Intensively with CPX-351

Author

Yannick Le Bris, Simon Bouzy, Audrey Ménard, Pascal Turlure, Patrice Chevallier, Marie-Pierre Gourin, Pierre-Yves Dumas, Sylvain Thepot, Anna Berceanu, Sophie Park, Marie-Anne Hospital, Thomas Cluzeau, Jose-Miguel Torregrosa Diaz, Louis Devron, Rosa Sapena, Fatiha Chermat, Sophie Dimicoli Salazar, Marie C Béné, Pierre Fenaux, and Pierre Peterlin

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022

30. Dynamics of Mortality and Transformation Risk within Different Risk Groups of Patients with Myelodysplastic Syndromes Stratified According to the IPSS-R - Comparison of Treated and Untreated Patients and Consequences for the Description of Risk Categories

Author

Michael Pfeilstocker, Heinz Tuechler, Lionel Ades, Jaroslav Cermak, Fatiha Chermat, Matteo G. Della Porta, Pierre Fenaux, Guillermo Garcia-Manero, Ulrich Germing, Detlef Haase, Andrea Kuendgen, Michael Luebbert, Silvia Maria Meira Magalhaes, Luca Malcovati, Yasushi Miyazaki, Guillermo Sanz, Valeria Santini, Mikkael A. Sekeres, Matthew J Walter, Peter Valent, and Peter L Greenberg

Subjects
Immunology, Cell Biology, Hematology, Biochemistry
Published
2022

31. Red blood cell transfusion burden in myelodysplastic syndromes ( <scp>MDS</scp> ) with ring Sideroblasts ( <scp>RS</scp> ): A retrospective multicenter study by the Groupe Francophone des Myélodysplasies ( <scp>GFM</scp> )

Author

Claire Jouzier, Amina Cherait, Pascale Cony‐Makhoul, Jean‐François Hamel, Melanie Veloso, Sylvain Thepot, Thomas Cluzeau, Aspasia Stamatoullas, Alice Garnier, Agnès Guerci‐Bresler, Sophie Dimicoli‐Salazar, Gian Matteo Pica, Stéphane Cheze, Clémence Santana, Fatiha Chermat, Pierre Fenaux, and Sophie Park

Subjects
Myelodysplastic Syndromes, Immunology, Humans, Immunology and Allergy, Anemia, Hematology, Erythrocyte Transfusion, Iron Chelating Agents, Retrospective Studies
Abstract

MDS-RS patients are characterized by chronic anemia and a low risk of Acute Myeloid Leukemia (AML) progression and they generally become Red Blood Cell (RBC) transfusion dependent (TD).We performed a retrospective "real-life" observational study of 6 months in 100 MDS-RS TD patients, recruited in 12 French centers, to describe transfusion characteristics, and evaluate the frequency and causes of hospitalizations, health costs, and morbidity, associated with transfusion dependency, in a French population of RBC transfusion-dependent MDS-RS patients.79% of the patients had high transfusion burden (HTB) and 21% low transfusion burden (LTB). HTB patients had a longer disease duration (6 vs. 3.7 years, p = 0.0078), more frequent iron chelation (82% vs. 50%, p = 0.0052) and higher serum ferritin (p = 0.03). During the 6-month study period, 22% of the patients required inpatient hospitalization, 36% of them for symptomatic anemia requiring emergency RBC transfusion. The 6-month median transfusion costs, including the cost of the day care facility, transportation to and from the hospital, iron chelation, and lab tests, was 16,188€/patient.MDS-RS represents the archetypal type of chronically transfused lower-risk MDS. Most of those patients have a high transfusion burden and thus frequently need visits to the hospital's day care facility, and frequent hospitalizations, with an overall high median treatment cost. Those costs should be compared with costs of new treatments potentially able to avoid RBC transfusion dependence and to reduce the complications of chronic anemia in MDS-RS patients.

Published
2022

32. A randomised phase <scp>II</scp> study of azacitidine ( <scp>AZA</scp> ) alone or with Lenalidomide ( <scp>LEN</scp> ), Valproic acid ( <scp>VPA</scp> ) or Idarubicin ( <scp>IDA</scp> ) in <scp>higher‐Risk MDS</scp> or low blast <scp>AML</scp> : <scp>GFM</scp> 's 'pick a winner' trial, with the impact of somatic mutations

Author

Lionel Adès, Nicolas Duployez, Agnes Guerci‐Bresler, Kamel Laribi, Pierre Peterlin, Norbert Vey, Sylvain Thepot, Stefan Wickenhauser, Hacene Zerazhi, Aspassia Stamatoullas, Eric Wattel, Christian Recher, Andrea Toma, Sophie Dimicoli‐Salazar, Thorsten Braun, Odile Beyne‐Rauzy, Jean‐Pierre Marolleau, Stéphane Cheze, Sophie Park, Thomas Cluzeau, Stanislas Nimubona, Dominique Bordessoule, Riad Benramdane, Bruno Quesnel, Shanti Amé, Stéphane de Botton, Fathia Chermat, Claude Preudhomme, Sylvie Chevret, and Pierre Fenaux

Subjects
Hematology
Published
2022

34. دراسة قياسية لأثر التكنولوجيا في انتقال البلدان إلى مسارات أعلى للدخل خلال الفترة (1990-2018)

Author

Tahar Chermat, Ahmed Hadroug, and Ali Djouadi

Abstract

تهدف هذه الدراسة إلى قياس أثر التكنولوجيا في انتقال البلدان من مسارات منخفضة ومتوسطة للدخل إلى مسارات أعلى للدخل، والخروج من فخ الدخل المتوسط والدخل المنخفض وإحداث عملية التقارب الاقتصادي، ولتحقيق هدف الدراسة تم اختيار عينة تضم 105 بلدا متباينة اقتصاديا، ومن ثم بناء نموذج لوجيستي خلال الفترة (1990- 2018). ومن أهم ما توصلت إليه الدراسة وجود أثر معنوي للتكنولوجيا في زيادة احتمال انتقال البلدان منخفضة ومتوسطة الدخل إلى مسارات عليا للدخل وإحداث عملية التقارب الاقتصادي فيما بين هذه الدول.

Published
2022

35. Radiotherapy on-chip: microfluidics for translational radiation oncology

Author

Rodin Chermat, Maryam Ziaee, David Y. Mak, Elena Refet-Mollof, Francis Rodier, Philip Wong, Jean-François Carrier, Yuji Kamio, and Thomas Gervais

Subjects
Lab-On-A-Chip Devices, Drug Discovery, Microfluidics, Radiation Oncology, Biomedical Engineering, Humans, Bioengineering, General Chemistry, Precision Medicine, Biochemistry
Abstract

The clinical importance of radiotherapy in the treatment of cancer patients justifies the development and use of research tools at the fundamental, pre-clinical, and ultimately clinical levels, to investigate their toxicities and synergies with systemic agents on relevant biological samples. Although microfluidics has prompted a paradigm shift in drug discovery in the past two decades, it appears to have yet to translate to radiotherapy research. However, the materials, dimensions, design versatility and multiplexing capabilities of microfluidic devices make them well-suited to a variety of studies involving radiation physics, radiobiology and radiotherapy. This review will present the state-of-the-art applications of microfluidics in these fields and specifically highlight the perspectives offered by radiotherapy on-a-chip in the field of translational radiobiology and precision medicine. This body of knowledge can serve both the microfluidics and radiotherapy communities by identifying potential collaboration avenues to improve patient care.

Published
2022

36. Decitabine Versus Hydroxyurea for Advanced Proliferative Chronic Myelomonocytic Leukemia: Results of a Randomized Phase III Trial Within the EMSCO Network

Author

Itzykson, Raphael, primary, Santini, Valeria, additional, Thepot, Sylvain, additional, Ades, Lionel, additional, Chaffaut, Cendrine, additional, Giagounidis, Aristoteles, additional, Morabito, Margot, additional, Droin, Nathalie, additional, Lübbert, Michael, additional, Sapena, Rosa, additional, Nimubona, Stanislas, additional, Goasguen, Jean, additional, Wattel, Eric, additional, Zini, Gina, additional, Torregrosa Diaz, Jose Miguel, additional, Germing, Ulrich, additional, Pelizzari, Anna Maria, additional, Park, Sophie, additional, Jaekel, Nadja, additional, Metzgeroth, Georgia, additional, Onida, Francesco, additional, Navarro, Robert, additional, Patriarca, Andrea, additional, Stamatoullas, Aspasia, additional, Götze, Katharina, additional, Puttrich, Martin, additional, Mossuto, Sandra, additional, Solary, Eric, additional, Gloaguen, Silke, additional, Chevret, Sylvie, additional, Chermat, Fatiha, additional, Platzbecker, Uwe, additional, and Fenaux, Pierre, additional

Published
2022
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38. P095 - Topic: AS06-Prognosis/AS06a-Prognostic factors of outcome and risk assessment: ACCELERATED FAILURE TIME MODELS AS A CLINICALLY PLAUSIBLE WAY TO DESCRIBE SURVIVAL IN MDS – AN ANALYSIS OF THE IWG-PM DATABASE

Author

Pfeilstöcker, M., Tuechler, H., Ades, L., Cermak, J., Chermat, F., Della Porta, M., Fenaux, P., Garcia-Manero, G., Germing, U., Haase, D., Kündgen, A., Lübbert, M., Magalhaes, S., Malcovati, L., Miyazaki, Y., Sanz, G., Santini, V., Sekeres, M., Walter, M., Valent, P., and Greenberg, P.

Published
2023
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39. Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms – results of the EUROPE trial by EMSCO

Author

Anne Sophie Kubasch, Aristoteles Giagounidis, Georgia Metzgeroth, Anna Jonasova, Regina Herbst, Jose Miguel Torregrosa Diaz, Benoit De Renzis, Katharina S. Götze, Marie-Luise Huetter-Kroenke, Marie-Pierre Gourin, Borhane Slama, Sophie Dimicoli-Salazar, Pascale Cony-Makhoul, Kamel Laribi, Sophie Park, Katja Jersemann, Dorothea Schipp, Klaus H. Metzeler, Oliver Tiebel, Katja Sockel, Silke Gloaguen, Anna Mies, Fatiha Chermat, Christian Thiede, Rosa Sapena, Richard F. Schlenk, Pierre Fenaux, Uwe Platzbecker, and Lionel Adès

Subjects
Hemoglobins, Cancer Research, Treatment Outcome, Thrombopoietin, Oncology, Myelodysplastic Syndromes, Neoplasms, Recombinant Fusion Proteins, Humans, Receptors, Fc, Hematology, Biomarkers
Abstract

The EUROPE phase 2 trial investigated the predictive value of biomarkers on the clinical efficacy of single agent romiplostim (ROM) treatment in patients with lower-risk myelodysplastic neoplasms (LR-MDS) and thrombocytopenia within the ‘European Myelodysplastic Neoplasms Cooperative Group‘ (EMSCO) network. A total of 77 patients with LR-MDS and a median platelet count of 25/nl were included, all patients received ROM at a starting dose of 750 μg by SC injection weekly. Thirty-two patients (42%) achieved a hematologic improvement of platelets (HI-P) with a median duration of 340 days. Neutrophil (HI-N) and erythroid (HI-E) responses were observed in three (4%) and seven (9%) patients, respectively. We could not confirm previous reports that HI-P correlated with baseline endogenous thrombopoietin levels and platelet transfusion history, but SRSF2 mutation status and hemoglobin levels at baseline were significantly linked to HI-P. Sequential analysis of variant allelic frequency of mutations like SRSF2 did not reveal an impact of ROM on clonal evolution in both responders and non-responders. In summary, our study confirms the safety and efficacy of ROM in LR-MDS patients and may allow to better define subgroups of patients with a high likelihood of response.

Published
2022

41. Molecular Response Analysis By High Throughput Sequencing in Higher Risk Myelodysplastic Syndrome (HR-MDS) Treated Intensively with CPX-351

Author

Le Bris, Yannick, primary, Bouzy, Simon, additional, Ménard, Audrey, additional, Turlure, Pascal, additional, Chevallier, Patrice, additional, Gourin, Marie-Pierre, additional, Dumas, Pierre-Yves, additional, Thepot, Sylvain, additional, Berceanu, Anna, additional, Park, Sophie, additional, Hospital, Marie-Anne, additional, Cluzeau, Thomas, additional, Torregrosa Diaz, Jose-Miguel, additional, Devron, Louis, additional, Sapena, Rosa, additional, Chermat, Fatiha, additional, Dimicoli Salazar, Sophie, additional, Béné, Marie C, additional, Fenaux, Pierre, additional, and Peterlin, Pierre, additional

Published
2022
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42. Dynamics of Mortality and Transformation Risk within Different Risk Groups of Patients with Myelodysplastic Syndromes Stratified According to the IPSS-R - Comparison of Treated and Untreated Patients and Consequences for the Description of Risk Categories

Author

Pfeilstocker, Michael, primary, Tuechler, Heinz, additional, Ades, Lionel, additional, Cermak, Jaroslav, additional, Chermat, Fatiha, additional, Della Porta, Matteo G., additional, Fenaux, Pierre, additional, Garcia-Manero, Guillermo, additional, Germing, Ulrich, additional, Haase, Detlef, additional, Kuendgen, Andrea, additional, Luebbert, Michael, additional, Magalhaes, Silvia Maria Meira, additional, Malcovati, Luca, additional, Miyazaki, Yasushi, additional, Sanz, Guillermo, additional, Santini, Valeria, additional, Sekeres, Mikkael A., additional, Walter, Matthew J, additional, Valent, Peter, additional, and Greenberg, Peter L, additional

Published
2022
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45. S169: CLINICAL AND MOLECULAR MARKERS FOR PREDICTING RESPONSE TO ROMIPLOSTIM TREATMENT IN LOWER-RISK MYELODYSPLASTIC SYNDROMES

Author

Kubasch, A. S., primary, Giagounidis, A., additional, Metzgeroth, G., additional, Jonasova, A., additional, Herbst, R., additional, Diaz, J. M. T., additional, De Renzis, B., additional, Götze, K. S., additional, Huetter-Kroenke, M.-L., additional, Gourin, M.-P., additional, Slama, B., additional, Dimicoli-Salazar, S., additional, Cony-Makhoul, P., additional, Laribi, K., additional, Park, S., additional, Jersemann, K., additional, Schipp, D., additional, Metzeler, K. H., additional, Tiebel, O., additional, Sockel, K., additional, Gloaguen, S., additional, Mies, A., additional, Chermat, F., additional, Thiede, C., additional, Sapena, R., additional, Schlenk, R. F., additional, Fenaux, P., additional, Platzbecker, U., additional, and Ades, L., additional

Published
2022
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47. Red blood cell transfusion burden in myelodysplastic syndromes ( MDS ) with ring Sideroblasts ( RS ): A retrospective multicenter study by the Groupe Francophone des Myélodysplasies ( GFM )

Author

Jouzier, Claire, primary, Cherait, Amina, additional, Cony‐Makhoul, Pascale, additional, Hamel, Jean‐François, additional, Veloso, Melanie, additional, Thepot, Sylvain, additional, Cluzeau, Thomas, additional, Stamatoullas, Aspasia, additional, Garnier, Alice, additional, Guerci‐Bresler, Agnès, additional, Dimicoli‐Salazar, Sophie, additional, Pica, Gian Matteo, additional, Cheze, Stéphane, additional, Santana, Clémence, additional, Chermat, Fatiha, additional, Fenaux, Pierre, additional, and Park, Sophie, additional

Published
2022
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48. 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

Author

Adès, Lionel, primary, Duployez, Nicolas, additional, Guerci‐Bresler, Agnes, additional, Laribi, Kamel, additional, Peterlin, Pierre, additional, Vey, Norbert, additional, Thepot, Sylvain, additional, Wickenhauser, Stefan, additional, Zerazhi, Hacene, additional, Stamatoullas, Aspassia, additional, Wattel, Eric, additional, Recher, Christian, additional, Toma, Andrea, additional, Dimicoli‐Salazar, Sophie, additional, Braun, Thorsten, additional, Beyne‐Rauzy, Odile, additional, Marolleau, Jean‐Pierre, additional, Cheze, Stéphane, additional, Park, Sophie, additional, Cluzeau, Thomas, additional, Nimubona, Stanislas, additional, Bordessoule, Dominique, additional, Benramdane, Riad, additional, Quesnel, Bruno, additional, Amé, Shanti, additional, de Botton, Stéphane, additional, Chermat, Fathia, additional, Preudhomme, Claude, additional, Chevret, Sylvie, additional, and Fenaux, Pierre, additional

Published
2022
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49. 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

Author

Lionel, Adès, Nicolas, Duployez, Agnes, Guerci-Bresler, Kamel, Laribi, Pierre, Peterlin, Norbert, Vey, Sylvain, Thepot, Stefan, Wickenhauser, Hacene, Zerazhi, Aspassia, Stamatoullas, Eric, Wattel, Christian, Recher, Andrea, Toma, Sophie, Dimicoli-Salazar, Thorsten, Braun, Odile, Beyne-Rauzy, Jean-Pierre, Marolleau, Stéphane, Cheze, Sophie, Park, Thomas, Cluzeau, Stanislas, Nimubona, Dominique, Bordessoule, Riad, Benramdane, Bruno, Quesnel, Shanti, Amé, Stéphane, de Botton, Fathia, Chermat, Claude, Preudhomme, Sylvie, Chevret, Pierre, Fenaux, Hopital Saint-Louis [AP-HP] (AP-HP), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP), Université Paris Cité (UPCité), Cancer Heterogeneity, Plasticity and Resistance to Therapies - UMR 9020 - U 1277 (CANTHER), Institut Pasteur de Lille, Réseau International des Instituts Pasteur (RIIP)-Réseau International des Instituts Pasteur (RIIP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université de Lille-Centre Hospitalier Régional Universitaire [Lille] (CHRU Lille)-Centre National de la Recherche Scientifique (CNRS), Centre Hospitalier Universitaire de Nancy (CHU Nancy), Centre Hospitalier Le Mans (CH Le Mans), Centre hospitalier universitaire de Nantes (CHU Nantes), Nantes Université (Nantes Univ), Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC), Aix Marseille Université (AMU), Centre de Recherche en Cancérologie de Marseille (CRCM), Aix Marseille Université (AMU)-Institut Paoli-Calmettes, Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Fédération nationale des Centres de lutte contre le Cancer (FNCLCC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Stress Adaptation and Tumor Escape - SATE (CRCI2NA / Eq 7), Centre de Recherche en Cancérologie et Immunologie Intégrée Nantes-Angers (CRCI2NA ), Université d'Angers (UA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Nantes Université - UFR de Médecine et des Techniques Médicales (Nantes Univ - UFR MEDECINE), Nantes Université - pôle Santé, Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ)-Nantes Université - pôle Santé, Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ)-Université d'Angers (UA)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Nantes Université - UFR de Médecine et des Techniques Médicales (Nantes Univ - UFR MEDECINE), Nantes Université (Nantes Univ)-Nantes Université (Nantes Univ), Centre Hospitalier Universitaire de Nîmes (CHU Nîmes), Centre Hospitalier Henri Duffaut (Avignon), Laboratoire de biologie et modélisation de la cellule (LBMC UMR 5239), École normale supérieure de Lyon (ENS de Lyon)-Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS), Service Hématologie - IUCT-Oncopole [CHU Toulouse], Pôle Biologie [CHU Toulouse], Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Centre Hospitalier Universitaire de Toulouse (CHU Toulouse)-Pôle IUCT [CHU Toulouse], Centre Hospitalier Universitaire de Toulouse (CHU Toulouse), Hôpital Henri Mondor, Biothérapies des maladies génétiques et cancers, Université Bordeaux Segalen - Bordeaux 2-Institut National de la Santé et de la Recherche Médicale (INSERM), Charité - UniversitätsMedizin = Charité - University Hospital [Berlin], CHU Amiens-Picardie, HEMATIM - Hématopoïèse et immunologie - UR UPJV 4666 (HEMATIM), Université de Picardie Jules Verne (UPJV)-CHU Amiens-Picardie-Institut National de la Santé et de la Recherche Médicale (INSERM), CHU Caen, Normandie Université (NU)-Tumorothèque de Caen Basse-Normandie (TCBN), CHU Grenoble, XLIM (XLIM), Université de Limoges (UNILIM)-Centre National de la Recherche Scientifique (CNRS), CHU Pontchaillou [Rennes], Contrôle de la Réponse Immune B et des Lymphoproliférations (CRIBL), Institut National de la Santé et de la Recherche Médicale (INSERM)-Centre National de la Recherche Scientifique (CNRS)-Institut Génomique, Environnement, Immunité, Santé, Thérapeutique (GEIST), Université de Limoges (UNILIM)-Université de Limoges (UNILIM), Centre Hospitalier René Dubos [Pontoise], Département d'Oncologie et Hématologie [Strasbourg], Les Hôpitaux Universitaires de Strasbourg (HUS), Institut Gustave Roussy (IGR), Département d'hématologie [Gustave Roussy], Equipe 2 : ECSTRA - Epidémiologie Clinique, STatistique, pour la Recherche en Santé (CRESS - U1153), Centre de Recherche Épidémiologie et Statistique Sorbonne Paris Cité (CRESS (U1153 / UMR_A_1125 / UMR_S_1153)), Conservatoire National des Arts et Métiers [CNAM] (CNAM), HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-Université Sorbonne Paris Cité (USPC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE)-Conservatoire National des Arts et Métiers [CNAM] (CNAM), and HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-HESAM Université - Communauté d'universités et d'établissements Hautes écoles Sorbonne Arts et métiers université (HESAM)-Université Sorbonne Paris Cité (USPC)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Université Paris Cité (UPCité)-Institut National de Recherche pour l’Agriculture, l’Alimentation et l’Environnement (INRAE)

Subjects
Leukemia, Myeloid, Acute, Treatment Outcome, Valproic Acid, Antineoplastic Combined Chemotherapy Protocols, Mutation, Azacitidine, Humans, Idarubicin, Lenalidomide, [SDV.MHEP]Life Sciences [q-bio]/Human health and pathology
Abstract

International audience; In order to improve the outcome observed with azacitidine (AZA) in higher-risk Myelodysplastic syndrome (MDS), its combination with other drugs in MDS must be evaluated. So far, no combination has not been shown to be more effective than AZA alone. AZA-PLUS was a phase II trial that, in a "pick a winner" approach, randomly assigned patients with higher-risk MDS, CMML and low blast count AML to: AZA; AZA plus lenalidomide; AZA plus Valproic Acid or AZA plus Idarubicin. 322 patients were included. After six\,cycles, 69 (21.4%) CR\,+\,PR were observed with no benefit from any combination. Median EFS and OS were 17.2 and 19.7\,months in the whole cohort, respectively, with no difference across randomised arms. Infection and rates of hospitalisation during the first six\,cycles were higher in the AZA-LEN And AZA-IDA arm, related to increased myelosuppression. Factors associated with better response were IPSS, favourable or intermediate karyotype, haemoglobin, lower circulating blast count, fibrinogen level and lower LDH, while poorer survival was seen in therapy-related MDS and, in the case of TP53, PTPN11 or CSF3R mutation. The combinations used did not improve the outcome obtained with AZA alone. However, our "pick a winner" randomised strategy may remain useful with potentially more active drugs to be tested in combination with AZA.

Published
2022

50. Core set of patient-reported outcomes for myelodysplastic syndromes: an EUMDS Delphi study involving patients and hematologists

Author

Igor Stojkov, Hege Garelius, Pierre Fenaux, Marjan Arvandi, Elvira Mora, Fabio Efficace, Theo de Witte, David T. Bowen, Argiris Symeonidis, Annette Conrads-Frank, Karin A Koinig, Corine van Marrewijk, Reinhard Stauder, Fatiha Chermat, Sibylle Puntscher, Ursula Rochau, Moshe Mittelman, and Uwe Siebert

Subjects
medicine.medical_specialty, Activities of daily living, Delphi Technique, Cancer development and immune defence Radboud Institute for Molecular Life Sciences [Radboudumc 2], education, Delphi method, European LeukemiaNet, All institutes and research themes of the Radboud University Medical Center, Quality of life, hemic and lymphatic diseases, Activities of Daily Living, Health care, medicine, Humans, Patient Reported Outcome Measures, Prospective Studies, Rank correlation, business.industry, Myelodysplastic syndromes, Health Services and Outcomes, Hematology, medicine.disease, humanities, Myelodysplastic Syndromes, Family medicine, Quality of Life, Mann–Whitney U test, business
Abstract

Key Points Systematically developed set of core PROs in MDS, involving patients and hematologists in a 2-round Delphi survey.Core PROs will support unified outcome measurement and facilitate inclusion of reliable patient information in MDS disease care., Visual Abstract, Patient-reported outcomes (PROs) are relevant and valuable end points in the care of patients with myelodysplastic syndromes (MDS). However, a consensus-based selection of PROs for MDS, derived by both patients and hematologists, is lacking. We aimed to develop a core set of PROs for patients with MDS as part of the prospective European LeukemiaNet MDS (EUMDS) Registry. According to international guidelines, candidate PROs were identified from a comprehensive literature search of MDS studies. Overall, 40 PROs were selected and evaluated in a two-round Delphi survey by 40 patients with MDS and 38 hematologists in the first round and 38 patients and 32 hematologists in the second round. Based on an agreement scale and predefined inclusion criteria, both patients and hematologists selected “general quality of life” as a core PRO. Hematologists also selected “transfusion-dependency burden” and “ability to work/activities of daily living” as core PROs. The second Delphi round increased PRO rating agreements. Statistically significant rating differences between patients and hematologists were observed for 28 PROs (Mann-Whitney U test; P < .05) in the first round and for 19 PROs in the second round, with “disease knowledge” and “confidence in health care services” rated notably higher by patients. The overall mean PRO ratings correlation between the 2 groups was moderate (Spearman’s rank correlation coefficient = 0.5; P < .05). This first consensus on a core set of PROs jointly developed by patients and hematologists forms the basis for patient-centered care in daily practice and clinical research.

Published
2022

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