Prognostic significance and longitudinal assessment of Patient-Reported Outcomes in MYelodysplastic Syndromes (PROMYS): PROMYS International Registry
Working Writing Committee
Fabio Efficace, Gianluca Gaidano, David Bowen, Michael Lübbert, Frédéric Baron, Emanuele Angelucci, Maria Teresa Voso, Massimo Breccia, Marco Vignetti, Sergio Amadori , Franco Mandelli
Study and Project Management:
Laura Cannella,
GIMEMA Data Centre, Rome
Biostatistician :
Francesco Cottone,
GIMEMA Data Centre, Rome
Regulatory Affairs & Data Manager:
Francesca Tartaglia,
GIMEMA Data Centre, Rome
Primary objective:
To investigate the prognostic value of baseline patients’ reported fatigue for overall survival in newly diagnosed myelodysplastic syndrome (MDS) patients.
Inclusion criteria:
– Patients with newly diagnosed myelodysplastic syndrome (MDS) according to 2008 WHO classification with any known IPSS risk score category. The initial diagnosis of MDS is acceptable within 3 months before date of registration.
– Having a full baseline QoL evaluation completed (i.e. EORTC QLQ-C30; EQ-5D, FACIT-Fatigue and Control Preference Scale).
– Adult patients (≥ 18 years old).
-Written informed consent provided.
Sample Size :
The overall sample based on the primary endpoint is 1070.
Study duration:
The recruitment period is estimated in five years and follow-up period is planned over five years (from date of registration).
Full Length Publications
Preference for involvement in treatment decisions and request for prognostic information in newly diagnosed patients with higher-risk myelodysplastic syndromes.Efficace F, Gaidano G, Sprangers M, Cottone F, Breccia M, Voso MT, Caocci G, Stauder R, Di Tucci AA, Sanpaolo G, Selleslag D, Angelucci E, Platzbecker U, Mandelli F.Ann Oncol. 2014 Feb;25(2):447-54.
Prevalence, severity and correlates of fatigue in newly diagnosed patients with myelodysplastic syndromes. Efficace F, Gaidano G, Breccia M, Criscuolo M, Cottone F, Caocci G, Bowen D, Lübbert M, Angelucci E, Stauder R, Selleslag D, Platzbecker U, Sanpaolo G, Jonasova A, Buccisano F, Specchia G, Palumbo GA, Niscola P, Wan C, Zhang H, Fenu S, Klimek V, Beyne-Rauzy O, Nguyen K, Mandelli F. Br J Haematol. 2015 Feb;168(3):361-70.
Accuracy of physician assessment of treatment preferences and health status in elderly patients with higher-risk myelodysplastic syndromes. Caocci G, Voso MT, Angelucci E, Stauder R, Cottone F, Abel G, Nguyen K, Platzbecker U, Beyne-Rauzy O, Gaidano G, Invernizzi R, Molica S, Criscuolo M, Breccia M, Lübbert M, Sanpaolo G, Buccisano F, Ricco A, Palumbo GA, Niscola P, Zhang H, Fenu S, La Nasa G, Mandelli F, Efficace F. Leuk Res. 2015 Aug;39(8):859-65.
Prognostic value of self-reported fatigue on overall survival in patients with myelodysplastic syndromes: a multicentre, prospective, observational, cohort study. Efficace F, Gaidano G, Breccia M, Voso MT, Cottone F, Angelucci E, Caocci G, Stauder R, Selleslag D, Sprangers M, Platzbecker U, Ricco A, Sanpaolo G, Beyne-Rauzy O, Buccisano F, Palumbo GA, Bowen D, Nguyen K, Niscola P, Vignetti M, Mandelli F. Lancet Oncol. 2015 Nov;16(15):1506-14.
Conference abstract
Accuracy of physician assessment of treatment preferences and health status in patients with higher-risk myelodysplastic syndromes Caocci G, Voso MT, Angelucci E, Stauder R, Cottone F, Abel G, Nguyen KT, Platzbecker Y, Beyne-Rauzy O, Gaidano G, Invernizzi R, Molica S, Criscuolo M, Breccia M, Lübbert M, Sanpaolo G, Buccisano F, Ricco A, Palumbo G, Niscola P, Zhang H, Fenu S, La Nasa G, Mandelli F, Efficace F. 20th Congress of the European Hematology Association (EHA), Vienna Jun 12, 2015; in press
Age and Gender-Related Pretreatment Quality of Life Profiles in Patients with Higher-Risk Myelodysplastic Syndromes. Establishing Benchmark Data from an International Study. Fabio Efficace, Gianluca Gaidano, Massimo Breccia, Pasquale Niscola, Francesco Cottone, Luana Fianchi, Maria Teresa Voso, Giovanni Caocci, Francesco Buccisano, Emanuele Angelucci, Valeria Santini, Reinhard Stauder, Uwe Platzbecker, Alessandra Ricco, Nicola di Renzo, David Bowen, Franco Mandelli. 57th Annual Meeting and Exposition of the American Society of Hematology (ASH), Orlando, (FL), USA. December, 2015.
Profiling patients with high-risk myelodysplastic syndromes who prefer to receive survival prognostic information. Final results from a large international prospective study. F Efficace,G Gaidano, M Breccia, M Voso, G Caocci, R Stauder, E Angelucci, D Selleslag, M Sanpaolo, U Platzbecker, A Jonasova, B Rauzy, F Buccisano, A Ricco, G Palumbo, D Bowen, K Nguyen, P Niscola, Z Huiyong, C Wan, M Luebbert, S Fenu, S Molica, C Balduini, C Focan, F Ciceri, N Renzo, W Chie, S Ackroyd, M Lunghi, V Klimek, F Cottone, M Vignetti, F Mandelli. 18th Congress of the European Hematology Association, Stockholm, Sweden. Hematologica, June 2013, Volume 98, Issue supplement 1 (abs. 1081).
Patient-Reported Fatigue, Functional Aspects and Quality of Life in Elderly Patients with High-Risk Myelodysplastic Syndromes. Evidence From a Large Prospective International Study. F. Efficace, G. Gaidano, M.T. Voso, G. Caocci, M. Breccia, R. Stauder, D. Selleslag, G. Sanpaolo, U. Platzbecker, M. Criscuolo, A. Di Tucci, M. Lunghi, A. Jonasova, F. Buccisano, A. Ricco, Z. Huiyoung, D. Bowen, G.A. Palumbo, P. Niscola, F. Cottone, M. Vignetti , F. Mandelli. 54th Annual Meeting and Exposition of the American Society of Hematology (ASH), Atlanta, (GA), USA. Blood (ASH Annual Meeting Abstracts), 120: Abstract 3163, 2012.
Health related quality of life profile and request of prognostic information on survival at the time of diagnosis in patients with high-risk myelodysplastic syndromes. Fabio Efficace, Gianluca Gaidano, Reinhard Stauder, Giovanni Caocci, Maria Teresa Voso, Marianna Criscuolo, Dominik Selleslag, Anna Angela Di Tucci, Monia Lunghi, Massimo Breccia, Francesco Buccisano, Uwe Platzbecker, Francesco Cottone et al. 53rd Annual Meeting of the American Society of Hematology (ASH), San Diego, (CA), USA. Blood (ASH Annual Meeting Abstracts) 118: (abs.2078), 2011
Involvement in treatment decisions, desire for prognostic information and quality of life in high-risk myelodysplastic syndromes: the physician’s perspective. F. Efficace, G. Alimena, M. T. Voso, G. Caocci, A. Di Tucci, R. Stauder, G. Sanpaolo, G. Gaidano, M. Breccia, D. Selleslag, O. Beyne-Rauzy, S. Wood, F. Ciceri, G. Specchia, D. Bowen, F. Buccisano, B. Deschler, R. Neuwirtova, C. Focan, S. Ackroyd, M. Criscuolo, R. Invernizzi, C. Ravoet, R. Paolini, G. Matta, S. Fenu, R. De Bock, E. Morra, F. Cottone, M. Vignetti, F. Mandelli. 11th International Symposium on Myelodysplastic Syndromes. Edinburgh, (Scotland). Leukemia Research 35 (S1), S57 (abs.147), 2011.
Investigating preferences and factors associated with involvement in treatment decision making in newly diagnosed patients with high-risk myelodysplastic syndromes: an international multicenter study. Efficace F, Gaidano G, Voso MT, Caocci G, Breccia M, Di Tucci A, Stauder R, Selleslag D, Wood S, Wan C, Sanpaolo G, Lunghi M, Balducci L, Cottone F, Vignetti M, Mandelli F. 52nd Annual Meeting of the American Society of Hematology (ASH), Orlando, (FL), USA. Blood (ASH Annual Meeting Abstracts) 116: (abs.4953), 2010.
Investigating pretreatment quality of life and symptomatology in newly diagnosed high-risk patients with myelodysplasia.F Efficace, E Angelucci, M Sprangers, G Gaidano, G Caocci, M Breccia, MT Voso, R Stauder, D Selleslag, M Lübbert, N Cascavilla,B Rauzy, W Chonghua, G Specchia, W Chie, C Focan, F Cottone, et al.15th Congress of the European Hematology Association, Barcellona, Spain. Hematologica, June 2010, Volume 95, Issue supplement 2 (abs. 0460).