Clinical trial NCT00809575: Quality of Life and Symptoms in Patients With Newly Diagnosed Myelodysplastic Syndromes
Official title: Prognostic Significance and Longitudinal Assessment of Patient-reported Quality of Life and Symptoms in Myelodysplastic Syndromes. A Large-scale International, Observational Study: PROMYS Study
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Adult
Myelodysplastic Syndromes
Keywords: adult
de novo myelodysplastic syndromes
Quality of life
Patient reported outcomes
Lead sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor class: OTHER
First posted: 2008-12-17
Enrollment: 927
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients with myelodysplastic syndromes (MDS).

Brief summary: RATIONALE: Gathering information about quality of life, fatigue, and other symptoms from patients with myelodysplastic syndromes may help doctors learn more about the disease and may help plan treatment.

PURPOSE: This clinical trial is studying quality of life and symptoms in patients with newly diagnosed myelodysplastic syndromes.

Detailed description: This study will ultimately aim at providing the scientific community with additional patient-reported health status data to support and further facilitate the clinical decision-making process. This project has thus a number of goals. The main objective of the protocol is to improve our understanding of the possible added prognostic value of patients' judgment on their own health status and its potential clinical implications. This would aim at providing clinicians with an easy and brief to administer patient-reported health status scale or tool to be used to make more informed treatment decisions. In addition, along with other recent evidence (also looking at the prognostic value of patients' health status judgment in MDS patients), the data of this research could possibly serve to devise a patient-based prognostic index to be used in this higher risk population.

Eligibility criteria: Inclusion criteria

* Patients with newly diagnosed myelodysplastic syndrome (MDS) according to 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; EQ5D; FACIT-Fatigue and Control Preference Scale).
* Adult patients (≥ 18 years old).
* Written informed consent provided.

Exclusion criteria

* Patients who have received prior treatment other than platelets or RBC transfusions, iron chelation, antibiotic/virostatic drugs, vitamins;
* Patients with therapy related MDS.
* Having any kind of psychiatric disorder or major cognitive dysfunction.
* Not able to read and understand local language.

Primary outcomes:
measure: To investigate the prognostic value of baseline patients' reported fatigue for overall survival in newly diagnosed myelodysplastic syndrome (MDS) patients.
timeFrame: After 5 years from study entry.
Secondary outcomes:
measure: To investigate the prognostic value of changes overtime of QoL and symptoms for clinical outcomes.
timeFrame: After 5 years from study entry.
measure: To describe prospectively short and long-term symptom burden and QoL outcomes by risk group and by type of therapy.
timeFrame: After 5 years from study entry.
measure: To compare the QoL of lower risk patients who maintain stable disease versus those who have progressed to AML or higher-risk score categories.
timeFrame: After 5 years from study entry.
measure: To compare the QoL and symptoms baseline reference data to be used as benchmarks for comparisons in clinical trials.
timeFrame: After 5 years from study entry.
measure: To establish international QoL and symptoms baseline reference data to be used as benchmarks for comparisons in clinical trials.
timeFrame: After 5 years from study entry.
measure: To evaluate the impact of transfusion dependency at baseline and over time on survival and QoL outcomes.
timeFrame: After 5 years from study entry.
measure: To devise a prognostic patient-based index.
timeFrame: After 5 years from study entry.
measure: To evaluate accuracy of clinical prediction of survival.
timeFrame: After 5 years from study entry.
measure: To assess patients' preferences for involvement in treatment decision-making across different risk group and examine relationships between preferences and patient characteristics.
timeFrame: After 5 years from study entry.
Other outcomes:
Locations:
city: New York
country: United States
facility: Memorial Sloan-Kettering Cancer Center (MSKCC)
state: New York
city: Houston
country: United States
facility: The University of Texas, MD Anderson Cancer Center
state: Texas
city: Innsbruck
country: Austria
facility: Innsbruck University Hospital
city: Antwerpen
country: Belgium
facility: ZNA Middelheim
city: Brugge
country: Belgium
facility: AZ Sint-Jan AV, Department of Hematology
city: Bruxelles
country: Belgium
facility: CH Jolimont
city: Liege
country: Belgium
facility: CHC - Centre Hospitalier Chrétien
city: Curitiba
country: Brazil
facility: Curitiba Unidade de Hematologia - Hemoterapia e Oncologia. Setor de Transplante de Medula Ossea. Complexo Hospital de Clinicas da Universidade Federal do Parana (CHC-UFPR)
city: China
country: China
facility: Affiliated Hospital of Liaoning University of Traditional Chinese Medicine
city: Kunming
country: China
facility: Kunming Medical University
city: Rijeka
country: Croatia
facility: Hematology - Clinical Hospital Centre Rijeka - Faculty of Medicine - University of Rijeka
city: Prague
country: Czechia
facility: General Teaching Hospital
city: Saint-Cloud
country: France
facility: Saint - Cloud Medecin des CLCC Hematologie Service E Hopital ReneHuguenin - Institut Curie
city: Toulouse
country: France
facility: Purpan Hospital - Internal Medicine Department
city: Dresden
country: Germany
facility: Department of Hematology - Universitatsklinikum Carl Gustav Carus
city: Freiburg
country: Germany
facility: University of Freiburg Medical Center
city: San Giovanni Rotondo
country: Italy
facility: Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza
state: Foggia
city: Ivrea
country: Italy
facility: U.O.A. di Medicina Trasfusionale e DH di Ematologia - ASL TO 4 Ospedale di Ivrea
state: Torino
city: Asti
country: Italy
facility: Asl Di Asti - Ospedali Riuniti - Presidio Ospedaliero Cardinal G. Massaia - Sc Oncologia
city: Bari
country: Italy
facility: Unità Operativa Ematologia 1 - Università degli Studi di Bari
city: Cagliari
country: Italy
facility: ASL N.8 - Ospedale "A. Businco" - Unità Operativa di Ematologia e Trapianto di Midollo
city: Cagliari
country: Italy
facility: CTMO - Ematologia - Ospedale "Binaghi"
city: Catania
country: Italy
facility: Ospedale "Ferrarotto"
city: Catanzaro
country: Italy
facility: A.O. Pugliese Ciaccio
city: Cona
country: Italy
facility: Aou Arcispedale Sant'Anna - Uoc Ematologia E Fisiopatologia Della Coagulazione
city: Firenze
country: Italy
facility: Aou Careggi - Firenze - Sod Ematologia
city: Genova
country: Italy
facility: Divisione Ematologia - Azienda Ospedaliera Universitaria "San Martino""
city: Latina
country: Italy
facility: Divisione di Ematologia Ospedale "Santa Maria Goretti"
city: Lecce
country: Italy
facility: ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia
city: Meldola
country: Italy
facility: Istituto Scientifico Romagnoli per lo Studio e la Cura dei Tumori- IRST
city: Milano
country: Italy
facility: U.O. Ematologia e Trapianto di Midollo - Ist. Scientifico Ospedale San Raffaele
city: Milano
country: Italy
facility: Ospedale Niguarda "Ca Granda"
city: Modena
country: Italy
facility: UO Ematologia - AOU Policlinico di Modena
city: Monza
country: Italy
facility: Asst Di Monza - Ospedale S. Eugenio - Uo Ematologia E Cta
city: Novara
country: Italy
facility: S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
city: Orbassano
country: Italy
facility: Ospedale S. Luigi Gonzaga
city: Parma
country: Italy
facility: Aou Di Parma - Sc Ematologia E Centro Trapianti Midollo Osseo
city: Pavia
country: Italy
facility: Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo
city: Piacenza
country: Italy
facility: Asl Di Piacenza - Ospedale "Guglielmo Da Saliceto" - Ematologia E Centro Trapianti
city: Reggio Emilia
country: Italy
facility: Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova - Irccs - Sc Ematologia
city: Roma
country: Italy
facility: Divisione Ematologia - Università Campus Bio-Medico
city: Roma
country: Italy
facility: Università degli Studi - Policlinico di Tor Vergata
city: Roma
country: Italy
facility: Università degli Studi "La Sapienza" - Dip. Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia
city: Roma
country: Italy
facility: Università Cattolica del Sacro Cuore - Policlinico A. Gemelli
city: Roma
country: Italy
facility: Complesso Ospedaliero S. Giovanni Addolorata
city: Rome
country: Italy
facility: U.O.C. Ematologia - Ospedale S.Eugenio
city: Rovigo
country: Italy
facility: SOS Dipartimento di Oncoematologia Osp. Rovigo
city: Sassari
country: Italy
facility: Ematologia - Dipartimento di Medicina Clinica e Sperimentale
city: Torino
country: Italy
facility: Aou Città Della Salute E Della Scienza - Ospedale S. Giovanni Battista Molinette
city: Taipei
country: Taiwan
facility: National Taiwan University
city: Bradford
country: United Kingdom
facility: Bradford Institute of Health Research
city: Leeds
country: United Kingdom
facility: St James's University Hospital
city: London
country: United Kingdom
facility: London North West Healthcare NHS Trust
Study officials:
affiliation: Gruppo Italiano Malattie EMatologiche dell'Adulto
name: Fabio Efficace, PhD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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. Preference for involvement in treatment decisions and request for prognostic information in newly diagnosed patients with higher-risk myelodysplastic syndromes. Ann Oncol. 2014 Feb;25(2):447-54. doi: 10.1093/annonc/mdt557.
citation: 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, Lubbert M, Sanpaolo G, Buccisano F, Ricco A, Palumbo GA, Niscola P, Zhang H, Fenu S, La Nasa G, Mandelli F, Efficace F. Accuracy of physician assessment of treatment preferences and health status in elderly patients with higher-risk myelodysplastic syndromes. Leuk Res. 2015 Aug;39(8):859-65. doi: 10.1016/j.leukres.2015.05.012. Epub 2015 Jun 12.
citation: Efficace F, Gaidano G, Breccia M, Criscuolo M, Cottone F, Caocci G, Bowen D, Lubbert 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. Prevalence, severity and correlates of fatigue in newly diagnosed patients with myelodysplastic syndromes. Br J Haematol. 2015 Feb;168(3):361-70. doi: 10.1111/bjh.13138. Epub 2014 Oct 1.
citation: 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. Prognostic value of self-reported fatigue on overall survival in patients with myelodysplastic syndromes: a multicentre, prospective, observational, cohort study. Lancet Oncol. 2015 Nov;16(15):1506-1514. doi: 10.1016/S1470-2045(15)00206-5. Epub 2015 Sep 21.
citation: Efficace F, Cottone F, Abel G, Niscola P, Gaidano G, Bonnetain F, Anota A, Caocci G, Cronin A, Fianchi L, Breccia M, Stauder R, Platzbecker U, Palumbo GA, Luppi M, Invernizzi R, Bergamaschi M, Borin L, Di Tucci AA, Zhang H, Sprangers M, Vignetti M, Mandelli F. Patient-reported outcomes enhance the survival prediction of traditional disease risk classifications: An international study in patients with myelodysplastic syndromes. Cancer. 2018 Mar 15;124(6):1251-1259. doi: 10.1002/cncr.31193. Epub 2017 Dec 12.
citation: Efficace F, Cottone F, Oswald LB, Cella D, Patriarca A, Niscola P, Breccia M, Platzbecker U, Palumbo GA, Caocci G, Stauder R, Ricco A, Petranovic D, Caers J, Luppi M, Fianchi L, Frairia C, Capodanno I, Follini E, Sarlo C, Fazi P, Vignetti M. The IPSS-R more accurately captures fatigue severity of newly diagnosed patients with myelodysplastic syndromes compared with the IPSS index. Leukemia. 2020 Sep;34(9):2451-2459. doi: 10.1038/s41375-020-0746-8. Epub 2020 Feb 21.