Quality of Life and Symptoms in Patients With Newly Diagnosed Myelodysplastic Syndromes

Myelodysplastic Syndromes Clinical Trial

— PROMYS  

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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT00809575
• Other study ID #: QOL-MDS0108
• Secondary ID: GIMEMA-QOL-MDS-0
• Status: Active, not recruiting
• Start date: October 2, 2008
• Est. completion date: February 2021

Study information

• Verified date: December 2020
• Source: Gruppo Italiano Malattie EMatologiche dell'Adulto
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Other known NCT identifiers

• NCT00872768

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 927
• Est. completion date: February 2021
• Est. primary completion date: October 8, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Adult
• Myelodysplastic Syndromes
• Preleukemia
• Syndrome

Intervention

Other:
• questionnaire administration

• fatigue assessment and management

• observation

• quality-of-life assessment

Locations

Country	Name	City	State
Austria	Innsbruck University Hospital	Innsbruck
Belgium	ZNA Middelheim	Antwerpen
Belgium	AZ Sint-Jan AV, Department of Hematology	Brugge
Belgium	CH Jolimont	Bruxelles
Belgium	CHC - Centre Hospitalier Chrétien	Liege
Brazil	Curitiba Unidade de Hematologia - Hemoterapia e Oncologia. Setor de Transplante de Medula Ossea. Complexo Hospital de Clinicas da Universidade Federal do Parana (CHC-UFPR)	Curitiba
China	Affiliated Hospital of Liaoning University of Traditional Chinese Medicine	China
China	Kunming Medical University	Kunming
Croatia	Hematology - Clinical Hospital Centre Rijeka - Faculty of Medicine - University of Rijeka	Rijeka
Czechia	General Teaching Hospital	Prague
France	Saint - Cloud Medecin des CLCC Hematologie Service E Hopital ReneHuguenin - Institut Curie	Saint-Cloud
France	Purpan Hospital - Internal Medicine Department	Toulouse
Germany	Department of Hematology - Universitatsklinikum Carl Gustav Carus	Dresden
Germany	University of Freiburg Medical Center	Freiburg
Italy	Asl Di Asti - Ospedali Riuniti - Presidio Ospedaliero Cardinal G. Massaia - Sc Oncologia	Asti
Italy	Unità Operativa Ematologia 1 - Università degli Studi di Bari	Bari
Italy	ASL N.8 - Ospedale "A. Businco" - Unità Operativa di Ematologia e Trapianto di Midollo	Cagliari
Italy	CTMO - Ematologia - Ospedale "Binaghi"	Cagliari
Italy	Ospedale "Ferrarotto"	Catania
Italy	A.O. Pugliese Ciaccio	Catanzaro
Italy	Aou Arcispedale Sant'Anna - Uoc Ematologia E Fisiopatologia Della Coagulazione	Cona
Italy	Aou Careggi - Firenze - Sod Ematologia	Firenze
Italy	Divisione Ematologia - Azienda Ospedaliera Universitaria "San Martino""	Genova
Italy	U.O.A. di Medicina Trasfusionale e DH di Ematologia - ASL TO 4 Ospedale di Ivrea	Ivrea	Torino
Italy	Divisione di Ematologia Ospedale "Santa Maria Goretti"	Latina
Italy	ASL Le1 P.O. Vito Fazzi - U.O. di Ematologia	Lecce
Italy	Istituto Scientifico Romagnoli per lo Studio e la Cura dei Tumori- IRST	Meldola
Italy	Ospedale Niguarda "Ca Granda"	Milano
Italy	U.O. Ematologia e Trapianto di Midollo - Ist. Scientifico Ospedale San Raffaele	Milano
Italy	UO Ematologia - AOU Policlinico di Modena	Modena
Italy	Asst Di Monza - Ospedale S. Eugenio - Uo Ematologia E Cta	Monza
Italy	S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro	Novara
Italy	Ospedale S. Luigi Gonzaga	Orbassano
Italy	Aou Di Parma - Sc Ematologia E Centro Trapianti Midollo Osseo	Parma
Italy	Med. Int. ed Oncologia Medica IRCCS Policlinico S. Matteo	Pavia
Italy	Asl Di Piacenza - Ospedale "Guglielmo Da Saliceto" - Ematologia E Centro Trapianti	Piacenza
Italy	Ausl Di Reggio Emilia - Arcispedale Santa Maria Nuova - Irccs - Sc Ematologia	Reggio Emilia
Italy	Complesso Ospedaliero S. Giovanni Addolorata	Roma
Italy	Divisione Ematologia - Università Campus Bio-Medico	Roma
Italy	Università Cattolica del Sacro Cuore - Policlinico A. Gemelli	Roma
Italy	Università degli Studi "La Sapienza" - Dip. Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia	Roma
Italy	Università degli Studi - Policlinico di Tor Vergata	Roma
Italy	U.O.C. Ematologia - Ospedale S.Eugenio	Rome
Italy	SOS Dipartimento di Oncoematologia Osp. Rovigo	Rovigo
Italy	Istituto di Ematologia - IRCCS Ospedale Casa Sollievo della Sofferenza	San Giovanni Rotondo	Foggia
Italy	Ematologia - Dipartimento di Medicina Clinica e Sperimentale	Sassari
Italy	Aou Città Della Salute E Della Scienza - Ospedale S. Giovanni Battista Molinette	Torino
Taiwan	National Taiwan University	Taipei
United Kingdom	Bradford Institute of Health Research	Bradford
United Kingdom	St James's University Hospital	Leeds
United Kingdom	London North West Healthcare NHS Trust	London
United States	The University of Texas, MD Anderson Cancer Center	Houston	Texas
United States	Memorial Sloan-Kettering Cancer Center (MSKCC)	New York	New York

Sponsors (1)

Lead Sponsor	Collaborator
Gruppo Italiano Malattie EMatologiche dell'Adulto

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Brazil,  China,  Croatia,  Czechia,  France,  Germany,  Italy,  Taiwan,  United Kingdom, 

References & Publications (6)

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. 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. — View 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. — View 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. — View Citation

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. 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. — View 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. — View 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To investigate the prognostic value of baseline patients' reported fatigue for overall survival in newly diagnosed myelodysplastic syndrome (MDS) patients.		After 5 years from study entry.
Secondary	To investigate the prognostic value of changes overtime of QoL and symptoms for clinical outcomes.	i.e. overall survival, AML transformation, toxicity and response to therapy.	After 5 years from study entry.
Secondary	To describe prospectively short and long-term symptom burden and QoL outcomes by risk group and by type of therapy.		After 5 years from study entry.
Secondary	To compare the QoL of lower risk patients who maintain stable disease versus those who have progressed to AML or higher-risk score categories.		After 5 years from study entry.
Secondary	To compare the QoL and symptoms baseline reference data to be used as benchmarks for comparisons in clinical trials.		After 5 years from study entry.
Secondary	To establish international QoL and symptoms baseline reference data to be used as benchmarks for comparisons in clinical trials.		After 5 years from study entry.
Secondary	To evaluate the impact of transfusion dependency at baseline and over time on survival and QoL outcomes.		After 5 years from study entry.
Secondary	To devise a prognostic patient-based index.		After 5 years from study entry.
Secondary	To evaluate accuracy of clinical prediction of survival.		After 5 years from study entry.
Secondary	To assess patients' preferences for involvement in treatment decision-making across different risk group and examine relationships between preferences and patient characteristics.		After 5 years from study entry.