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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT00809575




Registration number
NCT00809575
Ethics application status
Date submitted
16/12/2008
Date registered
17/12/2008
Date last updated
22/12/2020

Titles & IDs
Public title
Quality of Life and Symptoms in Patients With Newly Diagnosed Myelodysplastic Syndromes
Scientific 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
Secondary ID [1] 0 0
GIMEMA-QOL-MDS-0108
Secondary ID [2] 0 0
QOL-MDS0108
Universal Trial Number (UTN)
Trial acronym
PROMYS
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Adult 0 0
Myelodysplastic Syndromes 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - questionnaire administration
Other interventions - fatigue assessment and management
Other interventions - observation
Other interventions - quality-of-life assessment

Other interventions: questionnaire administration


Other interventions: fatigue assessment and management


Other interventions: observation


Other interventions: quality-of-life assessment
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To investigate the prognostic value of baseline patients' reported fatigue for overall survival in newly diagnosed myelodysplastic syndrome (MDS) patients.
Timepoint [1] 0 0
After 5 years from study entry.
Secondary outcome [1] 0 0
To investigate the prognostic value of changes overtime of QoL and symptoms for clinical outcomes.
Timepoint [1] 0 0
After 5 years from study entry.
Secondary outcome [2] 0 0
To describe prospectively short and long-term symptom burden and QoL outcomes by risk group and by type of therapy.
Timepoint [2] 0 0
After 5 years from study entry.
Secondary outcome [3] 0 0
To compare the QoL of lower risk patients who maintain stable disease versus those who have progressed to AML or higher-risk score categories.
Timepoint [3] 0 0
After 5 years from study entry.
Secondary outcome [4] 0 0
To compare the QoL and symptoms baseline reference data to be used as benchmarks for comparisons in clinical trials.
Timepoint [4] 0 0
After 5 years from study entry.
Secondary outcome [5] 0 0
To establish international QoL and symptoms baseline reference data to be used as benchmarks for comparisons in clinical trials.
Timepoint [5] 0 0
After 5 years from study entry.
Secondary outcome [6] 0 0
To evaluate the impact of transfusion dependency at baseline and over time on survival and QoL outcomes.
Timepoint [6] 0 0
After 5 years from study entry.
Secondary outcome [7] 0 0
To devise a prognostic patient-based index.
Timepoint [7] 0 0
After 5 years from study entry.
Secondary outcome [8] 0 0
To evaluate accuracy of clinical prediction of survival.
Timepoint [8] 0 0
After 5 years from study entry.
Secondary outcome [9] 0 0
To assess patients' preferences for involvement in treatment decision-making across different risk group and examine relationships between preferences and patient characteristics.
Timepoint [9] 0 0
After 5 years from study entry.

Eligibility
Key inclusion 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Unknown status
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/10/2008
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2021
Actual
Sample size
Target
Accrual to date
Final
927
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
New York
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
Austria
State/province [3] 0 0
Innsbruck
Country [4] 0 0
Belgium
State/province [4] 0 0
Antwerpen
Country [5] 0 0
Belgium
State/province [5] 0 0
Brugge
Country [6] 0 0
Belgium
State/province [6] 0 0
Bruxelles
Country [7] 0 0
Belgium
State/province [7] 0 0
Liege
Country [8] 0 0
Brazil
State/province [8] 0 0
Curitiba
Country [9] 0 0
China
State/province [9] 0 0
China
Country [10] 0 0
China
State/province [10] 0 0
Kunming
Country [11] 0 0
Croatia
State/province [11] 0 0
Rijeka
Country [12] 0 0
Czechia
State/province [12] 0 0
Prague
Country [13] 0 0
France
State/province [13] 0 0
Saint-Cloud
Country [14] 0 0
France
State/province [14] 0 0
Toulouse
Country [15] 0 0
Germany
State/province [15] 0 0
Dresden
Country [16] 0 0
Germany
State/province [16] 0 0
Freiburg
Country [17] 0 0
Italy
State/province [17] 0 0
Foggia
Country [18] 0 0
Italy
State/province [18] 0 0
Torino
Country [19] 0 0
Italy
State/province [19] 0 0
Asti
Country [20] 0 0
Italy
State/province [20] 0 0
Bari
Country [21] 0 0
Italy
State/province [21] 0 0
Cagliari
Country [22] 0 0
Italy
State/province [22] 0 0
Catania
Country [23] 0 0
Italy
State/province [23] 0 0
Catanzaro
Country [24] 0 0
Italy
State/province [24] 0 0
Cona
Country [25] 0 0
Italy
State/province [25] 0 0
Firenze
Country [26] 0 0
Italy
State/province [26] 0 0
Genova
Country [27] 0 0
Italy
State/province [27] 0 0
Latina
Country [28] 0 0
Italy
State/province [28] 0 0
Lecce
Country [29] 0 0
Italy
State/province [29] 0 0
Meldola
Country [30] 0 0
Italy
State/province [30] 0 0
Milano
Country [31] 0 0
Italy
State/province [31] 0 0
Modena
Country [32] 0 0
Italy
State/province [32] 0 0
Monza
Country [33] 0 0
Italy
State/province [33] 0 0
Novara
Country [34] 0 0
Italy
State/province [34] 0 0
Orbassano
Country [35] 0 0
Italy
State/province [35] 0 0
Parma
Country [36] 0 0
Italy
State/province [36] 0 0
Pavia
Country [37] 0 0
Italy
State/province [37] 0 0
Piacenza
Country [38] 0 0
Italy
State/province [38] 0 0
Reggio Emilia
Country [39] 0 0
Italy
State/province [39] 0 0
Roma
Country [40] 0 0
Italy
State/province [40] 0 0
Rome
Country [41] 0 0
Italy
State/province [41] 0 0
Rovigo
Country [42] 0 0
Italy
State/province [42] 0 0
Sassari
Country [43] 0 0
Taiwan
State/province [43] 0 0
Taipei
Country [44] 0 0
United Kingdom
State/province [44] 0 0
Bradford
Country [45] 0 0
United Kingdom
State/province [45] 0 0
Leeds
Country [46] 0 0
United Kingdom
State/province [46] 0 0
London

Funding & Sponsors
Primary sponsor type
Other
Name
Gruppo Italiano Malattie EMatologiche dell'Adulto
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT00809575
Trial related presentations / publications
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.
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.
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.
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.
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.
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.
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Fabio Efficace, PhD
Address 0 0
Gruppo Italiano Malattie EMatologiche dell'Adulto
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT00809575