Analysis of Risk in MDS Over Time - Comparison of Treated vs Untreated Patients

Myelodysplastic Syndromes Clinical Trial

Official title:

Analysis of Risk in MDS Over Time - Comparison of Treated vs Untreated Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04676945
• Other study ID #: N/A-NI-MDSAML-PI-14023
• Status: Completed
• Start date: August 21, 2020
• Est. completion date: November 21, 2022

Study information

• Verified date: November 2022
• Source: Mein Hanusch-Krankenhaus
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

A comparison of treated vs untreated patients with MDS with a sample size of approximately 8000 patients in 11 countries.

Description:

Non-interventional multicentre retrospective study using chart reviews - study was submitted to the ethics committee of city of Vienna - due to the design of the study no specific approval necessary.

Inclusion of MDS (all subtypes and risk groups) patients according to WHO or oligoblastic AML (RAEB-T according to FAB), untreated and treated during their chronic MDS phase

The 26 centers of the IWG-PM have provided control data set of untreated, all centers will be asked for contribution of treated patients Data will be collected in Austria, Brazil, Czech Republic, France, Germany, Italy, Japan, Netherlands, Spain, UK, USA.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9179
• Est. completion date: November 21, 2022
• Est. primary completion date: August 20, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• MDS (all subtypes and risk groups) according to WHO or oligoblastic AML (RAEB-T according to FAB) patients untreated and treated during their chronic MDS phase

Exclusion Criteria:

• None

Related Conditions & MeSH terms

• Myelodysplastic Syndromes

Locations

Country	Name	City	State
Austria	Hanusch Krankenhaus, 3.Medizinische Abteilung	Vienna

Sponsors (2)

Lead Sponsor	Collaborator
Michael Pfeilstöcker	Celgene Corporation

Country where clinical trial is conducted

Austria, 

References & Publications (7)

Della Porta MG, Alessandrino EP, Bacigalupo A, van Lint MT, Malcovati L, Pascutto C, Falda M, Bernardi M, Onida F, Guidi S, Iori AP, Cerretti R, Marenco P, Pioltelli P, Angelucci E, Oneto R, Ripamonti F, Bernasconi P, Bosi A, Cazzola M, Rambaldi A; Gruppo Italiano Trapianto di Midollo Osseo. Predictive factors for the outcome of allogeneic transplantation in patients with MDS stratified according to the revised IPSS-R. Blood. 2014 Apr 10;123(15):2333-42. doi: 10.1182/blood-2013-12-542720. Epub 2014 Feb 20. — View Citation

Greenberg PL, Tuechler H, Schanz J, Sanz G, Garcia-Manero G, Solé F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Levis A, Malcovati L, Cazzola M, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Krieger O, Luebbert M, Maciejewski J, Magalhaes SM, Miyazaki Y, Pfeilstöcker M, Sekeres M, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012 Sep 20;120(12):2454-65. Epub 2012 Jun 27. — View Citation

Lamarque M, Raynaud S, Itzykson R, Thepot S, Quesnel B, Dreyfus F, Rauzy OB, Turlure P, Vey N, Recher C, Dartigeas C, Legros L, Delaunay J, Visanica S, Stamatoullas A, Fenaux P, Adès L. The revised IPSS is a powerful tool to evaluate the outcome of MDS patients treated with azacitidine: the GFM experience. Blood. 2012 Dec 13;120(25):5084-5. doi: 10.1182/blood-2012-09-453555. Erratum in: Blood. 2014 Jun 26;123(26):4152. — View Citation

Montalban-Bravo G, Garcia-Manero G. Myelodysplastic syndromes: 2018 update on diagnosis, risk-stratification and management. Am J Hematol. 2018 Jan;93(1):129-147. doi: 10.1002/ajh.24930. — View Citation

Pfeilstöcker M, Tüchler H, Schönmetzler A, Nösslinger T, Pittermann E. Time changes in predictive power of established and recently proposed clinical, cytogenetical and comorbidity scores for Myelodysplastic Syndromes. Leuk Res. 2012 Feb;36(2):132-9. doi: 10.1016/j.leukres.2011.09.007. Epub 2011 Oct 2. — View Citation

Pfeilstöcker M, Tuechler H, Sanz G, Schanz J, Garcia-Manero G, Solé F, Bennett JM, Bowen D, Fenaux P, Dreyfus F, Kantarjian H, Kuendgen A, Malcovati L, Cazzola M, Cermak J, Fonatsch C, Le Beau MM, Slovak ML, Levis A, Luebbert M, Maciejewski J, Machherndl-Spandl S, Magalhaes SM, Miyazaki Y, Sekeres MA, Sperr WR, Stauder R, Tauro S, Valent P, Vallespi T, van de Loosdrecht AA, Germing U, Haase D, Greenberg PL. Time-dependent changes in mortality and transformation risk in MDS. Blood. 2016 Aug 18;128(7):902-10. doi: 10.1182/blood-2016-02-700054. Epub 2016 Jun 22. — View Citation

Sekeres MA, Swern AS, Fenaux P, Greenberg PL, Sanz GF, Bennett JM, Dreyfus F, List AF, Li JS, Sugrue MM. Validation of the IPSS-R in lenalidomide-treated, lower-risk myelodysplastic syndrome patients with del(5q). Blood Cancer J. 2014 Aug 29;4:e242. doi: 10.1038/bcj.2014.62. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	1.Overall Survival	In *overall survival* two possible events are defined: *death* (regarded as complete observation); *end of follow up* (regarded as censored observation) Time is calculated from diagnosis to the first occurrence of one of the above listed events.	From date of diagnosis until the date of death or lost to follow-up, whichever came first. No administrative censoring will be applied to the retrospectively collected data, a minimum period of two months of stable disease will be required.
Primary	2.Time to transformation	In *time to transformation* three possible events are defined: *transformation into AML* (regarded as complete observation); *death without transformation* (regarded as censored observation); *end of follow up* (regarded as censored observation) Time is calculated from diagnosis to the first occurrence of one of the above listed events.; In case of *transformation into AML* this results in a complete observation, in case of *death without transformation* or *end of follow up* the observation is treated as censored.	From date of diagnosis until the date of transformation,death or lost to follow-up, whichever came first. No administrative censoring will be applied to the retrospectively collected data,a minimum period of two months of stable disease will be required.