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

• ClinicalTrials.gov Identifier: NCT04676945
• Recruitment Status: Active, not recruiting
• First Posted: December 21, 2020
• Last Update Posted: September 5, 2021
• Sponsor: Michael Pfeilstöcker
• Collaborator: Celgene Corporation
• Information provided by (Responsible Party): Michael Pfeilstöcker, Mein Hanusch-Krankenhaus

Study Description

Brief Summary:

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

Condition or disease
Myelodysplastic Syndromes

Detailed 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.

Study Design

• Study Type: Observational
• Estimated Enrollment: 8000 participants
• Observational Model: Cohort
• Time Perspective: Retrospective
• Official Title: Analysis of Risk in MDS Over Time - Comparison of Treated vs Untreated Patients
• Actual Study Start Date: August 21, 2020
• Actual Primary Completion Date: August 20, 2021
• Estimated Study Completion Date: May 1, 2022

Groups and Cohorts

Group/Cohort
untreated control group; NO MDS disease modifying therapy
treated patients; ANY MDS disease modifying therapy

Outcome Measures

Primary Outcome Measures :

1. 1.Overall Survival [ Time Frame: 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. ]

   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.
2. 2.Time to transformation [ Time Frame: 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. ]

   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.

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

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 Population derived from registries of participating centres.

Criteria

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

Contacts and Locations

Locations

Austria

Hanusch Krankenhaus, 3.Medizinische Abteilung

Vienna, Austria, 1140

Sponsors and Collaborators

Michael Pfeilstöcker

Celgene Corporation

More Information

Publications:

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.

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.

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.

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.

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.

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.

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.

• Responsible Party: Michael Pfeilstöcker, Univ.Prof.Dr.med.univ., Mein Hanusch-Krankenhaus
• ClinicalTrials.gov Identifier: NCT04676945
• Other Study ID Numbers: N/A-NI-MDSAML-PI-14023
• First Posted: December 21, 2020
• Last Update Posted: September 5, 2021
• Last Verified: September 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Michael Pfeilstöcker, Mein Hanusch-Krankenhaus:

MDS; IPSS-R

Additional relevant MeSH terms:

Myelodysplastic Syndromes; Bone Marrow Diseases; Hematologic Diseases