Registry on Hypomethylating Agents in Myeloid Neoplasms

Registry on Hypomethylating Agents in Myeloid Neoplasms, Including Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML) and Acute Myeloid Leukemia (AML)

This registry is set up to collect real-world experience in the management of patients with myeloid neoplasms, in particularly in patients with MDS, CMML or AML, treated with hypomethylating agents in Austria and potentially other participating countries. This registry will collect data in a retrospective as well as in a prospective manner at various sites. The aim is to gain valuable insights on both efficacy and toxicity of these drugs in a routine clinical setting in patients with various comorbidities.

Study Overview

• Status: Completed
• Conditions: Acute Myeloid Leukemia; Myelodysplastic Syndromes; Chronic Myelomonocytic Leukemia
• Intervention / Treatment: Other: non interventional

• Study Type: Observational
• Enrollment (Actual): 1500

Contacts and Locations

Study Locations

• Austria
  • Feldkirch, Austria, 6807
    • LKH Feldkirch, Innere Med. II, Interne E
  • Fürstenfeld, Austria, 8280
    • LKH Fürstenfeld
  • Graz, Austria, 8036
    • UK Graz: Universitätsklinik für Innere Medizin; Klinische Abteilung für Hämatologie
  • Innsbruck, Austria, 6020
    • Universitätsklinik für Innere Medizin V
  • Klagenfurt, Austria, 9020
    • Klinikum Klagenfurt: 1. Medizinische Abteilung
  • Krems an der Donau, Austria, 3500
    • UK Krems: Innere Medizin II
  • Leoben, Austria, 8700
    • LKH Hochsteiermark
  • Linz, Austria, 4010
    • Ordensklinikum Linz - BHS: Interne I: Medizinische Onkologie und Hämatologie
  • Linz, Austria, 4020
    • Ordensklinikum Linz - Elisabethinen: Interne I
  • Linz, Austria, 4021
    • Kepler Universitätsklinikum Linz, Med. Campus III., Univ.-Klinik für Hämatologie und Internistische Onkologie
  • Salzburg, Austria, 5020
    • Universitätsklinik für Innere Medizin III Universitätsklinik für Innere Medizin III der PMU Salzburg
  • Steyr, Austria, 4400
    • Klinikum Steyr: Innere Medizin II: Onkologie, Gastroenterologie, Angiologie
  • Wels, Austria, 4800
    • Klinikum Wels Grieskirchen
  • Wien, Austria, 1130
    • Krankenhaus Hietzing
  • Wien, Austria, 1140
    • Hanusch Krankenhaus
  • Wien, Austria, 1030
    • Klinik Landstraße: 1. Medizinische Abteilung
  • Wien, Austria, 1090
    • AKH Meduni Wien: Universitätsklinik für Innere Medizin I: Klinische Abteilung für Hämatologie und Hämostaseologie
  • Wien, Austria, 1160
    • Klinik Ottakring, 1. Med. Abteilung
  • Wien, Austria, 1220
    • Klinik Donaustadt: 2. Medizinische Abteilung
• Croatia
  • Zagreb, Croatia, 10000
    • Clinical Hospital Merkur

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with MDS, CMML, and AML, who begin with or already have received treatment with a hypomethylating agent

Description

Inclusion Criteria:

Patients with MDS, CMML, and AML

• who begin with or already have received treatment with with a hypomethylating agent
• who are willing to provide informed consent

Exclusion Criteria:

• Due to the non-interventional design of this program there are no specific exclusion criteria.

Study Plan

How is the study designed?

Design Details

• Observational Models: Other
• Time Perspectives: Other

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Hypomethylating Agents; Patients treated with hypomethylating agents	Other: non interventional

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall survival		up to 10 years
Response evaluation	hematological response, marrow response, cytogenetic response (if data is available), quantification of reduced need for transfusions, median response duration	up to 10 years

Secondary Outcome Measures

Outcome Measure	Time Frame
Documentation of adverse events and toxicities	up to 10 years
Number of treatment cycles	up to 10 years
Number and reasons of dose reductions	up to 10 years
Uni/multivariate analysis of various factors known or thought to influence overall survival in order to establish prognostic markers	up to 10 years

Collaborators and Investigators

• Sponsor: Arbeitsgemeinschaft medikamentoese Tumortherapie
• Investigators: Study Chair: Richard Greil, MD, Universitätsklinik für Innere Medizin III der PMU Salzburg; Principal Investigator: Lisa Pleyer, MD, Universitätsklinik für Innere Medizin III der PMU Salzburg

Publications and helpful links

General Publications

• Falantes J, Pleyer L, Thepot S, Almeida AM, Maurillo L, Martinez-Robles V, Stauder R, Itzykson R, Pinto R, Venditti A, Bargay J, Burgstaller S, Martinez MP, Seegers V, Cortesao E, Foncillas MA, Gardin C, Montesinos P, Musto P, Fenaux P, Greil R, Sanz MA, Ramos F; European ALMA + Investigators. Real life experience with frontline azacitidine in a large series of older adults with acute myeloid leukemia stratified by MRC/LRF score: results from the expanded international E-ALMA series (E-ALMA+). Leuk Lymphoma. 2018 May;59(5):1113-1120. doi: 10.1080/10428194.2017.1365854. Epub 2017 Aug 24.
• Leisch M, Weiss L, Lindlbauer N, Jungbauer C, Egle A, Rohde E, Greil R, Grabmer C, Pleyer L. Red blood cell alloimmunization in 184 patients with myeloid neoplasms treated with azacitidine - A retrospective single center experience. Leuk Res. 2017 Aug;59:12-19. doi: 10.1016/j.leukres.2017.05.006. Epub 2017 May 9.
• Huemer F, Weiss L, Faber V, Neureiter D, Egle A, Geissler K, Voskova D, Zebisch A, Burgstaller S, Pichler A, Stauder R, Sperr W, Lang A, Pfeilstocker M, Machherndl-Spandl S, Stampfl M, Greil R, Pleyer L. Establishment and validation of a novel risk model for estimating time to first treatment in 120 patients with chronic myelomonocytic leukaemia. Wien Klin Wochenschr. 2018 Feb;130(3-4):115-125. doi: 10.1007/s00508-018-1315-2. Epub 2018 Jan 30.
• Almeida AM, Prebet T, Itzykson R, Ramos F, Al-Ali H, Shammo J, Pinto R, Maurillo L, Wetzel J, Musto P, Van De Loosdrecht AA, Costa MJ, Esteves S, Burgstaller S, Stauder R, Autzinger EM, Lang A, Krippl P, Geissler D, Falantes JF, Pedro C, Bargay J, Deben G, Garrido A, Bonanad S, Diez-Campelo M, Thepot S, Ades L, Sperr WR, Valent P, Fenaux P, Sekeres MA, Greil R, Pleyer L. Clinical Outcomes of 217 Patients with Acute Erythroleukemia According to Treatment Type and Line: A Retrospective Multinational Study. Int J Mol Sci. 2017 Apr 14;18(4):837. doi: 10.3390/ijms18040837.
• Pleyer L, Dohner H, Dombret H, Seymour JF, Schuh AC, Beach CL, Swern AS, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova D, Sperr WR, Hojas S, Rogulj IM, Andel J, Greil R. Azacitidine for Front-Line Therapy of Patients with AML: Reproducible Efficacy Established by Direct Comparison of International Phase 3 Trial Data with Registry Data from the Austrian Azacitidine Registry of the AGMT Study Group. Int J Mol Sci. 2017 Feb 15;18(2):415. doi: 10.3390/ijms18020415.
• Pleyer L, Burgstaller S, Stauder R, Girschikofsky M, Sill H, Schlick K, Thaler J, Halter B, Machherndl-Spandl S, Zebisch A, Pichler A, Pfeilstocker M, Autzinger EM, Lang A, Geissler K, Voskova D, Geissler D, Sperr WR, Hojas S, Rogulj IM, Andel J, Greil R. Azacitidine front-line in 339 patients with myelodysplastic syndromes and acute myeloid leukaemia: comparison of French-American-British and World Health Organization classifications. J Hematol Oncol. 2016 Apr 16;9:39. doi: 10.1186/s13045-016-0263-4.
• Ramos F, Thepot S, Pleyer L, Maurillo L, Itzykson R, Bargay J, Stauder R, Venditti A, Seegers V, Martinez-Robles V, Burgstaller S, Recher C, Deben G, Gaidano G, Gardin C, Musto P, Greil R, Sanchez-Guijo F, Fenaux P; European ALMA Investigators. Azacitidine frontline therapy for unfit acute myeloid leukemia patients: clinical use and outcome prediction. Leuk Res. 2015 Mar;39(3):296-306. doi: 10.1016/j.leukres.2014.12.013. Epub 2014 Dec 31.
• Pleyer L, Burgstaller S, Girschikofsky M, Linkesch W, Stauder R, Pfeilstocker M, Schreder M, Tinchon C, Sliwa T, Lang A, Sperr WR, Krippl P, Geissler D, Voskova D, Schlick K, Thaler J, Machherndl-Spandl S, Theiler G, Eckmullner O, Greil R. Azacitidine in 302 patients with WHO-defined acute myeloid leukemia: results from the Austrian Azacitidine Registry of the AGMT-Study Group. Ann Hematol. 2014 Nov;93(11):1825-38. doi: 10.1007/s00277-014-2126-9. Epub 2014 Jun 21.
• Pleyer L, Germing U, Sperr WR, Linkesch W, Burgstaller S, Stauder R, Girschikofsky M, Schreder M, Pfeilstocker M, Lang A, Sliwa T, Geissler D, Schlick K, Placher-Sorko G, Theiler G, Thaler J, Mitrovic M, Neureiter D, Valent P, Greil R. Azacitidine in CMML: matched-pair analyses of daily-life patients reveal modest effects on clinical course and survival. Leuk Res. 2014 Apr;38(4):475-83. doi: 10.1016/j.leukres.2014.01.006. Epub 2014 Jan 18.
• Pleyer L, Stauder R, Burgstaller S, Schreder M, Tinchon C, Pfeilstocker M, Steinkirchner S, Melchardt T, Mitrovic M, Girschikofsky M, Lang A, Krippl P, Sliwa T, Egle A, Linkesch W, Voskova D, Angermann H, Greil R. Azacitidine in patients with WHO-defined AML - results of 155 patients from the Austrian Azacitidine Registry of the AGMT-Study Group. J Hematol Oncol. 2013 Apr 29;6:32. doi: 10.1186/1756-8722-6-32.
• Pleyer L, Leisch M, Kourakli A, Padron E, Maciejewski JP, Xicoy Cirici B, Kaivers J, Ungerstedt J, Heibl S, Patiou P, Hunter AM, Mora E, Geissler K, Dimou M, Jimenez Lorenzo MJ, Melchardt T, Egle A, Viniou AN, Patel BJ, Arnan M, Valent P, Roubakis C, Bernal Del Castillo T, Galanopoulos A, Calabuig Munoz M, Bonadies N, Medina de Almeida A, Cermak J, Jerez A, Montoro MJ, Cortes A, Avendano Pita A, Lopez Andrade B, Hellstroem-Lindberg E, Germing U, Sekeres MA, List AF, Symeonidis A, Sanz GF, Larcher-Senn J, Greil R. Outcomes of patients with chronic myelomonocytic leukaemia treated with non-curative therapies: a retrospective cohort study. Lancet Haematol. 2021 Feb;8(2):e135-e148. doi: 10.1016/S2352-3026(20)30374-4.
• Leisch M, Pfeilstocker M, Stauder R, Heibl S, Sill H, Girschikofsky M, Stampfl-Mattersberger M, Tinchon C, Hartmann B, Petzer A, Schreder M, Kiesl D, Vallet S, Egle A, Melchardt T, Piringer G, Zebisch A, Machherndl-Spandl S, Wolf D, Keil F, Drost M, Greil R, Pleyer L. Adverse Events in 1406 Patients Receiving 13,780 Cycles of Azacitidine within the Austrian Registry of Hypomethylating Agents-A Prospective Cohort Study of the AGMT Study-Group. Cancers (Basel). 2022 May 17;14(10):2459. doi: 10.3390/cancers14102459.
• Pleyer L, Sekeres MA. An early glimpse at azacitidine plus venetoclax for myelodysplastic syndromes. Lancet Haematol. 2022 Oct;9(10):e714-e716. doi: 10.1016/S2352-3026(22)00252-6. Epub 2022 Sep 2. No abstract available.

Study record dates

Study Major Dates

• Study Start (Actual): February 9, 2009
• Primary Completion (Actual): December 31, 2021
• Study Completion (Actual): December 31, 2021

Study Registration Dates

• First Submitted: May 7, 2012
• First Submitted That Met QC Criteria: May 8, 2012
• First Posted (Estimated): May 10, 2012

Study Record Updates

• Last Update Posted (Actual): March 25, 2024
• Last Update Submitted That Met QC Criteria: March 22, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Keywords: AML; MDS; registry; CMML; Vidaza
• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Histologic Type; Disease Attributes; Bone Marrow Diseases; Hematologic Diseases; Precancerous Conditions; Myelodysplastic-Myeloproliferative Diseases; Chronic Disease; Neoplasms; Myelodysplastic Syndromes; Leukemia; Leukemia, Myeloid; Leukemia, Myeloid, Acute; Preleukemia; Leukemia, Myelomonocytic, Chronic; Leukemia, Myelomonocytic, Juvenile
• Other Study ID Numbers: AGMT_HMA Register