Clinical trial NCT01595295: Registry on Hypomethylating Agents in Myeloid Neoplasms
Official title: Registry on Hypomethylating Agents in Myeloid Neoplasms, Including Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML) and Acute Myeloid Leukemia (AML)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Chronic Myelomonocytic Leukemia
Myelodysplastic Syndromes
Acute Myeloid Leukemia
Keywords: CMML
MDS
AML
registry
Vidaza
Lead sponsor: Arbeitsgemeinschaft medikamentoese Tumortherapie
Sponsor class: OTHER
First posted: 2012-05-10
Enrollment: 1500
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: OTHER
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

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Response evaluation
timeFrame: up to 10 years
measure: Overall survival
timeFrame: up to 10 years
Secondary outcomes:
measure: Documentation of adverse events and toxicities
timeFrame: up to 10 years
measure: Number of treatment cycles
timeFrame: up to 10 years
measure: Number and reasons of dose reductions
timeFrame: up to 10 years
measure: Uni/multivariate analysis of various factors known or thought to influence overall survival in order to establish prognostic markers
timeFrame: up to 10 years
Other outcomes:
Locations:
city: Feldkirch
country: Austria
facility: LKH Feldkirch, Innere Med. II, Interne E
city: Fürstenfeld
country: Austria
facility: LKH Fürstenfeld
city: Graz
country: Austria
facility: UK Graz: Universitätsklinik für Innere Medizin; Klinische Abteilung für Hämatologie
city: Innsbruck
country: Austria
facility: Universitätsklinik für Innere Medizin V
city: Klagenfurt
country: Austria
facility: Klinikum Klagenfurt: 1. Medizinische Abteilung
city: Krems an der Donau
country: Austria
facility: UK Krems: Innere Medizin II
city: Leoben
country: Austria
facility: LKH Hochsteiermark
city: Linz
country: Austria
facility: Ordensklinikum Linz - BHS: Interne I: Medizinische Onkologie und Hämatologie
city: Linz
country: Austria
facility: Ordensklinikum Linz - Elisabethinen: Interne I
city: Linz
country: Austria
facility: Kepler Universitätsklinikum Linz, Med. Campus III., Univ.-Klinik für Hämatologie und Internistische Onkologie
city: Salzburg
country: Austria
facility: Universitätsklinik für Innere Medizin III Universitätsklinik für Innere Medizin III der PMU Salzburg
city: Steyr
country: Austria
facility: Klinikum Steyr: Innere Medizin II: Onkologie, Gastroenterologie, Angiologie
city: Wels
country: Austria
facility: Klinikum Wels Grieskirchen
city: Wien
country: Austria
facility: Klinik Landstraße: 1. Medizinische Abteilung
city: Wien
country: Austria
facility: AKH Meduni Wien: Universitätsklinik für Innere Medizin I: Klinische Abteilung für Hämatologie und Hämostaseologie
city: Wien
country: Austria
facility: Krankenhaus Hietzing
city: Wien
country: Austria
facility: Hanusch Krankenhaus
city: Wien
country: Austria
facility: Klinik Ottakring, 1. Med. Abteilung
city: Wien
country: Austria
facility: Klinik Donaustadt: 2. Medizinische Abteilung
city: Zagreb
country: Croatia
facility: Clinical Hospital Merkur
Study officials:
affiliation: Universitätsklinik für Innere Medizin III der PMU Salzburg
name: Richard Greil, MD
role: STUDY_CHAIR
affiliation: Universitätsklinik für Innere Medizin III der PMU Salzburg
name: Lisa Pleyer, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.
citation: 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.