|
|||||
|
|
Azacitidine in 302 patients with WHO-defined acute myeloid leukemia: results from the Austrian Azacitidine Registry of the AGMT-Study Group |
|
| Authors: | Lisa Pleyer Sonja Burgstaller Michael Girschikofsky Werner Linkesch Reinhard Stauder Michael Pfeilstocker Martin Schreder Christoph Tinchon Thamer Sliwa Alois Lang Wolfgang R Sperr Peter Krippl Dietmar Geissler Daniela Voskova Konstantin Schlick Josef Thaler Sigrid Machherndl-Spandl Georg Theiler Otto Eckmüllner Richard Greil |
| Institution: | 1. 3rd Medical Department with Hematology and Medical Oncology, Hemostaseology, Rheumatology and Infectious Diseases, Laboratory for Immunological and Molecular Cancer Research, Oncologic Center, Paracelsus Medical University Hospital Salzburg, and Center for Clinical Cancer and Immunology Trials at Salzburg Cancer Research Institute, Müllner Hauptstrasse 48, 5020, Salzburg, Austria 2. Department for Internal Medicine IV, Hospital Wels-Grieskirchen, Wels, Austria 3. 1st Medical Department with Hematology, Stem Cell Transplantation, Hemostatsis and Medical Oncology, Elisabethinen Hospital, Linz, Austria 4. Department of Hematology, Medical University, Graz, Austria 5. Internal Medicine V (Hematology and Oncology), Innsbruck Medical University, Innsbruck, Austria 6. Third Medical Department, Hanusch Hospital, Vienna, Austria 7. First Department of Internal Medicine, Center for Oncology and Hematology, Wilhelminenspital, Vienna, Austria 8. Department for Hematology and Oncology, LKH Leoben-Eisenerz, Leoben, Austria 9. 5th Medical Department with Oncology und Palliative Medicine, Hietzing, Vienna, Austria 10. Internal Medicine, Hospital Feldkirch, Feldkirch, Austria 11. Division of Hematology and Hemostaseology, Department of Internal Medicine I, Medical University of Vienna, Vienna, Austria 12. Department for Internal Medicine, LKH Fuerstenfeld, Fuerstenfeld, Austria 13. Department for Internal Medicine, with Nephrology, Gastroenterology and Hepatology, Hematology and Medical Onkology, Intensive Care Unit, and Rheumatology, Klinikum Klagenfurt am W?rtersee, P?rtschach am W?rthersee, Austria 14. Internal Medicine 3, Center for Hematology and Medical Oncology, General Hospital-Linz GesmbH, Linz, Austria 15. Institut für Waldwachstumsforschung, Universit?t für Bodenkultur, Vienna, Austria |
| Abstract: | Data on efficacy and safety of azacitidine in acute myeloid leukemia (AML) with >30 % bone marrow (BM) blasts are limited, and the drug can only be used off-label in these patients. We previously reported on the efficacy and safety of azacitidine in 155 AML patients treated within the Austrian Azacitidine Registry (clinicaltrials.gov identifier NCT01595295). We herein update this report with a population almost twice as large (n?=?302). This cohort included 172 patients with >30 % BM blasts; 93 % would have been excluded from the pivotal AZA-001 trial (which led to European Medicines Agency (EMA) approval of azacitidine for high-risk myelodysplastic syndromes (MDS) and AML with 20–30 % BM blasts). Despite this much more unfavorable profile, results are encouraging: overall response rate was 48 % in the total cohort and 72 % in patients evaluable according to MDS-IWG-2006 response criteria, respectively. Median OS was 9.6 (95 % CI 8.53–10.7)?months. A clinically relevant OS benefit was observed with any form of disease stabilization (marrow stable disease (8.1 months), hematologic improvement (HI) (9.7 months), or the combination thereof (18.9 months)), as compared to patients without response and/or without disease stabilization (3.2 months). Age, white blood cell count, and BM blast count at start of therapy did not influence OS. The baseline factors LDH >225 U/l, ECOG ≥2, comorbidities ≥3, monosomal karyotype, and prior disease-modifying drugs, as well as the response-related factors hematologic improvement and further deepening of response after first response, were significant independent predictors of OS in multivariate analysis. Azacitidine seems effective in WHO-AML, including patients with >30 % BM blasts (currently off-label use). Although currently not regarded as standard form of response assessment in AML, disease stabilization and/or HI should be considered sufficient response to continue treatment with azacitidine. |
| Keywords: | |
| 本文献已被 SpringerLink 等数据库收录！ | |