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Platelet doubling after the first azacitidine cycle is a promising predictor for response in myelodysplastic syndromes (MDS), chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML) patients in the Dutch azacitidine compassionate named patient programme
Author(s) -
van der Helm Lieke H.,
Alhan Canan,
Wijermans Pierre W.,
van Marwijk Kooy Marinus,
Schaafsma Ron,
Biemond Bart J.,
Beeker Aart,
Hoogendoorn Mels,
van Rees Bastiaan P.,
de Weerdt Okke,
Wegman Jurgen,
Libourel Ward J.,
Luykxde Bakker Sylvia A.,
Minnema Monique C.,
Brouwer Rolf E.,
Croonde Boer Fransien,
Eefting Matthijs,
Jie KonSiong G.,
van de Loosdrecht Arjan A.,
Koedam Jan,
Veeger Nic J. G. M,
Vellenga Edo,
Huls Gerwin
Publication year - 2011
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1111/j.1365-2141.2011.08893.x
Subject(s) - azacitidine , medicine , myelodysplastic syndromes , chronic myelomonocytic leukemia , hazard ratio , oncology , confidence interval , gastroenterology , bone marrow , biology , biochemistry , gene expression , gene , dna methylation
Summary The efficacy of azacitidine in the treatment of high‐risk myelodysplastic syndromes (MDS), chronic myelomonocytic leukaemia (CMML) and acute myeloid leukaemia (AML) (20–30% blasts) has been demonstrated. To investigate the efficacy of azacitidine in daily clinical practice and to identify predictors for response, we analysed a cohort of 90 MDS, CMML and AML patients who have been treated in a Dutch compassionate named patient programme. Patients received azacitidine for a median of five cycles (range 1–19). The overall response rate (complete/partial/haematological improvement) was 57% in low risk MDS, 53% in high risk MDS, 50% in CMML, and 39% in AML patients. Median overall survival (OS) was 13·0 (9·8–16·2) months. Multivariate analysis confirmed circulating blasts [Hazard Ratio (HR) 0·48, 95% confidence interval (CI) 0·24–0·99; P = 0·05] and poor risk cytogenetics (HR 0·45, 95% CI 0·22–0·91; P = 0·03) as independent predictors for OS. Interestingly, this analysis also identified platelet doubling after the first cycle of azacitidine as a simple and independent positive predictor for OS (HR 5·4, 95% CI 0·73–39·9; P = 0·10). In conclusion, routine administration of azacitidine to patients with variable risk groups of MDS, CMML and AML is feasible, and subgroups with distinct efficacy of azacitidine treatment can be identified.