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Ann Oncol. 2017 Jul 1;28(7):1547-1553. doi: 10.1093/annonc/mdx154.

Feasibility of allogeneic stem-cell transplantation after azacitidine bridge in higher-risk myelodysplastic syndromes and low blast count acute myeloid leukemia: results of the BMT-AZA prospective study.

Author information

Division of Hematology, Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome.
Department of Hematology, Università Cattolica del Sacro Cuore, Rome.
Department of Statistical Sciences, La Sapienza University, Rome.
Department of Hematology, Centro Trapianti Midollo Osseo, Pescara.
Division of Hematology and BMT, Department of Experimental and Clinical Medical Sciences, Azienda Ospedaliero-Universitaria di Udine, Udine.
Department of Hematology, ASST Papa Giovanni XXIII, Bergamo.
Department of Hematology, A.O. Spedali Civili, Brescia.
Department of Hematology, Ospedale Niguarda, Milano.
Department of Hematology, Ospedale Sant'Orsola Malpighi, University of Bologna, Bologna.
Department of Hematology, HSCT Adult Unit, San Gerardo Hospital, Monza.
Department of Hematology, Azienda Ospedaliera Universitaria - Ospedali Riuniti di Ancona, Ancona.
Department of Hematology, Ospedale Opera Padre Pio, San Giovanni Rotondo.
Department of Hematology, Fondazione IRCCS Istituto Nazionale dei Tumori, Milan.
Department of Hematology Oncology, Foundation IRCCS Ca' Granda Ospedale Maggiore Policlinico and University of Milan, Milano.
Department of Hematology, "SS Antonio e Biagio" Hospital, Alessandria.
Department of Hematology, Policlinico San Matteo and Pavia University, Pavia, Italy.



Allogeneic stem-cell transplantation (HSCT) is the only curative treatment in myelodysplastic syndromes (MDS). Azacitidine (AZA) is increasingly used prior to HSCT, however in Europe it is only approved for patients who are not eligible for HSCT.

Patients and methods:

We conducted a phase II multicenter study to prospectively evaluate the feasibility of HSCT after treatment with AZA in 70 patients with a myelodysplastic syndrome (MDS), 19 with acute myeloid leukemia (AML), and 8 with chronic myelomonocytic leukemia (CMML). After a median of four cycles (range 1-11): 24% of patients achieved complete remission, 14% partial remission, 8% hematologic improvement, 32% had stable and 22% progressive disease. Ten patients discontinued treatment before the planned four cycles, due to an adverse event in nine cases.


A HSC donor was identified in 73 patients, and HSCT was performed in 54 patients (74% of patients with a donor). Main reasons for turning down HSCT were lack of a donor, an adverse event, or progressive disease (9, 12, and 16 patients, respectively). At a median follow-up of 20.5 months from enrolment, response to AZA was the only independent prognostic factor for survival. Compared to baseline assessment, AZA treatment did not affect patients' comorbidities at HSCT: the HCT-CI remained stable in 62% patients, and worsened or improved in 23% and 15% of patients, respectively.


Our study shows that HSCT is feasible in the majority of patients with HR-MDS/AML/CMML-2 after AZA treatment. As matched unrelated donor was the most frequent source of donor cells, the time between diagnosis and HSCT needed for donor search could be 'bridged' using azacitidine. These data show that AZA prior to HSCT could be a better option than intensive chemotherapy in higher-risk MDS. The trial has been registered with the EudraCT number 2010-019673-1.


allogeneic stem-cell transplantation; azacitidine; high-risk MDS; hypomethylating treatment

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