Impact of Azacitidine Before Allogeneic Stem-Cell Transplantation for Myelodysplastic Syndromes: A Study by the Société Française de Greffe de Moelle et de Thérapie-Cellulaire and the Groupe-Francophone des Myélodysplasies

• Published in: Journal of clinical oncology Vol. 30; no. 36; pp. 4533 - 4540
• Main Authors: DAMAJ, Gandhi, DUHAMEL, Alain, BULABOIS, Claude-Eric, HUYNH, Anne, BAY, Jacques-Olivier, LEGRAND, Faeyzeh, DECONINCK, Eric, FEGUEUX, Nathalie, CLEMENT, Laurence, DAURIAC, Charles, MAILLARD, Natacha, CORNILLON, Jérôme, ROBIN, Marie, ADES, Lionel, GUILLERM, Gaelle, SCHMIDT-TANGUY, Aline, MARJANOVIC, Zora, PARK, Sophie, RUBIO, Marie-Thérèse, MAROLLEAU, Jean-Pierre, GARNIER, Federico, FENAUX, Pierre, YAKOUB-AGHA, Ibrahim, BEGUIN, Yves, MICHALLET, Mauricette, MOHTY, Mohamad, VIGOUROUX, Stephane, BORIES, Pierre, GARNIER, Alice, EL CHEIKH, Jean
• Format: Journal Article
• Language: English
• Published: Alexandria, VA American Society of Clinical Oncology 20-12-2012
• Subjects: Antimetabolites, Antineoplastic; Antimetabolites, Antineoplastic - adverse effects; Antimetabolites, Antineoplastic - therapeutic use; Azacitidine; Azacitidine - adverse effects; Azacitidine - therapeutic use; Biological and medical sciences; Combined Modality Therapy; Female; Hematologic and hematopoietic diseases; Hematopoietic Stem Cell Transplantation; Hematopoietic Stem Cell Transplantation - methods; Humans; Immunology; Induction Chemotherapy; Leukemias. Malignant lymphomas. Malignant reticulosis. Myelofibrosis; Life Sciences; Male; Medical sciences; Middle Aged; Myelodysplastic Syndromes; Myelodysplastic Syndromes - drug therapy; Myelodysplastic Syndromes - etiology; Myelodysplastic Syndromes - surgery; Retrospective Studies; Transplantation, Homologous; Transplantation, Homologous - methods; Treatment Outcome; Tumors; Antineoplastic agent; Stem cell; Hematopoietic cell; Homograft; Malignant hemopathy; Myelodysplastic syndrome; Azanucleoside; Cancerology; Allogeneic hematopoietic stem cell transplantation; Antimetabolic; Azacitidine; Triazine derivatives; Graft; Cancer
• ISSN: 0732-183X; 1527-7755
• DOI: 10.1200/JCO.2012.44.3499

To investigate the impact of prior-to-transplantation azacitidine (AZA) on patient outcome after allogeneic stem-cell transplantation (alloSCT) for myelodysplastic syndrome (MDS).
Of the 265 consecutive patients who underwent alloSCT for MDS between October 2005 and December 2009, 163 had received cytoreductive treatment prior to transplantation, including induction chemotherapy (ICT) alone (ICT group; n = 98), AZA alone (AZA group; n = 48), or AZA preceded or followed by ICT (AZA-ICT group; n = 17). At diagnosis, 126 patients (77%) had an excess of marrow blasts, and 95 patients (58%) had intermediate-2 or high-risk MDS according to the International Prognostic Scoring System (IPSS). Progression to more advanced disease before alloSCT was recorded in 67 patients. Donors were sibling (n = 75) or HLA-matched unrelated (10/10; n = 88). They received blood (n = 142) or marrow (n = 21) grafts following either myeloablative (n = 33) or reduced intensity (n = 130) conditioning.
With a median follow-up of 38.7 months, 3-year outcomes in the AZA, ICT, and AZA-ICT groups were 55%, 48%, and 32% (P = .07) for overall survival (OS); 42%, 44%, and 29% (P = .14) for event-free survival (EFS); 40%, 37%, and 36% (P = .86) for relapse; and 19%, 20%, and 35% (P = .24) for nonrelapse mortality (NRM), respectively. Multivariate analysis confirmed the absence of statistical differences between the AZA and the ICT groups in terms of OS, EFS, relapse, and NRM.
With the goal of downstaging underlying disease before alloSCT, AZA alone led to outcomes similar to those for standard ICT.