The issue is the status of maintenance with any single agent drug. The most recent phase II study concluded: “5-azacytidine treatment is safe, feasible and may be of benefit in a subset of patients”.
The Scottish Medicines Consortium does not recommend azacitidine (Vidaza®) for use within NHS Scotland for the treatment of adults who are not eligible for haematopoietic stem cell transplantation (SCT) with intermediate-2 and high-risk myelodysplastic syndrome (MDS), chronic myelomonocytic leukaemia (CMML) or acute myeloid leukaemia (AML).
On May 19, 2004 the U.S. Food and Drug Administration approved azacitidine as injectable suspension (Vidaza) for treatment of patients with the following MDS subtypes: RA or RARS (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), RAEB, RAEB-T, and CMML. Azacitidine is the first agent approved for treatment of myelodysplastic syndrome. However, though approved for MDS, there is good evidence for its effectiveness in AML as well. Subset analysis of the international phase III trial (AZA-001) demonstrating that the overall survival benefit observed in higher-risk MDS patients extended to patients with acute myeloid leukemia (AML). It is listed by NCCN as a low-intensity option for AML.
Almost one third of the patients (113 of 358) enrolled in the AZA-001 study met the WHO criteria for AML (median 23% bone marrow blasts), which has a poor prognosis and does not respond well to conventional chemotherapy. This subset analysis of the AZA-001 study showed the median overall survival was 24.5 months with VIDAZA compared to 16.0 months with CCR (p=0.005). Additionally, 50 percent of the AML patients who were treated with VIDAZA survived at least two years, compared to only 16 percent of AML patients treated with CCR.
Michael Grövdal et al, Maintenance treatment with azacytidine for patients with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukaemia following MDS in complete remission after induction chemotherapy, British Journal of Haematology
Volume 150 Issue 3, Pages 293 – 302, 2010
Reference: No. (589/09)
Source: Scottish Medicines Consortium (SMC)
Date published: 12/04/2010 16:30
NCCN, AML-11, 2017
Marcos De Lima et al., Blood, 200Decitabine As Salvage Therapy for Relapse of AML and MDS after Allogeneic Stem Cell Transplantation – a Retrospective Multicenter Analysis on Behalf of the German Cooperative Transplant Study Group
Thomas Schroeder et al., Blood, 2016Prospective Phase II Study of Prophylactic Azacitidine and Donor Lymphocyte Infusions Following Allogeneic Hematopoietic Stem Cell Transplantation for High Risk Acute Myeloid Leukemia and Myelodysplastic Syndrome Blood, 2016