Details

Autor(en) / Beteiligte

• Oran, Betül
• de Lima, Marcos
• Garcia-Manero, Guillermo
• Thall, Peter F.
• Lin, Ruitao
• Popat, Uday
• Alousi, Amin M.
• Hosing, Chitra
• Giralt, Sergio
• Rondon, Gabriela
• Woodworth, Glenda
• Champlin, Richard E.

Titel

A phase 3 randomized study of 5-azacitidine maintenance vs observation after transplant in high-risk AML and MDS patients

Ist Teil von

• Blood advances, 2020-11, Vol.4 (21), p.5580-5588

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2020

Link zum Volltext

Quelle

EZB-FREE-00999 freely available EZB journals

Beschreibungen/Notizen

• This study investigated the efficacy and safety of azacitidine maintenance in the posttransplant setting based on the encouraging phase 1/2 reports for azacitidine maintenance in patients with acute myeloid leukemia/myelodysplastic syndrome (AML/MDS). Between 2009 and 2017, a total of 187 patients aged 18 to 75 years were entered into a randomized controlled study of posttransplant azacitidine if they were in complete remission. Patients randomized to the treatment arm (n = 93) were scheduled to receive azacitidine, given as 32 mg/m2 per day subcutaneously for 5 days every 28 days for 12 cycles. The control arm (n = 94) had no intervention. Eighty-seven of the 93 patients started azacitidine maintenance. The median number of cycles received was 4; a total of 29 patients relapsed on study, and 23 patients withdrew from the study due to toxicity, patient's preference, or logistical reasons. Median relapse-free survival (RFS) was 2.07 years in the azacitidine group vs 1.28 years in the control group (P = .43). There was also no significant difference for overall survival, with a median of 2.52 years vs 2.56 years in the azacitidine and control groups (P = .85), respectively. Multivariate Cox regression analysis revealed no improvement in RFS or overall survival with the use of azacitidine as maintenance compared with the control group (hazard ratios of 0.73 [95% confidence interval, 0.49-1.1; P = .14] and 0.84 [95% confidence interval, 0.55-1.29; P = .43]). This randomized trial with azacitidine maintenance showed that a prospective trial in the posttransplant setting was feasible and safe but challenging. Although RFS was comparable between the 2 arms, we believe the strategy of maintenance therapy merits further study with a goal to reduce the risk of relapse in patients with AML/MDS. This trial was registered at www.clinicaltrials.gov as #NCT00887068. •In this phase 3, open-label, randomized trial, azacitidine maintenance did not improve RFS after transplant in high-risk AML/MDS.•We believe that posttransplant maintenance strategy merits further study to decrease the risk of relapse in AML/MDS patients. [Display omitted]