Atezolizumab alone or in combination did not demonstrate a favorable risk-benefit profile in myelodysplastic syndrome

Aaron T. Gerds, Bart L. Scott, Peter Greenberg, Tara L. Lin, Daniel A. Pollyea, Amit Verma, Monique Dail, Yuning Feng, Cherie Green, Connie Ma, Bruno C. Medeiros, Mark Yan, Kasra Yousefi, William Donnellan

Research output: Contribution to journalArticlepeer-review

2 Scopus citations


We present primary results from the phase 1b GO29754 study evaluating the safety and tolerability of atezolizumab, a programmed death-ligand 1 inhibitor, alone and in combination with azacitidine, a hypomethylating agent (HMA), in patients with relapsed/refractory (R/R) or HMA-naïve myelodysplastic syndrome (MDS). Patients with R/R MDS received atezolizumab for 12 months (cohort A) or atezolizumab plus azacitidine for 6 cycles followed by atezolizumab as maintenance for 8 cycles (cohort B). Patients with HMA-naïve MDS received atezolizumab plus azacitidine until loss of clinical benefit (cohort C). Safety, activity, and exploratory end points were investigated. Forty-six patients were enrolled and received treatment (cohort A, n 5 11; cohort B, n 5 14; cohort C, n 5 21). All patients experienced $1 adverse event (AE) on study, and all patients discontinued atezolizumab. In cohort A, 7 patients (63.6%) died, and no patients responded. In cohort B, 8 patients (57.1%) discontinued azacitidine, 11 (78.6%) died, and 2 (14.3%) responded. In cohort C, all 21 patients discontinued azacitidine, 13 died (61.9%), and 13 (61.9%) responded. The study was terminated by the sponsor before completion of recruitment because of the unexpected high early death rate in cohort C (6 [46.2%] of 13 deaths were due to AEs and occurred within the first 4 treatment cycles.). The high death rate and poor efficacy observed in this study do not support a favorable risk-benefit profile for atezolizumab as a single agent or in combination with azacitidine in R/R or HMA-naïve MDS.

Original languageEnglish (US)
Pages (from-to)1152-1161
Number of pages10
JournalBlood Advances
Issue number4
StatePublished - Feb 22 2022

ASJC Scopus subject areas

  • Hematology


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