Children as well as adolescent and young adult (AYA) patients with acute lymphoblastic leukemia (ALL) who relapse following frontline therapy typically receive intensive chemotherapy followed by hematopoietic stem cell transplant (HSCT). Unfortunately, this treatment option is not always effective, and these patients tend to have poor outcomes. At the 2019 American Society of Hematology (ASH) Annual Meeting, Patrick Brown, MD (Johns Hopkins University, Boston, Massachusetts, United States), presented results from the Children’s Oncology Group Study ALL1331, which compared blinatumomab to chemotherapy in 208 patients aged 1 to 30 years with intermediate to high risk ALL who had relapsed following first-line therapy (Abstract LBA1).
At a median follow-up of 1.4 years, blinatumomab was associated with significant improvements in both disease-free survival (DFS) and overall survival (OS) compared to chemotherapy. The estimated 2-year DFS rate was 59.3% with blinatumomab and 41.0% with chemotherapy (P = .05). Similarly, the estimated 2-year OS was 79.4% in patients receiving blinatumomab, while chemotherapy was associated with a 2-year OS of 59.2% (P = .05). Blinatumomab treatment was also associated with significantly higher rates of minimal residual disease (MRD) negativity, a key prognostic indicator for patients with relapsed ALL, and a greater proportion of patients receiving blinatumomab subsequently underwent HSCT (79% vs 45%; P<.0001). Blinatumomab was well-tolerated, and adverse events (AEs) were consistent with the known safety profile of this agent. AEs occurred less frequently and were less severe in patients receiving blinatumomab compared to patients receiving chemotherapy.
Dr Brown concluded that blinatumomab is a safe and effective treatment option for children and AYA patients with intermediate/high-risk relapsed ALL, associated with improved outcomes and a better safety profile than chemotherapy. Blinatumomab should now be considered a standard of care in this setting, he said. Future studies will focus on evaluating the role of blinatumomab in the era of immunotherapy, including the combination of blinatumomab with immune checkpoint inhibitors and the use of chimeric antigen receptor modified (CAR)-T cells.
Read more about this study on Medscape Medical News.