Blinatumomab Improves Outcomes, Toxicity in High-Risk Pediatric ALL: Martin Schrappe, MD, PhD

Replacing high-dose combination chemotherapy with blinatumomab in newly diagnosed pediatric high-risk B-cell acute lymphoblastic leukemia (ALL) cut the risk of an adverse event by nearly 50% while also meaningfully reducing toxicity, explained Martin Schrappe, MD, PhD, who spoke with The American Journal of Managed Care^®. Schrappe is the lead investigator of “Replacement of High Dose Combination Chemotherapy With Blinatumomab in Newly Diagnosed Pediatric High-Risk B-Cell ALL Improves Efficacy and Safety in the Randomized Phase 3 AIEOP-BFM ALL2017 Trial” and is presenting the data during the plenary abstracts session at the European Hematology Association 2026 Congress in Stockholm, Sweden, this afternoon.

He explained that the phase 3 trial was designed around a critical gap in pediatric ALL care: a subset of patients, approximately 20% to 25%, for whom standard chemotherapy offers limited efficacy while still carrying substantial toxicity. Consequently, the study set out to both improve outcomes and meaningfully reduce treatment burden.

Most prior study groups incorporating blinatumomab (Blincyto; Amgen) into frontline ALL therapy had added the agent on top of an existing chemotherapy backbone. When this trial was designed approximately 7 years ago, Schrappe explained that it was not yet clear whether immunotherapy could replace chemotherapy rather than supplement it.

That uncertainty made the results particularly striking, according to Schrappe. Replacing chemotherapy with blinatumomab not only substantially reduced toxicity but also improved event-free survival, reducing the risk of an event by nearly 50% in most patients.

He also framed the minimal residual disease findings through a patient-centered lens. Although MRD negativity is an important early indicator of treatment success, Schrappe explained that patients do not directly experience changes in MRD status. Instead, they are more likely to value both lower recurrence rates and fewer treatment-related adverse events.

Regarding safety, severe mucositis and serious infections, which are both common complications of chemotherapy, declined sharply in patients treated with blinatumomab. Neurotoxicity, however, emerged as a distinct adverse effect associated with immunotherapy; Schrappe emphasized that it remains an important consideration for monitoring.

As for further research, he explained that most study groups are currently evaluating blinatumomab in medium- and high-risk patients, while the Children's Oncology Group has extended its investigation into standard-risk populations. Schrappe said he anticipates that blinatumomab will eventually be incorporated across all risk groups, with future research focused on determining how much chemotherapy can safely be replaced.

“We are starting a study in low-risk patients in a few weeks, in which we try to replace large parts of the chemotherapy with immunotherapy, with the help of immunotherapy to allow patients to stay basically all the time at home and not to go to the hospital for a long time; I think this is the way to go,” he concluded.