New Research from Children's Hospital of Philadelphia to Transform Standard of Care Across the Globe for Newly Diagnosed Children with B-cell Acute Lymphoblastic Leukemia

Results will be presented at the 66^th American Society of Hematology Annual Meeting

PHILADELPHIA, Dec. 7, 2024 /PRNewswire/ -- Researchers at Children's Hospital of Philadelphia (CHOP) and the Children's Oncology Group (COG) announced the results of a Phase 3 study that demonstrated adding the bi-specific T-cell engager, blinatumomab, to chemotherapy for newly diagnosed National Cancer Institute (NCI) standard risk (SR) B-cell acute lymphoblastic leukemia (B-ALL) pediatric patients significantly improves survival outcomes. The results were published today in the New England Journal of Medicine and will be presented during the plenary session at the 66^th American Society of Hematology (ASH) Annual Meeting & Exposition in San Diego on December 8.

Since its initial approval a decade ago as an addition to chemotherapy for children and adults with relapsed B-ALL, blinatumomab, a type of immunotherapy that treats certain kinds of leukemia by binding to cancer cells and immune cells, has already made a major impact on the B-ALL treatment landscape. However, relapsed B-ALL remains a major cause of childhood cancer-related deaths, with nearly half of relapses occurring in children with SR B-ALL. For this reason, researchers sought to determine if this regimen would be effective in children at the time of diagnosis.

"Our team is dedicated to the global improvement of cure rates and decreasing side effects for children, adolescents and young adults," said Stephen P. Hunger, MD, a study co-author, former COG ALL Disease Committee Chair, Chief of CHOP's Division of Oncology and Director of CHOP's Center for Childhood Cancer Research. "This approach defines a new treatment standard for children newly diagnosed with B-ALL."

In AALL173, the Phase 3 randomized trial, researchers evaluated whether two non-sequential 28-day blinatumomab cycles added to chemotherapy improved disease-free survival (DFS) in newly diagnosed SR B-ALL pediatric patients between June 2019 and June 2024. CHOP was the largest enrolling site for the study. SR average (SR-Avg) patients tend to have a lower white blood cell count and favorable genetic features at diagnosis, compared with SR high (SR-High) patients, who have a higher white blood cell count and specific genetic mutations that make the disease more aggressive and challenging to treat. Of those originally analyzed, 1,440 SR-Avg/SR-High patients, whose average age was four years-old, were randomized, including 835 SR-Avg and 605 SR-High.

Overall, the three-year DFS was 96.0% for patients treated with chemotherapy plus blinatumomab compared with 87.9% for those treated with only chemotherapy, the current standard of care. Among SR-Avg patients, 3-year DFS was 97.5% for patients treated with blinatumomab compared with 90.2% for those treated with only chemotherapy. For SR-High patients, 3-year DFS was 94.1% for those treated with blinatumomab compared with 84.8% for those treated with only chemotherapy.

"These findings mark a significant clinical advancement, cementing blinatumomab and chemotherapy together as the safest and most effective treatment for children with B-ALL," said David T. Teachey, MD, a study co-author, Chair of the Acute Lymphoblastic Leukemia Disease Committee in COG, and Director of Clinical Research at the Center for Childhood Cancer Research at CHOP. "Moving forward, we expect more findings on higher-risk pediatric patients, as well as a focus on improving access to this lifesaving treatment for all children and their families."

This study was supported by grants from the National Institutes of Health/National Cancer Institute (U10CA180899; U20CA180886) and from the St. Baldrick's Foundation.

Gupta et al. "Blinatumomab in standard risk pediatric B-acute lymphoblastic leukemia." NEJM. Online December 7, 2024. DOI: 10.1056/NEJMoa2411680.

About Children's Hospital of Philadelphia:  

A non-profit, charitable organization, Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, the hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country. The institution has a well-established history of providing advanced pediatric care close to home through its CHOP Care Network, which includes more than 50 primary care practices, specialty care and surgical centers, urgent care centers, and community hospital alliances throughout Pennsylvania and New Jersey, as well as the Middleman Family Pavilion and its dedicated pediatric emergency department in King of Prussia. In addition, its unique family-centered care and public service programs have brought Children's Hospital of Philadelphia recognition as a leading advocate for children and adolescents. For more information, visit https://www.chop.edu. 

Contact:
Jennifer Lee
Children's Hospital of Philadelphia
(267) 426-6084
[email protected]

SOURCE Children's Hospital of Philadelphia