A groundbreaking pediatric cancer trial combining blinatumomab with standard chemotherapy for children with B-cell acute lymphoblastic leukemia has received the prestigious David Sackett Trial of the Year Award from the Society for Clinical Trials. The Children's Oncology Group AALL1731 study demonstrated that adding blinatumomab to chemotherapy reduces the risk of relapse by approximately two-thirds, marking what researchers describe as the biggest breakthrough in childhood cancer treatment in decades.
The award, presented on May 20 at the Society for Clinical Trials annual meeting in Vancouver, British Columbia, recognizes randomized controlled trials that provide a basis for substantial change in healthcare and "improve the lot of humankind." Since 2008, the Sackett Award has honored trials published in the previous calendar year that demonstrate significant clinical impact.
Study Results Transform Treatment Standards
The Phase III AALL1731 trial revealed striking improvements in patient outcomes when blinatumomab was added to standard chemotherapy regimens. Children receiving the combination therapy achieved a three-year disease-free survival rate of 96%, compared to 87.9% for those receiving chemotherapy alone. This represents a 61% reduction in the risk of relapse, secondary malignancy, or remission-related death.
"The AALL1731 study results are truly practice-changing, further solidifying blinatumomab's role as the standard of care for a large number of children with B-ALL," said Dr. Sumit Gupta, co-chair of the Children's Oncology Group AALL1731 study and oncologist at The Hospital for Sick Children (SickKids). "These breakthrough data showing a significant improvement in disease-free survival are poised to bring substantial clinical value to children with newly diagnosed B-ALL."
The study enrolled 4,264 newly diagnosed National Cancer Institute standard risk B-cell acute lymphoblastic leukemia patients, with 2,334 risk-stratified at the end of induction therapy. The trial began in July 2019 and concluded early in July 2024 after the first interim analysis demonstrated better outcomes than expected with the combination therapy.
Clinical Significance and Global Impact
B-cell acute lymphoblastic leukemia represents the most common childhood cancer. While the disease has a high overall cure rate, relapse remains a leading cause of death among children with this condition. The AALL1731 findings have now established the blinatumomab-chemotherapy combination as the ideal standard of care for children with B-ALL worldwide.
"Receiving the Sackett Award is monumental," said study co-leader Dr. Rachel Rau, a pediatric hematologist-oncologist at Seattle Children's Hospital. "It really recognizes the significant impact and magnitude of our work, placing it in the context of world health. For me, it was a big picture moment. This practice-changing finding will lead to many lives saved, and given that the results apply to children, the years of life ultimately saved is really incredible to think about."
Research Collaboration and Implementation
The study was designed and conducted independently from industry, with sponsorship from the Cancer Therapy Evaluation Program of the National Cancer Institute. Amgen provided blinatumomab and support through an NCI Cooperative Research and Development Agreement. Multiple institutions contributed to the trial's success, including Miller Children's & Women's Hospital through the Jonathan Jaques Children's Cancer Institute.
"Children's Oncology Group protocols change how we treat children with cancer – this represents a landmark study integrating targeted therapy into standard chemotherapy," said Dr. Jacqueline Casillas, medical director of the Jonathan Jaques Children's Cancer Institute at Miller Children's & Women's Hospital.
The integration of targeted immunotherapies like blinatumomab represents a significant advancement in pediatric leukemia care, building upon traditional chemotherapy approaches to enhance outcomes for young patients. The success of AALL1731 has reshaped treatment protocols worldwide, offering families improved survival rates and quality of life prospects.
"Over the last decade, BLINCYTO has reshaped the treatment landscape for B-ALL, offering a critical lifeline for thousands of adult and pediatric patients," said Dr. Jay Bradner, executive vice president of Research and Development and chief scientific officer at Amgen. "These powerful new data leave us little doubt about the profound impact of this medicine for a large number of children affected by this disease."
