Although almost 90% of children with acute lymphoblastic leukemia (ALL) and ∼60% of children with acute myeloid leukemia are cured with frontline therapy, relapse and chemotherapy resistance are significant challenges that contribute to morbidity and mortality. Even with long-term survival, the acute and chronic burdens of therapy are major issues for patients and families. Long-term side effects occur, including cardiac, endocrinologic, neurcognitive, orthopedic, and psychosocial problems, and healthy survivorship is frequently compromised. With goals of minimizing relapse and/or decreasing traditional chemotherapy-associated toxicities, exploration of immunotherapeutic strategies has moved to the forefront in pediatric cancer. New immunotherapy approaches provide a major paradigm shift in oncology overall, often curing previously incurable patients. The past several years have yielded successful uses across a variety of malignancies, and enthusiasm continues to rise for applying these therapies more broadly. Herein we discuss current approaches incorporating the bispecific T-cell engager blinatumomab, the antibody-drug conjugate inotuzumab ozogamicin (InO), and CD19-directed chimeric antigen receptor T cells in children with relapsed/refractory B-cell ALL and discuss the potential for using these immunotherapies in the treatment of newly diagnosed children.
© 2019 by The American Society of Hematology. All rights reserved.
Conflict of interest statement
Conflict-of-interest disclosure: L.G. received travel reimbursement and honoraria for service on advisory and data safety and monitoring boards from Amgen, Genentech/Roche, and Novartis; has a spouse employed by, and has stock in, Anchiano Therapeutics; and has stock and/or stock options in Amgen, Blueprint Medicines, Celgene, Clovis Oncology, OnKure, Mirati, and Sanofi Paris. A.W. declares no competing financial interests.
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