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Immunotherapy in pediatric acute lymphoblastic leukemia

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Abstract

The 5-year survival rate for children and adolescents with acute lymphoblastic leukemia (ALL) has improved to more than 90% in high-income countries. However, further increases in the intensity of conventional chemotherapy would be associated with significant adverse effects; therefore, novel approaches are necessary. The last decade has seen significant advances in targeted therapy with immunotherapy and molecular therapeutics, as well as advances in risk stratification for therapy based on somatic and germline genetic analysis and monitoring of minimal residual disease. For immunotherapy, the approval of antibody-based therapy (with blinatumomab in 2014 and inotuzumab ozogamicin in 2017) and T cell–based therapy (with tisagenlecleucel in 2017) by the US Food and Drug Administration has significantly improved the response rate and outcomes in patients with relapsed/refractory B-ALL. These strategies have also been tested in the frontline setting, and immunotherapy against a new ALL-associated antigen has been developed. Incorporating effective immunotherapy into ALL therapy would enable the intensity of conventional chemotherapy to be decreased and thereby reduce associated toxicity, leading to further improvement in survival and quality of life for patients with ALL.

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The authors thank Keith A. Laycock, PhD, ELS, for scientific editing of the manuscript.

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  1. Department of Oncology, St. Jude Children’s Research Hospital, MS 260, 262 Danny Thomas Place, Memphis, TN, 38105-3678, USA

    Hiroto Inaba & Ching-Hon Pui

  2. Department of Pediatrics, University of Tennessee Health Science Center, Memphis, TN, USA

    Hiroto Inaba & Ching-Hon Pui

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Inaba, H., Pui, CH. Immunotherapy in pediatric acute lymphoblastic leukemia. Cancer Metastasis Rev 38, 595–610 (2019). https://doi.org/10.1007/s10555-019-09834-0

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