Advances in immunotherapy for pediatric acute myeloid leukemia

Challice L. Bonifant, Mireya Paulina Velasquez, Stephen Gottschalk

Research output: Contribution to journalReview articlepeer-review

9 Scopus citations


Introduction: Achieving better disease control in patients diagnosed with acute myeloid leukemia (AML) has proven challenging. Overall survival has been impacted by addressing treatment related mortality with focused supportive care measures. Despite this improvement, it remains difficult to induce durable leukemia remissions despite aggressive chemotherapeutic regimens. The addition of hematopoietic stem cell transplants (HSCT) has allowed further treatment intensification and provided the benefit of graft-versus-leukemia (GVL) effect. However, HSCT carries the risk of transplant related morbidities, particularly GVHD, and anti-tumor responsiveness is still suboptimal. Thus, there is a need for alternate therapies. Immunotherapy has the potential to address this need. Areas covered: This review highlights promises and challenges to immune-based therapies for AML. It aims to summarize immunotherapeutic strategies trialed in AML patients to date, inclusive of: antibodies, vaccines, and cellular therapy. It emphasizes those being used in the pediatric population, but also includes adult clinical trials and translational science that may ultimately extend to pediatric patients. Expert opinion: The elusiveness of an ideal surface antigen target together with an immunosuppressive leukemic microenvironment add to the already difficult challenge in developing AML-targeted immunotherapies. Though many hurdles remain, recent translational discovery and progressive clinical advances anticipate exciting future developments.

Original languageEnglish (US)
Pages (from-to)51-63
Number of pages13
JournalExpert Opinion on Biological Therapy
Issue number1
StatePublished - Jan 2 2018
Externally publishedYes


  • Acute myeloid leukemia
  • NK cell therapy
  • T cell therapy
  • antibodies
  • immunotherapy
  • vaccines

ASJC Scopus subject areas

  • Pharmacology
  • Drug Discovery
  • Clinical Biochemistry


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