Current and emerging pharmacotherapy for the treatment of childhood acute myeloid leukemia

ABSTRACT

Introduction

Pediatric acute myeloid leukemia (AML) is a rare disease that is profoundly heterogeneous at a molecular and clinical level. Although, in recent clinical trials, the 5-year event-free survival rates for childhood AML ranged between 49% and 64%, bone marrow relapse still occurs in up to one-third of cases. New therapies are required to continue progress in this aggressive hematological disease. Optimistically, we anticipate that the next challenge may be not a lack of appropriate therapies but an abundance of potentially effective strategies and a question of how best to incorporate them into pediatric clinical practice.

Areas covered

The focus of this review is to highlight all promising agents currently under investigation for pediatric AML, including nucleoside analogs, epigenetic modifiers, targeted small-molecule inhibitors, monoclonal antibodies, novel chemotherapeutics, and immunotherapies.

Expert opinion

While AML outcomes have improved over time for pediatric AML patients, our challenge is how to improve outcomes with our new knowledge of genetic and epigenetic aberrations. We posit to incorporate active therapy options into combination strategies and utilize targeted and immunotherapy approaches, as more opportunities are available.

KEYWORDS:

• Menin inhibitors
• menin
• AML
• children
• pediatric
• pediatric AML

Article highlights

• Pediatric acute myeloid leukemia (AML) is a rare disease that is profoundly heterogeneous at a molecular and clinical level with a variable prognosis.

• Progress over the past decade has significantly improved the understanding of leukemia’s genetic and epigenetic aberrations and impacted prognosis and risk stratification.

• Exciting novel agents have shown remarkable responses. The future challenge will be how to best integrate them into the limited number of pediatric AML trials.

This box summarizes key points contained in the article.

Acknowledgments

We thank Sarah Bronson, Scientific Editor in the Research Medical Library at The University of Texas MD Anderson Cancer Center, for editing this article.

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.