Hematopoietic stem cell transplantation and immunotherapy for pediatric acute myeloid leukemia: an open challenge

Abstract

The present review summarizes the role of immunotherapy in the treatment of pediatric acute myeloid leukemia (AML). Through the evaluation of recent clinical and pre-clinical studies, different approaches are taken into account: from the allogeneic transplantation as remission consolidation strategy, to the most advanced targeted cell therapy approaches for relapse prevention. The entangled question of immunotherapy's evolving role in AML is also addressed, as new diagnostic tools provide better risk stratification, classic chemotherapy allows for higher survival rates and targeted drugs became available for children too. Moreover, the need for collaborative, prospective studies in this field is highlighted. Only through sound data it will be possible to compare and possibly integrate new therapies with conventional approaches, with the final aim of achieving a higher survival rate and a better quality of life for our patients.

Keywords::

• acute myeloid leukemia
• cell therapy
• childhood leukemia
• immunotherapy
• stem cell transplantation

Financial & competing interests disclosure

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.

No writing assistance was utilized in the production of this manuscript.

Key issues

• Relapse represents nowadays the major hurdle for acute myeloid leukemia therapy in children.

• Risk assessment at diagnosis and during treatment plays a pivotal role in determining the intensity of the treatment, namely, in the indication to stem cell transplantation in first remission.

• Immunotherapy under various forms can play an active role in the remission consolidation as well as in the relapse/refractory setting.

• Different forms of immunotherapy have proven feasible and safe on small clinical scale, and cooperative studies are needed to document efficacy of these treatments.

• Long-term toxicity evaluation should be more and more integrated in the evaluation of immunotherapy compared with conventional treatment, as long-terms survivors, especially among children, will be increasingly parallel to the efficacy of these new therapies.