Title:Safety and Efficacy of Immune Checkpoint Inhibitors in Children and
Young Adults with Haematological Malignancies: Review and Future Perspectives
Volume: 20
Issue: 1
Author(s): Eleni Tsotridou, Eleni Vasileiou, Elpis Mantadakis and Athanasios Tragiannidis*
Affiliation:
- 2nd Department of Paediatrics, Childhood and Adolescent Hematology-Oncology Unit, School of Medicine, Faculty of
Health Sciences, Aristotle University of Thessaloniki, AHEPA Hospital, Thessaloniki, Greece
Keywords:
Immune checkpoint inhibitors, pembrolizumab, nivolumab, haematological malignancies, children, paediatrics.
Abstract: Despite the marked improvement in overall survival rates of paediatric patients with haematological
malignancies that has been achieved during the last decades, there is still a pressing
need for novel therapeutic approaches for the subset of patients with relapsed or refractory disease.
Immune checkpoint inhibitors aim to induce potent anti-tumour immune responses by targeted
blocking of inhibitory receptors and have shown promising results in preclinical models and
studies on the adult population. However, paediatric malignancies present unique features, and so
far, experience with these agents is limited. In the current review, we present an overview of efficacy
and safety data from case reports, case series, and clinical trials employing the use of immune
checkpoint inhibitors in children, adolescents, and young adults with haematological malignancies.
We also discuss new possibilities involving novel targets and combination treatments and provide
a summary of the currently registered clinical trials.