Challenges in the harmonization of immune monitoring studies and trial design for cell-based therapies in the context of hematopoietic cell transplantation for pediatric cancer patients.

Clinical trials aimed at improving results of hematopoietic cell transplantation (HCT) by adjuvant cell-based interventions in children have been limited by small numbers and pediatric-specific features. The need for a larger number of pediatric HCT centers to participate in trials has resulted in a demand for harmonization of disease-specific clinical trials and immune-monitoring. Thus far, most phase I/II trials select different end points evaluated at disparate time points, making inter-study comparisons difficult and, sometimes, impossible. In this review, we discuss the various aspects that are important to consider for harmonizing clinical trial design as well as the critical elements for standardized (immune)-monitoring protocols in cell-based intervention trials in the context of HCT. Comparison data from trials applying harmonized trial design will lead to optimized immunotherapeutic treatment protocols to maximize clinical efficacy while minimizing toxicity. Copyright © 2015 International Society for Cellular Therapy. Published by Elsevier Inc. All rights reserved.

Citation

Stefan Nierkens, Arjan C Lankester, R Maarten Egeler, Peter Bader, Franco Locatelli, Michael A Pulsipher, Catherine M Bollard, Jaap-Jan Boelens, Westhafen Intercontinental Group. Challenges in the harmonization of immune monitoring studies and trial design for cell-based therapies in the context of hematopoietic cell transplantation for pediatric cancer patients. Cytotherapy. 2015 Dec;17(12):1667-74

Mesh Tags

PMID: 26589751

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