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Review
. 2015 Dec;17(12):1667-74.
doi: 10.1016/j.jcyt.2015.09.008.

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

Stefan Nierkens  1 , Arjan C Lankester  2 , R Maarten Egeler  3 , Peter Bader  4 , Franco Locatelli  5 , Michael A Pulsipher  6 , Catherine M Bollard  7 , Jaap-Jan Boelens  8 ; Westhafen Intercontinental Group
Affiliations
Review

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

Stefan Nierkens et al. Cytotherapy. 2015 Dec.

Abstract

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.

Keywords: cell based therapy; clinical trial design; hematopoietic cell transplantation, immune monitoring; pediatric cancer.

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Conflict of interest statement

Disclosure of interests: The authors have no commercial, proprietary, or financial interest in the products or companies described in this article.

Figures

Figure 1.
Figure 1.
Relevant parameters for immune monitoring to be considered for harmonization early in the process of multi-center trial design.
See this image and copyright information in PMC

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