TY - JOUR
T1 - 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
AU - Nierkens, Stefan
AU - Lankester, Arjan C.
AU - Egeler, R. Maarten
AU - Bader, Peter
AU - Locatelli, Franco
AU - Pulsipher, Michael A.
AU - Bollard, Catherine M.
AU - Boelens, Jaap Jan
PY - 2015/12/1
Y1 - 2015/12/1
N2 - 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.
AB - 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.
KW - Cell based therapy
KW - Clinical trial design
KW - Hematopoietic cell transplantation, immune monitoring
KW - Pediatric cancer
UR - http://www.scopus.com/inward/record.url?scp=84947545452&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84947545452&partnerID=8YFLogxK
U2 - 10.1016/j.jcyt.2015.09.008
DO - 10.1016/j.jcyt.2015.09.008
M3 - Article
C2 - 26589751
AN - SCOPUS:84947545452
VL - 17
SP - 1667
EP - 1674
JO - Cytotherapy
JF - Cytotherapy
SN - 1465-3249
IS - 12
ER -