Will Post-Transplantation Cell Therapies for Pediatric Patients Become Standard of Care?

Arjan C. Lankester, Franco Locatelli, Peter Bader, Eva Rettinger, Maarten Egeler, Satyendra Katewa, Michael A. Pulsipher, Stefan Nierkens, Kirk Schultz, Rupert Handgretinger, Stephan A. Grupp, Jaap Jan Boelens, Catherine M. Bollard

Research output: Contribution to journalArticlepeer-review


Although allogeneic hematopoietic stem cell transplantation (HSCT) is a curative approach for many pediatric patients with hematologic malignancies and some nonmalignant disorders, some critical obstacles remain to be overcome, including relapse, engraftment failure, graft-versus-host disease (GVHD), and infection. Harnessing the immune system to induce a graft-versus-tumor effect or rapidly restore antiviral immunity through the use of donor lymphocyte infusion (DLI) has been remarkably successful in some settings. Unfortunately, however, the responses to DLI can be variable, and GVHD is common. Thus, manipulations to minimize GVHD while restoring antiviral immunity and enhancing the graft-versus-tumor effect are needed to improve outcomes after allogeneic HSCT. Cellular therapies, defined as treatment modalities in which hematopoietic or nonhematopoietic cells are used as therapeutic agents, offer this promise for improving outcomes post-HSCT. This review presents an overview of the field for pediatric cell therapies in the transplant setting and discusses how we can broaden applicability beyond phase I.

Original languageEnglish
Pages (from-to)402-411
Number of pages10
JournalBiology of Blood and Marrow Transplantation
Issue number3
Publication statusPublished - Mar 1 2015


  • Adoptive cellular therapy
  • Chimeric antigen receptor
  • Pediatrics
  • Stem cell transplantation

ASJC Scopus subject areas

  • Transplantation
  • Hematology


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