¹Division of Pediatric Hematology, Oncology, and Blood and Marrow Transplantation, Department of Pediatrics University of Minnesota Medical School, Minneapolis, MN, USA

Correspondence: Dr ML MacMillan, Department of Pediatrics, Blood and Marrow Transplant Program, University of Minnesota, MMC 484, 420 Delaware Street SE, Minneapolis, MN 55455, USA. E-mail: macmi002@umn.edu

Received 9 July 2008; Revised 18 September 2008; Accepted 24 September 2008; Published online 27 October 2008.

Abstract

Suboptimal neutrophil and platelet recovery after unrelated donor umbilical cord blood transplantation (UCBT) may be due in part to an impaired microenvironment after intensive chemoradiotherapy. In an attempt to speed hematopoietic recovery, 15 pediatric patients with high-risk acute leukemia were enrolled on a single-institution phase I–II clinical trial in which ex-vivo culture-expanded MSCs from haploidentical parental donors were infused at the time of UCBT. Eight patients received MSCs on day 0, with three patients having a second dose infused on day 21. No serious adverse events were observed with any MSC infusion. All eight evaluable patients achieved neutrophil engraftment at a median of 19 days. Probability of platelet engraftment was 75%, at a median of 53 days. With a median follow-up of 6.8 years, five patients remain alive and disease free. The results of this pilot study show that infusion of ex-vivo culture-expanded haploidentical MSCs into unrelated pediatric UCBT recipients can be performed safely. This encouraging safety profile with haploidentical MSCs supports the investigation of unrelated 'off the shelf' allogeneic HLA-mismatched MSC products.