Clinical outcomes of unrelated cord blood transplantation in children with malignant and non-malignant diseases: Multicenter experience in China

Pediatr Transplant. 2018 Feb;22(1). doi: 10.1111/petr.13090. Epub 2017 Dec 14.

Abstract

This multicenter retrospective study included 184 children with malignant and non-malignant diseases who underwent UCBT between January 1998 and August 2012. The malignant disease group included 101 children with ALL, AML, CML, JMML, and MDS, and the non-malignant disease group included 83 children with PID, β-thalassemia, IMD BMF, and HLH. The median duration to neutrophil and platelet engraftment was 16 and 35 days in the malignant disease group vs 15 and 38 days in the non-malignant disease group. The cumulative incidence of grade II-IV aGVHD and cGVHD was 25.6% and 13.5% in the malignant disease group vs 19.7% and 11.1% in the non-malignant disease group, respectively. The median duration and cumulative incidence of neutrophil and platelet engraftment, and the cumulative incidence of grade II-IV aGVHD and cGVHD were similar between the two groups. Of the 184 pediatric patients, 114 patients survived during a median follow-up period of 14 months (range 4-138). The 5-year OS and DFS were not statistically different between the two groups (56.3% and 46.1% in malignant disease group vs 68.5% and 52.8% in non-malignant disease group). The above results indicate that UCB is a viable source for HSCT for children with malignant or non-malignant diseases, especially in urgent cases.

Keywords: children; malignant diseases; non-malignant diseases; unrelated cord blood transplantation.

Publication types

  • Multicenter Study

MeSH terms

  • Adolescent
  • Bone Marrow Diseases / mortality
  • Bone Marrow Diseases / therapy*
  • Child
  • Child, Preschool
  • China
  • Cord Blood Stem Cell Transplantation / methods*
  • Cord Blood Stem Cell Transplantation / mortality
  • Female
  • Follow-Up Studies
  • Humans
  • Immunologic Deficiency Syndromes / mortality
  • Immunologic Deficiency Syndromes / therapy*
  • Infant
  • Infant, Newborn
  • Leukemia / mortality
  • Leukemia / therapy*
  • Lymphatic Diseases / mortality
  • Lymphatic Diseases / therapy*
  • Male
  • Metabolic Diseases / mortality
  • Metabolic Diseases / therapy*
  • Retrospective Studies
  • Treatment Outcome
  • Unrelated Donors*