Outcomes of unrelated umbilical cord blood transplantation in 158 pediatric patients consecutively transplanted at a single institution using cord blood units from a single bank


Journal Article

Over the past decade, umbilical cord blood (UCB) has been shown to provide adequate stem cells for bone marrow reconstitution after myeloablative therapy. Reports to date include 2 from registries and a few small pilot studies, but there have been no reports of a large series of patients transplanted at a single institution. We now report the outcomes of 158 consecutively transplanted pédiatrie patients (<18 years of age) with hématologie malignancies, bone marrow failure or immunodeficiency syndromes, or inborn errors of metabolism treated at a single institution and transplanted with UCB units from a single bank over the past 7 years. Factors associated with a favorable outcome are identified. Ninety-eight patients were transplanted for a high risk or recurrent malignancy (48 ALL, 29 ANLL, 6 MDS, 2 JCML, 6 CML, 5 Neuroblastoma, 2 NHL) and 60 for non-malignant conditions (13 for BM failure, 18 for immunodeficiency, 29 for inborn errors). The median age was 5.13 yrs (range 0.08-17.9 yrs), median weight 16.7 kg (range 3.9-93.3), 54% were CMV positive, 63% male, 26 % were of minority ethnic background, and 84% had high risk characteristics. Four pts received 6/6, 45 pts 4/6 and 15 pts 3/6 matched grafts. The median cell dose was 55 million cells/kg and median CD34 dose 489,000 cells/kg. The median days to an ANC of 500/mm3, platelet count of 50K/mm3 and RBC transfusion independence were 22, 74,and 48, respectively. The probabilities of reaching an ANC of 500 by days 42 and 60 were 89% and 95% while the probability of reaching an untransfused platelet count of 50K/mm3 by day 180 was 88%. The incidence of acute GvHD, 35% developed moderate (17%) to severe (18%) disease. The probability of chronic GvHD was 14%, 85%limited. Relapses occurred in 16% of patients with malignancies within a median of 5 months (range 1-22). The probability of overall EFS at 3 years is 45%. In univariate analysis.the factors associated with a favorable outcome were cell dose and age, in multivariate analysis, cell dose/kg was the only statistically significant variable predictive of a favorable outcome. We conclude that banked, unrelated UCB (even if HLA mismatched at 1-2 antigens) should be strongly considered as the unrelated donor source for pédiatrie patients lacking a matched related stem cell donor.

Duke Authors

Cited Authors

  • Kurtzberg, J; Martin, PL; Szabolcs, PM

Published Date

  • December 1, 2000

Published In

Volume / Issue

  • 96 / 11 PART I

International Standard Serial Number (ISSN)

  • 0006-4971

Citation Source

  • Scopus