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

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 Scholars

Author Joanne Kurtzberg Pediatrics, Transplant and Cellular Therapy

Author Paul Langlie Martin Pediatrics, Transplant and Cellular Therapy