Data regarding pediatric UCBT for nonmalignant diseases are limited to abstract reports59-62 and publications by Locatelli et al.63 and Staba et al.64 Locatelli et al. reported on 44 patients, three-quarters of whom had a hemoglobinopathy/thalassemia and one-quarter had sickle cell disease (SCD). The UCB donor was related in all transplants. Ninety-three percent of the grafts were HLA identical. Neutrophil recovery at 60 days occurred in 89% of the patients. The median time to neutrophil recovery was 23 days. Platelet recovery was reported in 90% of the patients at a median of 39 days. Acute GVHD developed in 11% of the patients. The 2-year Kaplan-Meier estimate of EFS was 79% for patients with thalassemia and 90% for those with SCD. OS was 100%.
Staba et al. reported on 20 consecutive patients with Hurler's syndrome. All UCB donors were unrelated. The median age was 11 months. One patient was HLA identical, 11 were disparate at one locus, and the remaining grafts were mismatched at >2 loci. The conditioning regimen was Busulfan(Bu)/Cy/ATG. GVHD prophylaxis consisted of cyclosporine and methyl-prednisolone. The mean cell dose was 8.34 X 107/kg
Table 97.5 Adult unrelated donor UCBT
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