Pediatric patients with non-malignant disorders can achieve “excellent overall survival” without significant GVHD after cord blood transplantation (CBT), according to results of a prospective trial presented at the BMT Tandem Meetings.
Three-year overall survival was 96% in 25 consecutive pediatric patients transplanted for severe combined immunodeficiency (SCID) (n=17), immunodysregulation polyendocrinopathy enteropathy X-linked syndrome (IPEX) (n=2), leukocyte adhesion deficiency (LAD) (n=1), Wiskott-Aldrich syndrome (n=1), Krabbe (n=1), mannosidosis (n=1), hemophagocytic lymphohistiocytosis (HLH) (n=1), and Diamond-Blackfan anemia (n=1).
Median age of the patients was 8 months (range, 2-66), and five patients (25%) had persistent viral infections prior to transplant, and 7 patients (30%) required mechanical ventilation prior to transplantation.
At a median follow up of 2 years (range, 10 days-5 years), all but one evaluable patient achieved full donor chimerism (>95% donor-origin cells in peripheral blood) by day 42. No patients experienced grade III-IV acute GVHD or chronic GVHD. Only one patient (4%) developed grade II acute GVHD (skin).