Intravenous infusion of banked autologous and sibling umbilicalcord blood (CB) is being studied in children with acquired neu-rological conditions and has demonstrated safety and feasibilityin phase I/II studies. To provide access to this investigationalprocedure while efficacy trials are conducted, an expandedaccess program (EAP) was developed.
More than 1,400 children have enrolled in the screening protocol to date. From November 2017 to June 2019, 276 children received 302 CB infusions under the EAP.
No child required treatment or developed an infection post-infusion. One-year follow-up questionnaires were completed by 54 of 83 (65%) families. Parental assessment of clinical improvements varied.