Duke University Durham, North Carolina, United States
Background/Case Studies: An Expanded Access Program (EAP) was established in 2017 for the use of umbilical cord blood (CB) infusion in children with certain acquired neurologic conditions. Initially, only autologous or at least haploidentical sibling CB units were utilized. Given growing evidence of the efficacy of CB infusion in children with cerebral palsy (CP) and safety of allogeneic unrelated donor (URD) CB infusion, the EAP was expanded in 2024 to allow URD CB infusion in select children with CP.
Study
Design/Methods: Children at least 6 months of age with CP due to hypoxic ischemic encephalopathy (HIE), periventricular leukomalacia (PVL), stroke/bleed, or congenital hydrocephalus were eligible for an URD CB infusion if they had evidence of motor impairment, were a Gross Motor Function Classification (GMFCS) levels I-IV (age 2 years and older), and did not have a qualified autologous or sibling CB unit (CBU) available. After remote screening, an URD CBU from the Carolinas Cord Blood Bank was identified. URD CBUs had to provide a pre-cryopreservation cell dose of >= 2x10e7 total nucleated cells (TNC)/kg, have negative sterility and maternal infectious disease markers, and be at least a 4/6 HLA match to the recipient. If the TNC permitted, a single CBU was utilized for multiple infusions, but each recipient could only receive infusions from a single donor. On the day of infusion, CBUs were thawed and infused via peripheral IV under direct supervision in the outpatient setting after premedication with diphenhydramine and methylprednisolone.
Results/Findings: From June 2024 to May 2025, 34 children received a total of 40 URD CB infusions through the EAP. Patient and CBU characteristics are listed in Table 1. All infusions were completed as intended. One child received a second dose of diphenhydramine immediately post-infusion due to eye swelling and itching that resolved completely. There were no other infusion reactions or related adverse events to date. Compared to previously reported autologous/sibling CB infusions, the median precryopreservation TNCC of URD CBUs was 3 times higher (132.5x10e7 vs 43.4x10e7) and there were no post-thaw positive sterility cultures.
Conclusions: URD CB infusion is safe and feasible in young children with CP in a well-equipped outpatient setting and provides access to children who lack a suitable autologous or sibling CBU. Efficacy studies are planned.
