Use of Unlicensed Unrelated Umbilical Cord Blood Expands Access to Underserved Patients: Report of 2466 Transplants in a Racially/Ethnically Diverse Population

Background Umbilical Cord Blood (UCB) transplant (UCBT) is a curative procedure for patients with hematologic malignancies and genetic disorders, and expands transplant options for a racially/ethnically diverse population, who are often unable to find a fully matched unrelated donor. In 2011, the Food and Drug Administration (FDA) required that all unrelated UCBT use either licensed UCB or unlicensed UCB via an Investigational New Drug (IND). Methods To allow continued access to unlicensed UCB units, the National Marrow Donor Program® (NMDP) manages a prospective distribution protocol for unlicensed UCB units, under an IND, 10-Cord Blood Access.  This is an interim report of outcomes of 2466 US patients receiving unlicensed UCB units from 2011-2016.  Of note, only 6% of available UCB units were licensed by December 2016. Results 1509 adults and 957 children (564 malignant disease and 393 non-malignant disease) received single or double UCBT between October 2011 and December 2016.  Median age was 31 years ( Conclusions Use of unlicensed UCB units is safe and effective, and provides an important graft source for a diverse population.