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

BackgroundUmbilical 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).MethodsTo 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.Results1509 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 (<1 to 81); 50% of children (Figure 1) and 35% of adults (Figure 2) were non-Caucasian. Median days to neutrophil engraftment (absolute neutrophil count ≥ 500/mm3) were 22, 19 and 18 days and the incidence of neutrophil engraftment at 42 days was 89%, 88%, and 90% for adult, pediatric malignant and pediatric non-malignant cohorts respectively. The incidence of acute GVHD Grades II-IV was 27% (95% confidence interval [CI]: 25-29%), 26% (95% CI: 22-29%), and 16% (95% CI:12-19%) for adults, pediatric malignant and pediatric non-malignant cohorts, respectively. The incidence of chronic GVHD was 24% (95% CI: 22-27%), 27% (95% CI: 23-30%) and 25% (95% CI: 21-30%) for adult, pediatric malignant and pediatric non-malignant cohorts, respectively. One year overall survival (OS) was 57% (95% CI: 54-59%), 71% (95% CI: 67-75%), and 79% (95% CI: 75-83%) for adults, pediatric malignant and pediatric non-malignant cohorts, respectively (Figure 3). One year disease-free survival was 57% (95% CI: 54-59%) and 70% (95% CI: 66-74%) for adults and children with malignancy, respectively. OS was similar among different racial/ethnic groups.ConclusionsUse of unlicensed UCB units is safe and effective, and provides an important graft source for a diverse population. 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). 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. 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 (<1 to 81); 50% of children (Figure 1) and 35% of adults (Figure 2) were non-Caucasian. Median days to neutrophil engraftment (absolute neutrophil count ≥ 500/mm3) were 22, 19 and 18 days and the incidence of neutrophil engraftment at 42 days was 89%, 88%, and 90% for adult, pediatric malignant and pediatric non-malignant cohorts respectively. The incidence of acute GVHD Grades II-IV was 27% (95% confidence interval [CI]: 25-29%), 26% (95% CI: 22-29%), and 16% (95% CI:12-19%) for adults, pediatric malignant and pediatric non-malignant cohorts, respectively. The incidence of chronic GVHD was 24% (95% CI: 22-27%), 27% (95% CI: 23-30%) and 25% (95% CI: 21-30%) for adult, pediatric malignant and pediatric non-malignant cohorts, respectively. One year overall survival (OS) was 57% (95% CI: 54-59%), 71% (95% CI: 67-75%), and 79% (95% CI: 75-83%) for adults, pediatric malignant and pediatric non-malignant cohorts, respectively (Figure 3). One year disease-free survival was 57% (95% CI: 54-59%) and 70% (95% CI: 66-74%) for adults and children with malignancy, respectively. OS was similar among different racial/ethnic groups. Use of unlicensed UCB units is safe and effective, and provides an important graft source for a diverse population.