Hematopoietic Stem Cell Transplantation from Sibling or Autologous Cord Blood Units: Report from a Single Cord Blood Unit (CBU) Bank.
Authors: Willert Jennifer , Brown Heather , LaBossierre Denise , Rosenthal Joseph 
Published: October 2019

Umbilical CBU are considered an acceptable alternative source for hematopoietic stem cells transplantation (HSCT) when HLA-identical donors are unavailable. Outcome after transplants with HLA-matched CBU appear to be favorable in comparison to matched unrelated marrow donors (Eapen et al Lancet369: 1947, 2007). This advancement has resulted in the establishment of not-for-profit and for-profit cord blood banks. There is a wide consensus against directed donor (DD) banking of autologous CBUs for several reasons: families may be vulnerable to marketing at the time of birth of a child; the life-time likelihood of using a privately banked CBU is unknown; there are no data regarding safety or effectiveness of DD banked CBU transplantation.

Objective: To report data regarding the potential use, safety and efficacy of DD CBU released for use in HSCT by a single privately owned CBU bank.

Methods: Cord Blood Registry (CBR) collects data regarding CBUs released for HSCT for quality control and for education purposes. Recipient data including, diagnosis, age, weight, HLA match and viability were collected by CBR at the time of release of CBU. Transplant centers reported to CBR engraftment and outcomes data.

Results: From December 1993 to June 2007 CBR has provided DD CBU for 53 transplants at 26 different transplant centers in 14 states. Thirty five CBUs were used in HLA matched sibling HSCT (ALLO) and 18 in autologous HSCT (AUTO). There were 26 ALLO for malignancy (AML n=8; ALL, n=16 and 2 for CML) and 16 ALLO for non-malignant indications (Thalassemia, n=3; sickle cell disease, n=2; severe aplastic anemia (SAA), n=6; Fanconi’s anemia, n=2; and one each for Diamond Blackfan Anemia, Hurler’s syndrome, and Hyper IgM syndrome). The recipient age in ALLO for malignant disorders ranged from 1 year to 16 years with one adult receiving a cord blood unit for CML at the age of 46. For non-malignant indications the age ranged from 4 months to 11 years. AUTO HSCT was performed in 4 patients with SAA (n=4).and for emerging indications (anoxic brain injury, n=7 3 for type I diabetes, n=3; rare immune disorder, n=1). Time of CBU storage ranged from 1 to 114 months; 1 – 47 months and 2–114 months, for ALLO and AUTO, respectively. One of the units was stored for 114 months with engraftment on day +21 and 99% viability. Neutrophil engraftment occurred on average day +18 and +22, for non-malignant and malignant indications, respectively. Post-thaw viability was excellent for all CBU thawed, at approximately 90% or higher. No significant infections were found in any of the CBU tested.

Conclusion: Our data supports the use of DD CBUs for HSCT. Directed donation programs provide access to sibling CBU, which may increase the odds of finding a suitable donor. We are in the process of evaluating overall patient outcomes as well as the incidence of acute and chronic GVHD for all transplants described above. We will analyze the data in order to compare our findings to the outcomes reported for unrelated donor CBU obtained through public CBU banks. The successful outcome in our patients as well as the favorable outcomes reported with use of HSCT from related matched or partially matched CBU should re-open the discussion regarding the value of integrating DD and public banking of CBUs.