Outcomes for Umbilical Cord Blood Transplantation in Severe Combined Immunodeficiency Disorders: Ten-Year Experience
Monday, March 7, 2016: 2:45 PM
Room 408A (Convention Center)
Carrie N. Caruthers, MD, , , ,
Rationale: Severe combined immune deficiency (SCID) is a lethal disorder of infants. Patients who undergo genotypic HLA-matched bone marrow transplantation have improved survival. Some patients are unable to find a suitable donor and may undergo umbilical cord blood transplantation (UCBT).

Methods: A retrospective chart review was performed on the 10 patients with SCID who underwent UCBT between 1996-2005 at Cardinal Glennon Children's Medical Center at Saint Louis University. We analyzed patient survival as well as T-cell, B-cell, NK-cell subsets and function, and infections prior to and post- transplant at months 3, 6, 12, 24, 60 and 120.

Results: Eight of ten patients were alive 10 years post-transplantation. One patient died 19 days after transplant from cardiac arrhythmia and presumed cardiomyopathy. The other patient died 32 days after transplant from graft versus host disease of the gut and adenovirus sepsis. Immune studies for the cohort prior to UCBT had mean Absolute Lymphocyte Count (ALC) of 1634/mm3; CD3+ 15.3%, 784 cells/mm3; CD4+ 12%, 497 cells/mm3; CD8+ 8.7%, 300 cells/mm3; CD20+ 43.2%, 515 cells/mm3 and CD56+ 19.6%, 239 cells/mm3.  Immune studies 10 years after UCBT had mean ALC of 2603/mm3; CD3+70.3%, 1797 cells/mm3; CD4+ 41%, 1042 cells/mm3; CD8+ 27.6%, 718 cells/mm3; CD20+ 11.5%, 383 cells/mm3 and CD56+13.9%, 364 cells/mm3.  Immunoglobulins and responses to vaccinations were normal except Streptococcus pneumoniae(51.4% protective). One patient was on IVIG treatment at 120 months.

Conclusions: Patients with SCID who do not have a suitable bone marrow donor and undergo unrelated UCBT have good and sustained immune reconstitution.