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Oral Abstract

Biotherapies/Cellular Therapies and Immunotherapies - Hematopoietic Cell Therapy/Transplant Immunotherapies (includes CAR T cells)

Oral Abstract Session - Advances in Cord Blood Therapy

#OA2-AM25-MN-18 - Umbilical Cord Blood Transplantation (UCBT) a Very Good Curative Option for Pediatric Patients with Congenital Disorders Even in the Setting of Underrepresented Minorities

Monday, October 27, 2025
10:00 AM - 11:30 AM  PT
Room: SDCC 32
Oral Abstracts

    Presenting Author(s)

  • CM

    Caridad Martinez, MD

    Chief, Cell Therapy and Bone Marrow Transplant Program
    Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital
    Sugar Land, Texas

    Disclosure information not submitted.

Background/Case Studies: Stem cell transplantation (SCT) is the ultimate cure for many congenital pediatric disorders.  An HLA matched related donor is the preferred option but only about 20% of the patients will have this available.  Delaying STC in these patients will increase the risk of infection, organ damage, or progression of their disease.  UCBT is ideal for these patients due to its availability, less strict HLA matching requirements, and reduced risk of viral transmission.  Here we report our single center experience of a uniform UCBT platform without the use of serotherapy.

Study

Design/Methods: We included 81 patients: Inborn Errors of Immunity (IEI) (67), Metabolic (11), and Hematologic disorders (7) receiving UCBT from 2008-2024 with a median age at transplant of 6 months, range: 1 mo -10 yrs. Most of the patients were of underrepresented minorities (74%) with Hispanic being the most common (57%).  Severe Combined Immune Deficiency (SCID) (47) was the most common IEI diagnosis. All patients received a myeloablative conditioning regimen consisting of Busulfan, cyclophosphamide, and fludarabine without serotherapy. Seventy-six patients received a mismatched 5/6 UCB unit.  

Results/Findings: The 2 yr and 5 yr overall survival for the cohort was 88% and 87% respectively with a median follow up of: 8 years, range: 6 mo-16 yrs.  The overall survival for the SCID patients was 91%. All evaluable patients achieved neutrophil and platelet engraftment. Only 1 patient developed secondary graft loss 2 years after UCBT.  Ten patients experienced acute graft-versus-host disease (GvHD), with only two cases classified as severe. Eleven patients developed chronic GvHD, with four cases being extensive.

Conclusions:

Conditioned Umbilical Cord Blood Transplantation (UCBT) without serotherapy is a highly effective treatment for pediatric congenital disorders, even within an underrepresented population. This approach achieves excellent overall survival rates while minimizing the risk of graft failure or GvHD.