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Umbilical Cord Blood Transplantation Can be an Effective Therapy for Patients with Primary Immunodeficiency Diseases Who Lack an HLA-Matched Related Donor

Track: Poster Abstracts
Wednesday, February 26, 2014, 6:45 PM-7:45 PM
Longhorn Hall E (Exhibit Level 1) (Gaylord Texan)
Caridad Martinez, MD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
William Shearer, MD, PhD , Immunology, Allergy and Rheumatology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Jordan Orange, MD, PhD , Immunology, Allergy and Rheumatology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Howard Rosenblatt, MD , Department of Allergy/Immunology, Baylor College of Medicine, Houston, TX
Kathryn Leung, MD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Swati Naik, MD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Stephen Gottschalk, MD , Center for Cell and Gene Therapy, Dept. of Pediatrics, Baylor College of Medicine, Houston Methodist Hospital and Texas Children's Hospital, Houston, TX
Carl Allen, MD, PhD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Nabil Ahmed, MD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Ghadir Sasa, MD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Malcolm K. Brenner, MD, PhD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, The Methodist Hospital, Houston, TX
Helen E. Heslop, MD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, The Methodist Hospital, Houston, TX
Ann M. Leen, PhD , Center for Cell and Gene Therapy, Dept. of Pediatrics, Baylor College of Medicine, Houston Methodist Hospital and Texas Children's Hospital, Houston, TX
Imelda C Hanson, MD , Immunology, Allergy and Rheumatology, Baylor College of Medicine, Texas Children's Hospital, Houston, TX
Robert A. Krance, MD , Center for Cell and Gene Therapy, Baylor College of Medicine, Texas Children's Hospital, The Methodist Hospital, Houston, TX
Most patients with primary immune deficiency diseases (PIDDs) will require a stem cell transplant to cure their disease. There is, however, no consensus about the best alternative hemopoietic stem cell donor in the absence of an HLA-identical source. In particular, there are limited data comparing the outcomes between PIDD patients receiving umbilical cord blood transplant (UCBT) versus unrelated matched stem cell products. Fourteen children, median age 8 months (range, 12 – 20 months) with SCID (n=11), LAD (n=1), reticular dysgenesis (n=1) or Wiskott-Aldrich syndrome (n=1) received UCBT transplant at our institution between 2007 and 2013. Five of 14 patients had persistent viral infections prior to transplant: RSV (n=2), Parainfluenza 3 (n=1), VZV (n=1) and Norovirus (n=1), and 7/14 patients had required mechanical ventilation prior to transplantation. Seven of 14 patients were 6/6 HLA antigen matched with their stem cell donor, and 7 were one HLA antigen mismatched. All patients received myeloablative conditioning consisting of busulfan, cyclophosphamide and fludarabine and one patient also received campath. The median total nucleated cell dose in the transplant was 10x107/kg (range, 5.9 – 25.4). The median time to neutrophil recovery was 19 days (range, 11-30) and the median time to platelet recovery was 42 days (range, 27-127). All evaluable patients achieved full donor chimerism (defined as >95% donor cells in peripheral blood by day +42). No severe aGVHD or cGvHD has occurred and only one patient developed grade II aGvHD (skin). All patients are alive with a median follow up of 2 years (range, 10 days – 6 years).  All patients with viral infections at the time of transplant were able to clear the infection at a median time of 51 days (range, 44-54) and none developed significant new viral infections after transplant. ELISPOT analyses of peripheral blood at the time that infections were cleared showed T cell responses against the pertinent viruses. All evaluable patients have normalization of their immune defect including adequate B cell function in SCID patients. Hence, we have shown that UCBT for PIDDs produces speedy functional engraftment and immune-reconstitution in the absence of significant GvHD, leading to excellent overall survival. UCBT therefore seems an appropriate therapeutic alternative for patients with PIDDs who lack an HLA matched related donor.
Disclosures:
Nothing To Disclose
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