131 Successful Autologous Cord Blood Transplantation in a Child with Acquired Severe Aplastic Anemia

Track: Contributed Abstracts
Wednesday, February 13, 2013, 6:45 PM-7:45 PM
Hall 1 (Salt Palace Convention Center)
David Buchbinder, MD , Children's Hospital of Orange County, Orange, CA
Steven M Neudorf, MD , Children's Hospital of Orange County, Orange, CA
Richard Childs, MD , Hematology Branch, National Institutes of Health, Bethesda, MD
Carla Daum, RN, CPON , Children's Hospital of Orange County, Orange, CA
Loan Hsieh, MD , Children's Hospital of Orange County, Orange, CA
Van Huynh, MD , Children's Hospital of Orange County, Orange, CA
Ivan Kirov, MD , Children's Hospital of Orange County, Orange, CA
Edna Klinger, RN, BS , Children's Hospital of Orange County, Orange, CA
Nancy Kuntz, RN, PNP , Children's Hospital of Orange County, Orange, CA
David Margolis, MD , Children's Hospital, Medicial College of Wisconsin, Milwaukee, WI
Theodore B Moore, MD , Pediatrics, David Geffen School of Medicine at UCLA, Los Angeles, CA
Geetha Puthenveetil, MD , Children's Hospital of Orange County, Orange, CA
Elyssa Rubin, MD , Children's Hospital of Orange County, Orange, CA
Leonard Sender, MD , Oncology, UC Irvine Medical Center, Orange, CA
Amit Soni, MD , Children's Hospital of Orange County, Orange, CA
Jill Stites, FNP , Children's Hospital of Orange County, Orange, CA
Lilibeth Torno, MD , Children's Hospital of Orange County, Orange, CA
Diane Jean Nugent, MD , Children's Hospital of Orange County, Orange, CA
Background:

Over 400 cases of pediatric severe aplastic anemia (SAA) occur annually in the United States.  A growing number of children with SAA may have had their stem cells harvested through cord blood collection.  We describe a 9-year-old male with SAA treated successfully with an autologous cord blood transplant following immunoablative chemotherapy. With increasing numbers of cord blood cryopreservations, the use of autologous cord blood in the treatment of SAA might be considered as initial therapy.  

 Methods:

A previously healthy 9-year-old Hispanic male was diagnosed with acquired SAA . Due to ongoing transfusion support, and the lack of a sibling donor, anti-thymocyte globulin and cyclosporine was initiated.   Six months following the start of immunosuppressive therapy, the patient had no marrow recovery.  An unrelated bone marrow donor search revealed one 9 out of 10 matched unrelated bone marrow donor (HLA A antigen mismatch).  One 10 out of 10 matched cord blood unit was available in the public cord blood registry and further inquiry revealed that this donated cord blood unit was an autologous unit.  Given the lack of response to immunosuppressive therapy, the risks associated with the use of a mismatched unrelated donor hematopoietic cell transplantation (HCT), and the availability of the patients own cord blood unit, a decision was made to proceed with autologous cord blood HCT.  

 Results:

The HCT preparative regimen included:  fludarabine 30 mg / m2 / day from Day -6 to Day -4 for a total dose of 90 mg / m2.  Cyclophosphamide 60 mg / kg /day was given from Day -3 to Day -2 for a total dose of 120 mg / kg together with MESNA prophylaxis.  This was followed by a day of rest on Day -1 and an infusion of 3.0 x 10e7 TNC / kg and 0.9 x 10e6 CD34+ / kg using an autologous cord blood unit on Day 0.   His absolute neutrophil count exceeded 500 cell / uL on Day + 21 and has subsequently remained above this threshold without growth factor support.  His platelet count was greater than 20,000 / uL on Day + 36 with his last platelet and packed red blood cell transfusion given on Day +20.  The patient was discharged at Day +27 and remains transfusion independent at the time of his most recent clinical assessment at Day +100.  His Lansky performance status was 100 at discharge and has remained so now at over 3 months following transplant.

Conclusion:

To our knowledge, this is the first description of a successful application of autologous cord blood HCT in a pediatric patient with acquired SAA using an immunoablative preparative regimen consisting of fludarabine and cyclophosphamide.   We found autologous cord blood HCT following an immunoablative combination of fludarabine and cyclophosphamide to be an effective and safe alternative to the potential complications as well as morbidity and mortality associated with the use of matched unrelated donor HCT or the use of other alternative donor HCT approaches.

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