Unrelated Umbilical Cord Blood Transplantation for an... : Journal of Pediatric Hematology/Oncology (original) (raw)

Clinical And Laboratory Observations

Unrelated Umbilical Cord Blood Transplantation for an Infant With β-Thalassemia Major

Hall, Jennifer G. MD; Martin, Paul L. MD, PhD; Wood, Susan PNP; Kurtzberg, Joanne MD

From the Pediatric Bone Marrow and Stem Cell Transplant Program, Duke University Medical Center, Durham, North Carolina.

Received for publication December 10, 2003; accepted April 8, 2004.

Reprints: Dr. Jennifer G. Hall, Suite 1400 North Pavilion, Box 3350, 2400 Pratt Street, Duke University Medical Center, Durham, NC 27710 (e-mail: [email protected]).

Abstract

Background:

β-thalassemia major, one of the most prevalent hemoglobinopathies throughout the world, can be cured by allogeneic stem cell transplantation therapy. Many patients, however, lack a suitably matched related donor. Unrelated umbilical cord blood can be used as an alternative stem cell source for some of these patients. This report describes the successful transplantation of a 2-month-old infant with β-thalassemia major using partially HLA-matched unrelated umbilical cord blood.

Methods:

After cytoreduction with busulfan, cyclophosphamide, and antithymocyte globulin (ATG), the patient underwent transplantation at the age of 2 months with a 4/6 HLA matching umbilical cord blood unit from an unrelated donor.

Results:

The patient engrafted promptly with 100% donor chimerism. His only major complication was an autoimmune hemolytic anemia that resolved 2 years after transplantation. He is currently surviving, event-free, 5 years after transplantation with normal growth and cognitive development and full donor chimerism without evidence of β-thalassemia.

Conclusions:

Umbilical cord blood transplantation from related and unrelated donors should be considered for patients with β-thalassemia major who lack traditional bone marrow donors. As most newborns undergo screening for hemoglobinopathies, those with disease could be transplanted early in life before experiencing the morbidity and mortality caused by transfusion therapy, alloimmunization, and iron overload, increasing the likelihood of successful transplantation therapy.