Allogeneic bone marrow transplantation is limited by the availability of suitable marrow donors and risk of graft-versus-host disease (GVHD) and opportunistic infection. In an attempt to ameliorate these limitations, umbilical cord blood has been postulated as an alternative source of allogeneic haemopoietic stem cells for transplantation. From September, 1994, umbilical cord blood from sibling donors has been used to reconstitute haemapoiesis in 44 children with acquired or congenital lympho-haemapoietic disorders, neuroblastoma, or metabolic diseases. Patients who had HLA-identical and HLA-1 antigen disparate grafts, had a probability of engraftment at 50 days after transplantation of 85%. No patient had late graft failure. The probability of grade II-IV GVHD at 100 days was 3% and the probability of chronic GVHD at one year was 6%. With a median follow-up of 1·6 years, the probability of survival for recipients of HLA-identical or HLA-1 antigen disparate grafts is 72%. We conclude that umbilical cord blood is a sufficient source of transplantable haemopoietic stem cells for children with HLA-identical or HLA-1 antigen disparate sibling donors with very low risk of acute or extensive chronic GVHD. The feasibility of umbilical-cord-blood transplantation with HLA-2 and HLA-3 antigen disparate sibling donors remains to be determined.
Bibliographical noteFunding Information:
R Pahwa, Schneider Children’s Hospital, New Hyde Park, New York, USA (n=l); N Bunin, Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania, USA (n= 1); S Neudorf, Children’s Hospital of Pittsburgh, Pittsburgh, Pennsylvania, USA (n= 1); A Nagler, Hadassah University, Jerusalem, Israel (n=l); and F Falkenberg, University Medical Center, Leiden, The Netherlands (n= 1). The authors gratefully acknowledge Todd DeFor, for his diligence and careful analysis of the data, and Judith Bard for her critical review of the manuscript. The study was supported by grants from the Childrens Cancer Research Fund (JEW, MS), National Institutes of Health Grant No POI-CA21737 (MS) and American Cancer Society Career Development Award No 91-56 (JEW) ; National Institutes of Health Grant Cytokine No PO1-AI32918 (NAK) and Leukemia No P01-CA23766 (NAK) and Zelda Weintraub Cancer Foundation (NAK); National Institutes of Health Grant No ROI-HL46549 (HEB).