Umbilical cord blood stem cell transplantation from unrelated HLA-matched donor in an infant with severe congenital neutropenia.
We report here a 6-month-old boy with severe congenital neutropenia (SCN) successfully treated by cord blood stem cell transplantation (CBSCT) from an unrelated donor. He had recurrent life-threatening respiratory infection due to severe neutropenia that was refractory to recombinant human granulocyte colony-stimulating factor (rhG-CSF). Because he had no HLA-matched sibling and no time to wait for unrelated donor, he received HLA-matched unrelated CBSCT as determined by DNA typing. A total of 6.4 x 10(7) CB nucleated cells/kg was infused after conditioning with busulfan/horse antihuman thymocyte serum/cyclophosphamide. No GVHD developed under the treatment with cyclosporin A and methyl prednisolone. The neutrophil count reached 0.5 x 10(9)/l on day 14, reticulocyte 1% on day 13 and platelet count over 50 x 10(9)/l on day 31. We conclude that unrelated CBSCT can be an indication for some cases of SCN, who have recurrent life-threatening infections and are refractory to rhG-CSF, and have no HLA-matched sibling.
Department of Pediatrics, Hokkaido University School of Medicine, Sapporo, Japan. emino@med.hokudai.ac.jp
E
E
Mino
R
R
Kobayashi
M
M
Yoshida
Y
Y
Suzuki
M
M
Yamada
K
K
Kobayashi
D002066:Busulfan / Q000494:pharmacology; D036101:Cord Blood Stem Cell Transplantation / Q000379:methods*; D003520:Cyclophosphamide / Q000494:pharmacology; D016572:Cyclosporine / Q000494:pharmacology; D016172:DNA Fingerprinting; D004252:DNA Mutational Analysis; D016179:Granulocyte Colony-Stimulating Factor / Q000627:therapeutic use; D006680:HLA Antigens / Q000737:chemistry*; D006650:Histocompatibility Testing; D006801:Humans; D007223:Infant; D008297:Male; D008775:Methylprednisolone / Q000494:pharmacology; D009503:Neutropenia / Q000628:therapy*; D009504:Neutrophils / Q000378:metabolism; D011994:Recombinant Proteins / Q000627:therapeutic use; D012141:Respiratory Tract Infections / Q000209:etiology; D013997:Time Factors; D014019:Tissue Donors; D014057:Tomography, X-Ray Computed; D019172:Transplantation Conditioning; D014184:Transplantation, Homologous
D006680:HLA Antigens; D011994:Recombinant Proteins; D016179:Granulocyte Colony-Stimulating Factor; D016572:Cyclosporine; D003520:Cyclophosphamide; D002066:Busulfan; D008775:Methylprednisolone