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Successful unrelated mismatched cord blood transplantation in a child with malignant infantile osteopetrosis
Author(s) -
Jaing TangHer,
Hou JiaWoei,
Chen ShihHsiang,
Huang IAnne,
Wang ChaoJan,
Lee WeiI
Publication year - 2006
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/j.1399-3046.2006.00537.x
Subject(s) - medicine , osteopetrosis , busulfan , cord blood , cyclophosphamide , hematopoietic stem cell transplantation , transplantation , surgery , total body irradiation , umbilical cord , haematopoiesis , stem cell , immunology , chemotherapy , biology , genetics
Allogeneic hematopoietic stem cell transplantation represents the only curative option for malignant infantile osteopetrosis (MIOP), a rare disease of infants and young children, characterized by excessive accumulation of mineralized bone and abnormal hematopoiesis. We report a case of successful engraftment and stable full‐donor chimerism in a patient with MIOP who underwent unrelated donor cord blood transplantation (CBT). The donor was 2‐loci human leukocyte antigen (HLA)‐mismatch. After a conditioning regimen based on the combination of busulfan, cyclophosphamide, total body irradiation, and antithymocyte globulin, the patient received a dose of 3.85 × 10 7 /kg of nucleated cells. Neutrophil and platelet engraftment had been achieved by day +33 and +82, respectively, and the patient was discharged home on day +89. A successful engraftment of donor hematopoiesis was demonstrated and the child experienced grade II acute graft‐vs.‐host disease (GVHD) involving the skin only. A remarkable but non‐progressive decrease in lumbar spine bone mineral density was observed in the first nine months post‐transplant. This case suggests that unrelated donor CBT may be a feasible option in case of unavailability of a fully HLA‐matched related or unrelated donor.