PMID- 16857002 OWN - NLM STAT- MEDLINE DCOM- 20061207 LR - 20060721 IS - 1397-3142 (Print) IS - 1397-3142 (Linking) VI - 10 IP - 5 DP - 2006 Aug TI - Successful unrelated mismatched cord blood transplantation in a child with malignant infantile osteopetrosis. PG - 629-31 AB - 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 x 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. FAU - Jaing, Tang-Her AU - Jaing TH AD - Division of Hematology/Oncology, Department of Pediatrics, Chang Gung University and Children's Hospital, Taoyuan, Taiwan. jaing001@cgmh.org.tw FAU - Hou, Jia-Woei AU - Hou JW FAU - Chen, Shih-Hsiang AU - Chen SH FAU - Huang, I-Anne AU - Huang IA FAU - Wang, Chao-Jan AU - Wang CJ FAU - Lee, Wei-I AU - Lee WI LA - eng PT - Case Reports PT - Journal Article PL - Denmark TA - Pediatr Transplant JT - Pediatric transplantation JID - 9802574 SB - IM MH - *Cord Blood Stem Cell Transplantation MH - Humans MH - Infant MH - Male MH - Osteopetrosis/*therapy MH - Transplantation, Homologous EDAT- 2006/07/22 09:00 MHDA- 2006/12/09 09:00 CRDT- 2006/07/22 09:00 PHST- 2006/07/22 09:00 [pubmed] PHST- 2006/12/09 09:00 [medline] PHST- 2006/07/22 09:00 [entrez] AID - PTR537 [pii] AID - 10.1111/j.1399-3046.2006.00537.x [doi] PST - ppublish SO - Pediatr Transplant. 2006 Aug;10(5):629-31. doi: 10.1111/j.1399-3046.2006.00537.x.