New Bone Formation by Allogeneic Mesenchymal Stem Cell Transplantation in a Patient with Perinatal Hypophosphatasia

Journal of Pediatrics

Volumn 154, Issue 6, 2009, Pages 924-930

  Tadokoro, Mika ^a   Kanai, Rie ^b   Taketani, Takeshi ^b   Uchio, Yuji ^b   Yamaguchi, Seiji ^b   Ohgushi, Hajime ^a  

^a NATIONAL INSTITUTE OF ADVANCED INDUSTRIAL SCIENCE AND TECHNOLOGY AIST   (Japan)

^b SHIMANE UNIVERSITY FACULTY OF MEDICINE   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

ALLOGENEIC STEM CELL TRANSPLANTATION; ARTICLE; BONE DEVELOPMENT; BONE MARROW TRANSPLANTATION; CASE REPORT; CELL CULTURE; CERAMICS; FEMALE; FETUS; HUMAN; HYPOPHOSPHATASIA; MESENCHYMAL STEM CELL; MESENCHYMAL STEM CELL TRANSPLANTATION; NEWBORN; ORGAN DONOR; OSSIFICATION; OSTEOBLAST; PERINATAL MORBIDITY; PRIORITY JOURNAL; RESPIRATORY TRACT DISEASE; SKELETON MALFORMATION;

ALKALINE PHOSPHATASE; BONE DENSITY; CELL DIFFERENTIATION; CELLS, CULTURED; FEMALE; HUMANS; HYPOPHOSPHATASIA; INFANT; INFUSIONS, INTRAOSSEOUS; INFUSIONS, INTRAVENOUS; MESENCHYMAL STEM CELL TRANSPLANTATION; OSTEOBLASTS; OSTEOGENESIS; RESPIRATION, ARTIFICIAL; TISSUE ENGINEERING; TRANSPLANTATION, HOMOLOGOUS;

EID: 65549127189     PISSN: 00223476     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jpeds.2008.12.021     Document Type: Article

References (31)

1. Weiss M.J., Cole D.E., Ray K., Whyte M.P., Lafferty M.A., Mulivor R.A., et al. A missense mutation in the human liver/bone/kidney alkaline phosphatase gene causing a lethal form of hypophosphatasia. Proc Natl Acad Sci USA 85 (1988) 7666-7669
2. Henthorn P.S., Raducha M., Fedde K.N., Lafferty M.A., and Whyte M.P. Different missense mutations at the tissue-nonspecific alkaline phosphatase gene locus in autosomal recessively inherited forms of mild and severe hypophosphatasia. Proc Natl Acad Sci USA 89 (1992) 9924-9928
3. Shohat M., Rimoin D.L., Gruber H.E., and Lachman R.S. Perinatal lethal hypophosphatasia; clinical, radiologic and morphologic findings. Pediatr Radiol 21 (1991) 421-427
4. Whyte M.P., Walkenhorst D.A., Fedde K.N., Henthorn P.S., and Hill C.S. Hypophosphatasia: levels of bone alkaline phosphatase immunoreactivity in serum reflect disease severity. J Clin Endocrinol Metab 81 (1996) 2142-2148
5. Fraser D., and Laidlaw J.C. Treatment of hypophosphatasia with cortisone. Lancet 1 (1956) 553
6. Whyte M.P., McAlister W.H., Patton L.S., Magill H.L., Fallon M.D., Lorentz Jr. W.B., and Herrod H.G. Enzyme replacement therapy for infantile hypophosphatasia attempted by intravenous infusions of alkaline phosphatase-rich Paget plasma: results in three additional patients. J Pediatr 105 (1984) 926-933
7. Barcia J.P., Strife C.F., and Langman C.B. Infantile hypophosphatasia: Treatment options to control hypercalcemia, hypercalciuria, and chronic bone demineralization. J Pediatr 130 (1997) 825-828
8. Deeb A.A., Bruce S.N., Morris A.A., and Cheetham T.D. Infantile hypophosphatasia: Disappointing results of treatment. Acta Paediatr 89 (2000) 730-733
9. Millán J.L., Narisawa S., Lemire I., Loisel T.P., Boileau G., Leonard P., et al. Enzyme replacement therapy for murine hypophosphatasia. J Bone Miner Res 23 (2008) 777-787
10. Horwitz E.M., Prockop D.J., Fitzpatrick L.A., Koo W.W., Gordon P.L., Neel M., et al. Transplantability and therapeutic effects of bone marrow-derived mesenchymal cells in children with osteogenesis imperfecta. Nat Med 5 (1999) 309-313
11. Horwitz E.M., Gordon P.L., Koo W.K., Marx J.C., Neel M.D., McNall R.Y., et al. Isolated allogeneic bone marrow-derived mesenchymal cells engraft and stimulate growth in children with osteogenesis imperfecta: Implications for cell therapy of bone. Proc Natl Acad Sci USA 99 (2002) 8932-8937
12. Whyte M.P., Kurtzberg J., McAlister W.H., Mumm S., Podgornik M.N., Coburn S.P., et al. Marrow cell transplantation for infantile hypophosphatasia. J Bone Miner Res 18 (2003) 624-636
13. Cahill R.A., Jones O.Y., Klemperer M., Steele A., Mueller T.O., el-Badri N., et al. Replacement of recipient stromal/mesenchymal cells after bone marrow transplantation using bone fragments and cultured osteoblast-like cells. Biol Blood Marrow Transplant 10 (2004) 709-717
14. Cahill R.A., Wenkert D., Perlman S.A., Steele A., Coburn S.P., McAlister W.H., et al. Infantile hypophosphatasia: transplantation therapy trial using bone fragments and cultured osteoblasts. J Clin Endocrinol Metab 92 (2007) 2923-2930
15. Caplan A.I. Mesenchymal stem cells. J Orthop Res 9 (1991) 641-650
16. Yoshikawa T., Ohgushi H., Ichijima K., and Takakura Y. Bone regeneration by grafting of cultured human bone. Tissue Eng 10 (2004) 688-698
17. Ohgushi H., Kotobuki N., Funaoka H., Machida H., Hirose M., Tanaka Y., et al. Tissue engineered ceramic artificial joint ex-vivo osteogenic differentiation of patient mesenchymal cells on total ankle joints for treatment of osteoarthritis. Biomaterials 26 (2005) 4654-4661
18. Ohgushi H., and Caplan A.I. Stem cell technology and bioceramics: from cell to gene engineering. J Biomed Mater Res 48 (1999) 913-927
19. Nasu M., Ito M., Ishida Y., Numa N., Komaru K., Nomura S., et al. Aberrant interchain disulfide bridge of tissue-nonspecific alkaline phosphatase with an Arg433>Cys substitution associated with severe hypophosphatasia. FEBS J 273 (2006) 5612-5624
20. Sawai H., Kanazawa N., Tsukahara Y., Koike K., Udagawa H., Koyama K., et al. Severe perinatal hypophosphatasia due to homozygous deletion of T at nucleotide 1559 in the tissue nonspecific alkaline phosphatase gene. Prenat Diagn 23 (2003) 743-746
21. Mornet E. Hypophosphatasia. Orphanet J Rare Dis 4 (2007) 2 :40. Review
22. Aslan H., Zilberman Y., Kandel L., Liebergall M., Oskouian R.J., Gazit D., et al. Osteogenic differentiation of noncultured immunoisolated bone marrow-derived CD105+ cells. Stem Cells 24 (2006) 1728-1737
23. Ohgushi H., Kitamura S., Kotobuki N., Hirose M., Machida H., Muraki K., et al. Clinical application of marrow mesenchymal stem cells for hard tissue repair. Yonsei Med J 30 (2004) 61-67
24. Neumann F., Graef T., Tapprich C., Vaupel M., Steidl U., Germing U., et al. Cyclosporine A and mycophenolate mofetil vs. cyclosporine A and methotrexate for graft-versus-host disease prophylaxis after stem cell transplantation from HLA-identical siblings. Bone Marrow Transplant 35 (2005) 1089-1093
25. Nakahori Y., Mitani K., Yamada M., and Nakagome Y. A human Y-chromosome specific repeated DNA family (DYZ1) consists of a tandem array of pentanucleotides. 1986. Nucleic Acids Res 10 (1986) 7569-7580
26. Rutherford R.B., Moalli M., Franceschi R.T., Wang D., Gu K., and Krebsbach P.H. Bone morphogenetic protein-transduced human fibroblasts convert to osteoblasts and form bone in vivo. Tissue Eng 8 (2002) 441-452
27. Okumura M., Ohgushi H., Tamai S., and Shors E.C. Primary bone formation in porous hydroxyapatite ceramic: A light and scanning electron microscopic study. Cell and Materials 1 (1991) 29-34
28. Goshima J., Goldberg V.M., and Caplan A.I. The origin of bone formed in composite grafts of porous calcium phosphate ceramic loaded with marrow cells. Clin Orthop Relat Res 269 (1991) 274-283
29. Dominici M., Marino R., Rasini V., Spano C., Paolucci P., Conte P., et al. Donor cell-derived osteopoiesis originates from a self-renewing stem cell with a limited regenerative contribution after transplantation. Blood 111 (2008) 4386-4391
30. Khosla S., and Eghbali-Fatourechi G.Z. Circulating cells with osteogenic potential. Ann N Y Acad Sci 1068 (2006) 489-497
31. Nauta A.J., and Fibbe W.E. Immunomodulatory properties of mesenchymal stromal cells. Blood 110 (2007) 3499-3506