Engraftment and survival following hematopoietic stem cell transplantation for osteopetrosis using a reduced intensity conditioning regimen

Bone Marrow Transplantation

Volumn 38, Issue 12, 2006, Pages 783-787

  Tolar, J ^a   Bonfim, C ^b   Grewal, S ^a   Orchard, P ^a  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b FEDERAL UNIVERSITY OF PARANÁ   (Brazil)

Author keywords

[No Author keywords available]

Indexed keywords

BUSULFAN; CORTICOSTEROID; CYCLOPHOSPHAMIDE; CYCLOSPORIN; FLUDARABINE; MYCOPHENOLIC ACID 2 MORPHOLINOETHYL ESTER; THYMOCYTE ANTIBODY;

ADULT; ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION; ARTICLE; AUTOIMMUNE HEMOLYTIC ANEMIA; AUTOSOMAL RECESSIVE DISORDER; BACTEREMIA; BLOOD DONOR; CHIMERA; CLINICAL ARTICLE; CONTROLLED STUDY; CORD BLOOD STEM CELL TRANSPLANTATION; DRUG DOSE INCREASE; GRAFT FAILURE; GRAFT RECIPIENT; GRAFT SURVIVAL; GRAFT VERSUS HOST REACTION; HUMAN; INFANT; LYMPHOPROLIFERATIVE DISEASE; MORTALITY; OSTEOPOROSIS; OUTCOME ASSESSMENT; PERIPHERAL BLOOD STEM CELL TRANSPLANTATION; PRESCHOOL CHILD; PRIORITY JOURNAL; REDUCED INTENSITY CONDITIONING; SINGLE DRUG DOSE; SURVIVAL RATE; SURVIVAL TIME; UMBILICAL CORD BLOOD;

CHILD, PRESCHOOL; FEMALE; FOLLOW-UP STUDIES; GENETIC DISEASES, INBORN; GRAFT SURVIVAL; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; INFANT; MALE; OSTEOPETROSIS; TRANSPLANTATION CONDITIONING;

EID: 33751535183     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/sj.bmt.1705533     Document Type: Article

References (35)

1. Teitelbaum SL, Coccia PF, Brown DM, Kahn AJ. Malignant osteopetrosis: A disease of abnormal osteoclast proliferation. Metab Bone Dis Relat Res 1981; 3: 99-105.
2. Tolar J, Teitelbaum SL, Orchard PJ. Osteopetrosis. N Engl J Med 2004; 351: 2839-2849.
3. Sobacchi C, Frattini A, Orchard P, Porras O, Tezcan I, Andolina M et al. The mutational spectrum of human malignant autosomal recessive osteopetrosis. Hum Mol Genet 2001; 10: 1767-1773.
4. Kornak U, Kasper D, Bosl MR, Kaiser E, Schweizer M, Schulz A et al. Loss of the ClC-7 chloride channel leads to osteopetrosis in mice and man. Cell 2001; 104: 205-215.
5. Kornak U, Schulz A, Friedrich W, Uhlhaas S, Kremens B, Voit T et al. Mutations in the a3 subunit of the vacuolar H(+)-ATPase cause infantile malignant osteopetrosis. Hum Mol Genet 2000; 9: 2059-2063.
6. Chalhoub N, Benachenhou N, Rajapurohitam V, Pata M, Ferron M, Frattini A et al. Grey-lethal mutation induces severe malignant autosomal recessive osteopetrosis in mouse and human. Nat Med 2003; 9: 399-406.
7. Sly WS, Hewett-Emmett D, Whyte MP, Yu YS, Tashian RE et al. Carbonic anhydrase II deficiency identified as the primary defect in the autosomal recessive syndrome of osteopetrosis with renal tubular acidosis and cerebral calcification. Proc Natl Acad Sci USA 1983; 80: 2752-2756.
8. Teitelbaum SL. Bone resorption by osteoclasts. Science 2000; 289: 1504-1508.
9. Coccia PF. Cells that resorb bone. N Engl J Med 1984; 310: 456-458.
10. Gerritsen EJ, Vossen JM, van Loo IH, Hermans J, Helfrich MH, Griscelli C et al. Autosomal recessive osteopetrosis: Variability of findings at diagnosis and during the natural course. Pediatrics 1994; 93: 247-253.
11. Loria-Cortes R, Quesada-Calvo E, Cordero-Chaverri C. Osteopetrosis in children: A report of 26 cases. J Pediatr 1977; 91: 43-47.
12. Dorantes LM, Mejia AM, Dorantes S. Juvenile osteopetrosis: Effects on blood and bone of prednisone and a low calcium, high phosphate diet. Arch Dis Child 1986; 61: 666-670.
13. Glorieux FH, Pettifor JM, Marie PJ, Delvin EE, Travers R, Shepard N. Induction of bone resorption by parathyroid hormone in congenital malignant osteopetrosis. Metab Bone Dis Relat Res 1981; 3: 143-150.
14. Key L, Carnes D, Cole S, Holtrop M, Bar-Shavit Z, Shapiro F et al. Treatment of congenital osteopetrosis with high-dose calcitriol. N Engl J Med 1984; 310: 409-415.
15. Key Jr LL, Ries WL, Rodriguiz RM, Hatcher HC. Recombinant human interferon gamma therapy for osteopetrosis. J Pediatr 1992; 121: 119-124.
16. Coccia PF, Krivit W, Cervenka J, Clawson C, Kersey JH, Kim TH et al. Successful bone-marrow transplantation for infantile malignant osteopetrosis. N Engl J Med 1980; 302: 701-708.
17. Eapen M, Davies SM, Ramsay NK, Orchard PJ. Hematopoietic stem cell transplantation for infantile osteopetrosis. Bone Marrow Transplant 1998; 22: 941-946.
18. Coccia PF. Hematopoietic cell transplantation for osteopetrosis. In: Blume KG, Forman SJ, Appelbaum FR (eds). Thomas' Hematopoietic Cell Transplantation, 3rd edn. Blackwell Publishing Ltd: Malden, MA, 2004, pp 1443-1454.
19. Gerritsen EJ, Vossen JM, Fasth A, Friedrich W, Morgan G, Padmos A et al. Bone marrow transplantation for autosomal recessive osteopetrosis. A report from the Working Party on Inborn Errors of the European Bone Marrow Transplantation Group. J Pediatr 1994; 125 (6 Part 1): 896-902.
20. Driessen GJ, Gerritsen EJ, Fischer A, Fasth A, Hop WC, Veys P et al. Long-term outcome of haematopoietic stem cell transplantation in autosomal recessive osteopetrosis: An EBMT report. Bone Marrow Transplant 2003; 32: 657-663.
21. Laughlin MJ, Eapen M, Rubinstein P, Wagner JE, Zhang MJ, Champlin RE et al. Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukemia. N Engl J Med 2004; 351: 2265-2275.
22. Solh H, Da Cunha AM, Giri N, Padmos A, Spence D, Clink H et al. Bone marrow transplantation for infantile malignant osteopetrosis. J Pediatr Hematol Oncol 1995; 17: 350-355
23. Steward CG, Pellier I, Mahajan A, Ashworth MT, Stuart AG, Fasth A et al. Severe pulmonary hypertension: A frequent complication of stem cell transplantation for malignant infantile osteopetrosis. Br J Haematol 2004; 124: 63-71.
24. Scharf SJ, Smith AG, Hansen JA, McFarland C, Erlich HA. Quantitative determination of bone marrow transplant engraftment using fluorescent polymerase chain reaction primers for human identity markers. Blood 1995; 85: 1954-1963.
25. Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, McGlave PB, Miller JS et al. Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood 2005; 105: 1343-1347.
26. Locatelli F, Beluffi G, Giorgiani G, Maccario R, Fiori P, Pession A et al. Transplantation of cord blood progenitor cells can promote bone resorption in autosomal recessive osteopetrosis. Bone Marrow Transplant 1997; 20: 701-705.
27. Jaing TH, Hou JW, Chen SH, Huang IA, Wang CJ, Lee WI. Successful unrelated mismatched cord blood transplantation in a child with malignant infantile osteopetrosis. Pediatr Transplant 2006; 10: 629-631.
28. Tsuji Y, Ito S, Isoda T, Kajiwara M, Nagasawa M, Morio T et al. Successful nonmyeloablative cord blood transplantation for an infant with malignant infantile osteopetrosis. J Pediatr Hematol Oncol 2005; 27: 495-498.
29. Nicholls BM, Bredius RG, Hamdy NA, Gerritsen EJ, Lankester AC, Hogendoorn PC et al. Limited rescue of osteoclast-poor osteopetrosis after successful engraftment by cord blood from an unrelated donor. J Bone Miner Res 2005; 20: 2264-2270.
30. Pozzi S, Lisini D, Podesta M, Bernardo ME, Sessarego N, Piaggio G et al. Donor multipotent mesenchymal stromal cells may engraft in pediatric patients given either cord blood or bone marrow transplantation. Exp Hematol 2006; 34: 934-942.
31. Wagner JE, Barker JN, DeFor TE, Baker KS, Blazar BR, Eide C et al. Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: Influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood 2002; 100: 1611-1618.
32. Wagner W, Wein F, Seckinger A, Frankhauser M, Wirkner U, Krause U et al. Comparative characteristics of mesenchymal stem cells from human bone marrow, adipose tissue, and umbilical cord blood. Exp Hematol 2005; 33: 1402-1416.
33. Erices A, Conget P, Minguell JJ. Mesenchymal progenitor cells in human umbilical cord blood. Br J Haematol 2000; 109: 235-242.
34. Bieback K, Kern S, Kluter H, Eichler H. Critical parameters for the isolation of mesenchymal stem cells from umbilical cord blood. Stem Cells 2004; 22: 625-634.
35. Kern S, Eichler H, Stoeve J, Kluter H, Bieback K. Comparative analysis of mesenchymal stem cells from bone marrow, umbilical cord blood, or adipose tissue. Stem Cells 2006; 24: 1294-1301.