Denosumab treatment for fibrous dysplasia

Journal of Bone and Mineral Research

Volumn 27, Issue 7, 2012, Pages 1462-1470

  Boyce, Alison M ^a,b   Chong, William H ^a   Yao, Jack ^c   Gafni, Rachel I ^a   Kelly, Marilyn H ^a   Chamberlain, Christine E ^c   Bassim, Carol ^a   Cherman, Natasha ^a   Ellsworth, Michelle ^d   Kasa Vubu, Josephine Z ^d   Farley, Frances A ^d   Molinolo, Alfredo A ^a   Bhattacharyya, Nisan ^a   Collins, Michael T ^a  

^a NATIONAL INSTITUTE OF DENTAL AND CRANIOFACIAL RESEARCH   (United States)

^b EUNICE KENNEDY SHRIVER NATIONAL INSTITUTE OF CHILD HEALTH AND HUMAN DEVELOPMENT   (United States)

^c NATIONAL INSTITUTES OF HEALTH   (United States)

^d UNIVERSITY OF MICHIGAN HEALTH SYSTEM   (United States)

Author keywords

Hypercalcemia; McCune Albright syndrome; Rank ligand

Indexed keywords

CALCITRIOL; CALCIUM; CARBOXY TERMINAL TELOPEPTIDE; DENOSUMAB; OSTEOCLAST DIFFERENTIATION FACTOR; PHOSPHORUS;

ARTICLE; BONE BIOPSY; BONE PAIN; BONE TURNOVER; CASE REPORT; CHILD; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG WITHDRAWAL; FEMUR FRACTURE; FIBROUS DYSPLASIA; HUMAN; HYPERCALCEMIA; HYPOPHOSPHATEMIA; IMMUNOHISTOCHEMISTRY; MALE; MINERAL METABOLISM; MORBIDITY; PROTEIN EXPRESSION; SCHOOL CHILD; SECONDARY HYPERPARATHYROIDISM; TREATMENT RESPONSE; TUMOR GROWTH;

ANTIBODIES, MONOCLONAL; ANTIBODIES, MONOCLONAL, HUMANIZED; BIOPSY; BONE AND BONES; BONE NEOPLASMS; CELL MEMBRANE; CELL PROLIFERATION; CHILD; FIBROUS DYSPLASIA OF BONE; HUMANS; IMMUNOHISTOCHEMISTRY; MALE; MUTATION; NEOPLASM METASTASIS; OSTEOPOROSIS; RANK LIGAND;

EID: 84861393976     PISSN: 08840431     EISSN: 15234681     Source Type: Journal    
DOI: 10.1002/jbmr.1603     Document Type: Article

References (43)

1. Collins MT, Bianco P. Fibrous dysplasia. In: Favus MJ, editor Primer on the metabolic bone diseases and disorders of mineral metabolism 6th ed. Washington, DC: American Society for Bone and Mineral Research; 2006. p. 415-8.
2. Lichtenstein L. Polyostotic fibrous dysplasia. Arch Surg. 1938;36:874-98.
3. Lichtenstein L, Jaffe HL. Fibrous dysplasia of bone: a condition affecting one, several or many bones, the graver cases of which may present abnormal pigmentation of skin, premature sexual development, hyperthyroidism or still other extraskeletal abnormalities. Arch Pathol. 1942;33:777-816.
4. McCune DJ. Osteitis fibrosa cystica; the case of a nine year old girl who also exhibits precocious puberty, multiple pigmentation of the skin and hyperthyroidism. Am J Dis Child. 1936;52:743-4.
5. Albright F, Butler AM, Hampton AO, Smith PH. Syndrome characterized by osteitis fibrosa disseminata, areas of pigmentation and endocrine dysfunction, with precocious puberty in females, report of five cases. N Engl J Med. 1937;216:727-46.
6. Dumitrescu CE, Collins MT. McCune-Albright syndrome. Orphanet J Rare Dis. 2008;3:12.
7. Collins MT, Chebli C, Jones J, Kushner H, Consugar M, Rinaldo P, Wientroub S, Bianco P, Robey PG. Renal phosphate wasting in fibrous dysplasia of bone is part of a generalized renal tubular dysfunction similar to that seen in tumor-induced osteomalacia. J Bone Miner Res. 2001;16(5):806-13. (Pubitemid 32332290)
8. Riminucci M, Collins MT, Fedarko NS, Cherman N, Corsi A, White KE, Waguespack S, Gupta A, Hannon T, Econs MJ, Bianco P, Gehron Robey P. FGF-23 in fibrous dysplasia of bone and its relationship to renal phosphate wasting. J Clin Invest. 2003;112(5):683-92. (Pubitemid 38057709)
9. Weinstein LS, Shenker A, Gejman PV, Merino MJ, Friedman E, Spiegel AM. Activating mutations of the stimulatory G protein in the McCune-Albright syndrome. N Engl J Med. 1991;325(24):1688-95.
10. Schwindinger WF, Francomano CA, Levine MA. Identification of a mutation in the gene encoding the a subunit of the stimulatory G protein of adenylyl cyclase in McCune-Albright syndrome. Proc Natl Acad Sci USA. 1992;89:5152-6.
11. Happle R. The McCune-Albright syndrome: a lethal gene surviving by mosaicism. Clin Genet. 1986;29:321-4. (Pubitemid 16116576)
12. Riminucci M, Saggio I, Robey PG, Bianco P. Fibrous dysplasia as a stem cell disease. J Bone Miner Res. 2006;21(Suppl 2):P125-31.
13. Riminucci M, Gehron Robey P, Saggio I, Bianco P. Skeletal progenitors and the GNAS gene: fibrous dysplasia of bone read through stem cells. J Mol Endocrinol. 2010 Dec; 45(6):355-64.
14. Riminucci M, Fisher LW, Shenker A, Spiegel AM, Bianco P, Gehron Robey P. Fibrous dysplasia of bone in the McCune-Albright syndrome: abnormalities in bone formation. Am J Pathol. 1997;151(6):1587-600. (Pubitemid 27527922)
15. Riminucci M, Liu B, Corsi A, Shenker A, Spiegel AM, Robey PG, Bianco P. The histopathology of fibrous dysplasia of bone in patients with activating mutations of the Gs alpha gene: site-specific patterns and recurrent histological hallmarks. J Pathol. 1999;187(2):249-58. (Pubitemid 29037056)
16. Bianco P, Kuznetsov SA, Riminucci M, Fisher LW, Spiegel AM, Robey PG. Reproduction of human fibrous dysplasia of bone in immunocompromised mice by transplanted mosaics of normal and Gsalpha-mutated skeletal progenitor cells. J Clin Invest. 1998;101(8):1737-44. (Pubitemid 28198504)
17. Piersanti S, Remoli C, Saggio I, Funari A, Michienzi S, Sacchetti B, Robey PG, Riminucci M, Bianco P. Transfer, analysis, and reversion of the fibrous dysplasia cellular phenotype in human skeletal progenitors. J Bone Miner Res. 2010;25(5):1103-16.
18. Stanton RP. Surgery for fibrous dysplasia. J Bone Miner Res. 2006; 21(Suppl 2):P105-9.
19. Collins MT, Kushner H, Reynolds JC, Chebli C, Kelly MH, Gupta A, Brillante B, Leet AI, Riminucci M, Robey PG, Bianco P, Wientroub S, Chen CC. An instrument to measure skeletal burden and predict functional outcome in fibrous dysplasia of bone. J Bone Miner Res. 2005;20(2):219-26.
20. Parisi MS, Oliveri B, Mautalen CA. Effect of intravenous pamidronate on bone markers and local bone mineral density in fibrous dysplasia. Bone. 2003;33(4):582-8. (Pubitemid 37222384)
21. Plotkin H, Rauch F, Zeitlin L, Munns C, Travers R, Glorieux FH. Effect of pamidronate treatment in children with polyostotic fibrous dysplasia of bone. J Clin Endocrinol Metab. 2003;88(10):4569-75. (Pubitemid 37357495)
22. Fizazi K, Bosserman L, Gao G, Skacel T, Markus R. Denosumab treatment of prostate cancer with bone metastases and increased urine N-telopeptide levels after therapy with intravenous bisphosphonates: results of a randomized phase II trial. J Urol. 2009;182(2):509-15; discussion 515-6.
23. McClung MR, Lewiecki EM, Cohen SB, Bolognese MA, Woodson GC, Moffett AH, Peacock M, Miller PD, Lederman SN, Chesnut CH, Lain D, Kivitz AJ, Holloway DL, Zhang C, Peterson MC, Bekker PJ. Denosumab in postmenopausal women with low bone mineral density. N Engl J Med. 2006;354(8):821-31. (Pubitemid 43290920)
24. Gonzalez-Suarez E, Jacob AP, Jones J, Miller R, Roudier-Meyer MP, Erwert R, Pinkas J, Branstetter D, Dougall WC. RANK ligand mediates progestin-induced mammary epithelial proliferation and carcinogenesis. Nature. 2010;468(7320):103-7.
25. Schramek D, Leibbrandt A, Sigl V, Kenner L, Pospisilik JA, Lee HJ, Hanada R, Joshi PA, Aliprantis A, Glimcher L, Pasparakis M, Khokha R, Ormandy CJ, Widschwendter M, Schett G, Penninger JM. Osteoclast differentiation factor RANKL controls development of progestin-driven mammary cancer. Nature. 2010;468(7320):98-102.
26. Mikami S, Katsube K, Oya M, Ishida M, Kosaka T, Mizuno R, Mochizuki S, Ikeda T, Mukai M, Okada Y. Increased RANKL expression is related to tumour migration and metastasis of renal cell carcinomas. J Pathol. 2009;218(4):530-9.
27. Sasaki A, Ishikawa K, Haraguchi N, Inoue H, Ishio T, Shibata K, Ohta M, Kitano S, Mori M. Receptor activator of nuclear factor-kappaB ligand (RANKL) expression in hepatocellular carcinoma with bone metastasis. Ann Surg Oncol. 2007;14(3):1191-9.
28. Roodman GD, Dougall WC. RANK ligand as a therapeutic target for bone metastases and multiple myeloma. Cancer Treat Rev. 2008;34(1):92-101. (Pubitemid 351181016)
29. Stopeck AT, Lipton A, Body JJ, Steger GG, Tonkin K, de Boer RH, Lichinitser M, Fujiwara Y, Yardley DA, Viniegra M, Fan M, Jiang Q, Dansey R, Jun S, Braun A. Denosumab compared with zoledronic acid for the treatment of bone metastases in patients with advanced breast cancer: a randomized, double-blind study. J Clin Oncol. 2010;28(35):5132-9.
30. Fizazi K, Carducci M, Smith M, Damiao R, Brown J, Karsh L, Milecki P, Shore N, Rader M, Wang H, Jiang Q, Tadros S, Dansey R, Goessl C. Denosumab versus zoledronic acid for treatment of bone metastases in men with castration-resistant prostate cancer: a randomised, double-blind study. Lancet. 2011;377(9768):813-22.
31. Molyneux SD, Di Grappa MA, Beristain AG, McKee TD, Wai DH, Paderova J, Kashyap M, Hu P, Maiuri T, Narala SR, Stambolic V, Squire J, Penninger J, Sanchez O, Triche TJ, Wood GA, Kirschner LS, Khokha R. Prkar1a is an osteosarcoma tumor suppressor that defines a molecular subclass in mice. J Clin Invest. 2010;120(9):3310-25.
32. Thomas D, Henshaw R, Skubitz K, Chawla S, Staddon A, Blay JY, Roudier M, Smith J, Ye Z, Sohn W, Dansey R, Jun S. Denosumab in patients with giant-cell tumour of bone: an open-label, phase 2 study. Lancet Oncol. 2010;11(3):275-80.
33. Reid IR, Miller PD, Brown JP, Kendler DL, Fahrleitner-Pammer A, Valter I, Maasalu K, Bolognese MA, Woodson G, Bone H, Ding B, Wagman RB, San Martin J, Ominsky MS, Dempster DW. Effects of denosumab on bone histomorphometry: the FREEDOM and STAND studies. J Bone Miner Res. 2010;25(10):2256-65.
34. Pelzmann KS, Nagel DZ, Salyer WR. Case report 114. Skel Radiol. 1980;5(2):116-8.
35. Ishida T, Dorfman HD. Massive chondroid differentiation in fibrous dysplasia of bone (fibrocartilaginous dysplasia). Am J Surg Pathol. 1993;17(9):924-30. (Pubitemid 23245926)
36. Kyriakos M, McDonald DJ, Sundaram M. Fibrous dysplasia with cartilaginous differentiation ("fibrocartilaginous dysplasia"): a review, with an illustrative case followed for 18 years. Skel Radiol. 2004;33(1):51-62. (Pubitemid 38111316)
37. Bone HG, Bolognese MA, Yuen CK, Kendler DL, Miller PD, Yang YC, Grazette L, San Martin J, Gallagher JC. Effects of denosumab treatment and discontinuation on bone mineral density and bone turnover markers in postmenopausal women with low bone mass. J Clin Endocrinol Metab. 2011;96(4):972-80.
38. Kong YY, Yoshida H, Sarosi I, Tan HL, Timms E, Capparelli C, Morony S, Oliveira-dos-Santos AJ, Van G, Itie A, Khoo W, Wakeham A, Dunstan CR, Lacey DL, Mak TW, Boyle WJ, Penninger JM. OPGL is a key regulator of osteoclastogenesis, lymphocyte development and lymph-node organogenesis. Nature. 1999;397(6717):315-23.
39. Prolia ™ (package insert), Thousand Oaks, CA. Amgen; 2010.
40. van Persijn van Meerten EL, Kroon HM, Papapoulos SE. Epi- and metaphyseal changes in children caused by administration of bisphosphonates. Radiology. 1992;184(1):249-54.
41. Rauch F, Travers R, Munns C, Glorieux FH. Sclerotic metaphyseal lines in a child treated with pamidronate: histomorphometric analysis. J Bone Miner Res. 2004;19(7):1191-3. (Pubitemid 41110572)
42. Giusti A, Hamdy NA, Papapoulos SE. Atypical fractures of the femur and bisphosphonate therapy: a systematic review of case/case series studies. Bone. 2010;47(2):169-80.
43. Gerstenfeld LC, Sacks DJ, Pelis M, Mason ZD, Graves DT, Barrero M, Ominsky MS, Kostenuik PJ, Morgan EF, Einhorn TA. Comparison of effects of the bisphosphonate alendronate versus the RANKL inhibitor denosumab on murine fracture healing. J Bone Miner Res. 2009;24(2):196-208.