Медицинские аспекты современного состояния практики лечения прогрессирующей оссифицирующей фибродисплазии



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F. Результаты лабораторных исследований ПОФ

Kaplan FS, Sawyer J, Connors S, Keough K, Shore E, Gannon F, Glaser D, Rocke D, Zasloff M, Folkman J. Urinary basic fibroblast growth factor: a biochemical marker for preosseous fibroproliferative lesions in patients with ПОФ. Clin Orthop 346: 59-65, 1998

Lutwak, L. Myositis ossificans progressiva: mineral, metabolic & radioactive calcium studies of the effects of the hormones. Am J Med 37: 269-293, 1964

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G. Иммунная система и ПОФ

Kaplan FS, Glaser DL, Shore EM, Pignolo RJ, Xu M, Zhang Y, Senitzer D, Forman SJ, Emerson SG. Hematopoietic stem-cell contribution to ectopic skeletogenesis. J Bone Joint Surg Am 89: 347-357, 2007

Kaplan FS, Shore EM, Gupta R, Billings PC, Glaser DL, Pignolo RJ, Graf D, Kamoun M. Immunological features of fibrodysplasia ossificans progressiva and the dysregulated BMP4 Pathway. Clin Rev Bone Miner Metab 3: 189-193, 2005

Lanchoney TF, Cohen RB, Rocke DM, Zasloff MA, Kaplan FS. Permanent heterotopic ossification at the injection site after diphtheria-tetanus-pertussis immunizations in children who have fibrodysplasia ossificans progressiva. J Pediatrics 126: 762-764, 1995

H. Ошибки при диагностике ПОФ

Kitterman JA, Kantanie S, Rocke DM, Kaplan FS. Iatrogenic harm caused by diagnostic errors in fibrodysplasia ossificans progressiva. Pediatrics 116: 654-661, 2005

Zaghloul KA, Heuer GG, Guttenberg MD, Shore EM, Kaplan FS, Storm PB. Lumbar puncture and surgical intervention in a child with undiagnosed fibrodysplasia ossificans progressiva. J Neursurg Pediatrics 1: 91-94, 2008

I. Эпидемиологические, генетические и сопутствующие факторы ПОФ

Hebela N, Shore EM, Kaplan FS. Three pairs of monozygotic twins with fibrodysplasia ossificans progressiva: the role of environment in the progression of heterotopic ossification. Clin Rev Bone Miner Metab 3: 205-208, 2005

Janoff HB, Muenke M, Johnson LO, Rosenberg A, Shore EM, Okereke E, Zasloff M, Kaplan FS. Fibrodysplasia ossificans progressiva in two half-sisters: evidence for maternal mosaicism. Am J Med Genetics 61: 320-324, 1996

Janoff HB, Tabas JA, Shore EM, Muenke M, Dalinka MK, Schlesinger S, Zasloff MA, Kaplan FS. Mild expression of fibrodysplasia ossificans progressiva. J Rheumatol 22: 976-978, 1995

Kaplan FS, McCluskey W, Hahn G, Tabas J, Muenke M, Zasloff MA. Genetic transmission of fibrodysplasia ossificans progressiva. J Bone Joint Surg Am 75: 1214-1220, 1993

Rogers JG, Chase GA. Paternal age effect in fibrodysplasia ossificans progressiva. J Med Genet 16: 147-148, 1979

Shore EM, Feldman GJ, Xu M, Kaplan FS. The genetics of fibrodysplasia ossificans progressiva. Clin Rev Bone Miner Metab 3: 201-204, 2005

Virdi AS, Shore EM, Oreffo RO, Li M, Connor JM, Smith R, Kaplan FS, Triffitt, JT. Phenotypic and molecular heterogeneity in fibrodysplasia ossificans progressiva. Calcified Tissue Int 65: 250-255, 1999

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J. ПОФ и сигнальные пути КМГП

Ahn J, Serrano de la Peña L, Shore EM, Kaplan FS. aresis of a bone morphogenetic protein – antagonist response in a genetic disorder of heterotopic skeletogenesis. J Bone Joint Surg Am 85: 667-674, 2003

Billings PC, Fiori JL, Bentwood JL, O’Connell MP, Jiao X, Nussbaum B, Caron RJ, Shore EM, Kaplan FS. Dysregulated КМГП signaling and enhanced osteogenic differentiation of connective tissue progenitor cells from patients with fibrodysplasia ossificans progressiva. J Bone Miner Res 23: 305-313, 2008

Fiori JL, Billings PC, Serrano de la Peña L, Kaplan FS, Shore EM. Dysregulation of the КМГП-p38 MAPK signaling pathway in cells from patients with fibrodysplasia ossificans progressiva. J Bone Miner Res 21: 902-909, 2006

Fukada T, Kohda M, Kanomata K, Nojima J, Kamizono J, Oda H, Nakayama K, Ohtake A, Miyazono K, Jimi E, Owan I, Okazaki Y, Katagiri T. A constitutively activated КМГП receptor, ALK2, induces heterotopic bone formation in patients with fibrodysplasia ossificans progressiva. J Bone Miner Res 22 (suppl. 1); S10, 2007

Gannon FH, Kaplan FS, Olmsted E, Finkel G, Zasloff MA, Shore EM. Bone morphogenetic protein 2/4 in early fibromatous lesions of fibrodysplasia ossificans progressiva. Hum Pathol 28: 339-343, 1997

Glaser DL, Economides AN, Wang L, Liu X, Kimble RD, Fandl JP, Wilson JM, Stahl, N, Kaplan FS, Shore EM. In vivo somatic cell gene transfer of an engineered noggin mutein prevents BMP4-induced heterotopic ossification. J Bone Joint Surg Am 12: 2332-2342, 2003

Hegyi L, Gannon FH, Glaser DL, Shore EM, Kaplan FS, Shanahan CM. Stromal cells of fibrodysplasia ossificans progressiva lesions express smooth muscle lineage markers and the osteogenic transcription factor Runx2/Cbfa-1: Clues to a vascular origin of heterotopic ossification? J Pathol 201: 141-148, 2003

Kaplan FS, Groppe J, Pignolo RJ, Shore EM. Morphogen receptor genes and metamorphogenes: skeleton keys to metamorphosis. Ann NY Acad Sci 1116: 113-133, 2007

Kaplan FS, Tabas JA, Zasloff MA. Fibrodysplasia ossificans progressiva: A clue from the fly? Calcif Tiss Int 47: 117-125, 1990

O’Connell MP, Billings PC, Fiori JL, Deirmengian G, Roach HI, Shore EM, Kaplan FS. HSPG modulation of КМГП signaling in fibrodysplasia ossificans progressiva cells. J Cell Biochem 102: 1493-1503, 2007

Roush W. Protein builds second skeleton. Science 273: 1170, 1996

Serrano de la Peña L, Billings PC, Fiori JL, Ahn J, Shore EM, Kaplan FS. Fibrodysplasia ossificans progressiva (ПОФ), a disorder of ectopic osteogenesis, misregulates cell surface expression and trafficking of BMPRIA. J Bone Miner Res 20: 1168-1176, 2005

Shafritz AB, Shore EM, Gannon FH, Zasloff MA, Taub R, Muenke M, Kaplan FS. Overexpression of an osteogenic morphogen in fibrodysplasia ossificans progressiva (ПОФ). N Engl J Med 335: 555-561, 1996

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Shen Q, Xu M, Little SC, Kaplan FS, Mullins MC, Shore EM. Activation of КМГП signaling by the ПОФ ACVR1 R206H mutation. J Bone Miner Res 22 (suppl. 1); S43, 2007



Shore EM, Xu M, Feldman GJ, Fenstermacher DA, Cho T-J, Choi IH, Connor JM, Delai P, Glaser DL, Le Merrer M, Morhart R, Rogers JG, Smith R, Triffitt JT, Urtizberea JA, Zasloff M, Brown MA, Kaplan FS. A recurrent mutation in the КМГП type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva. Nature Genetics 38: 525-527, 2006

K. Ген ПОФ

Couzin J. Bone disease gene finally found. Science 312: 514-515, 2006

Furuya H, Ikezoe K, Wang L, Ohyagi Y, Motomura K, Fujii N, Kira J, Fukumaki Y. A unique case of fibrodysplasia ossificans progressiva with an ACVR1 mutation, G356D, other than the common mutation (R206H). Am J Med Genet A 146: 459-463, 2008

Kaplan FS. The key to the closet is the key to the kingdom: a common lesson of rare diseases. Orphan Disease Update 24(3): 1-9, 2006

Kaplan FS, Groppe J, Pignolo RJ, Shore EM. Morphogen receptor genes and metamorphogenes: skeleton keys to metamorphosis. Ann NY Acad Sci 1116: 113-133, 2007

Kaplan FS, Xu M, Seemann P, Connor M, Glaser DL, Carroll L, Delai, P, Fastnact-Urban E, Forman SJ, Gillessen-Kaesbach G, Hoover-Fong J, Köster B, Morhart R, Pauli RM, Reardon W, Zaidi S-A, Zasloff M, Mundlos S, Groppe J, Shore EM. Classical and atypical ПОФ phenotypes caused by mutations in the КМГП type I receptor ACVR1. Human Mutation. In press, 2008

Shore EM, Xu M, Feldman GJ, Fenstermacher DA, Cho T-J, Choi IH, Connor JM, Delai P, Glaser DL, Le Merrer M, Morhart R, Rogers JG, Smith R, Triffitt JT, Urtizberea JA, Zasloff M, Brown MA, Kaplan FS. A recurrent mutation in the КМГП type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva. Nature Genetics 38: 525-527, 2006

Xu M-Q, Feldman G, Le Merrer M, Shugart YY, Glaser DL, Urtizberea JA, Fardeau M, Connor JM, Triffitt J, Smith R, Shore EM, Kaplan FS. Linkage exclusion and mutational analysis of the noggin gene in patients with fibrodysplasia ossificans progressiva. Clin Genet 58: 291-298, 2000



L. Протеиновая модель мутаций при ПОФ

Chen Y-G, Liu F, Massagué J. Mechanism of TGF-β receptor inhibition by FKBP12. The EMBO J 16: 3866-3876. 1997

Groppe JC, Shore EM, Kaplan FS. Functional modeling of the ACVR1(R206H) mutation in FOP. Clin Orthop Relat Res 462: 87-92, 2007

Kaplan FS, Groppe J, Pignolo RJ, Shore EM. Morphogen receptor genes and metamorphogenes: skeleton keys to metamorphosis. Ann NY Acad Sci 1116: 113-133, 2007

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Shen Q, Xu M, Little SC, Kaplan FS, Mullins MC, Shore EM. Activation of КМГП signaling by the FOP ACVR1 R206H mutation. J Bone Miner Res 22 (suppl. 1); S43, 2007



Shore EM, Xu M, Feldman GJ, Fenstermacher DA, Cho T-J, Choi IH, Connor JM, Delai P, Glaser DL, Le Merrer M, Morhart R, Rogers JG, Smith R, Triffitt JT, Urtizberea JA, Zasloff M, Brown MA, Kaplan FS. A recurrent mutation in the КМГП type I receptor ACVR1 causes inherited and sporadic fibrodysplasia ossificans progressiva. Nature Genetics 38: 525-527, 2006

Wang T, Li B-Y, Danielson PD, Shah PC, Rockwell, S, Lechleider RJ, Martin J, Manganaro T, Donahoe PK. The immunophilin FKBP12 functions as a common inhibitor of the TGF-β family type I receptors. Cell 86: 435-444, 1996

Yamaguchi T, Kurisaki A, Yamakawa N, Minakuchi K, Sugino H. FKBP12 functions as an adaptor of the Smad7-Smurf1 complex on activin type I receptor. J Mol Endocrinol 36: 569-579, 2006

M. Генетическое тестирование и ПОФ

Kaplan FS, Xu M, Glaser DL, Collins F, Connor M, Kitterman J, Sillence D, Zackai E, Ravitsky V, Zasloff M, Ganguly A, Shore EM. Early diagnosis of fibrodysplasia ossificans progressiva. Pediatrics 121: e1295-e1300, 2008.



N. Модель ПОФ на лабораторных животных

Fukada T, Scott G, Komatsu Y, Araya R, Kawano M, Ray, MK, Yamada M, Mishina Y. Generation of a mouse with conditionally activated signaling through the КМГП receptor, ALK2. Genesis 44: 159-167, 2006

Glaser DL, Economides AN, Wang L, Liu X, Kimble RD, Fandl JP, Wilson JM, Stahl, N, Kaplan FS, Shore EM. In vivo somatic cell gene transfer of an engineered noggin mutein prevents BMP4-induced heterotopic ossification. J Bone Joint Surg Am 12: 2332-2342, 2003

Kan L, Hu M, Gomes WA, Kessler JA. Transgenic mice overexpressing BMP4 develop a fibrodysplasia ossificans progressiva (ПОФ)-like phenotype. Am J Pathol 165: 1107-1115, 2004

Kaplan FS, Shore EM, Pignolo RJ Glaser DL. Animal models of fibrodysplasia ossificans progressiva. Clin Rev Bone Miner Metab 3: 229-234, 2005

Olmsted EA, Gannon FH, Wang Z-Q, Grigoriadis AE, Wagner EF, Zasloff MA, Shore EM, Kaplan FS: Embryonic overexpression of the c-fos proto-oncogene: a murine stem cell chimera applicable to the study of fibrodysplasia ossificans progressiva in humans. Clin Orthop 346: 81-94, 1998



O. Проблемы терапевтической оценки ПОФ

Auerbach AD, Landefeld CS, Shojania KG. The tension between needing to improve care and knowing how to do it. N Engl J Med 357: 608-613, 2007

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Hellman S, Hellman DS. Of mice but not men: problems of the randomized clinical trial. New Engl J Med 324: 1585-1589, 1991



Kaplan FS, Glaser DL, Pignolo RJ, Shore EM. A new era for fibrodysplasia ossificans progressiva: a druggable target for the second skeleton. Expert Opin Biol Ther 7: 705-712, 2007

Kaplan FS, Le Merrer M, Glaser DL, Pignolo RJ, Goldsby R, Kitterman JA, Groppe J, Shore EM. Fibrodysplasia ossificans progressiva (ПОФ). Best Practice & Research – Clinical Rheumatology 22: 191-205, 2008

Miller FG, Rosenstein DL. The therapeutic orientation to clinical trials. N Engl J Med 348: 1383-1386, 2003

Passamani E. Clinical trials - are they ethical? New Engl J Med 324: 1589-1591, 1991

Rosenstirn J. A contribution to the study of myositis ossificans progressiva. Ann Surg 68: 485-520, 591-637, 1918

Stephens T, Brynner R: Dark Remedy. The Impact of Thalidomide And Its Revival as a Vital Medicine. Perseus Books, 208 pages, 2001

Yu PB, Hong CC, Sachidanandan C, Babitt JL. Dong DY, Hoyng SA, Lin HY, Bloch KD, Peterson RT. Dorsomorphin inhibitors КМГП signals required for embryogenesis and iron metabolism. Nat Chem Biol 4: 33-41, 2008

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II. ПАТОЛОГИЧЕСКИЕ И ПАТОФИЗИОЛОГИЧЕСКИЕ ОСНОВЫ МЕТОДОВ СИМПТОМАТИЧЕСКОГО ЛЕЧЕНИЯ ПОФ


Introduction

Glaser DL, Kaplan FS. Treatment considerations for the management of fibrodysplasia ossificans progressiva. Clin Rev Bone Miner Metab 3: 243-250. 2005

Kaplan FS, Le Merrer M, Glaser DL, Pignolo RJ, Goldsby R, Kitterman JA, Groppe J, Shore EM. Fibrodysplasia ossificans progressiva (ПОФ). Best Practice & Research – Clinical Rheumatology 22: 191-205, 2008

B. Corticosteroids

Janoff HB, Zasloff MA, Kaplan FS. Submandibular swelling in patients with fibrodysplasia ossificans progressiva. Otolaryngol Head Neck Surg 114: 599-604, 1996

Rhen T & Cidlowski JA. Anti-inflammatory action of glucocorticoids – new mechanisms for old drugs. N Engl J Med 353: 1711-1723, 2005

Staa TPV, Cooper C, Leufkens HGM, Bishop N. Children and the risk of fractures caused by oral corticosteroids. J Bone Miner Res 18: 913-918, 2003

C. Ингибиторы мастоцитов

Gannon FH, Glaser D, Caron R, Thompson LDR, Shore EM, Kaplan FS. Mast cell involvement in fibrodysplasia ossificans progressiva. Hum Pathol 32: 842-848, 2001

Kaplan AP. Chronic urticaria and angioedema. N Engl J Med 346:175-179, 2002

Simmons FER. Advances in H1 –Antihistamines. N Engl J Med 351:2203-2217, 2004



D. Ингибиторы циклооксигеназы 2 (ЦЩГ-2) и нестероидные противовоспалительные препараты (НСПВП)

Deeks JJ, Smith LA, Bradley MD. Efficacy, tolerability, and upper gastrointestinal safety of celecoxib for treatment of osteoarthritis and rheumatoid arthritis: systematic review of randomized controlled trials. British Med J (Review) 325: 619, 2002

DiCesare PE, Nimni ME, Pen L, Yazdi M, Cheung DT. Effects of Indomethacin on demineralized bone-induced heterotopic ossification in the rat. J Orthop Res 9: 855-861, 1991

Fitzgerald GA. Coxibs and cardiovascular disease. N Engl J Med 351: 1709-1711, 2004

Grosser T, Fries S, Fitzgerald GA. Biological basis for the cardiovascular consequences of cox-2 inhibition: therapeutic challenges and opportunities. J Clin Invest 116: 4-15, 2006

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Jones, MK, Wang H, Peskar BM, Levin E, Itani RM Sarfeh IJ, Tarnawski AS. Inhibition of angiogenesis by nonsteroidal anti-inflammatory drugs: insight into mechanisms and implications for cancer growth and ulcer healing. Nature Med 5: 1418-1423, 1999



Katori M, Majima M. Cyclooxygenase-2: its rich diversity of roles and possible application of its selective inhibitors. Inflammation Res 49: 367-392, 2000

Levitz CL, Cohen RB, Zasloff MA, Kaplan FS. The role of prostaglandins in bone formation. Abstracts from The First International Symposium on Fibrodysplasia Ossificans Progressiva, September 25-26, 1991, Philadelphia, Pennsylvania. Calcif Tissue Int 50: 385-388, 1992

Merck & Co: Merck announces voluntary worldwide withdrawal of Vioxx. www.merck.com. September 30, 2004

Simon AM, Manigrasso MB, O’Connor JP. Cyclo-oxygenase 2 function is essential for bone fracture healing. J Bone Miner Res 17: 963-976, 2002

Stempak D, Gammon J, Klein J, Koren G, Baruchel S. Single-dose and steady-state pharmacokinetics of celecoxib in children. Clin Pharmacol Ther 72: 490-497, 2002

Topol EJ. Failing the public health – rofecoxib, Merck, and the FDA. N Engl J Med 351:1707-1709, 2004

Van Ryn J, Pairet M: Clinical experience with cyclooxygenase-2 inhibitors. Inflammation Res. 48: 247-254, 2000

Weinreb M, Suponitsky I, Keila S. Systemic administration of an anabolic dose of PGE2 in young rats increases the osteogenic capacity of bone marrow. Bone 20: 521-526, 1997

White WB, Faich G, Borer JS, Makuch RW. Cardiovascular thrombotic events in arthritis trials of the cyclooxygenase-2 inhibitor celecoxib Am J Cardiol 92: 411-418, 2003

White WB, Faich G, Whelton A, Maurath C, Ridge NJ, Verburg KM, Geis GS, Lefkowith JB. Comparison of thromboembolic events in patients treated with celecoxib, a cyclooxygenase-2 specific inhibitor, versus ibuprofen or diclofenac. Am J Cardiol 89: 425-430, 2002

Zhang X, Schwarz EM, Young DA, Puzas E, Rosier RN, O’Keefe RJ. Cyclo-oxygenase-2 regulates mesenchymal cell differentiation into the osteoblast lineage and is critically involved in bone repair. J Clin Invest 109: 1405-1415, 2002

E. Аминобисфосфонаты

Bilezikian JP. Osteonecrosis of the jaw – do bisphosphonates pose a risk? N Engl J Med 355: 2278-2281, 2006

Black DM, Delmas PD, Eastell R, Reid IR, Boonen S, Cauley JA, Cosman F, Lakatos P, Leung PC, Man Z, Mautalen C, Mesenbrink P, Hu H, Caminis J, Tong K, Rosario-Jansen T, Krasnow J, Hue TF, Sellmeyer D, Eriksen EF, Cummings SR. Horizon Pivotal Fracture Trial. Once yearly zoledronic acid for treatment of postmenopausal osteoporosis. N Engl J Med 356: 1809-1822, 2007

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Brantus JF, Meunier PJ. Effects of intravenous etidronate and oral corticosteroids in fibrodysplasia ossificans progressiva. Clin Orthop 346: 117-120, 1998



Brumsen C, Hamdy NAT, Papapoulos SE. Long-term effects of bisphosphonates on the growing skeleton (studies of young patients with severe osteoporosis). Medicine 76: 266-283, 1997

Body JJ. Dosing regimens and main adverse events of bisphosphonates. Semin Oncol (4 supplement 11): 49-53, 2001

DiMeglio LA, Peacock M. Two-year clinical trial of oral alendronate versus intravenous pamidronate in children with osteogenesis imperfecta. J Bone Miner Res 21: 132-140, 2006

Dunford JE, Thompson K, Coxon FP, Luckman SP, Hahn FM, Poulter CD, Ebetino FH, Rogers MJ. Structure-activity relationships for inhibition or farnesyl diphosphate synthase in vitro and inhibition of bone resorption in vivo by nitrogen-containing bisphosphonates. J Pharmacol Exp Therap 296: 235-242, 2001

Falk MJ, Heeger S, Lynch KA, DeCaro KR, Bohach D, Gibson KS, Warman ML. Intravenous bisphosphonate therapy in children with osteogenesis imperfecta. Pediatrics 111: 573-578, 2003

Fournier P, Boissier S, Filleur S, Guglielmi J, Cabon F, Colombel M, Clezardin P. Bisphosphonates inhibit angiogenesis in vitro and testosterone-stimulated vascular regrowth in the ventral prostate in castrated rats. Cancer Res 62: 6538-6544, 2002

Green JR. Preclinical pharmacology of zoledronic acid. Semin Oncol 29 (6 suppl. 21): 3-11, 2002

Green JR. Antitumor effects of bisphosphonates. Cancer 97 (supplement): 840-847, 2003

Idris AI, Rojas J, Greig IR, Van’t Hof RJ, Ralston SH. Aminobisphosphonates cause osteoblast apoptosis and inhibit bone nodule formation in vitro. Calcif Tissue Int 82: 191-201, 2008

Khosla S, Burr D, Cauley J, Dempster DW, Ebeling PR, Felsenberg D, Gagel RF, Gilsanz V, Guise T, Koka S, McCauley LK, McGowan J, McKee MD, Mohla S, Pendrys DG, Raisz LG, Ruggiero SL, Shafer DM, Shum L, Silverman SL, Van Poznak CH, Watts N, Woo SB, Shane E. Bisphosphonate-associated osteonecrosis of the jaw: report of a task force of The American Society for Bone and Mineral Research. J Bone Miner Res 22: 1479-1491, 2007

Marini JC. Do bisphosphonates make children’s bones better or brittle? New Engl J Med 349:423-426, 2003

Munns CFJ, Rauch F, Zeitlin L, Fassier F, Glorieux FH. Delayed osteotomy but not fracture healing in pediatric osteogenesis imperfecta patients receiving pamidronate. J Bone Min Res 19: 1779-1786, 2004

Nogginuera A, Ros JB, Pavia C, Alcover E, Valls C, Villaronga M, Gonzalez E. Bisphosphonates, a new treatment for glucocorticoid-induced osteoporosis in children. J Pediatr Endocrinol Metab 16: 529-536, 2003

Orcel P, Beaudreuil J. Bisphosphonates in bone disease other than osteoporosis. Joint Bone Spine 69: 19-27, 2002

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Pechersturfer M, Jilch R, Sauty A, Horn E, Keck AV, Zimmer-Roth I, Thiebaud D. Effect of first treatment with aminobisphosphonates pamidronate and ibandronate on circulating lymphocyte subpopulations. J Bone Miner Res 15: 147-154, 2000



Rauch F, Plotkin H, Zeitlin L, Glorieux FH. Bone mass, size, and density in children and adolescents with osteogenesis imperfecta: effect of intravenous pamidronate therapy. J Bone Miner Res 18: 610-614, 2003

Rauch F, Travers R, Plotkin H, Glorieux FH. The effects of intravenous pamidronate on the bone tissue of children and adolescents with osteogenesis imperfecta. J Clin Invest 110: 1293-1299, 2002

Rosen CJ, Brown S. Severe hypocalcemia after intravenous bisphosphonate therapy in occult vitamin D deficiency. N Engl J Med 348: 1503-1504, 2003

Sambrook PN, Kotowicz M, Nash P, Styles CB, Naganathan V, Henderson-Briffa KN, Eisman JA, Nicholson GC. Prevention and treatment of glucocorticoid-induced osteoporosis: a comparison of calcitriol, vitamin D plus calcium, and alendronate plus calcium. J Bone Miner Res 18: 919-924, 2003

Santini D, Vincenzi B, Avvisati G, Dicuonzo G, Battistoni F, Gavasci M, Salerno A, Denaro V, Tonini G. Pamidronate induces modifications of circulating angiogenetic factors in cancer patients. Clin Cancer Res: 8: 1080-1084, 2002

Schuetz P, Mueller B, Christ-Crain M, Dick W, Haas H. Amino-bisphosphonates in heterotopic ossification: first experience in five consecutive cases. Spinal Cord 43: 604-610, 2005

Staa TPV, Cooper C, Leufkens HGM, Bishop N. Children and the risk of fractures caused by oral corticosteroids. J Bone Miner Res 18: 913-918, 2003

Still K, Phipps RJ, Scutt A. Effects of risedronate, alendronate, and etidronate on the viability and activity of rat bone marrow stromal cells in vitro. Calcif Tissue Int 72: 143-150, 2003

Tassone P, Forciniti, S, Galea E, Morrone G, Turco MC, Martinelli V, Tagliaferri P, Venuta S. Growth inhibition and synergistic induction of apoptosis by zoledronate and dexamethasone in human myeloma cell lines. Leukemia 14: 841-844, 2000

Whyte MP, Wenkert D, Clements KL, McAlister WH, Mumm S. Bisphosphonate-induced osteopetrosis. N Engl J Med 349: 457-463, 2003

Wood J, Bonjean K, Ruetz S, Bellahcene A, Devy L, Foidart JM, Castronovo V, Green JR. Novel antiangiogenic effects of the bisphosphonate compound zoledronic acid. J Pharmacol Exp Therap 302: 1055-1061, 2002

F. Снятие острых и хронических болей, связанных с ПОФ

Kaplan FS, Le Merrer M, Glaser DL, Pignolo RJ, Goldsby R, Kitterman JA, Groppe J, Shore EM. Fibrodysplasia ossificans progressiva (ПОФ). Best Practice & Research – Clinical Rheumatology 22: 191-205, 2008

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G. Миорелаксанты

Glaser DL, Kaplan FS. Treatment considerations for the management of fibrodysplasia ossificans progressiva. Clin Rev Bone Miner Metab 3: 243-250. 2005



H. Химиотерапевтические препараты и лучевая терапия

Glaser DL, Kaplan FS. Treatment considerations for the management of fibrodysplasia ossificans progressiva. Clin Rev Bone Miner Metab 3: 243-250. 2005

Kaplan FS, Le Merrer M, Glaser DL, Pignolo RJ, Goldsby R, Kitterman JA, Groppe J, Shore EM. Fibrodysplasia ossificans progressiva (ПОФ). Best Practice & Research – Clinical Rheumatology 22: 191-205, 2008

I. Пересадка костного мозга

Kaplan FS, Glaser DL, Shore EM, Pignolo RJ, Xu M, Zhang Y, Senitzer D, Forman SJ, Emerson SG. Hematopoietic stem-cell contribution to ectopic skeletogenesis. J Bone Joint Surg Am 89: 347-357, 2007



J. Прочие препараты

Benetos IS, Mavrogenis AF, Themistocleous GS, Kanellopoulos AD, Papagelopoulos PJ, Soucacos PN. Optimal treatment of fibrodysplasia ossificans progressiva with surgical excision of heterotopic bone, indomethacin, and irradiation. J Surg Orthop Adv 15: 99-104, 2006

Moore, SE, Jump A, Smiley JD. Effect of warfarin sodium therapy on excretion of 4-carboxy-L-glutamic acid in scleroderma, dermatomyositis, and myositis ossificans progressiva. Arthritis Rheum 29: 344-351, 1986

Olsen NJ, Stein CM. New drugs for rheumatoid arthritis. N Engl J Med 350: 2167-2179, 2004

Wieder DL. Treatment of traumatic myositis ossificans with acetic acid iontophoresis. Phys Ther 72: 133-137, 1992

Zasloff MA, Rocke DM, Crofford LJ, Hahn GV, Kaplan FS. Treatment of patients who have fibrodysplasia ossificans progressiva with isotretinoin. Clin Orthop 346: 121-129, 1998


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