Ostegenezis İmperfekta Olan Çocuklarda Pamidronat ve Kalsitonin Tedavilerinin Karşılaştırılması

Neslihan Önenli Mungan; Fatih Gürbüz; Eda Mengen; Özden Özgür; Ali Kemal Topaloglu; Bilgin Yüksel

EN TR

Comparison of Calcitonin and Pamidronate Treatments in Children with Osteogenesis Imperfecta

Abstract

Purpose: The main objective of this study was to compare the treatments of calcitonin and pamidronate by clinical, biochemical, and radiological findings in children with osteogenesis imperfecta and evaluate the efficiency of pamidronate treatment. Patients and methods: A total of 12 patients, aged 41±38 (1-120) months were studied. Group 1 was consisted of six patients who had received intranasal calcitonin at a dosage of 4-6 U/kg three times a week before switching to pamidronate treatment. Group 2 was also consisted of six patients who had received only pamidronate infusion at a dosage of 0.5-2 mg/kg every two months. Results: Annual fracture rates decreased from 2.72 ± 0.80 to 0.40 ± 0.70 (p

Keywords

Osteogenesis imperfecta, calcitonin, pamidronate, children.

Kaynakça

Van Dijk FS, Pals G, van Rijn RR, Nikkels PG, Cobben JM: Classification of Osteogenesis Imperfecta revisited. Eur J Med Genet. 2010; 53: 1–5
Lindsay R. Modeling the benefits of pamidronate in children with osteogenesis imperfecta. J Clin Invest. 2002; 110: 1239-43.
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. 2002; 110: 1293-99.
Glorieux FH, Bishop NJ, Plotkin H, Chabot G, Lanoue G, Travers R. Cyclic administration of pamidronate in children with severe osteogenesis imperfecta. N Engl J Med. 1998; 339: 986-7.
Sillence DO, Senn A, Danks DM: Genetic heterogeneity in osteogenesis imperfecta. J Med Genet. 1979; 16: 101–16.
Glorieux FH, Rauch F, Plotkin H et al: Type V osteogenesis imperfecta: a new form of brittle bone disease. J Bone Miner Res. 2000; 15: 1650–8.
Glorieux FH, Ward LM, Rauch F, Lalic L, Roughly PJ, Travers R: Osteogenesis imperfecta type VI: a form of brittle bone disease with mineralization defect. J Bone Miner Res. 2002; 17: 30–7.
Byers PH, Bonadio JF, Steinmann B. Osteogenesis imperfecta: update and perspective. Am J Med Genet. 1984; 17: 429-35.
Barnes AM, Chang W, Morello R, Cabral WA, Weis M, et al: Deficiency of cartilage-associated protein in recessive lethal osteogenesis imperfecta. N Engl J Med. 2006; 355: 2757–64..
Morello R, Bertin TK, Chen Y, Hicks J, Tonachini L, et al: CRTAP is required for prolyl 3-hydroxylation and mutations cause recessive osteogenesis imperfecta. Cell .2006; 127: 291–304.

Cabral WA, Chang W, Barnes AM, Weis M, Scott MA, et al: Prolyl 3-hydroxylase 1 deficiency causes a recessive metabolic bone disorder resembling lethal/severe osteogenesis imperfecta. Nat Genet. 2007; 39: 359–65.
Van Dijk FS, Nesbitt IM, Zwikstra EH, Nikkels PG, Piersma SR, et al: PPIB mutations cause severe osteogenesis imperfecta. Am J Hum Genet. 2009;85: 521–
Barnes AM, Carter EM, Cabral WA, Weis M, Chang W, et al: Lack of cyclophilin B in osteogenesis imperfecta with normal collagen folding. N Engl J Med. 2010; 362: 521–8.
Christiansen HE, Schwarze U, Pyott SM, Al- Swaid A, Al Balwi M, et al: Homozygosity for a missense mutation in SERPINH1 , which encodes the collagen chaperone protein HSP47, results in severe recessive osteogenesis imperfecta. Am J Hum Genet. 2010; 86: 389–98.
Alanay Y, Avaygan H, Camacho N, Utine GE, Boduroglu K, et al: Mutations in the gene encoding the RER protein FKBP65 cause autosomal recessive osteogenesis imperfecta. Am J Hum Genet. 2010; 86: 551–9.
Lapunzina P, Aglan M, Temtamy S, Caparrós Martin JA, Valencia M, et al: Identification of a frameshift mutation in Osterix in a patient with recessive osteogenesis imperfecta. Am J Hum Genet. 2010; 87: 110–4).
Becker J, Semler O, Gilissen C, Li Y, Bolz HJ, et al: Exome sequencing identifies truncating mutations in human SERPINF1 in autosomal recessive osteogenesis imperfecta. Am J Hum Genet. 2011; 88: 362–71.
Lee YS, Low SL, Lim AL, Loke KY. Cyclic pamidronate infusion improves bone mineralization and reduces fracture incidence in osteogenesis imperfecta. Eur J Pediatr. 2001; 1: 1-4.
Moriwake T, Seino Y. Recent progress in diagnosis and treatment of osteogenesis imperfecta. Acta Pediatr Jpn. 1997; 39: 521-7.
Castell S, Colbert C, Chakrabarti C, Bachtell RS, Kassner EG, Yasumura S. Therapy for osteogenesis imperfecta with synthetic salmon calcitonin. J Pediatr. 1991; 43: 49-54.
Nishi Y, Hamato K, Kajiyama M, Ono H, Kihara M, Jinno K. The effect of long term calcitonin therapy by injection and nasal spray on incidence of fractures in osteogenesis imperfecta. J Pediatr. 1979; 95: 807-11.
Özer G, Mungan Önenl i N, Yüksel B, Yıldızdaş D, Teker Z, Satar M. Osteogenesis imperfektalı 15 hastanın değerlendirilmesi. Türk Pediatri Arşivi. 2001; 36: 155-9.
Glorieux FH. The use of bisphosphonates in children with osteogenesis imperfecta. J Pediatr Endocrinol Metab.. 2001; 14: 1491-5.
Vasikaran SD. Bisphosphonates: an overview with special reference to alendronate. Ann Clin Biochem. 2001; 38: 608-23.
Guillot M, Eckart P, Desrosieres H, Amiour M, alJazayri Z. Osteogenesis imperfecta: a new, early therapeutic approach with bisphosphonate. A case report. Arch Pediatr. 2001; 8: 172-5.
Bembi B, Parma A, Bottega M, Ceschel C, Zanatta M, Martini C, Ciana G. Intravenous pamidronate treatment in osteogenesis imperfecta. J Pediatr. 1997; 13: 622-5.
Glorieux FH. Bisphosphonate therapy for severe osteogenesis imperfecta. J Pediatr Endocrinol Metab.. 2000; 13: 989-92.
Astrom E, Soderhall S. Beneficial effect of long term intravenous bisphosphonate treatment of osteogenesis imperfecta. Arch Dis Child. 2002; 86: 356Falk MJ, Heeger SH, Lynch KA, DeCaro KR, Bohach D, Gibson KS, Warman ML. Intravenous bisphosphonates therapy in children with osteogenesis imperfecta. Pediatrics. 2003; 111: 573
Letocha AD, Cintas HL, Troendle JF, Reynolds JC, Cann CE, Chernoff EJ, Hill SC, Gerber LH, Marini JC. Controlled trial of pamidronate in children with types III and IV osteogenesis imperfecta confirms vertebral gains but not short-term functional improvement. J Bone Miner Res. 2005; 20: 977–86.
Gonzalez E, Pavia C, Ros J, Villaronga M, Valls C, Escola J. Efficacy of low dose schedule pamidronate infusion in children with osteogenesis imperfecta. J Pediatr Endocrinol Metab. 2001; 14: 529-33.
Nishi Y, Hamamoto K, Kajiyama M, Ono H, Kihara M, Jinno K. Effect of long-term calcitonin therapy by injection and nasal spray incidence of fractures in osteogenesis imperfecta. J Pediatr. 1992; 121: 477
Castells S, Colbert C, Chakrabarti C, Bachtell RS, Kassner EG, Yai S. Therapy of osteogenesis imperfecta with synthetic salmon calcitonin. J Pediatr. 1979; 95: 807-11.
Rebelo I, Silva LP, Blanco JCM, Monteiro ME, Ferreira NC. Effects of synthetic salmon calcitonin therapy in children with osteogenesis imperfecta. Int Med Res. 1989; 17: 401-5.
Gökşen D, Coker M, Darcan S, Köse T, Kara S. Low-dose intravenous pamidronate treatment in osteogenesis imperfecta. Turk J Pediatr. 2006; 48: 124Andiran N, Alikasifoglu A, Gonc N, Ozon A, Kandemir N, Yordam N. Cyclic pamidronate therapy in children with osteogenesis imperfecta: results of treatment and follow-up after discontinuation. J Pediatr Endocrinol Metab. 2008; 21: 63-72.
Salehpour S, Tavakkoli S. Cyclic pamidronate therapy in children with osteogenesis imperfecta. J Pediatr Endocrinol Metab. 2010; 23: 73-80.
Wade JP. Osteoporosis. CMAJ. 2001; 165: 45-50.
Pedersen U, Charles P, Hansen HH, Elbrand O. Lack of effects of human calcitonin in osteogenesis imperfecta. Acta Orthop Scand. 1985; 56: 260-4.
August GP, Shapiro J, Hung W. Calcitonin therapy of children with osteogenesis imperfecta. J Pediatr. 1977; 91: 1001-5.
Baratelli Mm, Rizzi M, Corradi A. Osteogenesis imperfecta and calcitonin. Considerations on years of Clinical experimentation. Arch Sci Med. 1983; 140: 379Zacharin M, Bateman J. Pamidronate treatment of osteogenesis imperfecta-lack of correlation between clinical severity, age at onset of treatment, predicted collagen mutation and treatment response. J Pediatr Endocrinol Metab.. 2002; 15: 163-74.
Brumsen C, Hamdy NA, Papapoulos SE. Long-term effects of bisphosphonates on the growing skeleton. Studies of young patients with severe osteoporosis. Medicine. 1997; 76: 266-8.
Yazışma Adresi / Address for Correspondence: Dr. Fatih GÜRBÜZ Çukurova Üniversitesi Tıp Fakültesi, Çocuk Endokrinolojisi Bilim Dalı ADANA e-mail: fggurbuz@yahoo.com geliş tarihi/received :01.04.2013 kabul tarihi/accepted:25.04.2013

Ayrıntılar

Birincil Dil

Türkçe

Konular

-

Bölüm

-

Yazarlar

Neslihan Önenli Mungan Bu kişi benim

Fatih Gürbüz Bu kişi benim

Eda Mengen Bu kişi benim

Özden Özgür Bu kişi benim

Ali Kemal Topaloglu Bu kişi benim

Bilgin Yüksel Bu kişi benim

Yayımlanma Tarihi

1 Aralık 2013

Gönderilme Tarihi

22 Temmuz 2014

Kabul Tarihi

-

Yayımlandığı Sayı

Yıl 1970 Cilt: 38 Sayı: 4

IZ

https://izlik.org/JA97MA67MP

MLA

Mungan, Neslihan Önenli, vd. “Ostegenezis İmperfekta Olan Çocuklarda Pamidronat ve Kalsitonin Tedavilerinin Karşılaştırılması”. Cukurova Medical Journal, c. 38, sy 4, Aralık 2013, ss. 667-74, https://izlik.org/JA97MA67MP.

Bu eser Creative Commons Atıf-Gayriticari-Türetilemez 4.0 Uluslararası Lisansı ile lisanslanmıştır.

Ostegenezis İmperfekta Olan Çocuklarda Pamidronat ve Kalsitonin Tedavilerinin Karşılaştırılması

Comparison of Calcitonin and Pamidronate Treatments in Children with Osteogenesis Imperfecta

Abstract

Keywords