Acta Medica Iranica 2017. 55(10):658-660.

Denosumab Treatment of Severe Disuse Osteoporosis in a Boy With Spinal Muscular Atrophy
Stepan Kutilek

Abstract


Denosumab is a fully human recombinant monoclonal antibody to the receptor activator of nuclear factor-κB ligand. Denosumab is used in the treatment of postmenopausal osteoporosis and cancer-related bone disorders. There are only very scarce data on denosumab treatment in children. 14-year-old boy with spinal muscular atrophy (SMA) and severe disuse osteoporosis (spinal bone mineral density L1-L4 BMD-6.2SD Z-score) and two prevalent fragility fractures was treated with denosumab. He received 60 mg  subcutaneous injection at the baseline and seven months later. Six months after the initial injection there was a 19% increase in L1-L4 BMD. The injections were well tolerated without any adverse reactions. Calcemia remained stable (2.3-2.4 mmol/L). He was scheduled for the third denosumab injection six months later. Prior to this date, he acquired pneumonia and died due to respiratory failure, which is a frequent cause of death in patients with SMA. There was no relation to the denosumab treatment. In conclusion, one dose of denosumab significantly increased BMD in a child with severe osteoporosis.


Keywords


Denosumab; Spinal muscular atrophy; Fractures; Bone; Osteoporosis; Bone mineral density

Full Text:

PDF

References


Lewiecki EM, Bilezikian JP. Denosumab for the treatment of osteoporosis and cancer-related conditions. Clin Pharmacol Ther 2012;91:123-33.

Cummings SR, San Martin J, McClung MR, Siris ES, Eastell R, Reid IR, et al. FREEDOM Trial.Denosumab for prevention of fractures in postmenopausal women with osteoporosis. N Engl J Med 2009;361:756-65.

Boyce AM, Chong,WH, Yao J,, Gafni RI, Kelly MH, Chamberlain CE, et al. Denosumab treatment for fibrous dysplasia. J Bone Miner Res 2012;27:1462-70.

Karras NA, Polgreen LE, Ogilvie C, Manivel JC, Skubitz KM, Lipsitz E. Denosumab treatment of metastatic giant-cell tumor of bone in a 10-year-old girl. J Clin Oncol 2013;31:200-2.

Naidu A, Malmquist MP, Denham CA, Schow SR Management of central giant cell granuloma with subcutaneous denosumab therapy. J Oral Maxillofac Surg 2014;72:2469-84.

Grasemann C, Schündeln MM, Hövel M, Schweiger B, Bergmann C, Herrmann R, et al. Effects of RANK-ligand antibody (denosumab) treatment on bone turnover markers in a girl with juvenile Paget's disease. J Clin Endocrinol Metab 2013;98:3121-6.

Lange T, Stehling C, Fröhlich B, Klingenhöfer M, Kunkel

P, Schneppenheim R, et al. Denosumab: a potential new and innovative treatment option for aneurysmal bone cysts. Eur Spine J 2013;22:1417-22.

Pelle DW, Ringler JW, Peacock JD, Kampfschulte K, Scholten DJ 2nd, Davis MM, et al. Targeting receptor-activator of nuclear kappaB ligand in aneurysmal bone cysts: verification of target and therapeutic response. Transl Res 2014;164:139-48.

Semler O, Netzer C, Hoyer-Kuhn H, Becker J, Eysel P, Schoenau E. First use of the RANKL antibody denosumab in osteogenesis imperfecta type VI. J Musculoskelet Neuronal Interact 2012;12:183-8.

Hoyer-Kuhn H, Semler O, Schoenau E.Effect of denosumab on the growing skeleton in osteogenesis imperfecta. J Clin Endocrinol Metab 2014;99:3954-5.

Shroff R, Beringer O, Rao K, Hofbauer LC, Schulz A.Denosumab for post-transplantation hypercalcemia in osteopetrosis. N Engl J Med 2012;367:1766-7.

Iascone DM, Henderson CE, Lee JC. Spinal muscular atrophy: from tissue specificity to therapeutic strategies. F1000Prime Rep 2015;7:4.

Bachrach LK, Ward LM. Clinical review: Bisphosphonate use in childhod osteoporosis. J Clin Endocrinol Metab 2009;94:400-9.


Refbacks

  • There are currently no refbacks.


Creative Commons Attribution-NonCommercial 3.0

This work is licensed under a Creative Commons Attribution-NonCommercial 3.0 Unported License which allows users to read, copy, distribute and make derivative works for non-commercial purposes from the material, as long as the author of the original work is cited properly.