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Çocuk ve Adölesanlarda Uygulanan Pamidronat Tedavisinin Etkinlik ve Güvenilirliği

Year 2026, Volume: 16 Issue: 1, 1 - 7, 23.01.2026

Abstract

Amaç: Pamidronat, çocuklarda osteoporoz ve hiperkalseminin tedavisinde tercih edilen ilaçtır. Bu çalışma, ilacın güvenilirliğini ve etkinliğini incelemeyi amaçlamaktadır.
Gereç ve Yöntemler: Osteoporoz ve hiperkalsemi nedeniyle pamidronat tedavisi uygulanan hastalar çalışmaya dahil edildi. Osteoporoz hastaları üç ayda bir üç gün boyunca 1 mg/kg/gün dozunda tedavi edilirken, hiperkalsemi hastaları tek seferlik 1 mg/kg dozunda tedavi edildi. Boy, kilo, vücut kitle indeksi, kemik mineral yoğunluğu (BMD), kırıklar, biyokimyasal veriler ve komplikasyonlar tedavi öncesinde ve tedaviden en az altı ay sonra değerlendirildi.
Bulgular: Çalışmaya dahil edilen 31 hastanın 21'i (%67,7) erkek, 10'u (%32,3) kadındı ve ortalama yaş 12,20±4,31 idi. 20 hastada (%64,5) sekonder osteoporoz, 7 hastada (%22,6) osteogenezis imperfekta ile ilişkili primer osteoporoz ve 4 hastada (%12,9) hiperkalsemi tanısı konuldu. Ortalama takip süresi 11,26±4,16 aydı. Osteoporoz için pamidronat ile tedavi edilen hastalarda tedavi öncesi ortalama BMD -3,53 ±1,43 iken, tedavi sonrası -1,42 ±1,81 idi (p<0,001). Tedavi öncesi ve sonrası dönemler karşılaştırıldığında, toplam kırık sayısı, vertebral kırık sayısı ve yılda birden fazla kez meydana gelen kırık sayısında azalma gözlendi (p<0,001). Altı hastada (%19,3) komplikasyonlar gözlendi, ancak bu yan etkilerin tümü geçiciydi.
Sonuç: Farklı gruplardaki hastalara çeşitli dozlarda ve farklı sürelerde uygulanan pamidronat tedavisi ile etkinlik veya güvenlik açısından herhangi bir farklılık olmadığı gözlendi. Bildirilen düşük komplikasyon oranları literatürle uyumlu olup tedavinin güvenliğini doğrulamaktadır.

Ethical Statement

Bu çalışma Helsinki Bildirgesi'ne uygun olarak gerçekleştirilmiş ve Ankara Bilkent Şehir Hastanesi Kurumsal İnceleme Kurulu (IRB) tarafından onaylanmıştır (IRB No. E2-22-1931).

References

  • 1. Arundel P, Bishop N. Primary osteoporosis. Endocr Dev. 2015;28:162-75. doi:10.1159/000381037.
  • 2. Korula S, Titmuss AT, Biggin A, Munns CF. A practical approach to children with recurrent fractures. Endocr Dev. 2015;28:210-25. doi:10.1159/000381047.
  • 3. Shuhart CR, Yeap SS, Anderson PA, Jankowski LG, Lewiecki EM, Morse LR, et al. Executive summary of the 2019 ISCD position development conference on monitoring treatment, DXA cross-calibration and least significant change, spinal cord injury, peri-prosthetic and orthopedic bone health, transgender medicine, and pediatrics. J Clin Densitom. 2019;22:453-71. doi:10.1016/j.jocd.2019.07.001.
  • 4. Russell RG. Bisphosphonates: mode of action and pharmacology. Pediatrics. 2007;119(suppl 2):150-62. doi:10.1542/peds.2006-2023H.
  • 5. Camacho PM, Petak SM, Binkley N, Diab DL, Eldeiry LS, Farooki A, et al. American association of clinical endocrinologists/american college of endocrinology clinical practice guidelines for the diagnosis and treatment of postmenopausal osteoporosis-2020 update. Endocr Pract. 2020;26(suppl 1):1-46. doi:10.4158/GL-2020-0524SUPPL.
  • 6. Bachrach LK, Ward LM. Clinical review 1: bisphosphonate use in childhood osteoporosis. J Clin Endocrinol Metab. 2009;94(2):400-9. doi:10.1210/jc.2008-1531.
  • 7. Jin HY, Noh ES, Hwang IT. Diagnosis and management of osteoporosis in children and adolescents. The Ewha Medical Journal. 2023;46(4):e18. doi:10.12771/emj.2023.e18
  • 8. Simm PJ, Biggin A, Zacharin MR, Rodda CP, Tham E, Siafarikas A, et al. APEG bone mineral working group. Consensus guidelines on the use of bisphosphonate therapy in children and adolescents. J Paediatr Child Health. 2018;54(3):223-33. doi:10.1111/jpc.13768.
  • 9. Nussbaum SR, Younger J, Vandepol CJ, Gagel RF, Zubler MA, Chapman R, et al. Single-dose intravenous therapy with pamidronate for the treatment of hypercalcemia of malignancy: comparison of 30-, 60-, and 90-mg dosages. Am J Med. 1993;95:297-304. doi:10.1016/0002-9343(93)90282-t.
  • 10. Sternlicht H, Glezerman IG. Hypercalcemia of malignancy and new treatment options. Ther Clin Risk Manag. 2015;11:1779-88. doi:10.2147/TCRM.S83681.
  • 11. Munns CF, Shaw N, Kiely M, Specker BL, Thacher TD, Ozono K, et al. Global consensus recommendations on prevention and management of nutritional rickets. Horm Res Paediatr. 2016;85:83-106. doi:10.1159/000443136.
  • 12. Demir K, Konakçı E, Özkaya G, Kasap Demir B, Özen S, Aydın M, et al. New features for child metrics: further growth references and blood pressure calculations. J Clin Res Pediatr Endocrinol. 2020;12:125-29. doi:10.4274/jcrpe.galenos.2019.2019.0127.
  • 13. Martinez-Soto T, Pacaud D, Stephure D, Trussell R, Huang C. Treatment of symptomatic osteoporosis in children: a comparison of two pamidronate dosage regimens. J Pediatr Endocrinol Metab. 2011;24:271-4. doi:10.1515/jpem.2011.182.
  • 14. Dwan K, Phillipi CA, Steiner RD, Basel D. Bisphosphonate therapy for osteogenesis imperfecta. Cochrane Database Syst Rev. 2016;10:CD005088. doi:10.1002/14651858.CD005088.pub4.
  • 15. Szczepaniak-Kubat A, Kurnatowska O, Jakubowska-Pietkiewicz E, Chlebna-Sokół D. Assessment of quality of life of parents of children with osteogenesis imperfecta. Adv Clin Exp Med. 2012;21(1):99-104.
  • 16. Marginean O, Tamasanu RC, Mang N, Mozos I, Brad GF. Therapy with pamidronate in children with osteogenesis imperfecta. Drug Des Devel Ther. 2017;11:2507-15. doi:10.2147/DDDT.S141075.
  • 17. Kok DH, Sakkers RJ, Janse AJ, Pruijs HE, Verbout AJ, Castelein RM, et al. Quality of life in children with osteogenesis imperfecta treated with oral bisphosphonates (Olpadronate): a 2-year randomized placebo-controlled trial. Eur J Pediatr. 2007;166:1155-61. doi:10.1007/s00431-006-0399-2.
  • 18. Lee M, Kwon A, Song K, Lee HI, Choi HS, Suh J, et al. Effectiveness and safety of pamidronate treatment in nonambulatory children with low bone mineral density. Ann Pediatr Endocrinol Metab. 2024;29:46-53. doi:10.6065/apem.2346028.014.
  • 19. Juszczak B, Sułko J. Patient-reported effectiveness and safety of pamidronate in NSAIDs-refractory chronic recurrent multifocal osteomyelitis in children. Rheumatol Int. 2022;42:699-706. doi:10.1007/s00296-021-04886-4.
  • 20. Alcausin MB, Briody J, Pacey V, Ault J, McQuade M, Bridge C, et al. Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age. Horm Res Paediatr. 2013;79:333-40. doi:10.1159/000351374.
  • 21. Shi CG, Zhang Y, Yuan W. Efficacy of bisphosphonates on bone mineral density and fracture rate in patients with osteogenesis imperfecta: a systematic review and meta-analysis. Am J Ther. 2016;23:894-904. doi:10.1097/MJT.0000000000000236.
  • 22. Ward LM. A practical guide to the diagnosis and management of osteoporosis in childhood and adolescence. Front Endocrinol (Lausanne). 2024;25:1266986. doi:10.3389/fendo.2023.1266986.
  • 23. Chilbule SK, Madhuri V. Complications of pamidronate therapy in paediatric osteoporosis. J Child Orthop. 2012;6:37-43. doi:10.1007/s11832-012-0383-5.
  • 24. Sees JP, Sitoula P, Dabney K, Holmes L Jr, Rogers KJ, Kecskemethy HH, et al. Pamidronate treatment to prevent reoccurring fractures in children with cerebral palsy. J Pediatr Orthop. 2016;36:193-7. doi:10.1097/BPO.0000000000000421.
  • 25. McNeilly JD, Boal R, Shaikh MG, Ahmed SF. Frequency and aetiology of hypercalcaemia. Arch Dis Child. 2016;101:344-7. doi:10.1136/archdischild-2015-309029.
  • 26. Stokes VJ, Nielsen MF, Hannan FM, Thakker RV. Hypercalcemic disorders in children. J Bone Miner Res. 2017;32:2157-70. doi:10.1002/jbmr.3296.
  • 27. Bharill S, Wu M. Hypocalcemia and hypercalcemia in children. Pediatr Rev. 2023;44:533-6. doi:10.1542/pir.2022-005578.
  • 28. Davies JH, Shaw NJ. Investigation and management of hypercalcaemia in children. Arch Dis Child. 2012;97:533-8. doi:10.1136/archdischild-2011-301284.
  • 29. Ward L, Tricco AC, Phuong P, Cranney A, Barrowman N, Gaboury I, et al. Bisphosphonate therapy for children and adolescents with secondary osteoporosis. Cochrane Database Syst Rev. 2007;2007(4):CD005324. doi:10.1002/14651858.CD005324.pub2.
  • 30. Plotkin H, Coughlin S, Kreikemeier R, Heldt K, Bruzoni M, Lerner G. Low doses of pamidronate to treat osteopenia in children with severe cerebral palsy: a pilot study. Dev Med Child Neurol. 2006;48:709-12. doi:10.1017/S0012162206001526.
  • 31. Ballard T, Chargui S. Pamidronate. In: StatPearls. Treasure Island (FL): StatPearls Publishing; 2023. Available from: https://www.ncbi.nlm.nih.gov/books/NBK551673/
  • 32. Papapetrou PD. Bisphosphonate-associated adverse events. Hormones (Athens). 2009;8(2):96-110. doi:10.14310/horm.2002.1226.

Efficacy and Safety of Intravenous Pamidronate Treatment in Children and Adolescents

Year 2026, Volume: 16 Issue: 1, 1 - 7, 23.01.2026

Abstract

Aim: Pamidronate is the preferred treatment for osteoporosis and hypercalcemia in children. This study examined its safety and effectiveness.
Material and Methods: Patients treated with pamidronate for osteoporosis and hypercalcemia were included in the study. Osteoporosis patients received 1 mg/kg/day every three months for three days, while hypercalcemia patients received a single 1 mg/kg dose. Evaluations of height, weight, body mass index, bone mineral density (BMD), fractures, biochemical data, and complications were performed before and at least six months after treatment.
Results: Of the 31 patients included in the study, 21 (67.7%) were male and 10 (32.3%) were female, with a mean age of 12.20 ±4.31 years. Secondary osteoporosis was diagnosed in 20 (64.5%) patients, primary osteoporosis associated with osteogenesis imperfecta in seven (22.6%) patients, and hypercalcemia in four (12.9%) patients. The average follow-up period was 11.26 ±4.16 months. The mean BMD before treatment in patients treated with pamidronate for osteoporosis was -3.53 ±1.43, while after treatment it was -1.42 ±1.81 (p<0.001). When comparing the pre- and post-treatment periods, a decrease was observed in the total number of fractures, the number of vertebral fractures, and the number of fractures occurring more than once per year (p<0.001). Complications were observed in six (19,3%) patients but all these side effects were temporary.
Conclusion: Pamidronate treatment was administered at varying doses and for differing durations to patients from multiple groups, with no significant differences in efficacy or safety observed. The low complication rates reported are consistent with the literature and substantiate the safety of the treatment.

Ethical Statement

This study was performed in accordance with the Declaration of Helsinki and approved by the Institutional Review Board (IRB) of Ankara Bilkent City Hospital (IRB No. E2-22-1931).

References

  • 1. Arundel P, Bishop N. Primary osteoporosis. Endocr Dev. 2015;28:162-75. doi:10.1159/000381037.
  • 2. Korula S, Titmuss AT, Biggin A, Munns CF. A practical approach to children with recurrent fractures. Endocr Dev. 2015;28:210-25. doi:10.1159/000381047.
  • 3. Shuhart CR, Yeap SS, Anderson PA, Jankowski LG, Lewiecki EM, Morse LR, et al. Executive summary of the 2019 ISCD position development conference on monitoring treatment, DXA cross-calibration and least significant change, spinal cord injury, peri-prosthetic and orthopedic bone health, transgender medicine, and pediatrics. J Clin Densitom. 2019;22:453-71. doi:10.1016/j.jocd.2019.07.001.
  • 4. Russell RG. Bisphosphonates: mode of action and pharmacology. Pediatrics. 2007;119(suppl 2):150-62. doi:10.1542/peds.2006-2023H.
  • 5. Camacho PM, Petak SM, Binkley N, Diab DL, Eldeiry LS, Farooki A, et al. American association of clinical endocrinologists/american college of endocrinology clinical practice guidelines for the diagnosis and treatment of postmenopausal osteoporosis-2020 update. Endocr Pract. 2020;26(suppl 1):1-46. doi:10.4158/GL-2020-0524SUPPL.
  • 6. Bachrach LK, Ward LM. Clinical review 1: bisphosphonate use in childhood osteoporosis. J Clin Endocrinol Metab. 2009;94(2):400-9. doi:10.1210/jc.2008-1531.
  • 7. Jin HY, Noh ES, Hwang IT. Diagnosis and management of osteoporosis in children and adolescents. The Ewha Medical Journal. 2023;46(4):e18. doi:10.12771/emj.2023.e18
  • 8. Simm PJ, Biggin A, Zacharin MR, Rodda CP, Tham E, Siafarikas A, et al. APEG bone mineral working group. Consensus guidelines on the use of bisphosphonate therapy in children and adolescents. J Paediatr Child Health. 2018;54(3):223-33. doi:10.1111/jpc.13768.
  • 9. Nussbaum SR, Younger J, Vandepol CJ, Gagel RF, Zubler MA, Chapman R, et al. Single-dose intravenous therapy with pamidronate for the treatment of hypercalcemia of malignancy: comparison of 30-, 60-, and 90-mg dosages. Am J Med. 1993;95:297-304. doi:10.1016/0002-9343(93)90282-t.
  • 10. Sternlicht H, Glezerman IG. Hypercalcemia of malignancy and new treatment options. Ther Clin Risk Manag. 2015;11:1779-88. doi:10.2147/TCRM.S83681.
  • 11. Munns CF, Shaw N, Kiely M, Specker BL, Thacher TD, Ozono K, et al. Global consensus recommendations on prevention and management of nutritional rickets. Horm Res Paediatr. 2016;85:83-106. doi:10.1159/000443136.
  • 12. Demir K, Konakçı E, Özkaya G, Kasap Demir B, Özen S, Aydın M, et al. New features for child metrics: further growth references and blood pressure calculations. J Clin Res Pediatr Endocrinol. 2020;12:125-29. doi:10.4274/jcrpe.galenos.2019.2019.0127.
  • 13. Martinez-Soto T, Pacaud D, Stephure D, Trussell R, Huang C. Treatment of symptomatic osteoporosis in children: a comparison of two pamidronate dosage regimens. J Pediatr Endocrinol Metab. 2011;24:271-4. doi:10.1515/jpem.2011.182.
  • 14. Dwan K, Phillipi CA, Steiner RD, Basel D. Bisphosphonate therapy for osteogenesis imperfecta. Cochrane Database Syst Rev. 2016;10:CD005088. doi:10.1002/14651858.CD005088.pub4.
  • 15. Szczepaniak-Kubat A, Kurnatowska O, Jakubowska-Pietkiewicz E, Chlebna-Sokół D. Assessment of quality of life of parents of children with osteogenesis imperfecta. Adv Clin Exp Med. 2012;21(1):99-104.
  • 16. Marginean O, Tamasanu RC, Mang N, Mozos I, Brad GF. Therapy with pamidronate in children with osteogenesis imperfecta. Drug Des Devel Ther. 2017;11:2507-15. doi:10.2147/DDDT.S141075.
  • 17. Kok DH, Sakkers RJ, Janse AJ, Pruijs HE, Verbout AJ, Castelein RM, et al. Quality of life in children with osteogenesis imperfecta treated with oral bisphosphonates (Olpadronate): a 2-year randomized placebo-controlled trial. Eur J Pediatr. 2007;166:1155-61. doi:10.1007/s00431-006-0399-2.
  • 18. Lee M, Kwon A, Song K, Lee HI, Choi HS, Suh J, et al. Effectiveness and safety of pamidronate treatment in nonambulatory children with low bone mineral density. Ann Pediatr Endocrinol Metab. 2024;29:46-53. doi:10.6065/apem.2346028.014.
  • 19. Juszczak B, Sułko J. Patient-reported effectiveness and safety of pamidronate in NSAIDs-refractory chronic recurrent multifocal osteomyelitis in children. Rheumatol Int. 2022;42:699-706. doi:10.1007/s00296-021-04886-4.
  • 20. Alcausin MB, Briody J, Pacey V, Ault J, McQuade M, Bridge C, et al. Intravenous pamidronate treatment in children with moderate-to-severe osteogenesis imperfecta started under three years of age. Horm Res Paediatr. 2013;79:333-40. doi:10.1159/000351374.
  • 21. Shi CG, Zhang Y, Yuan W. Efficacy of bisphosphonates on bone mineral density and fracture rate in patients with osteogenesis imperfecta: a systematic review and meta-analysis. Am J Ther. 2016;23:894-904. doi:10.1097/MJT.0000000000000236.
  • 22. Ward LM. A practical guide to the diagnosis and management of osteoporosis in childhood and adolescence. Front Endocrinol (Lausanne). 2024;25:1266986. doi:10.3389/fendo.2023.1266986.
  • 23. Chilbule SK, Madhuri V. Complications of pamidronate therapy in paediatric osteoporosis. J Child Orthop. 2012;6:37-43. doi:10.1007/s11832-012-0383-5.
  • 24. Sees JP, Sitoula P, Dabney K, Holmes L Jr, Rogers KJ, Kecskemethy HH, et al. Pamidronate treatment to prevent reoccurring fractures in children with cerebral palsy. J Pediatr Orthop. 2016;36:193-7. doi:10.1097/BPO.0000000000000421.
  • 25. McNeilly JD, Boal R, Shaikh MG, Ahmed SF. Frequency and aetiology of hypercalcaemia. Arch Dis Child. 2016;101:344-7. doi:10.1136/archdischild-2015-309029.
  • 26. Stokes VJ, Nielsen MF, Hannan FM, Thakker RV. Hypercalcemic disorders in children. J Bone Miner Res. 2017;32:2157-70. doi:10.1002/jbmr.3296.
  • 27. Bharill S, Wu M. Hypocalcemia and hypercalcemia in children. Pediatr Rev. 2023;44:533-6. doi:10.1542/pir.2022-005578.
  • 28. Davies JH, Shaw NJ. Investigation and management of hypercalcaemia in children. Arch Dis Child. 2012;97:533-8. doi:10.1136/archdischild-2011-301284.
  • 29. Ward L, Tricco AC, Phuong P, Cranney A, Barrowman N, Gaboury I, et al. Bisphosphonate therapy for children and adolescents with secondary osteoporosis. Cochrane Database Syst Rev. 2007;2007(4):CD005324. doi:10.1002/14651858.CD005324.pub2.
  • 30. Plotkin H, Coughlin S, Kreikemeier R, Heldt K, Bruzoni M, Lerner G. Low doses of pamidronate to treat osteopenia in children with severe cerebral palsy: a pilot study. Dev Med Child Neurol. 2006;48:709-12. doi:10.1017/S0012162206001526.
  • 31. Ballard T, Chargui S. Pamidronate. In: StatPearls. Treasure Island (FL): StatPearls Publishing; 2023. Available from: https://www.ncbi.nlm.nih.gov/books/NBK551673/
  • 32. Papapetrou PD. Bisphosphonate-associated adverse events. Hormones (Athens). 2009;8(2):96-110. doi:10.14310/horm.2002.1226.
There are 32 citations in total.

Details

Primary Language English
Subjects Clinical Sciences (Other)
Journal Section Research Article
Authors

Banu Turhan 0000-0001-8474-6835

Gönül Büyükyılmaz 0000-0001-7888-7295

Keziban Toksoy Adıgüzel 0000-0003-1606-4781

Emre Özer 0000-0001-8475-9070

Gönül Yardımcı 0000-0002-4656-1490

Pınar Kocaay 0000-0003-0850-0360

Derya Tepe 0000-0002-2935-3008

Fatih Gurbuz 0000-0003-2160-9838

Mehmet Boyraz 0000-0002-4289-9128

Submission Date August 9, 2025
Acceptance Date October 10, 2025
Publication Date January 23, 2026
Published in Issue Year 2026 Volume: 16 Issue: 1

Cite

Vancouver Turhan B, Büyükyılmaz G, Toksoy Adıgüzel K, Özer E, Yardımcı G, Kocaay P, et al. Efficacy and Safety of Intravenous Pamidronate Treatment in Children and Adolescents. VHS. 2026;16(1):1-7.