A Systematic Review and Meta Analysis of Non-Randomized Interventional Studies on the Pamidronate Treatment Efficacy for Patients with Bone Fibrous Dysplasia

Authors

  • Nicolas Daniel Widjanarko Faculty of Medicine and Health Sciences, Atma Jaya Catholic University of Indonesia, Indonesia
  • Anthony Ekaputra Faculty of Medicine and Health Sciences, Atma Jaya Catholic University of Indonesia, Indonesia
  • Jessica Felicia Ang Faculty of Medicine and Health Sciences, Atma Jaya Catholic University of Indonesia, Indonesia

DOI:

https://doi.org/10.33192/smj.v75i12.265453

Keywords:

Fibrous Dysplasia, Pamidronate, Bone Pain, Serum Alkaline Phosphatase, Bone Mineral Density

Abstract

Objective: Pamidronate is one of the main therapies for Fibrous Dysplasia (FD), with documented enhancements in patients' clinical characteristics. Nevertheless, its usage has yielded inconclusive results. Therefore, this review aimed to investigate pamidronate’s impact on several clinical and biochemical outcomes in FD patients.

Materials and Methods: This review was conducted under the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines. MEDLINE, ProQuest, Wiley, and EBSCO search databases were used to search the literature. Risk of Bias In Non-Randomized Studies of Interventions (ROBINS-I) was applied for quality assessment of the included studies and Review Manager (RevMan) 5.4 was employed in performing the meta-analysis.

Results: There are eight and seven studies used in the meta-analysis and systematic review, respectively. The results showed there are two studies with a low risk of bias and six with a moderate category. All papers included in this meta-analysis showed significant differences in the reduction of bone pain (p<0.00001) and serum alkaline phosphatase (SAP) (p=0.04) after pamidronate treatment compared to the before-treatment groups.

Conclusion: The findings of this study indicated that pamidronate therapy had been proven to significantly reduce bone pain and increase SAP in FD patients. However, trials with more age-specific samples and a lower risk of bias should be carried out to determine the statistical significance of overall results.

References

Chapurlat RD, Meunier PJ. Fibrous dysplasia of bone. Baillieres Best Pract Res Clin Rheumatol. 2000;14(2):385-98.

Lietman SA, Levine MA. Fibrous dysplasia. Pediatr Endocrinol Rev PER. 2013;10 Suppl 2:389-96.

Berglund JA, Tella SH, Tuthill KF, Kim L, Guthrie LC, Paul SM, et al. Scoliosis in Fibrous Dysplasia/McCune-Albright Syndrome: Factors Associated With Curve Progression and Effects of Bisphosphonates. J Bone Miner Res Off J Am Soc Bone Miner Res. 2018;33(9):1641-8.

Collins MT, Kushner H, Reynolds JC, Chebli C, Kelly MH, Gupta A, et al. An instrument to measure skeletal burden and predict functional outcome in fibrous dysplasia of bone. J Bone Miner Res Off J Am Soc Bone Miner Res. 2005;20(2):219-26.

Rudge ES, Chan AHY, Leeper FJ. Prodrugs of pyrophosphates and bisphosphonates: disguising phosphorus oxyanions. RSC Med Chem. 2022;13(4):375-91.

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.

Drake MT, Clarke BL, Khosla S. Bisphosphonates: Mechanism of Action and Role in Clinical Practice. Mayo Clin Proc Mayo Clin. 2008;83(9):1032-45.

Yoon JH, Choi Y, Lee Y, Yoo HW, Choi JH. Efficacy and safety of intravenous pamidronate infusion for treating osteoporosis in children and adolescents. Ann Pediatr Endocrinol Metab. 2021;26(2):105-11.

Brendan Chan, Margaret Zacharin. Pamidronate Treatment of Polyostotic Fibrous Dysplasia: Failure to Prevent Expansion of Dysplastic Lesions During Childhood. J Pediatr Endocrinol Metab. 2006;19(1):75-80.

Parisi MS, Oliveri B. Long-term pamidronate treatment of polyostotic fibrous dysplasia of bone: A case series in young adults. Curr Ther Res Clin Exp. 2009;70(2):161-72.

Liu J, Zhao C, Liu B, Liu H, Wang L. Analgesia and curative effect of pamidronate disodium combined with chemotherapy on elderly patients with advanced metastatic bone cancer. Oncol Lett. 2019;18(1):771-5.

Ren HY, Sun LL, Li HY, Ye ZM. Prognostic Significance of Serum Alkaline Phosphatase Level in Osteosarcoma: A Meta-Analysis of Published Data. BioMed Res Int. 2015;2015:160835.

Park BY, Cheon YW, Kim YO, Pae NS, Lee WJ. Prognosis for craniofacial fibrous dysplasia after incomplete resection: age and serum alkaline phosphatase. Int J Oral Maxillofac Surg. 2010;39(3):221-6.

Chapurlat RD, Hugueny P, Delmas PD, Meunier PJ. Treatment of fibrous dysplasia of bone with intravenous pamidronate: long-term effectiveness and evaluation of predictors of response to treatment. Bone. 2004;35(1):235-42.

Page MJ, McKenzie JE, Bossuyt PM, Boutron I, Hoffmann TC, Mulrow CD, et al. The PRISMA 2020 statement: an updated guideline for reporting systematic reviews. Syst Rev. 2021;10:89.

Standard Deviation Calculator [Internet]. [cited 2023 Sep 2]. Available from: https://www.calculator.net/standard-deviation-calculator.html

Chapurlat RD, Delmas PD, Liens D, Meunier PJ. Long-term effects of intravenous pamidronate in fibrous dysplasia of bone. J Bone Miner Res Off J Am Soc Bone Miner Res. 1997;12(10):1746-52.

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.

Isaia GC, Lala R, Defilippi C, Matarazzo P, Andreo M, Roggia C, et al. Bone turnover in children and adolescents with McCune-Albright syndrome treated with pamidronate for bone fibrous dysplasia. Calcif Tissue Int. 2002;71(2):121-8.

Lala R, Matarazzo P, Andreo M, Marzari D, Bellone J, Corrias A, et al. Bisphosphonate treatment of bone fibrous dysplasia in McCune-Albright syndrome. J Pediatr Endocrinol Metab JPEM. 2006;19 Suppl 2:583-93.

Lala R, Matarazzo P, Bertelloni S, Buzi F, Rigon F, de Sanctis C. Pamidronate treatment of bone fibrous dysplasia in nine children with McCune-Albright syndrome. Acta Paediatr 2000;89(2):188-93.

Zacharin M, O’Sullivan M. Intravenous pamidronate treatment of polyostotic fibrous dysplasia associated with the McCune Albright syndrome. J Pediatr. 2000;137(3):403-9.

Igelström E, Campbell M, Craig P, Katikireddi SV. Cochrane’s risk of bias tool for non-randomized studies (ROBINS-I) is frequently misapplied: A methodological systematic review. J Clin Epidemiol. 2021;140:22-32.

Hartley I, Zhadina M, Collins MT, Boyce AM. Fibrous Dysplasia of Bone and McCune-Albright Syndrome: a Bench to Bedside Review. Calcif Tissue Int. 2019;104(5):517-29.

Spencer T, Pan KS, Collins MT, Boyce AM. The clinical spectrum of McCune-Albright syndrome and its management. Horm Res Paediatr. 2019;92(6):347-56.

Laine J, Kadado A, James C, Novotny S. The Role of Bisphophonates in Pediatric Orthopaedics: What Do We Know After 50 Years? Current Concept Review. J Pediatr Orthop Soc N Am [Internet]. 2019 Nov 1 [cited 2023 Sep 1];1(1). Available from: https://www.jposna.org/index.php/jposna/article/view/33

Parisi MS, Oliveri MB, Mautalen CA. Bone mineral density response to long-term bisphosphonate therapy in fibrous dysplasia. J Clin Densitom Off J Int Soc Clin Densitom. 2001;4(2):167-72.

Lee JM, Kim JE, Bae SH, Hah JO. Efficacy of pamidronate in children with low bone mineral density during and after chemotherapy for acute lymphoblastic leukemia and non-Hodgkin lymphoma. Blood Res. 2013;48(2):99-106.

Chapurlat RD, Gensburger D, Jimenez-Andrade JM, Ghilardi JR, Kelly M, Mantyh P. Pathophysiology and medical treatment of pain in fibrous dysplasia of bone. Orphanet J Rare Dis. 2012;7(1):S3.

Majoor BCJ, Traunmueller E, Maurer-Ertl W, Appelman-Dijkstra NM, Fink A, Liegl B, et al. Pain in fibrous dysplasia: relationship with anatomical and clinical features. Acta Orthop. 2019;90(4):401-5.

Shu J, Tan A, Li Y, Huang H, Yang J. The correlation between serum total alkaline phosphatase and bone mineral density in young adults. BMC Musculoskelet Disord. 2022;23:467.

Wang J, Du Z, Li D, Yang R, XiaodongTang, Yan T, et al. Increasing serum alkaline phosphatase is associated with bone deformity progression for patients with polyostotic fibrous dysplasia. J Orthop Surg. 2020;15(1):583.

Ma J, Liang L, Gu B, Zhang H, Wen W, Liu H. A retrospective study on craniofacial fibrous dysplasia: preoperative serum alkaline phosphatase as a prognostic marker? J Cranio-Maxillo-fac Surg Off Publ Eur Assoc Cranio-Maxillo-fac Surg. 2013;41(7):644-7.

Vaisman DN, McCarthy AD, Cortizo AM. Bone-specific alkaline phosphatase activity is inhibited by bisphosphonates: role of divalent cations. Biol Trace Elem Res. 2005;104(2):131-40.

Greenblatt MB, Tsai JN, Wein MN. Bone Turnover Markers in the Diagnosis and Monitoring of Metabolic Bone Disease. Clin Chem. 2017;63(2):464-74.

Chilbule SK, Madhuri V. Complications of pamidronate therapy in paediatric osteoporosis. J Child Orthop. 2012;6(1):37-43.

Juszczak B, Sułko J. Patient-reported effectiveness and safety of Pamidronate in NSAIDs-refractory chronic recurrent multifocal osteomyelitis in children. Rheumatol Int. 2022;42(4):699-706.

Hart ES, Kelly MH, Brillante B, Chen CC, Ziran N, Lee JS, et al. Onset, progression, and plateau of skeletal lesions in fibrous dysplasia and the relationship to functional outcome. J Bone Miner Res Off J Am Soc Bone Miner Res. 2007;22(9):1468-74.

Lee SE, Lee EH, Park H, Sung JY, Lee HW, Kang SY, et al. The diagnostic utility of the GNAS mutation in patients with fibrous dysplasia: meta-analysis of 168 sporadic cases. Hum Pathol. 2012;43(8):1234-42.

Elli FM, de Sanctis L, Bergallo M, Maffini MA, Pirelli A, Galliano I, et al. Improved Molecular Diagnosis of McCune–Albright Syndrome and Bone Fibrous Dysplasia by Digital PCR. Front Genet. 2019;10:862.

Kushchayeva YS, Kushchayev SV, Glushko TY, Tella SH, Teytelboym OM, Collins MT, et al. Fibrous dysplasia for radiologists: beyond ground glass bone matrix. Insights Imaging. 2018;9(6):1035-56.

Bhadada SK, Bhansali A, Das S, Singh R, Sen R, Agarwal A, et al. Fibrous dysplasia & McCune-Albright syndrome: An experience from a tertiary care centre in north India. Indian J Med Res. 2011;133(5):504-9.

Published

01-12-2023

How to Cite

Widjanarko, N. D., Ekaputra, A., & Ang, J. F. (2023). A Systematic Review and Meta Analysis of Non-Randomized Interventional Studies on the Pamidronate Treatment Efficacy for Patients with Bone Fibrous Dysplasia. Siriraj Medical Journal, 75(12), 851–863. https://doi.org/10.33192/smj.v75i12.265453

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