Bisphosphonates for Paget's disease of bone in adults

Corral‐Gudino, Luis; Tan, Adrian; Pino-Montes, Javier del; Ralston, Stuart H.

doi:10.1002/14651858.cd004956.pub3

Cited by 39 publications

(54 citation statements)

References 70 publications

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“…In a recent Cochrane review, (66) no statistically significant difference was found in adverse effects with oral bisphosphonates compared with placebo (6 studies, 678 participants, risk…”

Section: Adverse Eventsmentioning

confidence: 96%

“…When the data were expressed as the proportion of patients whose SF36 physical summary score improved after treatment, the difference favored zoledronic acid (RR ¼ 1.30, 95% CI 1.18-1.42). (66) In an extension of the same study, (100) the mean change from baseline SF36 summary score favored zoledronic acid (mean difference 3.8, 95% CI 3.12-4.49). However, this analysis was not intention to treat, and was based on a selected group of patients who had normal total ALP values at the end of the core study.…”

Section: Quality Of Evidence Very Low Patient Values and Preferencesmentioning

confidence: 98%

“…(54,84) We have mainly focused on the use of bisphosphonates, which are currently considered to be the treatment of choice for PDB and which are the only agents that have been evaluated in randomized controlled trials. (66) Through the literature review, we identified studies of several osteoclast inhibitors that had been employed at some point in the treatment of PDB, including glucagon, (85) mithramycin, (86) actinomycin D, (87) and gallium nitrate. (88) These were not considered further because the guideline group felt they were of historical interest and were evaluated in uncontrolled studies with no comparator.…”

Section: Effects Of Drug Treatment In Paget's Diseasementioning

confidence: 99%

“…It has been shown that the decrease is greater with nitrogen-containing bisphosphonates as opposed to nonnitrogen-containing bisphosphonates, and that within the bisphosphonates, zoledronic acid is most effective at reducing total ALP. (66) Predicting the response of bone lesions Al Nofal and colleagues conducted a systematic review and meta-analysis of studies that compared changes in marker concentrations after bisphosphonate therapy with disease extent as assessed by quantitative radionuclide scintigraphy. (83) Decreases in bone ALP concentrations after treatment have been observed after treatment with various bisphosphonates.…”

Section: Costs and Use Of Resourcesmentioning

confidence: 99%

See 3 more Smart Citations

Diagnosis and Management of Paget's Disease of Bone in Adults: A Clinical Guideline

Ralston

Corral‐Gudino

Cooper

et al. 2019

Journal of Bone and Mineral Research

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133

169

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An evidence based clinical guideline for the diagnosis and management of Paget’s disease of Bone (PDB) was developed using GRADE methodology, by a Guideline Development Group (GDG) led by the Paget’s Association (UK). A systematic review of diagnostic tests, pharmacological and non-pharmacological treatment options was conducted which sought to address several key questions of clinical relevance. Twelve recommendations and five conditional recommendations were made but there was insufficient evidence to address eight of the questions posed. The following recommendations were identified as the most important. Radionuclide bone scans, in addition to targeted radiographs, are recommended as a means of fully and accurately defining the extent of the metabolically active disease in patients with PDB.Serum total alkaline phosphatase (ALP) is recommended as a first line biochemical screening test in combination with liver function tests in screening for the presence of metabolically active PDB.Bisphosphonates are recommended for the treatment of bone pain associated with Paget’s disease. Zoledronic acid is recommended as the bisphosphonate most likely to give a favourable pain response.Treatment aimed at improving symptoms is recommended over a treat-to-target strategy aimed at normalising total ALP in PDB.Total hip or knee replacements are recommended for patients with PDB who develop osteoarthritis in whom medical treatment is inadequate. There is insufficient information to recommend one type of surgical approach over another. The guideline was endorsed by the European Calcified Tissues Society, the International Osteoporosis Foundation, the American Society of Bone and Mineral Research, the Bone research Society (UK) and the British Geriatric Society. The GDG noted that there had been a lack of research on patient-focused clinical outcomes in PDB and identified several areas where further research was needed.

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“…In a recent Cochrane review, (66) no statistically significant difference was found in adverse effects with oral bisphosphonates compared with placebo (6 studies, 678 participants, risk…”

Section: Adverse Eventsmentioning

confidence: 96%

Section: Quality Of Evidence Very Low Patient Values and Preferencesmentioning

confidence: 98%

Section: Effects Of Drug Treatment In Paget's Diseasementioning

confidence: 99%

Section: Costs and Use Of Resourcesmentioning

confidence: 99%

See 2 more Smart Citations

Diagnosis and Management of Paget's Disease of Bone in Adults: A Clinical Guideline

Ralston

Corral‐Gudino

Cooper

et al. 2019

Journal of Bone and Mineral Research

Self Cite

133

169

View full text Add to dashboard Cite

show abstract

“…2 Since 1973, the pharmacological treatment of PDB contemplates the use of potent bisphosphonates that act by suppressing the excessive bone turnover from active disease to physiological range. 3,4 Genetically, PDB exhibits an autosomal dominant pattern of inheritance with incomplete penetrance. 5 To date, 28 different mutations in the SQSTM1 gene have been identified in 35% and 10% of individuals with familial and sporadic PDB, respectively, with the c.1215C>T, p.Pro392Leu mutation as the most common one.…”

Section: Introductionmentioning

confidence: 99%

ZNF687 mutations are frequently found in pagetic patients from South Italy: implication in the pathogenesis of Paget's disease of bone

et al. 2018

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Paget's disease of bone (PDB) is a skeletal disorder whose molecular basis is not fully elucidated. However, 10% of patients show a familial PDB and 35% of them carry mutations in the SQSTM1 gene. We recently reported a founder mutation (p.Pro937Arg) in the ZNF687 gene, underlying PDB complicated by giant cell tumor (GCT/PDB) and rarely occurring in PDB patients without neoplastic degeneration. Since 80% of Italian GCT/PDB patients derive from Avellino, we hypothesized that ZNF687 mutation rate was higher in this region than elsewhere. Interestingly, our molecular analysis on 30 PDB patients showed that 33% hosted ZNF687 mutations, with the p.Pro937Arg identified in 8 familial cases. Two novel ZNF687 mutations (p.Pro665Leu and p.Gln784Glu) were detected in 2 sporadic patients. Only 2 subjects were positive for the p.Pro392Leu mutation in SQSTM1. ZNF687-mutated patients showed a severe PDB, with a remarkable number of affected sites. in vitro studies revealed that the ZNF687-mutant osteoclasts appeared as giant sized with up to 150 nuclei, never described in PDB. Finally, we also confirmed the causality of the p.Pro937Arg mutation in 4 additional GCT/PDB cases deriving from the same geographic area, indicating that PDB and GCT/PDB represent 2 sides of the same coin.

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Synthesis of lesquerella‐based bisphosphonates

Boon,

Bamert,

Fayer

et al. 2023

J Americ Oil Chem Soc

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Hydroxy fatty acids (HFAs) have found wide use in today's market, ranging from industrial materials to pharmaceuticals. Castor oil, which is obtained from castor seeds, has served as a primary source of the most common HFA, ricinoleic acid, but also contains several undesirable compounds which pose severe health risks, the most notable being ricin, an unusually stable, toxic protein. A promising HFA alternative is lesquerella oil, an oil obtained from seeds of the Lesquerella fendleri species. Lesquerella oil is mainly comprised of lesquerolic acid, an HFA that is structurally similar to ricinoleic acid, the only difference being that lesquerolic acid possesses two additional methylene groups on the carboxyl end of the molecule. In addition, the bisphosphonate moiety has been shown to display interesting biological activities, primarily as osteoporosis drugs and anti‐cancer therapeutics. The synthesis of lesquerella‐based bisphosphonates, both an unsaturated and saturated series, have been produced in high yields and high purity and are reported here.

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Bisphosphonates for Paget's disease of bone in adults

Abstract: Trusted evidence. Informed decisions. Better health.

Cited by 39 publications

References 70 publications

Diagnosis and Management of Paget's Disease of Bone in Adults: A Clinical Guideline

Diagnosis and Management of Paget's Disease of Bone in Adults: A Clinical Guideline

ZNF687 mutations are frequently found in pagetic patients from South Italy: implication in the pathogenesis of Paget's disease of bone

Synthesis of lesquerella‐based bisphosphonates

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