Denosumab Therapy in Cherubism

Liles, Steele I; Hoppe, Ian C.; Arnold, Laura

doi:10.1177/10556656221113891

Cited by 4 publications

(3 citation statements)

References 12 publications

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“…[ 49,50 ] Most treatments were found to be well tolerated, and many of the known adverse effects [ 49,51‐56 ] were not observed, apart from a serious hypocalcemia and rebound hypercalcemia that occurred in three denosumab cases. [ 46,47,57 ] Although the drugs with positive outcomes mentioned in these incidental reports are promising, it is difficult to make a meaningful comparison with our results due to the lack of a comparative and systematic analysis. To select the ideal drug, dosing, and treatment protocol that considers both clinical outcomes and long‐term safety data in this vulnerable pediatric population, larger studies need to be designed.…”

Section: Discussionmentioning

confidence: 87%

“…[9,[33][34][35][36][37][38][39][40] Inhibitors of tumor necrosis factor α, produced by hyperactive macrophages, [37,38] were proposed but failed to improve cherubism in three pediatric cases. [41,42] Clinical outcomes after targeting the hyperactive osteoclasts in eight pediatric and two young adult cherubism cases by either calcineurin inhibitors (tacrolimus) [43] or receptor activator of NF-κB ligand inhibitors (denosumab) [44][45][46][47] were positive, while for bisphosphonates [16,42,44,48] results varied. An attempt to mitigate the protein product of the mutated SH3BP2 gene by a c-abl inhibitor (imatinib) demonstrated a partial regression of lesions in four pediatric cherubism cases.…”

Section: Discussionmentioning

confidence: 99%

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Efficacy and Toxicity of Calcitonin Treatment in Children with Cherubism: A Single-Center Cohort Study

Schreuder,

Meijer,

Cleven

et al. 2023

Journal of Bone and Mineral Research

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Cherubism is a rare autosomal dominant disease characterized by expansile osteolytic jawbone lesions. The effect and safety of off‐label calcitonin treatment during the progressive phase of the disease are not well described. In this retrospective study, we present data on the radiological response and adverse effects of subcutaneously administered calcitonin in a cohort of nine cherubism children (3 female, 6 male). Two of the nine patients underwent two separate treatment courses with a significant off‐treatment interval in between; therefore, a total of 11 treatment courses with a mean duration of 17.9 months (range <1–35, SD 10.8) were studied. To measure response, the cumulative volume of cherubism lesions was calculated from available three‐dimensional imaging. The primary outcome was the change in the volume of lesions during calcitonin treatment and only assessed for the eight treatment courses with a minimal duration of 6 months. A statistically significant reduction in the mean cumulative volume of lesions was seen regardless of treatment duration. Average volume reduction was highest in the first half year of treatment, with a gradual, ongoing reduction thereafter. For the secondary outcome, the change in the cumulative volume of lesions after treatment cessation was assessed for the seven treatment courses with follow‐up imaging available. After six of these seven treatment courses, the cumulative volume increased again but remained undoubtedly smaller than the initial volume at the start of therapy. Adverse effects were assessed for all 11 treatment courses and occurred in 73% of them. Most adverse effects were mild and low grade, with the most severe being one grade 3 symptomatic hypocalcemia requiring hospitalization and early treatment termination. Calcitonin treatment seems effective and tolerable in treating actively progressing cherubism in children. However, further research is required to better understand the pharmacological treatment of cherubism, including also other drugs, dosing, and protocols.This article is protected by copyright. All rights reserved.

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Section: Discussionmentioning

confidence: 87%

Section: Discussionmentioning

confidence: 99%

Efficacy and Toxicity of Calcitonin Treatment in Children with Cherubism: A Single-Center Cohort Study

Schreuder,

Meijer,

Cleven

et al. 2023

Journal of Bone and Mineral Research

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“…The last of these drugs, as reported in recent years, may be helpful in cases of cherubism resistant to treatment, as an alternative to surgical treatment. It can also be used after surgery to reduce the risk of recurrence [21,22].…”

Section: Introductionmentioning

confidence: 99%

Observations of spontaneous cherubism based on two cases and a literature review

Brodowski,

Pakla,

Myszka

et al. 2023

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Cherubism is a genetically determined illness characterized by osseus lesions of the facial part of the skull. X-ray and histopathological analysis of cherubism show it to be similar to fibrosis dysplasia, a brown tumour that occurs in the parathyroids or giant cells. Our paper describes 2 cases of cherubism. In each of them, X-ray examination, histopathological analysis and genetic tests led to a diagnosis. In this study, in the first case, there was no mutation in the SH3BP2 gene, which does not exclude the possibility that the mutation occurs in another gene. In the second case, in a patient diagnosed with cherubism after many years of observation and when clinical symptoms had worsened, genetic tests confirmed a mutation in exon 9 of the SH3BP2 gene. StreszczenieCherubizm to choroba uwarunkowana genetycznie, dotycząca kości części twarzowej czaszki. Schorzenie jest najczęściej diagnozowane u dzieci poniżej 5. roku życia. Obraz radiologiczny i histopatologiczny cherubizmu zbliżony jest do dysplazji włóknistej, guza brunatnego w nadczynności przytarczyc lub guzów olbrzymiokomórkowych. W artykule przedstawiono dwa przypadki cherubizmu. Oba przypadki rozpoznano na podstawie badania klinicznego potwierdzonego badaniami radiologicznymi, histopatologicznymi oraz testami genetycznymi. W niniejszej pracy w pierwszym przypadku nie stwierdzono mutacji genu SH3BP2, co nie wyklucza, że mutacja występuje w innym genie. W drugim przypadku u chorej, u której rozpoznanie cherubizm zostało ustalone po wieloletniej obserwacji i w momencie nasilenia objawów klinicznych, badania genetyczne potwierdziły mutację w egzonie 9 genu SH3BP2.

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Denosumab

2024

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Denosumab Therapy in Cherubism

Cited by 4 publications

References 12 publications

Efficacy and Toxicity of Calcitonin Treatment in Children with Cherubism: A Single-Center Cohort Study

Efficacy and Toxicity of Calcitonin Treatment in Children with Cherubism: A Single-Center Cohort Study

Observations of spontaneous cherubism based on two cases and a literature review

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