Efficacy and safety of denosumab treatment in a prepubertal patient with cherubism

Haruka Kawamura 1 , Satoshi Watanabehttp://orcid.org/https://orcid.org/0000-0003-1878-1717 1 , Takashi I 2 , Izumi Asahina 2 , Hiroyuki Moriuchi 1 ,  and Sumito Dateki 1
  • 1 Department of Pediatrics, Nagasaki University Hospital, Nagasaki, Japan
  • 2 Department of Oral and Maxillofacial Surgery, Nagasaki University Hospital, Nagasaki, Japan
Haruka Kawamura, Satoshi WatanabeORCID iD: https://orcid.org/0000-0003-1878-1717, Takashi I
  • Department of Oral and Maxillofacial Surgery, Nagasaki University Hospital, Nagasaki, Japan
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, Izumi Asahina
  • Department of Oral and Maxillofacial Surgery, Nagasaki University Hospital, Nagasaki, Japan
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  • degruyter.comGoogle Scholar
, Hiroyuki Moriuchi and Sumito Dateki

Abstract

Background

Denosumab is an inhibitor of receptor activator of nuclear factor kappa-B ligand, which strongly suppresses osteoclasts. Cherubism is a rare autosomal dominant disorder characterized by symmetrical swelling of the jaws, in which the bone is replaced by a fibrous granuloma containing osteoclast-like giant cells.

Case presentation

We report the efficacy and safety of denosumab treatment in a prepubertal boy with progressive cherubism. The treatment consisting of eight subcutaneous denosumab injections (120 mg/dose) in 6 months not only suppressed the expansion of the osteolytic lesions but also dramatically ossified them. However, a transiently decreased growth rate and rebounded asymptomatic hypercalcemia were associated with the treatment.

Conclusions

The present case demonstrated the therapeutic potential of denosumab for treatment of cherubism, although adverse effects, especially those on childhood growth, remain obscure. Further studies are needed to establish a safe and effective protocol for denosumab treatment of children.

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The Journal of Pediatric Endocrinology and Metabolism (JPEM) is the only international journal dedicated exclusively to endocrinology in the neonatal, pediatric and adolescent age groups, and publishes the results of clinical investigations in pediatric endocrinology and basic research. JPEM publishes Review Articles, Original Research, Case Reports, Short Communications and Letters to the Editor.

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