Camurati-Engelmann disease--a rare cause of bone pain

Conn Med. 2012 Jan;76(1):33-7.

Abstract

Objective: Camurati-Engelmann disease or progressive diaphyseal dysplasia is a rare autosomal dominant disease characterized by cortical thickening of the long-bone diaphyses accompanied by waddling gait, muscular weakness, hearing loss and chronic skeletal pain. We describe two cases of Camurati-Engelmann disease with differing presentations and review of the literature on several therapeutic options.

Methods: We present two cases of Camurati-Engelmann disease which responded to different medical therapies. Various diagnostic tests including radiographs, bone scan and genetic analysis are also described.

Results: These cases responded differently to the treatment options and provide an insight into the variable response of the disease.

Conclusion: Diagnosis of this disorder is based on the clinical history, family history, clinical examination and imaging results. Recently genetic testing has become available for TGF-beta1 mutation Several therapeutic agents including biphosphonates, NSAIDs, prednisone and losartan have been described as therapeutic options with mixed results, as described in our cases.

Publication types

  • Case Reports

MeSH terms

  • Adult
  • Angiotensin II Type 1 Receptor Blockers / therapeutic use
  • Bone Density Conservation Agents / therapeutic use
  • Camurati-Engelmann Syndrome / complications
  • Camurati-Engelmann Syndrome / diagnosis*
  • Camurati-Engelmann Syndrome / drug therapy
  • Camurati-Engelmann Syndrome / genetics
  • Female
  • Glucocorticoids / therapeutic use
  • Humans
  • Male
  • Middle Aged
  • Mutation
  • Pain / drug therapy*
  • Pain / etiology
  • Pain / physiopathology
  • Transforming Growth Factor beta1 / genetics

Substances

  • Angiotensin II Type 1 Receptor Blockers
  • Bone Density Conservation Agents
  • Glucocorticoids
  • Transforming Growth Factor beta1