The effect of enzyme replacement therapy on bone crisis and bone pain in patients with type 1 Gaucher disease

Clin Genet. 2007 Mar;71(3):205-11. doi: 10.1111/j.1399-0004.2007.00769.x.

Abstract

The effect of enzyme replacement therapy (ERT) on bone crisis and bone pain was investigated in patients with Gaucher disease (GD) type 1 followed over 4 years. Data from the International Collaborative Gaucher Group Gaucher Registry were used. Only patients with bone crisis and/or bone pain data for 1 year prior to ERT, and for each of 3 years after the start of ERT, were included. Bone crises were reported in 17% of patients during the year before starting ERT. The frequencies of bone crises decreased to 5%, <1% and 3% for 1, 2, and 3 years after initiation of treatment, respectively (p < 0.0001). Bone pain followed a similar pattern of response. Bone pain was reported in 49% of patients the year before treatment and decreased to 30% in the first year, 29% in the second year, and 30% in the third year of ERT (p < 0.0001). ERT is associated with a reduction in bone crisis and bone pain in patients with GD type 1 . This study shows that significant improvements in symptoms of skeletal disease are achievable clinical outcomes and treatment goals in GD type 1.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Bone Diseases / drug therapy*
  • Bone Diseases / etiology
  • Bone and Bones / drug effects
  • Bone and Bones / pathology
  • Child
  • Female
  • Follow-Up Studies
  • Gaucher Disease / complications
  • Gaucher Disease / drug therapy*
  • Glucosylceramidase / deficiency
  • Glucosylceramidase / therapeutic use*
  • Humans
  • Male
  • Pain / drug therapy
  • Registries
  • Treatment Outcome

Substances

  • Glucosylceramidase