Severe primary IGF-1 deficiency

Severe primary IGF-1 (insulin-like growth factor 1, a protein that mediates the effects of growth hormone) deficiency causes delayed growth in children and adolescents, as well as a range of anatomical, morphological and physiological disorders. Learn more about the condition, its diagnosis and the different treatment options available.

Growth hormone treatment is not effective for children with primary IGF-1 deficiency, because the GH/IGF-1 pathway is blocked.

Cases of PIGFD can be classed as:

  • anomalies of the GH/IGF-1 pathway that can be treated with recombinant IGF-1, or
  • anomalies outside this pathway, including anomalies in the IGF-1 receptor, which do not respond to this treatment.

Type 1 human insulin-like growth factor derived from recombinant DNA for children with severe primary IGF-1 deficiency caused by anomalies in the GH/IGF-1 pathway is a long term treatment option for delayed growth caused by SPIGFD. It must be prescribed by a specialist when not counter-indicated. The treatment is administered by daily subcutaneous injection and the efficacy is evaluated based on the speed of growth. The dosage can be increased gradually, according to how well the treatment is tolerated by the patient.

Last update 13/04/2017