Primary severe IGF1 deficiency

IGF1 deficiency (IGFD) can be caused by various conditions including growth hormone (GH) deficiency, nutritional deficiencies and chronic inflammatory diseases. However, IGFD may occur as a primary disorder, with no identifiable cause.  Furthermore, the prevalence of severe primary IGFD remains unclear.

Treatment with recombinant human form of IGF1 (rhIGF1) is now available. However, uncertainty remains regarding the number of children likely to benefit from rhIGF1 therapy.

Teissier et al. conducted an observation study in a prospective cohort, with the objective to characterize and assess the prevalence of severe primary IGFD in a large cohort of patients evaluated for short stature at a paediatric endocrinology unit in France.

They found that the prevalence of severe primary IGFD, defined using criteria for rhIGF1 treatment, was only 1.2% in a vast cohort of children with short stature, and that only 2% of patients were eligible for rhIGF1 therapy. However, the study noted the efficacy of rhIGF1 in carefully selected patients and concludes that it remains important to consider the use of rhIGF1 as no other effective treatments are available.

Read the full article at Teissier et al. (2014) European Journal of Endocrinology 170 (6) 847-854; DOI:10.1530/EJE-14-0071