Effectiveness and safety of rhIGF1 therapy in patients with or without Laron syndrome.

ABSTRACT:

Objective

The European Increlex® Growth Forum Database Registry monitors the effectiveness and safety of recombinant human insulin-like growth factor-1 (rhIGF1; mecasermin, Increlex®) therapy in patients with severe primary IGF1 deficiency (SPIGFD). We present data from patients with and without a reported genetic diagnosis of Laron syndrome (LS).

Design

Ongoing, open-label, observational registry (NCT00903110).

Methods

Children and adolescents receiving rhIGF1 therapy from 10 European countries were enrolled in 2008-2017 (n = 242). The treatment-naïve/prepubertal (NPP) cohort (n = 138) was divided into subgroups based on reported genetic diagnosis of LS (n = 21) or non-LS (n = 117). Multivariate analysis of the NPP-non-LS subgroup was conducted to identify factors pr

SUBMITTER: Bang P 

PROVIDER: S-EPMC7849377 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature