Pharmacokinetics of IGF-1 in PAPP-A2-Deficient Patients, Growth Response, and Effects on Glucose and Bone Density

What Was Tested

This clinical trial paper looks at IGF-1 LR3.

The researchers describe the growth response and effects on glucose metabolism and bone mineral density (BMD) after 1 year of rhIGF-1 therapy.

Why It Was Studied

The pregnancy-associated plasma protein A2 (PAPP-A2) cleaves insulinlike growth factor binding proteins 3 and 5, releasing free insulinlike growth factor 1 (IGF-1).

Homozygous mutations in PAPP-A2 result in growth failure with elevated total but low free IGF-1.

Who Took Part

The age groups tagged for this paper are children, teenagers, young adults and adults, both men and women.

Three affected siblings, their heterozygous parents, and two healthy controls participated.

The two younger siblings were started on rhIGF-1 treatment.

How the Study Was Run

The researchers set it up as a pilot study.

It is described as a pilot, a small early test of whether a larger IGF-1 LR3 study would be practical.

A time span of 1 year appears in the methods.

An oral glucose tolerance test and dual-energy X-ray absorptiometry scans were obtained at baseline and after 1 year of treatment.

What Was Measured

Topics PubMed links to this paper include Absorptiometry, Photon, Blood Glucose, Body Composition, Bone Density, Family, Glucose Tolerance Test and Growth.

What This Means in Plain Language

The paper is a pilot study of IGF-1 LR3 over 1 year.

Its findings take up roughly 7 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2017 (The Journal of clinical endocrinology and metabolism), a journal based in United States.

More context: the IGF-1 LR3 compound page.