A randomized, double blind, placebo-controlled trial on safety and efficacy of recombinant human insulin-like growth factor-I in children with growth hormone receptor deficiency

Jaime Guevara-Aguirre, Oswaldo Vasconez, Victor Martinez, Ana Lucia Martinez, Arlan L. Rosenbloom, Frank B. Diamond, Sharron E. Gargosky, Leni Nonoshita, Ronald (Ron) Rosenfeld

Research output: Contribution to journalArticle

123 Citations (Scopus)

Abstract

GH insensitivity due to GH receptor deficiency is a rare autosomal recessive condition, characterized by deletions or mutations of the GH receptor gene. Patients are refractory to both endogenous and exogenous GH, resulting in severe growth retardation. Therapy with recombinant human insulin-like growth factor-I (rhIGF-I) can bypass the defect in the GH receptor and potentially stimulate growth. We previously identified a genetically homogeneous group of patients in southern Ecuador, thus providing a patient base for a controlled clinical trial of rhIGF-I therapy. Seventeen prepubertal patients were entered in a randomized, double blind, placebo- controlled trial. Subjects received either a 12-month course of rhIGF-I (120 μg/kg, sc, daily) or 6 months of placebo followed by 6 months of rhIGF-I. Subjects receiving rhIGF-I showed a significant increase in growth rate, which was sustained over the 1-yr course of therapy (from 2.9 ± 0.6 to 8.6 ± 0.4 cm/yr). Incidents of hypoglycemia were equal in frequency in the placebo and rhIGF-I groups. One recipient of rhIGF-I developed papilledema, which resolved spontaneously. rhIGF-I therapy did not alter serum IGF- binding protein-3 concentrations. rhIGF-I treatment is effective in stimulating skeletal growth in GH receptor deficiency. Although the therapy proved to be safe, the potent metabolic actions of rhIGF-I and the persistently low levels of serum IGF carrier protein necessitate continued careful observation for side-effects.

Original languageEnglish (US)
Pages (from-to)1393-1398
Number of pages6
JournalJournal of Clinical Endocrinology and Metabolism
Volume80
Issue number4
DOIs
StatePublished - Apr 1995

Fingerprint

Laron Syndrome
Somatotropin Receptors
Insulin-Like Growth Factor I
Placebos
Safety
Growth
Therapeutics
Ecuador
Papilledema
Insulin-Like Growth Factor Binding Protein 3
Sequence Deletion
Controlled Clinical Trials
Hypoglycemia
Refractory materials
Blood Proteins
Carrier Proteins
Genes
Observation

ASJC Scopus subject areas

  • Biochemistry
  • Endocrinology, Diabetes and Metabolism

Cite this

A randomized, double blind, placebo-controlled trial on safety and efficacy of recombinant human insulin-like growth factor-I in children with growth hormone receptor deficiency. / Guevara-Aguirre, Jaime; Vasconez, Oswaldo; Martinez, Victor; Martinez, Ana Lucia; Rosenbloom, Arlan L.; Diamond, Frank B.; Gargosky, Sharron E.; Nonoshita, Leni; Rosenfeld, Ronald (Ron).

In: Journal of Clinical Endocrinology and Metabolism, Vol. 80, No. 4, 04.1995, p. 1393-1398.

Research output: Contribution to journalArticle

Guevara-Aguirre, J, Vasconez, O, Martinez, V, Martinez, AL, Rosenbloom, AL, Diamond, FB, Gargosky, SE, Nonoshita, L & Rosenfeld, RR 1995, 'A randomized, double blind, placebo-controlled trial on safety and efficacy of recombinant human insulin-like growth factor-I in children with growth hormone receptor deficiency', Journal of Clinical Endocrinology and Metabolism, vol. 80, no. 4, pp. 1393-1398. https://doi.org/10.1210/jc.80.4.1393
Guevara-Aguirre, Jaime ; Vasconez, Oswaldo ; Martinez, Victor ; Martinez, Ana Lucia ; Rosenbloom, Arlan L. ; Diamond, Frank B. ; Gargosky, Sharron E. ; Nonoshita, Leni ; Rosenfeld, Ronald (Ron). / A randomized, double blind, placebo-controlled trial on safety and efficacy of recombinant human insulin-like growth factor-I in children with growth hormone receptor deficiency. In: Journal of Clinical Endocrinology and Metabolism. 1995 ; Vol. 80, No. 4. pp. 1393-1398.
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