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J Clin Endocrinol Metab. 2013 Jun;98(6):2422-9. doi: 10.1210/jc.2013-1157. Epub 2013 Apr 4.

Macimorelin (AEZS-130)-stimulated growth hormone (GH) test: validation of a novel oral stimulation test for the diagnosis of adult GH deficiency.

Author information

1
Division of Endocrinology, Diabetes, and Metabolism, Michael E. DeBakey Veterans Affairs Medical Center, Baylor College of Medicine, 2002 Holcombe Boulevard, Building 109, Room 210, Houston, Texas 77030, USA. jgarcia1@bcm.edu

Abstract

CONTEXT:

In the absence of panhypopituitarism and low serum IGF-I levels, the diagnosis of adult GH deficiency (AGHD) requires confirmation with a GH stimulation test. Macimorelin is a novel, orally active ghrelin mimetic that stimulates GH secretion.

OBJECTIVE:

The objective of the study was to determine the diagnostic efficacy and safety of macimorelin in AGHD.

DESIGN:

This was a multicenter open-label study comparing the diagnostic accuracy of oral macimorelin with that of arginine+GHRH in AGHD patients and healthy, matched controls. After 43 AGHD patients and 10 controls were tested, the GHRH analog Geref Diagnostic [GHRH(1-29)NH2] became unavailable in the United States. The study was completed by testing 10 additional AGHD patients and 38 controls with macimorelin alone.

MAIN OUTCOME MEASURE:

Peak GH area under the receiver operating characteristic curve after macimorelin was measured.

RESULTS:

Fifty AGHD subjects and 48 controls were evaluated. Peak GH levels in AGHD patients and controls after macimorelin were 2.36 ± 5.69 and 17.71 ± 19.11 ng/mL, respectively (P < .0001). With macimorelin, the receiver operating characteristic analysis yielded an optimal GH cut point of 2.7 ng/mL, with 82% sensitivity, 92% specificity, and 13% misclassification rate. For subjects receiving both tests, macimorelin showed discrimination comparable with arginine+GHRH (area under the receiver operating characteristic curve 0.99 vs 0.94, respectively, P = .29). Obesity (body mass index > 30 kg/m(2)) was present in 58% of subjects, and peak GH levels were inversely associated with body mass index in controls (r = -0.37, P = .01). Using the separate cut points of 6.8 ng/mL for nonobese and 2.7 for obese subjects reduced the misclassification rate to 11%. Only 1 drug-related serious adverse event, an asymptomatic QT interval prolongation on the electrocardiogram, was reported.

CONCLUSION:

Oral macimorelin is safe, convenient, and effective in diagnosing AGHD with accuracy comparable with the arginine+GHRH test.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT00448747.

PMID:
23559086
PMCID:
PMC4207947
DOI:
10.1210/jc.2013-1157
[Indexed for MEDLINE]
Free PMC Article

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