Characterization of the IGF system in 15 patients with Alstrom syndrome.
Document Type
Article
Publication Date
2007
Keywords
Blindness, Body-Height, Case-Control-Studies, Deafness, Female, Growth-Disorders, Growth-Hormone, Heart-Failure-Congestive, Humans, Hypertriglyceridemia, Insulin-Resistance, Insulin-Like-Growth-Factor-Binding-Protein-2, Insulin-Like-Growth-Factor-I, Insulin-Like-Growth-Factor-II, Luteinizing-Hormone, Male, Obesity, Somatomedins, Statistics-Nonparametric, Syndrome, Testosterone
First Page
269
Last Page
275
JAX Location
see Reprint Collection (a pdf is available)
JAX Source
Clin Endocrinol (Oxf) 2007 Feb; 66(2):269-75.
Abstract
BACKGROUND: Alstrom syndrome (ALMS) is a rare recessively inherited progressive disease (OMIM 203800). Among its diverse spectrum of clinical features are phenotypes associated with deficiencies of the GH/IGF-I axis, including short stature, obesity, insulin resistance, hypertriglyceridaemia and heart failure. PATIENTS AND MEASUREMENTS: To characterize the IGF system in ALMS, we evaluated a subset of 15 young adults with ALMS for hepatic, renal and thyroid function. Glycaemic and hormone measurements such as insulin, GH, FSH, LH, testosterone and 17-beta-oestradiol were clinically assessed. In addition, we measured IGF-I, IGF-II, IGF binding-protein-3 (IGFBP-3) and acid labile subunit (ALS - the subunits that constitute the main somatomedin complex in the circulation), and IGFBP-1 and IGFBP-2 (known to influence the bioavailability of the IGFs). RESULTS: A significantly lower height was observed in ALMS patients compared to age-matched controls. ALMS patients were clinically obese (by weight and body mass index (BMI) standards) and leptin levels correlated with BMI. Renal and hepatic dysfunction was implicated in some patients by increased values of blood urea nitrogen (BUN) and creatinine, and transaminases, respectively. One-third of the patients presented with fasting hyperglycaemia and 80% were hyperinsulinaemic. TSH was slightly increased in 20% of patients. Baseline FSH and LH in females were within the normal range, while half of the males had abnormally low testosterone values. Male patients with hypogonadism showed significantly lower testosterone, oestrogen and ALS levels. Baseline GH values were not found to be increased. ALS and IGFBP-1 were significantly reduced and IGFBP-2 was markedly increased in ALMS patients compared to age-matched controls. The IGFs and IGFBPs were not significantly different between males and females affected with ALMS. No significant association was observed between IGFs or IGFBPs levels and weight, height, BMI, glycaemia, hyperinsulinaemia and testosterone levels. However, we found a significant association of gamma-glutamyltransferase (GGT) with IGFBP-2. IGF-I levels were significantly associated with LH in female patients. CONCLUSIONS: In summary, the reduction of ALS and the increase of IGFBP-2 points to a growth hormone deficiency (GHD) condition in ALMS. However, further tests, including GH dynamics, are needed to determine whether, or to what degree disturbances in the GH/IGF axis contribute to the relatively short stature.
Recommended Citation
Maffei P,
Boschetti M,
Marshall JD,
Paisey RB,
Beck S,
Resmini E,
Collin GB,
Naggert JK,
Milan G,
Vettor R,
Minuto F,
Sicolo N,
Barreca A.
Characterization of the IGF system in 15 patients with Alstrom syndrome. Clin Endocrinol (Oxf) 2007 Feb; 66(2):269-75.