Endocrine Abstracts

Concerns have been raised that undergraduate medical education in Endocrinology/Diabetes (E&D) is not preparing students adequately for clinical practice. This project assessed trainer perceptions of the delivered D&E curriculum and identified areas for improvement. We surveyed E&D consultants at the 12 hospitals which accept students from our medical school using an online questionnaire. Emails were sent to 50 consultants. The response rate was 46% (23/50 recipien...

Context: Serum IGF-binding protein-3 (IGFBP-3) and molar IGF-I to IGFBP-3 ratio can aid the diagnosis of GH-related diseases. However, their clinical utility is limited by lack of validated reference intervals.Objectives: To establish age-, gender- and Tanner stage-specific reference intervals for IGFBP-3 and IGF-I to IGFBP-3 ratio for Indian ethnicity.Setting and Participants: We conducted a cross-sectional epidemiological study (...

Context: To diagnose and monitor GH-related disorders, serum IGF-I is a cornerstone, but Indian ethnicity based data, following consensus criteria for establishment of normative data are not available.Objectives: To generate normative IGF-I data for chronological age, bone age (BA) [Greulich & Pyle] and Tanner stage for both genders.Setting and Participants: We conducted a cross-sectional epidemiological study for children (age...

Context: Serum IGF-1 levels are of paramount importance for diagnosis and management of growth related disorders. The reference range of IGF- 1 should be ethnicity and Tanner specific. Indian data on the same is scarce and there is a need to develop the same.Objective: To develop age, sex and Tanner based normative reference range of serum IGF-1 level for Indian children and correlate the same with peri-pubertal hormonal parameters.<p class="abstext"...

ATL1103 is a second generation antisense 20mer intended to inhibit expression of the GH receptor (GHR) gene. Phosphorothioate and 2′-O-methoxyethyl modifications to nucleotides increase its plasma half-life and affinity for the target RNA to allow post-hybridization RNaseH degradation. We previously reported a phase 2, randomised, open-label, parallel group study of ATL1103 in 26 patients with acromegaly which demonstrated a fall in serum IGF-I of 26% with 200 mg twice w...

ATL1103 is a second generation antisense oligomer directed at the GH receptor. It is a 20mer with a phosphorothioate backbone and 2′-O-methoxyethyl modifications of the five nucleotides at either end intended to increase its plasma half-life and affinity for the target RNA to allow post-hybridization RNaseH degradation. We report a phase 2 randomised, open-label, parallel group study of subcutaneously administered ATL1103 in patients with active acromegaly. Appr...

Background: Distinguishing non-specific signs/symptoms of acromegaly from treatment-emergent adverse events (TEAEs) in patients treated with somatostatin receptor ligands has proven difficult given limited data from placebo-controlled studies. The CHIASMA OPTIMALstudy provides a novel data set to evaluate the incidence of adverse events (AEs) in patients randomized to oral octreotide capsules (OOC) or placebo.Methods: A multinational, randomized, placebo...

Objective: The phase 3 CHIASMA OPTIMAL study assessed efficacy and safety of oral octreotide capsules (OOC) in patients with acromegaly controlled on injectable somatostatin receptor ligands (SRLs). Sensitivity analyses were conducted for efficacy endpoints using two methods of imputation (i.e., the process of replacing clinical data with substitution values) to address missing data points due to some subjects reverting back to their prior injectable SRL treatment.<p class...

The European Registry on Cushing’s syndrome (ERCUSYN) is designed to collect prospective and follow-up data at EU level on patients with Cushing’s syndrome (CS). Baseline data on 481 CS patients (390 females, 91 males; mean age (±S.D.): 44±14 years) collected from 36 centres in 23 countries. This cohort included new patients since 2008 and retrospective cases since 2005. Patients were divided into four major etiologic groups: pituitary-depend...

Gigantism is an extremely rare condition and hence the relevant literature is largely a series of case reports. We present data on patients with gigantism <20 years of age identified from Pfizer’s Acrostudy registry of patients treated with pegvisomant.Eleven patients (5M) were identified: IGF1 at diagnosis was 1.6×ULN (1.15–3.3), height +5 SDS (1.1–3.8) and age 14.5 years (4–19). The three youngest (4, 7 and 14 years) had pi...