Background: Final results from MEGHA study required by Health French Authorities (HAS).
Objective and hypotheses: Objectives are to carry out longitudinal follow-up during maximum 5 years of AGHD patients treated with Saizen, with a description of prescription modalities, demographic and clinical characteristics, patient compliance, product safety, and quality of life.
Method: MEGHA is a multicentric study, with prospective follow-up of patients every 6 months during the first year then every 12 months in the subsequent years.
Results: 90 GH-deficient patients (45 women; 49 childhood acquired deficiency (CO-group), 41 adulthood (AO-group), 31, 8±13 years; 26.4 kg/m2) were enrolled between December 2003 and October 2007. 94% of patients had at least one GH stimulation test. IGF1 was <−2 SDS in 62.5 and 41.7% of CO-group and AO-group patients. 95.6% of patients had at least one associated hypopituitary deficiency. Initial GH dose was between 0.15 and 0.3 mg/day for 51.4% of patients and <0.15 mg/day in 27.1% (median 0.24 mg/day). In 1 year after inclusion, IGF1 remains <−2 SDS for 48% of CO-group patients and 5% of AO-group. BMI, WHR, BP levels, lipids, and glycaemia were not significantly changed. The total mean treatment exposure was 39.8±22.7 months. 50% of patients discontinued their treatment at a median of 13 months after inclusion. An improved quality of life (PGWB questionnaire) was observed for the first 6 months with a subsequent stabilization particularly for AO-group. 13 patients had treatment-related adverse events (no serious AE) leading to treatment discontinuation in three patients.
Conclusion: In the great majority, prescription recommendations for Saizen in adults are observed and the treatment is well tolerated. Beside GH dose adaptation to improve IGF1 treatment response, adherence is probably key.
Funding: This work was sponsored by Merck Serono.
01 - 03 Oct 2015
European Society for Paediatric Endocrinology