Development of a Minimum Dataset for the Global Monitoring of the Safety and Efficacy of Growth Hormone Replacement in Adults With Growth Hormone Deficiency (AGHD)

OBJECTIVE: To identify the minimum dataset (MDS) for the monitoring of safety and effectiveness of GH in adults with growth hormone deficiency (AGHD). DESIGN: Systematic review and expert consensus. METHODS: Outcomes for AGHD were identified through a systematic literature search in PubMed, Science Direct and Cochrane. In addition, 17 clinical experts from 10 countries and two patient representatives assembled through the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (GloBE-Reg) provided data items that ideally should be collected routinely. These items were subsequently graded independently by participants on: (1) importance of the data field and (2) ease of data collection in routine clinical practice. RESULTS: The systematic review identified 35 studies with 6732 participants with AGHD with a median age of 49 (range, 22-82) years. The common outcome categories included were cardiovascular in 21 (60%) studies, serum IGF-I in 13 (37%) and IGF-I SDS in 8 (23%), adiposity measures in 15 (44%) and psychosocial outcomes in 10 (29%). A total of 190 items were provided by experts and 86 (45%) achieved sufficient consensus and alignment with reported outcomes to create a final MDS with 45 items to be assessed, of which only 43 are manually entered. CONCLUSIONS: This study has identified by consensus a minimum dataset considered necessary to provide consistency and comparability in global studies of AGHD.