The effects of Growth hormone treatment discontinuation in Adults on Metabolic profile, Body composition and quality Of Life (GAMBOL Study)

生长激素治疗停止对成人代谢状况、身体成分和生活质量的影响(GAMBOL 研究)

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Abstract

BACKGROUND: In adults, treatment of growth hormone deficiency with daily recombinant human growth hormone injections has shown to improve many clinical features associated with GHD. Currently, many adults with GHD receive GH treatment indefinitely. However, to date, no study has consistently demonstrated that GH treatment has a sustained beneficial effect in adults with GHD. A randomised controlled trial is needed to understand the impacts of discontinuation of long-term GH therapy in adults. However, prior to embarking on an RCT, the feasibility of a discontinuation study and the acceptability of randomisation to patients and clinicians need to be assessed. RESEARCH AIMS: (a) to explore the current practice of offering discontinuation of long-term GH treatment in adults in the UK and(b) to assess the feasibility of conducting an RCT looking at the effects of discontinuing long-term GH treatment in adults with GHD. METHODS: This mixed-method study which will be conducted in three phases.Phase 1: An online survey of endocrine clinicians' practice of offering discontinuation of long-term GH therapy in adult patients with GHD.Phase 2: Feasibility cohort study which involves recruiting two groups of adult patients with GHD (aged more than 25 years), who have been on GH treatment for at least 5 years.: (a) an intervention group consisting of 20-25 patients who will be discontinuing their long-term GH treatment for two years and (b) a control group consisting of 20-25 patients who will continue with their GH treatment.Phase 3: Qualitative study. 10-16 participants will be recruited to explore their experiences of participation in the feasibility study using semi-structured interviews. POTENTIAL BENEFITS: This study will provide evidence of current GH treatment discontinuation practice in the UK and determine the feasibility of any future RCT. Long-term, this could promote and underpin the development of the much-needed relevant clinical guidance.

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