Endocrine Abstracts (2008) 17 P11

GH treatment and quality of life: the practicalities of running a multi-centre research study

G Wales1, L Bailey2, C Eiser3, N Wright1 & G Butler2

1Sheffield Children’s Hospital, Sheffield, UK; 2University of Reading, Reading, UK; 3University of Sheffield, Sheffield, UK.

Background: The National Institute for Clinical Excellence (NICE, 2002) suggests that measurement of quality of life (QOL) is necessary to assess the efficacy of GH treatment (GHT). A pilot study (Sheppard 2006) showed a greater improvement in QOL over the first 6 months of GHT for patients with acquired GH deficiency (AGHD) compared with idiopathic GH deficiency (IGHD).

Method: In view of this, a longitudinal research study was set up to measure changes in QOL during the first year of GHT for patients diagnosed with IGHD, AGHD, and Turner Syndrome. The sample size of 240 (60 in each of the 3 treatment groups and 60 controls: <2nd centile) is based upon the primary outcome measure, PedsQL (Varni, 1999) and has sufficient power to detect differences between groups (95% confidence, 80% power).

Practicalities: This sample size necessitated a multi-centre approach and the employment of two site co-ordinators (0.8 WTE) to look after the logistics of recruiting patients and collating data from 19 sites across the UK. The site co-ordinators assisted local investigators with obtaining site specific and R&D approvals as well as providing Site Files, resource files, and patient packs, containing all the necessary recruitment documentation and questionnaires. Idiosyncrasies in patient appointment systems, staff involved in starting patients on GHT, as well as the various availabilities, duties and responsibilities of local team members were identified. Significant regional variations have been found in the documentation and time taken to obtain local R&D approval, resulting in protracted negotiations and delays in recruiting.

Progress: To date we have 95 recruits (28 IGHD, 23 AGHD, 10 Turner Syndrome, 34 Controls) and will continue recruiting until June 2009. An initial audit of two tertiary referral centres suggests that 41% of new GHT starters are eligible for the study and 51% of these have been recruited. Of those ineligible, 21% are not GHD or Turner Syndrome, and 19% have other excluded conditions, however there is marked inter-centre variability.

Conclusion: Regional diversities in patient administration and local governance pose significant challenges in running multi-centre studies, and necessitate diverse and imaginative teamwork from investigators, nurses and site co-ordinators.

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