Endocrine Abstracts

Background: The National Institute for Health and Clinical Excellence (NICE) previously conducted a Technology Appraisal for Genotropin in children and positive guidance was issued in 2002. This guidance is currently under review.

Aim: To perform a systematic review (SR) to identify studies investigating the efficacy and safety of Genotropin published since the previous NICE appraisal for the existing indications (GH deficiency (GHD), Prader-Willi syndrome (PWS), Turner syndrome (TS) and chronic renal failure (CRF)), and for the more recently approved indication of small for gestational age (SGA).

Data sources: Eight electronic databases, including MEDLINE and EMBASE, were searched for English-language studies published since the search undertaken to inform the previous appraisal (in April 2001) for the indications existing at that time. No date restrictions were applied for the new SGA indication. The manufacturer of Genotropin was contacted for unpublished data.

Selection criteria: Randomised controlled trials (RCTs) were included if they were conducted in children (aged <16 years) with one of the indications of interest. Studies comparing Genotropin with an active comparator, placebo or no intervention were included. The reference lists of relevant SRs and meta-analyses were checked for additional trials. Outcomes focused on those clinically relevant to children with growth deficiencies.

Data collection and analysis: Studies were assessed for inclusion against the pre-agreed criteria. Data extraction from identified studies was performed independently by two reviewers, and consensus regarding data extraction or study selection was reached by discussion.

Results: From 2610 citations identified by the searches, a total of 25 RCTs were identified for inclusion in the updated assessment (two for GHD, seven for PWS, one for TS and 15 for SGA). No studies published from 2001 were identified for CRF. The studies for SGA dated back to 1994. Studies reported data for a variety of outcomes, with the most common being change in height, height standard deviation score, and growth or height velocity. Improvements in these outcomes were consistently observed in all studies that reported them. As consistent with the previous appraisal, serious adverse effects were rare.

Conclusions: Few RCTs were identified that have been published since the previous NICE appraisal. Furthermore, no long-term RCTs following children to final height were identified. It is likely that data from observational trials or registries may be required to supplement the current 2008 NICE review.