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Feb 2009

RPR-GH. Retrospective study of Poor Responders to Growth Hormone treatment Nordic Study group: Bang P, Bjeknes R, Dahlgren J, Dunkel L, Gustafsson J, Juul A, Kriström B, Tapanainen P and Åberg V. Study Rationale

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Feb 2009

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  1. RPR-GH Retrospective study of Poor Responders to Growth Hormone treatment Nordic Study group: Bang P, Bjeknes R, Dahlgren J, Dunkel L, Gustafsson J, Juul A, Kriström B, Tapanainen P and Åberg V Study Rationale In preparation for a prospective Nordic study in short children with poor response to GH, define the proportion of patients with poor response in the Nordic countries 1 Feb 2009

  2. Inclusion Criteria Treated with GH for short stature for at least one year since Jan 1, 2003 Height < -2 SDS at start of GH treatment Pre-pubertal during the first year of GH treatment Auxological data available from at least 3 months before start of treatment 2 Feb 2009

  3. Study patients Approx. 900 patients were screened 481 patients included from 10 sites 25 excluded Not fulfilling inclusion criteria (2 pubertal, 14 with Height SDS > -2.0) 9 entries had incomplete data 456 patients included in study analyses • Implications for the prospective study: • Only approx. 50% of patients treated with GH were included 3 Feb 2009

  4. Diagnosis after re-classification (number of patients) Other Includes PWS, Noonan, SR, etc Other syndromes IGHD Turner Skeletal dysplasia ISS SGA GHD + other disease 4 Feb 2009

  5. Results to be published shortly • The data I presented are currently being analysed and will be shortly submitted for publication. It is hoped that they will further characterise children who have had poor responses to growth hormone treatment and particularly help to identify which of these patients may have primary IGF-I deficiency. We hope to present these definitive data at a later meeting after they have been accepted for publication.

  6. Conclusions 6 Across all diagnostic groups as many as 16% of GH treated patients are poor responders even with the least conservative definition More than 50% fullfill at least one definition of poor response Highest proportion of poor responders in SGA, Turner and Skeletal dysplasia but significant numbers in other groups Different definitions of poor response are more or less related to pre-treatment growth and age Feb 2009

  7. ..... and unresolved questions • Should a given response be defined as poor in one diagnostic group (e.g. IGHD) but not in another (e.g SGA)? • Should poor responders be considered for alternative treatment (IGF-I or IGF-I/GH) and will there be a better reponse? When should therapy be discontinued? • Which are the group(s) that will respond better to IGF-I or IGF-I/GH? GH resistance? IGF-I resistance? GH-IGF-independent growth failure? • Should alternative treatment only be evaluated in GH non-reponders? 7 Feb 2009

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