Michael B Ranke1, Anders Lindberg2, Martin Carlsson3, Cecilia Camacho-Hübner3, Raoul Rooman4. 1. Division of Paediatric Endocrinology, University Children's Hospital, Tübingen, Germany, michael.ranke@gmx.de. 2. Endocrine Care, Pfizer Health AB, Sollentuna, Sweden. 3. Endocrine Care, Pfizer Inc., New York, New York, USA. 4. PendoCon, Putte, Belgium.
Abstract
BACKGROUND/AIMS: There is little information how rhGH treatment affects height in NS. This study aims to analyze data from the NS patients assembled in KIGS over 25 years. PATIENTS/ METHODS: Of 613 (389 m/224 f) NS patients documented, 476 (302 m/174 f) were treated for 1 year, 237 (160 m/77 f) of which served to develop a 1st year height velocity (HV) prediction algorithm. One-hundred and forty (74 m/66 f) had reached near adult height (NAH). Factors affecting NAH on rhGH were determined. RESULTS: At the start of rhGH, the NAH groups were (median, m, f) 11.0 and 10.3 years, with a height SDS of -3.2 and -3.8 SDS (Prader), respectively. The total gain after 6.3 and 5.6 years on rhGH (0.27 and 0.30 mg/kg/week) was 1.2 and 1.3 SDS. Age at the start of rhGH (negative), height at the start of rhGH, rhGH dose, number of rhGH injections/wk and birth weight (all positive) explained 36% of the variability of 1st year HV. Height at the start of rhGH, 1st year growth on rhGH, birth weight, and gender explained 74% of the variability of NAH. Causes for rhGH treatment discontinuation and adverse events were also analyzed. CONCLUSION: rhGH treatment increases NAH in NS. Prediction algorithms may optimize treatment in the future.
BACKGROUND/AIMS: There is little information how rhGH treatment affects height in NS. This study aims to analyze data from the NSpatients assembled in KIGS over 25 years. PATIENTS/ METHODS: Of 613 (389 m/224 f) NSpatients documented, 476 (302 m/174 f) were treated for 1 year, 237 (160 m/77 f) of which served to develop a 1st year height velocity (HV) prediction algorithm. One-hundred and forty (74 m/66 f) had reached near adult height (NAH). Factors affecting NAH on rhGH were determined. RESULTS: At the start of rhGH, the NAH groups were (median, m, f) 11.0 and 10.3 years, with a height SDS of -3.2 and -3.8 SDS (Prader), respectively. The total gain after 6.3 and 5.6 years on rhGH (0.27 and 0.30 mg/kg/week) was 1.2 and 1.3 SDS. Age at the start of rhGH (negative), height at the start of rhGH, rhGH dose, number of rhGH injections/wk and birth weight (all positive) explained 36% of the variability of 1st year HV. Height at the start of rhGH, 1st year growth on rhGH, birth weight, and gender explained 74% of the variability of NAH. Causes for rhGH treatment discontinuation and adverse events were also analyzed. CONCLUSION: rhGH treatment increases NAH in NS. Prediction algorithms may optimize treatment in the future.
Authors: Lars Sävendahl; Michel Polak; Philippe Backeljauw; Joanne C Blair; Bradley S Miller; Tilman R Rohrer; Anita Hokken-Koelega; Alberto Pietropoli; Nicky Kelepouris; Judith Ross Journal: J Clin Endocrinol Metab Date: 2021-05-13 Impact factor: 5.958
Authors: Alicia Romano; Juan Pablo Kaski; Jovanna Dahlgren; Nicky Kelepouris; Alberto Pietropoli; Tilman R Rohrer; Michel Polak Journal: Endocr Connect Date: 2022-01-31 Impact factor: 3.335