کد مقاله کد نشریه سال انتشار مقاله انگلیسی نسخه تمام متن
6224519 1607482 2012 5 صفحه PDF دانلود رایگان
عنوان انگلیسی مقاله ISI
A Pilot Study of Discontinuous, Insulin-Like Growth Factor 1-Dosing Growth Hormone Treatment in Young Children with FGFR3 N540K-Mutated Hypochondroplasia
موضوعات مرتبط
علوم پزشکی و سلامت پزشکی و دندانپزشکی پریناتولوژی (پزشکی مادر و جنین)، طب اطفال و بهداشت کودک
پیش نمایش صفحه اول مقاله
A Pilot Study of Discontinuous, Insulin-Like Growth Factor 1-Dosing Growth Hormone Treatment in Young Children with FGFR3 N540K-Mutated Hypochondroplasia
چکیده انگلیسی

ObjectiveTo assess the growth promoting effect of a recombinant growth hormone (rGH) treatment protocol adjusted on insulin-like growth factor 1 (IGF-1) dosing in children affected by the most severe forms of FGFR3 N540K-mutated hypochondroplasia.Study designMidterm results of an open-label, single-center, nonrandomized, 2003-2020 pilot trial to final stature, including 6 children (mean age, 2.6 ± 0.7 years; mean height SDS, −3.0 ± 0.5) with the N540K mutation of FGFR3 gene who received an rGH dosage titrated to an IGF-1 level close to 1.5 SDS of the normal range. rGH therapy was interrupted 1 day per week, 1 month per year, and 6 months every 2 years.ResultsThe mean height SDS increased by 1.9 during the 6.1 ± 0.9-year study period, reaching −0.8 to −1.3 at age 8.7 ± 1 years. The mean ± SDS baseline IGF-1 value was −1.6 ± 0.5 before rGH treatment and 1.4 ± 0.3 during the last year of observation. The average cumulative rGH dose was 0.075 ± 0.018 mg/kg/day (range, 0.059-0.100 mg/kg/day). Trunk/leg disproportion was improved.ConclusionIGF-1-dosing rGH treatment durably improves growth and reduces body disproportion in children with severe forms of hypochondroplasia.

ناشر
Database: Elsevier - ScienceDirect (ساینس دایرکت)
Journal: The Journal of Pediatrics - Volume 160, Issue 5, May 2012, Pages 849-853
نویسندگان
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