TY - JOUR

T1 - Growth and Adult Height in Girls With Turner Syndrome Following IGF-1 Titrated Growth Hormone Treatment

AU - Cleemann Wang, Amanda

AU - Hagen, Casper P

AU - Nedaeifard, Leila

AU - Juul, Anders

AU - Jensen, Rikke Beck

N1 - © Endocrine Society 2020. All rights reserved. For permissions, please e-mail: journals.permissions@oup.com.

PY - 2020

Y1 - 2020

N2 - CONTEXT: Girls with Turner syndrome (TS) suffer linear growth failure, and TS is a registered indication for growth hormone (GH) treatment. GH is classically dosed according to body weight, and serum insulin-like growth factor-1 (IGF-1) concentrations are recommended to be kept within references according to international guidelines.OBJECTIVE: To assess the effect of long-term GH treatment in girls with TS following GH dosing by IGF-1 titration.DESIGN AND SETTING: A retrospective, real-world evidence, observational study consisting of data collected in a single tertiary center from 1991 to 2018.PATIENTS: A cohort of 63 girls with TS treated with GH by IGF-1 titration with a median duration of 6.7 years (interquartile range [IQR]: 3.4-9.7 years).MAIN OUTCOME MEASURES: Longitudinal measurements of height, IGF-1, and adult height (AH) following GH treatment were evaluated and compared between the different karyotypes (45,X, 45,X/46,XX, or miscellaneous).RESULTS: Using GH dose titration according to IGF-1, only 6% of girls with TS had supranormal IGF-1 levels. Median dose was 33 µg/kg/day (IQR: 28-39 µg/kg/day) with no difference between the karyotype groups. AH was reached for 73% who attained a median AH of 1.25 standard deviation score (SDS) for age specific TS references (IQR: 0.64-1.50 SDS), and a median gain in height (ΔHSDS: AH SDS minus baseline height SDS of TS references) of 0.50 SDS, equal to 3.2 cm (SD 7.68) for all karyotypes.CONCLUSION: Our real-world evidence study suggested that titration of GH dose to keep IGF-1 levels within the normal range resulted in a lower AH gain than in studies where a fixed dose was used.

AB - CONTEXT: Girls with Turner syndrome (TS) suffer linear growth failure, and TS is a registered indication for growth hormone (GH) treatment. GH is classically dosed according to body weight, and serum insulin-like growth factor-1 (IGF-1) concentrations are recommended to be kept within references according to international guidelines.OBJECTIVE: To assess the effect of long-term GH treatment in girls with TS following GH dosing by IGF-1 titration.DESIGN AND SETTING: A retrospective, real-world evidence, observational study consisting of data collected in a single tertiary center from 1991 to 2018.PATIENTS: A cohort of 63 girls with TS treated with GH by IGF-1 titration with a median duration of 6.7 years (interquartile range [IQR]: 3.4-9.7 years).MAIN OUTCOME MEASURES: Longitudinal measurements of height, IGF-1, and adult height (AH) following GH treatment were evaluated and compared between the different karyotypes (45,X, 45,X/46,XX, or miscellaneous).RESULTS: Using GH dose titration according to IGF-1, only 6% of girls with TS had supranormal IGF-1 levels. Median dose was 33 µg/kg/day (IQR: 28-39 µg/kg/day) with no difference between the karyotype groups. AH was reached for 73% who attained a median AH of 1.25 standard deviation score (SDS) for age specific TS references (IQR: 0.64-1.50 SDS), and a median gain in height (ΔHSDS: AH SDS minus baseline height SDS of TS references) of 0.50 SDS, equal to 3.2 cm (SD 7.68) for all karyotypes.CONCLUSION: Our real-world evidence study suggested that titration of GH dose to keep IGF-1 levels within the normal range resulted in a lower AH gain than in studies where a fixed dose was used.

KW - Adolescent

KW - Body Height/drug effects

KW - Child

KW - Dose-Response Relationship, Drug

KW - Drug Dosage Calculations

KW - Female

KW - Human Growth Hormone/administration & dosage

KW - Humans

KW - Insulin-Like Growth Factor I/analysis

KW - Karyotype

KW - Karyotyping

KW - Longitudinal Studies

KW - Reference Values

KW - Retrospective Studies

KW - Treatment Outcome

KW - Turner Syndrome/blood

U2 - 10.1210/clinem/dgaa274

DO - 10.1210/clinem/dgaa274

M3 - Journal article

C2 - 32421787

VL - 105

SP - 2566

EP - 2574

JO - Journal of Clinical Endocrinology and Metabolism

JF - Journal of Clinical Endocrinology and Metabolism

SN - 0021-972X

IS - 8

ER -