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Noonan syndrome and Turner syndrome patients respond similarly to 4 years' growth-hormone therapy: longitudinal analysis of growth-hormone-naïve patients enrolled in the NordiNet® International Outcome Study and the ANSWER Program.

Lee PA, Ross JL, Pedersen BT, Kotnik P, Germak JA, Christesen HT - Int J Pediatr Endocrinol (2015)

Bottom Line: The analyses showed a significant increase in HSDS over time for both NS and TS (P < 0.0001).ΔHSDS in NS was higher with younger baseline age; ΔHSDS in TS was higher for patients with younger baseline age and higher GH dose.NS and TS patients responded well and similarly over 4 years of GH treatment.

View Article: PubMed Central - PubMed

Affiliation: Penn State College of Medicine, The Milton S. Hershey Medical Center, 500 University Dr., MC-H085, Hershey, PA 17033-0850 USA.

ABSTRACT

Background: Turner syndrome (TS) and Noonan syndrome (NS) are distinct syndromes associated with short stature and other similar phenotypic features. We compared the responses to growth hormone (GH) therapy of TS and NS patients enrolled in the NordiNet® International Outcome Study (IOS) or the American Norditropin Studies: Web-Enabled Research (ANSWER) Program, which collect information on GH therapy in clinical practice.

Methods: Repeated-measures regression analysis was performed on change in height standard deviation score (HSDS) and target-height-corrected HSDS, based on national normal references and treatment-naïve disease-specific references. Models were adjusted for baseline age and HSDS, and average GH dose. The study population was paediatric patients with TS and NS in the NordiNet® IOS and ANSWER Program. Longitudinal growth responses over 4 years were evaluated.

Results: In 30 NS patients (24 males; baseline age 8.39 ± 3.45 years) and 294 TS patients (7.81 ± 3.22 years), 4-year adjusted ΔHSDS were +1.14 ± 0.13 and +1.03 ± 0.04, respectively (national references). Based on untreated, disease-specific references, 4-year adjusted ΔHSDS for NS and TS were +1.48 ± 0.10 and +1.79 ± 0.04. The analyses showed a significant increase in HSDS over time for both NS and TS (P < 0.0001). ΔHSDS in NS was higher with younger baseline age; ΔHSDS in TS was higher for patients with younger baseline age and higher GH dose.

Conclusions: NS and TS patients responded well and similarly over 4 years of GH treatment.

No MeSH data available.


Related in: MedlinePlus

a Adjusted estimated means of height standard deviation score (HSDS; ± SE) by indication and visit year; adjusted for age at treatment start, HSDS at baseline, and average GH dose. b Adjusted estimated means of the change in HSDS, ΔHSDS (± SE) from baseline by indication and visit year; adjusted for age at treatment start, HSDS at baseline and average GH dose
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Fig3: a Adjusted estimated means of height standard deviation score (HSDS; ± SE) by indication and visit year; adjusted for age at treatment start, HSDS at baseline, and average GH dose. b Adjusted estimated means of the change in HSDS, ΔHSDS (± SE) from baseline by indication and visit year; adjusted for age at treatment start, HSDS at baseline and average GH dose

Mentions: NS and TS patients responded similarly to GH therapy over time (Fig. 3a and b). Figure 3a provides the HSDS estimated means ± SE per indication based on the normal national references adjusted for age at treatment start, HSDS at baseline and average GH dose. The adjustment only changed the values marginally from the unadjusted, crude data (Additional file 1: Table S1). Figure 3b depicts the estimated means (± SE) for ΔHSDS from baseline to 4-year follow-up for each indication. This figure illustrates the similar response in the NS and TS patients; no significant difference between the indications was found (P = 0.6281). The adjustment changed the values only marginally (Additional file 1: Table S2).Fig. 3


Noonan syndrome and Turner syndrome patients respond similarly to 4 years' growth-hormone therapy: longitudinal analysis of growth-hormone-naïve patients enrolled in the NordiNet® International Outcome Study and the ANSWER Program.

Lee PA, Ross JL, Pedersen BT, Kotnik P, Germak JA, Christesen HT - Int J Pediatr Endocrinol (2015)

a Adjusted estimated means of height standard deviation score (HSDS; ± SE) by indication and visit year; adjusted for age at treatment start, HSDS at baseline, and average GH dose. b Adjusted estimated means of the change in HSDS, ΔHSDS (± SE) from baseline by indication and visit year; adjusted for age at treatment start, HSDS at baseline and average GH dose
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

License 1 - License 2
Show All Figures
getmorefigures.php?uid=PMC4562101&req=5

Fig3: a Adjusted estimated means of height standard deviation score (HSDS; ± SE) by indication and visit year; adjusted for age at treatment start, HSDS at baseline, and average GH dose. b Adjusted estimated means of the change in HSDS, ΔHSDS (± SE) from baseline by indication and visit year; adjusted for age at treatment start, HSDS at baseline and average GH dose
Mentions: NS and TS patients responded similarly to GH therapy over time (Fig. 3a and b). Figure 3a provides the HSDS estimated means ± SE per indication based on the normal national references adjusted for age at treatment start, HSDS at baseline and average GH dose. The adjustment only changed the values marginally from the unadjusted, crude data (Additional file 1: Table S1). Figure 3b depicts the estimated means (± SE) for ΔHSDS from baseline to 4-year follow-up for each indication. This figure illustrates the similar response in the NS and TS patients; no significant difference between the indications was found (P = 0.6281). The adjustment changed the values only marginally (Additional file 1: Table S2).Fig. 3

Bottom Line: The analyses showed a significant increase in HSDS over time for both NS and TS (P < 0.0001).ΔHSDS in NS was higher with younger baseline age; ΔHSDS in TS was higher for patients with younger baseline age and higher GH dose.NS and TS patients responded well and similarly over 4 years of GH treatment.

View Article: PubMed Central - PubMed

Affiliation: Penn State College of Medicine, The Milton S. Hershey Medical Center, 500 University Dr., MC-H085, Hershey, PA 17033-0850 USA.

ABSTRACT

Background: Turner syndrome (TS) and Noonan syndrome (NS) are distinct syndromes associated with short stature and other similar phenotypic features. We compared the responses to growth hormone (GH) therapy of TS and NS patients enrolled in the NordiNet® International Outcome Study (IOS) or the American Norditropin Studies: Web-Enabled Research (ANSWER) Program, which collect information on GH therapy in clinical practice.

Methods: Repeated-measures regression analysis was performed on change in height standard deviation score (HSDS) and target-height-corrected HSDS, based on national normal references and treatment-naïve disease-specific references. Models were adjusted for baseline age and HSDS, and average GH dose. The study population was paediatric patients with TS and NS in the NordiNet® IOS and ANSWER Program. Longitudinal growth responses over 4 years were evaluated.

Results: In 30 NS patients (24 males; baseline age 8.39 ± 3.45 years) and 294 TS patients (7.81 ± 3.22 years), 4-year adjusted ΔHSDS were +1.14 ± 0.13 and +1.03 ± 0.04, respectively (national references). Based on untreated, disease-specific references, 4-year adjusted ΔHSDS for NS and TS were +1.48 ± 0.10 and +1.79 ± 0.04. The analyses showed a significant increase in HSDS over time for both NS and TS (P < 0.0001). ΔHSDS in NS was higher with younger baseline age; ΔHSDS in TS was higher for patients with younger baseline age and higher GH dose.

Conclusions: NS and TS patients responded well and similarly over 4 years of GH treatment.

No MeSH data available.


Related in: MedlinePlus