Limits...
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 Comparison of adjusted estimated means of Turner syndrome (TS) height standard deviation score (HSDS) references (± SE): target height (TH)-corrected HSDS values, normal-height standard deviation score values, and HSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]. b Comparison of adjusted estimated means of change in TS HSDS references (± SE): TH-corrected and normal ΔHSDS, and ΔHSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

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

Fig2: a Comparison of adjusted estimated means of Turner syndrome (TS) height standard deviation score (HSDS) references (± SE): target height (TH)-corrected HSDS values, normal-height standard deviation score values, and HSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]. b Comparison of adjusted estimated means of change in TS HSDS references (± SE): TH-corrected and normal ΔHSDS, and ΔHSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]

Mentions: Figure 2 depicts HSDS data for the TS patients. HSDS and TH-corrected HSDS values were similar (Fig. 2a) (Additional file 1: Table S1 for unadjusted estimated means). Baseline HSDS (mean ± SE) was –2.67 ± 0.03, increasing to –1.65 ± 0.03 at year 4. Baseline HSDS (mean ± SE) based on the Ranke TS reference was 0.30 ± 0.03, increasing to 2.09 ± 0.03 after 4 years’ GH treatment. Similar trends were observed using the Cabrol TS and Westerlaken TS references.Fig. 2


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 Comparison of adjusted estimated means of Turner syndrome (TS) height standard deviation score (HSDS) references (± SE): target height (TH)-corrected HSDS values, normal-height standard deviation score values, and HSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]. b Comparison of adjusted estimated means of change in TS HSDS references (± SE): TH-corrected and normal ΔHSDS, and ΔHSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]
© Copyright Policy - OpenAccess
Related In: Results  -  Collection

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

Fig2: a Comparison of adjusted estimated means of Turner syndrome (TS) height standard deviation score (HSDS) references (± SE): target height (TH)-corrected HSDS values, normal-height standard deviation score values, and HSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]. b Comparison of adjusted estimated means of change in TS HSDS references (± SE): TH-corrected and normal ΔHSDS, and ΔHSDS values based on the reference populations of Ranke [19], Cabrol [20] and Westerlaken [21]
Mentions: Figure 2 depicts HSDS data for the TS patients. HSDS and TH-corrected HSDS values were similar (Fig. 2a) (Additional file 1: Table S1 for unadjusted estimated means). Baseline HSDS (mean ± SE) was –2.67 ± 0.03, increasing to –1.65 ± 0.03 at year 4. Baseline HSDS (mean ± SE) based on the Ranke TS reference was 0.30 ± 0.03, increasing to 2.09 ± 0.03 after 4 years’ GH treatment. Similar trends were observed using the Cabrol TS and Westerlaken TS references.Fig. 2

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