Evaluation of Growth Characteristics and Final Heights of Cases Diagnosed with Noonan Syndrome on GH Treatment
Zeynep Şıklar1, Merih Berberoğlu1, Sirmen Kızılcan Çetin1, Melek Yıldız2, Serap Turan3, Şükran Darcan4, Semra Çetinkaya5, Nihal Hatipoğlu6, Ruken Yıldırım7, Korcan Demir8, Öznur Vermezoğlu9, Zehra Yavaş Abalı3, Deniz Özalp Kızılay4, Nilay Görkem Erdoğan5, Ülkü Gül Şiraz6, Zerrin Orbak10, İlker Tolga Özgen11, Aysun Bideci12, Beray Selver Eklioğlu13, Esin Karakılıç Özturan3, Gürkan Tarçın14, Abdullah Bereket3, Feyza Darendeliler21Ankara University School of Medicine, Department of Pediatric Endocrinology2İstanbul University School of Medicine, Department of Pediatric Endocrinology
3Marmara University School of Medicine, Department of Pediatric Endocrinology
4Ege University School of Medicine, Department of Pediatric Endocrinology
5University of Health Science, Dr. Sami Ulus Child Health and Diseases Health Implementation and Research Center
6Erciyes University School of Medicine, Department of Pediatric Endocrinology
7Diyarbakır Child’s Hospital Pediatric Endocrinology
8Dokuz Eylül University School of Medicine, Department of Pediatric Endocrinology
9Trakya University School of Medicine, Department of Pediatric Endocrinology
10Atatürk University, School of Medicine, Department of Pediatric Endocrinology and Diabetes
11Biruni University Taculty of Medicine Division of Pediatric Endocrinology
12Gazi University School of Medicine, Department of Pediatric Endocrinology
13Necmettin Erbakan University Faculty of Medicine Department of Pediatric Endocrinology
14Adana City Training and Research Hospital
INTRODUCTION: Proportional short stature is one of the most important features of Noonan Syndrome, and adult height often remains below the 3rd percentile. Although the pathophysiology of short stature in NS patients is not fully understood, it has been shown that GH treatment is beneficial in NS, and it significantly improves the height in respect to the results of short and long-term GH treatment.
METHODS: In this study, the efficacy of GH therapy was evaluated in children and adolescents with Noonan syndrome who attained final height. In this national cohort study, 67 cases with NS who reached final height from 14 centers were evaluated.
RESULTS: A total of 53 cases (mean follow-up time 5.6 years) received GH treatment. Height SDS of the subjects who were started on GH tended to be shorter than those who did not receive GH (-3.26± 1.07 vs. -2.53 ±1.23) at initial presentation. The mean final height and final height SDS in girls using GH vs those not using GH were 150.1 cm and -2.17 SD vs 47.4 cm and-2.8 SD, respectively. The mean final height and final height SDS in boys using GH vs. not using GH were 162.48 ± 6.19 cm and -1.81 SD vs 157.46 ± 10.16 cm and -2.68 ± 1.42 SD, respectively. The Δheight SDS value of the cases was significantly higher in the group receiving GH than in those not receiving GH (1.36 ± 1.12 SD vs. -0.2 ± 1.24, p<0.001). Cardiac findings remained stable in two patients with hypertrophic cardiomyopathy who received GH treatment. No significant side effects were observed in the cases during follow-up.
DISCUSSION AND CONCLUSION: In patients with Noonan syndrome who reach their final height, a significant increase in height is observed with GH treatment, and an increase of approximately +1.4 SDS can be achieved. It has been concluded that GH treatment is safe and effective.
Manuscript Language: English
