Evaluation of Growth Characteristics and Final Heights of Cases Diagnosed with Noonan Syndrome on GH Treatment.

Zeynep Şıklar, Merih Berberoğlu, Sirmen Kızılcan Çetin, Melek Yıldız, Serap Turan, Şükran Darcan, Semra Çetinkaya, Nihal Hatipoğlu, Ruken Yıldırım, Korcan Demir, Öznur Vermezoğlu, Zehra Yavaş Abalı, Deniz Özalp Kızılay, Nilay Görkem Erdoğan, Ülkü Gül Şiraz, Zerrin Orbak, İlker Tolga Özgen, Aysun Bideci, Beray Selver Eklioğlu, Esin Karakılıç Özturan, Gürkan Tarçın, Abdullah Bereket, Feyza Darendeliler
Author Information
  1. Zeynep Şıklar: Ankara University School of Medicine, Department of Pediatric Endocrinology.
  2. Merih Berberoğlu: Ankara University School of Medicine, Department of Pediatric Endocrinology.
  3. Sirmen Kızılcan Çetin: Ankara University School of Medicine, Department of Pediatric Endocrinology.
  4. Melek Yıldız: İstanbul University School of Medicine, Department of Pediatric Endocrinology.
  5. Serap Turan: Marmara University School of Medicine, Department of Pediatric Endocrinology.
  6. Şükran Darcan: Ege University School of Medicine, Department of Pediatric Endocrinology.
  7. Semra Çetinkaya: University of Health Science, Dr. Sami Ulus Child Health and Diseases Health Implementation and Research Center.
  8. Nihal Hatipoğlu: Erciyes University School of Medicine, Department of Pediatric Endocrinology.
  9. Ruken Yıldırım: Diyarbakır Child's Hospital Pediatric Endocrinology.
  10. Korcan Demir: Dokuz Eylül University School of Medicine, Department of Pediatric Endocrinology.
  11. Öznur Vermezoğlu: Trakya University School of Medicine, Department of Pediatric Endocrinology.
  12. Zehra Yavaş Abalı: Marmara University School of Medicine, Department of Pediatric Endocrinology.
  13. Deniz Özalp Kızılay: Ege University School of Medicine, Department of Pediatric Endocrinology.
  14. Nilay Görkem Erdoğan: University of Health Science, Dr. Sami Ulus Child Health and Diseases Health Implementation and Research Center.
  15. Ülkü Gül Şiraz: Erciyes University School of Medicine, Department of Pediatric Endocrinology.
  16. Zerrin Orbak: Atatürk University, School of Medicine, Department of Pediatric Endocrinology and Diabetes.
  17. İlker Tolga Özgen: Biruni University Taculty of Medicine Division of Pediatric Endocrinology.
  18. Aysun Bideci: Gazi University School of Medicine, Department of Pediatric Endocrinology.
  19. Beray Selver Eklioğlu: Necmettin Erbakan University Faculty of Medicine Department of Pediatric Endocrinology.
  20. Esin Karakılıç Özturan: Marmara University School of Medicine, Department of Pediatric Endocrinology.
  21. Gürkan Tarçın: Adana City Training and Research Hospital.
  22. Abdullah Bereket: Marmara University School of Medicine, Department of Pediatric Endocrinology.
  23. Feyza Darendeliler: İstanbul University School of Medicine, Department of Pediatric Endocrinology.

Abstract

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.
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.

Keywords

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