Büyüme hormonu eksikliği etiyolojisi, demografik veriler ve tedavi sonuçlarının değerlendirilmesi: Türkiye verileri. KIGS (Pfizer Uluslar Arası Büyüme Veri Tabanı) analiz sonuçları

Amaç: Türkiye'de büyüme hormonu (BH) eksikliği tanısı alan ve BH tedavisi başlanmış olan hastaların tanı ve tedavisine ilişkin verilerini değerlendirmek. Yöntem: Türkiye'deki 26 merkezin KİGS (Pfizer Uluslararası Büyüme Veri Tabanı) veri tabanında 1989 yılından itibaren kayıtlı BH eksikliği (BHE) vakalarının kayıtları alınarak Ocak 2004 tarihi itibarıyla 1008 hastanın verileri değerlendirildi. Bu veri tabanından yararlanılarak BH eksikliği vakalarının doğum boyu, doğum ağırlığı ve anne-baba boy değerleri, tedavi öncesi ve tedavi sırasında yaş, boy, vücut ağırlığı, hesaplanan vücut kitle indeksi, büyüme hızı ve hedef boy değerleri incelendi ve değerler standart deviasyon skor (SDS) olarak ifade edildi. BH eksikliği hastalarına büyüme hormonu (Genotropin®) haftada medyan 6 kez olarak subkutan olarak başlanmıştı. Tüm değerler medyan (10/90.persantil) olarak verildi. Bulgular: BH eksikliği vakalarının 822'si idiyopatik, 186'sı ise organik nedenli idi. Organik nedenler içinde kraniyofaringioma (n: 19), medulloblastom (n: 13) ve yapısal anomaliler (n: 89) en sık nedenleri oluşturuyordu. Tedavi başlangıcında idiyopatik BHE'de yaş 11.3 (5.4/15.1) yıl, boy SDS-3.1 (-5.2/-1.9) büyüme hızı 3.6 (1.6/6.2) cm/yıl ve boy-hedef boy farkı (SDS)-1.7 (-4.0/0.1)'ydı. BH 0.20 (0.15/0.24) mg/kg/hafta dozunda kullanılmıştı. Longitudinal olarak izlenen prepubertal idiyopatik BHE vakalarında büyüme hızı 1. yılda 8.7 (5.6/13.1) cm/yıl, 2 ve 3. yıllarda 7.0 (3.9/9.5) ve 5.9 (3.2 /8.1) cm/yıl oldu. Boy SDS'sinde özellikle 1. yılda anlamlı artış gözlenerek [-2.8 (-1.6 /4.4)] 3. yılda boy SDS-2.5 (-1.3/-3.8)'e vardı. Diğer etiyolojik gruplarda da benzer sonuçlar alındı. Sonuç: Türkiye'de BH eksikliği vakaları geç tanı almakta ve geç tedaviye başlanmaktadır. Kullanılan BH dozu önerilen doz aralığının alt sınırlarındadır. BH tedavisine yanıt literatür verileri ile uyumludur ve özellikle 1. yılda belirgindir. İlerleyen yıllarda kullanılan doz ile beklenen bir büyüme yanıtı sağlanmıştır. 2150 BH tedavi yılı açısından yan etki görülme sıklığı nadirdir.

Anahtar Kelimeler:

Büyüme hormonu, Çocuk, Eksiklik hastalıkları, Demografi, Türkiye

Growth hormone deficiency-etiology, demography and evaluation of treatment results in Turkish children: An analysis of KIGS (Pfizer International Growth Study) database

Aim: To evaluate the baseline demographic characteristics and the response to growth hormone (GH) treatment in Turkish children with GH deficiency (GHD). Methods: The data of 1008 GHD patients registered from 26 centers in Turkey in the KIGS (Pfizer's International Growth Study) database were evaluated as of January 2004. The baseline measurements of the patients including birth weight and length and parental heights; height, weight, age, calculated body mass index, height velocity and mid parental height (MPH) at onset and during GH treatment were retrieved from the database. All measurements were expressed as standard deviation scores (SDS) and given as median (10th/90th centiles) values. GH (Genotropin®) was given to all patients at a median frequency of 6 times per week subcutaneously. Results: The etiology of GHD was mainly idiopathic (n: 822). Organic causes were identified in 186 cases. Cranio-faryngioma (n: 19), medulloblastoma (n: 13) and structural central nervous system abnormalities (n: 89) were among the most frequent causes of organic etiology. In idiopathic GHD, age at onset of therapy was 11.3 (5.4/15.1) years. Height SDS at onset of therapy was -3.1 (-5.2/-1.9). Pre treatment height velocity was 3.6 (1.6/6.2) cm/year and height-MPH (SDS)-1.7 (-4.0/0.1). GH was given in a dose of 0.20 (0.15/0.24) mg/kg/week subcutaneously, 6 -7 times per week. Height velocity increased to 8.7 (5.0/13.1) cm/year in the first year of therapy. Height velocity was 7.0 (3.9/9.5) cm/year and 5.9 (3.2/8.1) cm/year in the 2nd and 3rd years of therapy (p<0.05) in the longitudinally followed prepubertal idiopathic GHD patients. Height SDS showed a significant increase to -2.8 (-4.4/-1.6) in the first and to -2.5 (-3.8 /-1.3) in the 3rd year of therapy. Similar values were obtained for the other groups. Conclusion: Age at diagnosis of GHD and age of onset of GH therapy are delayed in our children. The dose of GH used is at the lower end of the dose range recommended currently. The growth response to GH treatment is similar to the reported values in the literature on similar doses. There were no major side effects during 2150 years of GH therapy.

Keywords:

Growth Hormone, Child, Deficiency Diseases, Demography, Turkey,

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