Optimal Therapy of Growth Hormone Deficiency in the Child and Adolescent

Abstract

Paediatric endocrinologists aspire to provide the best care for children, including improving outcomes in growth hormone deficiency (GHD).The challenging issues are sometimes simple ones, such as which children should be seen in the endocrine clinic for evaluation of their growth, or standards for diagnosis of GHD, or methods of delivering standard therapy for GHD.1 Historically accepted solutions require review as knowledge and technology advance. 2-4This article addresses ways to optimise treatment strategy for children diagnosed with GHD. Optimal therapy of growth disorders depends on accurate diagnosis of the aetiology and identification of the goals of therapy. It is important to understand normal patterns of growth hormone (GH) and insulin-like growth factor (IGF) secretion in order to appreciate differences between normal physiology and hormone patterns induced by therapy. Finally, monitoring for efficacy and safety, identification of interfering factors and making appropriate dose adjustment are all part of optimising GH therapy in childhood GHD. Accurate DiagnosisMany factors besides GH participate in the regulation of normal growth (nutrition, thyroid hormone, genetics, prenatal health, family growth patterns and psychosocial adjustment [see Figure 1]). GH has been administered to children with severe GHD since 1958. Of course, there is no difficulty in making the diagnosis of GHD when the condition is severe. However, with the increasing availability of synthetic recombinant GH, GH therapy has been used in more conditions (see Figure 2). 5 There continues to be controversy about methods for accurate diagnosis of milder degrees of GHD, as well as controversy about which children should be treated with GH.Standardisation of GH assays and cutoff values for normal GH secretion may permit more uniform diagnosis, enabling therapy decisions to be based on reliable data from assays. Goals of TherapyThe purpose of GH treatment must be considered. GH therapy clearly speeds up linear growth in the GHD child and adolescent. However, GHD leads to metabolic effects in addition to slowed linear growth. In untreated GHD, body composition is altered with increased fat mass, decreased lean body mass and decreased accrual of bone mineral. 8-10In addition, individuals with GHD may have decreased exercise tolerance and a sense of fatigue, along with a raised risk for AbstractOptimal therapy of growth disorders depends on accurate diagnosis and clear goals for therapy. Understanding normal patterns of growth hormone (GH) and insulin-like growth factor (IGF) secretion are necessary to appreciate the different hormone pattern induced by therapy.Finally, monitoring efficacy and safety, identifying interfering factors and adjusting doses are all part of optimising GH therapy in childhood GH deficiency (GHD). Prevention of development of GHD would avoid the need for therapy. Options for optimising GH therapy in childhood GHD include initiating treatment as young as possible, facilitating adherence to a therapy plan and adjusti...

Optimizing growth hormone threapy in chilhood growth hormone defiency

“…BH eksikliği (6)(7)(8)(9)(10) , SGA (11,12) ve Turner sendromunun (13,14) tanısı ve BH ile tedavisinde uzlaşı sağlanmış algoritmalar mevcuttur. Ancak, BH tedavisinin etkinliği konusunda kanıta dayalı çalışmalar olmakla beraber, BH'ye yanıtın bireysel farklılıklar gösterebileceği ve hastalığın ağırlığı, uygulanan tedavi çizelgeleri ve tanı kriterleri açısından değişkenlik nedeniyle farklı hasta grupları arasında etkililik karşı-laştırması yapmanın zor olduğu unutulmamalıdır (5) .…”

“…Bunların yanı sıra uzun süreli tedavide birinci yıl büyüme yanıtı, ergenlik öncesi kazanılan boy ve tedaviye uyum da dikkate alınması gereken faktörler-dir (2,5) . Hastanın BH sekresyon kapasitesi, beslenme durumu ve varsa diğer hormon eksikliklerinin kontrolü de önemlidir (16) .…”

“…Hastanın BH sekresyon kapasitesi, beslenme durumu ve varsa diğer hormon eksikliklerinin kontrolü de önemlidir (16) . BH tedavisi altında gerçekleşen büyüme hızı doğum ağırlığı, mevcut ağırlık, ebeveyn boyları ile uyumsuzluk ve BH tedavi dozu ve enjeksiyon sıklığı ile pozitif, uyarılmış doruk BH değeri ve tedaviye başlama yaşı ile negatif korelasyon göster-mektedir (5,17) .…”

“…Bu matematiksel modellerin hedefi tedavi yanıtı değiş-kenliğini çoklu regresyon analizleri ile tanımlanabilir kıstaslar üzerinden sunarak, klinisyenlerin bireye göre tedavi dozu adaptasyonu yapabilmesine olanak sağlamaktır (5,17,22) .…”

“…Büyüme bozukluklarının optimal tedavisi etiyolojinin doğru tanılanması ve gerçekçi tedavi hedeflerinin belirlenmesi ile olası olup, tedavi altındaki hastaların etkililik ve güvenlilik açısından yakın izlemi, izlem (5) .…”