Identification and management of poor response to growth‐promoting therapy in children with short stature

Abstract

SummaryGrowth hormone (GH) is widely prescribed for children with short stature across a range of growth disorders. Recombinant human (rh) insulin-like growth factor-1 (rhIGF-1) therapy is approved for severe primary IGF-I deficiency -a state of severe GH resistance. Evidence is increasing for an unacceptably high rate of poor or unsatisfactory response to growth-promoting therapy (i.e. not leading to significant catch up growth) in terms of change in height standard deviation score (SDS) and height velocity (HV) in many approved indications. Consequently, there is a need to define poor response and to prevent or correct it by optimizing treatment regimens within accepted guidelines. Recognition of a poor response is an indication for action by the treating physician, either to modify the therapy or to review the primary diagnosis leading either to discontinuation or change of therapy. This review discusses the optimal investigation of the child who is a candidate for GH or IGF-1 therapy so that a diagnosis-based choice of therapy and dosage can be made. The relevant parameters in the evaluation of growth response are described together with the definitions of poor response. Prevention of poor response is addressed by discussion of strategy for first-year management with GH and IGF-1. Adherence to therapy is reviewed as is the recommended action following the identification of the poorly responding patient. The awareness, recognition and management of poor response to growth-promoting therapy will lead to better patient care, greater cost-effectiveness and increased opportunities for clinical benefit.

Growth hormone and noonan syndrome: update in dysfunctional signaling aspects and in therapy for short stature

“…This therapy is currently used to treat patients with severe GH insensitivity (e.g., patients with Laron syndrome, caused by absent or defective GH receptor) [67]. Therefore, even if the efficacy of rhIGF-1 to treat severe GH resistance is currently debated [68], this treatment could be helpful in NS patients, possibly in combination with GH therapy to improve its efficiency.…”

Optimizing growth hormone threapy in chilhood growth hormone defiency

“…İzlem 3-6 aylık aralarla boy ve ağırlık ölçülerek büyüme hızı ve kilo alımının tespiti, 1. yıl tedavi yanıtının değerlendirilerek yetersiz yanıt durumunda tedavi uyumu ve doğru tanı açısından değerlendirme-lerin ve gerektiğinde IGF-1 SDS bazında BH doz titrasyonunun yapılmasını içerir (Şekil 1) (3) .…”

“…BH tedavisi ile ilgili temel sorunlar BH eksikliği tanısı koymadaki zorluklar, aynı tanı grubundaki bireysel değişkenlik ve bu nedenle bireysel yanıtı, ayrıca ergenlikteki tedavi etkinliğini öngörme güçlü-ğü ve büyüme yanıtını etkileyen oksolojik, çevresel ve genetik faktörlerin varlığı olarak sıralanabilir (3,4) .…”

Evaluation of Prepubertal Patients with Suspected Neurosecretory Dysfunction of Growth Hormone Secretion: Diagnostic Steps and Treatment Response

“…Generally growth data after one year of treatment with recombinant GH are regarded significant for the response to therapy also during the following years and for the final height outcome [11][12][13][14][15][16][17][18][19]. For our whole group of NSD-patients we found a mean gain of H-SDS of 0,77 ± 0,48, HV almost doubled (8,24 ± 1,58 cm/year versus 4,32 ± 1,68 cm/year at baseline) and HV-SDS increased by 4,40 ± 3,5.…”