ESPE2021 ePoster Category 1 Growth Hormone and IGFs A (10 abstracts)
Safety and effectiveness of pediatric growth hormone therapy: Results from the full cohort in KIGS
Mohamad Maghnie 1 , Michael B Ranke 2 , Mitchell E Geffner 3 , Elpis Vlachopapadopoulou 4 , Helmuth G Dörr 5 , Kerstin Albertsson Wikland 6 , Lourdes Ibáñez 7,8 , Martin Carlsson 9 , Wayne Cutfield 10 , Raoul Rooman 11 , Roy Gomez 12 , Michael P Wajnrajch 9 , Agnès Linglart 13 , Renata Stawerska 14,15 , Michel Polak 16 & Adda Grimberg 17
1University of Genova, Genova, Italy; 2University Children´s Hospital, Tübingen, Germany; 3Children’s Hospital Los Angeles, Los Angeles, USA; 4Aglaia Kyriakou Children’s Hospital, Athens, Greece; 5Friedrich-Alexander University of Erlangen-Nürnberg, Erlangen, Germany; 6University of Gothenburg, Gothenburg, Sweden; 7Sant Joan de Déu, Barcelona, Spain; 8Health Institute Carlos III, Madrid, Spain; 9Pfizer, New York, USA; 10Liggins Institute, University of Auckland, Auckland, New Zealand; 11PendoCon, Putte, Belgium; 12Pfizer, Brussels, Belgium; 13AP-HP, Paris, France; 14Polish Mother’s Memorial Hospital-Research Institute, Lodz, Poland; 15Medical University of Lodz, Lodz, Poland; 16Université Paris Descartes; Hopital Necker Enfants Malades, Paris, France; 17The Children’s Hospital of Philadelphia, Philadelphia, USA
Objective: KIGS (Pfizer International Growth Survey) was a large, international database of pediatric patients who received recombinant human growth hormone (rhGH) as prescribed in real-world clinical settings. This analysis evaluated the long-term safety and efficacy data from all participants until KIGS close in 2012.
Methods: Children with growth disorders and treated with rhGH (Genotropin® [somatropin]; Pfizer, NY) were enrolled. Under informed consent/assent, safety and growth data were collected during their visits. Safety was evaluated in all rhGH-treated patients, and effectiveness in those who had ≥1 year of treatment and completed height assessment at year 1. The near-adult-height (NAH) subgroup included patients who received rhGH for ≥5 years (≥2 years of prepubertal treatment) and achieved NAH, defined by height velocity <2 cm/year during the last year and age >14 years (females) or >16 years (males). Adverse events (AEs), serious AEs (SAEs), and auxological data were collected. Height standard deviation score (Ht-SDS) and Ht-SDS minus mid-parental height SDS (DiffSDS) were based on the Prader reference. Continuous variables are presented as mean±SD.
Results: Globally, 83,803 patients (58% male, 42% female) were enrolled in KIGS. Indications for treatment included idiopathic GH deficiency (IGHD; 46.9%), small for gestational age (SGA; 9.5%), Turner syndrome (TS; 9.2%), idiopathic short stature (ISS; 8.2%), and other syndromes or growth disorders (26.2%). Patients were 10.2±4.0 years old and received rhGH at 24.3±10.0 to 42.9±17.1 mg/kg/day for different indications at treatment start. Duration of rhGH treatment was 3.3±2.8 years (0.0-19.8 years), with 3.8±3.1 years of follow-up (0.0-24.1 years). AEs were reported for 12,055 (14.4%) patients and SAEs for 3108 (3.7%). The most common SAEs included craniopharyngioma recurrence (n = 151), neoplasm recurrence (n = 99), scoliosis (n = 91), and recurrent malignancy (n = 91). Death was reported for 307 (0.4%) patients, most commonly caused by neoplasms (n = 84). Increases in height SDS with rhGH were observed. In particular, in the effectiveness cohort (n = 55,284), delta Ht-SDS at year 1 versus treatment start was 0.63±0.51, 0.67±0.43, 0.54±0.43, and 0.48±0.38 for patients with IGHD, SGA, TS, and ISS, respectively, and total gain to NAH Ht-SDS was 2.08±1.23, 1.54±0.92, 1.09±0.79, and 1.51±0.90, respectively, in the NAH subgroup. DiffSDS increased at NAH versus treatment start for all diagnoses.
Conclusion: Data from KIGS, the largest and longest running database of rhGH-treated children, confirm the safety and effectiveness of pediatric rhGH therapy as used in clinical practice.
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