Elżbieta Petriczko1, Tomasz Jackowski1, Anita Horodnicka-Józwa1, Beata Wikiera2, Anna Noczyńska2, Maria Korpal-Szczyrska3, Dorota Birkholz-Walerzak3, Ewa Małecka-Tendera4, Barbara Kalina-Fraska4, Maria Kalina5, Ewa Barg6, Iwona Beń-Skowronek7, Leszek Szewczyk7, Maciej Hilczer8,9, Joanna Smyczyńska8, Renata Stawerska8, Andrzej Lewiński10,11, Katarzyna Ziora12, Artur Bossowski13, Edyta Pietrewicz13, Beata Pyrżak14, Andrzej Kędzia15, Mieczysław Szalecki16,17, Agnieszka Kilian1, Mieczysław Walczak1. 1. Department of Paediatrics, Endocrinology, Diabetology, Metabolic Disorders, and Cardiology of Developmental Age, Pomeranian Medical University, Szczecin, Poland. 2. Department of Endocrinology and Diabetology of Children, and Adolescents, Wroclaw Medical University, Wroclaw, Poland. 3. Department of Paediatrics, Diabetology, and Endocrinology, Medical University of Gdansk, Gdansk, Poland. 4. Department of Paediatrics, Endocrinology, and Diabetes, Medical University of Silesia, Katowice, Poland. 5. Division of Clinical Genetics, Department of Molecular Biology, and Genetics, School of Medicine in Katowice, Medical University of Silesia, Katowice, Poland. 6. Department of Basic Medical Sciences, Wroclaw Medical University, Wroclaw, Poland. 7. Department of Paediatric Endocrinology, and Diabetology, Medical University Children Hospital, Lublin, Poland. 8. Department of Endocrinology and Metabolic Diseases, Polish Mother's Memorial Hospital - Research Institute, Lodz, Poland. 9. Department of Paediatric Endocrinology, Medical University of Lodz, Lodz, Poland. 10. Department of Endocrinology and Metabolic Diseases, Medical University of Lodz, Lodz, Poland, Poland. alewin@csk.umed.lodz.pl. 11. Department of Endocrinology and Metabolic Diseases, Polish Mother's Memorial Hospital - Research Institute, Lodz, Poland. alewin@csk.umed.lodz.pl. 12. Department of Paediatrics, Medical University of Silesia, Katowice, Poland. 13. Department of Paediatrics, Endocrinology, Diabetology with Cardiology Division, Medical University of Bialystok, Bialystok, Poland. 14. Department of Paediatrics and Endocrinology, Medical University of Warsaw, Warsaw, Poland. 15. Department of Clinical Auxology and Paediatric Nursing, Poznan University of Medical Sciences, Poznan, Poland. 16. Department of Endocrinology and Diabetology, Children's Memorial Health Institute, Warsaw, Poland. 17. The Faculty of Medicine and Health Sciences, Jan Kochanowski University, Kielce, Poland.
Abstract
INTRODUCTION: The objective of this study was to analyse the effects of the first three years of treatment with recombinant human insulinlike growth factor 1 (rhIGF-1) in patients from the Polish population. MATERIAL AND METHODS: Twenty-seven children (22 boys and five girls) aged 2.8 to 16.0 years old were qualified for treatment with rhIGF-1 (mecasermin) in different treatment centres, according to Polish criteria: body height below -3.0 SD and IGF-1 concentration below percentile 2.5 with normal growth hormone (GH) levels. Mecasermin initial dose was 40 μg/kg bw twice a day and was subsequently increased to an average of 100 μg/kg bw twice a day. Body height, height velocity, weight, body mass index (BMI), and adverse events were measured. RESULTS: Mecasermin treatment resulted in a statistically significant increase in body height (1.45 ± 1.06 SD; p < 0.01) and height velocity in comparison with pre-treatment values. The biggest change in height velocity happened during the first year and diminished during subsequent years. Body weight and BMI also increased significantly after treatment (1.16 ± 0.76 SD and 0.86 ± 0.75 SD, respectively; p < 0.01). Eight patients reported adverse events. These were mild and temporary and did not require treatment modification except in two patients. CONCLUSIONS: Treatment with rhIGF-1 was effective and safe in Polish patients with primary IGF-1 deficiency. It had a clear beneficial effect on the height of the patients and significantly accelerated the height velocity, particularly in the first year of treatment.
INTRODUCTION: The objective of this study was to analyse the effects of the first three years of treatment with recombinant humaninsulinlike growth factor 1 (rhIGF-1) in patients from the Polish population. MATERIAL AND METHODS: Twenty-seven children (22 boys and five girls) aged 2.8 to 16.0 years old were qualified for treatment with rhIGF-1 (mecasermin) in different treatment centres, according to Polish criteria: body height below -3.0 SD and IGF-1 concentration below percentile 2.5 with normal growth hormone (GH) levels. Mecasermin initial dose was 40 μg/kg bw twice a day and was subsequently increased to an average of 100 μg/kg bw twice a day. Body height, height velocity, weight, body mass index (BMI), and adverse events were measured. RESULTS: Mecasermin treatment resulted in a statistically significant increase in body height (1.45 ± 1.06 SD; p < 0.01) and height velocity in comparison with pre-treatment values. The biggest change in height velocity happened during the first year and diminished during subsequent years. Body weight and BMI also increased significantly after treatment (1.16 ± 0.76 SD and 0.86 ± 0.75 SD, respectively; p < 0.01). Eight patients reported adverse events. These were mild and temporary and did not require treatment modification except in two patients. CONCLUSIONS: Treatment with rhIGF-1 was effective and safe in Polish patients with primary IGF-1 deficiency. It had a clear beneficial effect on the height of the patients and significantly accelerated the height velocity, particularly in the first year of treatment.