open access

Vol 70, No 1 (2019)
Original paper
Submitted: 2018-09-20
Accepted: 2018-09-27
Published online: 2018-10-23
Get Citation

Treatment of severe primary IGF-1 deficiency using rhIGF-1 preparation — first three years of Polish experience

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 Hilczer89, Joanna Smyczyńska8, Renata Stawerska8, Andrzej Lewiński108, Katarzyna Ziora11, Artur Bossowski12, Edyta Pietrewicz12, Beata Pyrżak13, Andrzej Kędzia14, Mieczysław Szalecki1516, Agnieszka Kilian1, Mieczysław Walczak1
DOI: 10.5603/EP.a2018.0074
·
Pubmed: 30351442
·
Endokrynologia Polska 2019;70(1):20-27.
Affiliations
  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
  11. Department of Paediatrics, Medical University of Silesia, Katowice, Poland
  12. Department of Paediatrics, Endocrinology, Diabetology with Cardiology Division, Medical University of Bialystok, Bialystok, Poland
  13. Department of Paediatrics and Endocrinology, Medical University of Warsaw, Warsaw, Poland
  14. Department of Clinical Auxology and Paediatric Nursing, Poznan University of Medical Sciences, Poznan, Poland
  15. Department of Endocrinology and Diabetology, The Children’s Memorial Health Institute, Warsaw, Poland
  16. The Faculty of Medicine and Health Sciences, Jan Kochanowski University, Kielce, Poland

open access

Vol 70, No 1 (2019)
Original Paper
Submitted: 2018-09-20
Accepted: 2018-09-27
Published online: 2018-10-23

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.

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.

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Keywords

insulin-like growth factor-1 (IGF-1); growth hormone insensitivity; IGF-1 deficiency; short stature; growth-promoting therapy; mecasermin; Poland

About this article
Title

Treatment of severe primary IGF-1 deficiency using rhIGF-1 preparation — first three years of Polish experience

Journal

Endokrynologia Polska

Issue

Vol 70, No 1 (2019)

Article type

Original paper

Pages

20-27

Published online

2018-10-23

DOI

10.5603/EP.a2018.0074

Pubmed

30351442

Bibliographic record

Endokrynologia Polska 2019;70(1):20-27.

Keywords

insulin-like growth factor-1 (IGF-1)
growth hormone insensitivity
IGF-1 deficiency
short stature
growth-promoting therapy
mecasermin
Poland

Authors

Elżbieta Petriczko
Tomasz Jackowski
Anita Horodnicka-Józwa
Beata Wikiera
Anna Noczyńska
Maria Korpal-Szczyrska
Dorota Birkholz-Walerzak
Ewa Małecka-Tendera
Barbara Kalina-Fraska
Maria Kalina
Ewa Barg
Iwona Beń-Skowronek
Leszek Szewczyk
Maciej Hilczer
Joanna Smyczyńska
Renata Stawerska
Andrzej Lewiński
Katarzyna Ziora
Artur Bossowski
Edyta Pietrewicz
Beata Pyrżak
Andrzej Kędzia
Mieczysław Szalecki
Agnieszka Kilian
Mieczysław Walczak

References (13)
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  4. Petriczko E, Wikiera B, Horodnicka-Józwa A, et al. A two year observation of the process of applying recombinant IGF-1 to treat short stature in children with primary IGF-1 deficiency — case reports of 3 patients. Pediatr Endocrinol Diabetes Metab. 2011; 17(4): 233–238.
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  10. Guevara-Aguirre J, Rosenbloom AL, Vasconez O. Two-year treatment of growth hormone (GH) receptor deficiency with recombinant insulin-like growth factor I in 22 children: comparison of two dosage levels and to GH-treated GH deficiency. J Clin Endocrinol Metab. 1997; 82(2): 629–633.
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  12. Midyett LK, Rogol AD, Van Meter QL, et al. MS301 Study Group. Recombinant insulin-like growth factor (IGF)-I treatment in short children with low IGF-I levels: first-year results from a randomized clinical trial. J Clin Endocrinol Metab. 2010; 95(2): 611–619.
  13. Ahn SY, Pi LQ, Hwang ST, et al. Effect of IGF-I on Hair Growth Is Related to the Anti-Apoptotic Effect of IGF-I and Up-Regulation of PDGF-A and PDGF-B. Ann Dermatol. 2012; 24(1): 26–31.

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