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Treatment of severe primary IGF-1 deficiency using rhIGF-1 preparation - first three years of Polish experience
Elżbieta Petriczko1, Tomasz Jackowski1, Anita Horodnicka-Józwa1
1Department of Paediatrics, Endocrinology, Diabetology, Metabolic Disorders, and Cardiology of Developmental Age, Pomeranian Medical University, Szczecin, Poland.
Insights
Recombinant human insulinlike growth factor 1 (rhIGF-1) treatment significantly improved height and height velocity in Polish children with IGF-1 deficiency. The therapy was found to be safe and effective over three years.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Metabolic Disorders
Background:
- Primary Insulin-like Growth Factor 1 (IGF-1) deficiency affects children's growth.
- Recombinant human IGF-1 (rhIGF-1) is a potential therapeutic agent.
- Assessing rhIGF-1 efficacy in diverse populations is crucial.
Purpose of the Study:
- To analyze the effects of rhIGF-1 treatment over three years in Polish children.
- To evaluate safety and efficacy in a specific patient cohort.
- To assess impact on height, growth velocity, weight, and BMI.
Main Methods:
- Twenty-seven Polish children meeting specific height and IGF-1 criteria received rhIGF-1 (mecasermin).
- Dosing started at 40 μg/kg twice daily, increasing to an average of 100 μg/kg twice daily.
- Key metrics included body height, height velocity, weight, BMI, and adverse events.
Main Results:
- Significant increases in body height (1.45 ± 1.06 SD) and height velocity were observed.
- Height velocity gains were most pronounced in the first year.
- Body weight and BMI also showed significant increases (1.16 ± 0.76 SD and 0.86 ± 0.75 SD).
Conclusions:
- rhIGF-1 (mecasermin) treatment is effective for Polish children with primary IGF-1 deficiency.
- The therapy demonstrated a clear beneficial effect on patient height and accelerated height velocity.
- Adverse events were generally mild, temporary, and did not necessitate treatment modification in most cases.
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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