Autoimmune Hyperthyroidism in Prepubertal Children

Официальное название

Autoimmune Hyperthyroidism in Prepubertal Children With Onset Before 6 Years of Age: Characteristics at Diagnosis and Follow-Up Trends.

Обзор

The investigators propose a multicenter retrospective study to assess clinical, biochemical, and auxological characteristics at diagnosis and during follow-up in a cohort of Caucasian pediatric patients diagnosed with autoimmune hyperthyroidism before puberty. These prepubertal patients will be compared with a control group of post-pubertal patients with Graves' disease. This study aims to enhance the understanding of autoimmune hyperthyroidism in prepubertal patients by providing a detailed evaluation of disease onset, therapeutic response, and growth-related outcomes. The inclusion of a carefully matched post-pubertal control group will allow for robust comparative analysis and identification of age-dependent clinical patterns and prognostic indicators, ultimately supporting more tailored and effective management strategies in pediatric populations at this particular age.

Подробное описание

There are only few studies in literature focusing on the analysis of cohorts of patients with autoimmune hyperthyroidism with onset exclusively in early childhood. The investigators therefore propose this study with the aim of retrospectively assessing characteristics at onset and during follow-up in a cohort of Caucasian hyperthyroid subjects diagnosed with the disease before puberty.

OBJECTIVES AND ENDPOINTS

* Primary Objective To evaluate the clinical and biochemical features at the onset of autoimmune hyperthyroidism in patients diagnosed in prepubertal age Secondary Objectives

1. To evaluate of biochemical parameters during medical therapy 2. To evaluate of auxological parameters at diagnosis and during follow-up 3. To compare the previous data with the clinical and biochemical features at diagnosis and during the follow-up in patients diagnosed with Graves during the pubertal age (\> Tanner 2)

Inclusion Criteria 1. Subjects with autoimmune hyperthyroidism 2. Prepubertal stage (Tanner 1) at diagnosis Exclusion Criteria 3. Subjects with hyperthyroidism not of autoimmune aetiology 4. Pubertal stage (\>Tanner 2) at diagnosis

Sample size: * 50 hyperthyroid subjects diagnosed with the disease before puberty * 50 hyperthyroid subjects diagnosed with the disease during and post puberty

The data will then be analyzed using PRISM software. The principal investigator will verify the completeness, correctness, consistency and congruence of the reported data.

Вмешательства

• Другое restrospective observational study
  we collect clinical, hormonal and follow up data and compare data in prepubertal and pubertal patients affected by autoimmune hyperthyroidism

Первичные конечные точки

• Number of clinical signs of hyperthyroidism at diagnosis [Срок оценки: Day 0 (retrospective assessment at diagnosis)]
• Number of clinical symptoms at diagnosis [Срок оценки: Day 0]
• Serum free triiodothyronine (FT3) levels at diagnosis [Срок оценки: Day 0]
• Serum free thyroxine (FT4) levels at diagnosis [Срок оценки: Day 0]
• Serum thyroid-stimulating hormone (TSH) levels at diagnosis [Срок оценки: Day 0]
• Serum TSH receptor antibody (TRAb) levels at diagnosis [Срок оценки: Day 0]
• Thyroid autoantibody positivity at diagnosis (TPOAb and TgAb) [Срок оценки: Day 0]

Вторичные конечные точки (9)

• Duration of antithyroid drug therapy [Срок оценки: Baseline (treatment initiation) to treatment discontinuation (up to 24 months)]
• Remission rate after antithyroid drug therapy [Срок оценки: At 24 months after treatment initiation]
• Rate of definitive treatment (thyroidectomy or radioactive iodine therapy) [Срок оценки: Baseline to 24 months]
• Serum TSH receptor antibody (TRAb) levels at time of the definitive treatment [Срок оценки: At time of definitive treatment (up to 24 months)]
• Time to normalization of thyroid function [Срок оценки: Baseline to normalization (up to 12 months)]
• Aauxological parameters at diagnosis and during follow-up [Срок оценки: day 0, diagnosis; at 6 months; at 12 months]
• Number of antithyroid drug discontinuation attempts [Срок оценки: Baseline to 24 months]
• Annual growth velocity during follow-up [Срок оценки: Baseline to 24 months]
• Bone age during follow-up [Срок оценки: At baseline, 12 months, and 24 months]

Критерии участия

Критерии включения

-Subjects with autoimmune hyperthyroidism

Критерии исключения

• Subjects with hyperthyroidism not of autoimmune aetiology

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Случай-контроль

Центры проведения

Италия · 1 центр

• IRCCS Ospedale San Raffaele — Milan

Публикации

• Gu Y, Liang X, Liu M, Wu D, Li W, Cao B, Li Y, Su C, Chen J, Gong C. Clinical features and predictors of remission in children under the age of 7 years with Graves' disease. Pediatr Investig. 2020 Sep 27;4(3):198-203. doi: 10.1002/ped4.12219. eCollection 2020 Sep. PMID 33150314
• Chen J, Eng L & Lam L. MON-263 Graves' disease presenting as chronic diarrhea in a toddler. Journal of the Endocrine Society 2019
• Jonak O, Polubok J, Barg E. Graves' disease in 2.5 years old girl - 6-years-long observation. Pediatr Endocrinol Diabetes Metab. 2016;22(2):76-79. doi: 10.18544/PEDM-22.02.0055. PMID 28329777
• Azova S, Rajabi F, Modi BP, Mansfield L, Jonas MM, Drobysheva A, Boyd TK, Wassner AJ, Smith JR. Graves' disease in a five-month-old boy with an unusual treatment course. J Pediatr Endocrinol Metab. 2020 Dec 15;34(3):401-406. doi: 10.1515/jpem-2020-0549. Print 2021 Mar 26. PMID 33675208
• Shulman DI, Muhar I, Jorgensen EV, Diamond FB, Bercu BB, Root AW. Autoimmune hyperthyroidism in prepubertal children and adolescents: comparison of clinical and biochemical features at diagnosis and responses to medical therapy. Thyroid. 1997 Oct;7(5):755-60. doi: 10.1089/thy.1997.7.755. PMID 9349579
• Lazar L, Kalter-Leibovici O, Pertzelan A, Weintrob N, Josefsberg Z, Phillip M. Thyrotoxicosis in prepubertal children compared with pubertal and postpubertal patients. J Clin Endocrinol Metab. 2000 Oct;85(10):3678-82. doi: 10.1210/jcem.85.10.6922. PMID 11061522
• Francis N, Francis T, Lazarus JH, Okosieme OE. Current controversies in the management of Graves' hyperthyroidism. Expert Rev Endocrinol Metab. 2020 May;15(3):159-169. doi: 10.1080/17446651.2020.1754192. Epub 2020 Apr 21. PMID 32315207
• Mooij CF, Cheetham TD, Verburg FA, Eckstein A, Pearce SH, Leger J, van Trotsenburg ASP. 2022 European Thyroid Association Guideline for the management of pediatric Graves' disease. Eur Thyroid J. 2022 Jan 1;11(1):e210073. doi: 10.1530/ETJ-21-0073. PMID 34981748

Идентификаторы

NCT: NCT07544290 · CET 164-2025

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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