Menu
Enrolling by invitation NCT07544290

Autoimmune Hyperthyroidism in Prepubertal Children

— Observational Autoimmune Hyperthyroidism Children

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: restrospective observational study.
Who it may be relevant to
Registry conditions: Autoimmune Hyperthyroidism, Children. Basic parameters: 2 years — 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Italy
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

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

Overview

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.

Detailed description

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.

Interventions

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

Primary outcome measures

  • Number of clinical signs of hyperthyroidism at diagnosis [Time frame: Day 0 (retrospective assessment at diagnosis)]
  • Number of clinical symptoms at diagnosis [Time frame: Day 0]
  • Serum free triiodothyronine (FT3) levels at diagnosis [Time frame: Day 0]
  • Serum free thyroxine (FT4) levels at diagnosis [Time frame: Day 0]
  • Serum thyroid-stimulating hormone (TSH) levels at diagnosis [Time frame: Day 0]
  • Serum TSH receptor antibody (TRAb) levels at diagnosis [Time frame: Day 0]
  • Thyroid autoantibody positivity at diagnosis (TPOAb and TgAb) [Time frame: Day 0]
Secondary outcome measures (9)
  • Duration of antithyroid drug therapy [Time frame: Baseline (treatment initiation) to treatment discontinuation (up to 24 months)]
  • Remission rate after antithyroid drug therapy [Time frame: At 24 months after treatment initiation]
  • Rate of definitive treatment (thyroidectomy or radioactive iodine therapy) [Time frame: Baseline to 24 months]
  • Serum TSH receptor antibody (TRAb) levels at time of the definitive treatment [Time frame: At time of definitive treatment (up to 24 months)]
  • Time to normalization of thyroid function [Time frame: Baseline to normalization (up to 12 months)]
  • Aauxological parameters at diagnosis and during follow-up [Time frame: day 0, diagnosis; at 6 months; at 12 months]
  • Number of antithyroid drug discontinuation attempts [Time frame: Baseline to 24 months]
  • Annual growth velocity during follow-up [Time frame: Baseline to 24 months]
  • Bone age during follow-up [Time frame: At baseline, 12 months, and 24 months]

Eligibility criteria

Inclusion criteria

-Subjects with autoimmune hyperthyroidism

Exclusion criteria

  • Subjects with hyperthyroidism not of autoimmune aetiology

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Case-control

Study locations

Italy · 1 center
  • IRCCS Ospedale San Raffaele — Milan

Publications

  • 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

Identifiers

NCT: NCT07544290 · CET 164-2025

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗