Autoimmune Hyperthyroidism in Prepubertal Children

NCT07544290 | Enrolling By Invitation

• 1 other identifier: CET 164-2025
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autoimmune Hyperthyroidism; Children

Keywords

pubertal age; autoimmune hyperthyroidism; graves; prepubertal age; antithyroid drugs

Outcome Measures

Primary Outcomes (7)

• Number of clinical signs of hyperthyroidism at diagnosis

  Total number of clinical signs (including goiter, exophthalmos, and tachycardia) present at diagnosis, recorded as a count per patient.

  Day 0 (retrospective assessment at diagnosis)

• Number of clinical symptoms at diagnosis

  Total number of symptoms per patient, categorized as none (0), mild (1-2), moderate (3-5), or severe (\>5).

  Day 0

• Serum free triiodothyronine (FT3) levels at diagnosis

  Measurement of serum FT3 (pmol/L) at diagnosis.

  Day 0

• Serum free thyroxine (FT4) levels at diagnosis

  Measurement of serum FT4 (pmol/L) at diagnosis.

  Day 0

• Serum thyroid-stimulating hormone (TSH) levels at diagnosis

  Measurement of serum TSH (mIU/L) at diagnosis.

  Day 0

• Serum TSH receptor antibody (TRAb) levels at diagnosis

  Measurement of TRAb levels (IU/L) at diagnosis.

  Day 0

• Thyroid autoantibody positivity at diagnosis (TPOAb and TgAb)

  Presence or absence of thyroid autoantibodies (TPOAb and TgAb), reported as positive/negative.

  Day 0

Secondary Outcomes (9)

• Duration of antithyroid drug therapy

  Baseline (treatment initiation) to treatment discontinuation (up to 24 months)

• Remission rate after antithyroid drug therapy

  At 24 months after treatment initiation

• Rate of definitive treatment (thyroidectomy or radioactive iodine therapy)

  Baseline to 24 months

• Serum TSH receptor antibody (TRAb) levels at time of the definitive treatment

  At time of definitive treatment (up to 24 months)

• Time to normalization of thyroid function

  Baseline to normalization (up to 12 months)

Study Arms (2)

50 patients affected by autoimmune hyperthyroidism in prepubertal age

Other: restrospective observational study

50 patients affected by autoimmune hyperthyroidism in pubertal age

Other: restrospective observational study

Interventions

restrospective observational study OTHER

we collect clinical, hormonal and follow up data and compare data in prepubertal and pubertal patients affected by autoimmune hyperthyroidism

50 patients affected by autoimmune hyperthyroidism in prepubertal age; 50 patients affected by autoimmune hyperthyroidism in pubertal age

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

We enrolled all patients affected by autoimmune hyperthyroidism in prepubertal age and pubertal age to compare 2 groups

You may qualify if:

• Subjects with autoimmune hyperthyroidism

You may not qualify if:

• Subjects with hyperthyroidism not of autoimmune aetiology

Sponsors & Collaborators

• IRCCS Ospedale San Raffaele: lead
• University of Campania Luigi Vanvitelli: collaborator
• University of Genova: collaborator
• Arcispedale S. Anna, Ferrara: collaborator
• IRCCS Azienda Ospedaliero-Universitaria di Bologna: collaborator
• Azienda Ospedaliero-Universitaria di Parma: collaborator
• Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari: collaborator
• Azienda Ospedaliera Universitaria Integrata Verona: collaborator
• Fondazione IRCCS San Gerardo dei Tintori: collaborator
• Azienda Ospedaliera Universitaria Policlinico "G. Martino": collaborator
• Azienda Ospedaliera SS. Antonio e Biagio e Cesare Arrigo di Alessandria: collaborator
• Università di Torino, Torino: collaborator
• Federico II University: collaborator
• Buzzi Children's Hospital: collaborator

Study Sites (1)

IRCCS Ospedale San Raffaele

Milan, Italy, 20132, Italy

Location

Related Publications (10)

• 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.

  BACKGROUND

• Chen J, Eng L & Lam L. MON-263 Graves' disease presenting as chronic diarrhea in a toddler. Journal of the Endocrine Society 2019

  BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• 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 BACKGROUND

• Francis N, Francis T, Lazarus JH, et al. Current controversies in the management of Graves' hyperthyroidism. Expert Rev Endocrinol Metab 2020;15:159-69

  BACKGROUND

• Mooij CF, Cheetham TD, Verburg FA, et al. 2022 European Thyroid Association Guideline for the Management of Pediatric Graves' Disease. Eur Thyroid J 2022;11:e210073.

  BACKGROUND

• Kaplowitz PB, Vaidyanathan P. Update on pediatric hyperthyroidism. Curr Opin Endocrinol Diabetes Obes. 2020 Feb;27(1):70-76. doi: 10.1097/MED.0000000000000521.

  PMID: 31789723 BACKGROUND

• Léger J, Olivieri A, Donaldson M, et al.; ESPE-PES-SSLEP-JSPE-APEG-APPES-ISPAE;Congenital HHypothyroidism Consensus Conference GGroup European Society for Paediatric Endocrinology consensus guidelines on screening, diagnosis, and management of congenital hhypothyroidism Horm Res Paediatr 22014;81:80-10

  BACKGROUND

MeSH Terms

Conditions

Graves Disease

Condition Hierarchy (Ancestors)

Exophthalmos; Orbital Diseases; Eye Diseases; Goiter; Thyroid Diseases; Endocrine System Diseases; Hyperthyroidism; Autoimmune Diseases; Immune System Diseases

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: medical doctor - Specialist in Pediatrics Expert in Pediatric Endocrinology Head of the Thyroid Pathology Outpatient Clinic

Study Record Dates

• First Submitted: February 26, 2026
• First Posted: April 22, 2026
• Study Start: January 1, 2026
• Primary Completion: January 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: April 22, 2026

Record last verified: 2026-02

Locations

IT (1)