Autoimmune Hyperthyroidism, Children
Conditions
Keywords
pubertal age, autoimmune hyperthyroidism, graves, prepubertal age, antithyroid drugs
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.
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
OTHERrestrospective observational study
we collect clinical, hormonal and follow up data and compare data in prepubertal and pubertal patients affected by autoimmune hyperthyroidism
Sponsors
IRCCS Ospedale San Raffaele
University of Campania Luigi Vanvitelli
University of Genova
Arcispedale S. Anna, Ferrara
IRCCS Azienda Ospedaliero-Universitaria di Bologna
Azienda Ospedaliero-Universitaria di Parma
Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari
Azienda Ospedaliera Universitaria Integrata Verona
Fondazione IRCCS San Gerardo dei Tintori
Azienda Ospedaliera Universitaria Policlinico "G. Martino"
Azienda Ospedaliera SS. Antonio e Biagio e Cesare Arrigo di Alessandria
Università di Torino, Torino
Federico II University
Buzzi Children's Hospital
Study design
Observational model
CASE_CONTROL
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
-Subjects with autoimmune hyperthyroidism
Exclusion criteria
* Subjects with hyperthyroidism not of autoimmune aetiology
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of clinical signs of hyperthyroidism at diagnosis | Day 0 (retrospective assessment at diagnosis) | Total number of clinical signs (including goiter, exophthalmos, and tachycardia) present at diagnosis, recorded as a count per patient. |
| Number of clinical symptoms at diagnosis | Day 0 | Total number of symptoms per patient, categorized as none (0), mild (1-2), moderate (3-5), or severe (\>5). |
| Serum free triiodothyronine (FT3) levels at diagnosis | Day 0 | Measurement of serum FT3 (pmol/L) at diagnosis. |
| Serum free thyroxine (FT4) levels at diagnosis | Day 0 | Measurement of serum FT4 (pmol/L) at diagnosis. |
| Serum thyroid-stimulating hormone (TSH) levels at diagnosis | Day 0 | Measurement of serum TSH (mIU/L) at diagnosis. |
| Serum TSH receptor antibody (TRAb) levels at diagnosis | Day 0 | Measurement of TRAb levels (IU/L) at diagnosis. |
| Thyroid autoantibody positivity at diagnosis (TPOAb and TgAb) | Day 0 | Presence or absence of thyroid autoantibodies (TPOAb and TgAb), reported as positive/negative. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Duration of antithyroid drug therapy | Baseline (treatment initiation) to treatment discontinuation (up to 24 months) | Time in months from initiation of antithyroid drug therapy to treatment discontinuation or switch to definitive therapy, whichever occurs first. |
| Remission rate after antithyroid drug therapy | At 24 months after treatment initiation | Proportion of patients achieving sustained normalization of thyroid function (TSH, FT3, FT4) for at least 12 months after discontinuation of therapy. |
| Rate of definitive treatment (thyroidectomy or radioactive iodine therapy) | Baseline to 24 months | Proportion of patients requiring definitive treatment, defined as thyroidectomy or radioactive iodine therapy. |
| Serum TSH receptor antibody (TRAb) levels at time of the definitive treatment | At time of definitive treatment (up to 24 months) | Measurement of serum TRAb levels (IU/L) at the time of transition to definitive therapy. |
| Time to normalization of thyroid function | Baseline to normalization (up to 12 months) | Time in weeks from initiation of antithyroid therapy to normalization of FT3, FT4, and TSH levels. |
| Aauxological parameters at diagnosis and during follow-up | day 0, diagnosis; at 6 months; at 12 months | delta target height |
| Number of antithyroid drug discontinuation attempts | Baseline to 24 months | Number of attempts to discontinue antithyroid therapy per patient, categorized as successful or unsuccessful based on biochemical remission. |
| Annual growth velocity during follow-up | Baseline to 24 months | Change in height per year (cm/year). |
| Bone age during follow-up | At baseline, 12 months, and 24 months | Bone age assessed using Greulich and Pyle method. |
Countries
Italy
Outcome results
None listed