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| Name | Class |
|---|---|
| University of Campania Luigi Vanvitelli | OTHER |
| University of Genova | OTHER |
| S. Anna Hospital | OTHER |
| IRCCS Azienda Ospedaliero-Universitaria di Bologna |
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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.
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
To evaluate of biochemical parameters during medical therapy
To evaluate of auxological parameters at diagnosis and during follow-up
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 50 patients affected by autoimmune hyperthyroidism in prepubertal age |
| ||
| 50 patients affected by autoimmune hyperthyroidism in pubertal age |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| restrospective observational study | Other | we collect clinical, hormonal and follow up data and compare data in prepubertal and pubertal patients affected by autoimmune hyperthyroidism |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Duration of antithyroid drug therapy | Time in months from initiation of antithyroid drug therapy to treatment discontinuation or switch to definitive therapy, whichever occurs first. | Baseline (treatment initiation) to treatment discontinuation (up to 24 months) |
| Remission rate after antithyroid drug therapy |
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Inclusion Criteria:
-Subjects with autoimmune hyperthyroidism
Exclusion Criteria:
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We enrolled all patients affected by autoimmune hyperthyroidism in prepubertal age and pubertal age to compare 2 groups
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| IRCCS Ospedale San Raffaele | Milan | Italy | 20132 | Italy |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 33150314 | Background | 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. | |
| Background | Chen J, Eng L & Lam L. MON-263 Graves' disease presenting as chronic diarrhea in a toddler. Journal of the Endocrine Society 2019 | ||
| 28329777 |
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| ID | Term |
|---|---|
| D006111 | Graves Disease |
| ID | Term |
|---|---|
| D005094 | Exophthalmos |
| D009916 | Orbital Diseases |
| D005128 | Eye Diseases |
| D006042 | Goiter |
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| OTHER |
| Azienda Ospedaliero-Universitaria di Parma | OTHER |
| Azienda Ospedaliero-Universitaria Consorziale Policlinico di Bari | OTHER |
| Azienda Ospedaliera Universitaria Integrata Verona | OTHER |
| Fondazione IRCCS San Gerardo dei Tintori | OTHER |
| Azienda Ospedaliera Universitaria Policlinico "G. Martino" | OTHER |
| Azienda Ospedaliera SS. Antonio e Biagio e Cesare Arrigo di Alessandria | OTHER |
| Università di Torino, Torino | UNKNOWN |
| Federico II University | OTHER |
| Buzzi Children's Hospital | OTHER |
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Proportion of patients achieving sustained normalization of thyroid function (TSH, FT3, FT4) for at least 12 months after discontinuation of therapy. |
| At 24 months after treatment initiation |
| Rate of definitive treatment (thyroidectomy or radioactive iodine therapy) | Proportion of patients requiring definitive treatment, defined as thyroidectomy or radioactive iodine therapy. | Baseline to 24 months |
| Serum TSH receptor antibody (TRAb) levels at time of the definitive treatment | Measurement of serum TRAb levels (IU/L) at the time of transition to definitive therapy. | At time of definitive treatment (up to 24 months) |
| Time to normalization of thyroid function | Time in weeks from initiation of antithyroid therapy to normalization of FT3, FT4, and TSH levels. | Baseline to normalization (up to 12 months) |
| Aauxological parameters at diagnosis and during follow-up | delta target height | day 0, diagnosis; at 6 months; at 12 months |
| Number of antithyroid drug discontinuation attempts | Number of attempts to discontinue antithyroid therapy per patient, categorized as successful or unsuccessful based on biochemical remission. | Baseline to 24 months |
| Annual growth velocity during follow-up | Change in height per year (cm/year). | Baseline to 24 months |
| Bone age during follow-up | Bone age assessed using Greulich and Pyle method. | At baseline, 12 months, and 24 months |
| 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. |
| 33675208 | 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. |
| 9349579 | 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. |
| 11061522 | 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. |
| 32315207 | Background | 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. |
| 34981748 | Background | 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. |
| 31789723 | 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. |
| 24662106 | Background | Leger J, Olivieri A, Donaldson M, Torresani T, Krude H, van Vliet G, Polak M, Butler G; ESPE-PES-SLEP-JSPE-APEG-APPES-ISPAE; Congenital Hypothyroidism Consensus Conference Group. European Society for Paediatric Endocrinology consensus guidelines on screening, diagnosis, and management of congenital hypothyroidism. Horm Res Paediatr. 2014;81(2):80-103. doi: 10.1159/000358198. Epub 2014 Jan 21. |
| D013959 |
| Thyroid Diseases |
| D004700 | Endocrine System Diseases |
| D006980 | Hyperthyroidism |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |