Treatment of Congenital Hypothyroidism: Comparison Between L-Thyroxine Oral Solution and Tablet Formulations up to 3 years of age

2021 ◽  
Author(s):  
Maria Cristina Vigone ◽  
Rita Ortolano ◽  
Gaia Vincenzi ◽  
Clara Pozzi ◽  
Micol Ratti ◽  
...  
Keyword(s):  
Congenital Hypothyroidism ◽  
Oral Solution ◽  
Negative Effects ◽  
Developmental Quotient ◽  
Long Term Follow Up ◽  
Tablet Formulations ◽  
Hand Coordination ◽  
Group D

Objective. Oral solution and tablet formulations of Levo-Thyroxine (L-T4) are both used in the treatment of Congenital Hypothyroidism (CH). However, few studies and with a limited follow-up period have been published comparing these two formulations in children. Design. The aim of this multicenter study was to compare the effectiveness of L-T4 oral solution (with ethanol as excipient) and tablet formulation in children with CH up to 3 years of age. Methods. Children diagnosed with CH between 2006 and 2015 were enrolled and divided into two groups according to the L-T4 formulation used: solution in drops (Group D) or tablets (Group T). Auxological parameters, TSH and FT4 values and L-T4 dose were collected at diagnosis and at 15 days, 1, 3, 6, 12, 24 and 36 months of treatment. The Developmental Quotient at 1 and 3 years of age was evaluated using Griffiths’ Scale. Results. 254 children were enrolled. 117 were treated with solution and 137 with tablets. Auxological parameters, dose and thyroid function values at diagnosis, 3, 6, 12, 24, 36 months were not significantly different. TSH at 15 days (p=0.002) and 1 month (p=0.009) was significantly reduced in group D. At 2-year follow-up, median TSH was significantly lower in group T (p=0.03). No statistical difference was detected between the median Developmental Quotient, however group D showed lower values in the language subscale at 12 months and in eye-hand coordination at 36 months. Conclusions. Both therapeutic strategies are effective on treatment of CH. A higher risk of overtreatment in the first months of therapy seems to be associated with oral solution L-T4; therefore, a different strategy should be considered when starting and adjusting the dose. No negative effects in cognitive development were observed. The data obtained are encouraging but long-term follow-up is needed.

scholarly journals An Overview of Iron in Term Breast-Fed Infants

2015 ◽  
Vol 9 ◽  
pp. CMPed.S26572 ◽  
Author(s):  
Wafaa A. Qasem ◽  
James K. Friel
Keyword(s):  
Current Knowledge ◽  
Normal Growth ◽  
Psychomotor Development ◽  
Negative Effects ◽  
Normal Term ◽  
Long Term Follow Up ◽  
Essential Nutrient ◽  
Breast Fed

Background Iron is an essential nutrient for normal growth and neurodevelopment of infants. Iron deficiency (ID) remains the most common micronutrient deficiency worldwide. There are convincing data that ID is associated with negative effects on neurological and psychomotor development. Objectives In this review, we provide an overview of current knowledge of the importance of iron in normal term breast-fed infants with a focus on recommendations, metabolism, and iron requirements. Conclusions Health organizations around the world recommend the introduction of iron-rich foods or iron supplements for growing infants to prevent ID. However, there is no routine screening for ID in infancy. Multicenter trials with long-term follow-up are needed to investigate the association between iron fortification/supplementation and various health outcomes.

Treatment Strategy and Long Term Follow Up of Congenital Hypothyroidism

1989 ◽  
pp. 245-252 ◽  
Author(s):  
G. Van Vliet ◽  
Th. Barboni ◽  
M. Klees ◽  
F. Cantraine ◽  
R. Wolter
Keyword(s):  
Congenital Hypothyroidism ◽  
Treatment Strategy ◽  
Long Term Follow Up

scholarly journals Long Term Follow-Up of Borderline Congenital Hypothyroidism † 409

1998 ◽  
Vol 43 ◽  
pp. 73-73
Author(s):  
A Daliva ◽  
B Linder ◽  
J Martino-Nardi ◽  
P Saenger
Keyword(s):  
Congenital Hypothyroidism ◽  
Long Term Follow Up

scholarly journals Neonatal Screening for Congenital Hypothyroidism: What Can We Learn From Discordant Twins?

2019 ◽  
Vol 104 (12) ◽  
pp. 5765-5779 ◽  
Author(s):  
Emanuela Medda ◽  
Maria Cristina Vigone ◽  
Alessandra Cassio ◽  
Francesca Calaciura ◽  
Pietro Costa ◽  
...  
Keyword(s):  
Environmental Factors ◽  
Congenital Hypothyroidism ◽  
Screening Program ◽  
Phenotypic Variance ◽  
Fetal Life ◽  
Long Term Follow Up ◽  
Discordant Twins ◽  
Discordant Twin

Abstract Context Newborn screening program for congenital hypothyroidism (CH) adopting rescreening in at-risk neonates. Objectives To estimate the concordance rate for CH in twin pairs discordant at the first screening; to verify whether long-term follow-up of healthy cotwins belonging to CH discordant pairs may be useful to diagnose thyroid hypofunction during development; to evaluate the importance of genetic and environmental influences on liability to permanent and transient CH. Design and Patients Forty-seven screening discordant twin pairs were investigated. Proband was defined as the twin in the pair with a positive test at the first screening and a confirmed diagnosis of CH. Results Seven screening discordant twin pairs became concordant for CH within the first month of life (pairwise concordance of 14.9%) because seven screening negative cotwins showed high TSH values when retested. During long-term follow-up (range, 3 to 21 years), hypothyroidism was diagnosed in two monozygotic screening negative cotwins at the age of 9 months and 12 years, respectively. Furthermore, the twin analysis showed that 95% of liability to transient CH was explained by genetic factors and 5% by environmental (unshared) factors, whereas 64% of phenotypic variance of permanent CH was explained by common environmental factors (shared during the fetal life) and 36% by unshared environmental factors. Conclusions This study showed that the introduction of rescreening permits the diagnosis of CH in a greater number of twins. It also showed the importance of long-term follow-up in both twins in the pair, and the role of nongenetic factors in the etiology of permanent CH.

scholarly journals Examinations under anaesthesia as a measure of disease burden in unilateral retinoblastoma: the London experience

2019 ◽  
Vol 104 (1) ◽  
pp. 17-22 ◽  
Author(s):  
Ido Didi Fabian ◽  
Vishal Shah ◽  
Noa Kapelushnik ◽  
Zishan Naeem ◽  
Zerrin Onadim ◽  
...  
Keyword(s):  
Disease Burden ◽  
Conservative Therapy ◽  
Primary Treatment ◽  
Long Term Follow Up ◽  
Intraocular Retinoblastoma ◽  
Group B ◽  
Group D

BackgroundEarly diagnosis strategies and advances in retinoblastoma (Rb) management have resulted in nearly 100% survival. More attention should, therefore, be given to quality of life considerations. We aimed to quantify the number of examinations under anaesthesia (EUAs) in a cohort of patients with Rb, as a measure of disease burden.MethodsA retrospective analysis of patients with unilateral Rb that presented to the London Rb service from 2006 to 2013, were treated and had long-term follow-up. Correlations of clinical variables to number of EUAs were investigated.ResultsA total of 107 patients with Rb were included that presented at a mean age of 26.51 ± 22.68 months. The International Intraocular Retinoblastoma Classification (IIRC) was group B in 5 (5%), C in 13 (12%), D in 48 (45%) and E in 41 (38%) of the cases. Primary treatment was intravenous chemotherapy in 36 (34%) and enucleation in 71 (66%) of the cases. Mean number of EUAs was 20.67 ± 6.62, 12.52 ± 6.23 and 11.15 ± 6.91 for combined groups B/C, group D and group E patients (p < 0.001), respectively. On analysis, early age atpresentation and conservative treatments were found to significantly correlate with increased number of EUAs (p < 0.001). Mean follow-up time was 74.42 ± 25.16 months and no metastasis or death were reported.ConclusionFamilies should be counselled regarding the number of EUAs associated with the patient's IIRC group, with B/C eyes undergoing twice the number as compared with D/E eyes. For group D cases, where both enucleation and conservative therapy are valid options, treatment choice has a significant impact on the number of EUAs.

scholarly journals Congenital Hypothyroidism Long-Term Follow-up Project: Navigating the Rough Waters of a Multi-Center, Multi-State Public Health Project

2014 ◽  
Vol 24 (3) ◽  
pp. 464-472 ◽  
Author(s):  
Kupper Wintergerst ◽  
Gina Gembel ◽  
Tracey Kreipe ◽  
Patrick Zeller ◽  
Erica Eugster ◽  
...  
Keyword(s):  
Public Health ◽  
Congenital Hypothyroidism ◽  
Long Term Follow Up ◽  
Health Project

scholarly journals A molecular analysis and long-term follow-up of two siblings with severe congenital hypothyroidism carrying the IVS30+1G>T intronic thyroglobulin mutation

2008 ◽  
Vol 52 (8) ◽  
pp. 1337-1344 ◽  
Author(s):  
Ileana G. S. Rubio ◽  
Ana Luiza Galrao ◽  
Viviane Pardo ◽  
Meyer Knobel ◽  
Roberta F. Possato ◽  
...  
Keyword(s):  
Molecular Analysis ◽  
Congenital Hypothyroidism ◽  
Thyroid Tissue ◽  
Endoplasmatic Reticulum ◽  
Iodine Nutrition ◽  
Genes Expression ◽  
Long Term Follow Up ◽  
Tissue Specimens

OBJECTIVE: To extend the molecular analysis of the IVS30+1G>T intronic thyroglobulin (TG) mutation, and to report the eleven year follow-up of the affected patients. METHOSD: Two siblings with severe congenital hypothyroidism with fetal and neonatal goiter, harboring the IVS30+1G>T mutation were included. Nodular and non-nodular thyroid tissue specimens were collected. Specific thyroid genes expression was evaluated by real-timePCR and by immunohistochemistry. RESULTS: In non-nodular tissue specific thyroid genes mRNA were reduced when compared to normal thyroid sample. In the nodule, TPO and NIS expression was very low. Microscopic examinations showed very large follicular-lumina and swollen vesicles of endoplasmatic-reticulum. Strong cytoplasmatic and low follicular-lumen TG immunostaining were detected. Intracellular NIS, membrane TPO and TSHR immunostaining had higher positivity in non-nodular sample. Both patients had a long-term adequate developmental outcome, besides one patient have been lately-treated. CONCLUSIONS: IVS30+1G>T mutation not only lead to very enlarge endoplasmatic-reticulum, but also to alterations of specific thyroid genes expression. The clinical evolution of patients harboring these mutations strengthen the concept of the influence of environment, like iodine nutrition, to determine the final phenotypic appearance.

Therapy and prevention for mental health: What if mental diseases are mostly not brain disorders?

2019 ◽  
Vol 42 ◽  
Author(s):  
John P. A. Ioannidis
Keyword(s):  
Mental Health ◽  
Mental Disease ◽  
Brain Disorders ◽  
Social Stressors ◽  
Labor Education ◽  
Mental Diseases ◽  
Long Term Follow Up ◽  
Political Decisions

AbstractNeurobiology-based interventions for mental diseases and searches for useful biomarkers of treatment response have largely failed. Clinical trials should assess interventions related to environmental and social stressors, with long-term follow-up; social rather than biological endpoints; personalized outcomes; and suitable cluster, adaptive, and n-of-1 designs. Labor, education, financial, and other social/political decisions should be evaluated for their impacts on mental disease.

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