-
The Cochrane Database of Systematic... 2000Iodine deficiency is the leading preventable cause of intellectual impairment in the world. Although iodine supplementation is generally considered to be safe, there is... (Review)
Review
BACKGROUND
Iodine deficiency is the leading preventable cause of intellectual impairment in the world. Although iodine supplementation is generally considered to be safe, there is a possibility of high doses of iodine suppressing maternal thyroid function.
OBJECTIVES
The objective of this review was to assess the effects of iodine supplementation before or during pregnancy in areas of iodine deficiency.
SEARCH STRATEGY
We searched the Cochrane Pregnancy and Childbirth Group trials register.
SELECTION CRITERIA
All acceptably controlled trials of maternal iodine supplementation during pregnancy with clinical outcomes.
DATA COLLECTION AND ANALYSIS
Eligibility and trial quality were assessed by two reviewers.
MAIN RESULTS
Three trials involving 1551 women were included. In two trials, iodine supplementation was associated with a statistically significant reduction in deaths during infancy and early childhood (relative risk 0.71, 95% confidence interval 0. 56 to 0.90). Iodine supplementation was associated with decreased prevalence of endemic cretinism at the age of four years (relative risk 0.27, 95% confidence interval 0.12 to 0.60) and better psychomotor development scores between four to 25 months of age.
REVIEWER'S CONCLUSIONS
Iodine supplementation in a population with high levels of endemic cretinism results in an important reduction in the incidence of the condition with no apparent adverse effects.
Topics: Child, Preschool; Congenital Hypothyroidism; Deficiency Diseases; Dietary Supplements; Female; Humans; Infant; Iodine; Preconception Care; Pregnancy; Prenatal Care
PubMed: 10796152
DOI: 10.1002/14651858.CD000135 -
European Journal of Endocrinology May 2011Context Recombinant human GH (rhGH) is prescribed for the treatment of adults with GH deficiency (GHD). However, conflicting data are available on the efficacy of rhGH... (Review)
Review
Context Recombinant human GH (rhGH) is prescribed for the treatment of adults with GH deficiency (GHD). However, conflicting data are available on the efficacy of rhGH treatment in elderly GHD patients. Objective To assess the efficacy of rhGH treatment in elderly GHD subjects. Methods We searched the available literature in PubMed, Cochrane Library, Web of Science and EMBASE. Study selection Studies on GHD patients, aged >60 years, treated with rhGH were eligible for inclusion. Data extraction was performed by two reviewers independently. Results We found 11 eligible studies with a total of 534 patients. Only two studies had prospective, randomized, placebo-controlled study designs of rhGH treatment with a duration of 6 (n=15) and 12 months (n=62), respectively. Treatment with rhGH decreased total and low density lipoprotein (LDL) cholesterol levels by 4-8 and 11-16%, respectively, but did not alter high density lipoprotein or triglyceride levels. RhGH did not affect body mass index, but decreased waist circumference (by ∼3 cm) and waist/hip ratio. RhGh did not consistently affect blood pressure or bone mineral density. RhGH increased lean body mass by 2-5% and decreased total fat mass by 7-10% in four studies, but did not affect body composition in two other studies. RhGH consistently improved quality of life (QoL) parameters reflected in AGHDA-scores. There were no explicit data on elderly GHD patients aged >80 years. Conclusion RhGH replacement in elderly subjects with GHD decreases LDL cholesterol levels and improves QoL, but the effects on other parameters are not unequivocal. There were no data on the efficacy and safety of rhGH treatment in octogenarians with GHD.
Topics: Age Factors; Aged; Animals; Dwarfism, Pituitary; Human Growth Hormone; Humans; Randomized Controlled Trials as Topic; Recombinant Proteins
PubMed: 21339335
DOI: 10.1530/EJE-10-1170 -
Hormone Research in Paediatrics 2023The link between the effects of recombinant human growth hormone (rhGH) therapy in patients with growth hormone deficiency (GHD) and Chiari malformation type I (CM-1) is...
BACKGROUND
The link between the effects of recombinant human growth hormone (rhGH) therapy in patients with growth hormone deficiency (GHD) and Chiari malformation type I (CM-1) is controversial.
SUMMARY
We report the case of a patient with an unusual association of GHD due to ectopic posterior pituitary and CM-1. Our patient developed a headache and worsening of CM-1 after the initiation of rhGH therapy. Following an atlo-occipital decompression surgery, the patient was able to resume therapy with a marked growth improvement. Based on this observation, we provide a systematic review of the current literature about these two pathologies.
KEY MESSAGES
A careful follow-up of all patients with CM-1 treated with GH is mandatory, paying particular attention to the appearance of any neurological signs and symptoms.
Topics: Humans; Arnold-Chiari Malformation; Human Growth Hormone; Dwarfism, Pituitary; Recombinant Proteins
PubMed: 36001954
DOI: 10.1159/000526617 -
The Cochrane Database of Systematic... Nov 2018Iodine deficiency is the main cause of potentially preventable mental retardation in childhood, as well as causing goitre and hypothyroidism in people of all ages. It is...
BACKGROUND
Iodine deficiency is the main cause of potentially preventable mental retardation in childhood, as well as causing goitre and hypothyroidism in people of all ages. It is still prevalent in large parts of the world.
OBJECTIVES
To assess the effects of iodine supplementation overall, and of different forms and dosages of iodine supplementation separately, in the prevention of iodine deficiency disorders in children.
SEARCH METHODS
The Cochrane Library, MEDLINE, EMBASE and reference lists, databases of ongoing trials and the Internet were searched.
SELECTION CRITERIA
We included randomised controlled trials and prospective controlled trials not using randomisation of iodine supplementation in children living in areas of iodine deficiency.
DATA COLLECTION AND ANALYSIS
Two reviewers did the initial data selection and quality assessment of trials independently. As the studies identified were not sufficiently similar and not of sufficient quality, we did not do a meta-analysis but summarised the data in a narrative format.
MAIN RESULTS
Twenty-six prospective controlled trials were related to our question, assessing a total of 29613 children. Twenty of them were classified as being of low quality, six of moderate quality. Most studies used iodised oil as a supplement, but other supplements were also used. The intervention groups were compared to a non-supplemented control group, different doses or different forms of iodine supplementation.There was a clear tendency towards goitre reduction with iodine supplementation; this was significant in several studies. Significant differences in physical development were not seen, except in one study. Results for differences in cognitive and psychomotor measures were mixed, with only few studies showing a positive intervention effect. One study suggested that infant mortality was lowered after iodine supplementation.Most studies showed a significant increase in urinary iodine excretion and levels recommended by the WHO were reached in most cases after supplementation. Thyroid-stimulating hormone (TSH) levels were significantly reduced in one study. In 1.8% of the children investigated, adverse effects were found, most of them were minor and transient.
AUTHORS' CONCLUSIONS
Despite most of the included studies being of low quality, the results suggest that iodine supplementation, especially iodised oil, is an effective means of decreasing goitre rates and improving iodine status in children. Indications of positive effects on physical and mental development and mortality were seen, although results were not always significant. Adverse effects were generally minor and transient. Insufficient evidence was available on non-oil supplements. High quality controlled studies investigating relevant long term outcome measures are needed to address the question of the best form of iodine supplementation in different population groups and settings.
Topics: Child; Congenital Hypothyroidism; Controlled Clinical Trials as Topic; Dietary Supplements; Goiter; Humans; Iodates; Iodine; Iodized Oil; Myxedema; Potassium Compounds; Potassium Iodide; Sodium Chloride, Dietary
PubMed: 30489630
DOI: 10.1002/14651858.CD003819.pub3 -
Hormone Research in Paediatrics 2011The US Food and Drug Administration approved use of recombinant human growth hormone (GH) for the treatment of idiopathic short stature (ISS) in children; however, few... (Review)
Review
The US Food and Drug Administration approved use of recombinant human growth hormone (GH) for the treatment of idiopathic short stature (ISS) in children; however, few studies have evaluated metabolic outcomes. This article addresses whether children with ISS treated with GH experience the same metabolic benefits as children with GH deficiency (GHD) treated with GH. A systematic review of all published studies of GH treatment in children with ISS that included data on metabolic outcomes identified five studies. No meta-analysis has been performed.Studies show a metabolic response to GH treatment in children with ISS similar to that observed in children with GHD; effects include a transient decrease in insulin sensitivity and a dose-dependent increase in insulin-like growth factor I. However, no increase in the risk of diabetes was found. Children with ISS seem to benefit from GH treatment in terms of height gain without any severe negative metabolic outcomes.
Topics: Adipose Tissue; Adolescent; Body Height; Child; Dwarfism; Dwarfism, Pituitary; Glucose; Growth Disorders; Human Growth Hormone; Humans; Insulin; Insulin Resistance; Insulin-Like Growth Factor I; Leptin; Lipid Metabolism; Male; Recombinant Proteins
PubMed: 21912170
DOI: 10.1159/000330165 -
Journal of Endocrinological... Sep 2013Congenital hypothyroidism (CH) is a frequent disease occurring with an incidence of about 1/2500 newborns/year. In 80-85% of the cases CH is caused by alterations in... (Review)
Review
BACKGROUND
Congenital hypothyroidism (CH) is a frequent disease occurring with an incidence of about 1/2500 newborns/year. In 80-85% of the cases CH is caused by alterations in thyroid morphogenesis, generally indicated by the term "thyroid dysgenesis" (TD). TD is generally a sporadic disease, but in about 5% of the cases a genetic origin has been demonstrated. In these cases, mutations in genes playing a role during thyroid morphogenesis (NKX2-1, PAX8, FOXE1, NKX2-5, TSHR) have been reported.
AIM
This work reviews the main steps of thyroid morphogenesis and all the genetic alterations associated with TD and published in the literature.
Topics: Animals; Congenital Hypothyroidism; DNA-Binding Proteins; Female; Forkhead Transcription Factors; Homeobox Protein Nkx-2.5; Homeodomain Proteins; Humans; Male; Mice; Nuclear Proteins; PAX8 Transcription Factor; Paired Box Transcription Factors; Receptors, Thyrotropin; Thyroid Dysgenesis; Thyroid Gland; Thyroid Nuclear Factor 1; Transcription Factors
PubMed: 23698639
DOI: 10.3275/8973 -
Growth Hormone & IGF Research :... 2021We sought to obtain a better understanding of the burden of short stature using a systematic literature review.
OBJECTIVE
We sought to obtain a better understanding of the burden of short stature using a systematic literature review.
METHODS
Studies of the burden of short stature, of any cause in adults and children, were searched using Embase, MEDLINE and Cochrane databases in April 2020, capturing publications from 2008 onwards. Case series and populations with adult-onset growth hormone deficiency (GHD) were excluded.
RESULTS
Of 1684 publications identified, 41 studies (33 in children, 8 in adults) were included. All studies assessed human burden. Most study populations in children included short stature due to GHD, idiopathic short stature (ISS) and short stature after being born small for gestational age (SGA). In these populations, four studies showed that quality of life (QoL) in children with short stature was significantly worse than in children with normal stature. A significant association between QoL and short stature was observed in children with chronic kidney disease (CKD) (3 studies), achondroplasia (1 study) and transfusion-dependent β-thalassaemia (1 study), and in samples with mixed causes of short stature (3 studies). Three studies (one in GHD/ISS/SGA and two in CKD) found no significant association between short stature and QoL, and several studies did not report statistical significance. Approximately half of adult studies showed that QoL was reduced with short stature, and the other half showed no association. Two studies, one in adults with Prader-Willi syndrome and one in children with GHD, suggested a potential association between short stature and poorer cognitive outcomes. Three studies demonstrated an increased caregiver burden in parents of children with short stature.
CONCLUSIONS
Evidence suggests that, compared with those with normal stature, children and adults with short stature of any cause may experience poorer QoL. Further research could extend our understanding of the human burden in this field.
Topics: Achondroplasia; Adult; Body Height; Caregiver Burden; Child; Cost of Illness; Growth Disorders; Human Growth Hormone; Humans; Infant, Small for Gestational Age; Parents; Quality of Life; Renal Insufficiency, Chronic; beta-Thalassemia
PubMed: 33975197
DOI: 10.1016/j.ghir.2021.101392 -
Frontiers in Endocrinology 2022Pediatric patients with growth hormone deficiency (GHD) are currently treated with daily injections of recombinant human growth hormone (rhGH) to promote linear growth...
Treatment Adherence to Injectable Treatments in Pediatric Growth Hormone Deficiency Compared With Injectable Treatments in Other Chronic Pediatric Conditions: A Systematic Literature Review.
BACKGROUND
Pediatric patients with growth hormone deficiency (GHD) are currently treated with daily injections of recombinant human growth hormone (rhGH) to promote linear growth and enable attainment of normal adult height. One of the main reasons for suboptimal growth during rhGH therapy is non-adherence to treatment. The objective of this systematic literature review was to examine the recent literature on pediatric adherence to injectable treatments for chronic conditions (focusing on rhGH) to characterize levels of adherence and identify the factors/barriers associated with adherence.
METHODS
The Embase and MEDLINE databases (January 2015-October 2020) were searched to identify publications describing studies of pediatric patients (aged ≤17 years) with GHD and other chronic conditions requiring daily or weekly injectable treatments; a similar targeted search of Chinese literature was also performed. Adherence data were extracted from the included studies and summarized. Risk of bias was determined using the Cochrane Risk of Bias tool 2 or the Newcastle-Ottawa Scale.
RESULTS
A total of 23 publications were included, with all publications except for one (multiple sclerosis) focused on pediatric GHD studies: there were two clinical trials, 18 observational studies and three survey studies. Study sample sizes ranged from 30 to 13,553 patients (median: 95 patients). The definition of adherence varied between studies and included mean adherence rate, median adherence rate, and the percentage of patients within pre-specified adherence categories. Of the publications assessing adherence to daily rhGH, 11 studies reported 12-month mean adherence rate (range: 73.3%- 95.3%) and eight studies reported median adherence (range: 91%- 99.2%). The barriers to treatment adherence identified included self-administration, increased administration frequency, age (adolescence), longer treatment duration, device design, and insufficient family education, awareness, and/or engagement. Recommendations for increasing adherence included using adherence reminder tools, increasing patient engagement/education, and improving injection device design and drug product.
CONCLUSIONS
Adherence to rhGH treatment was high (>80%) for many studies, though comparability between studies was limited given the substantial heterogeneity in the way adherence was defined, measured, and reported. To address this heterogeneity, we recommend standardizing how adherence is defined and reported and encourage the use of standardized study designs and outcome measures.
Topics: Adolescent; Adult; Child; Dwarfism, Pituitary; Human Growth Hormone; Humans; Recombinant Proteins; Surveys and Questionnaires; Treatment Adherence and Compliance
PubMed: 35299969
DOI: 10.3389/fendo.2022.795224 -
BMJ (Clinical Research Ed.) Mar 2011To systematically determine the impact of growth hormone therapy on adult height of children with idiopathic short stature. (Review)
Review
OBJECTIVE
To systematically determine the impact of growth hormone therapy on adult height of children with idiopathic short stature.
DESIGN
Systematic review.
DATA SOURCES
Cochrane Central Register of Controlled Trials, Medline, and the bibliographic references from retrieved articles of randomised and non-randomised controlled trials from 1985 to April 2010.
DATA EXTRACTION
Height in adulthood (standard deviation score) and overall gain in height (SD score) from baseline measurement in childhood.
STUDY SELECTION
Randomised and non-randomised controlled trials with height measurements for adults. Inclusion criteria were initial short stature (defined as height >2 SD score below the mean), peak growth hormone responses >10 μg/L, prepubertal stage, no previous growth hormone therapy, and no comorbid conditions that would impair growth. Adult height was considered achieved when growth rate was <1.5 cm/year or bone age was 15 years in females and 16 years in males.
RESULTS
Three randomised controlled trials (115 children) met the inclusion criteria. The adult height of the growth hormone treated children exceeded that of the controls by 0.65 SD score (about 4 cm). The mean height gain in treated children was 1.2 SD score compared with 0.34 SD score in untreated children. A slight difference of about 1.2 cm in adult height was observed between the two growth hormone dose regimens. In the seven non-randomised controlled trials the adult height of the growth hormone treated group exceeded that of the controls by 0.45 SD score (about 3 cm).
CONCLUSIONS
Growth hormone therapy in children with idiopathic short stature seems to be effective in partially reducing the deficit in height as adults, although the magnitude of effectiveness is on average less than that achieved in other conditions for which growth hormone is licensed. The individual response to therapy is highly variable, and additional studies are needed to identify the responders.
Topics: Adolescent; Adult; Body Height; Child; Dwarfism; Female; Human Growth Hormone; Humans; Male; Randomized Controlled Trials as Topic; Treatment Outcome
PubMed: 21398350
DOI: 10.1136/bmj.c7157 -
Clinical Endocrinology Nov 2021The potential of harm to infants or their parents from a false positive (FP) newborn screening (NBS) result for congenital hypothyroidism (CH) is often cited as an...
OBJECTIVES
The potential of harm to infants or their parents from a false positive (FP) newborn screening (NBS) result for congenital hypothyroidism (CH) is often cited as an argument against lowering of screening thresholds for CH. This systematic review (SR) examines the evidence of harm and factors that possibly contribute.
STUDY DESIGN
PRISMA guidelines were followed and the protocol was registered online (Prospero, ID CRD42019123950, 20 August 2019) before the search was conducted. Multiple electronic databases and grey literature were searched. Articles were included/excluded based on predetermined eligibility criteria. Included articles were appraised for quality, using the relevant Critical Appraisal Skills Program (CASP) tool. Data were extracted and results were tabulated and summarised as part of a narrative synthesis.
RESULTS
A total of six studies met the inclusion criteria. All were qualitative and three were based on the same cohort. Studies were published between 1983 and 1996. CASP appraisals scored 2/6 studies as moderate quality and 4/6 as low quality. Studies reported that FP results on CH screening may cause initial stress for parents and poorly defined behavioural disturbance in a small number of children, though these effects were generally not long-lasting. Poor screening processes and inadequate communication with parents, increased the risk of harm to parents and children, from FP results.
CONCLUSION
This SR found a small number of dated, qualitative studies of low to moderate quality, conducted soon after the initiation of NBS for CH. Conclusive evidence of the risks of harm from FP results and ways to mitigate harm, awaits further, well-designed studies.
Topics: Child; Cohort Studies; Congenital Hypothyroidism; Humans; Infant; Infant, Newborn; Neonatal Screening
PubMed: 34302303
DOI: 10.1111/cen.14562