2.Advancement in the research of growth hormone in treating pediatric burn patients.
Chinese Journal of Burns 2013;29(1):18-21
The serious and persistent hypercatabolic state caused by severe burn injury leads to weight loss, weakening of muscles, immunity decline, and delayed wound healing in injured patients. Pediatric burn patients also suffer growth retardation. How to ameliorate the persistent hypercatabolic response is what the medical professionals should concern. Recombinant human growth hormone (rhGH) can attenuate the hypercatabolic response and alleviate the growth retardation. This article reviews the researches on the use of rhGH in the treatment of burn injury in children.
Burns
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drug therapy
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Child
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Human Growth Hormone
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therapeutic use
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Humans
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Recombinant Proteins
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therapeutic use
4.Clinical effect of letrozole in treatment of idiopathic short stature in adolescent boys.
Chinese Journal of Contemporary Pediatrics 2019;21(10):977-982
OBJECTIVE:
To evaluate the therapeutic effect and safety of letrozole in the treatment of adolescent boys with idiopathic short stature (ISS).
METHODS:
A retrospective analysis was performed for the clinical data of 16 adolescent boys with ISS who had a bone age of ≥14 years. Among these boys, 8 were initially treated with recombinant human growth hormone (rhGH), followed by rhGH combined with letrozole during a bone age of 14-15.5 years. The other 8 boys were initially treated with rhGH combined with letrozole since their bone age was ≥14 years at diagnosis. Of the 16 boys, 16 were treated for not less than 6 months, 12 were treated for not less than 1 year, and 5 were treated for not less than 1.5 years. The increase in bone age, predicted adult height (PAH), final adult height, sex hormones, and adverse reactions after treatment were analyzed.
RESULTS:
After 6 months, 1 year, and 1.5 years of treatment, median bone age was increased by 0 year, 0.5 year, and 0.5 year respectively, which was significantly lower than the increase in age (P<0.05). There was a significant increase in PAH after treatment (P<0.05). Seven boys reached final height, which was significantly higher than PAH before treatment (P<0.05). All the 16 boys had significant increases in luteinizing hormone, follicle-stimulating hormone, and testosterone levels after treatment (P<0.05), with a significant reduction in the estradiol level and a significant increase in the insulin level at 1 year of treatment (P<0.05). There was a significant increase in the insulin-like growth factor-1 level at 6 months and 1 year of treatment (P<0.05). There were no significant changes in blood glucose, blood lipids, uric acid, and the three indices for thyroid function as monitored during treatment (P>0.05).
CONCLUSIONS
In adolescent boys with ISS and a high bone age, rhGH combined with letrozole can safely and effectively delay the increase in bone age and improve PAH and final adult height, with little adverse effect.
Adolescent
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Body Height
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Dwarfism
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Growth Disorders
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Human Growth Hormone
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Humans
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Letrozole
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therapeutic use
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Male
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Retrospective Studies
6.Sensitivity of supplementation of thyroid hormone on treatment of idiopathic short-stature children during therapy with recombinant human growth hormone.
Wei WANG ; Shuqin JIANG ; Zhirui CUI ; Xiangyang LUO ; Lingli SHI ; Heli ZHENG
Frontiers of Medicine 2018;12(5):580-585
This study aimed to evaluate the effects of thyroid hormone supplementation on growth rate of children with idiopathic short stature (ISS) and low-normal serum free thyroxine FT4 who were receiving growth hormone therapy. We selected 64 prepubertal children with FT4 levels in the lowest third of the normal range as the lower FT4 group, and these children were divided randomly into two subgroups: L-thyroxine (L-T4)-treated subgroup was treated with L-T4 (0.5-3.0 g/(kg·d)) from the beginning of the study, and the non-L-T4-treated subgroup received placebo. We also selected 39 ISS children with FT4 in the upper two-thirds of the normal range as the higher FT4 group. During the first year, the lower FT4 group featured lower FT3, FT4, thyroid stimulating hormone (TSH), and insulin-like growth factor-I standard deviation score (IGF-I SDS) and significantly lower height velocity (HV) compared with the higher FT4 group. However, in the lower FT4 group, the L-T4-treated subgroup presented higher FT4, FT3, TSH, and IGF-I SDS concentrations and significantly higher HV compared with children in the non-L-T4-treated subgroup. In children with ISS, the negative effect of thyroid hormone deficiency on growth rate should be considered when FT4 level lies in the low-normal range prior to recombinant human growth hormone treatment.
Child
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Female
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Growth Disorders
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blood
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drug therapy
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Human Growth Hormone
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therapeutic use
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Humans
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Insulin-Like Growth Factor I
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metabolism
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Male
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Recombinant Proteins
;
therapeutic use
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Thyrotropin
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blood
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Thyroxine
;
blood
7.Prader-Willi syndrome: A case report.
Chinese Journal of Contemporary Pediatrics 2006;8(6):521-523
8.Long-term follow-up of isolated-growth hormone deficiency typeIA: the clinical analysis of 2-sister cases.
Guo-ying CHANG ; Zhi-ya DONG ; Wei WANG ; De-fen WANG
Chinese Journal of Pediatrics 2010;48(12):944-946
OBJECTIVETo elucidate the curative and adverse effect of recombinant human growth hormone (rhGH) in 2 patients with isolated-growth hormone deficiency type IA (IGHDIA), to track sexual development and pregnancy, and reassess the quality of life in the adulthood.
METHODThe authors summarized the data of 2-sister cases with IGHDIA; followed up for assessment of height, weight, blood pressure and sexual development; detected fasting blood lipids, glucose, insulin, insulin growth factor-1 (IGF-1) and insulin-like growth factor binding protein-3 (IGFBP-3); made an investigation of education and occupation, and so on.
RESULTAfter 6.2 and 7.3 years treatment with rhGH, the two sisters had considerably improved height from -7.8 SDS, -8.8 SDS to -2.6 SDS and -1.3 SDS respectively. No evident side effect was observed. They had normal sexual development and pregnancy. The levels of IGF-1 and IGFBP-3 were still low, in the elder sister they were 46.6 µg/L, 2460 µg/L, and in the younger 52.4 µg/L, 2430 µg/L. No hyperlipidemia, diabetes or obesity occurred.
CONCLUSIONLong term therapy with rhGH may improve final adult height of individuals with IGHDIA. They can have normal sexual development and pregnancy. Metabolic syndrome did not occur during the follow-up period.
Child ; Dwarfism, Pituitary ; classification ; therapy ; Female ; Follow-Up Studies ; Human Growth Hormone ; therapeutic use ; Humans ; Siblings
9.Efficacy of letrozole in treatment of male adolescents with idiopathic short stature.
Yanhong LI ; Minlian DU ; Huamei MA ; Qiuli CHEN ; Hongshan CHEN ; Jun ZHANG
Journal of Zhejiang University. Medical sciences 2020;49(3):308-314
To evaluate the efficacy and safety of aromatase inhibitor letrozole in treatment of male adolescents with idiopathic short stature (ISS). Seventy five boys with height less than 2 standard deviation (SD) below the mean who had entered puberty were enrolled in our study from 2004 to 2017, in the Pediatric Department of the First Affiliated Hospital, Sun Yat-Sen University. Among 75 patients, 28 in letrozole group received letrozole and spironolactone, 30 in gonadotrophin releasing hormone analogue (GnRHa) group received GnRHa injection and 17 had no intervention. Height velocity (HV), increment of bone age/chronological age (ΔBA/ΔCA), the final adult height (FAH) were compared among groups and the safety of letrozole treatment was evaluated. HV maintained faster during letrozole treatment when compared with other groups. HV during GnRHa treatment showed slightly decline in the first 6 months, but decreased remarkably after 6 months, and was significantly lower than that in letrozole group ( < 0.05). The maturation of BA slowed down in both letrozole and GnRHa groups. But the ΔBA/ΔCA in letrozole group during the first and the second year of treatment were significantly higher (0.67±0.09, 0.50±0.15, respectively) when compared with GnRHa group (0.59±0.16, 0.44±0.13, respectively) ( =2.78 and 2.20, all < 0.05). FAH in letrozole group and GnRHa group were (170±4) cm and (170±6)cm, there was no significant differences between the two groups ( >0.05), and both were higher than that in no intervention group (162±4 cm, < 0.01). After 6 months of letrozole treatment, testicular volumes and serum testerone levels increased; 39.2% (11/28) boys had clinical manifestations of hyperandrogenemia, and 82.1% (23/28) boys had decreased serum high-density lipoprotein (HDL) levels. Serum levels of HDL and testerone returned normal and the hyperandrogenemia disappeared after the cessation of letrozole treatment. No significant changes in serum triglyceride, serum low-density lipoprotein (LDL), fating serum levels of insulin and glucose, HOMA-IR were observed. No abnormal liver function, myalgia, scoliosis or aggravations of scoliosis was found. Long term letrozole therapy during puberty in boys with ISS can delay bone maturation without significant decrease of linear growth, and thus can improve the final adult height. No severe adverse reactions were found.
Adolescent
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Body Height
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Bone Development
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Child
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Gonadotropin-Releasing Hormone
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Growth Disorders
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Humans
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Letrozole
;
therapeutic use
;
Male
10.Clinical characteristics of four children with 3M syndrome and a literature review.
Ningan XU ; Kangxiang LIU ; Yan ZHONG
Chinese Journal of Medical Genetics 2023;40(7):795-801
OBJECTIVE:
To analyze the clinical features of 3M syndrome and effect of growth hormone therapy.
METHODS:
Clinical data of four children diagnosed with 3M syndrome by whole exome sequencing at Hunan Children's Hospital from January 2014 to February 2022 were retrospectively analyzed, which included clinical manifestation, results of genetic testing and recombinant human growth hormone (rhGH) therapy. A literature review was also carried our for Chinese patients with 3M syndrome.
RESULTS:
The clinical manifestations of the 4 patients included severe growth retardation, facial dysmorphism and skeletal malformations. Two patients were found to harbor homozygous variants of CUL7 gene, namely c.4717C>T (p.R1573*) and c.967_993delinsCAGCTGG (p.S323Qfs*33). Two patients were found to harbor 3 heterozygous variants of the OBSL1 gene including c.1118G>A (p.W373*), c.458dupG (p.L154Pfs*1002) and c.690dupC (p.E231Rfs*23), among which c.967_993delinsCAGCTGG and c.1118G>A were unreported previously. Eighteen Chinese patients with 3M syndrome were identified through the literature review, including 11 cases (11/18, 61.1%) carrying CUL7 gene variants and 7 cases (7/18, 38.9%) carrying OBSL1 gene variants. The main clinical manifestations were in keeping with previously reported. Four patients were treated with growth hormone, 3 showed obvious growth acceleration, and no adverse reaction was noted.
CONCLUSION
3M syndrome has a typical appearance and obvious short stature. To attain accurate diagnosis, genetic testing should be recommended for children with a stature of less than -3 SD and facial dysmorphism. The long-term efficacy of growth hormone therapy for patients with 3M syndrome remains to be observed.
Humans
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Child
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Retrospective Studies
;
Dwarfism/genetics*
;
Muscle Hypotonia/genetics*
;
Growth Hormone/therapeutic use*
;
Cytoskeletal Proteins/genetics*