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1.
J Pediatr Endocrinol Metab ; 37(9): 779-788, 2024 Sep 25.
Artículo en Inglés | MEDLINE | ID: mdl-39135509

RESUMEN

OBJECTIVES: This study aimed to analyze the height growth pattern and the incidence of significant growth deceleration in girls with CPP and EFP on GnRHa treatment, and thereby identify relevant predictors of growth deceleration. METHODS: The data of 99 girls diagnosed with CPP and 47 girls with EFP were included in this retrospective analysis. The incidence of growth deceleration was calculated in both the first and second years. Multivariate logistic regression analysis was used to identify predictors indicative of growth deceleration. RESULTS: Growth velocity (GV) trajectories showed gradual decreases to the nadir at 18 months of treatment, and then they recovered till the 24th month of treatment, especially in girls with CPP. Nevertheless, the recovery was significantly greater in the CPP group than EFP. In the first year, no significant difference in the incidence of growth deceleration was found between the CPP group and the EFP group [17.35 vs. 25.53 %, p=0.249]; in the second year, the CPP group had a lower incidence than the EFP group [42.86 vs. 76.92 %, p=0.027]. The multivariate logistic regression analysis suggested that bone age (BA) was an independent predictor of growth deceleration (OR=2.264, 95 % CI: 1.268-4.042, p=0.006). The result of ROC curves showed the cut-off value of BA was 11.05 years. CONCLUSIONS: GV varies at different periods during GnRHa treatment. GnRHa should be used with more caution for EFP treatment than for CPP. BA can be used to predict the occurrence of growth deceleration during GnRHa treatment.


Asunto(s)
Estatura , Humanos , Femenino , Estudios Retrospectivos , Niño , Estudios Longitudinales , Estatura/efectos de los fármacos , Trastornos del Crecimiento/tratamiento farmacológico , Pubertad Precoz/tratamiento farmacológico , Pronóstico , Estudios de Seguimiento , Adolescente , Desarrollo Infantil/efectos de los fármacos
2.
J Pediatr Endocrinol Metab ; 37(8): 680-685, 2024 Aug 27.
Artículo en Inglés | MEDLINE | ID: mdl-39089289

RESUMEN

OBJECTIVES: Clinical benefits of growth hormone (GH) in Prader-Willi syndrome (PWS) are proven and scoliosis is a known association of both PWS and GH therapy. The aims of this study were to assess GH prescribing practices and growth outcomes over time, the prevalence and predictors of scoliosis in GH-treated PWS children, and the near-final height of GH-treated PWS patients. DESIGN AND METHODS: This is a retrospective, descriptive study evaluating data from all clinic visits of patients aged 0-18 years with PWS, seen through the Children's Hospital at Westmead between March 1992 and May 2022 (n=75). RESULTS: A total of 64 patients were treated with GH (visits = 1,414). In the recent decade, the diagnosis of PWS and GH commencement were made significantly earlier in life. The prevalence of scoliosis was 41 %, in which age was the only significant predictor for scoliosis (odds ratio 1.19: 95 % CI [1.08-1.31; p=0.001]) adjusted for other predictors. In patients with data available at the age 16 years (23/28 treated with GH), those who were GH treated had significantly higher height SDS vs. nontreated group (SDS -0.67 vs. -2.58; p=0.0001) and lower BMI SDS (1.18 vs. 2.37; p<0.001). CONCLUSIONS: Significant improvements in growth and body composition were seen in the GH-treated group vs. non-treated group of children with PWS. There were no significant modifiable clinical predictors of scoliosis in children with PWS, but our findings confirm the high prevalence of scoliosis in GH-treated children with PWS reinforcing the need for close surveillance.


Asunto(s)
Hormona de Crecimiento Humana , Síndrome de Prader-Willi , Escoliosis , Humanos , Síndrome de Prader-Willi/tratamiento farmacológico , Niño , Masculino , Femenino , Estudios Retrospectivos , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/administración & dosificación , Adolescente , Preescolar , Lactante , Escoliosis/epidemiología , Escoliosis/tratamiento farmacológico , Escoliosis/etiología , Recién Nacido , Estudios de Seguimiento , Pronóstico , Resultado del Tratamiento , Estatura/efectos de los fármacos , Centros de Atención Terciaria , Prevalencia
3.
J Coll Physicians Surg Pak ; 34(8): 932-935, 2024 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-39113512

RESUMEN

OBJECTIVE: To determine the auxological response to recombinant human growth hormone (rhGH) therapy in children with growth hormone deficiency (GHD) presenting at the National Institute of Child Health, Karachi, Pakistan. STUDY DESIGN:  Observational study. Place and Duration of the Study: Department of Paediatric Endocrinology, National Institute of Child Health, Karachi, Pakistan, from January 2022 to December 2023. METHODOLOGY:  All pre-pubertal children with short stature aged 3-12 years diagnosed with GHD and who were prescribed rhGH therapy were included in the study. Children with any other underlying reason for short stature or any other comorbidity were excluded. Patients' demographics and baseline growth parameters were recorded in a pre-designed proforma. Patients were then followed up every three months till one year. Response to rhGH therapy was evaluated through comparison of growth parameters before and after one year of therapy. RESULTS: A total of 90 children including 47 (52.2%) males and 43 (47.8%) females with GHD were enrolled. Mean age of these patients was 7.92 ± 2.647 years. A statistically significant change in height (SD), Weight (SD), and BMI (SD) was observed before and after one year of therapy (p <0.001). Response to therapy in terms of height did not differ significantly with respect to gender (p = 0.955) or stimulated growth hormone levels (p = 0.911). However, response to rhGH therapy was significantly better in terms of increase in height, weight, and BMI in patients presenting earlier i.e. at age ≤8 years. CONCLUSION: Recombinant human growth hormone therapy was effective in children with short stature to achieve desirable growth. Children diagnosed and treated at a younger age (≤8years) achieve better height outcomes as compared to those presenting late. KEY WORDS:  Short stature, Growth hormone deficiency, Recombinant human growth hormone.


Asunto(s)
Estatura , Trastornos del Crecimiento , Hormona de Crecimiento Humana , Proteínas Recombinantes , Humanos , Femenino , Masculino , Niño , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/deficiencia , Preescolar , Estatura/efectos de los fármacos , Proteínas Recombinantes/uso terapéutico , Trastornos del Crecimiento/tratamiento farmacológico , Pakistán , Resultado del Tratamiento , Enanismo Hipofisario/tratamiento farmacológico
4.
Eur J Pediatr ; 183(10): 4531-4539, 2024 Oct.
Artículo en Inglés | MEDLINE | ID: mdl-39158594

RESUMEN

Several evidence gaps exist regarding the use of long-acting polyethylene glycol recombinant human growth hormone (PEG-rhGH) in children with idiopathic short stature (ISS), particularly studies conducted in real-world settings, with long-term follow-up, involving varied dosing regimens, and in comparison with daily rhGH. The study aimed to evaluate the effectiveness, safety, and adherence of once-weekly PEG-rhGH for catch-up growth in children with prepubertal ISS compared to daily rhGH. A real-world retrospective cohort study was conducted in prepubertal children with ISS in China. Children who voluntarily received once-weekly PEG-rhGH or daily rhGH were included and were followed up for 2 years. Ninety-five children were included, 47 received PEG-rhGH 0.2-0.3 mg/kg weekly and 48 received daily rhGH. Outcome measures included effectiveness in catch-up growth, adverse events, and treatment adherence. Height velocity increased significantly in both groups during rhGH therapy. In children who received PEG-rhGH treatment, height velocity was 10.59 ± 1.37 cm/year and 8.75 ± 0.86 cm/year in the first and second year, respectively, which were significantly more than those who received daily rhGH (9.80 ± 1.05 cm/year, P = 0.002, and 8.03 ± 0.89 cm/year, P < 0.001). The height standard deviation score improved at the end of the second year for all children (P < 0.001). However, children who received PEG-rhGH showed more excellent improvement than those with daily rhGH (1.65 ± 0.38 vs. 1.50 ± 0.36, P = 0.001). In children who received PEG-rhGH, lower missed doses were observed than those with daily rhGH (0.75 ± 1.06 vs. 4.4 ± 2.0, P < 0.001). No serious adverse events were observed. CONCLUSION: PEG-rhGH demonstrated superior effectiveness and adherence compared to daily rhGH in the treatment of children with ISS. The safety profiles were similar between the two treatments. WHAT IS KNOWN: • Recombinant human growth hormone (rhGH) has been used to increase adult height in children with idiopathic short stature (ISS), and its safety profile is comparable to other indications for growth hormone treatment. • The use of long-acting rhGH in children with ISS is still an area of uncertainty. WHAT IS NEW: • This 2-year real-world study provides new evidence that PEGylated rhGH (PEG-rhGH) is more effective than daily rhGH in promoting catch-up growth in children with ISS. • PEG-rhGH also demonstrated superior treatment adherence compared to daily rhGH in children with ISS. • The safety profiles of PEG-rhGH and daily rhGH were found to be similar.


Asunto(s)
Estatura , Trastornos del Crecimiento , Hormona de Crecimiento Humana , Polietilenglicoles , Proteínas Recombinantes , Humanos , Estudios Retrospectivos , Masculino , Hormona de Crecimiento Humana/administración & dosificación , Hormona de Crecimiento Humana/uso terapéutico , Femenino , Niño , Trastornos del Crecimiento/tratamiento farmacológico , Polietilenglicoles/administración & dosificación , Polietilenglicoles/efectos adversos , Polietilenglicoles/uso terapéutico , Proteínas Recombinantes/administración & dosificación , Proteínas Recombinantes/uso terapéutico , Estatura/efectos de los fármacos , China , Resultado del Tratamiento , Preescolar , Estudios de Seguimiento , Esquema de Medicación
5.
J Clin Endocrinol Metab ; 109(10): e1955-e1958, 2024 Sep 16.
Artículo en Inglés | MEDLINE | ID: mdl-38958114

RESUMEN

The increased availability of recombinant human GH (rhGH), albeit at a relatively high cost, has increased a demand for treatment of children and adolescents of normal height to increase their adult stature. There are no scientific reports on the efficacy and safety of rhGH therapy in this condition; therefore, the authors comment on the possible causes and consequences based on their personal opinion and experience. As in gigantism, when GH action and end-organ are normal, enough GH is expected to result in increased growth velocity. Short-term adverse effects related to rhGH therapy for approved indications of short stature in children have been very rare. Data on long-term adverse effects are still scarce. A small increase in height might be statistically significant but not functionally or socially relevant. Considering that an increase in height represents more a desire than a need, physicians should emphasize the normality and qualities of these children, discuss with families the alternatives, such as counseling, and refrain from supporting the concept that taller is better.


Asunto(s)
Estatura , Trastornos del Crecimiento , Hormona de Crecimiento Humana , Humanos , Estatura/efectos de los fármacos , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/efectos adversos , Niño , Adolescente , Trastornos del Crecimiento/tratamiento farmacológico , Proteínas Recombinantes/uso terapéutico
6.
Artículo en Inglés | MEDLINE | ID: mdl-39026482

RESUMEN

INTRODUCTION: Short stature in growth hormone deficiency (GHD) can be treated with recombinant human growth hormone (rhGH), which is proven to be both safe and effective. However, a considerable number of patients does not achieve satisfying therapy outcomes. AIM OF THE STUDY: To evaluate the predictive effect of height increase in the first year of rhGH treatment on long-term therapy outcomes. MATERIAL AND METHODS: 165 short-stature children (mean age 10.72 ±3.33 years; 63% males), diagnosed with GHD, treated with rhGH for at least one year (mean follow-up 4.32 ±1.80 years), divided into 2 groups according to the change in height standard deviation score (SDS) after the first year of rhGH treatment: good responders (GR) and poor responders (PR). Then, in one-year intervals, patient's chronological age, bone age, height, weight, insulin-like growth factor level, and rhGH dose were all assessed. RESULTS: In the GR group, mean height velocity SDS up to five years of observation was 1.19 ±0.41/year and in the PR group 0.59 ±0.38/year. The differences were statistically significant (p < 0.05). CONCLUSIONS: The primary response to the rhGH treatment in GHD children seems to be a good predictor for long-term therapy outcomes.


Asunto(s)
Estatura , Hormona de Crecimiento Humana , Humanos , Niño , Masculino , Femenino , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/deficiencia , Estatura/efectos de los fármacos , Resultado del Tratamiento , Adolescente , Trastornos del Crecimiento/tratamiento farmacológico , Estudios de Seguimiento , Proteínas Recombinantes/uso terapéutico
7.
J Clin Res Pediatr Endocrinol ; 16(2): 235-242, 2024 May 31.
Artículo en Inglés | MEDLINE | ID: mdl-38828521

RESUMEN

A rarely reported phenomenon of rapid-tempo puberty in which the physical changes of puberty and testosterone levels increase very rapidly has not been reported outside apart from in two reviews. The resulting rapid advancement of skeletal age causes early completion of growth with shorter adult stature than expected. This appears to be genetic given its occurrence in the present report in two families, one with three brothers, one with two. We also describe potential treatments and found for the youngest that early initiation of standard therapy preserved or reclaimed adult height (AH) potential. The foreshortened AH in this situation involves rapidly advancing puberty resulting from high circulating testosterone levels leading to rapid advance in skeletal age. This was recognized earlier among younger brothers and treatment with gonadotropin-releasing analogues, growth hormone (GH) and/or aromatase inhibitor therapy (AIT) was tried. Two brothers in family A and family B were treated. Case 5 started treatment early enough so his AH was within target height (mid-parental height) range. Cases 2, 3, 4 were tried on GH and/or AIT with outcomes suggesting benefit. The prevalence and mechanism of rapid-tempo puberty requires further study. Furthermore, as illustrated by two of the current cases, this phenomenon may have a heightened prevalence, or at least may occur, in children previously diagnosed with constitutional delay of growth, underscoring the need to be cautious in assurance of a normal AH outcomes in this population, based on data from a single assessment.


Asunto(s)
Estatura , Pubertad , Adolescente , Adulto , Niño , Femenino , Humanos , Masculino , Inhibidores de la Aromatasa/uso terapéutico , Estatura/efectos de los fármacos , Hormona Liberadora de Gonadotropina/análogos & derivados , Trastornos del Crecimiento/tratamiento farmacológico , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/administración & dosificación , Pubertad/efectos de los fármacos , Pubertad/fisiología , Pubertad Precoz/tratamiento farmacológico , Testosterona/uso terapéutico , Testosterona/sangre , Testosterona/administración & dosificación
8.
Pediatr Transplant ; 28(5): e14803, 2024 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-38899494

RESUMEN

BACKGROUND: Growth retardation and short final height is a common complication of chronic kidney disease (CKD) beginning in childhood, with profound deleterious effects on quality of life, mental health, and social achievement. Despite optimal treatments of causative factors for growth retardation in children with CKD, more than 50% of patients reach end-stage renal failure with a height >2 SD below the mean, and most do not demonstrate "catch-up" growth after receiving a kidney transplant. Four decades ago, recombinant human growth hormone (rhGH) treatment was introduced after studies showed increased growth velocity and improved height SDS in uremic subjects. Since then, an abundance of published data showed significant improvements in health-related quality of life, and most studies revealed no significant adverse effects. Clinical practice guidelines recommended rhGH treatment in CKD Stages 3-5D and after transplantation. Despite these guidelines, this therapy remained underutilized. Most commonly cited barriers to the implementation of rhGH treatment were the need for daily injections, financial challenges, physicians' unfamiliarity with guidelines, and fear of adverse events. CONCLUSIONS: rhGH has been shown to improve growth and final height in short children with CKD, with minimal adverse effects. Despite data of its successful use generated over 3 decades, this treatment is underutilized. More judicious utilization of the treatment should emphasize educating patients, their care givers, and members of the multidisciplinary treating team. Additional studies are needed to assess the longer-term rhGH treatment in larger cohorts of patients, leading to additional supportive data and clearer recommendations.


Asunto(s)
Trastornos del Crecimiento , Hormona de Crecimiento Humana , Trasplante de Riñón , Calidad de Vida , Humanos , Niño , Hormona de Crecimiento Humana/uso terapéutico , Trastornos del Crecimiento/etiología , Estatura/efectos de los fármacos , Fallo Renal Crónico/cirugía , Fallo Renal Crónico/complicaciones , Insuficiencia Renal Crónica/complicaciones , Resultado del Tratamiento , Proteínas Recombinantes/uso terapéutico , Guías de Práctica Clínica como Asunto , Adolescente
9.
Medicine (Baltimore) ; 103(25): e38350, 2024 Jun 21.
Artículo en Inglés | MEDLINE | ID: mdl-38905369

RESUMEN

Treatment outcomes for different causes of childhood dwarfism vary widely, and there are no studies on the economic burden of treatment in relation to outcomes. This paper compared the efficacy and healthcare costs per unit height of recombinant human growth hormone (rhGH) for the treatment of growth hormone deficiency (GHD) and idiopathic short stature (ISS) with a view to providing a more cost-effective treatment option for children. We retrospectively analyzed 117 cases (66 cases of GHD and 51 cases of ISS) of short-stature children who first visited Weifang People's Hospital between 2019.1 and 2022.1 and were treated with rhGH for 1 to 3 years to track the treatment effect and statistically analyzed by using paired t tests, non-parametric tests, and chi-square tests, to evaluate the efficacy of rhGH treatment for GHD and ISS children and the medicinal cost. The annual growth velocity (GV) of children with GHD and ISS increased the fastest during 3 to 6 months after treatment and then gradually slowed down. The GV of the GHD group was higher than that of the ISS group from 0 to 36 months after treatment (P < .05 at 3, 6, 9, and 12 months); the height standard deviation scores (HtSDS) of the children in the GHD and ISS groups increased gradually with the increase of the treatment time, and the changes in the height standard deviation scores (ΔHtSDS) of the GHD group were more significant than those of the ISS group (P < .05 at 3, 6, 9, and 12 months). (2) The medical costs in the pubertal group for a 1-cm increase in height were higher than those of children in the pre-pubertal group at the same stage (3 to 24 months P < .05). The longer the treatment time within the same group, the higher the medical cost of increasing 1cm height. RhGH is effective in treating children with dwarfism to promote height growth, and the effect on children with GHD is better than that of children with ISS; the earlier the treatment time, the lower the medical cost and the higher the comprehensive benefit.


Asunto(s)
Estatura , Enanismo , Hormona de Crecimiento Humana , Proteínas Recombinantes , Humanos , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/economía , Niño , Estudios Retrospectivos , Masculino , Femenino , Enanismo/tratamiento farmacológico , Enanismo/economía , Proteínas Recombinantes/uso terapéutico , Proteínas Recombinantes/economía , Proteínas Recombinantes/administración & dosificación , Estatura/efectos de los fármacos , Resultado del Tratamiento , Preescolar , Trastornos del Crecimiento/tratamiento farmacológico , Trastornos del Crecimiento/economía , Trastornos del Crecimiento/etiología , Economía Farmacéutica , Análisis Costo-Beneficio , Costos de la Atención en Salud/estadística & datos numéricos , Adolescente
10.
Nutrients ; 16(11)2024 May 25.
Artículo en Inglés | MEDLINE | ID: mdl-38892545

RESUMEN

Enteral zinc supplementation in preterm infants has been reported to improve short-term weight and height gain. This study aims to evaluate whether early enteral zinc supplementation in preterm infants admitted to the neonatal intensive care unit (NICU) affects their physical measurements at discharge, and to periodically test serum copper levels. Of the 221 patients admitted to the NICU, 102 were in the zinc group and 119 were in the no-zinc group. The zinc group was administered 3 mg/kg/day of zinc. Body weight, height, and head circumference at discharge (or on the expected delivery date) were evaluated, and the factors affecting these parameters were examined. Serum zinc and copper levels were also evaluated on admission and monthly thereafter. Multivariate analysis was performed and showed that the weeks of gestational age and small for gestational age (SGA) status affected the height and weight at discharge. SGA also affected the head circumference. Serum copper levels were within the reference range for all patients at 3 months of age. Enteral zinc supplementation of 3 mg/kg/day in preterm infants did not affect the weight, height, or head circumference at discharge, but was shown to be relatively safe.


Asunto(s)
Cobre , Suplementos Dietéticos , Nutrición Enteral , Recien Nacido Prematuro , Unidades de Cuidado Intensivo Neonatal , Alta del Paciente , Zinc , Humanos , Zinc/sangre , Zinc/administración & dosificación , Zinc/deficiencia , Cobre/sangre , Recién Nacido , Recien Nacido Prematuro/sangre , Masculino , Femenino , Nutrición Enteral/métodos , Edad Gestacional , Antropometría , Estatura/efectos de los fármacos , Recién Nacido Pequeño para la Edad Gestacional , Peso Corporal
11.
J Pediatr Endocrinol Metab ; 37(6): 525-531, 2024 Jun 25.
Artículo en Inglés | MEDLINE | ID: mdl-38717038

RESUMEN

OBJECTIVES: Somatrogon is a long-acting recombinant human growth hormone used to treat patients with paediatric growth hormone deficiency (pGHD). This global phase 3 study compared the efficacy and safety of once-weekly somatrogon with once-daily somatropin in children with GHD. METHODS: Prepubertal patients were randomized 1:1 to once-weekly somatrogon (0.66 mg/kg/week) or once-daily somatropin (0.24 mg/kg/week) for 12 months. The primary endpoint was height velocity (HV) at month 12; secondary endpoints included HV at month 6 and change in height standard deviation score (SDS) at months 6 and 12 and insulin-like growth factor 1 (IGF-1) SDS. RESULTS: This post hoc subgroup analysis focused specifically on Asian children (somatrogon: n=24 and mean age=7.76 years; somatropin: n=21 and mean age=8.10 years) across eight countries. Mean HV at month 12 was 10.95 cm/year (somatrogon) and 9.58 cm/year (somatropin); the treatment difference of 1.38 cm/year favoured somatrogon. The lower bound of the two-sided 95 % CI of the treatment difference (somatrogon-somatropin) was -0.20, similar to the overall study population (-0.24). Compared with the somatropin group, the somatrogon group had numerically higher HV at month 6 (8.31 vs. 11.23 cm/year); a similar trend was observed for height SDS and IGF-1 SDS at months 6 and 12. Safety and tolerability were similar between treatment groups; adverse events occurred in 83 % of somatrogon-treated children and 76 % of somatropin-treated children. CONCLUSIONS: This subgroup analysis demonstrated that somatrogon efficacy and safety in Asian children were consistent with the overall study population, where once-weekly somatrogon was non-inferior to once-daily somatropin. Clinicaltrials.gov: NCT02968004.


Asunto(s)
Hormona de Crecimiento Humana , Humanos , Femenino , Niño , Masculino , Hormona de Crecimiento Humana/administración & dosificación , Hormona de Crecimiento Humana/deficiencia , Hormona de Crecimiento Humana/efectos adversos , Hormona de Crecimiento Humana/uso terapéutico , Estatura/efectos de los fármacos , Trastornos del Crecimiento/tratamiento farmacológico , Pueblo Asiatico , Estudios de Seguimiento , Resultado del Tratamiento , Proteínas Recombinantes/administración & dosificación , Proteínas Recombinantes/efectos adversos , Proteínas Recombinantes/uso terapéutico , Esquema de Medicación , Preescolar , Pronóstico
12.
Endocr Pract ; 30(9): 837-846, 2024 Sep.
Artículo en Inglés | MEDLINE | ID: mdl-38821179

RESUMEN

OBJECTIVE: To describe adherence to daily somatropin treatment and impact on height velocity within 1 year of treatment start among patients with pediatric growth hormone deficiency in a real-world US population. METHODS: This retrospective cohort study included pediatric patients aged ≥3 years to <16 years with pediatric growth hormone deficiency prescribed somatropin by a pediatric endocrinologist at a US-based center of excellence between January 1, 2015 and December 31, 2020. Patient data were collected using hospital electronic health records linked to a specialty pharmacy patient prescription records. Adherence, evaluated over 12 months, was measured using the proportion of days covered metric and patients were categorized as adherent if their proportion of days covered ≥80%. Height velocity was annualized to compare across adherent and nonadherent patients. RESULTS: One hundred eighty-one patients were identified and included in this study, of which 70.2% were male,73.5% were white, and mean age (standard deviation [SD]) at index was 12.1 (2.8). In the height velocity analysis, 174 patients were included and the mean (SD) annualized change in height was 10.2 (5.7) cm/y in the adherent group (n = 108) and 9.8 (7.6) in the nonadherent group (n = 66). The difference in height velocity between the groups was not statistically significant. CONCLUSIONS: Minor improvements in average height velocity were observed in the patient group who were adherent to somatropin therapy, although not statistically significant. Lack of observed significance may be due to small sample sizes, short observation period, a likely heterogenous population in terms of growth hormone prescribing, data bias due to single-center origin, or potential patient misclassification.


Asunto(s)
Estatura , Hormona de Crecimiento Humana , Cumplimiento de la Medicación , Humanos , Masculino , Niño , Femenino , Estudios Retrospectivos , Hormona de Crecimiento Humana/deficiencia , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/administración & dosificación , Estatura/efectos de los fármacos , Adolescente , Preescolar , Cumplimiento de la Medicación/estadística & datos numéricos , Trastornos del Crecimiento/tratamiento farmacológico , Estudios de Cohortes , Enanismo Hipofisario/tratamiento farmacológico
13.
Endocr J ; 71(7): 687-694, 2024 Jul 12.
Artículo en Inglés | MEDLINE | ID: mdl-38710621

RESUMEN

Short stature with IGF-1 receptor (IGF1R) gene alteration is known as small-for-gestational-age (SGA) short stature with elevated serum IGF1 levels. Its prevalence and clinical characteristics remain unclear. No adapted treatment is available for short stature related to IGF1R gene alteration in Japan, and genetic testing is not yet widely accessible. We investigated short stature with IGF1R gene alterations and analyzed the clinical data of 13 patients using the results of questionnaires issued to the Japanese Society for Pediatric Endocrinology. Four cases were caused by a deletion of chromosome 15q26.3, and eight were caused by heterozygous pathogenic variants in the IGF1R gene. Cases with deletions showed a more severe degree of growth impairment (-4.5 ± 0.43 SD) than those caused by pathological variants (-2.71 ± 0.15 SD) and were accompanied by neurodevelopmental delay. However, cases caused by pathological variants lacked distinctive features. Only three of the 12 cases demonstrated serum IGF1 values exceeding +2 SD, and the other three had values below 0 SD. Four patients did not meet the criteria for SGA at birth. Six patients received GH therapy for SGA short stature and showed improvement in growth rate without any side effects or elevated serum IGF1 levels during treatment. Elevated IGF1 levels (over +2 SD) after GH treatment should be considered a suspicious finding. Owing to the lack of distinctive features, there was a possibility of undiagnosed cases of this condition. Promoting genetic testing and clinical trials on GH administration for this condition is recommended.


Asunto(s)
Trastornos del Crecimiento , Hormona de Crecimiento Humana , Recién Nacido Pequeño para la Edad Gestacional , Receptor IGF Tipo 1 , Humanos , Receptor IGF Tipo 1/genética , Femenino , Masculino , Niño , Hormona de Crecimiento Humana/uso terapéutico , Trastornos del Crecimiento/tratamiento farmacológico , Trastornos del Crecimiento/genética , Preescolar , Recién Nacido Pequeño para la Edad Gestacional/crecimiento & desarrollo , Factor I del Crecimiento Similar a la Insulina/metabolismo , Adolescente , Enanismo/tratamiento farmacológico , Enanismo/genética , Japón , Estatura/efectos de los fármacos , Resultado del Tratamiento
14.
Pediatr Blood Cancer ; 71(8): e31117, 2024 Aug.
Artículo en Inglés | MEDLINE | ID: mdl-38804882

RESUMEN

BACKGROUND: Aromatase inhibitors (AI) may improve height in short stature conditions; however, the effect in childhood cancer survivors (CCS) is unknown. We assessed final adult height (FAH) in CCS treated with AI and GH compared with those treated with GH alone. METHODS: Retrospective cohort study of GH-deficient male CCS treated between 2007 and 2023. FAH was noted as the height at the fusion of growth plates or 18 years of age. Multivariable linear regression was used to examine treatment association with FAH, adjusting for other risk factors. RESULTS: Ninety-two patients were included; 70 were treated with GH and 22 with combination AI/GH. The mean age at GH initiation did not differ between groups. The mean age at AI initiation was 13.7 ± 1.9 years. A greater proportion of patients in the AI/GH group were treated with stem cell transplantation, abdominal radiation, total body irradiation, and cis-retinoic acid (p < .01). Multivariable linear regression demonstrated no significant treatment association with FAH Z-score (ß = 0.04, 95% CI: -0.9 to 0.9). History of spinal radiation (ß = -0.93, 95% CI: -1.7 to -0.2), lower starting height Z-score (ß = -0.8, 95% CI: -1.2 to -0.4), and greater difference between bone age and chronological age (ß = -0.3, 95% CI: -0.5 to -0.07) were associated with lower FAH Z-score. CONCLUSIONS: Adjuvant AI was not associated with increased FAH in male CCS compared with GH monotherapy. Future work is needed to determine the optimal adjunctive treatment to maximize FAH for this population.


Asunto(s)
Inhibidores de la Aromatasa , Estatura , Supervivientes de Cáncer , Hormona de Crecimiento Humana , Neoplasias , Humanos , Masculino , Inhibidores de la Aromatasa/uso terapéutico , Estudios Retrospectivos , Estatura/efectos de los fármacos , Adolescente , Hormona de Crecimiento Humana/deficiencia , Niño , Neoplasias/tratamiento farmacológico , Estudios de Seguimiento , Trastornos del Crecimiento/tratamiento farmacológico , Trastornos del Crecimiento/etiología , Trastornos del Crecimiento/patología , Adulto , Pronóstico , Quimioterapia Adyuvante
15.
Curr Opin Pediatr ; 36(4): 463-466, 2024 Aug 01.
Artículo en Inglés | MEDLINE | ID: mdl-38747200

RESUMEN

PURPOSE OF REVIEW: Congenital adrenal hyperplasia (CAH) is a relatively common disorder and one of the most challenging conditions seen by pediatric endocrinologists. Poor linear growth in CAH has been recognized for many years. There are new insights to explain this abnormality and shed light on strategies to promote normal growth. RECENT FINDINGS: Published data suggest that the dose of hydrocortisone during two critical periods of rapid growth, namely infancy and at puberty, has a fundamental effect on growth velocity, and by definition adult height. To prevent over-treatment, hydrocortisone dosage should remain within the range of 10-15 mg/m 2 body surface area per day. Precursor steroids such as 17-hydroxy progesterone (17OHP) should not be suppressed to undetectable levels. In fact, 17OHP should always be measurable, as complete suppression suggests over-treatment. SUMMARY: CAH is a challenging disorder. High-quality compliance within the consultation setting, with the patient seeing the same specialist at every visit, will be rewarded by improved long-term growth potential. Quality auxological monitoring can avoid phases of growth suppression. New therapy with CRH receptor antagonists may lead to a more nuanced approach by allowing fine tuning of hydrocortisone replacement without the need to suppress ACTH secretion.


Asunto(s)
Hiperplasia Suprarrenal Congénita , Hidrocortisona , Humanos , Hiperplasia Suprarrenal Congénita/tratamiento farmacológico , Hiperplasia Suprarrenal Congénita/diagnóstico , Niño , Adolescente , Hidrocortisona/uso terapéutico , Estatura/efectos de los fármacos , Trastornos del Crecimiento/tratamiento farmacológico , Trastornos del Crecimiento/etiología , Lactante , Preescolar
16.
Endocr Pract ; 30(7): 639-646, 2024 Jul.
Artículo en Inglés | MEDLINE | ID: mdl-38723894

RESUMEN

OBJECTIVE: There have been rare data on letrozole for height improvement in girls. This study aimed to clarify the efficacy and safety of combination therapy with recombinant human growth hormone (rhGH), GnRHa, and letrozole in improving the height of girls with short stature and advanced bone age. METHODS: This was a hospital record-based retrospective study. Follow-up was conducted on girls with short stature who received treatment with rhGH, GnRHa, and letrozole in our hospital. The treatment group included a total of 29 participants. Before treatment, the mean age of the patients was 11.17 years, and the mean treatment duration was 17.31 months. The control group consisted of 29 short-statured girls who received rhGH/GnRHa treatment, with the mean age and treatment duration of 12.43 years and 16.59 months, respectively. RESULTS: The predicted adult heights (PAHs) before and after treatment were 155.38 and 161.32 cm (P < .001). The ΔPAH in the treatment group was 4 cm higher than that in the control group (5.85 vs 1.82 cm, P < .001). Significant differences were noted in the height standard deviation scores of bone age (P < .001) and chronological age (P = .003) before and after treatment. There was an increasing body mass index during therapy (P = .039). The height gain was 8.71 ± 4.46 cm, and the growth rate was 6.78 ± 3.84 cm per year. CONCLUSION: Combined treatment with GH, GnRHa, and letrozole can enhance the adult height and PAH in short-statured girls, and no significant side effects have been reported.


Asunto(s)
Estatura , Hormona Liberadora de Gonadotropina , Trastornos del Crecimiento , Hormona de Crecimiento Humana , Letrozol , Humanos , Letrozol/uso terapéutico , Letrozol/administración & dosificación , Femenino , Estudios Retrospectivos , Estatura/efectos de los fármacos , Niño , Adolescente , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/administración & dosificación , Hormona Liberadora de Gonadotropina/agonistas , Trastornos del Crecimiento/tratamiento farmacológico , Nitrilos/uso terapéutico , Triazoles/uso terapéutico , Triazoles/administración & dosificación , Quimioterapia Combinada , Inhibidores de la Aromatasa/uso terapéutico
17.
Gynecol Endocrinol ; 40(1): 2358227, 2024 Dec.
Artículo en Inglés | MEDLINE | ID: mdl-38807420

RESUMEN

OBJECTIVE: The aim of this study was to explore the impact of growth hormone (GH) therapy on the onset and progression of puberty in girls with idiopathic short stature. METHODS: This study included 541 girls aged between 4.5 and 10.6 years who were receiving GH treatment, monitored over a 22-year follow-up period. Of these, 126 girls have been followed up to the onset of menarche. The participants were divided into two groups: a ISS control group (n = 66) and a group receiving daily GH treatment at a dose of 0.15 iu/kg (n = 60). We assessed the pubertal development and GH usage of these girls every three months. RESULTS: (1) There was no significant difference in the onset of puberty between the growth hormone (GH) treatment group and the control group; however, the average duration of puberty was longer in the treatment group compared to the control group. (2) During puberty, there were no significant differences in height growth between the treated and untreated groups. (3) The duration of GH treatment showed a significant negative correlation with the age at onset of gonadal development and the age at menarche in females within the treatment group. CONCLUSION: GH treatment does not seem to accelerate the onset of puberty but may extend its duration, without significantly impacting height growth during puberty. Additionally, longer GH treatment duration is linked to earlier gonadal development and menarche in females.


Asunto(s)
Estatura , Trastornos del Crecimiento , Hormona de Crecimiento Humana , Menarquia , Pubertad , Humanos , Femenino , Niño , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/administración & dosificación , Pubertad/efectos de los fármacos , Trastornos del Crecimiento/tratamiento farmacológico , Menarquia/efectos de los fármacos , Estatura/efectos de los fármacos , Preescolar , Estudios de Seguimiento , Adolescente
18.
Front Endocrinol (Lausanne) ; 15: 1390674, 2024.
Artículo en Inglés | MEDLINE | ID: mdl-38737553

RESUMEN

Introduction: Gonadotropin-releasing hormone (GnRH) analogs are the standard treatment for central precocious puberty (CPP). Although there are numerous varieties of GnRH agonists, the effectiveness of 1-monthly compared with 3-monthly Leuprolide acetate is still restricted. The objective of this study was to evaluate the outcomes of CPP treatment with Leuprolide acetate at a 1-monthly dosage of 3.75 mg, in comparison to a dosage of 11.25 mg administered every 3 months. Method: This retrospective cohort study involved 143 girls diagnosed with CPP with 72 of them receiving the monthly treatment regimen and 71 receiving the 3-monthly treatment regimen. Anthropometric measurements were compared at the start and end of the therapy. The rates and level of LH suppression were assessed six months after therapy. Results: The regimen administered every 3 months showed more significant suppression of LH. The 3-monthly group showed lower actual height and degree of bone age advancement at the end of therapy. However, the predicted adult height (PAH) remained comparable in both groups. Conclusion: The 3-monthly treatment showed greater hormonal and growth suppression effects, but there was no significant difference in PAH between the two groups.


Asunto(s)
Leuprolida , Pubertad Precoz , Humanos , Leuprolida/administración & dosificación , Leuprolida/uso terapéutico , Pubertad Precoz/tratamiento farmacológico , Femenino , Estudios Retrospectivos , Niño , Resultado del Tratamiento , Hormona Luteinizante/sangre , Estatura/efectos de los fármacos , Esquema de Medicación , Hormona Liberadora de Gonadotropina/agonistas , Preescolar
19.
Pediatr Nephrol ; 39(11): 3167-3175, 2024 Nov.
Artículo en Inglés | MEDLINE | ID: mdl-38607423

RESUMEN

Recombinant human growth hormone therapy, which was introduced in the 1980s, is now routine for children with advanced chronic kidney disease (CKD) who are exhibiting growth impairment. Growth hormone usage remains variable across different centers, with some showing low uptake. Much of the focus on growth hormone supplementation has been on increasing height because of social and psychological effects of short stature. There are, however, numerous other changes that occur in CKD that have not received as much attention but are biologically important for pediatric growth and development. This article reviews the current knowledge about the multisystem effects of growth hormone therapy in pediatric patients with CKD and highlights areas where additional clinical research is needed. We also included clinical data on children and adults who had received growth hormone for other indications apart from CKD. Ultimately, having robust clinical studies which examine these effects will allow children and their families to make more informed decisions about this therapy.


Asunto(s)
Estatura , Trastornos del Crecimiento , Hormona de Crecimiento Humana , Insuficiencia Renal Crónica , Humanos , Niño , Hormona de Crecimiento Humana/uso terapéutico , Hormona de Crecimiento Humana/administración & dosificación , Trastornos del Crecimiento/etiología , Trastornos del Crecimiento/tratamiento farmacológico , Estatura/efectos de los fármacos , Adolescente
20.
Nutr Hosp ; 41(3): 690-701, 2024 Jun 27.
Artículo en Inglés | MEDLINE | ID: mdl-38666332

RESUMEN

Introduction: Introduction: early exposure to cadmium toxic metal has been suggested to be associated with reduced infants/children growth; nevertheless, the available evidence is contradictory. Objective: this meta-analysis aimed to examine the association of cadmium exposure through biological samples to growth measurements of infants/children, including body weight, height, body mass index (BMI), BMI-for-age (BMI Z-score), weight-for-age (WAZ), height-for-age (HAZ), and weight-for-height (WHZ) z-scores. Methods: a systematic search in PubMed and Scopus was implemented to obtain the related studies. The standardized beta coefficients (ß) and 95 % confidence intervals (95 % CI) were used as effect sizes to test the associations using the random effects analysis. Results: a total of 15 studies with 6,181 participants were included in the meta-analysis. In the overall analysis, pooled analysis of available data revealed that cadmium exposure was inversely linked to height (ß = -0.06, 95 % CI = -0.12 to -0.01) and WAZ (ß = -0.01, 95 % CI = -0.02 to -0.003). These relationships were also supported by prospective cohort studies and urinary cadmium exposure. In the stratified analysis, cadmium exposure was negatively linked to the weight of children in prospective cohort studies, in studies that assessed urinary cadmium exposure. No significant association was detected between cadmium exposure and BMI, BMI Z-score, WHZ, and HAZ in the overall and subgroup analyses. Conclusions: this meta-analysis emphasized the importance of cadmium exposure as a risk factor for growth failure in infants/children.


Introducción: Introducción: se ha sugerido que la exposición temprana al metal tóxico cadmio se asocia a un crecimiento reducido de los bebés y niños; sin embargo, la evidencia disponible es contradictoria. Objetivo: este metanálisis tuvo como objetivo examinar la asociación de la exposición al cadmio a través de muestras biológicas con las mediciones de crecimiento de bebés/niños, incluidos el peso corporal, la altura, el índice de masa corporal (IMC), el IMC para la edad (puntuación Z del IMC) y las puntuaciones z de peso para la edad (WAZ), altura para la edad (HAZ) y peso para la altura (WHZ). Métodos: se implementó una búsqueda sistemática en PubMed y Scopus para obtener los estudios relacionados. Los coeficientes beta estandarizados (ß) y los intervalos de confianza del 95 % (IC 95 %) se utilizaron como tamaños del efecto para probar las asociaciones mediante el análisis de efectos aleatorios. Resultados: se incluyeron en el metanálisis un total de 15 estudios, con 6.181 participantes. En el análisis general, el análisis conjunto de los datos disponibles reveló que la exposición al cadmio estaba inversamente relacionada con la altura (ß = -0,06, IC del 95 % = -0,12 a -0,01) y WAZ (ß = -0,01, IC del 95 % = -0,02 a -0,003). Estas relaciones también fueron respaldadas por estudios de cohortes prospectivos y exposición al cadmio en orina. En el análisis estratificado, la exposición al cadmio se relacionó negativamente con el peso de los niños en estudios de cohortes prospectivos, en estudios que evaluaron la exposición al cadmio en la orina. No se detectó ninguna asociación significativa entre la exposición al cadmio y el IMC, la puntuación Z del IMC, el WHZ y el HAZ en los análisis generales y de subgrupos. Conclusiones: este metanálisis enfatizó la importancia de la exposición al cadmio como factor de riesgo del retraso del crecimiento en lactantes/niños.


Asunto(s)
Cadmio , Humanos , Cadmio/orina , Niño , Lactante , Preescolar , Trastornos del Crecimiento/inducido químicamente , Metales Pesados/orina , Metales Pesados/efectos adversos , Índice de Masa Corporal , Estatura/efectos de los fármacos , Peso Corporal/efectos de los fármacos
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