Therapeutic Advances in Endocrinology and Metabolism最新文献

{"title":"Prediction of cardiac autonomic neuropathy using a machine learning model in patients with diabetes.","authors":"Ahmad Shaker Abdalrada, Jemal Abawajy, Tahsien Al-Quraishi, Sheikh Mohammed Shariful Islam","doi":"10.1177/20420188221086693","DOIUrl":"10.1177/20420188221086693","url":null,"abstract":"<p><strong>Background: </strong>Cardiac autonomic neuropathy (CAN) is a diabetes-related complication with increasing prevalence and remains challenging to detect in clinical settings. Machine learning (ML) approaches have the potential to predict CAN using clinical data. In this study, we aimed to develop and evaluate the performance of an ML model to predict early CAN occurrence in patients with diabetes.</p><p><strong>Methods: </strong>We used the diabetes complications screening research initiative data set containing 200 CAN-related tests on more than 2000 participants with type 2 diabetes in Australia. Data were collected on peripheral nerve functions, Ewing's tests, blood biochemistry, demographics, and medical history. The ML model was validated using 10-fold cross-validation, of which 90% were used in training the model and the remaining 10% was used in evaluating the performance of the model. Predictive accuracy was assessed by area under the receiver operating curve, and sensitivity, specificity, positive predictive value, and negative predictive value.</p><p><strong>Results: </strong>Of the 237 patients included, 105 were diagnosed with an early stage of CAN while the remaining 132 were healthy. The ML model showed outstanding performance for CAN prediction with receiver operating characteristic curve of 0.962 [95% confidence interval (CI) = 0.939-0.984], 87.34% accuracy, and 87.12% sensitivity. There was a significant and positive association between the ML model and CAN occurrence (<i>p</i> < 0.001).</p><p><strong>Conclusion: </strong>Our ML model has the potential to detect CAN at an early stage using Ewing's tests. This model might be useful for healthcare providers for predicting the occurrence of CAN in patients with diabetes, monitoring the progression, and providing timely intervention.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.9,"publicationDate":"2022-03-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8943459/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45205723","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Testosterone concentrations and prescription patterns of 1% testosterone gel in transgender and gender diverse individuals.","authors":"Brendan J Nolan, Sav Zwickl, Alex F Q Wong, Peter Locke, Satu Simpson, Ling Li, Jeffrey D Zajac, Ada S Cheung","doi":"10.1177/20420188221083512","DOIUrl":"10.1177/20420188221083512","url":null,"abstract":"<p><strong>Background: </strong>Masculinising hormone therapy with testosterone is used to align an individual's physical characteristics with their gender identity. Standard testosterone doses and formulations recommended for hypogonadal cisgender men are typically administered, although there are currently limited data evaluating the use of 1% testosterone gel in gender-affirming hormone therapy regimens.</p><p><strong>Objectives: </strong>The objective of the study was to assess the prescription patterns and serum total testosterone concentrations achieved with 1% testosterone gel in trans and gender diverse individuals.</p><p><strong>Materials and methods: </strong>A retrospective cross-sectional analysis was undertaken of trans individuals at a primary and secondary care clinic in Melbourne, Australia. Sixty-seven individuals treated with 1% testosterone gel were included. Primary outcomes were testosterone dose and serum total testosterone concentration achieved.</p><p><strong>Results: </strong>Median age was 25 (22-30) years and median duration of testosterone therapy was 12 (7-40) months. Thirty-five (52%) individuals had a nonbinary gender identity. Initial median testosterone dose was 25 mg (12.5-31.3) daily. Fifty-two (78%) individuals commenced doses <50 mg daily, the recommended starting dose for hypogonadal cisgender men. Median total testosterone concentration achieved was 11.9 nmol/l (7.3-18.6). Polycythaemia (haematocrit >0.5) was documented in eight of 138 (6%) laboratory results in six individuals.</p><p><strong>Discussion and conclusions: </strong>One percent testosterone gel achieves serum total testosterone concentrations in the cisgender male reference range. A high proportion of individuals had a nonbinary gender identity and most individuals commenced a lower dose than that typically administered to hypogonadal cisgender men, potentially related to slow or 'partial' masculinisation goals.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.9,"publicationDate":"2022-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8918760/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46626242","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pharmacological management of severe Cushing's syndrome: the role of etomidate.","authors":"Andrea Pence,&nbsp;Megan McGrath,&nbsp;Stephanie L Lee,&nbsp;Douglas E Raines","doi":"10.1177/20420188211058583","DOIUrl":"https://doi.org/10.1177/20420188211058583","url":null,"abstract":"<p><p>Cushing's syndrome (CS) is an endocrine disease characterized by excessive adrenocortical steroid production. One of the mainstay pharmacological treatments for CS are steroidogenesis enzyme inhibitors, including the antifungal agent ketoconazole along with metyrapone, mitotane, and aminoglutethimide. Recently, osilodrostat was added to this drug class and approved by the US Food and Drug Administration (FDA) for the treatment of Cushing's Disease. Steroidogenesis enzyme inhibitors inhibit various enzymes along the cortisol biosynthetic pathway and may be used preoperatively to lower cortisol levels and reduce surgical risk associated with tumor resection or postoperatively when surgery and/or radiation therapies are not curative. Because their selectivities for steroidogenic enzymes vary, they may even be administered in combination to achieve relatively rapid control of severe hypercortisolemia. Unfortunately, all currently available inhibitors are accompanied by serious adverse side effects that limit dosing and often result in treatment failures. Although more commonly known as a general anesthetic induction agent, etomidate is another member of the steroidogenesis enzyme inhibitor drug class. It suppresses cortisol production primarily by inhibiting 11β-hydroxylase and is the only inhibitor that may be given parenterally. However, the sedative-hypnotic actions of etomidate limit its use as an acute management option for CS. Thus, some have recommended that it be used only in intensive care settings. In this review, we discuss the initial development of etomidate as an anesthetic agent, its subsequent development as a treatment for CS, and the recent advances in dosing and drug development that dissociate sedative-hypnotic and adrenostatic drug actions to facilitate CS treatment in non-critical care settings.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2022-02-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ba/8f/10.1177_20420188211058583.PMC8848075.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39814806","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-world evidence of the use of glucocorticoids for severe COVID-19.","authors":"Alejandra Albarrán-Sánchez, Claudia Ramírez-Rentería, Moisés Mercado, Miriam Sánchez-García, Corazón de Jesús Barrientos-Flores, Aldo Ferreira-Hermosillo","doi":"10.1177/20420188211072704","DOIUrl":"10.1177/20420188211072704","url":null,"abstract":"<p><strong>Introduction: </strong>Currently, only glucocorticoids have proved to impact adverse outcomes in COVID-19. However, their risk/benefit balance remains inconclusive and populations' characteristics should be considered.</p><p><strong>Objective: </strong>The objective was to evaluate the real-life use of glucocorticoids in patients with severe COVID-19 hospitalized in a third-level referral center and to determine the type, accumulated doses, and the in-hospital outcomes related with their use.</p><p><strong>Methods: </strong>We evaluated a retrospective cohort of 737 patients with criteria for severe COVID-19 and a positive polymerase chain reaction (PCR) test for SARS-CoV-2. We extracted data for epidemiological analysis, medical history, and medications, as well as baseline laboratory tests. Data were analyzed using SPSS 21.0 and nonparametric tests, medians, and interquartile ranges (IQR). A <i>p</i> < 0.05 was considered significant.</p><p><strong>Results: </strong>A total of 65.3% were men, with a median age of 59 years (IQR 46-70) and a median of 10 days of hospital stay (IQR 6-16), more than 40% had diabetes, hypertension, and/or obesity, and 0.8% used steroids chronically. At the time of the study, 54.0% had been discharged due to improvement and 40.8% died. The most common treatment used was dexamethasone 6 mg/day/10 days (46.6%). Patients with a complete dexamethasone scheme [as proposed by the Randomized Evaluation of COVID-19 Therapy (RECOVERY) study] had a lower mortality risk [hazard ratio (HR) 0.441, 95% confidence interval (CI) 0.232-0.840] in comparison with patients with lower doses (HR 1.803, 95% CI 1.080-3.012). Patients with methylprednisolone or several steroids tended to have higher cumulative doses (equivalent to >675 mg of prednisolone).</p><p><strong>Conclusion: </strong>The use of steroids in severe COVID-19 reduces mortality only at the dose proposed in the RECOVERY study in the younger population. No benefit of the use of steroids was observed in patients with older age or higher number of comorbidities.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.9,"publicationDate":"2022-01-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/48/1e/10.1177_20420188211072704.PMC8777321.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39942737","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Qualitative evaluation of the symptoms and quality of life impacts of long-chain fatty acid oxidation disorders.","authors":"Rebecca Williams-Hall,&nbsp;Katie Tinsley,&nbsp;Eliza Kruger,&nbsp;Chloe Johnson,&nbsp;Alexandra Bowden,&nbsp;Tricia Cimms,&nbsp;Adam Gater","doi":"10.1177/20420188211065655","DOIUrl":"https://doi.org/10.1177/20420188211065655","url":null,"abstract":"<p><strong>Background: </strong>Long-chain fatty acid oxidation disorders (LC-FAOD) are a group of rare autosomal-recessive genetic disorders characterized by metabolic deficiencies in which the body is unable to convert long-chain fatty acids into energy. To date, however, there is limited understanding of the patient experience of LC-FAOD.</p><p><strong>Methods: </strong>The symptoms, observable signs, and quality of life (QoL) impacts associated with LC-FAOD were explored via a focus group (<i>n</i> = 8) and semi-structured interviews (<i>n</i> = 6) with patients and caregivers of patients with LC-FAOD, and interviews (<i>n</i> = 4) with expert clinicians. Data were analyzed via thematic analysis and summarized in a conceptual model.</p><p><strong>Results: </strong>Participants reported a wide range of signs and symptoms associated with LC-FAOD, broadly categorized as musculoskeletal, endocrine/nutritional/metabolic, neurological, gastrointestinal/digestive, sensory, cardiovascular, respiratory, urological, and constitutional. LC-FAOD were reported to have a significant impact on various aspects of patients' lives including physical functioning, participation in daily activities, emotional/psychological wellbeing, and social functioning. Lifestyle modifications (such as diet and exercise restrictions) were necessary because of the condition. Symptoms were typically episodic in presentation often arising or exacerbated during catabolic conditions such as prolonged exercise, fasting, physiological stress, and illness/infection. Symptoms were also commonly reported to lead to emergency room visits, hospitalization, and clinical complications.</p><p><strong>Conclusion: </strong>LC-FAOD have a considerable impact on patients' lives. There is a high degree of concordance in the signs, symptoms, and impacts of LC-FAOD reported by patients, caregivers, and clinicians; however, there were many symptoms and impacts that were only reported by patients and caregivers, thus demonstrating that insights from patient/caregiver experience data are integral for informing medical product development and facilitating patient-centered care.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2022-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/83/ea/10.1177_20420188211065655.PMC8755934.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39825217","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Lipid ratios and obesity indices are effective predictors of metabolic syndrome in women with polycystic ovary syndrome.","authors":"Małgorzata Kałużna,&nbsp;Magdalena Czlapka-Matyasik,&nbsp;Pola Kompf,&nbsp;Jerzy Moczko,&nbsp;Katarzyna Wachowiak-Ochmańska,&nbsp;Adam Janicki,&nbsp;Karolina Samarzewska,&nbsp;Marek Ruchała,&nbsp;Katarzyna Ziemnicka","doi":"10.1177/20420188211066699","DOIUrl":"https://doi.org/10.1177/20420188211066699","url":null,"abstract":"<p><strong>Background: </strong>Insulin resistance (IR) is common in women with polycystic ovary syndrome (PCOS). Metabolic syndrome (MS) involves IR, arterial hypertension, dyslipidemia, and visceral fat accumulation. Therefore, fatness indices and blood lipid ratios can be considered as screening markers for MS. Our study aimed to evaluate the predictive potential of selected indirect metabolic risk parameters to identify MS in PCOS.</p><p><strong>Methods: </strong>This cross-sectional study involved 596 women aged 18-40 years, including 404 PCOS patients diagnosed according to the Rotterdam criteria and 192 eumenorrheic controls (CON). Anthropometric and blood pressure measurements were taken, and blood samples were collected to assess glucose metabolism, lipid parameters, and selected hormone levels. Body mass index (BMI), waist-to-height ratio (WHtR), homeostasis model assessment for insulin resistance index (HOMA-IR), visceral adiposity index (VAI), lipid accumulation product (LAP), non-high-density lipoprotein cholesterol (non-HDL-C), and triglycerides-to-HDL cholesterol ratio (TG/HDL-C) were calculated. MS was assessed using the International Diabetes Federation (IDF) and the American Heart Association/National Heart, Lung, and Blood Institute (AHA/NHLBI) criteria.</p><p><strong>Results: </strong>MS prevalence was significantly higher in PCOS <i>versus</i> CON. Patients with both MS and PCOS had more unfavorable anthropometric, hormonal, and metabolic profiles <i>versus</i> those with neither MS nor PCOS and <i>versus</i> CON with MS. LAP, TG/HDL-C, VAI, and WHtR were the best markers and strongest indicators of MS in PCOS, and their cut-off values could be useful for early MS detection. MS risk in PCOS increased with elevated levels of these markers and was the highest when TG/HDL-C was used.</p><p><strong>Conclusions: </strong>LAP, TG/HDL-C, VAI, and WHtR are representative markers for MS assessment in PCOS. Their predictive power makes them excellent screening tools for internists and enables acquiring accurate diagnoses using fewer MS markers.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2022-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/6b/b4/10.1177_20420188211066699.PMC8755932.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39825219","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Recent advancement in the treatment of boys and adolescents with hypogonadism.","authors":"Rodolfo A Rey","doi":"10.1177/20420188211065660","DOIUrl":"https://doi.org/10.1177/20420188211065660","url":null,"abstract":"<p><p>Clinical manifestations and the need for treatment varies according to age in males with hypogonadism. Early foetal-onset hypogonadism results in disorders of sex development (DSD) presenting with undervirilised genitalia whereas hypogonadism established later in foetal life presents with micropenis, cryptorchidism and/or micro-orchidism. After the period of neonatal activation of the gonadal axis has waned, the diagnosis of hypogonadism is challenging because androgen deficiency is not apparent until the age of puberty. Then, the differential diagnosis between constitutional delay of puberty and central hypogonadism may be difficult. During infancy and childhood, treatment is usually sought because of micropenis and/or cryptorchidism, whereas lack of pubertal development and relative short stature are the main complaints in teenagers. Testosterone therapy has been the standard, although off-label, in the vast majority of cases. However, more recently alternative therapies have been tested: aromatase inhibitors to induce the hypothalamic-pituitary-testicular axis in boys with constitutional delay of puberty and replacement with GnRH or gonadotrophins in those with central hypogonadism. Furthermore, follicle-stimulating hormone (FSH) priming prior to hCG or luteinizing hormone (LH) treatment seems effective to induce an enhanced testicular enlargement. Although the rationale for gonadotrophin or GnRH treatment is based on mimicking normal physiology, long-term results are still needed to assess their impact on adult fertility.</p>","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2022-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/ea/b6/10.1177_20420188211065660.PMC8753232.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39825218","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pharmacotherapy to delay the progression of diabetic kidney disease in people with type 2 diabetes: past, present and future","authors":"Ritwika Mallik, T. Chowdhury","doi":"10.1177/20420188221081601","DOIUrl":"https://doi.org/10.1177/20420188221081601","url":null,"abstract":"Diabetic kidney disease (DKD) is a leading cause of morbidity and mortality among people living with diabetes, and is one of the most important causes of end stage renal disease worldwide. In order to reduce progression of DKD, important management goals include treatment of hypertension, glycaemia and control of cardiovascular risk factors such as lipids, diet, smoking and exercise. Use of angiotensin converting enzyme inhibitors or angiotensin receptor blockers has an established role in prevention of progression of DKD. A number of other agents such as endothelin-1 receptor antagonists and bardoxolone have had disappointing results. Recent studies have, however, suggested that newer antidiabetic agents such as sodium-glucose transporter-2 inhibitors (SGLT-2i) and glucagon-like peptide-1 analogues have specific beneficial effects in patients with DKD. Indeed most recent guidance suggest that SGLT-2i drugs should be used early in DKD, irrespective of glucose control. A number of pathways are hypothesised for the development and progression of DKD, and have opened up a number of newer potential therapeutic targets. This article aims to discuss management of DKD with respect to seminal trials from the past, more recent trials informing the present and potential new therapeutic options that may be available in the future.","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45125516","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Exploring the weight bias of professionals working in the field of obesity with a mobile IAT: a pilot study","authors":"Tobias Jungnickel, U. von Jan, S. Engeli, U. Albrecht","doi":"10.1177/20420188221098881","DOIUrl":"https://doi.org/10.1177/20420188221098881","url":null,"abstract":"Background: Obesity is common in many industrialized nations and often accompanied by related health issues. Furthermore, individuals living with overweight or obesity are often confronted with stigmatization in their daily lives. These problems may be aggravated if the objectivity of health care professionals is compromised due to (unconscious) prejudices. If pharmaceutical companies, regulatory agencies, and health insurers are also susceptible to these biases, decisions related to the development, approval, and reimbursement of obesity-related therapies may be negatively impacted. Materials and Methods: The ‘Implicit Association Test’ (IAT) is a psychometric test allowing to measure these attitudes and could therefore assist to reveal unconscious preferences. A self-developed mobile version, in the form of a ResearchKit-based IAT app was employed in the presented study. The objective was to determine (potential) weight bias and its characteristics for professionals attending a national obesity-related conference in Germany (G1), compared to a control group (without stated interest in the topic, G2) – both using the mobile app – and a historical control (G3) based on data provided by Project Implicit acquired by a web app. Results: Explicit evaluations of G1 were neutral at a higher percentage compared with G2 and G3, while implicit preference toward lean individuals did not differ significantly between G2 and G3, and G1. Conclusion: The greater discrepancy between the (more neutral) explicit attitude and the unconscious preference pointing in the anti-obesity direction could indicate an underestimated bias for the professional participants in G1. Implicit preference is often ingrained from childhood on, and difficult to overcome. Thus, even for professionals, it may unconsciously influence decisions made in the care they provide. Professionals in any given health care sector directed at obesity care should thus be made aware of this inconsistency to enable them to consciously counteract this potential effect.","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45397757","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Factors influencing height gain in children born small for gestational age treated with recombinant growth hormone: what extent is puberty involved?","authors":"Ramón Arroyo Ruiz, Aránzazu Ballester Pérez, I. Leiva-Gea, MªJosé Martínez-Aedo, J. López-Siguero","doi":"10.1177/20420188221083534","DOIUrl":"https://doi.org/10.1177/20420188221083534","url":null,"abstract":"Objective: The objective was to analyze the efficacy of recombinant human growth hormone (rhGH) treatment in children born small for gestational age (SGA) without catch-up growth treated before the onset of puberty, with follow-up until adult height. The influence of demographic and auxological factors on the final response evaluated as adult height and height gain was assessed. Patients and methods: A prospective longitudinal observational study performed in a tertiary hospital, involving SGA patients, who started treatment with rhGH between October 2003 and April 2015. Potential response predictors were evaluated by multiple regression analysis and receiver operating characteristic curves. Results: Of the initial 96 patients included, 61 patients (28 boys and 33 girls) reached adult height. Adult height gain in standard deviation (SDS) was 0.99 (0.8) and 1.49 (0.94), respectively (p < 0.05). An adult height greater than −2 SDS was reached in 75% of the girls but only in 53% of the boys. The pubertal height gain was 22.6 (5.8) cm in boys and 18.8 (4.5) cm in girls. The multiple regression model obtained for total height gain explained 42% of the variability in this variable including sex, height gain during the first year, and the difference from target height at the start of treatment. A first-year height gain of 0.69 SDS was the optimal point for assessing a final height gain greater than 1.5 SDS with a specificity of 70% and a sensitivity of 71%. Conclusion: Most SGA patients achieve normalization of height above −2 SD, the percentage being higher in girls. According to our predictive model, height gain in the first year is the most important variable for predicting good response to treatment. During puberty, there is a loss of height SDS, probably due to a lower total pubertal gain with respect to the reference population, which is more marked in boys.","PeriodicalId":22998,"journal":{"name":"Therapeutic Advances in Endocrinology and Metabolism","volume":null,"pages":null},"PeriodicalIF":3.8,"publicationDate":"2022-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47814168","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}