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OBJECTIVE: A nonclassic form of 11ß-hydroxylase deficiency (NC11ß-OHD) has been reported to cause mild androgen excess symptoms. Currently, the gold standard for biochemical diagnosis is elevated 11-deoxycortisol (11-DOC) levels after corticotropin stimulation test (ACTHstimT). However, there are no clear 11-DOC level cutoffs. One of the accepted references for 11-DOC levels for the paediatric population was published in 1991 by Lashansky et al. AIM: To determine the correlation between 11-DOC levels measured during ACTHstimT and clinical symptoms attributed to NC11ß-OHD. DESIGN: A retrospective study including all paediatric patients who underwent ACTHstimT at Shamir Medical Center between 2007 and 2015. Clinical data were collected from the patients' medical files. Outcome measures included the number of patients with hyperandrogenism signs and predefined elevated 11-DOC cut-off levels according to Lashansky for sex and age, and according to commercial kit cut-offs. RESULTS: Data were complete at presentation for 136 patients. Long-term clinical data were documented for 98 patients, mean follow-up duration of 3.1 years (1.37-5.09). There was no statistically significant difference in the number of cases with elevated 11-DOC according to both cut-offs and early puberty, premature adrenarche nor acne. Follow-up data demonstrated no statistically significant difference in the number of cases with elevated 11-DOC levels among patients with compromised final adult height, polycystic ovarian syndrome or hyperandrogenism. CONCLUSIONS: Basal and corticotropin stimulated 11-DOC levels were not significantly elevated above the 1.5 times cut-offs according to paediatric-specific norms or the commercial assay in paediatric individuals with possible clinical suspicion of NC11ß-OHD.
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Hiperplasia Suprarrenal Congênita , Cortodoxona/sangue , Hiperandrogenismo , Puberdade Precoce , Hiperplasia Suprarrenal Congênita/diagnóstico , Hormônio Adrenocorticotrópico , Criança , Feminino , Humanos , Masculino , Oxigenases de Função Mista , Estudos RetrospectivosRESUMO
OBJECTIVE: To compare clinical outcomes of 3 treatment regimens-glucocorticoids (GCs), oral contraceptives (OCs), or a combination of both-administered to adolescents and young women diagnosed in childhood with nonclassical congenital adrenal hyperplasia (NCCAH), who had been treated with GCs until their adult height was achieved. METHODS: A retrospective study of medical records of 53 female patients with NCCAH followed in 3 tertiary pediatric endocrinology institutes. The 3 treatment groups were compared for the prevalence of hirsutism and acne, standardized body mass index (BMI)-standard deviation score (SDS), and androgen levels at the attainment of adult height (baseline), 1-year later, and at the last documented visit. RESULTS: At baseline, there were no significant differences among groups in BMI-SDS, androgen levels, hirsutism prevalence, acne, or irregular menses. From baseline to the last visit, the rate of hirsutism declined significantly only in the OC group (37.5% vs 6.2%, respectively; P = .03). The rate of acne declined in the combined group (50% vs 9%, respectively; P = .03) with a similar tendency in the OC group (50% vs 12.5%, respectively; P = .05). No significant changes were observed in BMI-SDS for the entire cohort or any subgroup during follow-up. A significant rise in androstenedione (P < .001), testosterone (P < .01), and 17-hydroxyprogesterone (P < .01) levels was observed only in the OC group. CONCLUSION: In girls diagnosed in childhood with NCCAH, who require treatment for hyperandrogenism following completion of linear growth, management should be tailored individually using a patient-centered approach. Treatment with OCs might be better than that with GCs for regression of hirsutism and acne. The long-term effects of elevated levels of androgens associated with this treatment regimen should be further studied.
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Hiperplasia Suprarrenal Congênita , Glucocorticoides , Adolescente , Hiperplasia Suprarrenal Congênita/tratamento farmacológico , Hiperplasia Suprarrenal Congênita/epidemiologia , Adulto , Androstenodiona , Criança , Anticoncepcionais Orais , Feminino , Hirsutismo/tratamento farmacológico , Hirsutismo/epidemiologia , Humanos , Estudos RetrospectivosRESUMO
INTRODUCTION: Pheochromocytoma is a rare, catecholamine secreting tumor arising from chromaffin cells of the adrenal medulla and it is responsible for 0.5-2% of pediatric hypertension cases. We hereby present a case of a 13-year-old girl with obesity, excessive hypertension, prediabetes and secondary nocturnal enuresis, who was eventually diagnosed with pheochromocytoma. Most symptoms significantly improved after treating the tumor. We suggest that this diagnosis should be considered in pediatric patients presenting with similar symptoms.
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Neoplasias das Glândulas Suprarrenais , Hipertensão , Enurese Noturna , Feocromocitoma , Adolescente , Neoplasias das Glândulas Suprarrenais/complicações , Neoplasias das Glândulas Suprarrenais/diagnóstico , Criança , Feminino , Humanos , Hipertensão/diagnóstico , Hipertensão/etiologia , Enurese Noturna/diagnóstico , Enurese Noturna/etiologia , Feocromocitoma/complicações , Feocromocitoma/diagnósticoRESUMO
Our aim was to assess the efficacy, safety, and tolerability of alpha-1 antitrypsin (AAT) as a therapeutic modality for ß-cell preservation in patients with recent-onset type 1 diabetes. Seventy type 1 diabetes patients (37 males; mean age 13.1 ± 4.1years) were randomized to treatment with 22 infusions of AAT (Glassia®) (60 or 120 mg/kg) or placebo. The primary outcome was the area under the curve (AUC) of C-peptide from a 2-h mixed-meal tolerance test after 52 weeks. At week 52, C-peptide was 0.9, 0.45, and 0.48 pmol/mL in the AAT-120, AAT-60, and placebo groups (p = 0.170 and p = 0.866 vs. placebo, respectively). The declines in C-peptide glycated hemoglobin (HbA1c) and the total insulin dose (U/kg) were similar across groups. Within the predefined 12-18-years subgroup, the C-peptide AUC decreased significantly in the placebo and AAT-60 groups (-0.34 and -0.54 pmol/mL, respectively, p < 0.01), with a borderline decrease in the AAT-120 group (-0.29 pmol/mL, p = 0.047). The mean HbA1c level was significantly lower in the AAT-120 group compared to the placebo (6.7% ± 0.9% vs. 8.2 ± 1.4%, p = 0.05), and a higher percentage of patients attained HbA1c ≤ 7% (75% vs. 25%, p = 0.05). AAT was tolerated well, with a similar safety profile between groups. The AAT intervention showed promise in the subgroup of adolescents with recent-onset type 1 diabetes. Further studies are warranted to determine the impact and proposed mechanism of action of AAT in ß-cell preservation.
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Diabetes Mellitus Tipo 1/tratamento farmacológico , Fatores Imunológicos/uso terapêutico , alfa 1-Antitripsina/uso terapêutico , Adolescente , Adulto , Criança , Diabetes Mellitus Tipo 1/patologia , Método Duplo-Cego , Feminino , Humanos , Fatores Imunológicos/efeitos adversos , Células Secretoras de Insulina/efeitos dos fármacos , Células Secretoras de Insulina/patologia , Masculino , Efeito Placebo , Resultado do Tratamento , Adulto Jovem , alfa 1-Antitripsina/efeitos adversosRESUMO
BACKGROUND: Poorly controlled adolescents living with type 1 diabetes (T1D) and pump failure of insulin delivery leading to diabetic ketoacidosis (DKA) are still challenging in the western world. AIM: To investigate the effect of a combination modality of long-acting insulin for basal coverage and a pump for boluses, on the incidence of DKA and glycemic parameters in pediatric and young adults with poorly controlled T1D. METHODS: This multicenter, observational retrospective study included 55 patients (age range 3-25 years, 52.7% males) who were treated with the combination modality for a median of 18 months [(IQR)12,47], as part of their clinical care. Data were retrieved at initiation of the combined modality, after 6 months, and at last visit. RESULTS: Cohort's median age at combination modality initiation was 14.5 years [IQR12.4,17.3], and its median HbA1c level was 9.2% [IQR 8.2,10.2]. The main reasons for combination modality initiation were: (a) concern about sustained hyperglycemia on current management in 41.8%, (b) previous DKA episodes in 30.8%, and (c) refusal to wear a pump continuously in 14.6%. The percent of patients experiencing DKA who used the modality till end decreased from 25.4 to 8.8%. The frequency of DKA events per patient month decreased after 6 months from 0.073 (min 0, max 0.5) to 0.020 (min 0, max 0.5), p = 0.01, and at end to 0.016 (min 0, max 0.25), p = 0.007. CONCLUSIONS: The combination modality of once-daily long-acting insulin and pump for boluses is safe, feasible, and effective in preventing DKA among poorly controlled young people living with T1D, unable or un-willing to use advanced closed pumps.
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INTRODUCTION: Activating mutation of the calcium-sensing receptor gene (CASR) reduces parathyroid hormone secretion and renal tubular reabsorption of calcium, defined as autosomal dominant hypocalcemia type 1 (ADH1). Patients with ADH1 may present with hypocalcemia-induced seizures. Calcitriol and calcium supplementation in symptomatic patients may exacerbate hypercalciuria, leading to nephrocalcinosis, nephrolithiasis, and compromised renal function. METHODS: We report on a family with seven members over three generations with ADH1 due to a novel heterozygous mutation in exon 4 of CASR: c.416T>C. RESULTS: This mutation leads to substitution of isoleucine with threonine in the ligand-binding domain of CASR. HEK293T cells transfected with wild type or mutant cDNAs demonstrated that p.Ile139Thr substitution led to increased sensitivity of the CASR to activation by extracellular calcium relative to the wild-type CASR (EC50 of 0.88 ± 0.02 m
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Hipocalcemia , Nefrocalcinose , Nefrolitíase , Humanos , Hipocalcemia/genética , Receptores de Detecção de Cálcio/genética , Cálcio , Hipercalciúria/genética , Creatinina , Células HEK293 , Mutação , ConvulsõesRESUMO
AIMS: To report a novel mutation associated with developmental delay, epilepsy, and neonatal diabetes-DEND Syndrome, responsive to a novel management combination. METHODS: We describe the investigation, treatment, and genetic diagnosis of a newborn diagnosed with DEND syndrome. RESULTS: The patient was found to be de-novo heterozygous for pathogenic KCNJ11 missense variant: c.190G > A, p. (Val64Met), associated with DEND syndrome, responsive to a combination of super high doses of sulfonylurea (SU) and oral high-dose steroids. A single case was reported so far due to this mutation, presenting with severe DEND syndrome, treated by insulin only. His phenotypic description and management during 18 months, demonstrates this mutation is responsive to super-high doses of SU combined with high dose 6 weeks steroids protocol. CONCLUSIONS: We have identified a heterozygous missense mutation as the etiology for severe DEND syndrome in a one-day old neonate, presenting with asymptomatic hyperglycemia, responsive to a novel management combination.
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Diabetes Mellitus , Canais de Potássio Corretores do Fluxo de Internalização/genética , Diabetes Mellitus/tratamento farmacológico , Diabetes Mellitus/genética , Heterozigoto , Humanos , Hipoglicemiantes , Recém-Nascido , Masculino , Mutação , Prednisolona , Compostos de SulfonilureiaRESUMO
CONTEXT: The rare hypoparathyroidism-retardation-dysmorphism (HRD) syndrome (OMIM #241410) is caused by the mutated tubulin chaperone E (TBCE) gene. This gene encodes a critical protein in the microtubule assembly pathway. OBJECTIVE: To evaluate the endocrine profile of patients with HRD. METHODS: The study used a retrospective analysis of a large cohort of patients in a single university medical center. Sixty-three patients were diagnosed with HRD during 1990 to 2019; 58 of them had an endocrine evaluation. MAIN OUTCOME MEASURES: We investigated somatic growth parameters, the prevalence of hypoglycemia, growth hormone deficiency, hypothyroidism, hypogonadism, and cortisol deficiency. RESULTS: All patients were born small for gestational age, and severe growth retardation was found in all patients with mean height standard deviation score (SDS) of -8.8 (range: -5.1 to -15.1) and weight SDS -18 (range: -5.1 to -61.2). Serum insulin-like growth factor-1 concentrations were very low among the 21 studied patients: -2.32 SDS (range: -0.6 to -2.7). Four out of 14 (28%) investigated patients had growth hormone deficiency, and 55% of patients were hospitalized due to symptomatic hypoglycemia. Adrenal glucocorticoid insufficiency was diagnosed in 22% of those tested. Hypothyroidism was found in 36% of patients. Both hypogonadotrophic and hypergonadotrophic hypogonadism were observed. The main magnetic resonance imaging findings were small anterior pituitary gland, small hippocampus, brain atrophy, thin corpus callosum, Chiari type I malformation, and septo-optic dysplasia. CONCLUSION: Multiple endocrine abnormalities are common in patients with HRD syndrome. Periodic screening of thyroid and adrenal functions is recommended.
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Anormalidades Múltiplas/patologia , Doenças do Sistema Endócrino/patologia , Transtornos do Crescimento/complicações , Hipoparatireoidismo/complicações , Deficiência Intelectual/complicações , Osteocondrodisplasias/complicações , Convulsões/complicações , Anormalidades Múltiplas/epidemiologia , Anormalidades Múltiplas/etiologia , Adolescente , Adulto , Criança , Pré-Escolar , Doenças do Sistema Endócrino/epidemiologia , Doenças do Sistema Endócrino/etiologia , Feminino , Seguimentos , Humanos , Incidência , Lactente , Recém-Nascido , Israel/epidemiologia , Masculino , Prognóstico , Estudos Retrospectivos , Fatores de Risco , Adulto JovemRESUMO
AIMS: Children with chronic diseases were unable to receive their usual care during COVID-19 lockdown. We assessed the feasibility and impact of telehealth visits on the time-in-range (TIR) of paediatric individuals with type 1 diabetes (T1D). METHODS: An observational multicentre real-life study. Patients scheduled for an in-clinic visit during the lockdown were offered to participate in a telehealth visit. Sociodemographic, clinical, continuous glucose monitor and pump data were recorded 2 weeks prior and 2 weeks after telehealth visit. The primary endpoint was change in relative-TIR, i.e. change in TIR divided by the percent of possible change (∆TIR/(100-TIRbefore)*100). RESULTS: The study group comprised 195 individuals with T1D (47.7% males), mean±SD age 14.6 ± 5.3 years, and diabetes duration 6.0 ± 4.6 years. Telehealth was accomplished with 121 patients and their parents (62.0%); 74 (38.0%) did not transfer complete data. Mean TIR was significantly higher for the two-week period after the telehealth visit than for the two-week period prior the visit (62.9 ± 16.0, p < 0.001 vs. 59.0 ± 17.2); the improvement in relative-TIR was 5.7±26.1%. Initial higher mean glucose level, lower TIR, less time spent at <54 mg/dl range, longer time spent at 180-250 mg/dl range, higher daily insulin dose, and single-parent household were associated with improved relative-TIR. Multiple regression logistic analysis demonstrated only initial lower TIR and single-parent household were significant, odds ratio: -0.506, (95%CI -0.99,-0.023), p=0.04 and 13.82, (95%CI 0.621, 27.016), p=0.04, respectively. CONCLUSIONS: Paediatric and young adult patients with T1D benefited from a telehealth visit during COVID-19. However, this modality is not yet suitable for a considerable proportion of patients.
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COVID-19/epidemiologia , Controle de Doenças Transmissíveis/tendências , Diabetes Mellitus Tipo 1/epidemiologia , Diabetes Mellitus Tipo 1/terapia , Controle Glicêmico/tendências , Telemedicina/tendências , Adolescente , Glicemia/metabolismo , Automonitorização da Glicemia/métodos , Automonitorização da Glicemia/tendências , COVID-19/prevenção & controle , Criança , Pré-Escolar , Estudos de Coortes , Controle de Doenças Transmissíveis/métodos , Diabetes Mellitus Tipo 1/sangue , Feminino , Controle Glicêmico/métodos , Humanos , Israel/epidemiologia , Masculino , Telemedicina/métodos , Adulto JovemRESUMO
BACKGROUND: Use of a continuous glucose-monitoring system (CGMS) in the management of type 1 diabetes (T1D) may cause local skin irritation. OBJECTIVE: To examine the effects of fluticasone propionate aqueous nasal solution (nsFP), sprayed topically prior to CGMS insertion among youth with T1D. METHODS: This is a case series observational report, including real-life 6-month follow-up data from one pediatric diabetes center. All patients suffering from local skin irritation due to CGMS adhesives were offered prevention form skin irritation by spraying 2 puffs of nsFP on the skin area prior to adhesion of CGMS. Data were collected from their charts after 6 months. Outcome measures included the difference in degree of skin irritation, number of days of CGMS use, BMI SDS, mean glucose, and HbA1c, prior to use and during 6 months after use. RESULTS: Twelve patients used nsFP prior to CGMS insertion, mean age 8.6 ± 4.9 years and 66.7% males. Ten patients, median age 6.1 years (5.3-9.5) and 56% males, continued using nsFP for a mean of 0.56 ± 0.11 years, with no recurrence of local irritation nor dermatitis to same adhesive material. No differences were found before and after use of nsFP in CGMS mean glucose 180 mg/dl (153-202) versus 165 mg/dl (150-192). BMI SDS was slightly higher 0.44 (- 0.9-1.2) versus 0.25 (- 0.47-1.06), P = 0.05. CONCLUSIONS: This small-scale, single-site description of a simple intervention by nsFP and favorable outcome provides valuable insight for a simple solution for skin irritation and dermatitis in the pediatric population with T1D.
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Diabetes Mellitus Tipo 1/sangue , Equipamentos e Provisões/efeitos adversos , Fluticasona/administração & dosagem , Dermatopatias/tratamento farmacológico , Dermatopatias/etiologia , Administração Tópica , Adolescente , Propelentes de Aerossol , Glicemia/análise , Automonitorização da Glicemia/efeitos adversos , Automonitorização da Glicemia/instrumentação , Criança , Pré-Escolar , Feminino , Seguimentos , Humanos , Lactente , Masculino , Resultado do Tratamento , Adulto JovemRESUMO
AIMS: Billions of people have been under lockdown in an attempt to prevent COVID-19 spread. Lifestyle changes during lockdown could lead to deterioration of glycemic control in type 1 diabetes (T1D). We aimed to assess the impact of COVID-19 lockdown on the glycemic control of pediatric patients with T1D. METHODS: This observational real-life study from the AWeSoMe Group assessed continuous glucose monitoring (CGM) metrics of 102 T1D patients (52.9% males, mean age 11.2 ± 3.8 years, mean diabetes duration 4.2 ± 3.8 years) who used Dexcom G5. The data were accessed without any interface between patients, caregivers, and the diabetes team. Study variables from CGM metrics were: mean glucose level, time-in-range (TIR, 70-180 mg/dL; 3.9-10 mmol/L), hypoglycemia (< 54 mg/dL; < 3 mmol/L), hyperglycemia (> 250 mg/dL; > 13.3 mmol/L), coefficient of variation (CV), and time CGM active before and during lockdown. Delta-variable = lockdown variable minus before-lockdown variable. RESULTS: The mean TIR was 60.9 ± 14.3% before lockdown, with no significant change during lockdown (delta-TIR was 0.9 ± 7.9%). TIR during lockdown was significantly correlated with TIR before lockdown (r = 0.855, P < 0.001). Patients with improved TIR (delta-TIR > 3%) were significantly older than patients with stable or worse TIR (P = 0.028). Children aged < 10 years had a significantly higher CV before lockdown and during lockdown than children aged ≥ 10 years (P = 0.02 and P = 0.005, respectively). Among children aged < 10 years, a multiple linear regression model revealed associations of age and lower socioeconomic cluster with delta-TIR (F = 4.416, P = 0.019) and with delta-mean glucose (F = 4.459, P = 0.018). CONCLUSIONS: CGM metrics in pediatric patients with T1D were relatively stable during a nationwide lockdown. Intervention plans should focus on younger patients with lower socioeconomic position.
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Automonitorização da Glicemia/métodos , Glicemia/análise , Infecções por Coronavirus/epidemiologia , Diabetes Mellitus Tipo 1/metabolismo , Pneumonia Viral/epidemiologia , Adolescente , Glicemia/metabolismo , Automonitorização da Glicemia/instrumentação , COVID-19 , Criança , Diabetes Mellitus Tipo 1/diagnóstico , Feminino , Humanos , Estudos Longitudinais , Masculino , PandemiasRESUMO
Background The purpose of this study was to assess the benefit of a short workshop addressing knowledge and confidence of inpatient caregivers (physicians and nurses) treating pediatric type 1 diabetes mellitus (T1DM) patients. Methods Questionnaires to determine familiarity with T1DM management and confidence in care were distributed at three university-affiliated medical centers. A 5-h practical-skills workshop was conducted at one center. Same questionnaires were filled out immediately after the workshop and 3 months later. Evaluation of knowledge was based on 16 multiple-choice questions, and confidence based on 10 categorical questions. Results Nurses and physicians (total 135, 106 women, 85 nurses) completed the questionnaires. The median knowledge score was 53.33 (40-66) and 46.7 (26.7-66.7) for nurses and physicians, respectively (p = 0.76). The confidence scores were 1.44 (1.1-1.9) and 1.56 (1.2-1.8), respectively (p = 0.7). More experience among nurses (>10 years) was associated with higher confidence scores (p = 0.04). Twenty-one physicians and 52 nurses, 66 women, attended the workshop. The median knowledge grade improved from 60 (47-67) to 81 (69-81), p < 0.001, immediately after, and to 69 (63-81), p < 0.001, 3 months post-workshop. The median confidence score improved from 1.8 (1.6-2) to 2.4 (2.1-2.6), p < 0.001, and to 2.2 (2-2.6), p < 0.001, respectively. There was no association with profession or seniority. Conclusions Knowledge and confidence in care of medical staff treating inpatient T1DM pediatric patients are lacking. Both improved significantly after the workshop, independent of seniority or profession.
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Competência Clínica/normas , Diabetes Mellitus Tipo 1/terapia , Conhecimentos, Atitudes e Prática em Saúde , Hospitalização/estatística & dados numéricos , Pacientes Internados/estatística & dados numéricos , Corpo Clínico/educação , Administração dos Cuidados ao Paciente/normas , Criança , Estudos Transversais , Feminino , Seguimentos , Humanos , Masculino , Inquéritos e QuestionáriosRESUMO
BACKGROUND: The use of insulin pumps in pediatric patients with type 1 diabetes (T1D) has expanded; however, data comparing devices remain scarce. We compared glycemic control, technical difficulties, and quality of life (QOL) between pump devices of three companies. METHODS: This prospective observational trial is based on clinical data retrieved during 12 months of follow-up of pediatric patients who started pump therapy as part of their clinical care in four medical centers. The devices fully reimbursed by the national health insurance are as follows: MiniMed™ 640G, MiniMed® Veo™, Animas® Vibe®, and Abbott OmniPod®. Parameters investigated included discontinuation rate, glycated hemoglobin (HbA1c), mean self-monitoring of blood glucose (SMBG), total daily dose of insulin (TDD), pump setting parameters, body mass index (BMI), frequency of technical difficulties, significant skin reactions, and QOL. RESULTS: Of 113 children (mean age 9.1 ± 4.1 years, 46% females), 68 (60%) used Medtronic devices, 33 (29%) OmniPod, and 12 (11%) Animas devices. Twelve percent of the cohort stopped using the pump during the study period, with no difference according to device. There were no differences between groups regarding mean SMBG values (P = 0.86), insulin TDD (P = 0.24), BMI (P = 0.87), level of insertion site pain or local reaction (P = 0.51), technical problems (P = 0.66), and QOL (P = 0.38). Changes in HbA1c from study initiation to end were also not statistically significant for any of the devices: from 7.99% ± 1.14% to 7.93% ± 0.99% for Medtronic, from 7.71% ± 1.29% to 7.92% ± 1.38% for OmniPod, and from 8.75% ± 1.3% to 7.70% ± 1.33% for Animas (P = 0.63). CONCLUSIONS: Pump devices were comparable regarding glycemic control, weight gain, and satisfaction among pediatric patients with T1D.
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Automonitorização da Glicemia/instrumentação , Diabetes Mellitus Tipo 1/tratamento farmacológico , Diabetes Mellitus Tipo 1/psicologia , Sistemas de Infusão de Insulina/estatística & dados numéricos , Satisfação do Paciente/estatística & dados numéricos , Adolescente , Glicemia/análise , Automonitorização da Glicemia/psicologia , Criança , Diabetes Mellitus Tipo 1/sangue , Feminino , Hemoglobinas Glicadas/análise , Humanos , Hipoglicemiantes/administração & dosagem , Insulina/administração & dosagem , Sistemas de Infusão de Insulina/psicologia , Masculino , Estudos Prospectivos , Qualidade de VidaRESUMO
Unfortunately, the co-author name was misspelled as "Avivit Brenner" instead of "Avivit Brener" in the original publication and the correct author name is updated here.
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AIMS: Frequent glucose testing is required for optimal management of type 1 diabetes (T1D). Limited data are available regarding real-world experience of the novel technology for monitoring by continuous interstitial fluid glucose (IFG), using flash glucose-sensing technology (FSL-CGM). We aimed to assess the effect of FSL-CGM in a real-life clinical setting on glycemic control parameters, compliance, and adverse events among pediatric and young adult T1D patients. METHODS: This observational multi-center study assessed FSL-CGM use (6-12 months) in T1D patients (mean ± SD age 13.4 ± 4.9 years) who purchased the device out-of-pocket. Outcome measures included HbA1c, mean IFG levels, CGM metrics [time in hypoglycemia (< 54 mg/dL; < 3 mmol/L), in target range (70-180 mg/dL; 3.9-10 mmol/L), and in hyperglycemia > 240 mg/dL; > 13.3 mmol/L)], frequency of self-monitoring of blood glucose, acute complications, skin reactions, and reasons for initiation/discontinuation. RESULTS: Among patients with regular use of the FSL-CGM (n = 59), mean HbA1c decreased from 8.86 ± 0.23 to 8.05 ± 0.2% (73.3-64.5 mmol/mol) in 3 months (p = 0.0001) and plateaued thereafter. A clinically significant reduction in HbA1c (defined as a decrease of ≥ 0.5%) was associated with shorter diabetes duration. Of 71 patients who initiated use of the FSL-CGM, 12 (16.9%) discontinued during the study period. No statistically significant changes were found after FSL-CGM use, in mean and standard deviation IFG levels, and in time of glucose levels in target, hypoglycemia, and hyperglycemia ranges. One patient with hypoglycemia unawareness was found dead-in-bed while using FSL-CGM. CONCLUSIONS: Real-life observational data in a self-selected young T1D population demonstrated a significant and sustained reduction in HbA1c with FSL-CGM in one-third of the participants. Surveillance of glucose monitoring should be individualized, especially for patients with hypoglycemia unawareness.
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Técnicas Biossensoriais/métodos , Glicemia/análise , Diabetes Mellitus Tipo 1/sangue , Adolescente , Adulto , Técnicas Biossensoriais/instrumentação , Automonitorização da Glicemia/instrumentação , Criança , Pré-Escolar , Feminino , Humanos , Hiperglicemia/sangue , Hiperglicemia/diagnóstico , Hipoglicemia/sangue , Hipoglicemia/diagnóstico , Masculino , Cooperação do Paciente , Adulto JovemRESUMO
OBJECTIVE: To evaluate the effect of disease severity and colchicine treatment on height and weight parameters in children with familial Mediterranean fever (FMF). METHODS: Thirty prepubertal children (19 M, 11 F) were studied retrospectively. Z-score values of height, growth velocity, weight and body mass index were obtained over 1.84 +/- 1.14 years before and 2.58 +/- 1.55 years during colchicine therapy. Disease severity was evaluated by a specific score for FMF. RESULTS: By comparison to growth before treatment, during colchicine therapy height SDS increased from -1.00 +/- 1.17 to -0.54 +/- 0.96 (p < 0.001) and weight SDS increased from -0.74 +/- 1.09 to -0.47 +/- 1.06 (p = 0.008). An effect of disease severity on growth pattern could not be detected. Height SDS during therapy was negatively correlated with age at colchicine initiation. CONCLUSIONS: Colchicine therapy has a positive effect on both height and weight parameters in children with FMF. Early initiation of treatment is beneficial for height gain.