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2.
Pharmacol Res ; 195: 106882, 2023 09.
Artigo em Inglês | MEDLINE | ID: mdl-37543096

RESUMO

Type 1 diabetes (T1D) is the most frequent form of diabetes in pediatric age, affecting more than 1.5 million people younger than age 20 years worldwide. Early and intensive control of diabetes provides continued protection against both microvascular and macrovascular complications, enhances growth, and ensures normal pubertal development. In the absence of definitive reversal therapy for this disease, achieving and maintaining the recommended glycemic targets is crucial. In the last 30 years, enormous progress has been made using technology to better treat T1D. In spite of this progress, the majority of children, adolescents and young adults do not reach the recommended targets for glycemic control and assume a considerable burden each day. The development of promising new therapeutic advances, such as more physiologic insulin analogues, pioneering diabetes technology including continuous glucose monitoring and closed loop systems as well as new adjuvant drugs, anticipate a new paradigm in T1D management over the next few years. This review presents insights into current management of T1D in youths.


Assuntos
Diabetes Mellitus Tipo 1 , Adulto Jovem , Humanos , Adolescente , Criança , Adulto , Diabetes Mellitus Tipo 1/tratamento farmacológico , Hipoglicemiantes/uso terapêutico , Controle Glicêmico , Automonitorização da Glicemia , Glicemia , Insulina/uso terapêutico , Sistemas de Infusão de Insulina
4.
Rev. Rol enferm ; 46(4): 17-27, abr. 2023. tab, ilus
Artigo em Espanhol | IBECS | ID: ibc-219130

RESUMO

Objetivo: Determinar en población infantil con Diabetes tipo 1 (DT1) en tratamiento con infusión subcutánea continua de insulina (ISCI), si asumir responsabilidades de autocuidado tras recibir un programa estructurado de educación terapéutica (PEET) se relaciona con el control metabólico y la calidad de vida (CV). Métodos: Estudio observacional, transversal. Se realizó un sub-análisis retrospectivo. Se incluyeron sujetos con DT1 (edad 9-17 años) en terapia ISCI (>1año) que habían recibido el mismo PEET al inicio de ISCI. Se registraron: grado en que asumían responsabilidades de autocuidado acordes a su edad, control metabólico, CV, nivel de conocimientos sobre diabetes y uso de funciones específicas del dispositivo. Resultados: Se incluyeron 44 pacientes. Los niños que asumieron responsabilidades de autocuidado acordes a su edad presentaron valores de hemoglobina glicada (HbA1c) significativamente menores que los niños que no las asumieron (8,0±0,7% vs. 9,2±1,1%, respectivamente, p<0,001), así como una mayor puntuación en los cuestionarios de CV y de conocimientos (CV 84,3±9,3 vs. 79,4±10,6, p<0,01; conocimientos 27,9±4,2 vs. 26,5±4,3, respectivamente, n.s). El uso de las funciones específicas de la bomba se observó principalmente en aquellos que asumieron esas responsabilidades de autocuidado presentando valores más bajos de HbA1c que aquellos niños que no las utilizaron (7,9±1,0% vs. 8,4±0,8%, p<0,05). Conclusiones: Los pacientes con DT1 en tratamiento con ISCI que asumieron responsabilidades de autocuidado de su diabetes acorde a su edad, mostraron mejor control de HbA1c y mejor CV que aquellos que no lo hicieron. Se necesitan más estudios para profundizar en el conocimiento de estos aspectos. (AU)


Objective: The aim of this study was to determine if children and adolescents with type 1 diabetes (DT1) managed with continuous subcutaneous insulin infusion (ISCI) who assume self-care responsibilities tailored to the age after a specific structured education program (PEET), present better metabolic control and quality of life (CV). Methods: A observational, cross-sectional study was conducted. A retrospective sub-analysis was performed. Subjects with DT1 (aged 9-17 years) who have been using ISCI (>1year) were included. All patients received the same structured PEET when initiating ISCI treatment. The degree of self-care age-appropriate responsibilities assumed by children was registered. Data related to metabolic control, diabetes knowledge, use of different pump features, and quality of life were also collected. Results: Forty-four patients were included. Children assuming age-appropriate self-care responsibilities had a significantly lower glycated hemoglobin (HbA1c) value compared to those children who did not take on these responsibilities (8,0±0,7% vs. 9,2±1,1%, p<0,001). as well as higher scores in the CV and knowledge questionnaires (84,3±9,3 vs. 79,4±10,6 respectively, p<0,01; knowledge 27,9±4,2 vs. 26,5±4,3, respectively, n.s). The use of specific pump features was mainly observed in those who assumed age-appropriate self-care responsibilities and showed lower HbA1c values than those children who did not take on these responsibilities (7,9±1,0% vs. 8,4±0,8%, p<0,05). Conclusion: Patients with DT1 managed with ISCI, who assumed age-appropriate responsibilities on disease self- management, showed better HbAc1 and better CV than those who did not. More studies are needed to deepen the knowledge of these topics. (AU)


Assuntos
Humanos , Masculino , Feminino , Criança , Adolescente , Infusões Subcutâneas , Diabetes Mellitus Tipo 1/tratamento farmacológico , Insulina/uso terapêutico , Pediatria , Estudos Transversais , Qualidade de Vida
5.
JAMA Netw Open ; 6(2): e230077, 2023 02 01.
Artigo em Inglês | MEDLINE | ID: mdl-36808243

RESUMO

Importance: Continuous glucose monitoring (CGM) devices have demonstrated efficacy in adults and more recently in youths and older adults with type 1 diabetes. In adults with type 1 diabetes, the use of real-time CGM compared with intermittently scanned CGM was associated with improved glycemic control, but there are limited data available for youths. Objective: To assess real-world data on achievement of time in range clinical targets associated with different treatment modalities in youths with type 1 diabetes. Design, Setting, and Participants: This multinational cohort study included children, adolescents, and young adults younger than 21 years (hereinafter referred to collectively as youths) with type 1 diabetes for a duration of at least 6 months who provided CGM data between January 1, 2016, and December 31, 2021. Participants were enrolled from the international Better Control in Pediatric and Adolescent Diabetes: Working to Create Centers of Reference (SWEET) registry. Data from 21 countries were included. Participants were divided into 4 treatment modalities: intermittently scanned CGM with or without insulin pump use and real-time CGM with or without insulin pump use. Exposures: Type 1 diabetes and the use of CGM with or without an insulin pump. Main Outcomes and Measures: Proportion of individuals in each treatment modality group achieving recommended CGM clinical targets. Results: Among the 5219 participants (2714 [52.0%] male; median age, 14.4 [IQR, 11.2-17.1] years), median duration of diabetes was 5.2 (IQR, 2.7-8.7) years and median hemoglobin A1c level was 7.4% (IQR, 6.8%-8.0%). Treatment modality was associated with the proportion of individuals achieving recommended clinical targets. Adjusted for sex, age, diabetes duration, and body mass index standard deviation score, the proportion achieving the recommended greater than 70% time in range target was highest with real-time CGM plus insulin pump use (36.2% [95% CI, 33.9%-38.4%]), followed by real-time CGM plus injection use (20.9% [95% CI, 18.0%-24.1%]), intermittently scanned CGM plus injection use (12.5% [95% CI, 10.7%-14.4%]), and intermittently scanned CGM plus insulin pump use (11.3% [95% CI, 9.2%-13.8%]) (P < .001). Similar trends were observed for less than 25% time above (real-time CGM plus insulin pump, 32.5% [95% CI, 30.4%-34.7%]; intermittently scanned CGM plus insulin pump, 12.8% [95% CI, 10.6%-15.4%]; P < .001) and less than 4% time below range target (real-time CGM plus insulin pump, 73.1% [95% CI, 71.1%-75.0%]; intermittently scanned CGM plus insulin pump, 47.6% [95% CI, 44.1%-51.1%]; P < .001). Adjusted time in range was highest among real-time CGM plus insulin pump users (64.7% [95% CI, 62.6%-66.7%]). Treatment modality was associated with the proportion of participants experiencing severe hypoglycemia and diabetic ketoacidosis events. Conclusions and Relevance: In this multinational cohort study of youths with type 1 diabetes, concurrent use of real-time CGM and an insulin pump was associated with increased probability of achieving recommended clinical targets and time in range target as well as lower probability of severe adverse events compared with other treatment modalities.


Assuntos
Diabetes Mellitus Tipo 1 , Insulinas , Adulto Jovem , Humanos , Masculino , Adolescente , Criança , Idoso , Feminino , Diabetes Mellitus Tipo 1/tratamento farmacológico , Hipoglicemiantes/uso terapêutico , Glicemia , Estudos de Coortes , Automonitorização da Glicemia , Insulinas/uso terapêutico
6.
Eur J Pediatr ; 182(5): 1949-1963, 2023 May.
Artigo em Inglês | MEDLINE | ID: mdl-36809498

RESUMO

Hybrid closed loop (HCL) systems are the combination of a pump for insulin delivery and a glucose sensor for continuous glucose monitoring. These systems are managed by an algorithm, which delivers insulin on the basis of the interstitial glucose levels. The MiniMed™ 670G system was the first HCL system available for clinical purpose. In this paper, we reviewed the literature about metabolic and psychological outcomes in children, adolescents and young adults with type 1 diabetes treated with MiniMed™ 670G. Only 30 papers responded to the inclusion criteria and thus were considered. All the papers show that the system is safe and effective in managing glucose control. Metabolic outcomes are available up to 12 months of follow-up; longer study period are lacking. This HCL system may improve HbA1c up to 7.1% and time in range up to 73%. The time spent in hypoglycaemia is almost neglectable. Better improvement in blood glucose control is observed in patients with higher HbA1c at HCL system start and larger daily use of auto-mode functionality.     Conclusion: The Medtronic MiniMed™ 670G is safe and well accepted, without any increase in the burden for patients. Some papers report an improvement in the psychological outcomes, but other papers do not confirm this finding. So far, it significantly improves the management of diabetes mellitus in children, adolescents and young adults. Proper training and support by the diabetes team are mandatory. Studies for a period longer than 1 year would be appreciated to better understand the potentiality of this system. What is Known: • The Medtronic MiniMedTM 670G is a hybrid closed loop system which combines a continuous glucose monitoring sensor with an insulin pump. • It has been the first hybrid closed loop system available for clinical purpose. Adequate training and patients support play a key role in diabetes management. What is New: • The Medtronic MiniMedTM 670G may improve HbA1c and CGM metrics up to 1-year of follow-up, but the improvement appears lower than advanced hybrid closed loop systems. This system is effective to prevent hypoglycaemia. • The psychosocial effects remain less understood in terms of improvement of psychosocial outcomes. The system has been considered to provide flexibility and independence by the patients and their caregivers. The workload required to use this system is perceived as a burden by the patients who decrease the use of auto-mode functionality over time.


Assuntos
Diabetes Mellitus Tipo 1 , Hipoglicemia , Humanos , Criança , Adolescente , Adulto Jovem , Hipoglicemiantes/uso terapêutico , Hemoglobinas Glicadas , Glicemia , Automonitorização da Glicemia , Diabetes Mellitus Tipo 1/tratamento farmacológico , Insulina/uso terapêutico , Hipoglicemia/tratamento farmacológico
7.
J Diabetes Sci Technol ; 17(5): 1326-1336, 2023 09.
Artigo em Inglês | MEDLINE | ID: mdl-35470692

RESUMO

New metrics for assessing glycemic control beyond HbA1c have recently emerged due to the increasing use of continuous glucose monitoring (CGM) in diabetes clinical practice. Among them, time in range (TIR) has appeared as a simple and intuitive metric that correlates inversely with HbA1c and has also been newly linked to the risk of long-term diabetes complications. The International Consensus on Time in Range established a series of target glucose ranges (TIR, time below range and time above range) and recommendations for time spent within these ranges for different diabetes populations. These parameters should be evaluated together with the ambulatory glucose profile (AGP). Using standardized visual reporting may help people with diabetes and healthcare professionals in the evaluation of glucose control in frequent clinical situations. The objective of the present review is to provide practical insights to quick interpretation of patient-centered metrics based on flash glucose monitoring data, as well as showing some visual examples of common clinical situations and giving practical recommendations for their management.


Assuntos
Glicemia , Diabetes Mellitus , Humanos , Automonitorização da Glicemia , Benchmarking , Hemoglobinas Glicadas , Controle Glicêmico , Diabetes Mellitus/terapia , Assistência Centrada no Paciente
8.
J Pediatr Nurs ; 72: 146-152, 2023.
Artigo em Inglês | MEDLINE | ID: mdl-36446693

RESUMO

Evaluation of the degree of adherence to self-care among Spanish type 1 diabetes (T1DM) pediatric population lacks of a validated tool. PURPOSE: To cross-culturally adapt and determine the psychometric properties of the Spanish version of the Diabetes Management Questionnaire to assess the degree of adherence to self-care among children with T1DM. METHODS: Translation, back-translation, and patient suggestions, were considered to obtain the Spanish version (DMQ-Sp). A cross-sectional study was conducted with 323 children (aged 8-18 years) with T1DM and their parents to determine internal reliability, structural validity, and external validity. Responsiveness to change was analyzed through a prospective longitudinal study involving 102 newly diagnosed T1DM patients. Psychometrics were evaluated for the entire sample and stratified by age (8-12 and 13-18 years). RESULTS: A total of 323 children with T1DM [49.8% female; age 13.3 ± 2.8 years; 155 aged 8-12; glycated hemoglobin (HbA1c) value 7.7 ± 1.0%] answered the Spanish final version. The internal consistency Cronbach's alpha was 0.76 and intraclass correlation coefficient 0.84. Test-retest reliability was r = 0.84 (p < 0.001). Fit index of structural validity was >0.7. External validity correlated inversely with HbA1c (r = -0.39; p < 0.001). The DMQ-Sp score increased significantly after 6 months of receiving the full therapeutic education program (TEP) (baseline 57.07 ± 10.81 vs. 6 months 78.80 ± 10.31; p < 0.001). CONCLUSION: The DMQ-Sp is reliable, valid, and sensitive to change in a large sample of children (aged 8-18 years) with T1DM and their parents. PRACTICE IMPLICATIONS: DMQ-Sp can be a useful tool for diabetes teams to identify adherence to different tasks and to evaluate TEPs.

10.
Patient Prefer Adherence ; 16: 2229-2246, 2022.
Artigo em Inglês | MEDLINE | ID: mdl-36017376

RESUMO

Introduction: The management of type 1 diabetes (T1D) is especially complex during adolescence when youths are transferred to adult care centres. The objectives of this qualitative study were to: a) determine the expectations of young T1D patients prior to transfer, b) evaluate the transfer process between the 2 centres, and c) evaluate the therapeutic education and care programme (TECP) in the adult centre from their point of view. Material and Methods: Opinion sampling of adolescents from 2018-2019 was performed: Phase 1: adolescents with T1D prior to transfer to the adult hospital; Phase 2: adolescents with T1D one or two years after transfer and having undergone TECP. A focus group (1.5h) and semi-structured interviews (45 min) were performed and taped, transcribed, and sent to the participants for confirmation. Date analysis was performed of the transcriptions of the focus group and interviews. Fragments were selected and meta-categories created. Results: Eleven youths accepted to participate: 7 in Phase 1, 4 repeated in Phase 2 and 4 more transferred 2 years previously were added. The meta-categories obtained were: 1) perception of the quality of care and therapeutic education in the paediatric hospital. 2) transfer to the adult hospital. 3) experience of the youths 1-2 years after transfer and having undergone TECP. The data are presented in narrative form and are supported with text fragments of the participants' discussions. Discussion and conclusions: Analysis of patients' experience complemented by clinical-educational evaluation of TECP provides understanding of the perspectives of youths on the complexity of living with a chronic disease since childhood. It also provides information regarding the factors favouring quality care and therapeutic education, the complexity of transfer from paediatric to adult care, determination of the strong and weak points and the establishment of strategies to improve the programme.

11.
Pediatr Diabetes ; 23(5): 536-544, 2022 08.
Artigo em Inglês | MEDLINE | ID: mdl-35872603

RESUMO

OBJECTIVE: The following report describes the evaluation of the ISPAD Science School for Physicians (ISSP) and for Healthcare Professionals (ISSHP) in terms of their efficiency and success. METHODS: All past attendees from 2000-2019 ISSP and 2004-2019 ISSHP programs were invited to respond to an online survey to assess perceived outcomes of the programs on career development, scientific enhancement, scientific networking, and social opportunities. RESULTS: One-third of the past ISSP (129/428), and approximately 43% of the past ISSHP attendees (105/245) responded to the surveys. Most of ISSP attendees reported that the programs supported their career (82%) by helping to achieve a research position (59%), being engaged with diabetes care (68%) or research (63%) or starting a research fellowship (59%). Responders indicated that ISSP was effective in increasing interest in diabetes research (87%) and enhancing the number (66%) and quality (83%) of scientific productions, and promotion of international collaborations (86%). After the ISSP, 34% of responders received research grants. From the first round of the ISSHP survey (2004-2013), responders reported have improved knowledge (60%), gained more confidence in research (69%), undertaken a research project (63%), and achieved a higher academic degree (27%). From the second round (2014-2019), participants indicated that the program was valuable/useful in workplace (94%) through understanding (89%) and conducting (68%) research and establishing communication from other participants (64%) or from faculty (42%). After the ISSHP, 17% had received awards. CONCLUSIONS: From the participants' viewpoint, both programs were effective in improving engagement with diabetes research, supporting career opportunities, increasing scientific skills, and enhancing networking and research activities.


Assuntos
Diabetes Mellitus , Instituições Acadêmicas , Adolescente , Criança , Diabetes Mellitus/terapia , Pessoal de Saúde , Humanos
12.
J Clin Med ; 11(15)2022 Jul 26.
Artigo em Inglês | MEDLINE | ID: mdl-35893428

RESUMO

Objective: To assess the impact of the COVID-19 pandemic and lockdown measures on the presenting characteristics (age at diagnosis, severity, monthly distribution) of newly diagnosed type 1 diabetes in Spanish children. Research Design and Methods: An ambispective observational multicenter study was conducted in nine Spanish tertiary-level hospitals between January 2015 and March 2021. Inclusion criteria: new cases of type 1 diabetes in children (0-14 years) recording age, sex, date of diagnosis, presence of diabetic ketoacidosis (DKA) at onset, and severity of DKA. Data were compared before and during the pandemic. Results: We registered 1444 new cases of type 1 diabetes in children: 1085 in the pre-pandemic period (2015-2019) and 359 during the pandemic (2020-March 2021). There was a significant increase in the group aged ≤4 years in the pandemic period (chi-squared = 10.986, df 2, p = 0.0041). In 2020-2021, cases of DKA increased significantly by 12% (95% CI: 7.2-20.4%), with a higher percentage of moderate and severe DKA, although this increase was not significant. In 2020, there was a sharp decrease in the number of cases in March, with a progressive increase from May through November, higher than in the same months of the period 2015-2019, highlighting the increase in the number of cases in June, September, and November. The first three months of 2021 showed a different trend to that observed both in the years 2015-2019 and in 2020, with a marked increase in the number of cases. Conclusions: A change in monthly distribution was described, with an increase in DKA at onset of type 1 diabetes. No differences were found in severity, although there were differences in the age distribution, with an increase in the number of cases in children under 4 years of age.

13.
Pediatr Diabetes ; 22(8): 1115-1119, 2021 12.
Artigo em Inglês | MEDLINE | ID: mdl-34741569

RESUMO

BACKGROUND: Telemedicine for routine care of people with diabetes (PwD) during the COVID-19 pandemic rapidly increased in many countries, helping to address the several barriers usually seen. OBJECTIVE: This study aimed to describe healthcare professionals' (HCPs) experience on telemedicine use in diabetes care and investigate the changes and challenges associated with its implementation. METHODS: A cross-sectional electronic survey was distributed through the global network of JENIOUS members of ISPAD. Respondents' professional and practice profiles, clinic sizes, their country of practice, and data regarding local telemedicine practices during COVID-19 pandemic were investigated. RESULTS: Answers from 209 HCPs from 33 countries were analyzed. During the pandemic, the proportion of PwD receiving telemedicine visits increased from <10% (65.1% of responders) to >50% (66.5%). There was an increase in specific privacy requirements for remote visits (37.3% to 75.6%), data protection policies (42.6% to 74.2%) and reimbursement for remote care (from 41.1% to 76.6%). Overall, 83.3% HCPs reported to be satisfied with the use of telemedicine. Some concerns (17.5%) about the complexity and heterogeneity of the digital platforms to be managed in everyday practice remain, feeding the need for unifying and making interoperable the tools for remote care. Also, 45.5% of professionals reported to feel stressed by the need for extra-time for telemedicine consultations. CONCLUSIONS: Telemedicine was rapidly and broadly adopted during the pandemic globally. Some issues related to its use were promptly addressed by local institutions. Challenges with the use of different platforms and for the need of extra-time still remain to be solved.


Assuntos
Diabetes Mellitus/terapia , Telemedicina , Adulto , Idoso , Idoso de 80 Anos ou mais , COVID-19 , Estudos Transversais , Feminino , Pessoal de Saúde , Humanos , Internacionalidade , Internet , Masculino , Pessoa de Meia-Idade , Pandemias , Privacidade , Inquéritos e Questionários
14.
Diabetes Care ; 44(5): 1176-1184, 2021 05.
Artigo em Inglês | MEDLINE | ID: mdl-33653821

RESUMO

OBJECTIVE: Insulin delivery methods, glucose-monitoring modalities, and related outcomes were examined in a large, international, diverse cohort of children and adolescents with type 1 diabetes from the Better Control in Pediatric and Adolescent Diabetes: Working to Create Centers of Reference (SWEET) -Registry. RESEARCH DESIGN AND METHODS: Participants with type 1 diabetes of ≥1 year, aged ≤18 years, and who had documented pump or sensor usage during the period August 2017-July 2019 were stratified into four categories: injections-no sensor (referent); injections + sensor; pump-no sensor; and pump + sensor. HbA1c and proportion of patients with diabetic ketoacidosis (DKA) or severe hypoglycemia (SH) were analyzed; linear and logistic regression models adjusted for demographics, region, and gross domestic product per capita were applied. RESULTS: Data of 25,654 participants were analyzed. The proportions of participants (adjusted HbA1c data) by study group were as follows: injections-no sensor group, 37.44% (8.72; 95% CI 8.68-8.75); injections + sensor group, 14.98% (8.30; 95% CI 8.25-8.35); pump-no sensor group, 17.22% (8.07; 95% CI 8.03-8.12); and pump + sensor group, 30.35% (7.81; 95% CI 7.77-7.84). HbA1c was lower in all categories of participants who used a pump and/or sensor compared with the injections-no sensor treatment method (P < 0.001). The proportion of DKA episodes was lower in participants in the pump + sensor (1.98%; 95% CI 1.64-2.48; P < 0.001) and the pump-no sensor (2.02%; 95% CI 1.64-2.48; P < 0.05) groups when compared with those in the injections-no sensor group (2.91%; 95% CI 2.59-3.31). The proportion of participants experiencing SH was lower in pump-no sensor group (1.10%; 95% CI 0.85-1.43; P < 0.001) but higher in the injections + sensor group (4.25%; 95% CI 3.65-4.95; P < 0.001) compared with the injections-no sensor group (2.35%; 95% CI 2.04-2.71). CONCLUSIONS: Lower HbA1c and fewer DKA episodes were observed in participants using either a pump or continuous glucose monitoring (CGM) or both. Pump use was associated with a lower rate of SH. Across SWEET centers, use of pumps and CGM is increasing. The concomitant use of pump and CGM was associated with an additive benefit.


Assuntos
Diabetes Mellitus Tipo 1 , Adolescente , Glicemia , Automonitorização da Glicemia , Criança , Diabetes Mellitus Tipo 1/tratamento farmacológico , Hemoglobinas Glicadas/análise , Humanos , Hipoglicemiantes/efeitos adversos , Insulina/efeitos adversos , Sistemas de Infusão de Insulina , Sistema de Registros
15.
Endocrine ; 71(3): 626-633, 2021 03.
Artigo em Inglês | MEDLINE | ID: mdl-33527330

RESUMO

BACKGROUND: Wolfram Syndrome is a very rare genetic disease causing diabetes mellitus, blindness, deafness, diabetes insipidus, and progressive brainstem degeneration. Neurologic symptoms of affected patients include ataxia, sleep apnea, loss of bladder control, dysphagia, loss of taste, and accompanying psychiatric symptoms as a sign of progressive neurodegeneration. Its genetic cause is mainly biallelic mutations of the Wolframin endoplasmatic reticulum transmembrane glycoprotein gene Wfs1. These result in increased ER stress, which in turn induces apoptosis and leads to the depletion of the corresponding cells and a loss of their physiological functions. Though diabetes mellitus is mostly treated by insulin, there is still no proven cure for the disease in general. It leads to premature death in affected individuals-usually within the 4th decade of live. CURRENT RESEARCH AND TREATMENT TRIALS: Clinical studies are currently being conducted at various locations worldwide to test a therapy for the disease using various approaches. POTENTAIL OF VIRTUAL NETOWRKING: As rare diseases in general represent a major challenge for individual clinicians and researchers due to the rarity of diagnosis, the lack of evidence and of value of existing research, international cooperation, coordination and networking leading to an alignment of different stakeholders is necessary to support patients and increase knowledge about these diseases, like wolfram syndrome. CONCLUSION: ENDO-ERN and EURRECA are two EU-funded networks that aim to promote knowledge sharing, education and research on rare endocrine diseases.


Assuntos
Transtornos Mentais , Síndrome de Wolfram , Humanos , Proteínas de Membrana/genética , Mutação , Síndrome de Wolfram/diagnóstico , Síndrome de Wolfram/genética , Síndrome de Wolfram/terapia
16.
Endocrinol Diabetes Nutr (Engl Ed) ; 68(2): 82-91, 2021 Feb.
Artigo em Inglês, Espanhol | MEDLINE | ID: mdl-32912806

RESUMO

OBJECTIVE: Evaluate the results of a healthcare and therapeutic education programme (TEP) aimed at young patients with type 1 diabetes (T1D) transferred from a paediatric centre. METHODOLOGY: This was a prospective, pre-postest in young T1D patients transferred from 2005-2015. The programme has four phases: coordinated transfer, evaluation and objective pacting, knowledge (DKQ2) adherence (SCI-R.es) and quality of life (DQoL and SF12). Results were compared according to Multiple Daily Injections (MDI) vs. Continuous Subcutaneous Insulin Infusión (CSII) and adherence (SCI-R.es < 65 vs. > 65%). RESULTS: A total of 330 patients were transferred (age 18.19 ± 0.82 years, 49% females, glycated haemoglobin [HbA1c] 8.6 ± 1.4%). The programme was completed by 68%, and 61% did a group course. While no changes in HbA1c were observed at one year (8.3 ± 1.4 vs. 8.2 ± 1.4%), there were changes in severe hypoglycaemias/patient/year (0.23 ± 0.64 to 0.05 ± 0.34 p < 0.001) and mild > 5 hypoglycaemias/patient/week (6.9% vs. 3.9% p = 0.09). DQK2 knowledge increased (25.7 ± 3.6 vs. 27.8 ± 3.8 p < 0.001), with no changes in quality of life or grade of adherence. Patients with CSII (n = 21) performed more blood glucose controls and showed greater programme adherence with no changes in metabolic control. Patients with the best initial adherence presented the best control (p < 0.0001). A lower initial HbA1c and receiving the group course were associated with better clinical HbA1c results ≥ 0.5% (p < 0.05) CONCLUSIONS: The TEP improved some parameters of metabolic control without modifying the quality of life in young T1D patients. When comparing patients on MDI vs. CSII, there were no differences in metabolic control but there were when differences were evaluated considering treatment adherence.

17.
Diabetes Res Clin Pract ; 171: 108587, 2021 Jan.
Artigo em Inglês | MEDLINE | ID: mdl-33307134

RESUMO

COVID-19 has gravely threatened high-risk populations, such as people with diabetes and other noncommunicable diseases, leading to disproportionate hospitalizations and deaths worldwide. It is well documented from previous outbreaks that diabetes increases the risk for poor outcomes due to SARS infection. In the present review, we bring evidence that the country and global level health crisis caused by COVID-19 could have been avoided or extremely minimized if measures to protect high-risk populations were implemented timely. In addition to general lockdowns, testing, tracing, isolation and hygiene measures, other specific interventions for diabetes and comorbidities management were shown crucial to allow the continuation of care services during the pandemic. These interventions included: teleconsultation, digital remote education andmonitoring, e-prescriptions, medicine delivery options, mobile clinics, and home point-of-care tests. In conclusion, we recommend prompt actions to protect the most vulnerable groups, valuing knowledge and experiences from previous outbreaks and lessons learned during the COVID-19 pandemic, in order to shield communities, health systems and the global economy.


Assuntos
COVID-19/complicações , Saúde Global , Doenças não Transmissíveis/epidemiologia , SARS-CoV-2/isolamento & purificação , COVID-19/transmissão , COVID-19/virologia , Comorbidade , Humanos
18.
Pediatr Diabetes ; 22(2): 202-206, 2021 03.
Artigo em Inglês | MEDLINE | ID: mdl-33205546

RESUMO

The severe acute respiratory syndrome coronavirus 2 (SARS-Cov-2), responsible for the coronavirus disease COVID-19, was first identified in Wuhan, China in December 2019. Diabetes, as well as other cardiovascular comorbidities, has been recognized as a major risk factor for outcomes and mortality in adults with COVID-19, particularly in the elderly with type 2 diabetes. Based on these conclusions, COVID-19 data on adults have been generalized to youth with diabetes. Nevertheless, experience from pediatric diabetes practices in China (Wuhan), Italy, Spain (Catalonia), and the United States (San Francisco Bay Area) consistently report only a single severe case of COVID-19 in a 20-year-old female youth with type 1 diabetes (T1D) that was hospitalized for bilateral pneumonia and was subsequently discharged without complications. In Italy, information on COVID-19 in all children with diabetes is collected on a weekly basis and those with positive swab test or infection-related symptoms reported to a dedicated national registry. Of a total of 15 500 children tested, 11 subjects with T1D (age 8-17y) tested positive for COVID-19; 6/11 were asymptomatic and the rest presented with mild symptoms. In the rest of locations, youths with T1D diagnosed with COVID-19 were based on clinical suspicion and a confirmatory PCR test (Wuhan:0; Catalonia-HSJD:3; California-Stanford:2). All of them were asymptomatic or had a mild course. We suggest that COVID-19 data from adults should not be generalized to children, adolescents, and youth with diabetes as their outcomes and prognosis seem to be similar to their non-diabetic-peers and consistently milder than adults with diabetes.


Assuntos
COVID-19/epidemiologia , Diabetes Mellitus/epidemiologia , Hospitalização/estatística & dados numéricos , SARS-CoV-2 , Adolescente , Criança , Comorbidade , Diabetes Mellitus/terapia , Saúde Global , Humanos , Pandemias , Fatores de Risco
19.
Artigo em Inglês | MEDLINE | ID: mdl-32699114

RESUMO

INTRODUCTION: Compare cost of the interstitial liquid glucose flash monitoring (FM) system (FreeStyle Libre 2) versus self-monitoring of blood glucose (SMBG) in adults with type 1 diabetes mellitus (T1DM) in Spain. RESEARCH DESIGN AND METHODS: A model was developed to estimate, with the perspective of the Spanish health system, the annual costs associated with glucose monitoring and hypoglycemic events management in T1DM population, with multiple insulin daily doses (MDI). According to published evidence, rate of severe hypoglycemia (SHE) of 4.90 episodes per patient-year was applied. Reduction of SHE (58.6%) was modeled associated with FM use. Published rates of hospital care (20.2%) and subsequent admission (16%) were assumed for SHE. The daily consumption of strips and lancets was 9 in patients with SMBG (before and after 4 daily intakes and at bedtime) and 0.5 for FM users (according to IMPACT trial findings). Annual consumption of 26 FM sensors was considered (1 every 14 days). Unit costs (in € of 2019, excluding VAT) were obtained from literature and national databases. Sensitivity analyses (SA) were carried out to evaluate the model robustness. RESULTS: The total annual cost/patient was €4437 for SMBG and €2526 for FM. The use of FM would be associated with an annual savings in the costs of monitoring and managing hypoglycemic events of €1911 per patient-year. In a hypothetical cohort of 1000 patients with T1DM MDI, FM could avoid in 1 year 4900 SHE, 93 hospitalizations for SHE. In addition, the use of FM would generate total savings of up to €1 910 000 per year. In the SA with alternative hypoglycemia events rates and use of strips and lancets, and including non-SHE episodes, savings from €370 000 to €1 760 000 were observed with FM. CONCLUSIONS: The use of the FM system to monitor glucose in adults with T1DM treated with MDI, would reduce hypoglycemic events and would result in cost savings for the health system.


Assuntos
Diabetes Mellitus Tipo 1 , Adulto , Glicemia , Automonitorização da Glicemia , Diabetes Mellitus Tipo 1/tratamento farmacológico , Diabetes Mellitus Tipo 1/epidemiologia , Humanos , Hipoglicemiantes/uso terapêutico , Espanha/epidemiologia
20.
Endocrinol. diabetes nutr. (Ed. impr.) ; 67(6): 394-400, jun.-jul. 2020. tab
Artigo em Espanhol | IBECS | ID: ibc-193364

RESUMO

OBJETIVO: Evaluar la frecuencia de las hipoglucemias desapercibidas (HD) en pacientes con diabetes tipo 1, trasladados de pediatría, que siguen programa específico de atención y educación terapéutica (PAET) en el hospital de adultos. PACIENTES Y MÉTODOS: Jóvenes trasladados entre 2009-2011. El PAET incluyó proceso de traslado coordinado, visitas individuales y en grupo. Al inicio y a los 12 meses se valoran: HbA1c, frecuencia de hipoglucemias graves paciente/año (HG) y no graves. Los pacientes fueron clasificados y comparados en 2 grupos: hipoglucemia percibida e HD, según los resultados del Test de Clarke < 3R o > 3R respectivamente. RESULTADOS: Realizaron PAET 56 pacientes (edad 18,1±0,3 años, 46% chicas, HbA1c 8,0 ± 1,2%). En la valoración inicial el 16% presentaba HD. El número de episodios de HG fue superior en el grupo HD (0,33 ± 0,50 vs. 0,09 ± 0,28 p < 0,05). El porcentaje de pacientes con > 2 hipoglucemias no graves/semana fue superior en el grupo HD, aunque sin significación estadística (66% vs. 34%, p = 0,06). A los 12 meses todavía un 11% de pacientes presentaba HD. El número de HG siguió siendo superior en el grupo con HD (0,38 ± 1,06 vs. 0,02 ± 0,15, p = 0,04). CONCLUSIONES: El porcentaje de jóvenes con diabetes tipo 1 e HD es considerable en el momento del traslado. El PAET mejora su pronóstico, pero no lo soluciona a medio plazo. Los pacientes con HD presentan mayor frecuencia de HG. La detección de HD es necesaria para reducir las HG que todavía son una asignatura pendiente


OBJECTIVE: To evaluate frequency of hypoglycaemia unawareness (HU) in patients with type 1 diabetes (T1D) transferred from Paediatrics following a specific therapeutic education programme (TEP) in an adult hospital. PATIENTS AND METHODS: Young patients transferred from 2009-2011 were evaluated. The TEP included a coordinated transfer process, individual appointments and a group course. At baseline and at 12 months we evaluated glycated haemoglobin (HbA1c) frequency of severe (SH) hypoglycaemia/patient/year and non severe hypoglycaemia (NSH). The patients were classified into two groups and compared: hypoglycaemia awareness (HA) and HU according to the Clarke Test < 3R or > 3R respectively. RESULTS: Fifty-six patients (age 18.1 ± 0.3 years, 46% females, HbA1c 8.0 ± 1.2%) underwent the TEP. In the baseline evaluation 16% presented HU. The number of SH was higher in the HU Group (0.33 ± 0.50 vs. 0.09 ± 0.28 P < .05). The percentage of patients with > 2 NSH/week was higher, albeit not significantly, in the HU group (66% vs. 34%, p = 0.06). At 12 months 11% of the patients continued to present HU. The number of SH remained higher in the HU group (0.38 ± 1.06 vs. 0.02 ± 0.15 P = .04). CONCLUSIONS: The percentage of young people with T1D with HU is quite high at transfer. Although the TEP improves hypoglycaemia awareness it does not solve this important problem. Patients with HU more frequently present SH. It is necessary to identify HU in order to reduce SH which continues to be a problem in people with T1D


Assuntos
Humanos , Masculino , Feminino , Adolescente , Adulto Jovem , Diabetes Mellitus Tipo 1/complicações , Hipoglicemia/etiologia , Hipoglicemia/epidemiologia , Conscientização , Educação de Pacientes como Assunto , Hipoglicemia/diagnóstico , Hipoglicemia/tratamento farmacológico , Mudança das Instalações de Saúde , Estudos Retrospectivos , Índice de Massa Corporal , Desenvolvimento de Programas
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