RESUMEN
BACKGROUND: Accurately monitoring adherence to treatment with recombinant human growth hormone (r-hGH) enables appropriate intervention in cases of poor adherence. The electronic r-hGH auto-injector, easypod™, automatically records the patient's adherence to treatment. This study evaluated adherence to treatment of children who started using the auto-injector and assessed opinions about the device. METHODS: A multicentre, multinational, observational 3-month survey in which children received r-hGH as part of their normal care. Physicians reviewed the recorded dose history and children (with or without parental assistance) completed a questionnaire-based survey. Children missing ≤2 injections per month (92% of injections given) were considered adherent to treatment. Adherence was compared between GH treatment-naïve and treatment-experienced children. RESULTS: Of 834 recruited participants, 824 were evaluated. The median (range) age was 11 (1-18) years. From the recorded dose history, 87.5% of children were adherent to treatment over the 3-month period. Recorded adherence was higher in treatment-naïve (89.7%, n = 445/496) than in treatment-experienced children (81.7%, n = 152/186) [Fisher's exact test FI(X) = 7.577; p = 0.0062]. According to self-reported data, 90.2% (607/673) of children were adherent over 3 months; 51.5% (421/817) missed ≥1 injection over this period (mainly due to forgetfulness). Concordance between reported and recorded adherence was 84.3%, with a trend towards self-reported adherence being higher than recorded adherence. Most children liked the auto-injector: over 80% gave the top two responses from five options for ease of use (720/779), speed (684/805) and comfort (716/804). Although 38.5% (300/780) of children reported pain on injection, over half of children (210/363) considered the pain to be less or much less than expected. Given the choice, 91.8% (732/797) of children/parents would continue using the device. CONCLUSIONS: easypod™ provides an accurate method of monitoring adherence to treatment with r-hGH. In children who received treatment with r-hGH using easypod™, short-term adherence is good, and significantly higher in treatment-naïve children compared with experienced children. Children/parents rate the device highly. The high level of acceptability of the device is reflected by a desire to continue using it by over 90% of the children in the survey.
RESUMEN
Context: Studies of the clinical and immunological features of autoimmune Addison disease (AAD) are needed to understand the disease burden and increased mortality. Objective: To provide upgraded data on autoimmune comorbidities, replacement therapy, autoantibody profiles, and cardiovascular risk factors. Design, Setting, and Participants: A cross-sectional, population-based study that included 660 AAD patients from the Swedish Addison Registry (2008-2014). When analyzing the cardiovascular risk factors, 3594 individuals from the population-based survey in Northern Sweden, MONICA (monitoring of trends and determinants of cardiovascular disease), served as controls. Main Outcome Measures: The endpoints were the prevalence of autoimmune comorbidities and cardiovascular risk factors. Autoantibodies against 13 autoantigens were determined. Results: The proportion of 21-hydroxylase autoantibody-positive patients was 83%, and 62% of patients had ≥1 associated autoimmune diseases, more frequently coexisting in females (P < 0.0001). AAD patients had a lower body mass index (P < 0.0001) and prevalence of hypertension (P = 0.027) compared with controls. Conventional hydrocortisone tablets were used by 89% of the patients, with a mean dose of 28.1 ± 8.5 mg/d. The mean hydrocortisone equivalent dose normalized to the body surface was 14.8 ± 4.4 mg/m2/d. A greater hydrocortisone equivalent dose was associated with a greater incidence of hypertension (P = 0.046). Conclusions: Careful monitoring of AAD patients is warranted to detect associated autoimmune diseases. Contemporary Swedish AAD patients did not have an increased prevalence of overweight, hypertension, type 2 diabetes mellitus, or hyperlipidemia. However, high glucocorticoid replacement doses could be a risk factor for hypertension.
Asunto(s)
Enfermedad de Addison/inmunología , Enfermedad de Addison/complicaciones , Enfermedad de Addison/tratamiento farmacológico , Enfermedad de Addison/epidemiología , Adolescente , Adulto , Anciano , Autoanticuerpos/sangre , Enfermedades Autoinmunes/epidemiología , Enfermedades Autoinmunes/inmunología , Enfermedades Cardiovasculares/epidemiología , Enfermedades Cardiovasculares/etiología , Niño , Preescolar , Comorbilidad , Estudios Transversales , Esquema de Medicación , Femenino , Terapia de Reemplazo de Hormonas/métodos , Humanos , Hidrocortisona/administración & dosificación , Hidrocortisona/uso terapéutico , Lactante , Recién Nacido , Masculino , Persona de Mediana Edad , Sistema de Registros , Factores de Riesgo , Suecia/epidemiología , Adulto JovenRESUMEN
Autoimmune polyglandular syndrome type I (APS I) is an autosomal recessive disorder characterized by a combination of autoimmune manifestations affecting endocrine and non-endocrine organs. APS I usually presents in childhood. The three most common manifestations are chronic mucocutaneous candidiasis, hypoparathyroidism and Addison's disease. At least two of these must be present to fulfill the diagnostic criteria of this syndrome. The spectrum of other associated diseases includes gonadal insufficiency, alopecia, vitiligo and chronic active hepatitis. APS I is caused by a mutation in the AIRE-gene (autoimmune regulator) located on chromosome 21. Analysis of specific autoantibodies against intracellular enzymes, particularly enzymes in the synthesis of steroids and neurotransmittors, can be used in the diagnosis of APS I and to predict different manifestations of the disease.
Asunto(s)
Poliendocrinopatías Autoinmunes , Enfermedad de Addison/genética , Enfermedad de Addison/inmunología , Insuficiencia Suprarrenal/genética , Insuficiencia Suprarrenal/inmunología , Adulto , Alopecia/genética , Alopecia/inmunología , Alopecia/patología , Autoanticuerpos/genética , Candidiasis Mucocutánea Crónica/genética , Candidiasis Mucocutánea Crónica/inmunología , Niño , Cromosomas Humanos Par 21/genética , Diabetes Mellitus Tipo 1/genética , Diabetes Mellitus Tipo 1/inmunología , Hepatitis Autoinmune/genética , Hepatitis Autoinmune/inmunología , Humanos , Hipoparatiroidismo/genética , Hipoparatiroidismo/inmunología , Síndromes de Malabsorción/genética , Síndromes de Malabsorción/inmunología , Poliendocrinopatías Autoinmunes/genética , Poliendocrinopatías Autoinmunes/inmunología , Poliendocrinopatías Autoinmunes/patología , Vitíligo/genética , Vitíligo/inmunologíaAsunto(s)
Transexualidad , Adolescente , Edad de Inicio , Trastorno del Espectro Autista/epidemiología , Niño , Comorbilidad , Trastornos del Desarrollo Sexual/diagnóstico , Trastornos del Desarrollo Sexual/epidemiología , Trastornos del Desarrollo Sexual/psicología , Trastornos de Alimentación y de la Ingestión de Alimentos/epidemiología , Disforia de Género/diagnóstico , Disforia de Género/epidemiología , Disforia de Género/psicología , Humanos , Comunicación Interdisciplinaria , Legislación Médica , Grupo de Atención al Paciente , Cirugía de Reasignación de Sexo/psicología , Suecia , Transexualidad/diagnóstico , Transexualidad/epidemiología , Transexualidad/psicología , Adulto JovenAsunto(s)
Disforia de Género , Transexualidad , Adolescente , Niño , Preservación de la Fertilidad , Disforia de Género/diagnóstico , Disforia de Género/tratamiento farmacológico , Disforia de Género/metabolismo , Disforia de Género/psicología , Hormona Liberadora de Gonadotropina/efectos adversos , Hormona Liberadora de Gonadotropina/uso terapéutico , Humanos , Salud Mental , Padres/psicología , Pubertad/metabolismo , Calidad de Vida , Transexualidad/diagnóstico , Transexualidad/tratamiento farmacológico , Transexualidad/metabolismo , Transexualidad/psicología , Adulto JovenRESUMEN
AIM: To examine perceived height during the first 24 months of growth hormone (GH) treatment in short prepubertal children. METHODS: Ninety-nine 3- to 11-year-old short prepubertal children with either isolated GH deficiency (n = 32) or idiopathic short stature (n = 67) participated in a 24-month randomized trial of individualized or fixed-dose GH treatment. Children's and parents' responses to three perceived height measures: relative height (Silhouette Apperception Test), sense of height (VAS short/tall), and judgment of appropriate height (yes/no) were compared to measured height. RESULTS: Children and parents overestimated height at start (72%, 54%) and at 24 months (52%, 30%). Short children described themselves as tall until 8.2 years (girls) and 9 years (boys). Prior to treatment, 38% of children described their height as appropriate and at 3 months, 63%. Mother's height, parental sense of the child's tallness and age explained more variance in children's sense of tallness (34%) than measured height (0%). CONCLUSION: Short children and parents overestimate height; a pivotal age exists for comparative height judgments. Even a small gain in height may be enough for the child to feel an appropriate age-related height has been reached and to no longer feel short.