RESUMO
INTRODUCTION: According to the Rome III Criteria, functional dyspepsia (FD) is classified as postprandial distress syndrome (PDS) and epigastric pain syndrome (EPS). On the other hand, the satiety test (ST) has been used to evaluate gastric accommodation and emptying, distinguishing healthy individuals from those with dyspepsia. AIMS: To determine whether the ST can distinguish dyspeptic individuals from healthy ones and to evaluate its usefulness in differentiating the two FD subtypes. METHODS: Adults with FD were consecutively enrolled in a cross-sectional study within the time frame of August 2011 and October 2012. Healthy subjects participated as controls. The ST consisted of the intake of a nutritional supplement (Fortisip®, Nutricia Bagó®) at a constant speed; satiety was graded at 5-minute intervals (1 to 5 points). Intake was suspended when the maximum score was reported. The total ingested volume and caloric intake was recorded and the Mann-Whitney U test was used in the statistical analysis. RESULTS: The study included 39 dyspeptic patients and 20 control individuals. The patients were predominantly women (84.6 vs. 25%; p < 0.0001) and they were similar in age (39.59 ± 13.53 vs. 34.70 ± 9.85 years) and BMI (24.32 ± 3.52 vs. 25.82 ± 3.34 kg/m2) with respect to the controls. The FD subtype percentages were PDS: 61%, EPS: 31%, and Mixed syndrome: 8%. There was a lower ingested volume and caloric intake on the part of the dyspeptic patients (185 vs. 300 ml and 277 vs. 520 Kcal, respectively. Both: P<.001). No differences in the ST were observed between the two pure dyspepsia subtypes. CONCLUSIONS: There was a difference in the ST between healthy individuals and those with dyspepsia, but the ingested volume and caloric intake in the two FD subtypes were similar.
Assuntos
Dispepsia/diagnóstico , Resposta de Saciedade/fisiologia , Adulto , Idoso , Estudos Transversais , Dispepsia/classificação , Dispepsia/fisiopatologia , Feminino , Esvaziamento Gástrico/fisiologia , Humanos , Masculino , Pessoa de Meia-Idade , Estudos Prospectivos , Saciação , Estômago/fisiopatologia , Inquéritos e Questionários , Adulto JovemRESUMO
OBJECTIVES: To investigate a 4-year prospective clinical trial of agalsidase alfa in children with Fabry disease, an X-linked metabolic disorder caused by a deficiency of the lysosomal enzyme alpha-galactosidase A. STUDY DESIGN: Seventeen (16 boys, 1 girl; age range, 7.3 to 18.4 years) of the 24 children who completed a 6-month, open-label agalsidase alfa study enrolled in a 3.5-year extension study that investigated the safety and potential efficacy of long-term treatment. All 17 patients completed the initial 6-month study, and 10 patients (9 boys) completed the extension study. RESULTS: Agalsidase alfa was well tolerated. In treated boys, there were sustained, statistically-significant improvements in the clinical features of Fabry disease, including reduced plasma globotriaosylceramide levels, reduced pain severity assessed by the Brief Pain Index, and improved heart rate variability. Mean urine globotriaosylceramide levels were reduced to normal range (P < .05 compared with baseline during 1.5 to 4 years). Kidney function and left ventricular mass indexed to height remained stable throughout. CONCLUSIONS: This clinical trial demonstrates that treatment with agalsidase alfa was well tolerated and associated with improvement of Fabry disease-related features.
Assuntos
Doença de Fabry/tratamento farmacológico , alfa-Galactosidase/uso terapêutico , Adolescente , Tamanho Corporal , Criança , Doença de Fabry/metabolismo , Doença de Fabry/fisiopatologia , Feminino , Humanos , Isoenzimas/efeitos adversos , Isoenzimas/uso terapêutico , Rim/fisiopatologia , Masculino , Medição da Dor , Proteínas Recombinantes , Suor/fisiologia , Triexosilceramidas/sangue , Triexosilceramidas/urina , Função Ventricular Esquerda , alfa-Galactosidase/efeitos adversosRESUMO
OBJECTIVE: To evaluate the safety and explore the efficacy of enzyme replacement therapy with agalsidase beta (recombinant human alpha-galactosidase A; Fabrazyme [Genzyme Corporation, Cambridge, MA]) in pediatric patients with Fabry disease, a genetic disorder in which deficient endogenous enzyme causes pathogenic tissue accumulation of globotriaosylceramide (GL-3). STUDY DESIGN: Fourteen male and 2 female patients, 8 to 16 years old, were treated in this open-label study. A 12-week observation period to collect baseline data preceded the 48-week treatment period when agalsidase beta (1 mg/kg) was infused intravenously every 2 weeks. No primary efficacy end point was specified. RESULTS: Before treatment, results of skin biopsies from 12 male patients showed moderate or severe GL-3 accumulation in superficial dermal capillary endothelial cells; with treatment, these cells were completely cleared of GL-3 in week-24 biopsies from all 12 male patients and in all available week-48 biopsies. With treatment, reports of gastrointestinal symptoms declined steadily. Patient diaries documented significant reductions in school absences due to sickness. Agalsidase beta was generally well tolerated; most treatment-related adverse events were mild or moderate infusion-associated reactions involving rigors, fever, or rhinitis. CONCLUSIONS: Agalsidase beta safely and effectively reduced the GL-3 accumulation in dermal endothelium already evident in children with Fabry disease. Early intervention may prevent irreversible end-organ damage from chronic GL-3 deposition.