ABSTRACT
Resumo O objetivo deste artigo é avaliar a prevalência e os fatores associados à adesão a terapia de reposição enzimática em adolescentes com Fibrose cística. Estudo transversal, descritivo e observacional. Foram coletados dados sociodemográficos e clínicos. Os instrumentos utilizados para avaliar adesão foram: questionário de Morisky-Green e a dispensação de medicação na farmácia e para os fatores associados, entrevista com questionário estruturado. Foram entrevistados 44 adolescentes. Segundo o método de análise de dispensação da farmácia e o questionário de Morisky-Green, encontramos uma adesão de 45,5% e 11,4%, respectivamente. A maior adesão foi observada naqueles com diagnóstico precoce e a menor nos adolescentes mais velhos e nas meninas. Os fatores com a maior prevalência de não adesão foram: não levar as enzimas ao comer fora de casa, tomar enzimas somente nas grandes refeições, função pulmonar normal, com obstrução grave e muito grave. A prevalência de adesão a enzima foi pequena. Informações relacionadas à doença e ao tratamento devem ser aprimoradas, principalmente em adolescentes mais velhos e com comprometimento da função pulmonar, com criação de estratégias e estudos longitudinais para identificar fatores que interferem na adesão.
Abstract This article sets out to evaluate the prevalence and factors associated with adherence to enzyme replacement therapy among adolescents with cystic fibrosis. It is a cross-sectional, descriptive and observational study. Sociodemographic and clinical data were collected. The instruments used to assess adherence were: the Morisky-Green questionnaire and medication dispensation at the pharmacy, and interviews with structured questionnaires for the associated factors. Forty-four adolescents were interviewed. According to the method of the pharmacy medication dispensation analysis and the Morisky-Green questionnaire, the adherence of 45.5% and 11.4% was found, respectively. The higher adherence was observed in those with early diagnosis and the lowest in older adolescents and girls. The factors with the highest prevalence of non-adherence were: not taking enzymes when eating out of the home; only taking enzymes with major meals; normal lung function; with severe and very severe obstruction. The prevalence of adhesion to enzymes was low. Information related to the disease and treatment should be improved, especially among older adolescents and with impairment of lung function, with the creation of strategies and longitudinal studies to identify factors that interfere with adherence.
Subject(s)
Humans , Male , Female , Child , Adolescent , Young Adult , Cystic Fibrosis/therapy , Medication Adherence/statistics & numerical data , Enzyme Replacement Therapy/statistics & numerical data , Cross-Sectional Studies , Surveys and Questionnaires , Cystic Fibrosis/diagnosis , Early DiagnosisABSTRACT
Gaucher disease is a multisystemic lipidosis characterized by hematologic problems, organomegally, skeletal involvement with or without neurological affection. The aim of the study is to identify the multisystemic involvements in children with Gaucher disease and to evaluate the effect of enzyme replacement therapy [ERT] on such patients. This study included 40 patients with Gaucher disease attending the Hematology Unit, Pediatric University Hospital during the period from January to December 2012. All cases were subjected to full history taking, thorough clinical examination, complete blood count, abdominal ultrasonography, radiological examination of the chest, distal end of femur and proximal end of tibia, EEC, echocardiography, Doppler echocardiography, tissue Doppler echocardiography and High Resolution C.T chest [HRCT]. Results: Significant differences were detected between cases and controls in weight, height and skull circumference [p value < 0,001]. Patients during ERT showed a significant decrease in frequency of epistaxis and blood transfusion than those before ERT [p value < 0.05]. Patients during ERT showed a significant increase in mean Hb level and platelet count than those before ERT [p value < 0.05]. There were no significant differences between the two groups of patients regarding the radiological bone changes and neurological aspects of Gaucher disease. Conclusion: Gaucher disease must be suspected in any child with organomegally, bone problems, bleeding tendency with or without neurological affection. ERT is effective in the management of hematological and visceral aspects of Gaucher disease. ERT has no role in the neurological aspect of Gaucher disease