ABSTRACT
Objective: To characterize adherence to Phenylketonuria (PKU) management practices among PKU patients treated at reference sites around Argentina, Brazil, and Mexico. Methods: This is a retrospective, observational, multicenter, and multinational survey-based study using aggregate data. From an initial list of 40 sites, 22 clinicians expressed interest in completing the survey, with 20 clinicians from 20 unique sites fulfilling all the study criteria. The Survey contained 28 questions, including respondent's clinic characteristics, clinic PKU treatment recommendations, and patient adherence to clinic recommendations. Survey was available in local languages, and the respondents were asked to consult their clinic records to complete their responses. Adherence was assessed by target blood phenylalanine (Phe), target blood testing frequency, and clinic visits. Results: A total of 1077 (out of 1377) actively managed PKU patients (seen in the clinic in the last 3 years) from 13 clinics in Brazil, six in Argentina, and one in Mexico were analyzed. Upper blood Phe target was set over 360 µMol/L in 70% of the clinics for adult patients. Around 40% of the patients >30 years old had Phe blood tests done twice a year or less, with 60% of the clinics recommending semestral visits for adults <30 years old. Twice a month was the most common frequency of visits for <1 year old. The COVID-19 pandemic was a disruptor for frequency of visits and exams. Conclusions: These results show that there is still room for improvement in terms of adherence, namely in adults and older children. More efforts must be made to educate patients and healthcare professionals about the importance of treatment adherence, accompanied by public policies that expand access to pharmacological and dietary treatment with diversity and quality to improve adherence to adequate blood Phe levels.
ABSTRACT
OBJECTIVE:: to assess the severity of obesity in children and adolescents through the presence of comorbidities and the potential indication of bariatric surgery. METHODS:: we conducted a cross-sectional study with clinical and laboratory data of the first consultation of patients at the childhood obesity clinic at a tertiary hospital from 2005 to 2013. We divided the patients into groups with or without potential indication for surgery, and recorded age, gender, birth weight, age of obesity onset, BMI Z score, presence of acanthosis nigricans, blood pressure, total cholesterol and fractions, triglycerides, blood glucose and fasting insulin, HOMA1-IR, CRP and ESR. The group with potential indication for surgery included: BMI > 40 or between 35-40 with comorbidities (Triglycerides >130mg/dl, glucose levels >100mg/dl, HOMA1-IR >3.16, Total Cholesterol >200mg/dl, LDL >130mg/dl and HDL <45mg/dl), regardless of age, epiphysis consolidation and previous treatment. RESULTS:: of the 296 patients included in the study, 282 (95.3%) were younger than 16 years. The most frequent change was the HDL (63.2%), followed by HOMA1-IR (37.5%). Of the group of 66 patients with potential indication for surgery (22.3%), only ten (15.1%) had more than 16 years. Acanthosis nigricans, the average HOMA1-IR, insulin, CRP, ESR, age, BMI Z score and systolic and diastolic blood pressure were significant in the group with potential surgical indication. CONCLUSION:: bariatric surgery might be indicated by BMI and comorbidities in children and adolescents under 16 years. OBJETIVO:: avaliar a gravidade da obesidade em crianças e adolescentes pela presença de comorbidades e pela potencial indicação de cirurgia bariátrica. MÉTODOS:: estudo transversal com dados clínicos e laboratoriais da primeira consulta de pacientes do ambulatório de obesidade infantil em um hospital terciário no período de 2005 a 2013. Os pacientes foram divididos em grupos com ou sem potencial indicação cirúrgica, e associados com idade, sexo, peso de nascimento, idade de início da obesidade, escore z de IMC, presença de acantose nigricans, pressão arterial, colesterol total e frações, triglicérides, glicemia e insulina de jejum, HOMA1-IR, PCR e VHS. O grupo com potencial indicação cirúrgica incluiu: IMC >40 ou IMC entre 35-40 com comorbidades (Triglicérides >130mg/dl, Glicemia >100mg/dl, HOMA1-IR >3,16, Colesterol total >200mg/dl, LDL >130mg/dl e HDL <45mg/dl), independente da idade, consolidação das epífises e tratamento prévio. RESULTADOS:: de 296 pacientes incluídos no estudo, 282 (95,3%) tinham menos de 16 anos. A alteração mais frequente foi a do HDL (63,2%), seguido do HOMA1-IR (37,5%). Do grupo de 66 pacientes com potencial indicação cirúrgica (22,3%), apenas dez (15,1%) tinham mais de 16 anos. Acantose nigricans, as médias de HOMA1-IR, insulina, PCR, VHS, idade, escore z de IMC e pressões sistólica e diastólica foram significantes no grupo com potencial indicação cirúrgica. CONCLUSÃO:: os resultados sugerem que a cirurgia bariátrica, poderia estar indicada pelo IMC e presença de comorbidades, em crianças e adolescentes com menos de 16 anos.
