Minimal Symptom Expression' in Patients With Acetylcholine Receptor Antibody-Positive Refractory Generalized Myasthenia Gravis Treated With Eculizumab

The efficacy and tolerability of eculizumab were assessed in REGAIN, a 26-week, phase 3, randomized, double-blind, placebo-controlled study in anti-acetylcholine receptor antibody-positive (AChR+) refractory generalized myasthenia gravis (gMG), and its open-label extension

Post-intervention Status in Patients With Refractory Myasthenia Gravis Treated With Eculizumab During REGAIN and Its Open-Label Extension

OBJECTIVE: To evaluate whether eculizumab helps patients with anti-acetylcholine receptor-positive (AChR+) refractory generalized myasthenia gravis (gMG) achieve the Myasthenia Gravis Foundation of America (MGFA) post-intervention status of minimal manifestations (MM), we assessed patients' status throughout REGAIN (Safety and Efficacy of Eculizumab in AChR+ Refractory Generalized Myasthenia Gravis) and its open-label extension. METHODS: Patients who completed the REGAIN randomized controlled trial and continued into the open-label extension were included in this tertiary endpoint analysis. Patients were assessed for the MGFA post-intervention status of improved, unchanged, worse, MM, and pharmacologic remission at defined time points during REGAIN and through week 130 of the open-label study. RESULTS: A total of 117 patients completed REGAIN and continued into the open-label study (eculizumab/eculizumab: 56; placebo/eculizumab: 61). At week 26 of REGAIN, more eculizumab-treated patients than placebo-treated patients achieved a status of improved (60.7% vs 41.7%) or MM (25.0% vs 13.3%; common OR: 2.3; 95% CI: 1.1-4.5). After 130 weeks of eculizumab treatment, 88.0% of patients achieved improved status and 57.3% of patients achieved MM status. The safety profile of eculizumab was consistent with its known profile and no new safety signals were detected. CONCLUSION: Eculizumab led to rapid and sustained achievement of MM in patients with AChR+ refractory gMG. These findings support the use of eculizumab in this previously difficult-to-treat patient population. CLINICALTRIALSGOV IDENTIFIER: REGAIN, NCT01997229; REGAIN open-label extension, NCT02301624. CLASSIFICATION OF EVIDENCE: This study provides Class II evidence that, after 26 weeks of eculizumab treatment, 25.0% of adults with AChR+ refractory gMG achieved MM, compared with 13.3% who received placebo

Frequência de ocorrência de carcinoma basocelular palpebral na região Centro-Oeste paulista e características dos portadores

OBJETIVO: Observar a frequência de ocorrência do carcinoma basocelular palpebral na região Centro-Oeste paulista e descrever o perfil demográfico dos portadores. MÉTODOS: Estudo transversal, de amostragem aleatória, realizado em doze cidades da região Centro-Oeste do Estado de São Paulo. Foram examinados 11.167 indivíduos. Os pacientes foram avaliados em uma Unidade Móvel, com atendimento oftalmológico completo. O diagnóstico de carcinoma basocelular palpebral foi feito através do exame clínico e biomicroscópico da lesão. Os pacientes diagnosticados foram encaminhados ao Ambulatório de Plástica Ocular da Faculdade de Medicina de Botucatu para tratamento. Os dados foram submetidos à análise de frequência de ocorrência. RESULTADOS: Cinco casos de carcinoma basocelular palpebral foram identificados na amostra, correspondendo a uma frequência de ocorrência de 0,045%. Quatro pacientes eram do sexo feminino, a maioria com idade maior ou igual a 70 anos e todos os casos tinham a cor da pele branca. Apenas três indivíduos encaminhados compareceram ao serviço para exérese da lesão e confirmação diagnóstica. CONCLUSÃO: O carcinoma basocelular palpebral acomete 0,045% dos habitantes da região Centro-Oeste paulista, afetando principalmente o sexo feminino, na faixa etária próxima dos 70 anos.PURPOSE: To observe the frequency of occurrence of eyelid basal cell carcinoma in the centralwest region of São Paulo State and to describe the demographic profile of the basal cell carcinoma carriers. METHODS: Transversal study, using a random sampling, carried out in 12 cities in the centralwest region of São Paulo State evolving 11,167 individuals. Patients were evaluated in a Mobile Unit, with complete ophthalmologic evaluation. The diagnosis of eyelid basal cell carcinoma was done through clinical examination and biomicroscopy of the lesion if desirable. The basal cell carcinoma carriers diagnosed were referred to Oculoplastic Clinic of Faculdade de Medicina de Botucatu for treatment. Data were submitted to analysis of frequency of occurrence. RESULTS: Five cases of eyelid basal cell carcinoma were identified in the sample, corresponding to a frequency of occurrence of 0.045%. Four patients were female, most with age equal or greater than 70 year-old and all the cases had white skin color. Only three individuals conveyed attended the service for excision of the lesion and diagnostic confirmation. CONCLUSION: The eyelid basal cell carcinoma affects 0.045% of the inhabitants of the centralwest region of São Paulo State, affecting mainly the 70 year-old female range.Fundação de Amparo à Pesquisa do Estado de São Paulo (FAPESP

Enxerto dermoadiposo em cavidades anoftálmicas secundárias - estudo retrospectivo e revisão da literatura

Purpose: To evaluate the dermofat graft in the anophthalmic socket treatment. Methods: A retrospective study including 28 anophthalmic cavity carriers treated with dermofat graft was carried out. The patients were analyzed according to age, sex, socket contraction degree, first surgery performed, reason for graft choice, procedure's results and complications. The data were analyzed according to the frequency of occurrence. Results: Nineteen (67.8%) of the 28 analyzed patients were male. According to age the majority of patients were between 40 to 60 years old (13 patients - 46.4%) and the most common cause of eyeball loss was trauma (seven cases - 25%). Anophthalmic socket type III was the most frequently observed (32.1%). The evisceration was the first procedure in 13 cases, enucleation in 11 and skin graft was done in two patients. Two patients came to the service after evisceration and did not provide any information about the first surgery. The choice to use the dermofat graft was to replace volume at the socket of 12 patients (42.8%). Most of the patients (53.6%) had no complications and graft necrosis was the problem most frequently observed (32.1%). The surgery was considered good in 71.4% and not good in only 7.1%. Conclusion: the dermofat graft is an efficient and easy technique to reconstruct the anophthalmic cavity. The authors suggest this procedure must be introduced in the medical training services since it provide good results

Carcinoma de Meibômio como segundo tumor em portadores de retinobastoma: relato de dois casos

O retinoblastoma pode acometer um ou os dois olhos, ocorrendo de forma hereditária ou esporádica. O portador dessa doença pode desenvolver, a longo prazo, outros tipos de tumores não oculares. Relatamos dois pacientes: o filho, portador de cavidade anoftálmica bilateral após remoção dos olhos devido ao retinoblastoma, que apresentou segundo tumor palpebral unilateral, afetando a pálpebra superior; e o pai, portador de cavidade anoftálmica unilateral também por remoção de retinoblastoma e que apresentou tumoração de crescimento rápido na pálpebra superior ipsilateral ao olho enucleado. O exame imuno-histoquímico de ambas as lesões excisadas revelou que se tratava de carcinoma de glândulas sebáceas. Os pacientes evoluíram bem após a remoção do tumor palpebral e, até o momento, não apresentam sinais de recidiva ou metástase