| 1 | Screening of Fabry disease in patients with end-stage renal disease of unknown etiology:the first Thailand study显示文摘We aimed to explore the prevalence of Fabry disease in Thai patients who were diagnosed with end-stage renal disease(ESRD) of an unknown origin.Venous blood samples were collected from ESRD patients for biochemical and molecular studies.Alpha-galactosidase A(a-GAL A) screening was performed from dried-blood spots using fluorometry.Molecular confirmation was performed using DNA sequencing of the GLA gene.A total of 142 male and female patients were included in this study.Ten patients(7.04%) exhibited a significant decrease in a-GAL A activity.There were no definitive pathogenic mutations observed in the molecular study.However,four patients revealed a novel nucleotide variant at c.l-10 C>T,which was identified as a benign variant following screening in the normal population.In conclusion,the a-GAL A assay utilizing dried-blood spots revealed a significant false positive rate.There was no definitive Fabry disease confirmed in Thai patients diagnosed with ESRD of unknown etiology. | Objoon Trachoo Paisan Jittorntam Sarunpong Pibalyart Saowanee Kajanachumphol Norasak Suvachittanont Suthep Patputthipong Piyatida Chuengsaman Arkom Nongnuch | 2017 | The Journal of Biomedical Research2017，31，1： | 1 |
| 2 | Cetuximab plus irinotecan in pretreated metastatic colorectal cancer patients:The ELSIE study显示文摘AIM:To evaluate the efficacy and safety of cetuxim-ab plus irinotecan in irinotecan-refractory metastatic colorectal cancer (mCRC) patients from South-East Asia and Australia. METHODS:In this open-label,phase Ⅱ study,the main eligibility criteria were epidermal growth factor receptor-positive mCRC with progressive disease within 3 mo of an irinotecan-based regimen as the most recent chemotherapy. Patients received cetuximab 400 mg/m2 initially,then 250 mg/m2 every week,with the same regimen of irinotecan on which the patients had progressed (4 pre-defined regim-ens allowed). The prim-ary objective was evaluation of progression-free survival (PFS) at 12 wk. Secondary objectives included a further investigation of PFS,and an assessment of the overall response rate (ORR),duration of response,time to treatment failure (TTF),overall survival and the safety profile. RESULTS:One hundred and twenty nine patients were enrolled from-25 centers in the Asia-Pacific region and of these 123 received cetuximab plus irinotecan. The most common recent irinotecan regimen used was 180 mg/m2 every 2 wk which had been used in 93 patients (75.6%). The PFS rate at 12 wk was 50% (95% confidence interval (CI,41-59) and m-edian PFS tim-e was 12.1 wk (95% CI:9.7-17.7). The ORR was 13.8% (95% CI:8.3-21.2) and disease control rate was 49.6% (95% CI:40.5-58.8). Median duration of response was 31.1 wk (95% CI:18.0-42.6) and median overall survival was 9.5 mo (95% CI,7.5-11.7). The median TTF was 11.7 wk (95% CI:9.1-17.4). Treatment was generally well tolerated. The most common grade 3/4 adverse events were diarrhea (13.8%),neutropenia (8.9%),rash (5.7%) and vomiting (5.7%).CONCLUSION:In patients from Asia and Australia,this study confirm-s the activity and safety of cetuxim-ab plus irinotecan observed in previous studies in Europe and South America. | Robert Lim Yan Sun Seock-Ah Im Ruey-Kuen Hsieh Tsz Kok Yau Anthony Bonaventura Arkom Cheirsilpa Regina Esser Matthias Mueser Suresh Advani | 2011 | World Journal of Gastroenterology2011，17，14： | 1 |
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