Eun Mi Yang1,2, Sang Taek Lee3, Hyun Jin Choi2, Hee Yeon Cho3, Joo Hoon Lee4, Hee Gyung Kang5,6, Young Seo Park4,7, Hae Il Cheong8, Il-Soo Ha2,8. 1. Department of Pediatrics, Chonnam National University Hospital, Gawngju, Korea. 2. Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Korea. 3. Department of Pediatrics, Samsung Medical Center, Sungkynkwan University School of Medicine, Seoul, Korea. 4. Department of Pediatrics, Asan Medical Center Children's Hospital, University of Ulsan College of Medicine, Seoul, Korea. 5. Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Korea. pedneph@snuh.org. 6. Research Coordination Center for Rare Diseases, Seoul National University Hospital, Seoul, Korea. pedneph@snuh.org. 7. Research Coordination Center for Rare Diseases, Seoul National University Hospital, Seoul, Korea. 8. Kidney Research Institute, Medical Research Center, Seoul National University College of Medicine, Seoul, Korea.
Abstract
BACKGROUND: Cyclosporine A and tacrolimus (TAC) are often used as a second-line treatment for children with refractory nephrotic syndrome (NS). This study was undertaken to investigate the efficacy and safety of Tacrobell®, a locally produced generic form of TAC. METHODS: This study was a one-year prospective, open-label, single-arm, multicenter trial. Fourty-four children with steroid-dependent NS (SDNS) and 33 children with steroid-resistant NS (SRNS) were enrolled. The primary endpoints were defined as the remission rates, whereas the secondary endpoints were recognized as the duration of remission and adverse effects of TAC. RESULTS: After one-year treatment, 34 (77.3%) of the 44 patients with SDNS were in complete remission, and 6 (13.6%) were in partial remission. Nineteen (43.2%) patients did not relapse during the study; for those who did relapse, the mean duration of remission was 4.6±2.9 months. The number of relapse episodes during the study period (0.90 per patient-year) was significantly lower than that in the preceding year (2.8 per patient-year). After treatment for 3 and 6 months, 12 (36.4%) of the 33 patients with SRNS were in remission, and after treatment for 12 months, the number of patients had increased to 13 (39.4%). The mean time to achieve remission was 4.0±3.2 months. After remission (duration, 3.7±2.7 months), 12 (54.5%) of 22 patients relapsed. The fasting blood glucose and blood pressure levels during the therapy were similar to those at the time of study entry. CONCLUSIONS: Treatment with Tacrobell® was effective and safe for children with refractory NS. The efficacy of this generic form of TAC was better than that of the original TAC formula.
BACKGROUND:Cyclosporine A and tacrolimus (TAC) are often used as a second-line treatment for children with refractory nephrotic syndrome (NS). This study was undertaken to investigate the efficacy and safety of Tacrobell®, a locally produced generic form of TAC. METHODS: This study was a one-year prospective, open-label, single-arm, multicenter trial. Fourty-four children with steroid-dependent NS (SDNS) and 33 children with steroid-resistant NS (SRNS) were enrolled. The primary endpoints were defined as the remission rates, whereas the secondary endpoints were recognized as the duration of remission and adverse effects of TAC. RESULTS: After one-year treatment, 34 (77.3%) of the 44 patients with SDNS were in complete remission, and 6 (13.6%) were in partial remission. Nineteen (43.2%) patients did not relapse during the study; for those who did relapse, the mean duration of remission was 4.6±2.9 months. The number of relapse episodes during the study period (0.90 per patient-year) was significantly lower than that in the preceding year (2.8 per patient-year). After treatment for 3 and 6 months, 12 (36.4%) of the 33 patients with SRNS were in remission, and after treatment for 12 months, the number of patients had increased to 13 (39.4%). The mean time to achieve remission was 4.0±3.2 months. After remission (duration, 3.7±2.7 months), 12 (54.5%) of 22 patients relapsed. The fasting blood glucose and blood pressure levels during the therapy were similar to those at the time of study entry. CONCLUSIONS: Treatment with Tacrobell® was effective and safe for children with refractory NS. The efficacy of this generic form of TAC was better than that of the original TAC formula.
Authors: K Falkiewicz; D Kamińska; W Nahaczewska; M Boratyńska; H Owczarek; M Klinger; M Woźniak; D Patrzałek; P Szyber Journal: Transplant Proc Date: 2006 Jan-Feb Impact factor: 1.066