论文部分内容阅读
AIM: To investigate efficacy and safety for granulocyte, monocyte apheresis in a population of pediatric patients with ulcerative colitis.METHODS: The ADAPT study was a prospective, openlabel, multicenter study in pediatric patients with moderate, active ulcerative colitis with pediatric ulcerative colitis activity index(PUCAI) of 35-64. Patients received one weekly apheresis with Adacolumn~ granulocyte, monocyte/macrophage adsorptive(GMA) apheresis over 5 consecutive weeks, optionally followed by up to 3 additional apheresis treatments over 3 consecutive weeks. The primary endpoint was the change in mean PUCAI between baseline and week 12; the secondary endpoint was improvement in PUCAI categorized as(Significant Improvement, PUCAI decrease of ≥ 35), Moderate Improvement(PUCAI decrease of 20 < 35), Small Improvement(PUCAI decrease of 10 < 20) or No change(PUCAI decrease of < 10). RESULTS: Twenty-five patients(mean age 13.5 years; mean weight 47.7 kg) were enrolled. In the intention-to-treat set(ITT), the mean value for PUCAI improvement was 22.3 [95%CI: 12.9-31.6; n = 21]. In the per-protocol(PP) set, the mean improvement was 36.3 [95%CI: 31.4-41.1; n = 8]. Significant Improvement was recorded for 9 out of 20 patients(45%); 5 out of 20 patients(25%) had Moderate Improvement and one patient(5%) had No Change in PUCAI score at week 12. In the PP set, six out of eight patients(75%) showed Significant Improvement; and in two out of eight patients(25%) Moderate Improvement was recorded. The endoscopic activity index(EAI) decreased by 3 points on average. Seven(7) out of 21(33%) patients in ITT and 4 out of 8(50%) patients in PP have used steroids during the clinical investigation. The mean steroid dosage for these patients in the ITT set decreased from a mean 12.4 mg to 10 mg daily on average from Baseline to week 12.CONCLUSION: Adacolumn~; GMA apheresis treatment was effective in pediatric patients with moderate active Ulcerative Colitis. No new safety signals were reported. The present data contribute to considering GMA apheresis as a therapeutic option in pediatric patients having failed first line therapy.
AIM: To investigate efficacy and safety for granulocyte, monocyte apheresis in a population of pediatric patients with ulcerative colitis. METHODS: The ADAPT study was a prospective, openlabel, multicenter study in pediatric patients with moderate, active ulcerative colitis with pediatric ulcerative colitis activity index (PUCAI) of 35-64. Patients received one weekly apheresis with Adacolumn ~ granulocyte, monocyte / macrophage adsorptive (GMA) apheresis over 5 consecutive weeks, preferably followed by up to 3 additional apheresis treatments over 3 consecutive weeks. The primary endpoint was the change in mean PUCAI between baseline and week 12; the secondary endpoint was improved in PUCAI categorized as (Significant Improvement, PUCAI decrease of ≥ 35), Moderate Improvement (PUCAI decrease of 20 <35), Small Improvement (PUCAI decrease of 10 < RESULTS: Twenty-five patients (mean age 13.5 years; mean weight 47.7 kg) were enrolled. In the intenti (PUCAI decrease of <10) The mean value for PUCAI improvement was 22.3 [95% CI: 12.9-31.6; n = 21]. In the per- protocol set of (PP) 5 out of 20 patients (25%) had Moderate Improvement and one patient (5%) had No Change in PUCAI (45%); 5 out of 20 patients score at week 12. In the PP set, six out of eight patients (75%) showed Significant Improvement; and in two out of eight patients (25%) Moderate Improvement was recorded. The endoscopic activity index (EAI) decreased by 3 points Seven (7) out of 21 (33%) patients in ITT and 4 out of 8 (50%) patients in PP have used steroids during the clinical investigation. The mean steroid dosage for these patients in the ITT set decreased from a mean 12.4 mg to 10 mg daily on average from Baseline to week 12. CONCLUSION: Adacolumn ~ ; GMA apheresis treatment was effective in pediatric patients with moderate active Ulcerative Colitis. No new safety signals were rreported. The present data contribute to considering GMA apheresis as a therapeutic option in pediatric patients having failed first line therapy.