Efficacy and safety of Choudongning Capsule in children with Tourette’s syndrome of spleen deficiency and phlegm accumulation in phase II clinic / 中草药

Objective To evaluate the efficacy and safety of Choudongning (CDN) Capsule in children with Tourette’s syndrome of spleen deficiency and phlegm accumulation. Methods A stratified-block randomized, parallel-controlled, double-blind, double- dummy, multi-center trials were designed for clinical study. A total of 240 patients were enrolled and separated randomly into experimental group and positive control group in the rate of 1:1. Patients in the experimental group orally took CDN Capsule and simulated Tiapridal Tablets; The patients in positive control group took Tiapridal Tablets and simulated CDN Capsule. Treatment course lasted for 6 weeks. The clinical efficacy and integrals of YGTSS twitch, the factor scores of motor tics and vocal tics, YGTSS social function damage, traditional Chinese medicine syndrome efficacy were observed to evaluate its security. Results The total effective rate of YGTSS twitch clinical efficacy was 71.19% in the experimental group and 67.50% in the positive control group, respectively. The results of non-inferiority test indicated that the efficacy of experimental group was not worse than that in the positive control group. There was no significant difference between two groups in YGTSS twitch integrals, motor tics and vocal tics’ factor scores, YGTSS social function damage, and traditional Chinese medicine syndrome efficacy. Only two ADRs (1.67%) in the experimental group and six ADRs (5%) in the positive control group were observed during clinical trials. The incidence of ADRs in the experimental group was lower than that in the positive control group with no statistical difference. Conclusion CDN Capsule have similar efficacy on the treatment of Tourette’s syndrome of spleen deficiency and phlegm accumulation contrast to Tiapridal Tablets, with lower adverse reactions and better safety.

Phase II Study of Irinotecan and Cisplatin Combination Chemotherapy in Metastatic, Unresectable Esophageal Cancer / Journal of the Korean Cancer Association, 대한암학회지

PURPOSE: The objective of this multicenter phase II study was to evaluate the efficacy and safety of irinotecan and cisplatin combination chemotherapy in metastatic, unresectable esophageal cancer. MATERIALS AND METHODS: Patients were treated with irinotecan 65 mg/m² and cisplatin 30 mg/m² on days 1 and 8 of each 21-day treatment cycle. The primary endpoint was response rate, and secondary endpoints were survival, duration of response, initial metabolic response rate, and toxicity. RESULTS: A total of 27 patients with squamous cell histology were enrolled in the study. The median age of the patients was 61 years. The objective response rate of the 20 patients in the perprotocol group was 30.0% (90% confidence interval [CI], 13.2 to 46.9). The median follow-up duration was 10.0 months, and the median progression-free survival and overall survival were 4.5 months (95% CI, 1.6 to 6.2) and 8.8 months (95% CI, 4.7 to 10.5), respectively. Four of 13 patients (30.8%) evaluated showed initial metabolic response. The median duration of response for partial responders was 5.0 months (range, 3.4 to 8.0 months). The following grade 3/4 treatment-related hematologic toxicities were reported: neutropenia (40.7%), anaemia (22.2%), and thrombocytopenia (7.4%). Two patients experienced febrile neutropenia. The most common grade 3/4 non-hematologic toxicities were asthenia (14.8%) and diarrhoea (11.1%). CONCLUSION: Irinotecan and cisplatin combination chemotherapy showed modest anti-tumour activity and manageable toxicity for patients with metastatic, unresectable esophageal cancer.

Treatment of chronic graft-versus-host disease: Past, present and future / 대한혈액학회지

Chronic GVHD was recognized as a complication of allogeneic hematopoietic cell transplantation more than 30 years ago, but progress has been slowed by the limited insight into the pathogenesis of the disease and the mechanisms that lead to development of immunological tolerance. Only 6 randomized phase III treatment studies have been reported. Results of retrospective studies and prospective phase II clinical trials suggested overall benefit from treatment with mycophenolate mofetil or thalidomide, but these results were not substantiated by phase III studies of initial systemic treatment for chronic GVHD. A comprehensive review of published reports showed numerous deficiencies in studies of secondary treatment for chronic GVHD. Fewer than 10% of reports documented an effort to minimize patient selection bias, used a consistent treatment regimen, or tested a formal statistical hypothesis that was based on a contemporaneous or historical benchmark. In order to enable valid comparison of the results from different studies, eligibility criteria, definitions of individual organ and overall response, and time of assessment should be standardized. Improved treatments are more likely to emerge if reviewers and journal editors hold authors to higher standards in evaluating manuscripts for publication.

Efficacy of Gemcitabine Chemotherpy in Advanced Non-small cell Lung Cancer ( NSCLC ): A Phase 2 Study / 대한암학회지

PURPOSE: To evaluate the efficacy and safety of gemcitabine, a pyrimidine antimetabolite against advanced non-small cell lung cancer (NSCLC). MATERIALS AND METHODS: Forty patients with unresectable stage IIIb to IV, pathologacally documented NSCLC were evaluated. Patients received gemcitabine 1000 mg/m, as a 30 to 60-min, intravenous infusion on days 1, 8 and 15, which was repeated every 28 days. Responses were assessed every two courses. Twenty-five to fifty percent dose reduction was permitted, ptovided that overall toxicity was severe according to World Health Organization (WHO) toxicity criteria. RESULTS: Of all 40 patients (32 men, 8 women; age range 37 to 73 years; median 63 years), 3S patients were assessable for response. 15 patients had stage IIIb disease and 25 had stage IV. Nineteen patients were histologically classified as adenocarcinoma (47.5%), 17 as squamous cell carcinoma (42.5%), 1 as large cell carcinoma (2.5%), 1 as mixed carcinoma (2.5%) and 2 as undifferentiated carcinoma (5.0%). The overall response rate was 20%. None of the patients showed complete response while 7 showed partial response (20%), 5 had stable diseases (23%) and 23 had progressive diseases (57%). During a total of 119 courses, hematologic toxicity was negligible. Granulo- cytopenia worse than WHO grade 3 occured in 11.8%, anemia in O.S% and thrombocytopenia in 0.8%, respectively. Non-hematologic toxicity was minor and easily controlled. There was no case of febrile neutropenia or treatment-related death. CONCLUSION: The single agent efficacy of gemcitabine is comparable to other agents commonly used to treat NSCLC. Gemcitabine has unusually mild side effect profile for such an active agent. This significant activity in conjunction with a very favorable toxicity profile supports further investigation in combination with other agents in patients with inoperable NSCLC.