In order to better incorporate patient input in clinical trials, the US Food and Drug Administration has included "patient-focused drug development" in the selection and development of clinical outcome assessments, and formulated a series of guidelines. Based on the third guiding principle, "Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments", this article summarizes the clinical outcome assessments from five aspects: concept, development process, scoring mechanism, interference factors and sensitivity, and introduces four different types of clinical outcome assessments, providing new ideas for "patient-focused drug development" efficacy evaluation in clinical trials.
Objective To investigate the effects of component blood transfusion combined with heparin therapy on coagulation function and clinical outcomes in pregnant women with acute disseminated intravascular coagulation (DIC). Methods A retrospective analysis was conducted on the clinical data of 65 pregnant women with acute DIC who were treated in Obstetrics Department of Luzhou People’ s Hospital between March 2020 and March 2022. Pregnant women treated with component blood transfusion were included in the control group, while those treated with component blood transfusion combined with heparin were included in the observation group. Before and after treatment, the DIC scoring system was used for score evaluation. Coagulation function indicators and routine blood indicators were compared between the two groups of pregnant women. Adverse clinical outcomes and adverse reactions were observed in both groups of pregnant women. Results The study enrolled 65 pregnant women, comprising 30 in the observation group and 35 in the control group. Before treatment, there was no statistical difference in DIC score, coagulation function indicators, or routine blood indicators between the two groups (P>0.05). After treatment, the DIC score, prothrombin time, activated partial thromboplastin time, thrombin time, and D-dimer significantly decreased in both groups (P<0.05), and the above indicators in the observation group [3.39±0.48, (13.28±2.28) s, (24.68±2.06) s, (14.27±1.82) s, and (2.23±0.88) mg/L, respectively] were lower than those in the control group [4.11±1.56, (15.02±2.45) s, (26.79±3.18) s, (15.61±1.91) s, and (2.87±0.74) mg/L, respectively] (P<0.05). The levels of fibrinogen, platelet count, hemoglobin, and hematocrit significantly increased in both groups (P<0.05), and the levels in the observation group [(4.29±1.05) g/L, (175.36±20.46)×109/L, (84.09±7.27) g/L, and (25.49±3.13)%, respectively] were higher than those in the control group [(3.44±1.27) g/L, (145.77±21.12)×109/L, (76.58±7.13) g/L, and (23.03±3.05)%, respectively] (P<0.05). The observation group had a lower incidence rate of adverse clinical outcomes compared to the control group (33.3% vs. 74.3%, P<0.05). The incidence rates of adverse reactions were not statistically different between the two groups (P>0.05). Conclusions Component blood transfusion combined with heparin therapy for pregnant women with acute DIC can effectively improve their coagulation function, reduce the risk of bleeding, and further improve adverse clinical outcomes such as postpartum hemorrhage and hysterectomy. Additionally, this treatment approach demonstrates a high safety profile.
ObjectiveTo evaluate the impact of using alanyl-glutamine dipeptide on clinical outcome for gastric cancer patients with nutritional risk after total gastrectomy. MethodsThis study was carried out in the period from March to August 2015. The nutritional risk was screened by continuous sampling method in the new hospitalized patients with gastric cancer who would undergo total gastrectomy. The patients were grouped randomly. Alanyl-glutamine was given to the experimental group patients. The clinical data of the two groups were analyzed, such as the laboratory parame-ters of nutritional status and hepatorenal function, complications of surgery, the nutrition-related hospitalization day, etc. ResultsThe preoperative data were consistent in the two groups of the included 40 cases. The results showed, in the third and seventh days after surgery, the level of plasma albumin was higher in the experimental group than in the control group〔(33.9±5.6) g/L vs. (30.8±4.0) g/L and (36.6±3.9) g/L vs. (33.9±4.2) g/L, respectively). Also, the CD4+/CD8+ cells immune index was significantly improved in the experimental group after surgery (1.7±0.7 vs. 1.2±0.3, P < 0.05). The recovery time of intestinal function〔(65.7±5.3) h vs. (71.6±7.2)h, P < 0.01)〕and nutrition-related hospitalization day〔(10.1±1.8) d vs. (11.7±1.9)d, P < 0.01)〕in alanyl-glutamine dipeptide group were shorted than that in the control group. No serious adverse drug reactions were found in the patients during the treatment period. ConclusionApplication alanyl-glutamine to the patients with nutritional risk after total gastrectomy could partly improve clinical outcome indicators.
Objective To investigate the scientificity of patient-reported outcomes instrument for asthma ( Asthma-PRO) , which maybe used to evaluate the efficacy of anti-asthma drugs in clinical trials and clinical practice.Methods 366 asthma patients and 100 healthy subjects were face-to-face interviewed by well-trained investigators, and the data of Asthma-PRO instrument were collected. The psychometric performance such as reliability, validity, responsiveness and clinical feasibility in the Asthma-PRO instrument was evaluated. Results The split-half reliabilities of the Asthma-PRO instrument and each dimension were greater than 0.8. In the analysis of internal consistency of each dimension, the cronbach’s alpha coefficient was greater than 0.7. Factor analysis showed that the instrument has good construct validity. The scores of each of the facets and total scores between the asthma patients and the healthy subjects were different. The recovery rate and the efficient rate of the questionnaire were more than 95%, and the time required to complete a questionnaire was within 20 minutes, indicating that the scale had a high clinical feasibility. Conclusion The Asthma-PRO instrument has good reliability, validity, responsiveness and clinical feasibility.
Objective To summarize the application of enteral nutrition support in hospitalized patients. Methods The related literatures about enteral nutrition support in recent years were reviewed. Results The rates of malnutrition and nutritional risk were still high in patients. Enteral nutrition support could improve the condition and result of these cases in better clinical outcome, such as shorten hospitalization time and reduced the cost of hospital expenditure. Enteral nutritional support included both oral supplementation and tube-feeding techniques. Artificial nutrition may be provided by nasal tube (nasogastric or nasojejunal tube) or surgically placed tube (gastrostomy, jejunostomy, percutaneous endoscopic gastrostomy or percutaneous endoscopic gastrostomy-jejunostomy). More attention should be paid to the issues relating to feeding intolerance, including abdominal distension, diarrhea, reflux, and aspiration, especially for postoperative patients with early enteral nutrition support. Conclusion Enteral nutrition support requires highly individually and specialized tailored management.
To ensure the scientific and recognized evaluation of patient-centered clinical research outcomes, the drug development tool qualification certification website of the Center for Drug Evaluation and Research and the Center for Biologics Evaluation and Research have disclosed the information of clinical outcome assessment (COA) submitted for certification, and encouraged the use of certified COA. This paper searched all COA and approval decisions on the website, analyzed the submitted information and certification status of COA, and interpreted the certification technology and process of COA, so as to provide references and suggestions for the development of patient-centered clinical research outcome evaluation.
ObjectiveTo investigate the prevalence of the nutritional risks, the relationship between application of nutritional support and the clinical outcome of patients with gastrointestinal major surgery in the Frist People's Hospital of Shuangliu. MethodsGastrointestinal major surgery patients in Department of General Surgery in the Frist People's Hospital of Shuangliu from March 2010 to March 2014 were consecutively enrolled. Patients who provided informed consent were screened by NRS 2002, tracking nutrition support status and analysis the relationship between nutrition support and clinical outcome. In this study, the clinical outcome index included postoperative complications and hospitalization time. ResultsThere were totally 130 cases enrolled, 112 cases completed assessment by NRS 2002. The prevalence of nutritional risk was 75.9%(85/112), there were totally 57 patients(50.9%) received nutrition support, and all for parenteral nutrition. The prevalence of postoperative complication was 46.4%(52/112). The prevalence of postoperative complication in patients who had nutritional risk and received nutritional support was 41.7%(15/36), whereas, in patients who had nutritional risk but not received nutritional support was 73.5%(36/49), there was statistically significant difference between the 2 groups(P=0.002). In patients who not had nutritional risk, the postoperative complication rate was only 3.7%(1/27). ConclusionsBecause of noninvasive and easy to operate, NRS 2002 are adpted to hospitalized patients with gastrointestinal major surgery. Because of the specific of disease metabolism, the higher nutritional risk occurres in patients with gastrointestinal major surgery, appropriate nutritional support for this kinds of patients can reduce the incidence of postoperative complication, and improve the prognosis.
ObjectiveTo evaluate the reliability,validity and feasibility of a patient-reported outcomes (PRO) scale in the subjects with respiratory failure. Methods364 patients with chronic respiratory failure and 97 healthy subjects were face-to-face interviewed by well-trained investigators,and the data of respiratory failure -PRO instrument were collected. The psychometric performance such as reliability,validity,responsiveness and clinical feasibility in the respiratory failure -PRO instrument was evaluated. ResultsThe Cronbach's alpha coefficient of the respiratory failure -PRO instrument and each dimension were greater than 0.7. Factor analysis showed that the instrument had good construct validity. The scores of each of the facets and total scores between the patients and the healthy subjects were different. The recovery rate and the efficient rate of the questionnaire were more than 95%,and the time required to complete a questionnaire was within 15 minutes,indicating that the scale had a high clinical feasibility. ConclusionThe respiratory failure -PRO instrument has good reliability,validity,responsiveness and clinical feasibility.
【摘要】 目的 观察提前去除卵丘细胞对胚胎质量的影响，进一步探讨其临床应用价值。 方法 将2009年7-9月行体外受精-胚胎移植治疗的患者随机分为两组，试验组共137个周期，于授精后5 h去除卵丘细胞；对照组共146个周期，于授精后16～18 h去除卵丘细胞。两组均于授精后16～18 h观察受精情况，72 h对胚胎进行评分，选择1～3枚优质胚胎进行胚胎移植。分别比较两组的年龄、不育年限、获卵数、受精率、卵裂率、优质胚胎率、胚胎种植率和临床妊娠率。 结果 两组的优质胚胎率差异有统计学意义（Plt;0.01）；但年龄、不育年限、获卵数、受精率、卵裂率、胚胎种植率和临床妊娠率比较，差异均无统计学意义（Pgt;0.05）。 结论 体外受精中提前去除卵丘细胞优质胚胎增加，有助于提高临床累积妊娠率。【Abstract】 Objective To evaluate the effect of denuding the oocyte granulose cells soon after fertilization in vitro (IVF). Methods A total of 283 IVF cycles in patients from July to September 2009 were divided into a trial group and a control group randomly. The trial group contained 137 cycles, being denuded the oocyte granulose cells only 5 hours after IVF. The controlled group contained 146 cycles, being denuded the oocyte granulose cell and observed the pronuclear 18 hours after IVF. The outcomes of female age, infertile duration, the number of oocyte, fertilization rate,abnormal fertilization rate, good quality embryo rate, and clinical pregnancy rate were measured. Results There was no difference in any of the outcomes between the two groups except for the high quality embryo rate (Plt;0.01). The clinical pregnancy rate was higher in the trial group without significant differences (Pgt;0.05). Conclusions Denuding the oocyte granulose cells soon after IVF helps to improve the embryo quality and the clinical accumulated pregnancy rate.