Use In Chinese Patients Research Articles

Efficacy and Safety of Asciminib in Chinese Patients with Philadelphia Chromosome(Ph)-Positive Leukaemias

Background Asciminib is a first-in-class STAMP (Specifically Targeting the ABL Myristoyl Pocket) tyrosine kinase inhibitor (TKI). Its efficacy and safety profile in patients with chronic-phase chronic myeloid leukaemia (CML) have been shown in the Phase 3 ASCEMBL study, comparing asciminib 40 mg twice daily versus bosutinib 500 mg once daily. However, asciminib use in Chinese patients has hitherto not been reported. Moreover, the safety of high-dose asciminib (≥150 mg twice daily) in the Asian population is yet to be addressed. Here we present the real-world clinical outcomes of Chinese patients with Ph+ leukaemias in Hong Kong who received asciminib through a Managed Access Program (MAP). Methods Queen Mary Hospital (QMH) is a university-affiliated tertiary hospital in Hong Kong. Patients with Ph+ leukaemias under the care of QMH for whom asciminib was requested through MAP were included. Details on patient demographics, disease history, prior treatment lines, asciminib usage, adverse events, efficacy and death were collected. Results At data cut-off for analysis on 30 June 2023, 13 patients for whom asciminib was approved had data available. One patient passed away before the commencement of asciminib, and thus was excluded (Table 1). There were 7 patients with CML, who on diagnosis were all in chronic phase at a median age of 53 (22-70) years. All had received imatinib and at least one newer generation TKI, with 5 patients (71%) having received ponatinib. The majority (5 patients, 71%) received asciminib because of lack of efficacy of prior treatment, with 4 patients also having the T315I mutation. The median age at asciminib treatment (median dose: 400 mg, 80-400 mg) was 64 (28-76) years, with a median time from diagnosis of 81 (13-314) months. Two patients received imatinib concomitantly. The median duration of treatment was 12 (6-38) months. Two patients were already in at least major molecular response (MMR) before treatment, and have maintained that response. Two patients had ≥ 1-log reduction of BCR::ABL1 transcript level. Three patients had stable disease. There was one death unrelated to CML (Figure 1). There were 5 patients with Ph-positive acute lymphoblastic leukaemia (ALL), at a median age of 57 (18-68) years at diagnosis. All had received prior chemotherapy in combination with a TKI (imatinib, N=3; dasatinib, N=5; ponatinib, N=5), with 3 patients having undergone allogeneic haematopoietic stem cell transplantation (allo-HSCT). Three patients had detectable kinase domain mutation (T315I, N=2; E255K + E459K, N=1). The majority (3 patients, 60%) received asciminib because of lack of efficacy of prior treatment. The median age at asciminib treatment (median dose: 240 mg, 80-400 mg) was 58 (19-72) years, with a median time from diagnosis of 22 (7-47) months. The median duration of treatment was 16 (2-19) months. Four patients received another ATP-competitive TKI concomitantly (imatinib, N=1; dasatinib, N=1; ponatinib, N=2). For patients receiving asciminib due to prior treatment failure (N=3), complete molecular response (CMR) was achieved in 2 patients with measurable residual BCR:: ABL1; 1 patient with frank leukaemia achieved complete remission with incomplete hematologic recovery with molecular response at 0.06%. For two patients receiving asciminib because of intolerance to prior treatment, one further improved from MR4 to CMR while pre-existing CMR was maintained in the other patient. There were 3 deaths; 2 due to COVID-19 and 1 due to refractory leukaemia (Figure 1). Asciminib showed an acceptable safety profile, with raised lipase leading to dose reduction only in 1 patient. Grade 3 or above toxicities were all haematological, observed in 5 patients, 2 of whom had previous allo-HSCT. One patient on asciminib-ponatinib treatment developed grade 2 renal impairment. Conclusion Asciminib demonstrated promising clinical efficacy with satisfactory tolerance in this cohort of Ph+ leukaemias failing multiple lines of therapy. Further investigation of its therapeutic role in Ph+ ALL is warranted.

Psychometric assessment of the Adherence to Asthma Medication Questionnaire in asthma patients in China: a validation study

Background Patients’ medication adherence plays a critical role in the treatment and rehabilitation of disease. However, few tools are currently available that can be used to identify the reasons for their nonadherence with their medication regimens. It is possible to evaluate both the level of medication adherence of a patient as well as the reasons for it using the Adherence to Asthma Medication Questionnaire (AAMQ). The purpose of this study was to adapt the AAMQ for use in Chinese patients and a variety of asthma patients. Methods A total of 242 asthma patients were recruited from Jinzhou in China. The Adherence to Asthma Medication Questionnaire was translated and back-translated using the Brislin translation model. Test-retest reliability, internal consistency, and stability all play a large role in determining the reliability of a scale. Confirmatory factor analysis was used to examine the scale’s construct validity, and expert consultation was used to verify the scale’s content validity. Statistics were deemed significant at p < 0.05. Results The Cronbach’s α value of the Chinese version of the AAMQ was 0.866, and the coefficient values for the three domains ranged between 0.702 and 0.798. The split-half reliability and stability values were 0.794 and 0.772, respectively. The content validity index of the scale (S-CVI) was 0.923, and the content validity index of the level of scale entry was 0.857–1.000. According to the confirmatory factor analysis, the chi-square degreed of freedom were 1.484, and the model fitting indices were all within normal limits. Conclusions The AAMQ had good reliability and validity for asthmatic patients. The results of the scale’s assessment can be used as a criterion for medication adherence among asthmatic patients and to understand the causes. This study provides a reference for solving the problem of medication adherence among asthma patients and implementing targeted nursing measures. Patient or public contribution Gratitude is extended to all individuals collaborating in completing the survey and to the author.

Efficacy of interpersonal psychotherapy in mainland China: a systematic review and meta-analysis.

Interpersonal Psychotherapy (IPT) is an evidence-based therapy. There have been increasing demand and training opportunities of IPT in China. Reviewing current evidence on its use in Chinese patients can help us understand the applicability of IPT in China and identify knowledge gaps to encourage and better future research in this field. We did a comprehensive search of three major electronic databases: PubMed (English), Chinese National Knowledge Infrastructure (CNKI) and WanFang Data (Chinese). We examined overall study design, outcome measures, data analyses and other parameters. We only selected articles of Randomized Clinical Trials (RCT) for this review. All study findings were grouped and summarized per psychiatric diagnoses. The meta-analysis and forest plots were performed whereas studies could be combined. After a full text review of 132 articles, 40 were selected for the final review. Comparing with control groups, evidences supported the efficacy of IPT in Chinese patients with Major Depressive Disorder (MDD), Postpartum depression, Generalized Anxiety Disorder (GAD), Social Anxiety Disorder, Post Stress Traumatic Disorder (PTSD), and Post-psychotic Depression. It was also beneficial to college students and Chinese first-time mothers. Meta-analysis using a random-effects model consistently yielded significant score differences between the IPT and control groups (p < 0.0001) on MDD. This systematic review has identified the current best evidence for IPT efficacy in Chinese population. The findings support IPT as an effective treatment in Chinese with certain psychiatric conditions, consistent with those from many other studies throughout the world.

Palbociclib plus endocrine therapy in hormone receptor-positive and HER2 negative metastatic breast cancer: a multicenter real-world study in the northwest of China

BackgroundReal-world data of Palbociclib are insufficient in China. This study aimed to investigate the treatment pattern and real-world outcomes in hormone receptor positive and human epidermal growth factor 2 receptor negative (HR+/HER2-) metastatic breast cancer (MBC) patients treated with Palbociclib in the northwest of China.MethodsHR+/HER2- MBC patients who received Palbociclib in 8 centers from July 2017 to September 2019 were retrospectively included in this study. Real-world objective response rate (ORR), progression-free survival (PFS) and safety profiles were analyzed. The survival curves were plotted by the Kaplan-Meier method to analyze PFS, which was verified by the log-rank test.ResultsIn total, 211 women were eligible for the analysis. A total of 85 patients (40.3%), 78 (37.0%), and 48 (22.7%) received Palbociclib in the first-, second-, third- or later-line setting, respectively. 46 patients achieved partial response and 145 patients experienced stable disease, with an ORR of 21.8% and a disease control rate of 90.5%. Following a median follow-up period of 14.2 months, the median PFS was 12.2 months (95% confidence interval, 10.1-14.3 m), and the median overall survival was not reached. Early Palbociclib initiation, sensitivity or acquired resistance to endocrine therapy, estrogen receptor and progesterone receptor double positivity, less than 3 metastatic sites, without visceral metastasis, bone metastasis only, without prior chemotherapy or endocrine therapy were associated with a prolonged PFS in MBC (All P < 0.05). The most common grade 3 or 4 adverse events (AE) was neutropenia (36.5%), and the most common nonhematologic AE was fatigue (10.9%). No patient experienced AE leading to treatment discontinuation.ConclusionPalbociclib plus endocrine therapy exhibited favorable effectiveness and manageable toxicities in the real-world setting, supporting their use in Chinese patients with HR+/HER2 − MBC.

Translation and adaptation of the EORTC QLQ-LC 29 for use in Chinese patients with lung cancer

BackgroundThe latest European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Lung Cancer 29 (QLQ-LC29) has been translated and validated in several languages but not yet in simplified Chinese. This study aimed to translate this questionnaire into simplified Chinese and adapt it for use in Chinese patients with lung cancer.MethodsThe translation and adaptation process followed the EORTC translation procedure, and consisted of eight steps, namely, translation preparation, forward translations, reconciled translation, back translations, a back translation report, proofreading, pilot testing, and finalisation. The pilot testing included 10 patients with lung cancer.ResultsWe obtained the permission to perform the EORTC QLQ-LC29 translation work on November 17, 2020. Thereafter, it took 3 weeks to complete the forward translations, reconciled translation, and back translations. After several rounds of discussion with the EORTC Translation Unit, 19 items used the existing translations from the EORTC Item Library (a database of EORTC questionnaire items and their translations), and 10 items were translated from scratch. The 10 patients included in the pilot testing phase had a median age of 64 years (range 31–69 years); five were male, five had an educational level of high school or above, and six had undergone surgery. Eight items received comments from patients (six items by one patient alone and the other two items by three patients). No patients commented on the instructions or the format used for responses. After discussion with the EORTC Translation Unit, we modified the Chinese wording in item 50 to ensure that the meaning of “lifeless” was clear. No changes were made to the remaining items.ConclusionsThe simplified Chinese version of the EORTC QLQ-LC29 is now available on the EORTC website. This translation may contribute to the application of the EORTC QLQ-LC29 scale in both research and clinical practice in the Chinese population with lung cancer. Further evaluation of the psychometric properties of the translated EORTC QLQ-LC29 is warranted.

Efficacy and safety of eltrombopag in Chinese patients with chronic immune thrombocytopenia: stage 2 results from a multicenter phase III study

This phase III, randomized, placebo-controlled study conducted in three stages (6-week, randomized, placebo-controlled stage 1; 24-week, open-label stage 2; and continuous extension stage 3) assessed the long-term efficacy and safety of eltrombopag use in Chinese patients with chronic immune thrombocytopenia (ITP). This article presents the results from stage 2. Overall, 150 patients (placebo-eltrombopag [P-E], 50; eltrombopag-eltrombopag [E-E], 100) received open-label eltrombopag. The median platelet count was maintained between 41 × 109/L and 80 × 109/L. Most patients in both groups (P-E, 90.0%; E-E, 81.8%) achieved platelet counts ≥30 × 109/L and ≥2 times the baseline platelet count at least once with eltrombopag treatment. Overall, 32% of patients achieved platelet counts ≥50 × 109/L in ≥75% of platelet count assessments. Both groups showed a decreased tendency to infrequent bleeding and clinically significant bleeding events during stage 2 compared with baseline. Among patients who received ≥1 ITP medication at baseline, 70.4% in the P-E group and 40.8% in the E-E group reduced or permanently stopped ≥1 of their ITP medications. The stage 2 results further demonstrated a sustainable long-term efficacy and good tolerability of eltrombopag with a favorable benefit–risk ratio in Chinese chronic ITP patients. Trial registration: Clinicaltrials.gov NCT01762761. Registered 8 January 2013, https://clinicaltrials.gov/ct2/show/NCT01762761

Linguistic and Psychometric Validation of the Chinese Version of the Control Attitudes Scale-Revised in Patients With Chronic Heart Failure.

The concept of perceived control reflects the belief that one has resources needed to cope with negative events and the ability to positively influence consequences of those negative events. In patients with heart failure, perceived control is associated with a variety of health outcomes. Perceived control is commonly measured using the Control Attitudes Scale-Revised (CASR). There is no Chinese version of the CAS-R (CCAS-R). The purpose of this article was to perform linguistic validation and psychometric evaluation of the CCAS-R. The CAS-R was translated into Chinese according to Brislin's model. Then, a multicenter observational study was performed. Floor and ceiling effects, internal consistency, structural validity, and hypothesis testing were all assessed for psychometric validation of the CCAS-R. A total of 227 patients with chronic heart failure were included. There were no ceiling or floor effects detected. Cronbach α was 0.94, indicating a high reliability. The results of the confirmatory factor analysis showed that the 1-factor structure as proposed by the original CAS-R fits the data well. The results of the principal component analysis suggested that the 1-factor structure was optimal as well, accounting for 71.6% of the total variance. The a priori hypothesis was supported by a statistically significant correlation between the CCAS-R and 3 theoretically related variables. We developed a semantically equivalent version of the CAS-R in Chinese. The evaluation of the instruments' psychometric properties demonstrated that the CCAS-R has good reliability and validity for use in Chinese patients with chronic heart failure.

Efficacy and Safety of Budesonide/Glycopyrrolate/Formoterol Fumarate Metered Dose Inhaler in Chinese Patients with COPD: A Subgroup Analysis of KRONOS.

IntroductionThis pre-specified subgroup analysis evaluated the efficacy and safety of budesonide/glycopyrrolate/formoterol fumarate metered dose inhaler (BGF MDI) triple therapy versus corresponding dual therapies in the China subgroup of the phase III, double-blind KRONOS study in patients with moderate to very severe chronic obstructive pulmonary disease (COPD).MethodsPatients were randomized 2:2:1:1 to BGF MDI 320/18/9.6 μg, glycopyrrolate/formoterol fumarate (GFF) MDI 18/9.6 μg, budesonide/formoterol fumarate (BFF) MDI 320/9.6 μg, or budesonide/formoterol fumarate dry powder inhaler (BUD/FORM DPI) 400/12 μg twice daily for 24 weeks. The primary endpoint was change from baseline in morning pre-dose trough forced expiratory volume in 1 s (FEV1) over weeks 12–24. Secondary endpoints included symptoms, health-related quality of life, and safety. Rate of moderate/severe COPD exacerbations was an additional efficacy endpoint.ResultsIn the China subgroup (n = 432; 22.7% of the KRONOS population), BGF MDI demonstrated nominally significant improvements in the primary endpoint versus BFF MDI (least squares mean (LSM) difference 68 mL; P = 0.0035) and BUD/FORM DPI (LSM difference 78 mL; P = 0.0010) but not GFF MDI (LSM difference − 4 mL; P = 0.8316). BGF MDI demonstrated at least numerical improvements versus comparators in secondary lung function and symptom endpoints. BGF MDI reduced the rate of moderate/severe COPD exacerbations versus GFF MDI (rate ratio 0.41; P = 0.0030), with numerical benefits versus BFF MDI and BUD/FORM DPI. All treatments were well tolerated.ConclusionsResults demonstrated that BGF MDI showed benefits on lung function (vs inhaled corticosteroid/long-acting β2-agonist), as well as symptoms and exacerbations relative to dual therapies. Findings support BGF MDI use in Chinese patients with moderate to very severe COPD.Clinical Trial RegistrationClinicalTrials.gov NCT02497001.Electronic supplementary materialThe online version of this article (10.1007/s12325-020-01266-5) contains supplementary material, which is available to authorized users.

Warfarin therapy in Chinese patients with atrial fibrillation treated with percutaneous coronary intervention: a 5 year follow-up retrospective cohort study

Objective: To evaluate warfarin use in Chinese patients with atrial fibrillation (AF) undergoing percutaneous coronary intervention (PCI) by investigating the stroke and major adverse cardiac and cerebral events (MACCEs) and bleeding events.Methods: Retrospective cohort study of the 5 year follow-up of 1134 patients with AF who underwent PCI. The patients were grouped according to whether they received warfarin or not. Baseline characteristics and the occurrence of MACCEs and bleeding events were compared between the two groups using the CHA2DS2-VASc and HAS-BLED scoring. Cox regression analysis was used to identify factors related to the occurrence of MACCEs and bleeding.Results: Overall MACCE (p = .008) and mortality (p = .004) rates were significantly lower in the warfarin group compared with the non-warfarin group. Major bleeding, minor bleeding and overall bleeding were comparable in the two groups. Recurrent myocardial infarction (HR = 10.129, 95% CI = 4.737–21.655; p < .001) and a baseline CHA2DS2-VASc score >4 (HR = 2.035, 95% CI = 1.121–3.692; p = .019) were independent predictors of MACCEs in the warfarin group. A baseline HAS-BLED score ≥3 (HR = 5.498, 95% CI = 3.773–8.013; p < .001) and previous bleeding (HR = 3.058, 95% CI = 1.319–7.088; p = .009) were independent predictors of bleeding.Conclusions: Warfarin reduces the incidence of MACCEs but does not increase bleeding events in Chinese patients with AF who underwent PCI. For patients taking warfarin, recurrent myocardial infarction and a baseline CHA2DS2-VASc score >4 were related to MACCE occurrence.

A phase II study of apatinib combined with S-1 in previously treated refractory metastatic colorectal cancer.

e15016 Background: Although regorafenib and fruquintinib have been recommended as the third-line treatment for patients with refractory metastatic colorectal cancer (mCRC), the median survival time of mCRC is still only for 4-8 months and the low response rate and unpleasant side effects limit their use in Chinese patients. Apatinib, an oral VEGFR-2 inhibitor, has been approved as a third line treatment in metastatic gastric cancer. In addition, apatinib has demonstrated good safety, tolerability, and efficacy in the treatment of advanced solid tumors. The aim of this study was to assess the efficacy and safety of apatinib combined with S-1 in the treatment of refractory mCRC. Methods: In this prospective, open-label, single-arm, multicenter, phase II study, patients after failure of second-line chemotherapy were enrolled and took apatinib (250mg, daily) combined with S-1 (standard dose). The primary endpoint was progression free survival (PFS) and the second endpoint was response rate and overall survival time. Results: From December 2017, 22 patients (14 male) with a median age of 56y (range: 34-71 y) were enrolled and eligible for evaluation of the PFS, response rate and safety. The median PFS was 105d (95% CI: 79.01-130.98). two patients (9%) achieved partial response, 15 (68.18%) achieved stable disease, and 5 (22.72%) were progressive disease. The objective response rate and the disease control rate were 9% and 77.27%, respectively. Median overall survival was not reached. The common adverse effects were abnormal liver function (7/22; 31.81%), leukopenia (5/22; 22.72%) and thrombocytopenia (4/22; 18.18%). The incidence for grade 3-4 side effect was very low. One patient experienced grade 3 proteinuria and there were no toxic deaths. Conclusions: This preliminary result indicated that apatinib combined with S-1 may extend the PFS in mCRC, with well-tolerated toxicities, making it a promising therapeutic target for mCRC treatment. Clinical trial information: NCT03397199 .

Cost-effectiveness of Insulin Degludec Versus Insulin Glargine in Insulin-naive Chinese Patients With Type 2 Diabetes

PurposeThe goal of this study was to investigate the long-term economic outcomes of insulin degludec versus insulin glargine use in Chinese patients with type 2 diabetes mellitus (T2DM) whose oral antidiabetic drugs did not provide sufficient glycemic control. MethodsA published and validated Chinese diabetes health policy model, which reflects Chinese T2DM epidemiologic profiles, was used to assess the lifetime economic outcomes of microvascular and macrovascular complications and mortality. Efficacy and safety, medical expenditure, and utility data were derived from the literature, which were assigned to model variables for estimating the quality-adjusted life-years (QALYs) and costs, as well as incremental cost-effectiveness ratios. The analysis was conducted from the perspective of Chinese health care service providers. One-way and probabilistic sensitivity analyses were performed. FindingsCompared with insulin glargine, insulin degludec was associated with 0.0053 QALY at an additional cost of $3278 in our simulated cohort. This outcome resulted in an incremental cost-effectiveness ratio of insulin degludec over insulin glargine of $613,443 per QALY gained. The one-way sensitivity analyses indicated that the results were sensitive to several model inputs. ImplicationsInsulin degludec is unlikely to be cost-effective compared with insulin glargine for Chinese patients with T2DM whose disease is inadequately controlled with oral antidiabetic drugs.

Validation of the Centrality of Pain Scale in Chinese-Speaking Patients with Painful Temporomandibular Disorders.

The present study aimed to validate the Centrality of Pain Scale (COPS) for use in Chinese patients with painful temporomandibular disorders (TMDs). The Centrality of Pain Scale was firstly translated and cross-culturally adapted following international guidelines. In total, 166 patients with TMD were recruited to complete the Chinese version of the COPS (COPS-C). In addition to the COPS-C, the patients were also administered the Pain Catastrophizing Scale (PCS) and the Pain Self-Efficacy Questionnaire (PSEQ). The reliability of the COPS-C was evaluated using internal consistency and test-retest methods. The construct validity of the COPS-C was evaluated using exploratory factor analysis (EFA). Convergent validity was determined by analyzing the correlations between COPS-C scores and the scores of the PCS and PSEQ. Cronbach's alpha for the total COPS-C score was 0.942. The interitem correlations ranged from 0.356 to 0.901. The intraclass correlation coefficient values of the COPS-C ranged between 0.815 and 0.929. The results of the EFA indicated a one-factor solution for the measure, accounting for 70.4% of the total observed variance. The factor loadings of all items ranged from 0.713 to 0.917. Regarding convergent validity, the COPS-C had moderate correlations with the PCS and the PSEQ. The results provide initial evidence that the COPS-C is a reliable and valid measure. It can be used as a suitable instrument for Chinese patients with TMD.

Validation of the Pain Resilience Scale in Chinese-speaking patients with temporomandibular disorders pain.

To validate the Pain Resilience Scale (PRS) for use in Chinese patients with temporomandibular disorders (TMD) pain. According to international guidelines, the original PRS was first translated and cross-culturally adapted to formulate the Chinese version of PRS (PRS-C). A total of 152 patients with TMD pain were recruited to complete series of questionnaires. Reliability of the PRS-C was investigated using internal consistency and test-retest reliability. Validity of the PRS-C was calculated using cross-cultural validity and convergent validity. Cross-cultural validity was evaluated by examining the confirmatory factor analysis (CFA). And convergent validity was examined through correlating the PRS-C scores with scores of 2 commonly used pain-related measures (the Connor-Davidson Resilience Scale [CD-RISC] and the Tampa Scale for Kinesiophobia for Temporomandibular Disorders [TSK-TMD]). The PRS-C had a high internal consistency (Cronbach's alpha=0.92) and good test-retest reliability (intra-class correlation coefficient [ICC]=0.81). The CFA supported a 2-factor model for the PRS-C with acceptable fit to the data. The fit indices were chi-square/DF=2.21, GFI=0.91, TLI=0.97, CFI=0.98 and RMSEA=0.08. As regards convergent validity, the PRS-C evidenced moderate-to-good relationships with the CD-RISC and the TSK-TMD. The PRS-C shows good psychometric properties and could be considered as a reliable and valid measure to evaluate pain-related resilience in patients with TMD pain.

Development and pilot-testing of patient decision aid for use among Chinese patients with primary open-angle glaucoma

BackgroundA patient decision aid (PDA) is a tool for shared decision making (SDM), which emphasises patient empowerment. It is useful in chronic diseases and when there are multiple, no best...

Validation of the Social support and Pain Questionnaire (SPQ) in patients with painful temporomandibular disorders

BackgroundThe present study aimed to validate of Social support and Pain Questionnaire (SPQ) for use in Chinese patients with painful temporomandibular disorders (TMD).MethodsThe Chinese version of SPQ was produced by translation and cross-culturally adaptation of the original English version according to international guidelines. The Chinese version of SPQ was then distributed to a total of 118 patients with painful TMD. Reliability of the SPQ was evaluating using internal consistency and test-retest methods and validity of the SPQ was determined by construct validity and convergent validity. The exploratory factor analysis (EFA) was used to assess the construct validity of SPQ. And convergent validity was assessed by correlating the SPQ scores with the score of a global oral health question.ResultsThe Chinese version of SPQ has a high internal consistency (Cronbach’s alpha value, 0.926) and good test-retest reliability ((intraclass correlation coefficient (ICC), 0.784). Construct validity was evaluated by EFA, extracting one factor, accounting for 74.8% of the variance. All factor loadings of the six items had exceeded 0.80. As regards convergent validity, the SPQ showed good correlation with the global oral health question.ConclusionThese findings support that the Chinese version of SPQ can be used as a reliable and valid tool for Chinese patients with painful TMD.

Current status of antiarrhythmic drug use and safety assessment in Chinese patients with atrial fibrillation

Objective: To investigate the current status of antiarrhythmic drugs (AADs) use in Chinese patients with atrial fibrillation(AF) and assess the safety of AADs in this patient cohort. Methods: From January 2011 to December 2013, a total of 4 008 AF patients treated with AADs was enrolled in this study and patients were followed up for 24 months. Detailed information of prescribed drug, the causes of drug discontinuation and side effects were recorded. Results: Amiodarone was prescribed to 64.3%(2 579 cases) and propafenone to 31.1%(1 247 cases) of the enrolled patients, only 148 patients(3.7%) were treated with sotalol and 34 patients (0.8%) were treated with moracizine. The prevalence of heart failure (4.0%(102/2 579) vs. 1.4%(17/1 247, P<0.001), coronary heart disease (13.5% (348/2 579) vs. 7.4%(93/1 247), P<0.001) and non-ischemic cardiomyopathy (3.1%(78/2 579) vs. 0.7%(9/1 247), P<0.001) was significantly higher in patients treated with amiodarone than in the patients treated with propafenone. During the follow-up period, the discontinuation rate of amiodarone, propafenone, sotalol and moracizine was 28.8%(743/2 579), 25.1%(313/1 247), 14.2%(21/148) and 32.4%(11/34) respectively. The reasons of discontinuing amiodarone were: follow physicians' decision (75.7%, 563 cases), no effect (3.0%, 22 cases), side effects (4.3%, 32 cases) and patients' own decision (17.0%, 126 cases). The side effects of amiodarone included thyroid dysfunction (56.3%, 18 cases), bradycardia (12.5%, 4 cases), interstitial pneumonitis/pulmonary interstitial fibrosis (6.2%, 2 cases) and others (gastrointestinal symptom, rash, hepatic dysfunction, etc.). Conclusions: Amiodarone and propafenone are the most common AADs used in Chinese patients with atrial fibrillation. The prescription of AADs is essentially in accordance to the guideline of AF treatment. However, the discontinuation rates of AADs are high in Chinese AF patients. Lacking of better AADs is still a major problem in AF pharmacotherapy. Clinical Trial Registry Chinese Clinical Trial Registry, ChiCTR-OCH-13003729.

A large-scale, multicenter, retrospective study on efficacy of bivalirudin use during peri-percutaneous coronary intervention period for Chinese patients with coronary heart disease

To observe the efficacy and safety of bivalirudin use in Chinese patients with coronary heart disease (CHD) during the peri-percutaneous coronary intervention(PCI) period. A total of 3 271 patients who underwent PCI and received periprocedural bivalirudin treatment between July 2013 and October 2015 from 88 centers of China were involved in this study. The primary outcome was 30-day net adverse clinical events (NACE a composite of major adverse cardiac or cerebral events (MACE, all-cause death, reinfarction, urgent target vessel revascularization, or stroke) or bleeding), the secondary outcome was stent thrombosis at 30 days. The mean age of enrolled patients was (65.12±12.44) years old, 27.4%(889/3 244) of them were female. Percent of stable coronary disease (SCD), non-ST segment elevation acute coronary syndrome (NSTE-ACS) and ST elevation myocardial infarction (STEMI) was 5.0%(162/3 248), 44.6%(1 450/3 248) and 50.4%(1 636/3 248) respectively. Radial access was performed in 89.5% (2 879/3 271) patients, and 9.7% (316/3 271) and 34.1% (1 115/3 271) patients also received ticagrelor and tirofiban medication. 69.3% (2 266/3 271) patients received post-procedural bivalirudin infusion, in which 46.3% (1 050/2 266) was treated at PCI-does, with a median duration of 2.5(1.0, 4.0) h. During the 30-day follow-up, NACE occurred in 3.45% (103/2 988) patients, the incidence of MACE, death was 2.17% (65/2 994) and 1.03% (31/3 017), respectively and bleeding events were recorded in 1.37% (41/2 996) patients. Four cases (0.13%) of stent thrombosis (3 acute stent thrombosis) were recorded. Peri-PCI Bivalirudin use is safe and related with low bleeding risk in Chinese CHD patients.

Efficacy of sorafenib correlates with Memorial Sloan-Kettering Cancer Center (MSKCC) risk classification and bone metastasis in Chinese patients with metastatic renal cell carcinoma

Several prognostic models have been developed to assess the efficacy and safety of sorafenib for metastatic renal cell carcinoma (mRCC), but few studies have validated its use in Chinese patients. The objective of this single center, single arm retrospective study was to examine the efficacy and safety of sorafenib and its related prognostic clinico-pathologic factors in Chinese mRCC patients. One hundred thirty four mRCC patients were enrolled. All patients received 400 mg of sorafenib orally twice daily. The dose was subsequently adjusted in the event of treatment-induced toxicity. Tumor response, progression-free survival (PFS), overall survival (OS) and adverse events (AEs) were determined. The median PFS and OS were 10 months (1-36 months) and 22 months (2-37 months), respectively. Complete, partial, and stable disease were observed in two (1.49%), 24 (17.91%), and 99 (73.88%) patients, respectively. Hand/foot skin reactions, diarrhea and fatigue were the most commonly observed AEs following sorafenib treatment. Among the AEs, only 13 grades 3 and 4 were observed. Multivariate analysis revealed that independent predictive factors for PFS included Eastern Cooperative Oncology Group (ECOG) status, Memorial Sloan-Kettering Cancer Center (MSKCC) risk status, and bone metastasis (all p < 0.05). Factors associated with OS included MSKCC risk values, bone metastasis and sorafenib-induced hypertension (all p < 0.05). The introduction of sorafenib therapy for mRCC in Chinese patients may lead to a favorable disease control with acceptable tolerability. In addition, the parameters predicting favorable outcomes, including ECOG status, MSKCC risk status and bone metastasis, may have prognostic value in clinical practice.

Development and validation of new glomerular filtration rate predicting models for Chinese patients with type 2 diabetes

BackgroundPrevious researches has depicted that the performance of the recommended glomerular filtration rate (GFR)-estimating equations in the type 2 diabetic population is inferior to that in the non-diabetic population. We attempted to develop new GFR-predicting models for use in Chinese patients with type 2 diabetes in this study.MethodsWe enrolled 519 type 2 diabetic patients including a development data-set (n = 276), an internal validation data-set (n = 138) and an external validation data-set (n = 105) to establish new GFR-predicting models. 99mTc-DTPA-GFR revised by the dual sample method was referred to as the gold GFR standard.ResultsBased on sex, age, serum creatinine and new predictor variables [body mass index (BMI), hemoglobinA1C, and urinary albumin creatinine ratio], eight new regression models and eight artificial neural network (ANN) models were developed. In the external validation group, only ANN3 was superior in both precision and accuracy over the original CKD-EPI equation (precision, 20.5 vs. 24.2 mL/min/1.73 m2, P < 0.001; 30 % accuracy, 88.6 vs. 80.6 %, P = 0.02).ConclusionsANN3 based on sex, age, serum creatinine and BMI is the optimal model for GFR estimation in Chinese patients with type 2 diabetes.Electronic supplementary materialThe online version of this article (doi:10.1186/s12967-015-0674-y) contains supplementary material, which is available to authorized users.

PP03.17 – 2750: Bone turnover and its connection to penicillamine use in Chinese patients with Wilson disease

Objective The prevalence of osteoporosis is higher among patients with Wilson disease (WD) than the general public, of which the cause is still unclear. The purpose of this research is to analyze the possible causes for the high prevalence and connection between penicillamine and bone mineral density. Methods Eight-four WD patients (44 males and 40 females) and 76 controls (27 males and 49 females) were enrolled in this study. The patients were diagnosed via mutation analysis of ATP7B. Dual-energy X-ray Analysis (DXA) was used to evaluate the bone mineral density (BMD). Serum phosphate, calcium, magnesium, parathyroid hormone, calcitonin, osteocalcin, 25-(OH)-Vitamin D as well as alkaline phosphatase were measured to evaluate the bone turnover activity. Based on the information on imaging, laboratory test and previous medical history, we analyzed the correlations between BMD, bone turnover activity and patients' age, gender, clinical manifestations and maximum dosage of penicillamine. Results Fifty-eight patients received a DXA scan. The prevalence of osteoporosis in WD patients was 27.58% (16/58) whereas that in the controls was 19.74% (15/76). BMD, T. score and Z score of lumbar vertebrae as well as BMD, Z score of hip neck were significantly lower in WD patients. 25(OH)-Vitamin D, serum phosphate, serum calcium and ALP were significantly higher in WD patients than the controls. But among patients who have received different maximum daily dosage of penicillamine, larger dose was associated with lower BMD. Age, gender and clinical manifestations did not affect BMD among WD patients. Conclusion WD patients have a higher prevalence of osteoporosis, lower BMD and higher bone turnover activity compared with controls. The use of penicillamine is an important precipitating factor.