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Transparency Ecosystem for Research and Journals in Medicine (TERM) working group summarized the essential recommendations that should be considered to review and publish a high-quality guideline. These recommendations from editors and reviewers included 10 components of essential requirements: systematic review of existing relevant guidelines, guideline registration, guideline protocol, stakeholders, conflicts of interest, clinical questions, systematic reviews, recommendation consensus, guideline reporting and external review. TERM working group abbreviates them as PAGE (essential requirements for Publishing clinical prActice GuidelinEs), and recommends guideline authors, editors, and peer reviewers to use them for high-quality guidelines.

Rationale There are no randomized trials examining the best treatment for acute basilar artery occlusion in the 6–24-hour time window. Aims To assess the safety and efficacy of thrombectomy for stroke due to basilar artery occlusion in patients randomized within 6–24 h from symptom onset or time last seen well. Sample size For an estimated difference of 20% in proportions of the primary outcome between the two groups, 318 patients will be included for 5% significance and 90% power with a planned interim analysis after two-thirds of the sample size (212 patients) have achieved the 90 days follow-up. Methods and design A prospective, multi-center, randomized, controlled, open-label and blinded-endpoint trial. The randomization employs a 1:1 ratio of mechanical thrombectomy with the detachable Solitaire thrombectomy device and best medical therapy (BMT) vs. BMT alone. Study outcomes The primary outcome will be the proportion of patients achieving modified Rankin Scale (mRS) 0–3 at 90 days. Key secondary outcomes are: dramatic early favorable response, dichotomized mRS score (0–2 vs. 3–6 and 0–4 vs. 5–6) at 90 days, ordinal (shift) mRS analysis at 90 days, infarct volume at 24 h, vessel recanalization at 24 h in both treatment arms, and successful recanalization in the thrombectomy arm according to the modified thrombolysis in cerebral infarction (mTICI) classification defined as mTICI 2 b or 3. Safety variables are mortality at 90 days, symptomatic intracranial hemorrhage rates at 24 h, and procedure-related complications. Discussion Results from this trial will indicate whether mechanical thrombectomy is superior to medical management alone in achieving favorable outcomes in subjects with acute stroke caused by basilar artery occlusion presenting within 6–24 h from symptom onset. Trial registration: URL: http://www.clinicaltrials.gov . ClinicalTrials.gov Identifier: NCT02737189.

Background The main purpose was to determine basic epidemiological data on CKD among hospitalized pediatric patients in China. Methods Data from pediatric inpatients with CKD hospitalized from June 1, 2013 to May 31, 2017 were extracted from the electronic records of HQMS database, which includes over 14 million inpatients. Codes from the 10th revision of the International Classification of Diseases (ICD-10) were used to search the database. Results A total of 524 primary diseases of CKD were included in this study. In all, there were 278 231 pediatric inpatients with CKD, which accounted for 1.95 % of the 14 250 594 pediatric inpatients registered in the HQMS database. The number of pediatric inpatients with CKD was 67 498 in 2013, 76 810 in 2014, 81 665 in 2015 and 82 649 in 2016, which accounted for 1.93 %, 1.93 %, 1.99 and 2.09 %, respectively, of the total population of pediatric inpatients. The etiology of CKD was secondary nephrosis in 37.95 % of cases, which ranked first and followed by CAKUT with a percentage of 24.61 %. Glomerular diseases and cystic kidney disease accounted for 21.18 and 5.07 %, respectively. Among all 278 231 patients, 6 581 (2.37 %) had a primary discharge diagnosis of CKD. The renal pathology findings of CKD showed that IgA accounted for 51.17 %. Conclusions This study provides a descriptive analysis of the hospitalized population of pediatric CKD patients. Our study provides important, fundamental data for policy making and legislation, registry implementation and the diagnosis, treatment and prevention of CKD in China.

Objectives To evaluate the efficacy and safety of leflunomide (LEF) and glucocorticoids (GCs) combination therapy compared with GCs monotherapy in preventing relapse of IgG4-related disease (IgG4-RD). Methods A 12-month, randomized, open-label, controlled trial was conducted at a large academic medical center (ClinicalTrials.gov: NCT02703194). Enrolled patients with active IgG4-RD were randomly allocated to the GCs + LEF (20 mg/day) combination therapy or GCs monotherapy group. All patients received GCs with a predefined taper regimen starting from a dosage of 0.5–0.8 mg/kg/d. The primary outcome was the time to relapse. The secondary outcomes included complete response, remission, GCs dosage, and serum IgG4 level. Results Sixty-six patients with active IgG4-RD were enrolled (33 patients in each group). The demographic and disease characteristics showed no statistically significant differences between groups. Additionally, the initial GCs dosages were similar (50.00 vs. 50.00 mg/day, P = 0.295). Disease relapses occurred in 6 (18.2%) and 14 (42.4%) patients in the combination therapy group and GCs monotherapy group, respectively (P = 0.032). The combination therapy was significantly superior to GCs monotherapy regarding the primary outcome, the time to relapse (HR, 0.35; 95% confidence interval [CI], 0.13–0.90; P = 0.023), as well as the secondary outcome, the time to complete response (HR, 1.75; 95% CI, 1.01–3.02; P = 0.034). A longer duration of remission was observed in the combination therapy group (7.00 vs. 3.00 months, P = 0.002) and less cumulative dosage of GCs was used (5103.13 vs. 5637.50 mg, P = 0.031). Additionally, a higher proportion of patients in the combination therapy group (54.5%) were able to reach a daily GCs dose of ≤5 mg/day compared with the GCs monotherapy group (18.2%) (P = 0.006). The incidences of adverse events were similar in the 2 groups (P = 0.325). Conclusion LEF in combination with GCs therapy is well-tolerated and significantly superior to GCs monotherapy in preventing the relapse of IgG4-RD. LEF can be used as a steroid-sparing agent in the management of IgG4-RD.

Background Peripheral-blood (PB) and bone marrow (BM) are both widely used in hematopoietic stem cell transplantation (HSCT). However, it is unclear whether PB or BM produces a more satisfactory outcome in haploidentical HSCT, particularly for patients using post-transplant cyclophosphamide (PTCy), which is the standard therapy. However, to date, no meta-analysis focusing on this issue has been published. Methods We systematically searched PubMed, MEDLINE, Web of Science, the Cochrane Library and the ClinicalTrials.gov website for studies regarding the use of BM or PB in haploidentical HSCT for hematological malignancies in adults using PTCy. Data were analyzed using Open Meta-Analyst statistical software. Results Fourteen studies were extracted including four comparative retrospective reports and ten single-arm reports, with a total of 1759 patients received PTCy haploidentical HSCT (462 patients received PBSCT, 1297 patients received BMT). The pooled outcomes of comparative retrospective studies showed significantly higher incidence of grade III-IV acute graft-versus-host disease (GVHD) (OR = 1.741, 95%CI 1.032-2.938), incidence of grade II IV acute GVHD (OR = 1.778, 95%CI 1.314, 2.406) and engraftment rate (OR = 1.843, 95%CI 1.066-3.185) in the PB group. No significant differences were found on the incidence of relapse, 2-year overall survival (OS) and disease-free survival (DFS), acute II IV GVHD and chronic GVHD between PBSCT or BMT. Conclusion The efficacy of PB is not inferior to BM for patients undergoing PTCy haploidentical HSCT with regard to primary outcomes, including OS, DFS, NRM and relapse. However, with regards to convenience and pain relief, PB graft is suitable for haploidentical HSCT, but with a higher risk of acute GVHD.

ObjectiveTo investigate the gap between real-world data and clinical research initiated by doctors in China, explore the potential reasons for this gap and collect different stakeholders’ suggestions.DesignThis qualitative study involved three types of hospital personnel based on three interview outlines. The data analysis was performed using the constructivist grounded theory analysis process.SettingSix tertiary hospitals (three general hospitals and three specialised hospitals) in Beijing, China, were included.ParticipantsIn total, 42 doctors from 12 departments, 5 information technology managers and 4 clinical managers were interviewed through stratified purposive sampling.ResultsElectronic medical record data cannot be directly downloaded into clinical research files, which is a major problem in China. The lack of data interoperability, unstructured electronic medical record data and concerns regarding data security create a gap between real-world data and research data. Updating hospital information systems, promoting data standards and establishing an independent clinical research platform may be feasible suggestions for solving the current problems.ConclusionsDetermining the causes of gaps and targeted solutions could contribute to the development of clinical research in China. This research suggests that updating the hospital information system, promoting data standards and establishing a clinical research platform could promote the use of real-world data in the future.

Background No therapeutics have yet been proven effective for the treatment of severe illness caused by SARS-CoV-2. Methods We conducted a randomized, controlled, open-label trial involving hospitalized adult patients with confirmed SARS-CoV-2 infection, which causes the respiratory illness Covid-19, and an oxygen saturation (Sao2) of 94% or less while they were breathing ambient air or a ratio of the partial pressure of oxygen (Pao2) to the fraction of inspired oxygen (Fio2) of less than 300 mm Hg. Patients were randomly assigned in a 1:1 ratio to receive either lopinavir–ritonavir (400 mg and 100 mg, respectively) twice a day for 14 days, in addition to standard care, or standard care alone. The primary end point was the time to clinical improvement, defined as the time from randomization to either an improvement of two points on a seven-category ordinal scale or discharge from the hospital, whichever came first. Results A total of 199 patients with laboratory-confirmed SARS-CoV-2 infection underwent randomization; 99 were assigned to the lopinavir–ritonavir group, and 100 to the standard-care group. Treatment with lopinavir–ritonavir was not associated with a difference from standard care in the time to clinical improvement (hazard ratio for clinical improvement, 1.31; 95% confidence interval [CI], 0.95 to 1.80). Mortality at 28 days was similar in the lopinavir–ritonavir group and the standard-care group (19.2% vs. 25.0%; difference, −5.8 percentage points; 95% CI, −17.3 to 5.7). The percentages of patients with detectable viral RNA at various time points were similar. In a modified intention-to-treat analysis, lopinavir–ritonavir led to a median time to clinical improvement that was shorter by 1 day than that observed with standard care (hazard ratio, 1.39; 95% CI, 1.00 to 1.91). Gastrointestinal adverse events were more common in the lopinavir–ritonavir group, but serious adverse events were more common in the standard-care group. Lopinavir–ritonavir treatment was stopped early in 13 patients (13.8%) because of adverse events. Conclusions In hospitalized adult patients with severe Covid-19, no benefit was observed with lopinavir–ritonavir treatment beyond standard care. Future trials in patients with severe illness may help to confirm or exclude the possibility of a treatment benefit. (Funded by Major Projects of National Science and Technology on New Drug Creation and Development and others; Chinese Clinical Trial Register number, ChiCTR2000029308. opens in new tab.)

Background To investigate the repeatability of superficial retinal vessel density measurements in healthy eyes with long axial length (AL) using optical coherence tomography angiography (OCTA). Methods There were 60 eyes of 31 volunteers enrolled in this cross-sectional observational study. All subjects underwent OCTA, AL and refraction test. The enrolled eyes were divided into the long AL group (26 mm ≤ AL

Purpose To compare the anatomical outcomes of different extents of internal limiting membrane (ILM) peeling in idiopathic macular hole surgery. Methods Prospective, parallel‐group, randomized clinical trial. A total of 121 eyes of 121 patients with idiopathic macular hole underwent pars plana vitrectomy, and peeling of the ILM with a diameter of two disk diameters (DD) or 4DD based on randomization. The main outcome was the proportion of eyes with complete hole closure at 12 months. The second outcome was the hole closure grading stratified by macular hole closure index (MHCI) at each visit. Results At 12 months, there was no significant difference in anatomical outcomes with complete closure achieved in 52 (82.5%) of 63 eyes in the 2DD group and 53 (91.4%) of 58 eyes in the 4DD group (p = 0.15). For subjects with MHCI ≤0.5 (n = 24), complete closure rate was significantly lower in the 2DD group compared to the 4DD group (p = 0.012; 18.2% versus 75.9%, respectively). Average BCVA was lower in 2DD group than 4DD group (p = 0.014). By contrast, when MHCI was >0.5, the complete closure rate between the two groups showed no significant difference: 96.2% (50 patients) versus 95.6% (43 patients), respectively (p = 0.185). Conclusion In patients with idiopathic full‐thickness macular hole and MHCI ≤0.5, a larger ILM peel of 4DD tends to achieve better anatomical outcomes than a more limited 2DD peel.

Background There are many public health issues to resolve regarding rare diseases, including a lack of data from large-scale studies. The objective of this study was to explore fundamental data for a list of rare diseases in China, based on a hospitalization summary reports (HSRs) database. The Target Rare Diseases List (TRDL) 2017 was generated using an expert consensus method in which experts listed diseases according to research priorities. Using codes of the 10th revision of the International Statistical Classification of Diseases and Related Health Problems (ICD-10) and key search terms of rare diseases in English and Chinese, data were obtained from HSRs of 96 hospitals, covering a population of over 15 million in China from 2014 to 2015. We extracted and analyzed information on demographics, hospitalizations, and readmissions. Results A total 281 rare diseases were included in the TRDL 2017. Altogether, 106,746 hospitalizations for a rare disease were captured from 1 January 2014 to 31 December 2015, accounting for 0.69% of inpatients during the same period. The top 10 rare diseases with most cases on the TRDL 2017 were thalassemia, idiopathic pulmonary arterial hypertension, pulmonary Langerhans cell histiocytosis, moyamoya disease, motor neuron disease, idiopathic pulmonary fibrosis, systemic sclerosis, hepatolenticular degeneration, coarctation of the aorta, and transposition of the great arteries. Among the 24 cities in the database, the five cities with the most types of the rare disease were Beijing, Changsha, Guangzhou, Shanghai, and Chengdu, with 191, 162, 143, 141, and 133 types, respectively. The five cities with most cases of the 281 rare diseases were Beijing, Guangzhou, Shanghai, Nanning, and Chengdu. The age distribution of rare diseases was 52% for the age group 25–64 years, and 27% of cases in the age group of 0–14 years were among children. The 10 highest readmission rates ranged from 35 to 65%. Conclusions This study provided the TRDL 2017 and descriptive analysis of 281 rare diseases in a hospitalized population. Our study reveals important fundamental information that will be useful in national policy making and legislation; registry implementation; and diagnosis, treatment, and prevention of rare diseases in China. Electronic supplementary material The online version of this article (10.1186/s13023-019-1137-y) contains supplementary material, which is available to authorized users.

Background This study examined the association between interaction of retinoid-related orphan receptor alpha (RORA) (rs8042149) genotype x trauma exposure and post-traumatic stress disorder (PTSD) symptoms. Methods A total of 1196 Chinese adults who suffered from a deadly 2008 Wenchuan earthquake participated in this study. PTSD symptoms were measured with the PTSD Checklist for DSM-5 (PCL-5). A custom-by-design 2 × 48-Plex SNPscan™Kit were used to genotype the RORA rs8042149 SNP. Results Our results showed that the interaction of rs8042149 genotype x trauma exposure was associated with total PTSD symptoms in males. Moreover, the rs8042149 genotype x trauma exposure interaction was only associated with severity of the negative affect, anhedonia and dysphoric arousal symptoms in males. Limitations A moderate sample exposed to a specific event was assessed with a self-reported PTSD measure. Conclusions This study provides preliminary evidence supporting the functional role of RORA in PTSD, and adds to the knowledge for understanding the genetic underpinnings of PTSD. Moreover, this study carries implications for understanding the comorbidity of PTSD and the sex-specific expression of PTSD’s symptoms.

To investigate whether a new macular hole closure index (MHCI) could predict anatomic outcome of macular hole surgery. A vitrectomy with internal limiting membrane peeling, air-fluid exchange, and gas tamponade were performed on all patients. The postoperative anatomic status of the macular hole was defined by spectral-domain OCT. MHCI was calculated as (M+N)/BASE based on the preoperative OCT status. M and N were the curve lengths of the detached photoreceptor arms, and BASE was the length of the retinal pigment epithelial layer (RPE layer) detaching from the photoreceptors. Postoperative anatomical outcomes were divided into three grades: A (bridge-like closure), B (good closure), and C (poor closure or no closure). Correlation analysis was performed between anatomical outcomes and MHCI. Receiver operating characteristic (ROC) curves were derived for MHCI, indicating good model discrimination. ROC curves were also assessed by the area under the curve, and cut-offs were calculated. Other predictive parameters reported previously, which included the MH minimum, the MH height, the macular hole index (MHI), the diameter hole index (DHI), and the tractional hole index (THI) had been compared as well. MHCI correlated significantly with postoperative anatomical outcomes (r = 0.543, p = 0.000), but other predictive parameters did not. The areas under the curves indicated that MHCI could be used as an effective predictor of anatomical outcome. Cut-off values of 0.7 and 1.0 were obtained for MHCI from ROC curve analysis. MHCI demonstrated a better predictive effect than other parameters, both in the correlation analysis and ROC analysis. MHCI could be an easily measured and accurate predictive index for postoperative anatomical outcomes.

Background The effects of fish oil supplements on lipid profile in dialysis patients are controversial. With increasing interest in the potential health benefits of fish oil, it is important to explore its real effects. Objective We aimed to identify and quantify the effects of fish oil on triglyceride (TG), total cholesterol (TC), high-density lipoprotein cholesterol (HDL-C), and low-density lipoprotein cholesterol (LDL-C) in dialysis patients. Methods PubMed, EMBASE and the Cochrane Central Register of Controlled Trials were searched for relevant trials of fish oil and lipid profile in dialysis patients. We identified 209 potential studies and included 13 randomized controlled trials. Eligible studies, determined by consensus using predefined criteria, were reviewed in accordance with the Preferred Reporting Items for Systematic Reviews and Meta-Analyses (PRISMA) guidelines and a meta-analysis was performed. Results Compared with the control group, serum TG and TC levels in the fish oil group were reduced by 0.23 mmol/L (95% CI, −0.31, −0.14, P 12w) demonstrated a tendency towards greater improvement of serum HDL-C and LDL-C levels compared with short-term intervention (≤12 w). However, both of the changes were not statistically significant in meta-regression analysis. There were no obvious difference in effects of different doses and components of fish oil on lipid levels. Conclusion Fish oil supplements reduced serum TG and TC levels, and increased HDL-C levels, without affecting LDL-C levels among dialysis patients. It should benefit patients at risk of cardiovascular diseases. Based on randomized controlled trials, we suggested a daily supplement dose of fish oil for dialysis patients of >1 g, but a high dose might not be necessary. Electronic supplementary material The online version of this article (doi:10.1186/1476-511X-13-127) contains supplementary material, which is available to authorized users.

Stathmin (STMN1) has been demonstrated as a regulator of fear processing across species, which implicates that it may be important in the etiopathogenesis of fear-related psychiatric disorders such as posttraumatic stress disorder (PTSD). This study examined the association between STMN1 rs182455 genotype, a single nucleotide polymorphism (SNP) located within or close to the putative transcriptional control region of STMN1 gene, and PTSD symptoms. A total of 326 Chinese adults who suffered from a deadly 2008 Wenchuan earthquake and unexpectedly lost their children during the disaster participated in this study. PTSD symptoms were measured with the PTSD Checklist (PCL). The Sequenom iPlex chemistries and the MassARRAY system were used to genotype the STMN1 rs182455 SNP. Our results indicated that the STMN1rs182455 genotype was not associated with severity of total PTSD symptoms in either females or males; however, it could significantly predict severity of PTSD's reexperiencing symptoms in females. The findings provide preliminary evidence supporting the important role of STMN1 in the development of PTSD, and expand extant knowledge on the genetic underpinnings of PTSD and the sex-specific expression of PTSD's symptoms.

Importance A randomized clinical trial is needed to evaluate what is the best photodynamic therapy (PDT) protocol to use for acute central serous chorioretinopathy. Objective To compare the efficacy and safety of a 50% dose of verteporfin (a method of PDT) with the efficacy and safety of a 30% dose for acute central serous chorioretinopathy. Design, Setting, and Participants A multicenter, noninferiority, double-masked, randomized, controlled, clinical trial in which 131 patients (131 eyes) with acute central serous chorioretinopathy for less than 6 months were recruited with a follow-up of 12 months from university-based ophthalmology practices. Interventions Patients were randomly assigned to either a 50% dose of verteporfin (the 50%-dose PDT group) or a 30% dose (the 30%-dose PDT group). Main Outcomes and Measures The 2 primary outcome measures were the proportion of eyes with complete absorption of subretinal fluid and the proportion of eyes with complete disappearance of fluorescein leakage at 6 and 12 months. The secondary outcome measures included the subretinal fluid recurrent rate, the fluorescein leakage recurrent rate at 12 months, the mean best-corrected visual acuity, the retinal thickness of the foveal center, and the maximum retinal thickness at each scheduled visit. Results The noninferiority of the 30%-dose PDT compared with the 50%-dose PDT for the primary outcomes was not demonstrated. The optical coherence tomography–based improvement rate in the 30%-dose PDT group was less than that in the 50%-dose PDT group both at 6 months (73.8% vs 92.9%; α = 0.0125, P = .006) and at 12 months (75.4% vs 94.6%; α = 0.0125, P = .004). The fluorescein angiography–based improvement rate in the 30%-dose PDT group was less than that in the 50%-dose PDT group both at 6 months (68.9% vs 91.1%; α = 0.0125, P = .003) and at 12 months (68.9% vs 92.9%; α = 0.0125, P = .001). The subretinal fluid recurrence rate in the 30%-dose PDT group was greater than that in the 50%-dose PDT group (24.0% vs 5.7% at 12 months; P = .010, determined by use of the log-rank test). The fluorescein leakage recurrent rate in the 30%-dose PDT group was significantly higher than that in the 50%-dose PDT group (16.7% vs 3.8% at 12 months; P = .03, determined by use of the log-rank test). No ocular adverse event was encountered in the study. Conclusions and Relevance A 50% dose of verteporfin may be more effective at resolving subretinal fluid and fluorescein leakage, and with better visual outcomes, than a 30% dose for acute central serous chorioretinopathy. Trial Registration clinicaltrials.gov Identifier:NCT01574430