Purpose/Significance To solve the problem that regional clinical research data are difficult to integrate efficiently,and to promote"Chinese evidence"and"Chinese protocol"in the global clinical research community.Method/Process Based on the standard-ization theory,the data standardization system is proposed,and the construction and application methods of the regional clinical research data platform are explored with the integration of multi-center clinical research data as the starting point.Result/Conclusion The theo-retical framework of the regional clinical research data platform has been preliminarily established,and the clinical research capabilities of tertiary hospitals in Shanghai have been significantly improved.

Objective:To analyze the efficacy of second-line regimens and prognostic factors in patients with first-relapsed multiple myeloma (MM) treated with bortezomib, cyclophosphamide, and dexamethasone (BCD).Methods:A retrospective cohort study. Clinical data were collected in first-relapsed MM patients after BCD treatment from three tertiary hospitals in north China from July 2009 to October 2022. Patients were classified according to the second-line regimen into the immunotherapy group, single novel agent group [either proteasome inhibitor (PI) or immunomodulatory drug (IMiD)], combination treatment group (both PI+IMiD), and traditional treatment group. Responses to second-line regimens and survival data were analyzed. The Kaplan-Meier method was used for survival analysis and the Cox proportional risk model was used for univariate and multivariate analyses.Results:A total of 217 patients were enrolled including 8.8% (19/217) in the immunotherapy group, 48.4% (105/217) in the PI/IMiD group, 29.9% (65/217) in the PI+IMiD group, and 12.9% (28/217) in the traditional treatment group. The median age was 62 years (range 31-83 years) and 56.2% (122/217) were males. The overall response rates (ORRs) in the four groups were 94.7% (18/19) vs. 56.2% (59/105) vs. 73.8% (48/65) vs. 32.1% (9/28) ( χ2=24.55; P<0.001), respectively. The progression-free survival (PFS) of the second-line regimens (2ndPFS) was 17.7 vs. 9.0 vs. 9.2 vs. 4.6 months ( χ2=22.74; P<0.001), respectively, among which patients in the PI/IMiD and PI+IMiD groups had comparable 2ndPFS ( χ2=1.76; P=0.923). Patients with high-risk cytogenetic abnormalities (HRCAs) achieved the longest 2ndPFS of 22.0 months in the immunotherapy group ( χ2=15.03; P=0.002). Multivariate analysis suggested that immunotherapy ( HR=0.11, 95% CI 0.05-0.27), achievement of efficacy of partial response or better ( HR=0.47, 95% CI 0.34-0.66), and non-aggressive relapse ( HR=0.25, 95% CI 0.17-0.37) were independent prognostic factors of 2ndPFS. Conclusion:In this real-world study, immunotherapy was associated with a more favorable efficacy and PFS for first-relapsed MM patients after BCD treatment, with similar outcomes in patients with HRCAs.

The skin manifestations of monoclonal(M)-proteinemia are rare and present in patients with monoclonal gammopathy of undetermined significance (MGUS), smoldering plasma cell myeloma (SMM) and multiple myeloma (MM). In this study, we reported 4 cases with M-proteinemia-related rare skin lesions, including pyoderma gangrenosum (PG), erythema elevatum diutinum (EED), cutis laxa (CL) and lichen myxedematosus(LM). These skin lesions are specific, where the potential mechanism was immune-mediated paraneoplastic syndrome rather than direct plasma cell infiltration. Anti-plasma cell treatment was effective in treating skin lesions. The clinical outcome of MM-related skin changes was correlated to tumor control, whereas the prognosis of MGUS or SMM related skin lesions was favorable. Skin involvement in M-proteinemia is extremely rare and less well-known, which greatly impairs quality of life. The diagnosis and treatment of these 4 cases support the need for futher study.

Objective:To investigate the efficacy of using a pediatric-inspired regimen for adolescents and young adults (AYA) with Philadelphia chromosome-negative (Ph -) acute lymphoblastic leukemia/lymphoblastic lymphoma (ALL/LBL) at a single center in China. Methods:Clinical data of 71 consecutive newly diagnosed AYA patients with Ph - ALL/LBL on a pediatric-inspired regimen in Peking Union Medical College Hospital from January 2012 to November 2018 were retrospectively analyzed. Results:Median age at diagnosis was 20 years (range: 15-38) , and 46 patients (64.8%) were male. Forty-nine (69.0%) had B-ALL/LBL. Among 62 ALL patients, 22 (35.5%) were high-risk. Complete remission rate was 93.0%. At follow-up with a median time of 44 months, the estimated 5-year disease-free survival (DFS) and overall survival (OS) was 56.3% and 64.3%, respectively. There was no significant difference in 5-year OS between allogeneic hematopoietic stem cell transplantation group and the continuous chemotherapy group after completion of 4 courses of chemotherapy. The 5-year DFS and OS for the non-high-risk group was 63.1% and 73.7%, respectively, which were significantly higher than 32.0% and 44.4% for the high-risk group, respectively ( P<0.001) . Conclusions:The use of pediatric-inspired regimen for AYAs with Ph - ALL/LBL was feasible and effective.

Objective To evaluate the effect of Levosimendan on the prognosis in patients with severe coronary heart disease after operation.Methods A total of 485 severe coronary disease patients undergoing coronary artery bypass grafting from Teda International Cardiovascular Hospital and the Cardiac Surgery Department of the First Affiliated Hospital of China Medical University from May 2014 to June 2016 were enrolled.Of them,45 cases receiving Levosimendan postoperatively were assigned to the Levosimendan group,and according to propensity score matching,another 45 cases were selected as the control group in this study.Clinical data before treatment had no difference between the groups (P ＞ 0.1).Postoperative prognosis was compared between the two groups.Results There were significant differences in heart rate,mean arterial pressure,central venous pressure,cardiac output and other hemodynamic parameters between the two groups 48h after operation.The heart ultrasound results showed that the left ventricular ejection fraction(IVEF) was increased [(0.53±0.12) ％vs.(0.46±0.09)％,t =2.594,P=0.002],the postoperative ventilation time was reduced [(46.8±11.3) h vs.(58.5±16.3) h,t=-2.031,P=0.045]and the onset of bowel sounds became early [(16.5±5.9) h vs.(18.7±10.1) h,t =1.592,P=0.039]in the levosimendan group than in the control group 48h after operation.The incidences of new-onset acute kidney injury(20 ％ and 40 ％,x2 =6.702,P =0.018),new-onset postoperative atrial fibrillation (15.6％ and 44.4％,x2 =6.156,P =0.023) and perioperative myocardial infarction(11.1 ％ and 33.3％,x2 =6.429,P =0.021) had significant differences between the two groups(P＜0.05),but there was no difference in ICU retention time,1-month mortality after operation,malignant arrhythmia incidence and auxiliary equipment use (P ＞ 0.05).Conclusions Levosimendan can improve the early prognosis of severe coronary disease patients undergoing coronary artery bypass grafting and reduce the occurrence of postoperative organ dysfunction.

Objective: To Evaluate the efficacy and safety of posaconazole as primary prevention of invasive fungal disease (IFD) in patients with severe aplastic anemia (SAA) treated with anti-thymus/lymphocyte immunoglobulin (ATG/ALG) combined with cyclosporine intensive immunosuppressive therapy (IST). Methods: A retrospective analysis of clinical data of 58 SAA patients who received IST of anti-thymocyte immunoglobulin combining cyclosporine and antifungal prophylaxis during April 2013 to May 2017 in Peking Union Medical College Hospital was performed. The patients were divided into posaconazole prophylaxis group and the control group (itraconazole or fluconazole). The disease characteristics, IFD prevention effect and adverse drug reaction, curative effect and prognosis of the two groups were compared. Results: Posaconazole was used to prevent fungal infection in 20 patients. The other 38 patients were used as the control group. Retrospective analysis showed comparable characteristics (gender, age, disease severity, etiology, interval between the onset of disease to treatment, ATG/ALG type) of both groups. The incidence of IFD were 0 and 15.8% in posaconazole prophylaxis group and the control group, respectively (P=0.084). In the control group, there were 6 cases diagnosed as IFD. Of them, 2 were confirmed, 2 suspected and 2 not identified. Five of the 6 cases were pulmonary infection, 1 bloodstream infections. Of the 6 IFD cases, 5 were very severe aplastic anemia (VSAA). There was no obvious adverse reaction in posaconazole prophylaxis group. Conclusion Posaconazole is safe and effective for primary prevention of fungal infection of SAA patients receiving IST, especially for the VSAA.

Objective: To improve the understanding of the rare clinical presentation and management of purpura fulminans (PF) in patients with paroxysmal nocturnal haemoglobinuria (PNH). Methods: A case of PF occurring in PNH is reported, while the related literature review is conducted. Results: A 49-year-old male patient suffered from one-week history of fever, greenish-brown colour urine, multiple well demarcated and painful purpura of the head and neck. He had been reported to have two thromboembolic events during the 22-year course of PNH. Skin biopsy displayed classic PF features. Laboratory testing showed a high PNH clone, intravascular hemolysis and coagulation system changes. After sufficient anticoagulation and short course of glucocorticoid therapy, the clinical conditions were improved correspondingly. During a follow-up period of 6 month, there was no recurrence of thrombosis. Conclusion PF should be considered in PNH patients with unexplained, quickly developed painful purpura. Extensive work-up should be performed to find out other potential thrombophilic risk factors after diagnosis of PF. Early diagnosis, adequate anticoagulation therapy and control hemolysis were essential to PF treatment occurring in PNH. The survival of patients and the qualities of life can be improved. The PNH clone detection is needed to evaluate the status of procoagulation and predict the risk of recurrent thrombosis.

Objective To analyze the risk factors and pathogen distribution of ventilator-associated pneumonia (VAP) in patients following coronary artery bypass grafting (CABG).Methods The clinical data of 1414 patients undergoing CABG in Teda International Cardiovascular Hospital from June 2014 to June 2016 were retrospectively analyzed .The VAP developed following CABG in 42 patients ( VAP group ) and not occurred in 1372 patients ( non-VAP group) .Multivariate logistic regression was used to analyze the risk factors of VAP in patients after CABG , and the microbial culture and drug sensitivity test were performed in VAP patients.Results The incidence rate of VAP after CABG was 2.97％ (42/1414).Multivariate logistic regression analysis showed that history of smoking ( OR =2.216, 95％ CI 1.018 -4.825, P <0.05), mechanical ventilation time >48 h (OR=7.457, 95％ CI 3.443-16.161, P<0.01), LVEF<40％(OR=3.524 , 95％ CI 1.203-10.325, P<0.05) and postoperative acute kidney injury (OR=16.239, 95％ CI 7.551 -34.924, P <0.01) were independent risk factors for VAP in patients after CABG.A total of 42 pathogen strains were detected in 42 patients with VAP, including 37 strains of Gram-negative bacteria, 2 strains of Gram-positive bacteria, and 3 strains of fungus.Gram-negative bacteria mainly were Klebsiella pneumoniae subspecies ( n =23, 54.76％) and Burkholderia cepacia ( n =6, 14.27％);the Gram-positive bacteria were Staphylococcus aureus ( n =2, 4.76％);the fungus was Candida albicans ( n =3, 7.14％).Klebsiella pneumoniae was sensitive to many antibiotics;and the resistance rate to amikacin , aztreonam , meropenem , and levofloxacin was <10％, the resistance rate to ceftazidime and piperacillin was <25％. Burkholderia cepacia was naturally resistant to amikacin , ampicillin, aztreonam, cefazolin, gentamicin and sulfamethoxazole .Conclusion The incidence of VAP was higher in patients after CABG , and the involved pathogens were mainly Gram-negative bacteria .Clinically , it is necessary to take necessary measures to prevent and treat VAP in order to improve the prognosis of patients undergoing CABG .

Objective: To evaluate the clinical characteristics, MYD88^L265P mutation, CXCR4^WHIM mutation and prognosis in patients with Waldenström macroglobulinemia (WM). Methods: The clinical characteristics, International Prognostic Scoring System for symptomatic WM (WPSS) , and overall survival (OS) were retrospectively assayed in 93 patients with newly diagnosed WM at Peking Union Medical College Hospital during January 2000 to August 2016. The MYD88^L265P mutation and CXCR4^WHIM mutation were tested among 34 patients. Results: The median age of the 93 patients was 64 years (range, 33-85 years) with a male-to-female ratio of 2.44. According to WPSS, we included 16 (17.2%) low-risk, 44 (47.3%) intermediate-risk and 33 (35.5%) high-risk patients. Eight patients had secondary amyloidosis. With a median follow-up of 44 (1-201) months, the median OS was 84 months. Cox regression multifactor analysis showed WPSS risk group (HR=2.342, 95% CI 1.111-4.950, P=0.025) , whether patients had secondary amyloidosis (HR=5.538, 95% CI 1.958-15.662, P=0.001) and whether patients received new drugs (HR=3.392, 95% CI 1.531-7.513, P=0.003) were independent factors associated with OS. We have investigated the presence of the MYD88^L265P and CXCR4^WHIM mutation in 34 patients and found that MYD88^L265P mutation was occurred in 32 patients (94.1%) and CXCR4^WHIM mutation was occurred in 8 patients (23.5%). Seven of 8 patients who harbored CXCR4^WHIM-mutated also exhibited the MYD88^L265P mutation. Patients with MYD88^L265PCXCR4^WHIM vs MYD88^L265PCXCR4^WT presented with more severe anemia, lower platelet level, higher M protein level and more hyper-viscosity syndrome. Conclusion WPSS risk group, whether patients had secondary amyloidosis or received new drugs are independent factors for OS in WM. MYD88^L265P and CXCR4^WHIM mutation, the most common somatic variants in WM, often occur together and impact the clinical presentation.