Fabry disease (FD) is a rare X-linked inherited lysosomal storage disorder caused by variants in the GLA gene, which encodes α-galactosidase A (α-Gal A). These mutations lead to reduced or absent α-Gal A activity, resulting in the accumulation of its substrates in various organs and tissues. This accumulation causes multi-system damage and can be life-threatening. This article reviews the current landscape of various treatment strategies for FD, including specific therapies (such as enzyme replacement therapy, chaperone therapy, substrate reduction therapy, and gene therapy), symptomatic management, as well as dietary and exercise interventions, aiming to provide a reference for the clinical management of FD.

Objective To investigate the correlation and consistency between the parameters of thromboelastography(TEG)and routine coagulation tests,and to evaluate the application value of the two methods in heparin anticoagulation monitoring and coagulation function monitoring in patients receiving extracorporeal membrane oxygenation(ECMO)therapy.Methods A total of 138 patients who recieved ECMO in the Department of Critical Care Medicine of the People's Hospital of Guangxi Zhuang Autonomous Region from October 2021 to December 2022 were selected.A total of 317 pairs of ordinary TEG and heparinase-modified TEG(hmTEG)parameters measured simultaneously were analyzed for correlation and consis-tency with activated partial thromboplastin time(APTT),fibrinogen(Fib),and platelet count(Plt),and the parameters tested when ECMO was established and 24 hours after ECMO operation were compared.Results The correlation coefficient between R values and APTT of hmTEG(r=0.441,P<0.05)was lower than that of ordinary TEG(r=0.547,P<0.05).The parameters α-Angle and K value of ordinary TEG were not correlated with Fib(P>0.05),while as for hmTEG,the correla-tion was 0.359(P<0.05)and-0.343(P<0.05),respectively.The correlation between MA value of hmTEG and Plt was 0.456(P<0.05),which was much lower than its correlation with Fib(r=0.715,P<0.05).APTT and hmTEG had moderate agreement in judging the anticoagulant effect of UFH(P<0.05).Plt at 24 hours after ECMO was significantly lower than that at establishment of ECMO(P<0.05).Fib,APTT and hmTEG parameters were not significantly different between the two groups(P>0.05).Conclusion The parameters of hmTEG can better reflect the real level of coagulation factors in patients receiving ECMO.The results of hmTEG and APTT are complementary to assess whether heparin in ECMO patients is over-dosed,and hmTEG has unique advantages.Routine coagulation tests and TEG cannot replace each other,and the combina-tion of them can achieve better anticoagulation and coagulation management.

【Objective】 To explore the model of treatment of refractory wounds by apheresis platelet-rich plasma (PRP), and evaluate its efficacy and safety. 【Methods】 PRP treatment was carried out for 34 patients with refractory wounds who were hospitalized in our hospital from June 2020 to December 2023. The patient′s autologous PRP was collected by the blood composition separator. PRP point-like injection and/or direct application along the edge of the wound, or platelet gel (PG) to cover the wound and fill the sinus were used to treat different types of wounds. The frequency of treatment was once to twice a week, with each course of treatment consisting of 4 to 5 sessions. The efficacy was observed and recorded, and the safety was evaluated. 【Results】 Before PRP collection, the basal platelet, white blood cell and red blood cell counts were (321.85±114.64) ×10⁹/L, (9.52±3.21) ×10⁹/L and (4.34±0.62) ×10¹²/L, respectively. The counts of platelets, white blood cells and red blood cells in PRP products were (1 438.53±376.89)×10⁹/L, (1.38±1.03)×10⁹/L and (0.03±0.01)×10¹²/L, respectively. The platelet concentration of PRP products increased by 4.47 times on average, and the sampled bacterial culture was negative. Out of 34 patients treated with PRP, 33 showed improvement in symptoms, while 1 did not respond well due to long-term bedridden and poor compliance. Four patients had mild adverse reactions during PRP collection or treatment, but all were able to complete PRP collection or treatment after standardized treatment. 【Conclusion】 Apheresis PRP products have the advantages of stable quality, safety, reliability and traceability, and can effectively promote the healing of a variety of refractory wounds. Its treatment process is safe and effective, and is worthy of popularization and application.

AIM: To compare the effects of different doses of alfentanil on tracheal intubation conditions, hemodynamic parameters and recovery quality in children undergoing tonsillectomy. METHODS: Ninety children undergoing tonsillectomy were randomly divided into 3 groups, and received alfentanil 20 μg/kg (A20 group), 40 μg/kg (A40 group) and 60 μg/kg (A60 group) for anesthesiainduction respectively, 30 cases in each group. The remaining anesthesia induction and maintenance protocols were the same. The Helbo-Hansen scores of the three groups were evaluated, and the MAP and HR before anesthesia induction (T0), before tracheal intubation (T1), immediately after tracheal intubation (T2), and 1 min after intubation (T3) as well as the recovery time of spontaneous breathing, eye opening time, extubated time, agitation score in PACU, and adverse drug reactions were recorded. RESULTS: Compared with A20 group, the total values of Helbo-Hansen score and cough scores in group A40 and A60 were lower (P<0.05). Compared with T0, the MAP at T1-T3 were decreased in group A40 and A60, and HR increased at T2 and T3 in group A20 while HR slowed down at T1 in group A40, and at T1-T3 in group A60 (P<0.05). Compared with A20 group, children in group A40 had lower MAP and slower HR at T1-T3, while those in group A60 had lower MAP and slower HR at T1-T3 (P<0.05). The recovery time of spontaneous breathe and extubated time were prolonged in group A60 (P<0.05). CONCLUSION: During the anesthesia induction period of tonsillectomy in children, both afentanil 40 μg/kg or 60 μg/kg combined with propofol 3 mg/kg and rocuronium 0.3 mg/kg can provide satisfactory intubation condition, while the vital signs are more stable during anesthesia induction in afentanil 40 μg/kg group and rapid extubation after operation can be achieved.

BACKGROUND: The clinical trials of new anti-tumor drugs are prospering in China. The acceptance of clinical trials in patients is an important factor affecting the speed and quality of clinical trials. Previous studies have investigated the acceptance of clinical trials in those cancer patients, who have never participated in a trial. This study is designed to investigate and compare the acceptance and related causes of clinical trials in cancer patients who have once participated in a clinical trial or not. METHODS: From June 2018 to April 2019, a standardized questionnaire-based survey was conducted among two groups of cancer patients classified by history of clinical trial participation in Cancer hospital, Chinese Academy of Medical Science, mainly focusing on their overall acceptance of clinical trials and related considerations, including the role of attending doctors, as well as group differences between the two participants. RESULTS: A total of 538 patients were enrolled with an average age of 53.5 years old, 51.1% of whom were males, and 43.3% of whom have never participated in a clinical trial. Overall, 502 patients (93.3%) were willing to or recommend their relatives or friends to participate in clinical trials, and patients with history of clinical trial participation had higher willingness (96.6% vs 90.8%, P=0.008). Patients were most likely to be motivated by expectation of optimal treatment (100.0% vs 99.3%) for both those who had once participated in a clinical trial or those not, respectively followed by financial burden reduction (56.0%) and recommendation by attending doctor (43.7%). The main reasons for unwillingness-to-participate for those who had once participated in a clinical trial were abandoning other treatment options, divided into control group or additional visits, while for those who had never participated in a clinical trial, ineffective treatment or serious adverse reactions were their main concerns. In the decision-making of clinical trial participation, 88% patients highly valued the role of recommendation by attending doctors. Among patients without trial participation history, 60.9% of those had no unwillingness-to-participate expressed that recommendation by attending doctors would change their decisions. The study also reported patients' preferences for information and access to clinical trials. CONCLUSIONS The acceptance of clinical trials in cancer patients in our hospital is generally high, especially in patients who had a history of trial participation. It's of substantial significance to give full play to the role of doctors in improving the acceptance of clinical trials of cancer patients in China.

BACKGROUND: Early investigation suggested patients' level of awareness regarding clinical trials was related with willingness to participation. This study was intended to evaluate the level of awareness of cancer patients regarding clinical trials and related influencing factors, and to compare the differences of awareness between patients who attended clinical trials before and not. METHODS: From Jun, 2018 to April, 2019, standardized question-naires were gathered from cancer patients (attended clinical trials vs not attended clinical trials) in our hospital regarding basic information and 10 other questions about awareness. The level of awareness was evaluated and patients were classified into "low cognition" and "high cognition" groups. Logistic regression analysis was performed to determine whether certain characteristics would predict for awareness. RESULTS: Of the 617 participants, 38.6% have attended clinical trials before. 338 (54.6%) patients had a correct overall understanding of clinical trials, while 44 (7.1%) patients still thought participants were the victim of scientific research. Except for the compensation of medical expenses (51.5% vs 48.7%) and related laws of clinical trials (52.3% vs 45.5%), other parts of understanding were elevated in patients attended clinical trials before comparing with patients who didn't, including significance (86.2% vs 77.6%), risk disclosure (91.2% vs 71.6%), confidentiality (73.2% vs 59.7%), voluntariness (95.8% vs 76.3%), withdrawal (86.6% vs 68.2%) and expenses (62.8% vs 39.2%). The proportion of participants who understand these components did not increase even in 239 patients who had attended clinical trials before. Participants who attended clinical trials before (OR=1.83, 95%CI: 1.11-3.00), unmarried/divorced (OR=5.04, 95%CI: 1.73-14.66), retired (OR=2.53, 95%CI: 1.16-5.50) had a higher level of awareness, while patients who had bad impression with doctors (OR=0.43, 95%CI: 0.26-0.72) had lower awareness. CONCLUSIONS The current level of awareness for clinical trials of cancer patients in our hospital was relatively low, even in patients who had attended clinical trials before. It's necessary to improve patients' awareness of clinical trial by promoting harmony relationship between patients and doctors, as well as by enhancing related propagation. Strengthening the adequacy and efficacy of informed consent in clinical trials also needs to be achieved in the future.

Objective? To explore the effects of fast track surgery (FTS) nursing on fatigue and quality of life among hepatocellular carcinoma patients. Methods? From June 2017 to June 2018, we selected 80 primary hepatic carcinoma patients with hepatocellular carcinoma resection of hepatobiliary surgery at Peking Union Medical College Cancer Hospital as subjects by convenience sampling. All of the patients were divided into control group and observation group with the method of random number table, 40 cases in each group. Observation group carried out FTS, while control group adopted routine nursing. The intervention effects were compared with the Cancer Fatigue Scale (CFS) and the Quality of Life Questionnaire Core 30 (QLQ-C30). Results? Before intervention, there was no statistical difference in the score of CFS between two groups (P＞0.05). After intervention, the total score and dimension scores of CRF of observation group were lower than those of control group with statistical differences (P＜ 0.01). Before intervention, there was no statistical difference in the score of QLQ-C30 between two groups (P＞ 0.05). After intervention, the total score of QLQ-C30 of observation group was higher than that of control group with a statistical difference (P＜ 0.01). Conclusions? FTS can effectively improve the postoperative fatigue of hepatocellular carcinoma patients and quality of life of patients which is worthy of clinical popularization and application.

ObjectiveTo evaluate the efficacy of problem-based learning (PBL) and case-based study(CBS)in health education for patients with hepatocellular carcinoma treated by transcatheter arterial chemoembolization(TACE). MethodsUsing convenience sampling method, 200 patients with hepatocellular carcinoma treated by TACE in the Department of Invasive Treatment, Cancer Hospital, Chinese Academy of Medical Sciences, Peking Union Medical College from October 2017 to August 2018 were selected as the study subjects. According to the admission time, they were divided into control group and intervention group with 100 cases each. The control group was given routine health education, while the intervention group was given PBL combined with CBS health education on the basis of routine health education. Anxiety level and mastery level of education contents were compared between the two groups. ResultsThere was no significant difference in level of anxiety between the two groups at admission (P＞ 0.05). When discharged from hospital, compared with the control group, the anxiety level of intervention group was lower and the mastery of education contents was higher, and the differences were statistically significant (P＜ 0.05). ConclusionsPBL combined with CBS in the health education for patients with liver HCC treated with TACE embodied the individualized nursing for patients and can effectively reduce the patients' anxiety degree and improve their mastery level towards the relevant knowledge, hence improve their quality of life as well as the disease outcome and recovery.

Objective To investigate the effect of comprehensive nursing for pain,quality of life and satisfaction of hepatocellular carcinoma (HCC) patients after TACE.Methods A total of 180 patients with HCC treated with TACE were enrolled.The patients were equally divided into intervention group and control group (each n＝ 90).Comprehensive nursing was performed,including routine nursing care,psychological support,painful health education,nursing interventions with relaxation therapies and pressing acupoints combined with drug analgesia in intervention group.The control group was given routine nursing care.The numerical rating scale (NRS) scores for pain assessment,satisfaction and quality of life were compared between the two groups.Results NRS scores at the 1st,2nd,3rd day after operation in the intervention group at resting state and exercise state were all lower than those in the control group (all P＜0.05).The satisfaction rate of the intervention group (94.11％ [85/90]) was higher than that of the control group (76.67％ [69/90];X2 ＝63.47,P＜0.05).The quality of life in intervention group was also significantly higher than that in control group (X2 ＝7.476,P＜0.05).Conclusion In HCC patients,comprehensive nursing can effectively reduce the pain from TACE and improve the satisfaction and quality of life.

Objective To analyze the characteristics of life expectancy and influencing factors in Chinese population in different areas and provide scientific evidence for policy-making on disease managements,medical care and risk factor intervention.Methods Based on the national census data from National Bureau of Statistics and the death registration data from the National Health and Family Planning Commission,we used exponential model,under-report adjustment model and abbreviated life tables to estimate the life expectancy and influence on disease in Chinese population in 2013.Results The Chinese life expectancy was 75.8 years in 2013,1 year higher than that in 2010.The life expectancy in urban area was 77.4 years,while it was 75.1 years that in rural area with the gap between the rural area and urban area was 2.3 years.The life expectancy was 77.2 years in the eastern area,75.8 years in middle area and 73.5 years in western area,the gap between the east and west was 3.6 years.In 2013,the first 10 leading diseases causing the life expectancy lost were cerebrovascular disease,ischemic heart disease,chronic obstructive pulmonary disease,lung cancer,road injury,liver cancer,stomach cancer,hypertensive heart disease,lower respiratory infection,esophagus cancer,resulting in 7.97 years of life expectancy lost.Conclusion The life expectancy in Chinese has already reached a relative high level,while the gap between different areas still exists.Different policies on disease management,medical care and risk factor interventions targeting different areas are needed to increase the life expectancy and improve the quality of life.