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Outcomes of haploidentical bone marrow transplantation in patients with severe aplastic anemia-II thatprogressed from non-severe acquired aplastic anemia

《医学前沿（英文）》 2021年 第15卷 第5期   页码 718-727 doi: 10.1007/s11684-020-0807-4

摘要： Severe aplastic anemia II (SAA-II) progresses from non-severe aplastic anemia (NSAA). The unavailability of efficacious treatment has prompted the need for haploidentical bone marrow transplantation (haplo-BMT) in patients lacking a human leukocyte antigen (HLA)-matched donor. This study aimed to investigate the efficacy of haplo-BMT for patients with SAA-II. Twenty-two patients were included and followed up, and FLU/BU/CY/ATG was used as conditioning regimen. Among these patients, 21 were successfully engrafted, 19 of whom survived after haplo-BMT. Four patients experienced grade II–IV aGvHD, including two with grade III–IV aGvHD. Six patients experienced chronic GvHD, among whom four were mild and two were moderate. Twelve patients experienced infections during BMT. One was diagnosed with post-transplant lymphoproliferative disorder and one with probable EBV disease, and both recovered after rituximab infusion. Haplo-BMT achieved 3-year overall survival and disease-free survival rate of 86.4%±0.73% after a median follow-up of 42 months, indicating its effectiveness as a salvage therapy. These promising outcomes may support haplo-BMT as an alternative treatment strategy for patients with SAA-II lacking HLA-matched donors.

关键词： severe aplastic anemia     non-severe acquired aplastic anemia     haploidentical bone marrow transplantation     outcomes    

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Fanconi anemia gene-associated germline predisposition in aplastic anemia and hematologic malignancies

《医学前沿（英文）》 2022年 第16卷 第3期   页码 459-466 doi: 10.1007/s11684-021-0841-x

摘要： Whether Fanconi anemia (FA) heterozygotes are predisposed to bone marrow failure and hematologic neoplasm is a crucial but unsettled issue in cancer prevention and family consulting. We retrospectively analyzed rare possibly significant variations (PSVs) in the five most obligated FA genes, BRCA2, FANCA, FANCC, FANCD2, and FANCG, in 788 patients with aplastic anemia (AA) and hematologic malignancy. Sixty-eight variants were identified in 66 patients (8.38%). FANCA was the most frequently mutated gene (n = 29), followed by BRCA2 (n = 20). Compared with that of the ExAC East Asian dataset, the overall frequency of rare PSVs was higher in our cohort (P = 0.016). BRCA2 PSVs showed higher frequency in acute lymphocytic leukemia (P = 0.038), and FANCA PSVs were significantly enriched in AA and AML subgroups (P = 0.020; P = 0.008). FA-PSV-positive MDS/AML patients had a higher tumor mutation burden, higher rate of cytogenetic abnormalities, less epigenetic regulation, and fewer spliceosome gene mutations than those of FA-PSV-negative MDS/AML patients (P = 0.024, P = 0.029, P = 0.024, and P = 0.013). The overall PSV enrichment in our cohort suggests that heterozygous mutations of FA genes contribute to hematopoietic failure and leukemogenesis.

关键词： Fanconi anemia     aplastic anemia     hematologic malignancy     germline predisposition    

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with composition of lung microbiota and important taxa predicting clinical prognosis in patients with severecommunity-acquired pneumonia

《医学前沿（英文）》 2022年 第16卷 第3期   页码 389-402 doi: 10.1007/s11684-021-0856-3

摘要： Few studies have described the key features and prognostic roles of lung microbiota in patients with severe community-acquired pneumonia (SCAP). We prospectively enrolled consecutive SCAP patients admitted to ICU. Bronchoscopy was performed at bedside within 48 h of ICU admission, and 16S rRNA gene sequencing was applied to the collected bronchoalveolar lavage fluid. The primary outcome was clinical improvements defined as a decrease of 2 categories and above on a 7-category ordinal scale within 14 days following bronchoscopy. Sixty-seven patients were included. Multivariable permutational multivariate analysis of variance found that positive bacteria lab test results had the strongest independent association with lung microbiota (R2=0.033; P=0.018), followed by acute kidney injury (AKI; R2=0.032; P=0.011) and plasma MIP-1β level (R2=0.027; P=0.044). Random forest identified that the families Prevotellaceae, Moraxellaceae, and Staphylococcaceae were the biomarkers related to the positive bacteria lab test results. Multivariable Cox regression showed that the increase in α-diversity and the abundance of the families Prevotellaceae and Actinomycetaceae were associated with clinical improvements. The positive bacteria lab test results, AKI, and plasma MIP-1β level were associated with patients’ lung microbiota composition on ICU admission. The families Prevotellaceae and Actinomycetaceae on admission predicted clinical improvements.

关键词： severe community-acquired pneumonia     lung microbiota     clinical improvements     7-category ordinal scale     Prevotellaceae    

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A randomized, controlled, open label non-inferiority trial of intravenous ferric carboxymaltose versusiron sucrose in patients with iron deficiency anemia in China

《医学前沿（英文）》 doi: 10.1007/s11684-023-1001-2

摘要： Iron deficiency (ID) and ID anemia (IDA) pose significant public health concerns in China. Although iron sucrose (IS) treatment is well-established in the country, ferric carboxymaltose (FCM) offers the advantage of higher doses and fewer infusions. This open label, randomized, controlled, non-inferiority trial was conducted at multiple sites in China to compare the outcomes of FCM (maximum of 2 doses, 500 or 1000 mg iron) and IS (up to 11 infusions, 200 mg iron) treatments in subjects with IDA. The primary endpoint was the achievement of hemoglobin (Hb) response (an increase of ≥2 g/dL from baseline) within 8 weeks, whereas secondary endpoints included changes in Hb, transferrin saturation, and serum ferritin levels. Among the 371 randomized subjects, a similar percentage of subjects treated with FCM and IS achieved Hb-response (FCM 99.4%, IS 98.3%), thereby confirming the non-inferiority of FCM compared with IS (difference 1.12 (−2.15, 4.71; 95% confidence interval (CI))). Furthermore, a significantly higher proportion of FCM-treated subjects achieved early Hb-response at Week 2 (FCM 85.2%, IS 73.2%; difference 12.1 (3.31, 20.65; 95% CI)). Additionally, the increase in TSAT and serum ferritin levels from baseline was significantly greater at all time points for FCM-treated subjects. The safety profiles of FCM and IS were comparable, with the exception of transient hypophosphatemia and pyrexia, which are consistent with FCM’s known safety profile. In conclusion, FCM proves to be an efficacious treatment for IDA, providing faster Hb-response and correction of ID with fewer administrations than IS.

关键词： iron deficiency     anemia     intravenous iron     ferric carboxymaltose     iron sucrose     Hb response     early response    

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Cross-sectional prevalence and pattern of non-anaemia severe malaria among 2–10 year olds in Sokoto in

《医学前沿（英文）》 2022年 第16卷 第6期   页码 969-974 doi: 10.1007/s11684-021-0912-z

摘要： Malaria is highly prevalent in Nigeria and accounts for approximately 40% of global malaria mortality. However, most reports on severe malaria in Nigeria are from hospital-based studies without accurate information from communities; thus, malaria-related deaths in the community are left untracked. This study aimed to describe the prevalence and pattern of severe malaria in a community in Northwestern Nigeria. A cross-sectional study was conducted among 2–10-year-old children in Sokoto, in August and December 2016, to determine the endemicity of malaria based on Plasmodium falciparum prevalence rate (PfPR2-10) and to describe the disease pattern. Severe malaria was diagnosed according to the World Health Organisation criteria. Data were described using Stata version 15. The prevalence of non-anaemia severe malaria was higher than expected (2.6%), considering the endemicity pattern which was mesoendemic based on a PfPR2-10 of 34.8%. The mean age of children with severe malaria was 3.73 years, and the male–female ratio was 2:1. However, 54.0% of the patients had hyperparasitaemia. A relatively high prevalence of non-anaemia severe malaria was found in Wamakko. This finding suggests the need to identify and treat cases in the community using modifications of current strategies, particularly seasonal malaria chemoprophylaxis.

关键词： severe malaria     nigeria     malaria mortality     PfPR2-10     intermediate malaria transmission    

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Targeting apoptosis to manage acquired resistance to third generation EGFR inhibitors

《医学前沿（英文）》 2022年 第16卷 第5期   页码 701-713 doi: 10.1007/s11684-022-0951-0

摘要： A significant clinical challenge in lung cancer treatment is management of the inevitable acquired resistance to third-generation epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (EGFR-TKIs), such as osimertinib, which have shown remarkable success in the treatment of advanced NSCLC with EGFR activating mutations, in order to achieve maximal response duration or treatment remission. Apoptosis is a major type of programmed cell death tightly associated with cancer development and treatment. Evasion of apoptosis is considered a key hallmark of cancer and acquisition of apoptosis resistance is accordingly a key mechanism of drug acquired resistance in cancer therapy. It has been clearly shown that effective induction of apoptosis is a key mechanism for third generation EGFR-TKIs, particularly osimertinib, to exert their therapeutic efficacies and the development of resistance to apoptosis is tightly associated with the emergence of acquired resistance. Hence, restoration of cell sensitivity to undergo apoptosis using various means promises an effective strategy for the management of acquired resistance to third generation EGFR-TKIs.

关键词： acquired resistance     EGFR inhibitor     apoptosis     lung cancer    

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Analysis of antibiotic usage for viral community-acquired pneumonia in adults

Rongmeng Jiang, Bing Han, Chang Dou, Fei Zhou, Bin Cao, Xingwang Li

《医学前沿（英文）》 2021年 第15卷 第1期   页码 139-143 doi: 10.1007/s11684-019-0736-2

摘要： The rationale for the antibiotic treatment of viral community-acquired pneumonia (CAP) in adults was analyzed to develop a clinical reference standard for this condition. Clinical data from 166 patients diagnosed with viral pneumonia across 14 hospitals in Beijing from November 2010 to December 2017 were collected. The indications for medications were evaluated, and the rationale for the use of antibiotics was analyzed. A total of 163 (98.3%) patients with viral pneumonia were treated with antibiotics. A combination of C-reactive protein (CRP) and procalcitonin (PCT) was used as markers to analyze the possible indications for antibiotic use. With threshold levels set at 0.25 µg/L for PCT and 20 mg/L for CRP, the rate of unreasonable use of antibiotics was 55.2%. By contrast, at a CRP level threshold of 60 mg/L, the rate of antibiotic misuse was 77.3%. A total of 39 of the 163 (23.9%) patients did not meet the guidelines for drug selection for viral CAP in adults. The unreasonable use of antibacterial drugs for the treatment of viral CAP in adults is a serious concern. Clinicians must reduce the unnecessary use of antibiotics.

关键词： adult     antibiotic     viral pneumonia    

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Treatment of severe acute pancreatitis through retroperitoneal laparoscopic drainage

Chun Tang, Baolin Wang, Bing Xie, Hongming Liu, Ping Chen

《医学前沿（英文）》 2011年 第5卷 第3期   页码 302-305 doi: 10.1007/s11684-011-0145-7

摘要： A treatment method based on drainage via retroperitoneal laparoscopy was adopted for 15 severe acute pancreatitis (SAP) patients to investigate the feasibility of the method. Ten patients received only drainage via retroperitoneal laparoscopy, four patients received drainage via both retroperitoneal and preperitoneal laparoscopy, and one patient received drainage via conversion to laparotomy. Thirteen patients exhibited a good drainage effect and were successfully cured without any other surgical treatment. Two patients had encapsulated effusions or pancreatic pseudocysts after surgery, but were successfully cured after lavage and B ultrasound-guided percutaneous catheter drainage. SAP treatment via retroperitoneal laparoscopic drainage is an effective surgical method, resulting in minor injury.

关键词： severe acute pancreatitis (SAP)     laparoscope     retroperitoneal drainage     treatment    

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Experience gained in analyzing severe accidents for WWER RP using CC SOCRAT

《能源前沿（英文）》 2021年 第15卷 第4期   页码 872-886 doi: 10.1007/s11708-021-0796-2

摘要： The current Russian regulatory documents on the safety of nuclear power plant (NPP) specify the requirements regarding design basis accidents (DBAs) and beyond design basis accidents (BDBAs), including severe accidents (SAs) with core meltdown, in NPP design (NP-001-15, NP-082-07, and others). For a rigorous calculational justification of BDBAs and SAs, it is necessary to develop an integral CC that will be in line with the requirements of regulatory documents on verification and certification (RD-03-33-2008, RD-03-34-2000) and will allow for determining the amount of data required to provide information within the scope stipulated by the requirements for the structure of the safety analysis report (SAR) (NP-006-16). The system of codes for realistic analysis of severe accidents (SOCRAT) (formerly, thermohydraulics (RATEG)/coupled physical and chemical processes (SVECHA)/behavior of core materials relocated into the reactor lower plenum (HEFEST)) was developed in Russia to analyze a wide range of SAs at NPP with water-cooled water-moderated power-generating reactor (WWER) at all stages of the accident. Enhancements to the code and broadening of its applicability are continually being pursued by the code developers (Nuclear Safety Institute of the Russian Academy of Sciences (IBRAE RAN)) with OKB Gidropress JSC and other organizations. Currently, the SOCRAT/1 code can be used as a base tool to obtain realistic estimates for all parameters important for computational justification of the reactor plant (RP) safety at the in-vessel stage of SAs with fuel melting. To perform analyses using CC SOCRAT/1, the experience gained during execution of thermohydraulic codes is applied, which allows for minimizing the uncertainties in the results at the early stage of an accident scenario. This study presents the results of the work performed in 2010–2020 in OKB Gidropress JSC using the CC SOCRAT/1. Approaches have been considered to develop calculational models and analyze SAs using CC SOCRAT. This process, which is clearly structured in OKB Gidropress JSC, provides a noticeable reduction in human involvement, and reduces the probability of erroneous results.

关键词： system of codes for realistic analysis of severe accidents (SOCRAT)     design basis accidents (DBAs)     severe accidents (SAs)     computer code (CC)     nuclear power plant (NPP) design     water-cooled water-moderated (WWER)     modeling     model     safety requirements    

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Clinical manifestations and outcomes in severe ulcerative colitis

YANG Xuesong, YAO Wei, LIU Wenbin, LI Jun, LU Yumin

《医学前沿（英文）》 2007年 第1卷 第2期   页码 192-195 doi: 10.1007/s11684-007-0036-0

摘要： In order to evaluate the clinical manifestations and outcomes of severe ulcerative colitis (UC), we retrospectively reviewed 41 patients with severe UC from 144 consecutively hospitalized UC cases from 1988 to 2004. Data recorded included onset, symptoms, signs, laboratory results, endoscopic, radiologic and pathologic findings, the clinical treatment process and follow-up. Of these severe cases, 92.7% (38/41) had pancolitis. Clinically, 36.9% (15/41) were categorized as first onset type, 36.9% (15/41) were chronic persistent and 26.8% (11/41) were chronic recurrent. Steroids played a main role in the remission of severe UC (61.0%). Thirty-one cases (75.6%) were relieved by drug therapy. Seven cases (17.1%) progressed to the need for operation. An early age of onset, pancolitis, low hemoglobin and serum albumin levels, and the need for intravenous steroids tended to be associated with the need for surgery. In conclusion, most of the severe UC patients respond well to drug therapy, but for individuals who are unresponsive to drug therapy, or for those depending on steroids, after a reasonable duration of treatment, the necessity for surgery should be considered.
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突发性灾害天气的结构预测与应急对策

欧阳首承,谢娜,郝丽萍

《中国工程科学》 2005年 第7卷 第9期   页码 9-13

摘要：

以V-3θ图的非规则信息结构分别分析了北京2004年7月10日和上海7月12日出现的突发性强对流降水、风暴和川东北的区域性特大暴雨天气过程的结构特征，并简单地讨论了突发灾害天气的预测原理和分析、预测方法。结果表明，局地强对流降水天气或区域性大暴雨在V-3θ图的结构上事先有明显的征兆而可预测；并作为有效的防灾、减灾和相应城市建设应考虑的排水、储水和防风等的特殊性应急措施。

关键词： 溃变     结构分析     突发性强对流     防灾措施    

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Diagnosis and management of acquired thrombotic thrombocytopenic purpura in southeast China: a single

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《医学前沿（英文）》 2016年 第10卷 第4期   页码 430-436 doi: 10.1007/s11684-016-0492-5

摘要：

Acquired thrombotic thrombocytopenic purpura (TTP) is a rare life-threatening thrombotic microangiopathy. This study aimed to provide a profile of the diagnosis and management of patients with acquired TTP collected in 10 years in a single center in southeast China. A total of 60 patients diagnosed with acute acquired TTP from March 2005 to August 2015 were enrolled. Among the 60 patients, 52 patients presented with their first episodes, and eight patients had two or more episodes. The median age at presentation was 49 (range, 17 to 78) years with a female predominance (male:female ratio, 1:1.60). ADAMTS 13 activity were analyzed in 43 patients, among whom 33 (76.7%) patients had a baseline level of<5%. Mortality was 30%. Plasma exchange (PEX) was performed in 62 of 69 (89.9%) episodes. Corticosteroids were administered in 54 of 69 (78.3%) episodes. Other immunosuppressants (e.g., vincristine, cyclosporine, and cyclosporin) were used in 7 of 69 (10.1%) episodes. Rituximab was documented in 4 patients with refractory/relapsed TTP for 5 episodes, showing encouraging results. In conclusion, the diagnosis of TTP depended on a comprehensive analysis of clinical data. Plasma ADAMTS13 activity assay helped confirm a diagnosis. PEX was the mainstay of the therapy, and rituximab can be used in relapsed/refractory disease.

关键词： thrombotic thrombocytopenic purpura     ADAMTS 13     plasma exchange    

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A pilot study on Paxlovid therapy for hemodialysis patients with severe acute respiratory syndrome coronavirus

《医学前沿（英文）》 doi: 10.1007/s11684-023-1011-0

摘要： We aimed to investigate the safety and efficacy of nirmatrelvir/ritonavir (Paxlovid) therapy for hemodialysis-dependent patients with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection. Thirteen hemodialysis patients infected with the Omicron variant of SARS-CoV-2 from April 3 to May 30, 2022, were recruited. Laboratory parameters and chest CT (computed tomography) imaging were analyzed. The treatment group included six patients who received 150 mg/100 mg of Paxlovid orally once daily for 5 days, whereas the control group included seven patients who received basic treatment. No serious adverse reactions or safety events were recorded. Four control patients progressed to moderate disease, and none in the treatment group showed progression of chest CT findings (P < 0.05). Paxlovid therapy tended toward early viral clearance and low viral load on Day 8. Moreover, 83.3% of the patients in the treatment group and 57.1% of the patients in the control group turned negative within 22 days. In the Paxlovid treatment group, we found significantly increased levels of lymphocytes (P=0.03) and eosinophils (P=0.02) and decreased levels of D-dimer on Day 8 compared with those on Day 1. Paxlovid therapy showed a potential therapeutic effect with good tolerance in hemodialysis patients. The optimal dose and effectiveness evaluation must be further investigated in a largeer cohort.

关键词： Paxlovid     hemodialysis     SARS-CoV-2     viral load     chest CT scan    

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Arsenic trioxide induced rhabdomyolysis, a rare but severe side effect, in an APL patient: a case report

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《医学前沿（英文）》 2017年 第11卷 第2期   页码 284-286 doi: 10.1007/s11684-017-0514-y

摘要：

Arsenic trioxide (ATO), a component of the traditional Chinese medicine arsenic sublimate, promotes apoptosis and induces leukemic cell differentiation. Combined with all-trans-retinotic acid (ATRA), ATO has become the first-line induction therapy in treating acute promyelocytic leukemia (APL). The most common side effects of ATO include hepatotoxicity, gastrointestinal symptoms, water-sodium retention, and nervous system damage. In this report, we present a rare side effect, rhabdomyolysis, in a 68-year-old female APL patient who was treated with ATO. After taking 10 mg ATO daily for 6 days, she presented shortness of breath, myodynia, elevated creatine kinase, and acute renal insufficiency. This report describes the first case of ATO-induced rhabdomyolysis.

关键词： arsenic trioxide     APL     rhabdomyolysis    

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Outcome of Stretta radiofrequency and fundoplication for GERD-related severe asthmatic symptoms

null

《医学前沿（英文）》 2015年 第9卷 第4期   页码 437-443 doi: 10.1007/s11684-015-0422-y

摘要：

This study aimed to investigate the outcome of treatment with Stretta radiofrequency (SRF) or laparoscopic Nissen fundoplication (LNF). A total of 137 gastroesophageal reflux disease (GERD) patients with severe asthmatic symptoms who responded inadequately to medical treatment for asthma were investigated. The patients were followed up 1 year and 5 years after SRF (n = 82) or LNF (n = 55) treatment. A questionnaire covering 29 related symptoms and medication use was employed. Digestive, respiratory, and ear-nose-throat (ENT) symptom scores significantly decreased after antireflux treatment. Symptom scores respectively changed from 17.2±10.1, 31.9±6.6, and 21.1±11.8 to 5.0±6.2, 11.5±10.2, and 6.3±6.8 at 1 year and to 5.6±6.5, 13.1±10.1, and 7.8±7.2 at 5 years (P <0.001). The outcome of LNF was significantly better than that of SRF in terms of digestive (P <0.001, P = 0.001), respiratory (P = 0.006, P = 0.001), and ENT symptoms (P = 0.006, P = 0.003) at both 1 year and 5 years. SRF and LNF were both effective against the digestive symptoms of GERD as well as GERD-related severe asthmatic and ENT symptoms, with better outcomes exhibited by the LNF group. Severe asthmatic symptoms and GERD were closely associated, and this finding warrants further study.

关键词： asthma     gastroesophageal reflux     Stretta radiofrequency     laparoscopic Nissen fundoplication    

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标题 作者 时间 类型 操作

Outcomes of haploidentical bone marrow transplantation in patients with severe aplastic anemia-II thatprogressed from non-severe acquired aplastic anemia

期刊论文

Fanconi anemia gene-associated germline predisposition in aplastic anemia and hematologic malignancies

期刊论文

with composition of lung microbiota and important taxa predicting clinical prognosis in patients with severecommunity-acquired pneumonia

期刊论文

A randomized, controlled, open label non-inferiority trial of intravenous ferric carboxymaltose versusiron sucrose in patients with iron deficiency anemia in China

期刊论文

Cross-sectional prevalence and pattern of non-anaemia severe malaria among 2–10 year olds in Sokoto in

期刊论文

Targeting apoptosis to manage acquired resistance to third generation EGFR inhibitors

期刊论文

Analysis of antibiotic usage for viral community-acquired pneumonia in adults

Rongmeng Jiang, Bing Han, Chang Dou, Fei Zhou, Bin Cao, Xingwang Li

期刊论文

Treatment of severe acute pancreatitis through retroperitoneal laparoscopic drainage

Chun Tang, Baolin Wang, Bing Xie, Hongming Liu, Ping Chen

期刊论文

Experience gained in analyzing severe accidents for WWER RP using CC SOCRAT

期刊论文

Clinical manifestations and outcomes in severe ulcerative colitis

YANG Xuesong, YAO Wei, LIU Wenbin, LI Jun, LU Yumin

期刊论文

突发性灾害天气的结构预测与应急对策

欧阳首承,谢娜,郝丽萍

期刊论文

Diagnosis and management of acquired thrombotic thrombocytopenic purpura in southeast China: a single

null

期刊论文

A pilot study on Paxlovid therapy for hemodialysis patients with severe acute respiratory syndrome coronavirus

期刊论文

Arsenic trioxide induced rhabdomyolysis, a rare but severe side effect, in an APL patient: a case report

null

期刊论文

Outcome of Stretta radiofrequency and fundoplication for GERD-related severe asthmatic symptoms

null

期刊论文