1.Venetoclax in the treatment of non-M3 acute myeloid leukemia:a rapid health technology assessment
Yazhuo ZHANG ; Huiling LI ; Yinyin DUAN ; Yuye SHI
Chinese Journal of Pharmacoepidemiology 2025;34(3):314-323
Objective To rapidly evaluate the efficacy,safety,and cost-effectiveness of venetoclax(Ven)in non-M3 acute myeloid leukemia(AML),and to provide an evidence-based basis for rational clinical treatment.Methods PubMed,Cochrane Library,Embase,CNKI,WanFang Data databases,and relevant health technology assessment(HTA)websites were searched to collect relevant literature and reports on Ven treatment for non-M3 AML,with a search timeframe from the establishment of the database/website to November 1st,2024.Two researchers independently screened literature,extracted data,and assessed quality,and then qualitatively described and analyzed the results.Results A total of 11 pieces of literature were included,including 5 systematic reviews/Meta-analysis,4 pharmacoeconomic studies,and 2 HTA reports.In terms of efficacy,compared with the control group,non-M3 AML patients receiving Ven treatment had a higher clinical remission rate(P<0.05),a longer event-free survival(EFS)(P<0.05)and a similar or longer overall survival(OS)(P<0.05).Regarding safety,compared to Azacitidine(Aza)monotherapy,Ven+Aza resulted in a higher likelihood of febrile neutropenia in non-M3 AML patients(P<0.05).Non-M3 AML patients receiving Ven+low-dose cytarabine(LDAC)had a higher risk of developing thrombocytopenia compared with LDAC monotherapy(P<0.05).However,the early 30-day mortality rate was lower in the Ven+chemotherapy group than that in the chemotherapy alone group(P<0.05),presenting an acceptable security profile overall.In terms of cost-effectiveness,Ven was cost-effective in non-M3 AML patients compared with the control group.Conclusion Ven has manifested remarkable efficacy and acceptable security profile among patients with non-M3 AML,thus proving to be a medium to long-term cost-effective treatment modality.
2.Venetoclax in the treatment of non-M3 acute myeloid leukemia:a rapid health technology assessment
Yazhuo ZHANG ; Huiling LI ; Yinyin DUAN ; Yuye SHI
Chinese Journal of Pharmacoepidemiology 2025;34(3):314-323
Objective To rapidly evaluate the efficacy,safety,and cost-effectiveness of venetoclax(Ven)in non-M3 acute myeloid leukemia(AML),and to provide an evidence-based basis for rational clinical treatment.Methods PubMed,Cochrane Library,Embase,CNKI,WanFang Data databases,and relevant health technology assessment(HTA)websites were searched to collect relevant literature and reports on Ven treatment for non-M3 AML,with a search timeframe from the establishment of the database/website to November 1st,2024.Two researchers independently screened literature,extracted data,and assessed quality,and then qualitatively described and analyzed the results.Results A total of 11 pieces of literature were included,including 5 systematic reviews/Meta-analysis,4 pharmacoeconomic studies,and 2 HTA reports.In terms of efficacy,compared with the control group,non-M3 AML patients receiving Ven treatment had a higher clinical remission rate(P<0.05),a longer event-free survival(EFS)(P<0.05)and a similar or longer overall survival(OS)(P<0.05).Regarding safety,compared to Azacitidine(Aza)monotherapy,Ven+Aza resulted in a higher likelihood of febrile neutropenia in non-M3 AML patients(P<0.05).Non-M3 AML patients receiving Ven+low-dose cytarabine(LDAC)had a higher risk of developing thrombocytopenia compared with LDAC monotherapy(P<0.05).However,the early 30-day mortality rate was lower in the Ven+chemotherapy group than that in the chemotherapy alone group(P<0.05),presenting an acceptable security profile overall.In terms of cost-effectiveness,Ven was cost-effective in non-M3 AML patients compared with the control group.Conclusion Ven has manifested remarkable efficacy and acceptable security profile among patients with non-M3 AML,thus proving to be a medium to long-term cost-effective treatment modality.
3.Tofacitinib for the treatment of ulcerative colitis:a rapid health technology assessment
Huiling LI ; Yinyin DUAN ; Qianqian LI ; Xiaoli WU
Chinese Journal of Pharmacoepidemiology 2024;33(12):1414-1424
Objective To evaluate the efficacy,safety and cost-effectiveness of tofacitinib in the treatment of ulcerative colitis using a rapid health technology assessment,and to provide an evidence-based basis for rational clinical use and decision-making.Methods PubMed,Embase,Cochrane Library,CNKI,WanFang Data,VIP and relevant HTA websites and databases were electronically searched to collect high-quality clinical evidence and economic evaluation literature of tofacitinib in the treatment of ulcerative colitis from inception to July 31,2024.The results were qualitatively analyzed by two researchers after independently screening the literature,extracting information and evaluating quality.Results A total of 17 studies were included,including 1 HTA report,9 systematic reviews/Meta-analyses,and 8 pharmacoeconomic studies.In terms of efficacy,the clinical response rate,clinical remission rate,mucosal healing rate,endoscopic remission rate and short-term colectomy rate were significantly improved by the treatment of tofacitnib in ulcerative colitis patients(P<0.05).In terms of safety,tofacitinib was similar to placebo in the incidence of adverse events,serious adverse events,withdrawal from treatment due to adverse events,incidence of serious infections,and overall mortality(P>0.05),with only a slight increase in the incidence of infections(P<0.05).In terms of cost-effectiveness,the treatment of moderate-to-severe ulcerative colitis with tofacitinib was cost-effective compared with conventional therapy or other biologics in many countries including China.Conclusion Tofacitinib is effective and safe in the treatment of ulcerative colitis,and is significantly cost-effective for moderate-to-severe ulcerative colitis patients.Since it is still an off-label drug used for treating ulcerative colitis in China,the efficacy,safety and cost-effectiveness of tofacitinib need to be further evaluated based on the real-world clinical research and medical background in China in the future.
4.Tofacitinib for the treatment of ulcerative colitis:a rapid health technology assessment
Huiling LI ; Yinyin DUAN ; Qianqian LI ; Xiaoli WU
Chinese Journal of Pharmacoepidemiology 2024;33(12):1414-1424
Objective To evaluate the efficacy,safety and cost-effectiveness of tofacitinib in the treatment of ulcerative colitis using a rapid health technology assessment,and to provide an evidence-based basis for rational clinical use and decision-making.Methods PubMed,Embase,Cochrane Library,CNKI,WanFang Data,VIP and relevant HTA websites and databases were electronically searched to collect high-quality clinical evidence and economic evaluation literature of tofacitinib in the treatment of ulcerative colitis from inception to July 31,2024.The results were qualitatively analyzed by two researchers after independently screening the literature,extracting information and evaluating quality.Results A total of 17 studies were included,including 1 HTA report,9 systematic reviews/Meta-analyses,and 8 pharmacoeconomic studies.In terms of efficacy,the clinical response rate,clinical remission rate,mucosal healing rate,endoscopic remission rate and short-term colectomy rate were significantly improved by the treatment of tofacitnib in ulcerative colitis patients(P<0.05).In terms of safety,tofacitinib was similar to placebo in the incidence of adverse events,serious adverse events,withdrawal from treatment due to adverse events,incidence of serious infections,and overall mortality(P>0.05),with only a slight increase in the incidence of infections(P<0.05).In terms of cost-effectiveness,the treatment of moderate-to-severe ulcerative colitis with tofacitinib was cost-effective compared with conventional therapy or other biologics in many countries including China.Conclusion Tofacitinib is effective and safe in the treatment of ulcerative colitis,and is significantly cost-effective for moderate-to-severe ulcerative colitis patients.Since it is still an off-label drug used for treating ulcerative colitis in China,the efficacy,safety and cost-effectiveness of tofacitinib need to be further evaluated based on the real-world clinical research and medical background in China in the future.
5.Establishment of whole-process intelligent pharmaceutical care model for peritoneal dialysis patients
Yongfu HANG ; Yan XU ; Xiaohua DAI ; Tiantian WU ; Yinyin DUAN ; Deyu XU ; Kun HU ; Xingxing LIU ; Jianguo ZHU ; Liyan MIAO ; Lin LI
China Pharmacy 2023;34(21):2644-2648
OBJECTIVE To develop a whole-process intelligent model of pharmaceutical care for peritoneal dialysis (PD) patients, and to provide a reference for clinical pharmacists to provide standardized PD pharmaceutical care. METHODS The pharmaceutical care mode of PD patients at home and abroad was investigated and analyzed. Based on the actual situation of the First Affiliated Hospital of Soochow University (hereinafter referred to as “our hospital”), with “home→PD center outpatient→ inpatient department” as the main node, the recycling process of medication reconciliation was optimized. The whole-process intelligent pharmaceutical care model of PD was illustrated by improving the Chinese version of the drug-related problems (DRPs) classification tool, developing the corresponding pharmaceutical care process, and presenting specific cases. RESULTS Based on the medication therapy management (MTM) platform, our hospital had built a closed-loop PD whole-process intelligent pharmaceutical care model of “in-hospital pharmaceutical care (building document)-PD outpatient MTM-home pharmaceutical care (online App management)”. A “double cycle” workflow of “admission→discharge→outpatient” medication reconciliation cycle and “discovery-analysis-intervention-follow-up-record-evaluation” DRPs cycle was formed. CONCLUSIONS The establishment of the whole-process intelligent pharmaceutical care model for PD in our hospital provides experience for standardizing pharmaceutical care for PD patients, and can reduce DRPs.
6.Inhibition effect of short hairpin RNA of transforming growth factor β1 on cytokines expression induced by albumin in HK2 cells
Shaobin DUAN ; Fuyou LIU ; Yinyin CHEN ; Fang LIU ; Ying LI ; Guanghui LING ; Li XIAO ; Hong LIU ; Youming PENG
Journal of Central South University(Medical Sciences) 2009;34(10):949-956
Objective To determine the effect of 2 transforming growth factor β1 (TGF-β1) short hairpin RNA (shRNA) expression plasmids (pcDU6-A1-A2 and pcDU6-B1-B2) on proliferation, TGF-β1, connective tissue growth factor (CTGF), and fibronectin (FN) expression induced by human serum albumin (HAS) in HK2 cells. Methods A vector plasmid containing the TGF-β1 shRNA was generated. An HK2 cell line was used in the study. The 2 TGF-β1 shRNA expression plasmids were transfected into cultured HK2 cells by lipofectamine 2000. Cellular proliferation was assessed by tetrazolium salt colorimetry. The semi-quantitative reverse transcriptive PCR was performed to detect the expression of TGF-β1,CTGF, and FN mRNA. Levels of TGF-β1 and FN protein were measured with a sandwich enzyme-linked immunosorbent assay. Results After treating with 5 g/L HAS for 24 hours in HK2 cells, cellular proliferating capacity increased significantly (P<0.05). The expression levels of TGF-β1, CTGF, and FN mRNA were upregulated in HK2 cells stimulated by 5 g/L HAS, and levels of TGF-β1 and FN protein in the culture supernatant increased (P<0.05). The introduction of pcDU6-A1-A2 and pcDU6-B1-B2 resulted in significant reduction of cellular proliferation activity, and the expression levels of TGF-β1, CTGF, and FN mRNA were downregulated (P<0.05). Levels of TGF-β1 and FN protein in the culture supernatant decreased (P<0.05) after 12 or 24 hours of TGF-β1 shRNA transfection into HK2 cells There was no significant difference in the expression levels of TGF-β1, CTGF, and FN mRNA between the 2 pcDU6 vector plasmid mediated TGF-β1 shRNA groups (P>0.05). Conclusion pcDU6 vector plasmid mediated TGF-β1 shRNAs could obviously inhibit the expression levels of TGF-β1, CTGF, FN and cellular proliferation stimulated by HAS in HK2 cells, which may be related to the mediation of TGF-β1.

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