scholarly journals Investigating the Efficacy and Safety of Thalidomide for Treating Patients With ß-Thalassemia: A Meta-Analysis

2022 ◽  
Vol 12 ◽  
Author(s):  
Yanfei Lu ◽  
Zhenbin Wei ◽  
Gaohui Yang ◽  
Yongrong Lai ◽  
Rongrong Liu

At present, the main therapies for ß-thalassemia patients include regular blood transfusion and iron chelation, associating with a number of limitations. Thalidomide, a fetal hemoglobin (HbF) inducer that promotes γ-globin gene expression, has been reported to be effective for ß-thalassemia. Thus, this meta-analysis was conducted to assess the efficacy and safety of thalidomide for treating patients with ß-thalassemia. We searched the related studies from eight databases published from inception until December 1, 2021. The R 4.0.5 language programming was used to perform meta-analysis. After screening of retrieved articles, 12 articles were included that enrolled a total of 451 patients. The Cochrane Collaboration risk assessment tool was used to evaluate the quality and the bias risk of the randomized controlled trials (RCTs), and non randomized trials were assessed using Newcastle-Ottawa Scale (NOS). After treatment with thalidomide, the pooled overall response rate (ORR) was 85% (95% confidence interval (CI): 80–90%), and the pooled complete response rate (CRR) was 54% (95% confidence interval: 31–76%). Compared with the placebo group, the thalidomide group had higher odds of overall response rate (odds ratio = 20.4; 95% CI: 6.75–61.64) and complete response rate (odds ratio = 20.4; 95% CI: 6.75–61.64). A statistically significant increase in hemoglobin level and HbF level after treatment, while there was no statistically significant difference in adult hemoglobin (HbA) level, spleen size, and serum ferritin. According to the results of ORR and CRR, transfusion-dependent thalassemia (TDT) patients showed remarkable efficacy of thalidomide, 83 and 52% respectively. So we analyzed 30 transfusion-dependent thalassemia patients from three studies and found that the most frequent ß-globin gene mutations were CD41-42 (-TCTT), while response to thalidomide did not show any statistically significant relationship with XmnI polymorphism or CD41-42 (-TCTT) mutation. About 30% of patients experienced mild adverse effects of thalidomide. Collectively, thalidomide is a relatively safe and effective therapy to reduce the blood transfusion requirements and to increase Hb level in patients with ß-thalassemia.

2021 ◽  
Vol 2021 ◽  
pp. 1-10
Author(s):  
Lu-Zhen Li ◽  
Jia-Ming Wu ◽  
Ting Chen ◽  
Liang-Chen Zhao ◽  
Juan-Na Zhuang ◽  
...  

Objective. Systematically evaluate the efficacy of physical ablation combined with TKI in the treatment of advanced non-small cell lung cancer (NSCLC). Methods. We performed a comprehensive search of databases including OVID, PubMed, EMBASE, the Cochrane Library, and three Chinese databases (China National Knowledge Infrastructure, Wanfang Database, and Chongqing Weipu Database). The aim was to identify randomized controlled trials (RCT) investigating physical ablation as the treatment for advanced NSCLC. We also evaluated the methodological quality of the included studies and summarized the data extracted for meta-analysis with Review Manager 5.3. Results. A total of 9 studies, including 752 patients, were evaluable. The meta-analysis results show that the complete response rate (CRR) (RR: 2.23, 95% CI: 1. 46 to 3.40, P 0.01), partial response rate (PRR) (RR: −2.25, 95% CI: 1.41 to 3.59, P 0.01), and disease control rate (DCR) (RR: −2.80, 95% CI: 1.64 to 4.80, P < 0.01) of patients with advanced NSCLC who received physical ablation combined with TKI therapy were higher than those who did not receive physical ablation therapy. The control groups from seven of the studies had a total of 606 patients with targeted therapies and chemotherapy. The complete response rate was (CRR) (RR: 2.48, 2.4895% CI: 1.55 to 2.47, P 0.01), partial response rate (PRR) (RR: −1.66, 95% CI: 1.20 to 2.31, P < 0.01), and disease control rate (DCR) (RR: −2.68, 95% CI: 1.41 to 5.06, P < 0.01) for patients with advanced NSCLC who had received physical ablation combined with targeted therapies and chemotherapy, compared to patients who had not received physical ablation therapy. This difference was statistically significant. Above all, these results showed that the clinical efficacy of physical ablation combined EGFR-TKIs therapy (regardless of whether it was combined with chemotherapy) was better than that of EGFR-TKIs therapy alone. Conclusion. Physical ablation combined with TKI treatment in patients with advanced NSCLC can improve efficacy.


Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4340-4340
Author(s):  
Yuejian Liu ◽  
Feili Chen ◽  
Xutao Guo ◽  
Pengcheng Shi ◽  
Jie Zha ◽  
...  

Abstract Abstract 4340 Background: Myelodysplastic syndrome (MDS) is a malignant hematological disease that comprises a heterogeneous group of clonal hematopoietic stem and progenitor cell disorders, with peripheral cytopenias, bone marrow hypercellularity, high-risk of evolving into acute myeloid leukemia (AML). MDS/AML is a special refractory and palindromic AML characterized by poor therapeutic effects and low complete response rate, as well as high treatment-related complications and mortality. Patients with MDS/AML are often elders and represent more intolerance to routine or intensive chemotherapies. Homoharringtonine, an alkaloid found as the major active component in Chinese plants cephatotaxus fortuneif., has been widely used in AML since the 1970s in China. Decitabine, a hypomethylating agent, is active and has been approved for the treatment of myelodysplastic syndrome (MDS) in recent years. Objective: In order to compare the efficacy, toxicity and long-term prognosis of two chemotherapies HA (Homoharringtonine and cytarabine) and Decitabine regiment in MDS/AML. Methods: A total of 26 MDS/AML patients consisting of 14 males and 12 females were included in this study. They were randomly assigned to receive either HA (H 4mg.d−1,d1–3; A 100mg.m−2d−1, d1–7) or decitabine£.. 20mg.m−2d−1, d1–5£© The effect measures used were hazard ratios (HR) for overall survival (OS), progression-free survival (PFS) and freedom from first progression. Relative risks were used to analyse complete response rate, total response rate, treatment-related mortality and adverse events. A Log-rank test was used in survival analysis, and a Chi-square test was performed for other outcomes. Results: The complete remission (CR) rate with HA regimen according to MDS/AML criteria was 33% and 36% with decitabine (P>0.05). HA group had no lower total response rate than Decitabine group (53% versus 64%, P>0.05). The freedom from first progression in chemotherapy with HA regiment and decitabine was 20% and 18% (P>0.05), respectively. PFS was not statistically significantly longer for two comparators with HR was 0.41(95% confidence interval (CI) 0.09722 to 1.740). There was no statistically significant difference in OS between the HA group and decitabine group with HR was 0.799 (95% CI 0.2992 to 2.133); median survival: 300 days vs 291 days (P>0.05,95% confidence interval (CI) 0.6165 to 1.445). The treatment-related mortality was 13% with HA regimen versus 18% with decitabine at 3 weeks (P>0.05) and 40% with HA regiment versus 18% with decitabine at 3 months (P>0.05). The haematological toxicities and liver function lesion WHO grade III or IV were not significantly higher in the HA group than that in the decitabine group (P>0.05). The total secondary infection rates in all sections of chemotherapies were 58% and 19% (P=0.005) in the two groups, respectively. Secondary infection rate was significantly lower in the decitabine group than that in the HA group. Conclusions: This analysis showed that Homoharringtonine and cytarabine regiment in treating MDS/AML has a similar therapeutic effect and long-term benefit with decitabine, both regiments were associated with relatively safe and effective outcomes in patients with MDS/AML. However, HA regiment shows a higher risk of secondary infection than decitabine. Longer follow-up and further studies will evaluate prospectively the results of HA regiment versus decitabine in this setting. Disclosures: No relevant conflicts of interest to declare.


2021 ◽  
Vol 12 ◽  
Author(s):  
Yali Tao ◽  
Hui Zhou ◽  
Ting Niu

Background: Selinexor (SEL) is an orally bioavailable, highly-selective, and slowly-reversible small molecule that inhibits Exportin 1. Preclinical studies showed that SEL had synergistic antimyeloma activity with glucocorticoids, proteasome inhibitors (PIs) and immunomodulators. The combination of selinexor and dexamethasone (DEX) has been approved in the United States for patients with penta-refractory multiple myeloma in July 2019. This meta-analysis aimed to investigate the safety and efficacy of selinexor based treatment in Multiple myeloma.Methods: We systematically searched the Medline (PubMed), Embase, Web of Science, Cochrane Central Register of Controlled Trials Library databases and ClinicalTrials.gov. Outcome measures of efficacy included overall response rate (ORR), clinical benefit rate (CBR), stringent complete response rate (sCR), complete response rate (CR), very good partial response (VGPR), partial response rate (PR), minimal response (MR), rate of stable disease (SDR), rate of progressive disease (PDR) and median progression-free survival (mPFS). Safety was evaluated by the incidences of all grade adverse events and Grade≥3 adverse events. The subgroup analysis was conducted to analyze the difference in different combination treatment regimens (SEL + DEX + PIs vs SEL + DEX).Results: We included six studies with 477 patients. The pooled ORR, CBR, sCR, CR, VGPR, PR, MR, SDR, and PDR were 43% (18–67%), 55% (32–78%), 5% (−2–13%), 7% (4–11%), 14% (5–24%), 23% (15–31%), 11% (8–14%), 26% (14–38%) and 14% (4–23%), respectively. SEL + DEX + PIs treatment had higher ORR (54 vs 24%, p = 0.01), CBR (66 vs 37%, p = 0.01), sCR (10 vs 2%, p = 0.0008), and VGPR (23 vs 5%, p &lt; 0.00001) compared to SEL + DEX treatment, and lower PDR (4 vs 23%, p &lt; 0.00001) and SDR (17 vs 37%, p = 0.0006). The pooled incidences of any grade and grade≥3 were 45 and 30% in hematological AEs, and in non-hematological AEs were 40 and 30%, respectively. The most common all grade (68%) and grade≥3 (54%) hematological AE were both thrombocytopenia. Fatigue was the most common all grade (62%) and grade≥3 (16%) non-hematological AE. Compared to SEL + DEX treatment, SEL + DEX + PIs treatment had lower incidences of hyponatremia (39 vs 12%, p &lt; 0.00001), nausea (72 vs 52%, p &lt; 0.00001), vomiting (41 vs 23%, p &lt; 0.0001), and weight loss (42 vs 17%, p = 0.03) in all grade AEs. Meanwhile, SEL + DEX + PIs treatment had lower incidences of anemia (36 vs 16%, p = 0.02), fatigue (20 vs 13%, p = 0.04), hyponatremia (22 vs 5%, p &lt; 0.0001) than SEL + DEX treatment in grade≥3 AEs.Conclusion: Our meta-analysis revealed that selinexor-based regimens could offer reasonable efficacy and tolerable adverse events in patients with multiple myeloma. SEL + DEX + PIs treatments had higher efficacy and lower toxicities than SEL + DEX.


2021 ◽  
Author(s):  
Wen jie Xie ◽  
Shuai Zhang ◽  
Lei Su ◽  
Yan hong Li ◽  
Xi Zhang ◽  
...  

Aim: We performed an updated meta-analysis to evaluate the efficacy and safety of lenvatinib in cancer patients. Materials & methods: Databases were searched to identify relevant trials. Data were extracted to evaluate overall survival, progression-free survival, overall response rate and grade ≥3 adverse events. Results: The pooled analysis demonstrated that lenvatinib significantly improved progression-free survival (hazard ratio: 0.43; 95% CI: 0.23–0.80; p = 0.008), overall survival (hazard ratio: 0.85; 95% CI: 0.75–0.97; p = 0.013) and overall response rate (relative risk: 6.89; 95% CI: 2.22–21.36; p = 0.001) compared with control therapy. However, the use of lenvatinib can increase the risk of severe infection. Conclusion: Lenvatinib-containing regimens are associated with better progression-free survival, overall survival and overall response rate, but can induce severe infection.


Dermatology ◽  
2021 ◽  
pp. 1-9
Author(s):  
Wen-Li Xue ◽  
Jia-Qi Ruan ◽  
Hong-Ye Liu ◽  
Hong-Xia He

<b><i>Background:</i></b> Photodynamic therapy is an established treatment option for Bowen’s disease. Our meta-analysis was aimed at evaluating the efficacy and recurrence of photodynamic therapy or other topical treatments (5-fluorouracil, cryotherapy) and of photodynamic therapy alone or in combination with other therapies (ablative fractional CO<sub>2</sub> laser or plum-blossom needle) for the treatment of Bowen’s disease. <b><i>Methods:</i></b> Trials that met our inclusion criteria were identified from PubMed, EMBASE, Web of Science, and Cochrane Library databases, and meta-analyses were conducted with RevMan V.5.4. The risk of bias was estimated with the Cochrane Collaboration’s risk of bias tools. Complete response rate, recurrence, pain/visual analogue scale score, cosmetic outcome, and adverse events were considered as outcomes. <b><i>Results:</i></b> Of the 2,439 records initially retrieved, 8 randomized controlled trials were included in this meta-analysis. According to our analyses, photodynamic therapy exhibited a significantly higher complete response rate (RR = 1.36, 95% CI [1.01, 1.84], <i>I</i><sup>2</sup> = 86%, <i>p</i> = 0.04), less recurrence (RR = 0.53, 95% CI [0.30, 0.95], <i>I</i><sup>2</sup> = 0%, <i>p</i> = 0.03), and better cosmetic outcome (RR = 1.34, 95% CI [1.15, 1.56], <i>I</i><sup>2</sup> = 0%, <i>p</i> = 0.0002) compared with other treatments. Moreover, there was a significant difference between the complete response rate of photodynamic therapy combined with ablative fractional CO<sub>2</sub> laser and that of photodynamic therapy (RR = 1.85, 95% CI [1.38, 2.49], <i>I</i><sup>2</sup> = 0%, <i>p</i> &#x3c; 0.0001). Photodynamic therapy combined with ablative fractional CO<sub>2</sub> laser or plum-blossom needle also showed significantly less recurrence (RR = 0.21, 95% CI [0.09, 0.51], <i>I</i><sup>2</sup> = 0%, <i>p</i> = 0.0005) and a lower visual analogue scale score (RR = 0.51, 95% CI [0.06, 0.96], <i>I</i><sup>2</sup> = 0%, <i>p</i> = 0.03) than photodynamic therapy alone. However, there was no significant difference in the complete response rate between photodynamic therapy combined with ablative continuous CO<sub>2</sub> laser and photodynamic therapy combined with ablative fractional CO<sub>2</sub> laser (RR = 1.00, 95% CI [0.54, 1.86], <i>I</i><sup>2</sup> not applicable, <i>p</i> = 1.00). <b><i>Conclusions:</i></b> This meta-analysis shows that photodynamic therapy can be used in the treatment of Bowen’s disease with better efficacy, less recurrence, and better cosmetic outcomes than cryotherapy and 5-FU. Some methods, including ablative fractional CO<sub>2</sub> laser, can be applied in combination with photodynamic therapy to improve efficacy. However, which laser-assisted photodynamic therapy scheme has the most advantages in the treatment of Bowen’s disease warrants further exploration.


JRSM Open ◽  
2021 ◽  
Vol 12 (6) ◽  
pp. 205427042110106
Author(s):  
Yan Zhuang ◽  
Lin-feng Dai ◽  
Ming-qi Chen

Objective Several trials had compared the efficacy and safety between non-vitamin K antagonist oral anticoagulants and warfarin for acute venous thromboembolism, but the results were incomplete. This updated review comprehensively assessed the efficacy and safety of non-vitamin K antagonist oral anticoagulants for venous thromboembolism. Design Meta-analysis of randomised control trials. Six databases were searched from January 2000 to December 2018. Setting Adult patients had got non-vitamin K antagonist oral anticoagulants or warfarin for venous thromboembolism. Participants Randomised control trials that compared the efficacy and safety between non-vitamin K antagonist oral anticoagulants and warfarin. Main outcome measures The efficacy and safety of non-vitamin K antagonist oral anticoagulants . Results Seven studies involving 29,879 cases were included, among which 14,943 cases were assigned to non-vitamin K antagonist oral anticoagulants group and 14,936 cases to warfarin group. Meta-analysis showed that compared with warfarin, recurrent venous thromboembolism (odds ratio 0.94 [95% confidence interval 0.81 to 1.11]), death related to venous thromboembolism or fatal pulmonary embolism (odds ratio 1.00 [95% confidence interval 0.63 to 1.60]), symptomatic deep-vein thrombosis (odds ratio 0.88 [95% confidence interval 0.72 to 1.09]), symptomatic nonfatal pulmonary embolism (odds ratio 1.03 [(95% confidence interval 0.82 to 1.30]) and all deaths (odds ratio 0.92 [95% confidence interval 0.76 to 1.12]) are similar in non-vitamin K antagonist oral anticoagulants group, but major bleeding event (odds ratio 0.61 [95% confidence interval 0.50 to 0.75]) and clinically relevant non-major bleeding event (odds ratio [95% confidence interval 0.53 to 0.85]) are less in non-vitamin K antagonist oral anticoagulants group.  Conclusions For the treatment of venous thromboembolism, non-vitamin K antagonist oral anticoagulants is as effective as warfarin, and has a better safety profile than warfarin.


2018 ◽  
Vol 2018 ◽  
pp. 1-8 ◽  
Author(s):  
Bum Jun Kim ◽  
Hyeong Su Kim ◽  
Hyun Joo Jang ◽  
Jung Han Kim

Objective.Several recent reviews of published studies have shown that the eradication ofH. pyloriinfection in patients with ITP improved thrombocytopenia in about half of the cases. However, most included studies were observational case series. We performed the first meta-analysis of randomized trials to gain a better insight into the effect ofH. pylorieradication in ITP patients.Methods.A systematic computerized search of the electronic databases including PubMed, EMBASE, Google Scholar, and Cochrane Library (up to December 2017) was conducted.Results.From six studies, a total of 241 patients (125 in eradication group and 116 in control group) were included in the meta-analysis. Patients in the eradication group showed significantly higher overall platelet response rate than those in the control group (odds ratio = 1.93, 95% confidence interval: 1.01–3.71,P=0.05). In the subgroup analysis, however, children in the eradication group failed to show statistically better response rate than those in the noneradication group (odds ratio = 1.80, 95% confidence interval: 0.88–3.65,P=0.11).Conclusions.This meta-analysis indicates thatH. pylorieradication has a significant therapeutic effect in patients with ITP. Considering the intrinsic limits in the design and sample size of the included studies, however, large randomized controlled trials are warranted to validate the therapeutic impact ofH. pylorieradication in adults as well as children with ITP.


2001 ◽  
Vol 19 (8) ◽  
pp. 2232-2239 ◽  
Author(s):  
George Fountzilas ◽  
Christos Papadimitriou ◽  
Urania Dafni ◽  
Dimitrios Bafaloukos ◽  
Dimosthenis Skarlos ◽  
...  

PURPOSE: To compare the efficacy of two different schedules of epirubicin and paclitaxel, as first-line chemotherapy, in patients with advanced breast cancer (ABC). PATIENTS AND METHODS: From October 1997 until May 1999, 183 eligible patients with ABC entered the study. Chemotherapy in group A (93 patients) consisted of four cycles of epirubicin at a dose of 110 mg/m2 followed by four cycles of paclitaxel at a dose of 225 mg/m2 in a 3-hour infusion. All cycles were repeated every 2 weeks with granulocyte colony-stimulating factor support. The therapeutic regimen in group B (90 patients) consisted of epirubicin (80 mg/m2) immediately followed by paclitaxel (175 mg/m2 in a 3-hour infusion) every 3 weeks for six cycles. RESULTS: In total, 79 patients (85%) in group A and 72 patients (80%) in group B completed treatment. The median relative dose-intensity of epirubicin was 0.96 in both groups, and that of paclitaxel was 0.96 and 0.97 in groups A and B, respectively. The complete response rate was higher in group A (21.5% v 9% P = .02). Nevertheless, there was no significant difference in the overall response rate between the two groups (55% v 42%, P = .10). Severe neutropenia was more frequently observed with concurrent treatment. After a median follow-up of 16.5 months, median time to progression was 10 months in group A and 8.5 months in group B (P = .27), and median survival was 21.5 and 20 months, respectively (P = .17). CONCLUSION: The present study failed to demonstrate a significant difference in overall response rate between dose-dense sequential administration of epirubicin and paclitaxel compared with the combination of the two drugs given on the same day, even though the sequential treatment resulted in a significantly higher complete response rate.


Cancers ◽  
2019 ◽  
Vol 11 (1) ◽  
pp. 47 ◽  
Author(s):  
Wen-Liang Yu ◽  
Zi-Chun Hua

Chimeric antigen receptors T cells (CAR T) had been used for treating various tumor patients in clinic, and owned an incredible efficacy in part of malignancies. However, CAR T therapy remains controversial due to doubts about its efficacy and safety in the clinical treatment of various malignancies. A total of 997 tumor patients from 52 studies were included in this review. Eligible studies were searched and reviewed from the databases of PubMed, Web of Science, Wanfang and Clinicaltrials.gov. Then meta-analysis and subgroup analysis were used to investigate the overall response rate (ORR), complete response rate (CRR), common side effect rate (CSER) and relapse rate (RR) of CAR T therapy for patients in clinical researches, respectively. The results further confirmed that CAR T therapy had a higher response rate for hematologic malignancies. More importantly, CAR T therapy had a higher CSER in patients with hematologic malignancies, and it had a similar RR in patients with different malignancies. Cell cultured without the addition of IL-2 and total administration less than 108 cells were recommended. This study offers a reference for future research regarding the application in solid and hematologic malignancies, side effects and relapse, and even the production processes of CAR T cells.


2021 ◽  
Vol 11 ◽  
Author(s):  
Rui Zhang ◽  
Meng Zhou ◽  
Jiaqian Qi ◽  
Wenjing Miao ◽  
Ziyan Zhang ◽  
...  

BackgroundTransplant-associated thrombotic microangiopathy (TA-TMA) is a dangerous and life-threatening complication in patients undergoing hematopoietic stem cell transplantation (HSCT). Eculizumab has been used in the treatment of TA-TMA, and several studies have confirmed the benefit of Eculizumab in patients with TA-TMA. However, the results remain controversial. We conducted a systematic review and meta-analysis to evaluate the efficacy and safety of Eculizumab for TA-TMA.Materials and MethodsWe searched PubMed and Embase for studies on the efficacy and safety of Eculizumab in TA-TMA patients. Efficacy outcomes consisted of overall response rate (ORR), complete response rate (CRR), and survival rate at the last follow-up (SR). Safety outcomes were adverse events (AEs), including infection, sepsis, impaired liver function, infusion reactions, and death.ResultsA total of 116 patients from six studies were subjected to meta-analysis. The pooled estimates of ORR, CRR, and SR for TA-TMA patients were 71% (95% CI: 58–82%), 32% (95% CI: 11–56%), and 52% (95% CI: 40–65%), respectively. Only one patient presented with a severe rash, and infection was the most common AEs. The main causes of death were infection and GvHD.ConclusionCurrent evidence suggests that Eculizumab improves SR and ORR in patients with TA-TMA and that Eculizumab is well tolerated. However, the number of studies is limited, and the findings are based mainly on data from observational studies. Higher quality randomized controlled trials and more extensive prospective cohort studies are needed.


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