scholarly journals Use of Biologics in Pityriasis Rubra Pilaris Refractory to First-Line Systemic Therapy: A Systematic Review

2019 ◽  
Vol 24 (1) ◽  
pp. 73-78
Author(s):  
Avee Naidoo ◽  
Cathryn Sibbald ◽  
Patrick J. Fleming ◽  
Vincent Piguet
Keyword(s):  
Large Scale ◽  
Randomized Clinical Trials ◽  
Case Series ◽  
Efficacy And Safety ◽  
Inclusion Criteria ◽  
Treatment Resistant ◽  
First Line ◽  
Disease Treatment ◽  
Pityriasis Rubra Pilaris ◽  
Systemic Therapies

Pityriasis rubra pilaris (PRP) is an uncommon, inflammatory, papulosquamous skin disease. Treatment of PRP is challenging as the disease is often refractory to conventional therapies, such as retinoids and methotrexate. There has been an increasing number of studies reporting the successful use of biologic therapy in patients with PRP; however, the data on the efficacy and safety are limited. Our objective was to evaluate the existing evidence for utilizing biologics, whether alone or in combination with established systemic therapies, in patients with treatment-resistant PRP. We systematically reviewed evidence within Medline and Pubmed databases between January 1, 2000, to March 31, 2019. Articles consisted of patients diagnosed with PRP who have failed to respond sufficiently to first-line systemic therapies, or who had comorbidities that precluded their use. In total, 363 unique articles were identified, 56 of which were considered relevant to the clinical question. Of the 56 articles highlighted, 35 met the inclusion criteria and were limited to case series and case studies. Therapy with biologics was found to be successful for both monotherapy (81.1% [27/33]) and when used in combination with existing systemic therapies (87.5% [14/16]). The existing evidence suggests that biologics may be regarded as a tool for PRP treatment alone or in combination therapy with existing treatments, although large-scale randomized clinical trials are necessary to better assess their efficacy and safety.

scholarly journals Use of Ketamine Infusions for Treatment of Complex Regional Pain Syndrome: A Systematic Review

2021 ◽  
Author(s):  
Anand S. Patil ◽  
Ahish Chitneni ◽  
Suhani Dalal ◽  
Joe Ghorayeb ◽  
Yolanda Pham ◽  
...  
Keyword(s):  
Systematic Review ◽  
Pain Relief ◽  
Complex Regional Pain Syndrome ◽  
Large Scale ◽  
Randomized Clinical Trials ◽  
Cochrane Review ◽  
Pain Syndrome ◽  
Inclusion Criteria ◽  
Treatment Resistant ◽  
Ketamine Infusion

AbstractBackgroundThis systematic review aims to review clinical studies on the use of ketamine infusion for patients with treatment-resistant Complex Regional Pain Syndrome (CRPS).MethodsThe following systematic review was registered on PROSPERO (CRD42021228470). Studies for the systematic review were identified through 3 databases; PubMed, CINAHL, and Cochrane Review. Inclusion criteria for studies consisted of randomized clinical trials or cohort studies that conducted trials on the use of ketamine infusion for pain relief in patients with Complex Regional Pain Syndrome (CRPS). Exclusion criteria for studies included any studies that were systematic review, meta-analyses, case reports, literature reviews, or animal studies. In the included studies, the primary outcome of interest was post drug administration pain score.ResultsIn this systematic review, 14 studies met the inclusion criteria and were reviewed. In these studies, the dosage of ketamine infusion used ranged from 0.15 mg/kg to 7mg/kg with primary indication being treatment of Complex Regional Pain Syndrome (CRPS). In 13 of the studies, ketamine infusion resulted in a decrease in pain scores and relief of symptoms.ConclusionsPatients who received Ketamine infusion for treatment-resistant CRPS self-reported adequate pain relief with treatment. This suggests that ketamine infusion may be a useful form of treatment for patients with no significant pain relief with other conservative measures. Future large-scale studies, including randomized double-blind placebo-controlled trials on the use of ketamine infusion for CRPS must be conducted in a large-scale population to further assess the effectiveness of ketamine infusion in these populations.

scholarly journals Efficacy and Safety of Bevacizumab in Combination with Chemotherapy for Colorectal Cancer – A Real World Study in China

2020 ◽  
Author(s):  
Tao Shen ◽  
Xian-Shuo Cheng ◽  
Wei-Xun Chunyu ◽  
Hong-Tao Zhang ◽  
Cui-Feng Xia ◽  
...  
Keyword(s):  
Colorectal Cancer ◽  
Adverse Events ◽  
Real World ◽  
Large Scale ◽  
Progression Free Survival ◽  
Secondary Outcome ◽  
Second Line ◽  
Efficacy And Safety ◽  
First Line ◽  
Demographic Subgroups

Abstract Background Large scale randomized trials have demonstrated that bevacizumab in addition to chemotherapy as first-line or second-line treatment has significant survival benefits. We aim to explore the clinical impact of bevacizumab in combination with chemotherapy in first-line or second-line in patients with colorectal cancer (CRC). Methods The medical records of patients with CRC who received bevacizumab at first or second-line of treatment were collected retrospectively. The primary outcome of the study was to evaluate the efficacy of bevacizumab in combination with chemotherapy by survival endpoints i.e. overall survival (OS) and progression-free survival (PFS) and the secondary outcome was to evaluate its safety by incidence of adverse events (AE). Results Fifty-one patients with CRC had met the selection criteria for treatment with bevacizumab to either cetuximab or FOLFOX or both. The median age was 54 years. During follow-up, ten patients had exhibited progression after treatment while 5 patients died. The median OS and PFS of the overall population were not reached. The Cox proportional regression analysis revealed no significant prognostic factors of OS and PFS for treatment with bevacizumab in various demographic subgroups. The 1-year PFS rates of all 51 patients was 76%. The 1-year and 3-year OS rates for all 51 patients were 95% and 88%, respectively. Toxicities were usually mild in nature, with nausea, vomiting, hand and foot syndrome, neutropenia, asthenia and palpitation being the commonly reported adverse events. Conclusion In this real-world setting, the efficacy and safety of bevacizumab in combination with chemotherapy is limited and further research is warranted as to whether bevacizumab with chemotherapy is an optimal treatment as first-line or second-line therapy in Chinese CRC patients.

scholarly journals Efficacy and Safety of Docetaxel Plus Oxaliplatin and Capecitabine in the First Line Treatment of Advanced Gastric Adenocarcinoma

2013 ◽  
Vol 2013 ◽  
pp. 1-6
Author(s):  
Ying Liu ◽  
Zhengbao Ye ◽  
Wenqi Xi ◽  
Tao Ma ◽  
Min Shi ◽  
...  
Keyword(s):  
Gastric Adenocarcinoma ◽  
Large Scale ◽  
Objective Response ◽  
Line Treatment ◽  
Efficacy And Safety ◽  
First Line ◽  
Common Grade ◽  
Advanced Gastric Adenocarcinoma ◽  
Grade 3 ◽  
First Line Treatment

Objective.To evaluate the efficacy and safety of docetaxel plus oxaliplatin and capecitabine (DOX) in the first line treatment of advanced gastric adenocarcinoma.Methods.A total of 37 patients were enrolled into this study, and they received DOX regimen (docetaxel 75 mg/m2and oxaliplatin 130 mg/m2intravenous infusion on day 1, and capecitabine 1000 mg/m2orally twice daily on d1–14); treatment was repeated every 3 weeks.Results.All 37 patients were assessable for evaluation. The numbers of patients with complete response (CR), partial responses (PR), stable disease (SD), and progressive disease (PD) were 1, 10, 23, and 3, respectively. The objective response rate (ORR) was 29.7%, with the disease control rate (DCR) of 91.9%. Median progression-free survival (mPFS) and overall survival (mOS) were 197 days and 364 days, respectively. The most common grade 3/4 toxicities were hematological toxicities. The most common grade 3/4 nonhematological toxicities were fatigue, nausea, vomiting, anorexia, diarrhea, and hand-foot syndrome.Conclusion.The DOX regimen demonstrated a promising efficacy as the first line regimen in treating advanced gastric cancer patients with good performance status, the toxicities were tolerated and controllable. Large-scale clinical observation is necessary to get further evidence.

scholarly journals Efficacy and Safety Profile of Ixazomib Based Regimens in Relapsed/Refractory Multiple Myeloma: A Meta-Analysis of Clinical Trials

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 20-21
Author(s):  
Ahmad Iftikhar ◽  
Muhammad Ashar Ali ◽  
Anum Javaid ◽  
Muhammad Abu Zar ◽  
Atif Sohail ◽  
...  
Keyword(s):  
Multiple Myeloma ◽  
Clinical Trials ◽  
Safety Profile ◽  
Randomized Clinical Trials ◽  
Meta Analysis ◽  
Case Series ◽  
Pooled Analysis ◽  
Phase Iii ◽  
Efficacy And Safety ◽  
Grade 3

Background: Multiple myeloma (MM) is an incurable disease, and clinical trials with newer agents have shown improved patient outcomes. There is a need for effective and tolerable treatment for patients with relapsed/refractory MM (RRMM). Proteasome inhibitors (bortezomib, carfilzomib, ixazomib) remain an integral part of regimens used in RRMM or newly diagnosed (ND) MM. This meta-analysis aims to assess the efficacy and safety of ixazomib (Ixa) based regimens in RRMM. Methods: A comprehensive literature search was performed on PubMed, Cochrane, Embase, Web of Science, and clinicaltrials.gov. We used MeSH and Emtree terms, "ixazomib" AND "multiple myeloma" from the inception of literature till 06/01/2020. We screened 1529 articles and included 3 randomized clinical trials (RCT, N=907) and 8 non-randomized clinical trials (NRCT, N=321). We excluded case reports, case series, review articles, meta-analysis, observational studies, and clinical trials that didn't provide data about the efficacy and safety of Ixa in RRMM. We used the R programming language (version 4.0.2) to conduct a meta-analysis. Results: In 11 clinical trials (N=1228), the age range of patients was 30-91 years. In Phase III RCTs (N=837) comparing Ixa + Lenalidomide (Len) + dexamethasone (Dex) vs. placebo + Len + Dex, risk ratio of overall response rate (ORR), complete response (CR), and very good partial response (VGPR) were 1.14 (95% CI=1.05-1.24, I2=80%), 1.87 (95% CI=1.17-2.99, I2=0), and 1.15 (95% CI=0.95-1.40, I2=0), respectively in favor of Ixa + Len + Dex. (Fig 1-3) Grade 3 or higher treatment-related adverse events (TRAEs) thrombocytopenia, diarrhea, and rash were reported in 20%, 5.7% and 6.4% of the patients in the Ixa group vs. 10%, 2.1%, and 2.8% in the placebo group, respectively. In a Phase II RCT by Kumar et al (N=70) comparing the Ixa dosage, 4 mg Ixa + Dex yielded an ORR of 31%, CR 2.8%, and VGPR 17.1%, while 5.5 mg Ixa yielded improved ORR of 54%, CR 2.8%, and VGPR 25.7%. In a NRCT by Costello et al. (N=6), Ixa + daratumumab (Dara) + Pom + Dex yielded 100% ORR, CR 5% (95% CI=0.17-0.83), and VGPR 50% (95% CI=0.17-0.83). ≥Grade 3 TRAEs were hypertension (16%), and hematological (33%). Among 417 patients from two RCT in single arm who received Ixa + Len + Dex, pooled ORR was 70% (95% CI=0.53-0.82, I2=84%), pooled CR 11% (95% CI=0.8-0.14, I2=0), and pooled VGPR was 29% (95% CI=0.18-0.43, I2=66%). In a NRCT by Dhakal et al. (N=19), Ixa + bendamustine + Dex yielded an ORR 58% (95% CI=0.36-0.77), CR 0, and VGPR 11% (95% CI =0.03-0.34). ≥Grade 3 TRAEs were neutropenia 31%, thrombocytopenia 52%, and diarrhea 10%. In 2 NRCT (N=106), Ixa + cyclophosphamide (Cyc) + Dex yielded a pooled ORR 52% (95% CI=0.42-0.61, I2=0), CR 4% (95% CI=0.01-0.10, I2=0), and VGPR 17% (95% CI=0.11-0.25, I2=0). ≥Grade 3 TRAEs were thrombocytopenia (15%), and upper abdominal pain (4%). In a NRCT by Ludwig et al. (N=90), Ixa + thalidomide (Thal) + Dex yielded an ORR 51% (95% CI=0.41-0.61), CR 9% (95% CI=0.5-0.17), and VGPR 14% (95% CI=0.09-0.23). ≥Grade 3 TRAEs were anemia (17.8%), and infections (16.1%). In a NRCT by Krishnan et al. (N=31), Ixa + Pomalidomide (Pom) + Dex yielded an ORR 48% (95% CI=0.32-0.65) and VGPR 16% (95% CI=0.07-0.33). (Fig 4-6) ≥Grade 3 TRAEs were neutropenia (10%), and lymphopenia (35%). In 2 NRCT by Kumar et al. (N=70) of two drugs combination, Ixa + Dex yielded a pooled ORR 43% (95% CI=0.28-0.59, I2=47%), pooled CR 1% (95% CI=0-0.09, I2=0), and pooled VGPR 24% (95% CI=0.16-0.36, I2=0). ≥Grade 3 TRAEs were hematological (28%), and non-hematological (22.8%). In 2 NRCT of Ixa monotherapy (N=69), pooled ORR was 17% (95% CI=0.10-0.28, I2=0), and pooled CR 6% (95% CI=0.2-0.22, I2=0). (Fig 4-6) ≥Grade 3 TRAEs were anemia (11%), thrombocytopenia (5.4%), and neutropenia (2.7%). Conclusion: Our study provides useful insight into relative efficacy of various Ixa regimens for the treatment of RRMM. The pooled analysis of RCT showed that the combination of Ixa + Len + Dex yielded better response as compared to placebo. In the pooled analysis of outcomes in single arm NRCT, Ixa + Dara + Pom + Dex and Ixa + Len + Dex showed better efficacy outcomes as compared to Ixa + Dex in combination with Thal, Cyc, or Bendamustin. Three drugs Ixa combination regimens had better efficacy as compared to two drugs combination of Ixa + Dex and Ixa monotherapy. Ixa was well tolerated with acceptable safety profile. Additional multicenter, double-blind clinical trials are needed to confirm these results. Disclosures Anwer: Incyte, Seattle Genetics, Acetylon Pharmaceuticals, AbbVie Pharma, Astellas Pharma, Celegene, Millennium Pharmaceuticals.: Honoraria, Research Funding, Speakers Bureau.

Efficacy and safety of direct oral anticoagulants (DOACs) and low molecular weight heparin (LMWH) for primary prevention of venous thromboembolism (VTE) in ambulatory cancer patients.

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 12088-12088
Author(s):  
Mina Shenouda ◽  
Ruchi Desai ◽  
Michael Vishal Jaglal
Keyword(s):  
Cancer Patients ◽  
Randomized Clinical Trials ◽  
Meta Analysis ◽  
Oral Anticoagulants ◽  
Bleeding Event ◽  
Solid Organ ◽  
Efficacy And Safety ◽  
Inclusion Criteria ◽  
Safety Outcomes ◽  
Solid Organ Malignancy

12088 Background: Active malignancy is a well described risk factor for thrombosis. Randomized clinical trials (RCT) have evaluated anticoagulation (AC) with DOACs or LMWH for prevention of VTE in ambulatory cancer patients. This objective of this meta-analysis is to evaluate the efficacy and safety of DOACs and LMWH thromboprophylaxis in adult patients with active solid organ malignancy or lymphoma. Methods: We conducted a search of MEDLINE, EMBASE, and CENTRAL from 10/31/2009-11/31/2019. Data for meta-analysis was extracted from studies that met inclusion criteria (RCT, ambulatory patients age >18 years with active solid organ malignancy or lymphoma, prophylactic AC with DOAC or LMWH). Risk ratio (RR) were calculated for primary (efficacy) end point of VTE occurrence and secondary (safety) end points of major bleeding (MB) and clinically relevant non-major (CRNMB). Subgroup analyses of efficacy and safety endpoints were conducted based on AC and cancer types. Results: Eleven trials met inclusion criteria with total of 7741 participants. Two trials evaluated DOACs and nine trials evaluated LWMH for thromboprophylaxis. Efficacy results are noted in Table. Safety outcomes for MB and CRNMB for AC were RR 1.83 (95% CI 1.26, 2.65), p=0.001 and RR 1.36 (95% CI 1.05, 1.76), p=0.02. Safety outcomes for MB and CRNMB for DOAC were RR 1.95 (95% CI 0.88, 4.30), p=0.10 and RR 1.35 (95% CI 0.80, 2.27), p=0.26. Safety outcomes for MB and CRNMB for LMWH were RR 2.05 (95% CI 1.19, 3.51), p=0.009 and RR 1.44 (95% CI 1.01, 2.05), p=0.04. Conclusions: Both DOACs and LMWH decrease risk for VTE development in ambulatory adult cancer patients. MB and CRNMB were significantly increased in patients taking LMWH but not in patients taking DOACs. A large clinical trial using DOACs for thromboprophylaxis would help elucidate the thrombosis and bleeding event rate in ambulatory cancer patients. [Table: see text]

scholarly journals Hemostasis of idiopathic recurrent epistaxis in children with microwave ablation: a prospective pilot case series

2019 ◽  
Vol 48 (1) ◽  
Author(s):  
Zheng-cai Lou
Keyword(s):  
Microwave Ablation ◽  
Primary Outcome ◽  
Case Series ◽  
University Hospital ◽  
Efficacy And Safety ◽  
Inclusion Criteria ◽  
Recurrent Epistaxis ◽  
Safe Technique ◽  
Chemical Cautery

Abstract Objective We evaluated microwave ablation (MWA) for treatment of idiopathic recurrent anterior epistaxis (RAE) in children, in terms of technical feasibility, efficacy, and safety. Study design A prospective pilot case series. Setting Tertiary university hospital. Methods Children with idiopathic RAE were treated with endoscopic MWA and reevaluated at 1 and 4 weeks and at 6 months thereafter. The primary outcome was successful hemostasis on the day of the procedure. Secondary outcomes were the rebleeding rates after 1 and 4 weeks, and 6 months, and any complications. Results Of the 92 children with idiopathic RAE who met the inclusion criteria, the operation was interrupted in 7 children due to intraoperative noncooperation, and silver nitrate cautery was performed instead. All procedures were completed, and hemostasis was achieved within 10–20 s by MWA in 85 children. Two to four ablations were conducted. No recurrent epistaxis occurred and no severe MWA -related complications, such as septal perforation or synechiae formation, were observed at the 6-month follow-up. Conclusions Endoscopic MWA is a feasible and safe technique for the treatment of idiopathic pediatric RAE in the clinical setting, especially those cases that do not respond to in-office chemical cautery.

scholarly journals How Effective and Safe Is Bronchial Thermoplasty in “Real Life” Asthmatics Compared to Those Enrolled in Randomized Clinical Trials?

2016 ◽  
Vol 2016 ◽  
pp. 1-3 ◽  
Author(s):  
Rita Arrigo ◽  
Giuseppe Failla ◽  
Nicola Scichilone ◽  
Alba La Sala ◽  
Carla Galeone ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Randomized Clinical Trials ◽  
Real Life ◽  
Case Series ◽  
Limited Information ◽  
Efficacy And Safety ◽  
Exclusion Criteria ◽  
Bronchial Thermoplasty ◽  
Pretreatment Period ◽  
Ed Visits

There is limited information on the efficacy and safety of bronchial thermoplasty (BT) inreal life. We evaluated the outcomes of the randomized clinical trials for BT in severe asthmatics, in whom the exclusion criteria were not strictly controlled. A case series of seven asthmatics (M/F: 4/3; age:54.6±2.9years) is reported. Subjects had a statistically significant improvement in AQLQ (from a mean of3.96±1.1to4.5±1.2and5.5±0.6after 6 and 12 months of treatment;p=0.0007) and in the ACQ score (from2.77±0.8to1.83±1.2and1.5±0.8after 6 and 12 months;p<0.001). In the year after BT, severe exacerbations, salbutamol use, and OCS use were significantly lower compared with the 1-yr pretreatment period (p<0.001). No ED visits and hospitalization occurred in the year after BT. No changes in functional parameters were recorded. Our investigation confirms the safety and efficacy of BT in severe asthmatics inreal lifesettings.

scholarly journals COVID-19 convalescent plasma and randomized clinical trials: rebuilding confidence by explaining failures and finding signals of efficacy.

2021 ◽  
Author(s):  
Daniele Focosi ◽  
Massimo Franchini ◽  
Liise-anne Pirofski ◽  
Thierry Burnouf ◽  
Nigel Paneth ◽  
...  
Keyword(s):  
Large Scale ◽  
Randomized Clinical Trials ◽  
Antibody Therapy ◽  
Case Series ◽  
Clinical Settings ◽  
Therapeutic Effects ◽  
Frontline Treatment ◽  
And Function ◽  
Antibody Levels ◽  
Based Medicine

Convalescent plasma (CP) recurs as a frontline treatment in epidemics because it is available as soon as there are survivors. The COVID-19 pandemic represented the first large-scale opportunity to shed light into mechanisms of action, safety and efficacy of convalescent plasma using modern evidence-based medicine approaches. Studies ranging from observational case series to randomized controlled trials (RCT) have reported highly variable efficacy results for COVID-19 CP (CCP), resulting in more doubt than certainty. Reasons for CCP success and failure may be hidden in study details, which are usually difficult to explain to physicians and the public but provide fertile ground for designing next-generation studies. In this paper we analyzed variables associated with efficacy such as clinical settings, disease severity, CCP SARS-CoV-2 antibody levels and function, dose, timing of administration (variously defined as time from onset of symptoms, molecular diagnosis, diagnosis of pneumonia, or hospitalization, or by serostatus), outcomes (defined as hospitalization, requirement for ventilation, clinical improvement or mortality), CCP provenance and time for collection, and criteria for efficacy. Focusing only on the results from the 23 available RCT we noted that these were more likely to show signals of efficacy, including reductions in mortality, if the plasma neutralizing titer was ≥ 160 and the time to randomization was ≤ 9 days, consistent with passive antibody therapy efficacy requiring dosing with sufficient antibody. The fact that most studies revealed signals of efficacy despite variability in CCP and its use suggest robust therapeutic effects that become apparent despite the data noise.

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