scholarly journals Randomized Double-Blind Placebo-Controlled Trial of Acetyl-L-Carnitine for the Prevention of Taxane-Induced Neuropathy in Women Undergoing Adjuvant Breast Cancer Therapy

2013 ◽  
Vol 31 (20) ◽  
pp. 2627-2633 ◽  
Author(s):  
Dawn L. Hershman ◽  
Joseph M. Unger ◽  
Katherine D. Crew ◽  
Lori M. Minasian ◽  
Danielle Awad ◽  
...  
Keyword(s):  
Cancer Therapy ◽  
Functional Assessment ◽  
Controlled Trial ◽  
Nutritional Supplement ◽  
Primary Objective ◽  
Double Blind ◽  
Carnitine Level ◽  
Trial Outcome Index ◽  
Adjuvant Breast Cancer ◽  
Outcome Index

PurposeChemotherapy-induced peripheral neuropathy (CIPN) is common and leads to suboptimal treatment. Acetyl-L-carnitine (ALC) is a natural compound involved in neuronal protection. Studies have suggested ALC may be effective for the prevention and treatment of CIPN.Patients and MethodsA 24-week randomized double-blind trial comparing ALC (3,000 mg per day) with placebo in women undergoing adjuvant taxane-based chemotherapy was conducted. The primary objective was to determine if ALC prevents CIPN as measured by the 11-item neurotoxicity (NTX) component of the Functional Assessment of Cancer Therapy (FACT) –Taxane scale at 12 weeks. Secondary objectives included changes in 24-week end points, functional status (FACT–Trial Outcome Index [TOI]), fatigue (Functional Assessment of Chronic Illness Therapy [FACIT] –Fatigue), and NTX grade.ResultsA total of 409 patients were evaluable (208 received ALC; 201, placebo). In a multivariate linear regression, week-12 scores were 0.9 points lower (more CIPN) with ALC than placebo (95% CI, −2.2 to 0.4; P = .17), whereas week-24 scores were 1.8 points lower with ALC (95% CI, −3.2 to −0.4; P = .01). Patients receiving ALC were more likely to have a > 5-point decrease in FACT-NTX scores (38% v 28%; P = .05), and FACT-TOI scores were 3.5 points lower with ALC (P = .03). Grade 3 to 4 neurotoxicity was more frequent in the ALC arm (eight v one). No differences between arms were observed for FACIT-Fatigue or other toxicities. Serum carnitine level increased with ALC but remained stable with placebo.ConclusionThere was no evidence that ALC affected CIPN at 12 weeks; however, ALC significantly increased CIPN by 24 weeks. This is the first study to our knowledge showing that a nutritional supplement increased CIPN. Patients should be discouraged from using supplements without proven efficacy.

Use of the Functional Assessment of Cancer Therapy-Anemia (FACT-An) Instrument in Persons with MPN-Associated Myelofibrosis and Anemia

Blood ◽  
2012 ◽  
Vol 120 (21) ◽  
pp. 4706-4706
Author(s):  
Stacie Hudgens ◽  
Tom Tencer ◽  
Robert Peter Peter Gale ◽  
Zeba M. Khan
Keyword(s):  
Cancer Therapy ◽  
Functional Assessment ◽  
Myeloproliferative Neoplasm ◽  
Well Being ◽  
Change Score ◽  
Patient Reported Outcome ◽  
Double Blind ◽  
Patient Reported ◽  
Outcome Index ◽  
Rbc Transfusion

Abstract Abstract 4706 Background. Myeloproliferative neoplasm (MPN)-associated myelofibrosis is characterized by anemia in almost all affected persons. The Functional Assessment of Cancer Therapy (FACT) measurement system is a patient-reported outcome (PRO) developed to document symptoms to diverse aspects of cancer treatment. One FACT version, FACT-An, specifically addresses symptoms of anemia related to cancer- therapy. FACT-An has been validated in diverse cancer populations but not in MPN-associated myelofibrosis. Methods. Data were from a phase-2 randomized double-blind Bayesian “pick-the-winner” trial of pomalidomide and prednisone in subjects with MPN-associated myelofibrosis and anemia (including RBC-transfusion-dependence). Details of the study, including definitions of anemia, response, RBC-transfusion-dependence and –independence were published previously. Change in quality of life (QOL) from randomization to the last cycle of therapy was evaluated by using the FACT-An physical well-being (PWB), functional well-being (FWB), trial outcome index (TOI) and anemia (An) domains. Minimally clinically important differences (MID) were used to determine the smallest difference in scores which subjects perceived as beneficial in the FACT-An domains of interest. Subjects were classified as meeting MID for responsiveness if their change score from baseline was greater than one standard error of measurement (SEM), indicating improvement. Results. 85 subjects were studied. All FACT-An domains showed strong reliability as measured by Cronbach's alpha. 31 were classified as anemia responders by clinical and laboratory criteria. Anemia responders showed greater improvement in PWB, FWB and TOI scores than non-responders (TOI presented below) across all FACT-An domains. Improvement began at the 2nd 28 d cycle of therapy and was sustained. Conclusion. We show anemia response defined by IWG-MRT criteria correlates with improved QOL measured by the FACT-An instrument in persons with MPN-associated myelofibrosis. Disclosures: Tencer: Celgene Corporation: Employment. Gale:Celgene Corporation: Employment. Khan:Celgene: Employment.

Improvement in symptoms and quality of life (QoL) for patients (p) with non-small cell lung cancer (NSCLC) treated with erloninib: TargeT study

2007 ◽  
Vol 25 (18_suppl) ◽  
pp. 18140-18140
Author(s):  
V. Alberola ◽  
O. Gallego ◽  
G. López-Vivanco ◽  
C. Mesía ◽  
J. Oramas ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Lung Cancer ◽  
Cancer Therapy ◽  
Functional Assessment ◽  
Objective Response ◽  
Well Being ◽  
End Point ◽  
Trial Outcome Index ◽  
Outcome Index

18140 Background: Erlotinib is an EGFR TKI that is effective in the treatment of advanced NSCLC, in terms of longer survival, better quality of life and delayed symptom progression.We present here the outcome of a group of p from the TargeT study, whose QoL was assessed by the Functional Assessment of Cancer Therapy-Lung (FACT-L) questionnaire and the Lung Cancer Subscale (LCS). Methods: TargeT study was a multicenter, single-arm phase II study evaluating efficacy, safety, and tolerability of erlotinib (150 m/day) in p with stage IIIB or IV NSCLC, in 1st, 2nd and 3rd line treatment. Primary end-point was time to progression. QoL was a secondary end point as assessed monthly by the Functional Assessment of Cancer Therapy-Lung questionnaire (FACT- L) and its Lung Cancer Subscale. Physical and functional aspects of the QoL were measured by the Trial Outcome Index (TOI), which is the sum of the physical well being, functional well-being and LCS scores from the FACT-L questionnaire. Results: Data from 91 pts were available. QoL analysis showed that 53% of the p (95% IC 37–58%) had improvement in FACT-L or TOI. Similarly, 45% (95% IC35–56%) of improved their symptoms from baseline. Improvement was observed for each individual LCS item and specifically in the pulmonary items. In symptomatic p, shortened of breath was 17,6% at baseline vs 2.2% after treatment (p<0.001) and cough was 24.7% vs 8.8 % (p<0.001) after treatment. Those improvements in symptoms were rapid and, 73% of the patients who improved showed that recovery in the first cycle of treatment. In terms of association between efficacy of erlotinib and QoL, there is a statistically significative relationship between objective response and improvement in TOI or FACT-L (p<0.02). Conclusions: This QoL analysis confirms that erlotinib improves both symptoms and functional aspect of patients with NSCLC. The improvement in QoL is related with objective response. No significant financial relationships to disclose.

Effects of high-dose Asian ginseng (Panax ginseng) to improve cancer-related fatigue: Results of a double-blind, placebo-controlled randomized controlled trial.

2016 ◽  
Vol 34 (26_suppl) ◽  
pp. 209-209 ◽  
Author(s):  
Sriram Yennu ◽  
Nizar M. Tannir ◽  
Janet L. Williams ◽  
Kenneth R. Hess ◽  
Susan Frisbee-Hume ◽  
...  
Keyword(s):  
Functional Assessment ◽  
Controlled Trial ◽  
Depression Scale ◽  
Primary Objective ◽  
High Dose ◽  
Cancer Type ◽  
Double Blind ◽  
Ginseng Extract ◽  
Cancer Related Fatigue ◽  
Edmonton Symptom Assessment Scale

209 Background: Cancer related fatigue (CRF) is the most common and disabling symptom in cancer.Panax ginseng extract (PG) is frequently used as a nutraceutical treatment for fatigue. There are no well-powered placebo-controlled trials that used validated CRF outcome measures to investigate of PG effects in cancer patients. The primary objective of this trial was to evaluate the effects oral PG versus Placebo (PL) for CRF. Methods: Patients with cancer fatigue ≥ 4/10 on Edmonton Symptom Assessment Scale (ESAS) were eligible. Patients were randomized to either 400mg of standardized PG or matching PL orally twice a day for 28 days. The primary endpoint was change in the Functional Assessment of Chronic Illness-Fatigue (FACIT-F) fatigue subscale from baseline to Day 28. Secondary outcomes were Functional Assessment of Cancer Therapy-General (FACT-G), Hospital Anxiety and Depression Scale (HADS), and ESAS. Results: Total evaluable patients were 112 (56 for PG and PL groups). No significant differences in baseline characteristics between the two groups except cancer type (p = 0.002). There was significant improvement in FACIT fatigue and ESAS fatigue scores in PG and PL groups at Day 15 and Day 29. The mean (SD) of FACIT-F fatigue scores at baseline, Day 15, and Day 29 were 22.6 (10.4), 29.8(10.7), 30.1 (11.6) and 23.8 (9.1), 30.0 (10.1), 30.4 (11.6) for PG and PL respectively. Mean (SD) improvement in the FACIT-F subscale at Day 29 was not significantly different in PG than in the PL group [7.5 (12.7) vs 6.5 (9.9), P = 0.67]. Mean (SD) improvement in the ESAS fatigue, FACT-G, and HADS at Day 29 were also not significantly different in PG than in the PL group. In a multiple linear model analysis, the change in FACIT-F fatigue from Day 0 to Day 29 was negatively correlated with baseline FACIT-F fatigue (p = 0.0005), baseline HADS score (p = 0.032), and male gender (p = 0.023). There were a significantly higher number of any grade of toxicities in PL group than in PG group (33/62 vs. 28/64, p = 0.024). Conclusions: Both PG and Placebo result in a significant improvement in CRF at Day 15 and Day 29. PG was not significantly superior to placebo after 4 weeks of treatment. Further studies are needed. Clinical trial information: NCT01375114.

scholarly journals Early combination therapy with etanercept and methotrexate in JIA patients shortens the time to reach an inactive disease state and remission: results of a double-blind placebo-controlled trial

2021 ◽  
Vol 19 (1) ◽  
Author(s):  
Ekaterina Alexeeva ◽  
Gerd Horneff ◽  
Tatyana Dvoryakovskaya ◽  
Rina Denisova ◽  
Irina Nikishina ◽  
...  
Keyword(s):  
Combination Therapy ◽  
Controlled Trial ◽  
Randomized Study ◽  
Primary Objective ◽  
Disease State ◽  
Inactive Disease ◽  
Open Label ◽  
Standard Regimen ◽  
Double Blind ◽  
Early Combination Therapy

Abstract Background Remission is the primary objective of treating juvenile idiopathic arthritis (JIA). It is still debatable whether early intensive treatment is superior in terms of earlier achievement of remission. The aim of this study was to evaluate the effectiveness of early etanercept+methotrexate (ETA+MTX) combination therapy versus step-up MTX monotherapy with ETA added in refractory disease. Methods A multi-centre, double-blind, randomized study in active polyarticular JIA patients treated with either ETA+MTX (n = 35) or placebo+MTX (n = 33) for up to 24 weeks, followed by a 24-week open-label phase. The efficacy endpoints included pedACR30 criteria improvement at week 12, inactive disease at week 24, and remission at week 48. Patients who failed to achieve the endpoints at week 12 or at week 24 escaped to open-label ETA+MTX. Safety was assessed at each visit. Results By intention-to-treat analysis, more patients in the ETA+MTX group reached the pedACR30 response at week 12 (33 (94.3%)) than in the placebo+MTX group (20 (60.6%); p = 0.001). At week 24, comparable percentages of patients reached inactive disease (11 (31.4%) vs 11 (33.3%)). At week 48, 11 (31.4%) and eight (24.2%) patients achieved remission. The median (+/−IQR) times to achieve an inactive disease state in the ETA+MTX and placebo+MTX groups were 24 (14–32) and 32 (24–40) weeks, respectively. Forty-four (74/100 patient-years) adverse events (AEs) were reported, leading to treatment discontinuation in 6 patients. Conclusions Early combination therapy with ETA+MTX proved to be highly effective compared to the standard step-up regimen. Compared to those treated with the standard regimen, more patients treated with a combination of ETA+MTX reached the pedACR30 response and achieved inactive disease and remission more rapidly.

Optimizing chair time in infusion centers using intravenous cetirizine premedication for the prevention of hypersensitivity infusion reactions.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. e13508-e13508
Author(s):  
Julio Antonio Peguero ◽  
Ahmed Ayad ◽  
Stacia Young-Wesenberg ◽  
Teresa Yang ◽  
Janine North ◽  
...  
Keyword(s):  
Controlled Trial ◽  
Quality Care ◽  
Primary Objective ◽  
Double Blind ◽  
Acute Urticaria ◽  
Number Of Patients ◽  
Study Results ◽  
Secondary Endpoints ◽  
Infusion Reactions ◽  
Anti Cd20

e13508 Background: Oncology infusion centers are increasingly focused on improving operational efficiencies and patient satisfaction, while maintaining quality care. One key component is optimizing chair time, which has been especially important for patient safety during the COVID-19 pandemic to reduce risk of transmission. Many infusions require antihistamine premedication to reduce the risk of hypersensitivity infusion reactions (IRs). The two IV options are IV diphenhydramine and IV cetirizine, which have a quicker onset than oral options and can be administered IV push. In treating acute urticaria, IV cetirizine was shown to be comparable to IV diphenhydramine, with fewer side effects, and it may be effective for preventing IRs with improved chair time. Methods: A randomized, double-blind phase 2 study evaluating premedication with single dose IV cetirizine 10 mg versus IV diphenhydramine 50 mg was conducted in 34 patients receiving paclitaxel, rituximab, its biosimilar or obinutuzumab (first cycle, retreatment after 6 months or with persistent IRs). The primary objective was the incidence of IRs after premedication. Secondary endpoints included sedation due to antihistamines and time to readiness for discharge. Sedation was reported by patients on a scale of 0-4 (0 = none to 4 = extremely severe). No formal statistical analyses were planned given the exploratory nature of the study. Results: Adults primarily with cancer (n = 31 [91%]) were enrolled during the COVID-19 pandemic, from March 25 to November 23, 2020. The median age was 65 and 67 years in the IV cetirizine and diphenhydramine groups, respectively. The number of patients with IRs was 2/17 (11.8%) with IV cetirizine versus 3/17 (17.6%) with IV diphenhydramine. The mean sedation score in the IV cetirizine group compared to the IV diphenhydramine group was lower at all time points, including at discharge (0.1 vs 0.4, respectively). Mean time to discharge was 24 minutes less with IV cetirizine (4.3 hours [1.5]) versus IV diphenhydramine (4.7 hours [1.2]). This difference was greater (30 minutes less) in those ≥65 years of age (4.4 [1.3] vs 4.9 [1.0] hours). Regardless of whether patients received paclitaxel (n = 9) or an anti-CD20 (n = 25), patients had less chair time when premedicated with IV cetirizine. There were fewer treatment-related adverse events (AEs) with IV cetirizine (2 events) than with IV diphenhydramine (4 events). Conclusions: This was the first randomized, controlled trial evaluating IV antihistamine premedication for IRs and chair time. It was shown that IV cetirizine can prevent IRs, with less sedation, fewer related AEs and reduced chair time compared to IV diphenhydramine. This improves infusion center operations and patient experience. Clinical trial information: NCT04189588.

scholarly journals Feasibility randomised multicentre, double-blind, double-dummy controlled trial of anakinra, an interleukin-1 receptor antagonist versus intramuscular methylprednisolone for acute gout attacks in patients with chronic kidney disease (ASGARD): protocol study

BMJ Open ◽  
2017 ◽  
Vol 7 (9) ◽  
pp. e017121 ◽  
Author(s):  
Gowrie Balasubramaniam ◽  
Trisha Parker ◽  
David Turner ◽  
Mike Parker ◽  
Jonathan Scales ◽  
...  
Keyword(s):  
Chronic Kidney Disease ◽  
Kidney Disease ◽  
Controlled Trial ◽  
Interleukin 1 ◽  
Scale Up ◽  
General Information ◽  
Primary Objective ◽  
Retention Rates ◽  
Acute Gout ◽  
Double Blind

IntroductionAcute gout occurs in people with chronic kidney disease, who are commonly older people with comorbidities such as hypertension, heart disease and diabetes. Potentially harmful treatments are administered to these vulnerable patients due to a lack of clear evidence. Newly available treatment that targets a key inflammatory pathway in acute gout attacks provides an opportunity to undertake the first-ever trial specifically looking treating people with kidney disease. This paper describes the protocol for a feasibility randomised controlled trial (RCT) comparing anakinra, a novel interleukin-1 antagonist versus steroids in people with chronic kidney disease (ASGARD).Methods and analysisASGARD is a two-parallel group double-blind, double-dummy multicentre RCT comparing anakinra 100 mg, an interleukin-1 antagonist, subcutaneous for 5 days against intramuscular methylprednisolone 120 mg. The primary objective is to assess the feasibility of the trial design and procedures for a definitive RCT. The specific aims are: (1) test recruitment and retention rates and willingness to be randomised; (2) test eligibility criteria; (3) collect and analyse outcome data to inform sample and power calculations for a trial of efficacy; (4) collect economic data to inform a future economic evaluation estimating costs of treatment and (5) assess capacity of the project to scale up to a national multicentre trial. We will also gather qualitative insights from participants. It aims to recruit 32 patients with a 1:1 randomisation. Information from this feasibility study will help design a definitive trial and provide general information in designing acute gout studies.Ethics and disseminationThe London-Central Ethics Committee approved the protocol. The results will be disseminated in peer-reviewed journals and at scientific conferences.Trial registration numberEudraCT No. 2015-001787-19, NCT/Clinicalstrials.gov No.NCT02578394, pre-results, WHO Universal Trials Reference No. U1111-1175-1977. NIHR Grant PB-PG-0614–34090.

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