open label trial
Recently Published Documents


TOTAL DOCUMENTS

1184
(FIVE YEARS 246)

H-INDEX

84
(FIVE YEARS 10)

2022 ◽  
Vol 11 (1) ◽  
Author(s):  
Claire L Wood ◽  
Niamh Morrison ◽  
Michael Cole ◽  
Malcolm Donaldson ◽  
David B Dunger ◽  
...  

Objective Patients with thyrotoxicosis are treated with anti-thyroid drug (ATD) using block and replace (BR) or a smaller, titrated dose of ATD (dose titration, DT). Design A multi-centre, phase III, open-label trial of newly diagnosed paediatric thyrotoxicosis patients randomised to BR/DT. We compared the biochemical response to BR/DT in the first 6 months of therapy. Methods Patients commenced 0.75 mg/kg carbimazole (CBZ) daily with randomisation to BR/DT. We examined baseline patient characteristics, CBZ dose, time to serum thyroid-stimulating hormone (TSH)/free thyroxine (FT4) normalisation and BMI Z-score change. Results There were 80 patients (baseline) and 78 patients (61 female) at 6 months. Mean CBZ dose was 0.9 mg/kg/day (BR) and 0.5 mg/kg/day (DT). There was no difference in time to non-suppressed TSH concentration; 16 of 39 patients (BR) and 11 of 39 (DT) had suppressed TSH at 6 months. Patients with suppressed TSH had higher mean baseline FT4 levels (72.7 vs 51.7 pmol/L; 95% CI for difference 1.73, 31.7; P = 0.029). Time to normalise FT4 levels was reduced in DT (log-rank test, P = 0.049) with 50% attaining normal FT4 at 28 days (95% CI 25, 32) vs 35 days in BR (95% CI 28, 58). Mean BMI Z-score increased from 0.10 to 0.81 at 6 months (95% CI for difference 0.57, 0.86; P < 0.001) and was greatest in patients with higher baseline FT4 concentrations. Conclusions DT-treated patients normalised FT4 concentrations more quickly than BR. Overall, 94% of patients have normal FT4 levels after 6 months, but 33% still have TSH suppression. Excessive weight gain occurs with both BR and DT therapy.


2021 ◽  
Vol 10 (15) ◽  
pp. e289101522660
Author(s):  
Marina Carvalho Martins Madalão ◽  
Andrea Alves Simiqueli ◽  
Tarcísio Lima Filho ◽  
Márcia Cristina Teixeira Ribeiro Vidigal ◽  
Luis Antonio Minim ◽  
...  

This study was to evaluate the influence of information on the addition of omega-3 and its health benefits in the acceptance of dairy dessert. The hedonic thresholds methodology was applied in two trials: blind and open-label. In the blind trial, no information was provided to consumers regarding the formulation of the flans. In the open-label trial, consumers received the information on the addition of omega-3 and its health benefits. The value of compromised acceptance threshold (CAT) was not influenced by the information on the addition of omega-3 and its health benefits. However, there was a high increase in the value of hedonic rejection threshold (HRT) when the consumers were informed of the benefits of omega-3, and it was possible to increase the addition of the compound in the flans by up to 21.03% without causing product rejection, confirming the positive effect of information on health benefits in accptance.


2021 ◽  
Author(s):  
Paul V Suhocki ◽  
James S Ronald ◽  
Anna Mae E Diehl ◽  
David M Murdoch ◽  
P Murali Doraiswamy

Gut-microbiome-inflammation interactions have been linked to neurodegeneration in Alzheimers disease (AD) and other disorders. We hypothesized that treatment with rifaximin, a minimally absorbed gut-specific antibiotic, may modify the neurodegenerative process by changing gut flora and reducing neurotoxic microbial drivers of inflammation. In a pilot, open-label trial, we treated 10 subjects with mild to moderate probable AD dementia (MMSE = 17 + 3) with rifaximin for 3 months. Treatment was associated with a significant reduction in serum neurofilament-light levels (p <0.004) and a significant increase in fecal phylum Firmicutes microbiota. Serum pTau181 and GFAP levels were reduced (effect sizes of -0.41 and -0.48 respectively) but did not reach significance. There was also a non-significant downward trend in serum cytokine IL-6 and IL-13 levels. Increases in stool Erysipelatoclostridium were correlated significantly with reductions in serum pTau 181 and serum GFAP. Insights from this pilot trial are being used to design a larger placebo-controlled clinical trial to determine if specific microbial flora/products underlie neurodegeneration, and whether rifaximin is clinically efficacious as a therapeutic.


Author(s):  
Anja S. Petersen ◽  
Adam S. Pedersen ◽  
Mads C. J. Barloese ◽  
Per Holm ◽  
Ole Pedersen ◽  
...  

Author(s):  
Grzegorz S. Nowakowski ◽  
Wolfgang Willenbacher ◽  
Richard Greil ◽  
Thomas S. Larsen ◽  
Krish Patel ◽  
...  

Blood ◽  
2021 ◽  
Vol 138 (Supplement 1) ◽  
pp. 539-539
Author(s):  
Xinping Zhou ◽  
Fanjun Meng ◽  
Yanjuan Lin ◽  
Zheng Ge ◽  
Yuemin Kuang ◽  
...  

Abstract Background: HMAs are mainstay treatment of higher-risk myelodysplastic syndromes (MDS). However, clinical outcomes of patients treated with decitabine (DEC) monotherapy were far from satisfactory with an overall response rate (ORR) of 33%-55.4% and an overall survival (OS) of 17.7-22 months. Some clinical researches reported that the addition of all-trans retinoic acid (ATRA) to DEC increased response rate and prolonged survival of MDS and elderly acute myeloid leukemia (AML) patients. Our data showed ATRA enhanced the cytotoxic effect of DEC on MDS via activating RARα-Nrf2 complex (2021 EHA abstract EP891). These findings suggested that addition of ATRA to DEC in treatment-naive patients may improve response rate based on the synergetic function. We therefore conducted a study of combination of DEC and ATRA in MDS subtype excessive blasts (EB) patients. Methods: In this randomized, multicenter, open-label trial, patients with newly diagnosed MDS subtype EB based on the 2016 WHO classification from 7 different tertiary medical centers in China were included. Patients were randomized 1:1 to receive either oral ATRA (25mg/m 2/day on days 1-28) plus DEC (20 mg/m 2 daily on days 1-5) or DEC monotherapy(Figure 1). The primary endpoint was overall response rate ORR, defined as complete remission (CR), partial remission (PR), marrow complete remission (mCR), or hematological improvement (HI). Key secondary endpoints were mCR, HI, overall survival (OS), and progress free survival (PFS). Response was assessed after completion of four cycles of treatment. For patients who bridged to allo-HSCT later on and did not complete four cycles of treatments, response was defined as best response ever observed before receiving allo-HSCT. Here, we report results of the interim analysis. Results: Between May 2018 and July 2021, 165 patients were randomly allocated into either DEC plus ATRA (n=82) or DEC monotherapy (n=83). 63.6% of patients were male and 36.4% were female, with a median age of 62 years (range, 19 to 81 years). 38.8% of patients had EB1 and 61.2% had EB2. As of July 31, 2021, 126 patients were available for the assessment of treatment results. After a median follow up of 9.6 months, median number of courses on treatment was 4 courses (range, 1-14), 61 in DEC plus ATRA arm and 65 in DEC arm . OR was achieved in 85.2% of DEC plus ATRA patients compared to 56.9% in DEC monotherapy (p&lt;0.001). mCR rate was 73.8% in patients treated with DEC plus ATRA and 53.8% in those with DEC monotherapy (p=0.02). HI rate was 63.9% and 44.6% in patients with DEC plus ATRA and DEC monotherapy, respectively (p=0.03). The median OS and PFS were 18.8 months and 13.5 months for DEC plus ATRA arms, 19.2 months and 13.0 months for in DEC arm, respectively. 72.7% patients developed at least one adverse event (AE) during the trial, 73.3% in the DEC plus ATRA arm and 72.0% in DEC arm, respectively. Grade 3/4 Hematological toxicity occurred in 73.68 % of DEC plus ATRA arm and 76.92 % of DEC arm. Hyperlipidemia occurred in 31 patients in DEC plus ATRA arm (3.2% grade 3/4), and 18 (no grade 3/4) in DEC arm (p&lt;0.001). Incidence of headache was higher in DEC plus ATRA arm (14.7% vs 4.0% in DEC arm, respectively, p&lt;0.001). No other statistically significant differences were observed in the incidence of treatment-related AEs between the two groups. No early death occurred. Conclusion: Combination of DEC and ATRA achieved an OR rate of 85.2% in MDS subtype EB. Enrollment is ongoing to assess its efficacy and safety in treating EB. This therapy may be a new treatment option for EB subjects. Figure 1 Figure 1. Disclosures No relevant conflicts of interest to declare.


Sign in / Sign up

Export Citation Format

Share Document