scholarly journals Efficacy and safety of octanorm (cutaquig®) in adults with primary immunodeficiencies with predominant antibody deficiency: a prospective, open-label study

Immunotherapy ◽  
2020 ◽  
Vol 12 (5) ◽  
pp. 299-309
Author(s):  
Elena Latysheva ◽  
Yulia Rodina ◽  
Liudmila Sizyakina ◽  
Areg Totolian ◽  
Irina Tuzankina
Keyword(s):  
Quality Of Life ◽  
Bacterial Infections ◽  
Short Form ◽  
Primary Immunodeficiencies ◽  
Phase Iii ◽  
Efficacy And Safety ◽  
Open Label ◽  
Clinical Trial Registration ◽  
Registration Number

Aim: To evaluate efficacy and safety of octanorm (16.5% subcutaneous immunoglobulin) in adult patients with primary immunodeficiencies. Patients & methods: Primary immunodeficiencies patients (18–70 years) previously treated with intravenous immunoglobulin were included in this Phase III study. Octanorm was administered subcutaneously once weekly over 8 months. End points included infections, adverse events and quality of life. Results: 25 patients (mean age 35.2 years, female 60.0%) were recruited, 24 completed the study. Mean dose of octanorm was 0.11 g/kg/week. No serious bacterial infections occurred. Three patients (12.0%) had an adverse event (mild) assessed as related to octanorm. Both the mental and physical summary 36-item Short Form Health Survey scores were improved. Conclusion: Octanorm is effective, safe and improves quality of life. Clinical Trial registration number: NCT03988426.

Effects of BG-12 (dimethyl fumarate) on health-related quality of life in patients with relapsing–remitting multiple sclerosis: findings from the CONFIRM study

2013 ◽  
Vol 20 (2) ◽  
pp. 253-257 ◽  
Author(s):  
Mariko Kita ◽  
Robert J Fox ◽  
J Theodore Phillips ◽  
Michael Hutchinson ◽  
Eva Havrdova ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Multiple Sclerosis ◽  
Short Form ◽  
Dimethyl Fumarate ◽  
Phase Iii ◽  
Health Related Quality ◽  
Relapsing Remitting ◽  
Related Quality ◽  
Health Related

Multiple sclerosis (MS) has a significant impact on health-related quality of life (HRQoL) with symptoms adversely affecting many aspects of everyday living. BG-12 (dimethyl fumarate) demonstrated significant efficacy in the phase III studies DEFINE and CONFIRM in patients with relapsing–remitting MS. In CONFIRM, HRQoL was worse in patients with greater disability at baseline, and who relapsed during the study, and improved with BG-12 treatment. Mean Short Form-36 Physical Component Summary scores for BG-12 increased over 2 years and scores for placebo decreased. Coupled with clinical and neuroradiological benefits, these HRQoL results further support BG-12 as an effective oral treatment for relapsing MS.

An Open-Label, Analgesic Efficacy and Safety of Pituitary Radiosurgery for Patients With Opioid-Refractory Pain: Study Protocol for a Randomized Controlled Trial

Neurosurgery ◽  
2017 ◽  
Vol 83 (1) ◽  
pp. 146-153 ◽  
Author(s):  
Pierre-Yves Borius ◽  
Stéphanie Ranque Garnier ◽  
Karine Baumstarck ◽  
Frédéric Castinetti ◽  
Anne Donnet ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Pain Relief ◽  
Cancer Pain ◽  
Controlled Trial ◽  
Analgesic Efficacy ◽  
Standards Of Care ◽  
Control Group ◽  
Efficacy And Safety ◽  
Open Label

Abstract BACKGROUND Hypophysectomy performed by craniotomy or percutaneous techniques leads to complete pain relief in more than 70% to 80% of cases for opioid refractory cancer pain. Radiosurgery could be an interesting alternative approach to reduce complications. OBJECTIVE To assess the analgesic efficacy compared with standard of care is the primary goal. The secondary objectives are to assess ophthalmic and endocrine tolerance, drug consumption, quality of life, and mechanisms of analgesic action. METHODS The trial is multicenter, randomized, prospective, and open-label with 2 parallel groups. This concerns patients in palliative care suffering from nociceptive or mixed cancer pain, refractory to standard opioid therapy. Participants will be randomly assigned to the control group receiving standards of care for pain according to recommendations, or to the experimental group receiving a pituitary GammaKnife (Elekta, Stockholm, Sweden) radiosurgery (160 Gy delivered in pituitary gland) associated with standards of care. Evaluation assessments will be taken at baseline, day0, day4, day7, day14, day28, day45, month3, and month6. EXPECTED OUTCOMES We could expect pain improvement in 70% to 90% of cases at day4. In addition we will assess the safety of pituitary radiosurgery in a vulnerable population. The secondary endpoints could show decay of opioid consumption, good patient satisfaction, and improvement of the quality of life. DISCUSSION The design of this study is potentially the most appropriate to demonstrate the efficacy and safety of radiosurgery for this new indication. New recommendations could be obtained in order to improve pain relief and quality of life.

scholarly journals Experience of patisiran with transthyretin stabilizers in patients with hereditary transthyretin-mediated amyloidosis

2020 ◽  
Vol 10 (5) ◽  
pp. 289-300 ◽  
Author(s):  
Hollis Lin ◽  
Madeline Merkel ◽  
Cecilia Hale ◽  
Jing L Marantz
Keyword(s):  
Quality Of Life ◽  
Diabetic Neuropathy ◽  
Phase Ii ◽  
Phase Iii ◽  
Open Label ◽  
Safety And Efficacy ◽  
Impairment Score ◽  
Open Label Extension ◽  
Post Hoc

Aim: Examine safety and pharmacodynamics of patisiran alone or with concomitant transthyretin stabilizers from the Phase II open-label extension study and safety and efficacy of patisiran in patients with prior transthyretin stabilizer use from the Phase III APOLLO study. Patients & methods: Post hoc analyses in patients with hereditary transthyretin-mediated amyloidosis with polyneuropathy. Results: Patisiran safety was consistent regardless of concomitant or prior transthyretin stabilizers. In the Phase II open-label extension (n = 27), transthyretin reduction was similar over 24 months, regardless of concomitant transthyretin stabilizers. In APOLLO (n = 225), patisiran-treated groups showed stabilization or improvements in neurological function (modified Neuropathy Impairment Score +7) and quality of life (Norfolk Quality of Life-Diabetic Neuropathy questionnaire) at 18 months, regardless of prior transthyretin stabilizers. Conclusion: Patients benefit from patisiran regardless of transthyretin stabilizer use.

scholarly journals Quality of Life From Canadian Cancer Trials Group MA.17R: A Randomized Trial of Extending Adjuvant Letrozole to 10 Years

2018 ◽  
Vol 36 (6) ◽  
pp. 563-571 ◽  
Author(s):  
Julie Lemieux ◽  
Michael D. Brundage ◽  
Wendy R. Parulekar ◽  
Paul E. Goss ◽  
James N. Ingle ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Aromatase Inhibitor ◽  
Short Form ◽  
Phase Iii ◽  
Secondary Outcome ◽  
Secondary Outcome Measure ◽  
Change Scores ◽  
Sf 36 ◽  
Cancer Trials

Purpose MA.17R was a Canadian Cancer Trials Group–led phase III randomized controlled trial comparing letrozole to placebo after 5 years of aromatase inhibitor as adjuvant therapy for hormone receptor–positive breast cancer. Quality of life (QOL) was a secondary outcome measure of the study, and here, we report the results of these analyses. Methods QOL was measured using the Short Form-36 (SF-36; two summary scores and eight domains) and menopause-specific QOL (MENQOL; four symptom domains) at baseline and every 12 months up to 60 months. QOL assessment was mandatory for Canadian Cancer Trials Group centers but optional for centers in other groups. Mean change scores from baseline were calculated. Results One thousand nine hundred eighteen women were randomly assigned, and 1,428 women completed the baseline QOL assessment. Compliance with QOL measures was > 85%. Baseline summary scores for the SF-36 physical component summary (47.5 for letrozole and 47.9 for placebo) and mental component summary (55.5 for letrozole and 54.8 for placebo) were close to the population norms of 50. No differences were seen between groups in mean change scores for the SF-36 physical and mental component summaries and the other eight QOL domains except for the role-physical subscale. No difference was found in any of the four domains of the MENQOL Conclusion No clinically significant differences were seen in overall QOL measured by the SF-36 summary measures and MENQOL between the letrozole and placebo groups. The data indicate that continuation of aromatase inhibitor therapy after 5 years of prior treatment in the trial population was not associated with a deterioration of overall QOL.

scholarly journals A timed activity protocol to address sleep-wake disorders in home dwelling persons living with dementia: the healthy patterns clinical trial

2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Nancy A. Hodgson ◽  
Nalaka Gooneratne ◽  
Adriana Perez ◽  
Sonia Talwar ◽  
Liming Huang
Keyword(s):  
Quality Of Life ◽  
Clinical Trial ◽  
Trial Registration ◽  
Phase Iii ◽  
Poor Sleep ◽  
Sleep Wake Disorders ◽  
Clinical Trial Registration ◽  
Link Type ◽  
Home Based

Abstract Background Sleep-wake disorders occur in most persons living with dementia and include late afternoon or evening agitation, irregular sleep-wake rhythms such as daytime hypersomnia, frequent night awakenings, and poor sleep efficiency. Sleep-wake disorders pose a great burden to family caregivers, and are the principal causes of distress, poor quality of life, and institutionalization. Regulating the sleep-wake cycle through the use of light and activity has been shown to alter core clock processes and suggests that a combination of cognitive, physical, and sensory-based activities, delivered at strategic times, may be an effective mechanism through which to reduce sleep-wake disorders. Methods A definitive Phase III efficacy trial of the Healthy Patterns intervention, a home-based activity intervention designed to improve sleep-wake disorders and quality of life, is being conducted using a randomized two-group parallel design of 200 people living with dementia and their caregivers (dyads). Specific components of this one-month, home-based intervention involve 4 in-home visits and includes: 1) assessing individuals’ functional status and interests; 2) educating caregivers on environmental cues to promote activity and sleep; and 3) training caregivers in using timed morning, afternoon, and evening activities based on circadian needs across the day. The patient focused outcomes of interest are quality of life, measures of sleep assessed by objective and subjective indicators including actigraphy, subjective sleep quality, and the presence of neuropsychiatric symptoms. Caregiver outcomes of interest are quality of life, burden, confidence using activities, and sleep disruption. Salivary measures of cortisol and melatonin are collected to assess potential intervention mechanisms. Discussion The results from the ongoing study will provide fundamental new knowledge regarding the effects of timing activity participation based on diurnal needs and the mechanisms underlying timed interventions which can lead to a structured, replicable treatment protocol for use with this growing population of persons living with dementia. Clinical trial registration Clinicaltrials.gov # NCT03682185 at https://clinicaltrials.gov/; Date of clinical trial registration: 24 September 2018.

scholarly journals Efficacy and safety of two Ayurvedic dosage forms for Allergic rhinitis: Study protocol for an open-label randomized controlled trial

2019 ◽  
Author(s):  
JM Dahanayake ◽  
Pathirage Kamal Perera ◽  
P Galappaththy ◽  
D Samaranayake
Keyword(s):  
Quality Of Life ◽  
Allergic Rhinitis ◽  
Controlled Trial ◽  
Dosage Forms ◽  
Efficacy And Safety ◽  
Open Label ◽  
Randomized Controlled ◽  
Nasal Symptoms ◽  
Freeze Dried

Abstract Background: Allergic rhinitis (AR) is an immune response of the nasal mucosa to airborne allergens and involves nasal congestion, watery nasal discharge, itching of the nose and sneezing. The symptoms of allergic rhinitis may significantly affect a patient’s quality of life and can be associated with conditions such as fatigue, headache, cognitive impairment and sleep disturbances. Various complementary and alternative medicine treatments have been used for this condition in clinical practice. The Ayurveda system of medicine is the most common complementary medicine system practiced in Sri Lanka. The aim of this study is to examine the efficacy and safety of a decoction used in traditional Ayurveda for allergic rhinitis and it’s ready to use freeze dried formulation in comparison to an antihistamine over a period of 4 weeks on relief of symptoms in allergic rhinitis. Study design: This is a three arm, open label, non-inferiority, randomized controlled clinical trial enrolling patients with AR. Tamalakyadi decoction containing 12 ingredients (TMD12), used in traditional Ayurveda and its freeze dried formulation are the test products. The efficacy and safety of the two Ayurvedic dosage forms will be tested against the antihistamine loratidine Patients with symptoms of AR will be allocated randomly into the 3 arms after a 1 week run-in period and the medications will be given orally for 28 days. Total Nasal symptom Score (TNSS) of the patients will be used as the primary efficacy endpoint. TNSS will be recorded and compared between the 3 arms prior to visit 1, at the end of 28 days, end of the and second months of follow up. Symptom scores of daytime nasal symptoms, night time nasal symptoms, non-nasal symptoms and Health Related Quality of Life questionnaire are used as secondary end points. Discussion: This clinical trial will be able to provide evidence based scientific data on Ayurvedic dosage form, TMD12 and the freeze-dried formulation in the treatment of allergic rhinitis. This trial is expected to develop capacity to scientifically evaluate various Ayurvedic treatments that are claimed to have efficacy in treatment of various disease conditions. Trial registration:ISRCTN18149439 (06 May 2019) Keywords: Allergic rhinitis, Ayurvedic dosage forms, Tamalakyadi decoctions, Randomized controlled trial

scholarly journals Efficacy and safety of dolutegravir plus emtricitabine versus standard ART for the maintenance of HIV-1 suppression: 48-week results of the factorial, randomized, non-inferiority SIMPL’HIV trial

PLoS Medicine ◽  
2020 ◽  
Vol 17 (11) ◽  
pp. e1003421
Author(s):  
Delphine Sculier ◽  
Gilles Wandeler ◽  
Sabine Yerly ◽  
Annalisa Marinosci ◽  
Marcel Stoeckle ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Adverse Events ◽  
Viral Suppression ◽  
Safety Data ◽  
Risk Difference ◽  
Hiv Treatment ◽  
Efficacy And Safety ◽  
Open Label ◽  
Hiv 1

Background Dolutegravir (DTG)–based dual therapy is becoming a new paradigm for both the initiation and maintenance of HIV treatment. The SIMPL’HIV study investigated the outcomes of virologically suppressed patients on standard combination antiretroviral therapy (cART) switching to DTG + emtricitabine (FTC). We present the 48-week efficacy and safety data on DTG + FTC versus cART. Methods and findings SIMPL’HIV was a multicenter, open-label, non-inferiority randomized trial with a factorial design among treatment-experienced people with HIV in Switzerland. Participants were enrolled between 12 May 2017 and 30 May 2018. Patients virologically suppressed for at least 24 weeks on standard cART were randomized 1:1 to switching to DTG + FTC or to continuing cART, and 1:1 to simplified patient-centered monitoring versus standard monitoring. The primary endpoint was the proportion of patients virologically suppressed with <100 copies/ml through 48 weeks. The secondary endpoints included virological suppression at 48 weeks according to the US Food and Drug Administration (FDA) snapshot analysis. Non-inferiority of DTG + FTC versus cART for viral suppression was assessed using a stratified Mantel–Haenszel risk difference, with non-inferiority declared if the lower bound of the 95% confidence interval was greater than −12%. Adverse events were monitored to assess safety. Quality of life was evaluated using the PROQOL-HIV questionnaire. Ninety-three participants were randomized to DTG + FTC, and 94 individuals to cART. Median nadir CD4 count was 246 cells/mm3; median age was 48 years; 17% of participants were female. DTG + FTC was non-inferior to cART. The proportion of patients with viral suppression (<100 copies/ml) through 48 weeks was 93.5% in the DTG + FTC arm and 94.7% in the cART arm in the intention-to-treat population (risk difference −1.2%; 95% CI −7.8% to 5.6%). Per-protocol analysis showed similar results, with viral suppression in 96.5% of patients in both arms (risk difference 0.0%; 95% CI −5.6% to 5.5%). There was no relevant interaction between the type of treatment and monitoring (interaction ratio 0.98; 95% CI 0.85 to 1.13; p = 0.81). Using the FDA snapshot algorithm, 84/93 (90.3%) participants in the DTG + FTC arm had an HIV-1 RNA viral load of <50 copies/ml compared to 86/94 (91.5%) participants on standard cART (risk difference −1.1%; 95% CI −9.3% to 7.1%; p = 0.791). The overall proportion of patients with adverse events and discontinuations did not differ by randomization arm. The proportion of patients with serious adverse events was higher in the cART arm (16%) compared to the DTG + FTC arm (6.5%) (p = 0.041), but none was considered to be related to the study medication. Quality of life improved more between baseline and week 48 in the DTG + FTC compared to the cART arm (adjusted difference +2.6; 95% CI +0.4 to +4.7). The study’s main limitations included a rather small proportion of women included, the open label design, and its short duration. Conclusions In this study, DTG + FTC as maintenance therapy was non-inferior to cART in terms of efficacy, with a similar safety profile and a greater improvement in quality of life, thus expanding the offer of 2-drug simplification options among virologically suppressed individuals. Trial registration ClinicalTrials.gov NCT03160105.

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