Articles: disease.
-
Randomized Controlled Trial Multicenter Study
Acceptance and Commitment Therapy plus usual care for improving quality of life in people with motor neuron disease (COMMEND): a multicentre, parallel, randomised controlled trial in the UK.
Motor neuron disease is a progressive, fatal neurodegenerative disease for which there is no cure. Acceptance and Commitment Therapy (ACT) is a psychological therapy incorporating acceptance, mindfulness, and behaviour change techniques. We aimed to evaluate the effectiveness of ACT plus usual care, compared with usual care alone, for improving quality of life in people with motor neuron disease. ⋯ National Institute for Health and Care Research Health Technology Assessment and Motor Neurone Disease Association.
-
Randomized Controlled Trial Multicenter Study Comparative Study
Aspirin vs Placebo as Adjuvant Therapy for Breast Cancer: The Alliance A011502 Randomized Trial.
Observational studies of survivors of breast cancer and prospective trials of aspirin for cardiovascular disease suggest improved breast cancer survival among aspirin users, but prospective studies of aspirin to prevent breast cancer recurrence are lacking. ⋯ Among participants with high-risk nonmetastatic breast cancer, daily aspirin therapy did not improve risk of breast cancer recurrence or survival in early follow-up. Despite its promise and wide availability, aspirin should not be recommended as an adjuvant breast cancer treatment.
-
Randomized Controlled Trial Multicenter Study
Telephone Health Coaching and Remote Exercise Monitoring (TeGeCoach) in Peripheral Arterial Occlusive Disease—a Randomized Controlled Trial.
Supervised exercise programs are used to treat intermittent claudication (IC). Home-based exercise programs have been developed to lower barriers to participation. We studied the effects of one such exercise program (TeGeCoach) on self-reported walking ability in patients with IC. ⋯ Significant improvements regarding symptom burden demonstrate the benefit of a home-based exercise program and thus expand the opportunities for guideline-oriented treatment of IC. Future studies should additionally address the effect of home-based exercise programs on clinical variables by means of, for example, the 6-minute walk test.
-
Randomized Controlled Trial Multicenter Study
Single Ascending and Multiple-Dose Trial of Zerlasiran, a Short Interfering RNA Targeting Lipoprotein(a): A Randomized Clinical Trial.
Lipoprotein(a) is a causal risk factor for atherosclerotic cardiovascular disease (ASCVD) and calcific aortic stenosis, with no pharmacological treatments approved by regulatory authorities. ⋯ Zerlasiran was well tolerated and reduced lipoprotein(a) concentrations with infrequent administration.
-
Multicenter Study
Exagamglogene Autotemcel for Severe Sickle Cell Disease.
Exagamglogene autotemcel (exa-cel) is a nonviral cell therapy designed to reactivate fetal hemoglobin synthesis by means of ex vivo clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 gene editing of autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) at the erythroid-specific enhancer region of BCL11A. ⋯ Treatment with exa-cel eliminated vaso-occlusive crises in 97% of patients with sickle cell disease for a period of 12 months or more. (CLIMB SCD-121; ClinicalTrials.gov number, NCT03745287.).