How does risdiplam compare with other treatments for Types 1-3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison.

To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. In Type 1 SMA, studies of risdiplam and nusinersen included similar populations.

Comparative Efficacy of Atezolizumab plus Bevacizumab and Other Treatment Options for Patients with Unresectable Hepatocellular Carcinoma: A Network Meta-Analysis.

Background: Most phase 3 clinical trials of systemic therapy for first-line unresectable hepatocellular carcinoma (HCC) have failed, with the exception of SHARP, REFLECT, and IMbrave150. We conducted indirect comparisons of therapies evaluated for first-line HCC treatment.

Summary: We conducted a systematic review and meta-analysis of treatments for adults with locally advanced or metastatic unresectable HCC and no prior systemic treatment, including atezolizu-mab plus bevacizumab, sorafenib, lenvatinib, nivolumab, selective internal radiotherapy (SIRT), transarterial chemoembolization, and placebo or best supportive care.

Prognostic Factors and Treatment-Effect Modifiers in Spinal Muscular Atrophy.

Spinal muscular atrophy (SMA) is a rare, progressive neuromuscular disease characterized by loss of motor neurons and muscle atrophy. Untreated infants with type 1 SMA do not achieve major motor milestones, and death from respiratory failure typically occurs before 2 years of age. Individuals with types 2 and 3 SMA exhibit milder phenotypes and have better functional and survival outcomes.

Matching-adjusted indirect comparison: entrectinib versus crizotinib in fusion-positive non-small cell lung cancer.

To perform indirect treatment comparisons of entrectinib versus alternative fusion-positive non-small cell lung cancer treatments. Relevant studies with crizotinib and chemotherapy as comparators of interest identified by systematic literature review were selected for matching-adjusted indirect comparison by feasibility assessment. Matching was based on known prognostic/predictive factors and scenario analyses were used for unreported confounders in comparator trials.

Systematic Review and Network Meta-analysis of Idiopathic Pulmonary Fibrosis Treatments.

Background: The antifibrotics pirfenidone and nintedanib are both approved for the treatment of idiopathic pulmonary fibrosis (IPF) by regulatory agencies and are recommended by health technology assessment bodies. Other treatments such as N-acetylcysteine are used in clinical practice but have not received regulatory approval. No head-to-head trials have been conducted to directly compare the efficacy of these therapies in IPF.

Outcomes after orthopedic trauma.

Background: Clinical orthopedic research needs better ability to assess patient expectations with regard to orthopedic trauma surgery outcomes. The aim of this study was to investigate to which extent patient expectations prior to surgery could be met after surgery.

Methods: Patients (≥18 years) with surgical ankle fractures were prospectively recruited at 5 orthopedic trauma clinics in the United States (USA), Canada, and Brazil and followed up for 12 months.

Effect of pirfenidone on mortality: pooled analyses and meta-analyses of clinical trials in idiopathic pulmonary fibrosis.

Background: In clinical trials of idiopathic pulmonary fibrosis, rates of all-cause mortality are low. Thus prospective mortality trials are logistically very challenging, justifying the use of pooled analyses or meta-analyses. We did pooled analyses and meta-analyses of clinical trials of pirfenidone versus placebo to determine the effect of pirfenidone on mortality outcomes over 120 weeks.

Decrease of morbidity in road traffic accidents in a high income country - an analysis of 24,405 accidents in a 21 year period.

Background: The WHO initiated the "Decade of Action for Road Safety" because the fatality on road traffic accidents could become the fifth leading cause of death in 2030. On the contrary, fatalities continue to decrease in high income countries. The aim of the study was to find evidence for changes in injury severity of passenger car occupants after road traffic accidents in Germany over time, and to find contributing factors.

TEFTOM: A Promising General Trauma Expectation/Outcome Measure-Results of a Validation Study on Pan-American Ankle and Distal Tibia Trauma Patients.

Background. In orthopedics, there is no instrument specifically designed to assess patients' expectations of their final surgery outcome in general trauma populations. We developed the Trauma Expectation Factor Trauma Outcome Measure (TEFTOM) to investigate the fulfilment of patients' expectations one year after surgery as a measure of general trauma surgical outcomes.

How to document and report orthopedic complications in clinical studies? A proposal for standardization.

Introduction: The documentation of complications is critical for the evaluation of therapeutic interventions in orthopedics. However, there is a lack of accepted methodological standardization and definitions. We propose a concept to support the consensus development of a standardized management and classification of complications in clinical research.