Visualizing the target estimand in comparative effectiveness studies with multiple treatments.

Comparative effectiveness research using real-world data often involves pairwise propensity score matching to adjust for confounding bias. We show that corresponding treatment effect estimates may have limited external validity, and propose two visualization tools to clarify the target estimand. We conduct a simulation study to demonstrate, with bivariate ellipses and joy plots, that differences in covariate distributions across treatment groups may affect the external validity of treatment effect estimates.

Arthroscopic partial meniscectomy for the degenerative meniscus tear: a comparison of patients included in RCTs and prospective cohort studies.

Background And Purpose: Concerns exist regarding the generalizability of results from randomized controlled trials (RCTs) evaluating arthroscopic partial meniscectomy (APM) to treat degenerative meniscus tears. It has been suggested that study populations are not representative of subjects selected for surgery in daily clinical practice. Therefore, we aimed to compare patients included in trials and prospective cohort studies that received APM for a degenerative meniscus tear.

The potential value of disease-modifying therapy in patients with spinocerebellar ataxia type 1: an early health economic modeling study.

Objective: There currently is no disease-modifying therapy for spinocerebellar ataxia type 1 (SCA1). Genetic interventions, such as RNA-based therapies, are being developed but those currently available are very expensive. Early evaluation of costs and benefits is, therefore, crucial.

The value of reducing arthroscopic partial meniscectomy in the treatment of degenerative meniscus tears: a budget impact analysis.

Aims: Numerous studies have shown that arthroscopic partial meniscectomy (APM) is not (cost-) effective in patients with symptoms attributed to a degenerative meniscus tear. We aimed to assess the budget impact of reducing APM in routine clinical practice in this population.

Materials And Methods: A patient-level state transition model was developed to simulate patients recently diagnosed with a degenerative meniscus tear.

Bladder EpiCheck urine test in the follow-up of NMIBC: a cost analysis.

Purpose: In the Netherlands yearly more than 5000 patients are diagnosed with non-muscle invasive bladder cancer (NMIBC). With a specificity of 88.0% and a negative predictive value (NPV) for high grade NMIBC of 99.

Confounding adjustment methods in longitudinal observational data with a time-varying treatment: a mapping review.

Objectives: To adjust for confounding in observational data, researchers use propensity score matching (PSM), but more advanced methods might be required when dealing with longitudinal data and time-varying treatments as PSM might not include possible changes that occurred over time. This study aims to explore which confounding adjustment methods have been used in longitudinal observational data to estimate a treatment effect and identify potential inappropriate use of PSM.

Design: Mapping review.

Auditory brainstem response prior to MRI compared to standalone MRI in the detection of vestibular schwannoma: A modelling study.

Objectives: To determine the cost-effectiveness of auditory brainstem response prior to MRI (ABR-MRI) compared to standalone MRI to diagnose vestibular schwannoma.

Design: A state transition model was developed to simulate costs and effects (quality-adjusted life years [QALY]) for both diagnostic strategies for patients suspected of a vestibular schwannoma. Model input was derived from literature, hospital databases and expert opinions.

A cost-effectiveness analysis of three approaches for lymph node assessment in patients with low- and intermediate-risk endometrial cancer.

Objective: To assess the cost-effectiveness of sentinel lymph node mapping compared to risk factor assessment and routine full lymph node dissection for the assessment of lymph nodes in patients with low- and intermediate-risk endometrioid endometrial cancer.

Methods: A decision-analytic model was designed to compare three lymph node assessment strategies in terms of costs and effects: 1) sentinel lymph node mapping; 2) post-operative risk factor assessment (adjuvant therapy based on clinical and histological risk factors); 3) full lymph node dissection. Input data were derived from systematic literature searches and expert opinion.

Intra-articular corticosteroid injections increase the risk of requiring knee arthroplasty.

Aims: Recent studies have suggested that corticosteroid injections into the knee may harm the joint resulting in cartilage loss and possibly accelerating the progression of osteoarthritis (OA). The aim of this study was to assess whether patients with, or at risk of developing, symptomatic osteoarthritis of the knee who receive intra-articular corticosteroid injections have an increased risk of requiring arthroplasty.

Methods: We used data from the Osteoarthritis Initiative (OAI), a multicentre observational cohort study that followed 4,796 patients with, or at risk of developing, osteoarthritis of the knee on an annual basis with follow-up available up to nine years.

Arthroscopic meniscectomy versus non-surgical or sham treatment in patients with MRI confirmed degenerative meniscus lesions: a protocol for an individual participant data meta-analysis.

Introduction: Arthroscopic partial meniscectomy (APM) after degenerative meniscus tears is one of the most frequently performed surgeries in orthopaedics. Although several randomised controlled trials (RCTs) have been published that showed no clear benefit compared with sham treatment or non-surgical treatment, the incidence of APM remains high. The common perception by most orthopaedic surgeons is that there are subgroups of patients that need APM to improve, and they argue that each study sample of the existing trials is not representative for the day-to-day patients in the clinic.

Guidance from key organisations on exploring, confirming and interpreting subgroup effects of medical treatments: a scoping review.

Objectives: With the increasing interest in personalised medicine, the use of subgroup analyses is likely to increase. Subgroup analyses are challenging and often misused, possibly leading to false interpretations of the effect. It remains unclear to what extent key organisations warn for such pitfalls and translate current methodological research to detect these effects into research guidelines.