ReviewClinical pharmacology and therapeutics2022
Functional Loss and Mortality in Randomized Clinical Trials for Amyotrophic Lateral Sclerosis: To Combine, or Not to Combine-That is the Estimand.
Review in Clinical pharmacology and therapeutics, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers, 1 of them a synthesis that pooled it.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
7 citing papers in PubMed, 1 synthesis or guideline pooled it.
- Pooled it
- Heterogeneity in the Analysis of the ALSFRS-R in ALS Clinical Trials and its Effect on the Validity and Precision of Trial Conclusions.Neurology · 2026Article
- Design considerations for C9orf72 disease prevention trials.Brain : a journal of neurology · 2025Review
- Handling rescue therapy in myasthenia gravis clinical trials: why it matters and why you should care.Annals of clinical and translational neurology · 2025Review
- Role of mitochondrial quality control in neurodegenerative disease progression.Frontiers in cellular neuroscience · 2025Review
- Predicting risk factors for pediatric mortality in clinical trial research: A retrospective, cross-sectional study using a Healthcare Cost and Utilization Project database.Journal of clinical and translational science · 2023Article
- Joint modeling of endpoints can be used to answer various research questions in randomized clinical trials.Journal of clinical epidemiology · 2022Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
5 authors.
Funding
Abstract
Amyotrophic lateral sclerosis is a rapidly progressive disease leading to death in, on average, 3-5 years after first symptom onset. Consequently, there are frequently a non-negligible number of patients who die during the course of a clinical trial. This introduces bias in end points such as daily functioning, muscle strength, and quality of life. In this paper, we outline how the choice of strategy to handle death affects the interpretation of the trial results. We provide a general overview of the considerations, positioned in the estimand framework, and discuss the possibility that not every strategy provides a clinically relevant answer in each setting. The relevance of a strategy changes as a function of the intended trial duration, hypothesized treatment effect, and population included. It is important to consider this trade-off at the design stage of a clinical trial, as this will clarify the exact research question that is being answered, and better guide the planning, design, and analysis of the study.
Indexed as
Identifiers
What OpenQuestion holds
Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.