ReviewResearch and practice in thrombosis and haemostasis2019
Hemophilia trials in the twenty-first century: Defining patient important outcomes.
Review in Research and practice in thrombosis and haemostasis, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers, 2 of them syntheses 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
28 citing papers in PubMed, 2 syntheses or guidelines pooled it.
- Non-clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B.The Cochrane database of systematic reviews · 2024Pooled it
- Pooled it
- Novel use of a hash-based tokenization for sharing sickle cell disease data without sharing protected health information.Blood advances · 2026Article
- Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST).Thrombosis and haemostasis · 2025Article
- Patient-Centred Management of Well-Controlled Haemophilia: Obtaining Opinions and Definitions Through a Delphi Consensus.Journal of clinical medicine · 2025Review
- Economic Burden of Haemophilia from a Societal Perspective: A Scoping Review.PharmacoEconomics - open · 2025Review
- Bio-hybrid scaffolds combining polyvinyl alcohol and decellularized articular cartilage for the treatment of focal chondral lesions in hemophilic patients.Frontiers in pharmacology · 2025Article
- Mapping the Patient Experience in a Pediatric Hemophilia Unit: Our Patient Journey.Journal of clinical medicine · 2024Article
- [Treatment of haemophilia in Austria].Wiener klinische Wochenschrift · 2024Article
- Concizumab prophylaxis in persons with hemophilia A or B with inhibitors: patient-reported outcome results from the phase 3 explorer7 study.Research and practice in thrombosis and haemostasis · 2024Article
- Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities to transform the care of people with hemophilia.Expert review of hematology · 2023Article
- ACTIVLIM-Hemo: A new self-reported, unidimensional and linear measure of activity limitations in persons with haemophilia.Haemophilia : the official journal of the World Federation of Hemophilia · 2023Article
- Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.Haemophilia : the official journal of the World Federation of Hemophilia · 2022Article
- Untreated bleeds in people with hemophilia A in a noninterventional study and intrapatient comparison after initiating emicizumab in HAVEN 1-3.Research and practice in thrombosis and haemostasis · 2022Article
- Review
- Predictive Modeling Identifies Total Bleeds at 12-Weeks Postswitch to N8-GP Prophylaxis as a Predictor of Treatment Response.Thrombosis and haemostasis · 2022Article
- Patients' perspective on supposedly patient-relevant process and outcome parameters: a cross-sectional survey within the 'PRO patients study'.BMC health services research · 2022Article
- Haemophilia in France: Modelisation of the Clinical Pathway for Patients.International journal of environmental research and public health · 2022Article
- Incorporating the patient voice and patient engagement in GOAL-Hēm: Advancing patient-centric hemophilia care.Research and practice in thrombosis and haemostasis · 2022Article
- Cost-Effectiveness Analysis of rFVIIIFc Versus Contemporary rFVIII Treatments for Patients with Severe Hemophilia A Without Inhibitors in the United States.PharmacoEconomics - open · 2021Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Treatment for hemophilia has advanced dramatically over the past 5 decades. Success of prophylactic therapy in preventing bleeding and decreasing associated complications has established a new standard of care. However, with the advent of gene therapy and treatments that effectively mimic sustained coagulation factor replacement, outcome measures that worked well for assessing factor replacement therapies in past clinical trials need to be reassessed. In addition, while therapies have advanced, so has the science of outcome assessment, including recognition of the importance of patient important and patient reported outcomes. This manuscript reviews strengths and limitations of outcome measures used in hemophilia from both a provider and patient perspective.
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.