ArticleJournal of patient-reported outcomes2023
Clinically important change for the FACIT-Fatigue scale in paroxysmal nocturnal hemoglobinuria: a derivation from international PNH registry patient data.
Article in Journal of patient-reported outcomes, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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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.
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Who cites it
31 citing papers in PubMed, 26 citations in OpenAlex.
- Efficacy and safety of crovalimab versus eculizumab in complement inhibitor-naïve patients with paroxysmal nocturnal hemoglobinuria: Chinese subgroup analysis of the phase 3 COMMODORE 2 study.Annals of hematology · 2026Trial
- Efficacy and safety of vemircopan as monotherapy in patients with paroxysmal nocturnal hemoglobinuria.Blood advances · 2026Trial
- Psychometric performance of the Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue questionnaire among adults with paroxysmal nocturnal hemoglobinuria.Journal of patient-reported outcomes · 2026Trial
- The Effect of Yoga on Cancer-Related Fatigue and Quality of Life Among Racially and Ethnically Diverse Cancer Survivors: A Secondary Data Analysis.Journal of integrative and complementary medicine · 2025Trial
- Patient-reported outcomes in patients with paroxysmal nocturnal hemoglobinuria treated with crovalimab and approved C5 inhibitors in the phase III COMMODORE 2 and 1 studies.Annals of hematology · 2025Trial
- Patient-reported improvements in paroxysmal nocturnal hemoglobinuria treated with iptacopan from 2 phase 3 studies.Blood advances · 2025Trial
- Trial
- The effect of YOCAS©® yoga on cancer-related fatigue and quality of life in older (60+) vs. younger (≤ 59) cancer survivors: Secondary analysis of a nationwide, multicenter, phase 3 randomized controlled trial.Journal of geriatric oncology · 2024Trial
- Moving toward Individual Treatment Goals with Pegcetacoplan in Patients with PNH and Impaired Bone Marrow Function.International journal of molecular sciences · 2024Trial
- Predictors for improvement in patient-reported outcomes: post hoc analysis of a phase 3 randomized, open-label study of eculizumab and ravulizumab in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria.Annals of hematology · 2024Trial
- Physician and Patient Perception of Disease, Symptomatology, and Treatment Administration Preference in Paroxysmal Nocturnal Haemoglobinuria: Results from a Real-World Survey.Advances in therapy · 2026Article
- Long-Term Fatigue After Treatment for Breast Cancer.JAMA network open · 2026Article
- Disease burden and treatment patterns of paroxysmal nocturnal hemoglobinuria in Japan: a real-world survey.International journal of hematology · 2026Article
- Efficacy and Safety of Treatments for Paroxysmal Nocturnal Hemoglobinuria: A Systematic Literature Review.Journal of clinical medicine · 2026Review
- Real life use of ravulizumab in Italian patients with paroxysmal nocturnal hemoglobinuria: evidence from the REACTION observational study.Annals of hematology · 2026Observational
- Article
- Clinical Outcomes and Disease Management for Patients with Paroxysmal Nocturnal Haemoglobinuria in China: Results from a Real-World Study.Drugs - real world outcomes · 2025Article
- Characterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment.Blood advances · 2025Article
- Article
- Article
Corrections and comments
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Authors and funding
8 authors at 6 institutions in 5 countries.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundFatigue is the most common symptom associated with paroxysmal nocturnal hemoglobinuria (PNH). The objective of this analysis was to estimate values that would suggest a clinically important change (CIC) for the functional assessment of chronic illness therapy-fatigue scale (FACIT-Fatigue) in patients with PNH.
methodsAdults with PNH who initiated eculizumab within 28 days of enrollment in the International PNH Registry as of January 2021 with baseline FACIT-Fatigue scores were included in the analysis. Distribution-based estimates of likely difference were calculated using 0.5 × SD and SEM. Anchor-based estimates of CIC considered the European Organization for Research and Treatment of Cancer (EORTC) global health status/quality of life summary score and the EORTC Fatigue Scale score. Changes in anchors and high disease activity (HDA) shift from start of eculizumab treatment to each follow-up visit were then assessed by FACIT-Fatigue score change (≤ 1 CIC, no change, or ≥ 1 CIC).
resultsAt baseline, 93% of 423 patients had fatigue documented in their medical history. The distribution-based estimates for FACIT-Fatigue were 6.5 using 0.5 × SD and 4.6 using SEM; internal consistency was high (α = 0.87). For anchor-based estimates, the FACIT-Fatigue CIC ranged from 2.5 to 15.5, and generally supported 5 points as a reasonable lower end of the value for meaningful individual change. The percentage of patients who changed from having HDA at baseline to no HDA at eculizumab-treated follow-up visits increased over time.
conclusionThese results support the use of 5 points as the CIC for FACIT-Fatigue in patients with PNH, which is within range of the CICs reported in other diseases (3-5 points).
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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.