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Psychometric validation and interpretation thresholds of the Hidradenitis Suppurativa Quality of Life (HiSQOL©) questionnaire using pooled data from the phase III BE HEARD I & II trials of bimekizumab in hidradenitis suppurativa Open
Background Hidradenitis suppurativa (HS) is a chronic, inflammatory skin condition which negatively impacts patients’ physical and mental wellbeing. The HS Quality of Life questionnaire (HiSQOL©) was developed to assess HS-specific changes…
Psychometric properties and meaningful change thresholds for the QOL-E instrument in patients with myelodysplastic neoplasms Open
Background Myelodysplastic neoplasms (MDS) are characterized by ineffective hematopoiesis, peripheral blood cytopenias, and an increased risk of progression to acute myeloid leukemia. One of the main treatment goals is improving quality of…
View article: Health-Related Quality of Life of Luspatercept Versus Epoetin Alfa in Red Blood Cell Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes: Results from the Final Datacut of the Phase 3 COMMANDS Study
Health-Related Quality of Life of Luspatercept Versus Epoetin Alfa in Red Blood Cell Transfusion-Dependent Lower-Risk Myelodysplastic Syndromes: Results from the Final Datacut of the Phase 3 COMMANDS Study Open
Introduction: Treatment goals in lower-risk myelodysplastic syndromes (LR-MDS) include reduction of red blood cell (RBC) transfusion burden, improvement of cytopenias, and maintenance of, or improvement in, health-related quality of life (…
Luspatercept Improves Fatigue-Related Quality of Life through 5 Years of Treatment in Non-Transfusion Dependent Beta-Thalassemia Open
Introduction: Luspatercept has been shown to increase hemoglobin levels and improve health-related quality of life (HRQoL) in patients with non-transfusion-dependent beta-thalassemia (NTDT) participating in the phase 2, double-blind random…
Estimation of Health State Utility Values for Patients Undergoing First-Line Treatment of Advanced-Stage Classical Hodgkin Lymphoma in the HD21 Trial Open
Introduction: HD21 (NCT02661503) is a phase 3, randomized, open-label study evaluating 4 or 6 cycles of BrECADD vs eBEACOPP as a first-line treatment (tx) of advanced classical Hodgkin lymphoma.Results showed that BrECADD had a more favora…
Identifying thresholds for meaningful improvements in NTDT-PRO scores to support conclusions about treatment benefit in clinical studies of patients with non-transfusion-dependent beta-thalassaemia: analysis of pooled data from a phase 2, double-blind, placebo-controlled, randomised trial Open
Objectives To estimate thresholds for defining meaningful within-patient improvement from baseline to weeks 13–24 and interpreting meaningfulness of between-group difference for the non-transfusion-dependent beta-thalassaemia patient-repor…
Validity and score interpretation of the 12-item Psoriatic Arthritis Impact of Disease: an analysis of pooled data from two phase 3 trials of bimekizumab in patients with psoriatic arthritis Open
Objectives To investigate psychometric performance of the 12-item Psoriatic Arthritis Impact of Disease (PsAID-12) total and individual item scores in patients with psoriatic arthritis (PsA) and to estimate score change thresholds and scor…
Calibration and validation of modeled 5-year survival predictions among people with cystic fibrosis treated with the cystic fibrosis transmembrane conductance regulator modulator ivacaftor using United States registry data Open
Objectives Cystic fibrosis (CF) is a rare genetic disease characterized by life-shortening lung function decline. Ivacaftor, a CF transmembrane conductance regulator modulator (CFTRm), was approved in 2012 for people with CF with specific …
Psychometric evaluation of the NTDT-PRO questionnaire for assessing symptoms in patients with non-transfusion-dependent beta-thalassaemia Open
Objectives The non-transfusion-dependent beta-thalassaemia-patient-reported outcome (NTDT-PRO) questionnaire was developed for assessing anaemia-related tiredness/weakness (T/W) and shortness of breath (SoB) among patients with NTDT. Psych…
Impact of Lenalidomide Treatment on Overall Survival in Patients With Lower-Risk, Transfusion-Dependent Myelodysplastic Syndromes Open
This analysis of prospective trial data in patients with LR-MDS confirms lenalidomide may improve OS by reducing TB and serum ferritin. OS should be considered as an endpoint in future lower risk MDS clinical trials.
Modeling long-term health outcomes of patients with cystic fibrosis homozygous for <i>F508del-CFTR</i> treated with lumacaftor/ivacaftor Open
Background: Lumacaftor/ivacaftor combination therapy is efficacious and generally safe for patients with cystic fibrosis (CF) homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation. However, long-term survival be…