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Browsing by Author "Oliva, Esther Natalie"
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Appraisal of patient-reported outcome measures in analogous diseases and recommendations for use in phase II and III clinical trials of pyruvate kinase deficiency
Salek, Mir-Saeed Shayegan; Ionova, Tatyana; Johns, Jeffrey; Oliva, Esther Natalie (2018-11-19)Purpose: Pyruvate kinase deficiency (PKD) is a rare disease and understanding of its epidemiology and associated burden remains limited. With no current curative therapy, clinical manifestations can be life threatening, ... -
Guidelines for the Use and Reporting of Patient-Reported Outcomes in Multiple Myeloma Clinical Trials
Laane, Edward; Salek, Sam; Oliva, Esther Natalie; Bennink, Christine; Clavreul, Solène; Richardson, Paul G; Scheid, Christof; Weisel, Katja; Ionova, Tatyana (2023-12-08)Simple Summary: It is recognized that patients with multiple myeloma (MM) experience a high burden of disease and treatment-related symptoms that impact upon their quality of life (QoL). In these patients, patient-reported ... -
The Reporting, Use, and Validity of Patient-Reported Outcomes in Multiple Myeloma in Clinical Trials: A Systematic Literature Review
Salek, Sam; Ionova, Tatiana; Oliva, Esther Natalie; Andreas, Marike; Skoetz, Nicole; Kreuzberger, Nina; Laane, Edward (2022-12-06)Background: Patient-reported outcomes (PROs) are becoming increasingly important in supporting clinical outcomes in clinical trials. In multiple myeloma (MM), PRO measurement is useful to reveal how treatment affects ...