Psychometric evaluation of a patient-reported outcomes instrument for congenital thrombotic thrombocytopenic purpura

• Главные авторы: Abiola Oladapo (Автор), Diane Ito (Автор), Ana María Rodriguez (Автор), Stephanie Philpott (Автор), Robert Krupnick (Автор), Veleka Allen (Автор), Christopher Hibbard (Автор), Marie Scully (Автор), Bruce Ewenstein (Автор)
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• Опубликовано: SpringerOpen, 2023-07-01T00:00:00Z.
• Предметы: article
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Abstract Background Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra-rare, life-threatening hereditary disorder that causes patients to experience significant morbidity and decreased health-related quality of life (HRQoL). A cTTP disease-specific patient-reported outcome (PRO) instr...