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

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...

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Main Authors: Abiola Oladapo (Author), Diane Ito (Author), Ana María Rodriguez (Author), Stephanie Philpott (Author), Robert Krupnick (Author), Veleka Allen (Author), Christopher Hibbard (Author), Marie Scully (Author), Bruce Ewenstein (Author)
Format: Book
Published: SpringerOpen, 2023-07-01T00:00:00Z.
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