Validation of the PRUCISION Instruments in Pediatric Patients with Progressive Familial Intrahepatic Cholestasis.
Adv Ther
; 39(11): 5105-5125, 2022 11.
Article
em En
| MEDLINE
| ID: mdl-36066745
Progressive familial intrahepatic cholestasis (PFIC) is a collection of liver diseases that typically affects very young children. A problematic symptom of PFIC is extremely itchy skin, or pruritus, that can keep patients and their families up at night. The PRUCISION questionnaire was developed to measure the severity of a patient's pruritus and sleep disturbance from the perspective of the patient's caregiver. The current study had two primary goals: (1) to assess whether PRUCISION could reliably measure these symptoms and detect changes over time relative to other established rating scales that assess related concepts, and (2) to identify what score change on PRUCISION could be considered clinically meaningful. To do this, data from a clinical study, called PEDFIC 1, in patients with PFIC were used: patient's scores on PRUCISION from their caregiver's perspective were compared with scores on other established scales, first before any treatment was given in PEDFIC 1, and then again after 24 weeks of treatment with a drug called odevixibat. In general, there was good agreement between PRUCISION scores and scores on other scales. For example, when PRUCISION scores indicated that symptoms improved, this tended to correlate with improvement on other measures. Additionally, these analyses indicated that if the PRUCISION score drops by 1 point or more, that can be considered a clinically important change. Overall, this study found that the caregiver-reported PRUCISION questionnaire is valid for assessing changes in pruritus and sleep symptoms in patients with PFIC, which may benefit patients as new treatments are developed.
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Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Colestase Intra-Hepática
Tipo de estudo:
Diagnostic_studies
/
Etiology_studies
/
Prognostic_studies
Limite:
Child
/
Humans
Idioma:
En
Ano de publicação:
2022
Tipo de documento:
Article