THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPA
Not Applicable
- Conditions
- Paroxysmal Nocturnal Hemoglobinuria (PNH)
- Registration Number
- JPRN-UMIN000050211
- Lead Sponsor
- Swedish Orphan Biovitrum Japan
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- All
- Target Recruitment
- 68
Inclusion Criteria
Not provided
Exclusion Criteria
-Correspond to an inappropriate answer
Study & Design
- Study Type
- Others,meta-analysis etc
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Self-reported hematological and clinical measures (such as diagnosis of ongoing anemia, levels of hemoglobin, history of red blood cell transfusions, history of thrombotic events, and history of renal function impairment) Quality of life assessed using PRO scales (EQ-5D-5L and EORTC) Fatigue and depression state assessed using PRO scales (FACIT-Fatigue and PHQ-8) Impact on work productivity assessed using a PRO scale (WPAI) and impact of C5 inhibitor infusions and blood transfusions assessed using a Likert scale Self-reported health resource utilization (Number of times in ER and hospitalized)
- Secondary Outcome Measures
Name Time Method Calculation of summary statistics and number and percentage of respondents for the primary endpoints by subgroup described below -Subgroup Patients with reported hemoglobin levels of <12 (120) vs >_12 (120) g/dL (g/L) and <10.5 (105) vs >_10.5 (105) g/dL (g/L) Patients with vs. without a history of transfusion in the past 6 months Patients with vs. without a history of thrombotic events Patients classification as separately specified in the Statistical Analysis Plan (SAP)