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THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN

THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN - THE CLINICAL, HUMANISTIC, AND ECONOMIC BURDEN AMONG PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) IN JAPAN

Status
Active, not recruiting
Phases
Unknown
Study type
Unknown
Source
JPRN
Registry ID
JPRN-UMIN000050211
Enrollment
60
Registered
2023-02-02
Start date
2022-09-30
Completion date
Unknown
Last updated
2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Paroxysmal Nocturnal Hemoglobinuria (PNH)

Interventions

None listed

Sponsors

Swedish Orphan Biovitrum Japan
Lead Sponsor

Eligibility

Sex/Gender
All

Inclusion criteria

Inclusion criteria: -18 years old or older -Self-reported diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) -Self-reported that currently being treated with eculizumab or ravulizumab -Agree to informed consent for the study

Exclusion criteria

Exclusion criteria: -Correspond to an inappropriate answer

Design outcomes

Primary

MeasureTime frame
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

MeasureTime frame
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)—

Countries

Japan

Contacts

Public ContactMoemi Miura

Social Survey Research Information co., Ltd(SSRI) Health outcomes research, HBI/MA Service Division

hor@ssri.com03-6709-9710

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026