An Observational Post-Authorization Safety and Effectiveness Study of Ravulizumab in Adult Patients Aged 18 and Older Diagnosed With Paroxysmal Nocturnal Hemoglobinuria Using Multicenter Electronic Medical Records in South Korea (ESR-25-22871)
This multicenter, retrospective observational cohort study aims to evaluate the long-term real-world effectiveness and safety of ravulizumab in adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) in South Korea over a 4-year post-marketing period. By analyzing electronic medical records (EMR) from participating institutions, the study investigates intravascular hemolysis control, transfusion avoidance, breakthrough hemolysis (BTH) characteristics, and overall clinical outcomes in an Asian population.
Studieöversikt
Status
Betingelser
Studietyp
Inskrivning (Beräknad)
Kontakter och platser
Studieorter
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Seoul, Sydkorea
- Yonsei University College of Medicine
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Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
Tar emot friska volontärer
Kön som är behöriga för studier
Testmetod
Studera befolkning
Korean patients with Paroxysmal Nocturnal Hemoglobinuria whose retrospective clinical data are available in the electronic medical records (EMR) of the participating hospitals.
Beskrivning
Inclusion Criteria:
- Adult patients (18 years of age or older) with a confirmed diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) documented by flow cytometry.
- Patients who initiated ravulizumab treatment on or before February 28, 2025, and have at least 6 months of follow-up data available.
- To reflect real-world clinical practice, meningococcal (Neisseria meningitidis) vaccination status will be collected if available, but is not a mandatory requirement for study inclusion.
Exclusion Criteria:
- Less than 18 years of age at the time of ravulizumab treatment initiation.
- Known hypersensitivity to ravulizumab or any of its excipients.
- Untreated Neisseria meningitidis infection at the time of ravulizumab treatment initiation.
- Prior or concomitant use of other complement inhibitors for PNH (with the exception of prior eculizumab use).
Studieplan
Hur är studien utformad?
Designdetaljer
- Observationsmodeller: COHORT
- Tidsperspektiv: RETROSPECTIVE
Kohorter och interventioner
Grupp / Kohort |
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PNH patients treated with Ravulizumab
Patients diagnosed with PNH and prescribed Ravulizumab at multiple centers in South Korea, whose electronic medical records (EMR) are available for retrospective review. |
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Proportion of patients achieving lactate dehydrogenase (LDH) levels less than or equal to 1.5 times the upper limit of normal (ULN)
Tidsram: Up to 4 years after ravulizumab initiation
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Assessment of intravascular hemolysis control, defined as achieving and maintaining serum LDH levels at or below 1.5 times the ULN during the 4-year post-marketing observation period. |
Up to 4 years after ravulizumab initiation
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Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
|---|---|---|
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Incidence and clinical characteristics of breakthrough hemolysis (BTH)
Tidsram: hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
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BTH is defined as at least one new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia [hemoglobin less than 10 g/dL], major adverse vascular events [MAVE, including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated lactate dehydrogenase (LDH) levels of at least 2 times the upper limit of normal (ULN), after prior reduction of LDH to less than 1.5 times the ULN during therapy. Associated contextual factors, including complement-amplifying conditions, hospitalizations, transfusions, and the use of rescue medication, will also be recorded and characterized at each event. |
hroughout the follow-up period, assessed at each occurrence of a BTH event (up to 4 years)
|
Samarbetspartners och utredare
Sponsor
Studieavstämningsdatum
Studera stora datum
Studiestart (Faktisk)
Primärt slutförande (Beräknad)
Avslutad studie (Beräknad)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- 4-2026-0786
Plan för individuella deltagardata (IPD)
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