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The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
PNH-RECORD study aims to aims to collect data on the patients' characteristics and clinical outcomes of ravulizumab administered in the scope of routine clinical practice in PNH.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Ravulizumab group | Open-label |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Ravulizumab | Drug | open-label arm with ravulizumab administrated within the scope of routine clinical practice |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change in LDH levels from index date (first dose of ravulizumab) at every 6 month | up to 36 months | |
| Proportion of patients achieving or remaining at LDH<1.5 ULN at every 6 month | up to 36 months |
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of transfusion-free patients from index date at every 6 month | up to 36 months | |
| Number of blood units used per patient-year | up to 36 months | |
| To present patients characteristics |
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Inclusion Criteria:
Exclusion Criteria:
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Adult (aged ≥18) patients with PNH
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site | Bialystok | Poland | ||||
| Research Site |
Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.
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AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
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| ID | Term |
|---|---|
| D006457 | Hemoglobinuria, Paroxysmal |
| ID | Term |
|---|---|
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C000629409 | ravulizumab |
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Mean age (years) at PNH diagnosis |
| Baseline |
| To present patients characteristics | Mean age (years) at index date | Baseline |
| To present patients characteristics | Proportion (%) of men and women | Baseline |
| To present patients characteristics | Mean body mass index (kg/m²) | Baseline |
| To present clinical characteristics | Mean and median (years) duration of PNH | Baseline |
| To present clinical characteristics | Mean and median (months) time from diagnosis to complement inhibitors treatment initiation | Baseline |
| To present clinical characteristics | Proportion (%) of patients with complication of haemolysis, thrombosis, serious vascular event andaplastic anemia | Baseline |
| To present clinical characteristics | Proportion (%) of patients naïve to complement inhibitors | Baseline |
| Brzozów |
| Poland |
| Research Site | Bydgoszcz | Poland |
| Research Site | Gdansk | Poland |
| Research Site | Krakow | Poland |
| Research Site | Lodz | Poland |
| Research Site | Lublin | Poland |
| Research Site | Opole | Poland |
| Research Site | Szczecin | Poland |
| Research Site | Warsaw | Poland |
| Research Site | Wałbrzych | Poland |
| D009190 |
| Myelodysplastic Syndromes |
| D001855 | Bone Marrow Diseases |