Paroxysmal Nocturnal Haemoglobinuria
Conditions
Keywords
Paroxysmal nocturnal haemoglobinuria, PNH, ravulizumab
Brief summary
The PNH-RECORD study, a Polish multicenter observational (non-interventional), open-label, retrospective with prospective follow-up.
Detailed description
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.
Interventions
DRUGRavulizumab
open-label arm with ravulizumab administrated within the scope of routine clinical practice
Sponsors
AstraZeneca
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Adult (aged ≥18) patients with PNH receiving ravulizumab treatment in the frames of NDP in Poland. 2. Patients willing to participate in the study and signed ICF.
Exclusion criteria
1. Those who participated in ravulizumab clinical trial in the past, and/or those who participated/plans to participate in clinical trial on/after the date of first ravulizumab infusion through NDP. 2. Cognitive incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
Design outcomes
Primary
| Measure | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | Baseline | Mean age (years) at PNH diagnosis |
| To present clinical characteristics | Baseline | Mean and median (years) duration of PNH |
Countries
Poland
Outcome results
None listed