Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria

Real Life Use of Ravulizumab in Italian Patients With Paroxysmal Nocturnal Hemoglobinuria a Multicenter Observational Retrospective and Prospective Cohort Study.

This study will collect clinical response data on participants who were already treated with eculizumab for at least 26 weeks and who started ravulizumab treatment as a specific therapeutic strategy as per ordinary clinical practice.

This study is an Italian multi-center, observational (non-interventional), cohort study composed of both retrospective and prospective observation periods on the same Paroxysmal Nocturnal Hemoglobinuria (PNH) participants. After the First Participant In from different Italian study centers, participants will be consecutively enrolled for 9 months and they will be observed for 52 weeks after the start of ravulizumab.

Inclusion Criteria:

• Body weight of 10 kilogram or above
• Hemolysis with clinical symptom(s) indicative of high disease activity
• Documented diagnoses of PNH confirmed by high-sensitivity flow cytometry evaluation of red blood cells and white blood cells with granulocyte or monocyte clone size of ≥ 5%
• Clinically stable after having been treated with eculizumab for at least the past 6 months
• Participant already assigned to ravulizumab treatment as a specific therapeutic strategy within the current routine clinical practice (this decision has to be made independently and before the enrolment of the participant in the study)
• Vaccinated against Neisseria meningitidis (according to Summary of Product Characteristics) < 3 years before dosing or at least 2 weeks prior to initiating ravulizumab unless the risk of delaying ravulizumab therapy outweighs the risk of developing a meningococcal infection
• Signed written informed and privacy consent prior to study participation

Exclusion Criteria:

• History of hematopoietic stem cell transplantation (evaluated at baseline)
• Known pregnant or breastfeeding participant (evaluated at baseline)
• Participant unable to read and write in Italian language and to autonomously fill in questionnaires and scales (evaluated at enrolment)
• Participants enrolled in any clinical study receiving experimental treatments for PNH (evaluated at baseline)
• Hypersensitivity to the active substance or to any of the excipient of the study drug.
• Participants with unresolved N. meningitidis infection at treatment initiation
• Participants who are not currently vaccinated against N. meningitidis unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination