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

Prior treatment

• Participants enrolled in any clinical study receiving experimental treatments for PNH (evaluated at baseline)
• Participants who are not currently vaccinated against N. meningitidis unless they receive prophylactic treatment with appropriate antibiotics until 2 weeks after vaccination

Other illness

• History of hematopoietic stem cell transplantation (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

Other

• 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)

• 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