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

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. *…