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 evaluationof 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 6months

• Participant already assigned to ravulizumab treatment as a specific therapeuticstrategy within the current routine clinical practice (this decision has to be madeindependently and before the enrolment of the participant in the study)

• Vaccinated against Neisseria meningitidis (according to Summary of ProductCharacteristics) < 3 years before dosing or at least 2 weeks prior to initiatingravulizumab unless the risk of delaying ravulizumab therapy outweighs the risk ofdeveloping 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 inquestionnaires and scales (evaluated at enrolment)

• Participants enrolled in any clinical study receiving experimental treatments forPNH (evaluated at baseline)

• Hypersensitivity to the active substance or to any of the excipient of the studydrug.

• Participants with unresolved N. meningitidis infection at treatment initiation

• Participants who are not currently vaccinated against N. meningitidis unless theyreceive prophylactic treatment with appropriate antibiotics until 2 weeks aftervaccination