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.
Study Type
OBSERVATIONAL
Enrollment
120
Participants will be observed for 52 weeks after the start of ravulizumab.
Clinical Trial Site
Brescia, Italy
Clinical Trial Site
Catania, Italy
Clinical Trial Site
Lecce, Italy
Clinical Trial Site
Ragusa, Italy
Clinical Trial Site
Percentage Change In Lactate Dehydrogenase (LDH) From Baseline To End Of Observation
The analysis using descriptive statistics will be performed on the Full Analysis Set, including the participants having the LDH evaluation at both the baseline and at the end of observation.
Time frame: Baseline through up to Week 52
Percentage Change In LDH From Baseline To End Of Observation On Participants Treated With Ravulizumab With Respect To The Observed Treatment Period With Eculizumab
The difference of percentage change in LDH from baseline to end of observation will be calculated on participants treated with ravulizumab (over the 52 weeks after baseline) and on the same participants treated with eculizumab (during up to 52 weeks before baseline).
Time frame: Baseline through up to Week 52
Number of Transfusions During Treatment Period With Ravulizumab
The number of participants who needed transfusions, with relative frequency and percentage, and descriptive statistics on the total number of transfusions (number of packed red blood cell units transfused) will be calculated for both the treatment period with ravulizumab and the treatment period with eculizumab.
Time frame: Baseline through up to Week 52
Total Number Of Transfusion Sessions During The Treatment Period With Ravulizumab
The number of participants who needed transfusions, with relative frequency and percentage, and descriptive statistics on the number of transfusion sessions (number of days) will be calculated for both the treatment period with ravulizumab and the treatment period with eculizumab.
Time frame: Baseline through up to Week 52
Number Of Participants Undergoing Ravulizumab Without A ≥ 2 gram/deciliter (g/dL) Decrease In Hemoglobin Level In The Absence Of Transfusion
The proportion of participants without a ≥ 2 g/dL decrease in hemoglobin level in the absence of transfusion will be calculated for both the treatment period with ravulizumab and the treatment period with eculizumab.
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Roma, Italy
Clinical Trial Site
Salerno, Italy
Time frame: Baseline through up to Week 52
Breakthrough Hemolysis (BTH) In The Presence Of Elevated LDH During Treatment Period With Ravulizumab
BTH is defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, dyspnea, anemia \[hemoglobin \< 10 g/dL\], major adverse vascular event including thrombosis, dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥ 2 \* upper limit of normal (ULN) after prior LDH reduction to \< 1.5 \* ULN while on therapy.
Time frame: Baseline through up to Week 52
Change From Baseline To Each Timepoint Of Assessment Using Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants will score each item on a 5-point scale. Total scores range from 0 to 52, with a higher score indicating better Quality of Life (QoL).
Time frame: Baseline through up to Week 52
Change From Baseline To Each Timepoint Of Assessment Using European Organisation for Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire-Core 30 Scale
EORTC is a questionnaire developed to assess the QoL of cancer participants over the preceding 7 days. The questionnaire has 30 questions related to QoL, with the first 28 questions scored on a 4-point scale and the final 2 questions that probe the participant's overall health and QoL scored on a scale of 1 (very poor) to 7 (excellent). Each subscale has a range of 0 to 100%. Thus, a high score for a functional scale represents a high/healthy level of functioning, a high score for the global health status/QoL represents a high QoL, but a high score for a symptom scale/item represents a high level of symptomatology/problem.
Time frame: Baseline through up to Week 52
PNH-specific Patient Preference Questionnaire (PPQ)
PNH-PPQ is a participant-centered approach for evaluating preferences for the treatment of PNH. It contains 11 questions assessing overall treatment preference, evaluating treatment preference according to 9 treatment characteristics, assessing the most important treatment characteristic for participant overall medication preference, and evaluating those same aspects of treatment with ravulizumab.
Time frame: Week 52
Number of Participants Experiencing Adverse Events (AEs)
An AE is any untoward medical occurrence in a participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. AEs that will occur during the ravulizumab treatment will be collected and will be reported to the Investigator or qualified designee by the participant.
Time frame: Baseline through up to Week 52
Costs Related to PNH Sustained By National Health System (NHS) For Ravulizumab Treatment
The following medical costs related to PNH sustained by NHS will be collected: specialist visits, pharmacological and non-pharmacological treatments, hospital and emergency rooms admissions, and examinations (by examination type). This will be reported through descriptive statistics and will be appraised considering the quantity of resource consumption by medical resources and its unit costs.
Time frame: Baseline, Weeks 18, 34, and 52
Costs Sustained By The Participants Related To The Infusion Visits For Ravulizumab Treatment
The following costs sustained by the participants related to the infusion visits will be collected: average cost to reach the structure, overall time required for the infusion (time to reach the structure + time of infusion), retirement status of the participant, loss of working days, and need of a caregiver. This will be calculated in terms of the total cost of transport and overall time required for infusion.
Time frame: Baseline, Weeks 18, 34, and 52