Ravulizumab dose optimization

Conditions

Paroxysmal nocturnal hemoglobinuria (PNH)

Brief summary

Percentage of change in LDH compared to baseline LDH between standard dosing and individualized dosing, Difference in percentage target attainment (Ravulizumab concentration >100 mg/L) between the standard dosing regimen and the individualized dosing regimen

Detailed description

The proportion of patients with breakthrough hemolysis, defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2× the ULN after prior reduction of LDH to <1.5× the ULN on treatment, Quality of life assessed with the FACIT-Fatigue Scale and patient preference, Differences between number of transfusions between both dosing strategies, Differences in cumulative ravulizumab dose over 52 weeks, number of infusions and appointments with the physician as surrogate marker for treatment costs.

Related papers

No related papers found yet.

Papers published before 2007-09-01 may not appear yet. Historical indexing is in progress.

Interventions

DRUGUltomiris 300 mg/30 mL concentrate for solution for infusion

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Percentage of change in LDH compared to baseline LDH between standard dosing and individualized dosing, Difference in percentage target attainment (Ravulizumab concentration >100 mg/L) between the standard dosing regimen and the individualized dosing regimen	—

Secondary

Measure	Time frame
The proportion of patients with breakthrough hemolysis, defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2× the ULN after prior reduction of LDH to <1.5× the ULN on treatment, Quality of life assessed with the FACIT-Fatigue Scale and patient preference, Differences between number of transfusions between both dosing strategies, Differences in cumulative ravulizumab dose over 52 weeks, number of infusions and appointments with the physician as surrogate marker for treatment costs.	—

Measure

Time frame

The proportion of patients with breakthrough hemolysis, defined as at least 1 new or worsening symptom or sign of intravascular hemolysis (fatigue, hemoglobinuria, abdominal pain, shortness of breath [dyspnea], anemia [hemoglobin <10 g/dL], major adverse vascular event [including thrombosis], dysphagia, or erectile dysfunction) in the presence of elevated LDH ≥2× the ULN after prior reduction of LDH to <1.5× the ULN on treatment, Quality of life assessed with the FACIT-Fatigue Scale and patient preference, Differences between number of transfusions between both dosing strategies, Differences in cumulative ravulizumab dose over 52 weeks, number of infusions and appointments with the physician as surrogate marker for treatment costs.

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Countries

Netherlands

Outcome results

None listed