ACCESS-EXTSeguridad y Eficacia a Largo Plazo de la Combinación de Pozelimab y Cemdisiran en la Hemoglobinuria Paroxística Nocturna

Pozelimab

Cemdisiran

Pozelimab

Cemdisiran

Incidence of treatment-emergent serious adverse events (SAEs)

Severity of treatment-emergent SAEs

Incidence of treatment emergent adverse events of special interest (AESIs)

Severity of treatment emergent AESIs

Incidence of adverse events (AEs) leading to permanent treatment discontinuation

Severity of adverse events (AEs) leading to permanent treatment discontinuation

Percent change from baseline in lactate dehydrogenase (LDH)

Transfusion avoidance

Adequate control of hemolysis (LDH ≤1.5 × ULN)

Transfusion avoidance

Transfusion avoidance

Transfusion avoidance

Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)

Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)

Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)

Breakthrough hemolysis (defined as LDH ≥2 × ULN [subsequent to initial achievement of LDH ≤1.5 × ULN] concomitant with signs or symptoms associated with hemolysis)

Hemoglobin stabilization

Hemoglobin stabilization

Hemoglobin stabilization

Hemoglobin stabilization

Percent change in LDH

Percent change in LDH

Percent change in LDH

Change in fatigue

Change in fatigue

Change in fatigue

Change in fatigue

Change in physical function (PF) scores on the EORTC QLQ-C30

Change in PF scores on the EORTC QLQ-C30

Change in PF scores on the EORTC QLQ-C30

Change in PF scores on the EORTC QLQ-C30

Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30

Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30

Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30

Change in GHS/quality of life (QOL) scale on the EORTC QLQ-C30

Normalization of LDH

Rate of red blood cell (RBC) transfusion

Rate of RBC transfusion

Rate of RBC transfusion

Rate of RBC transfusion

Number of units of RBC transfusion

Number of units of RBC transfusion

Number of units of RBC transfusion

Number of units of RBC transfusion

Percentage of days with LDH ≤1.5x upper limit of normal (ULN)

Percentage of days with LDH ≤1.5x ULN

Percentage of days with LDH ≤1.5x ULN

Percentage of days with LDH ≤1.5x ULN

Change in hemoglobin levels

Change in hemoglobin levels

Change in hemoglobin levels

Change in hemoglobin levels

Change in total complement hemolytic activity assay (CH50)

Percent change in CH50

Concentrations of total pozelimab in serum

Concentrations of cemdisiran in plasma

Incidence of treatment-emergent anti-drug antibodies to pozelimab

Incidence of treatment-emergent anti-drug antibodies to cemdisiran

Concentration of total complement component 5 (C5) in plasma

Percent change of concentration of total C5 in plasma