Efficacy data for APPOINT: a study of complement inhibitor–naive adults with PNH1
The majority of patients taking FABHALTA experienced Hb improvements in the absence of RBC transfusions
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PRIMARY END POINT
Patients with sustained Hb increase of ≥2 g/dL* from baseline in the absence of RBC transfusions† after 24 weeks1
*Assessed between Days 126 and 168.1
†Assessed between Days 14 and 168. Requiring RBCs refers to any patient receiving transfusions or meeting protocol-defined criteria.2
Higher Hb levels in the absence of RBC transfusions are within reach1
FABHALTA is the first PNH treatment to evaluate a primary end point of the response rate of patients achieving sustained Hb increase of ≥2 g/dL, as opposed to Hb stabilization1,3-5
Patients taking FABHALTA experienced an increase in adjusted mean Hb level of +4.29 g/dL from baseline
ADDITIONAL END POINTS
Patients experienced increases in mean Hb by Week 1, with increases through Week 246,7
Mean Hb levels (g/dL) through Week 24 (values within 30 days of transfusion are considered missing)
The data from this additional analysis are exploratory; therefore, not subject to family-wise Type 1 error control, and presented for observation only. No formal conclusions can be made.
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Sustained Hb level of ≥12 g/dL* in the absence of RBC transfusions† after 24 weeks was observed in 47.5% of patients (n=19/40; 95% CI, 31.5-63.9)6
*Assessed between Days 126 and 168.1
†Assessed between Days 14 and 168. Requiring RBCs refers to any patient receiving transfusions or meeting protocol-defined criteria.2
RBC transfusion avoidance with FABHALTA over the 24-week treatment period
ADDITIONAL END POINT
The data from this additional analysis are exploratory; therefore, not subject to family-wise Type 1 error control, and presented for observation only. No formal conclusions can be made.
Patients achieving RBC transfusion avoidance assessed between Weeks 2 and 246
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In the 6 months before trial enrollment, 70% (n=28/40) of patients required a transfusion.2 Transfusion avoidance in APPOINT was defined as absence of administration of packed-RBC transfusions between Days 14 and 168.6
The impact of FABHALTA on LDH and ARC levels
ADDITIONAL END POINTS
The data from this additional analysis are exploratory; therefore, not subject to family-wise Type 1 error control, and presented for observation only. No formal conclusions can be made.
Absolute reticulocyte count (ARC) and lactate dehydrogenase (LDH) are 2 of the known biomarkers of hemolytic activity. ARC is a marker of both IVH and EVH, while LDH primarily reflects IVH.9-11
Adjusted mean change from baseline in LDH2,6,II
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IIMean of visits between Days 126 and 168.2
#Day 7.2
¶Day 14.2
**Day 168.2
Adjusted mean change from baseline in ARC2,6,II
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IIMean of visits between Days 126 and 168.2
With FABHALTA, the adjusted mean change from baseline in FACIT-Fatigue score was +10.8
ADDITIONAL END POINT
Patient-reported FACIT-Fatigue scores may be an underestimation or overestimation because patients were not blinded to treatment.
The data from this additional analysis are exploratory; therefore, not subject to family-wise Type 1 error control, and presented for observation only. Due to the explanatory nature, small sample size, single-arm and open-label design, no formal conclusions can be made.
Assessed between Weeks 18 and 24. The adjusted mean change* from baseline in FACIT-Fatigue score†† in patients taking FABHALTA was +10.8 (95% CI, 8.63-12.95)2,6
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In separate large-scale surveys, the mean FACIT-Fatigue score for the general population was 4412,13,§§
*Assessed between Days 126 and 168.1
††The Functional Assessment of Chronic Illness Therapy – Fatigue Scale (FACIT-Fatigue) is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function. The level of fatigue is measured on a 4-point Likert scale (in the study, 4=not at all fatigued to 0=very much fatigued), with 0 being the worst possible score and 52 the best.2
‡‡Baseline mean FACIT-Fatigue scores and adjusted mean change in FACIT-Fatigue scores at Day 168 were reported for 40 patients.2
§§The FACIT-Fatigue score for the general population was determined through the assessment of 1010 adults in the US in 2002 and 2426 adults in Germany in 2018.12,13