.

*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 

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 stabilization^1,3-5

ADDITIONAL END POINTS

Patients experienced increases in mean Hb by Week 1, with increases through Week 24^6,7

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.

Click on image to enlarge. 

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 

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

Click on image to enlarge. 

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

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 LDH^2,6,II

Click on image to enlarge.

^IIMean of visits between Days 126 and 168.^2  
#Day 7.^{2         
¶}Day 14.²         
**Day 168.²     
 

Adjusted mean change from baseline in ARC^2,6,II 

Click on image to enlarge.

^IIMean of visits between Days 126 and 168.²

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

Click on image to enlarge.

• In separate large-scale surveys, the mean FACIT-Fatigue score for the general population was 44^12,13,§§

*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