APPOINT: With FABHALTA oral monotherapy, substantial Hb improvements without the need for RBC transfusions are within reach

Patients with sustained Hb increase of ≥2 g/dL* from baseline in the absence of RBC transfusions^† after the 24-week core treatment period

*Assessed between Days 126 and 168.^{1,2   
†}Assessed between Days 14 and 168. Requiring RBCs refers to any patient receiving transfusions or meeting protocol-defined criteria.^2,3

^‡Based on observed and central laboratory data only. Adjusted mean assessed at a single Week 48 (Day 336) visit. Patients who received transfusions between Days 14 and 336 or were missing Day 336 assessment were considered nonresponders.^4
§Assessed at a single Week 48 (Day 336) visit.^4
‖The 'n' values reflect patients who responded based on observed data.⁴

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 experienced increases in mean Hb by Week 1, with increases through Week 24^2,3

Mean Hb levels (g/dL) through Week 24 (values within 30 days of transfusion are excluded and considered missing)

Click on image to enlarge. 

Sustained Hb levels 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)²

*Assessed between Days 126 and 168.^1,2
†Assessed between Days 14 and 168. Requiring RBCs refers to any patient receiving transfusions or meeting protocol-defined criteria.^2,3

^§Assessed at a single Week 48 (Day 336) visit.^4
IIThe ‘n’ values reflect patients who responded based on observed data.^4
¶Based on central laboratory data only. Adjusted mean assessed at a single week 48 (Day 336) visit. Hb data during core treatment period and within 30 days post-transfusion were considered missing.^4
#The ‘n’ values reflect patients with non-missing data.^4 
**Based on observed and central laboratory data only. Patients who received transfusions between Days 14 and 336 or were missing a Day 336 assessment were considered nonresponders.⁴

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 24²

^††Transfusion avoidance defined as patients who did not receive any transfusions between Days 14 and 336.^4 
‡‡Based on observed data considering only patients who received transfusions between Days 14 and 336 as failures.⁴

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.

ARC and LDH are 2 of the known biomarkers of hemolytic activity. ARC is a marker of both IVH and EVH, while LDH primarily reflects IVH.^6-8
 

Adjusted mean change from baseline in ARC^2,3,§§

^§§Mean of visits between Days 126 and 168.³
 

Adjusted mean change from baseline in LDH^2,3,††

^††Mean of visits between Days 126 and 168.^3 
¶¶Day 7.^3 
##Day 14.^3 
***Day 168.³

^†††Adjusted mean assessed at a single Week 48 (Day 336) visit. Analysis based on observed and central laboratory data only.^{4 
‡‡‡}The ‘n’ values reflect patients with non-missing change from baseline.⁴

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 exploratory 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)^2,3

^¶¶¶The ‘n’ values reflect patients with non-missing values.^4
###Adjusted mean assessed at a single Week 48 (Day 336) visit.⁴

*Assessed between Days 126 and 168.^3 
§§§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.^3
IIIIIIBaseline mean FACIT-Fatigue scores and adjusted mean change in FACIT-Fatigue scores at Day 168 were reported for 40 patients.³ 
****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.^9,10