Survey says… (Fatigue + PROs)
What is MWPI? (What does it stand for and what is the minimum point improvement from baseline in Neuro-QoL Fatigue scores?)
Meaningful Within Person Improvement, ≥6.7 point-improvement
What is the difference between unanchored MAIC and PCB-anchored Butcher ITC?
MAIC adjust for patient characteristics using individual patient data for NIPO and aggregate data for comparators; Butcher ITC uses aggregate published data with no adjustment
What was the rate of SAEs in the DB Phase vs OLE and how many discontinuations due to SAEs were there in both?
14.3% PBO v 9.2% NIPO DB; 23.9% PBO/NIPO vs 28.4% NIPO/NIPO OLE
What proportion of patients had MCI in MG-ADL at W48?
84.6% NIPO/NIPO and 63.3% PBO/NIPO
"What are the key findings on mortality compared to the general population in wAIHA patients?"
(Kjellander) The key findings show that patients with wAIHA have a significantly increased mortality risk compared to the general population, with a 2.2-fold higher risk in primary wAIHA and a 3.9-fold higher risk in secondary wAIHA. The excess mortality is highest among younger patients diagnosed under 60 years old, and up to 30% of the 5-year all-cause mortality is attributed to AIHA itself, infections, and cardiovascular complications. This underscores the persistent and substantial impact of wAIHA on patient survival.
What were the primary findings in the Vissing et al. Fatigue poster?
A higher proportion of NIPO patients had MWPI in Neuro-QoL Fatigue than PBO and NIPO patients had greater improvements in Fatigue scores as early as Week 2 and sustained through Week 24
What subgroups/populations were compared for the comparative analyses for NIPO vs EFGART and NIPO vs ROZA?
Vs EFGART: AChR+; vs ROZA: AChR+ and MuSK+
How many deaths were there in the DB phase and the OLE and what were they?
3 DB; 4 OLE
What proportion of patients achieved MSE in DB phase and OLE phase? How did MSE compare between DB Week 24 NIPO and OLE Week 24 PBO/NIPO?
18.4% DB, 26.9% OLE NIPO/NIPO; 44.3% OLE PBO/NIPO
What percentage of Taiwanese patients received an emergency blood transfusion in their first year of wAIHA diagnosis?
(Kao) National Health Insurance Research Database, covering >99% Taiwan population, evaluated ~1500 adults with AIHA (primary or secondary) for at least 6 months before enrollment and with at least 1 treatment such as OCS or NSIT between Jan 2017 through Dec 2022 to determine prevalence, treatment patterns, overall survival. Annual prevalence has increased
NIPO-treated patients demonstrated improvements in Neuro-QoL Fatigue Score as early as Week ______ compared with PBO.
Week 2
At what timepoint is NIPO favored over EFGART and over ROZA?
EFGART: Week 8-24; ROZA: Week 10-14
Describe the results for lipids in the DB phase and how these data correlate with blood pressure and/or cardiovascular risk?
Mild increases in total cholesterol, HDL, and LDL; total chol/HDL ratio remained under 4; no difference in rates of MACE events or CV risk for patients with lipid changes; mean systolic BP was lower than baseline in DB and OLE
What were mean MG-ADL scores at OLE Week 24 (Study Week 48)?
-5.19 NIPO/NIPO, -2.68 PBO/NIPO
What did a systematic review in primary and secondary wAIHA reveal regarding outcomes to inform the design of future studies?
(Rodrigues) A total of 401 outcomes were identified and categorized into 14 domains (Figure 2). Most outcomes reported were within the domains of hemolysis, hemolytic markers, response and treatment pattern. There was no consistent or universal definition for ‘response’ and 40% of the publications did not provide a definition. Quality of life was only reported in 3% of the studies. Validated patient reported outcome measures were identified in only 12 (4%) studies.
NIPO-treated patients were approximately how much more likely to have MWPI sustained for greater than 8, 12, 16, or 20 weeks?
Twice as likely
Decribe the results for the standardized mean AUC MG-ADL for nipocalimab vs other FcRn blockers.
Cumulative effect over time was greater with NIPO vs other FcRns
Describe the muscle spasms and peripheral edema observed in the trial? (rates for DB and OLE, severity, changes in rates over time)
DB: 12.2% for each; OLE 6.8% muscle spasms, 5.1% peripheral edema; all mild to moderate
What were mean QMG scores at OLE Week 24 (Study Week 48)?
-4.73 NIPO/NIPO, -3.24 PBO/NIPO
What constitutes a meaningful change in fatigue according to the qualitative review on the FACIT Fatigue scale for warm anemia?
3-point change in total score