Sutimlimab Improves Patient-Reported QOL in Cold Agglutinin Disease

An analysis of patient-reported outcomes from the pivotal CARDINAL trial of sutimlimab in cold agglutinin disease found that quality of life (QOL) improved and persisted throughout treatment.

Sutimlimab has shown efficacy in patients with cold agglutinin disease (CAD), but studies on patient-reported quality of life (QOL) outcomes throughout treatment are lacking. A study published in Annals of Hematology analyzed patient-reported outcomes (PROs) from the CARDINAL study of sutimlimab in CAD and found that patient-reported fatigue and other QOL measures improved with treatment.

In the pivotal phase 3, multicenter CARDINAL study, sutimlimab normalized bilirubin levels, improved hemoglobin levels, and significantly reduced the need for blood transfusions in patients with CAD who had a recent history of blood transfusion prior to the study. The first part of the study included a 6-week screening and a 26-week treatment period. Those who responded had the option to participate in part 2 and receive sutimlimab for a 2-year extension study on long-term safety, efficacy, and response durability.

From baseline to treatment assessment timepoint (TAT), patient-reported QOL measures included Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores, as well as responses to the EuroQol 5-dimension 5-level questionnaire (EQ-5D-5L) and the 12-Item Short Form Health Survey (SF-12). The EQ-5D-5L includes 5 domains and a visual analog scale (VAS) of a patient’s perceived overall health. The SF-12 includes 8 domains stratified into physical (PCS) and mental (MCS) scores. Patients also reported on CAD-related symptom incidence at each visit, and researchers evaluated Patient Global Impression of Change (PGIC) and Patient Global Impression of (fatigue) Severity (PGIS) at each visit during the study.

The analysis included 24 patients from 16 centers in 8 countries, all of whom had received at least 1 dose of sutimlimab. The mean age was 71 years, and 62.5% of the patients were women. Most of the patients had CAD-related symptoms, especially symptomatic anemia.

At baseline, the mean FACIT-Fatigue score was 32.5, and it increased quickly with sutimlimab treatment. The improvements in fatigue persisted throughout the 26-week treatment period, and the mean FACIT-Fatigue score was 44.3 at TAT. One patient who missed an infusion reported a FACIT-Fatigue score decline of 44% during the withdrawal period.

The mean SF-12 PCS score increased from 38.7 at baseline to 44.4 at week 5, and the mean SF-12 MCS score increased from 49.8 to 56.2. Both improvements persisted throughout treatment. These scores coincided with changes to hematological parameters. EQ-5D-5L index scores increased by 0.074, and VAS scores increased by 16.8 from baseline to week 26.

Most of the 16 patients who completed the PGIC felt their disease had improved by week 26, and none of the 17 patients who completed the PGIS reported severe fatigue at the end of the study. CAD-related hemoglobinuria and disabling circulatory symptoms were present at baseline but were absent at week 26, and there was a significant improvement in anemia symptoms from baseline to week 26.

The single-arm design and small patient cohort were limitations of this study, but the rarity of CAD limits study size in general for this population. All of the QOL end points except FACIT-Fatigue were also exploratory in the CARDINAL study.

“Despite these encouraging results, the exploratory nature of these end points reflects that the small sample size did not provide sufficient power to detect statistical significance,” the authors wrote.

Even so, the results reiterate that patients with CAD have abnormal self-reported QOL measures at baseline relative to the general population. The findings also suggest that patients who respond to sutimlimab experience QOL improvements along with the clinical improvements seen in the CARDINAL study.


Röth A, Barcellini W, Tvedt THA, et al. Sutimlimab improves quality of life in patients with cold agglutinin disease: results of patient-reported outcomes from the CARDINAL study. Ann Hematol. Published online August 23, 2022. doi:10.1007/s00277-022-04948-y

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