Enjaymo Improves Life Quality, Lessens Fatigue: Analysis
CADENZA Phase 3 clinical trial participants report significant improvements
People with cold agglutinin disease (CAD) reported clinically significant improvements in health-related quality of life after treatment with Enjaymo (sutimlimab-jome), according to a new analysis of data from the Phase 3 CADENZA clinical trial.
These findings “further augment the primary efficacy outcomes of the placebo-controlled Phase 3 CADENZA study, demonstrating that in addition to providing improvements in hematologic [blood-related] parameters, treatment with [Enjaymo] also results in considerable benefits in PROs [patient-reported outcomes] and QoL [quality of life],” the researchers wrote.
The study, “Sutimlimab provides clinically meaningful improvements in patient-reported outcomes in patients with cold agglutinin disease: Results from the randomised, placebo-controlled, Phase 3 CADENZA study,” was published in the European Journal of Haematology. The work was funded by Sanofi, which markets Enjaymo.
Enjaymo became the first disease-modifying treatment approved in the U.S. for adults with CAD earlier this year. Administered directly into the bloodstream every other week, Enjaymo is designed to block the activation of the classic complement pathway, a group of inflammatory proteins that are overactive in CAD.
The efficacy of Enjaymo was tested against a placebo in two Phase 3 clinical trials: CARDINAL (NCT03347396) and CADENZA (NCT03347422).
CARDINAL’s results showed that Enjaymo eased anemia and led to improvements in patient-reported life quality among CAD patients who had a blood transfusion in the six months before the study.
Previously published data from CADENZA demonstrated that the therapy also reduced anemia, fatigue, and the need for other treatments among patients without a recent history of transfusion.
Patient-reported data
Now, researchers evaluated whether Enjaymo also improved the quality of life of CAD patients with no recent transfusion. They analyzed patient-reported life quality data from the 42 CADENZA participants: 22 given Enjaymo, and 20 on a placebo.
The 12-Item Short Form Health Survey, or SF-12, is a standardized measure of health-related quality of life. On both the mental and physical subscores of the SF-12, patients treated with Enjaymo reported an average improvement of more than five points over the course of the 26-week study.
These improvements were markedly larger than those reported by patients on a placebo, but these differences failed to reach statistical significance. Still, the magnitude of change between study’s start and end on the Enjaymo group was considered clinically meaningful, according to the researchers.
Enjaymo also was associated with greater improvements in another measure of overall health-related quality of life, called the EuroQol visual analogue scale (EQ-VAS), than a placebo. But again, these differences were only close to reaching statistical significance.
On a third standardized measure of health-related life quality called the Patient Global Impression of Change, the researchers noted that nearly three-quarters (73.7%) of Enjaymo-treated patients reported some level of improvement after 26 weeks, as compared with less than a third (31.6%) of those on placebo.
Of note, 10.5% of patients receiving Enjaymo reported that their disease had “‘very much improved’, “compared with no patients in the placebo group,” the team wrote.
Measuring fatigue
Measures of patient-reported fatigue — the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) and Patient Global Impression of fatigue Severity — also showed better outcomes with Enjaymo than a placebo.
The researchers highlighted that a clinically meaningful improvement in FACIT-fatigue scores was seen as early as one week after starting treatment with Enjaymo, but no meaningful changes were seen for patients in the placebo group.
“Along with quick and sustained responses to treatment [blood-related parameters], treatment with [Enjaymo] resulted in rapid improvements in PROs … versus placebo-treated patients,” the researchers concluded.
“Together, the results support the effectiveness of targeting the classical complement pathway in the management of CAD independent of transfusion status,” they added.
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