Among patients with autoimmune hemolytic anemia (AIHA), parsaclisib may be an effective treatment that improves hemoglobin (Hgb) levels without excessive toxicity, according to research presented at the EHA 2022 Hybrid Congress.

AIHA, a rare autoimmune condition, is caused by red blood cell hemolysis. Furthermore, there are not many therapeutic options for AIHA other than rituximab or steroids.

Parsaclisib, an inhibitor of phosphoinositide 3-kinase-delta, has previously shown promise in the AIHA setting. For this ongoing open-label phase 2 study ( Identifier: NCT03538041), researchers are evaluating the safety and efficacy of parsaclisib among patients diagnosed with AIHA.

Continue Reading

All included patients are at least 18 years of age and have been previously diagnosed with warm AIHA (wAIHA), cold agglutinin disease, or mixed-type AIHA. Hgb levels in included patients are within 7 and 10 g/dL, and all patients must have failed at least 1 prior therapy line. The study’s primary endpoints are efficacy, as measured by the complete and partial response rates determined by Hgb levels, and safety.

Overall, 25 patients (16 wAIHA, 6 cold agglutinin disease, and 3 mixed AIHA) have been enrolled and treated in 1 of 2 cohorts, which included 10 and 15 patients, respectively. Eight patients in cohort 1 had a dose increase. The mean age in the overall population was 61.6 years, and 56% of patients were female sex; the mean baseline Hgb was 8.9 g/dL.

A total of 20 patients completed 12 weeks of parsaclisib. A complete response was noted in 32% of patients; 64% of patients had at least a partial response. Fifty percent of patients with wAIHA, furthermore, had a complete response, and Hgb improvements appeared to be sustained during the study period.

Adverse events were noted in 84% of patients during the 12-week treatment period, and 24% of patients had grade 3 or worse events. Grade 3 or worse events, or serious events, were also noted in 53% of patients in an extension period.

One fatal event, acute respiratory failure, was not related to the study therapy.


Jang JH, Gomez RD, Bumbea H, et al. Long-term efficacy and safety results from an ongoing open-label phase 2 study of parsaclisib for the treatment of autoimmune hemolytic anemia (AIHA). Presented at EHA 2022; June 9-12, 2022. Abstract S286.