Tacrolimus is effective in relapsed/refractory autoimmune cytopenias: results of a single-center retrospective study

The standard therapies for autoimmune cytopenias, including idiopathic thrombocytopenia (ITP), autoimmune hemolytic anemia (AIHA) and Evans syndrome (ES), are corticosteroids and intravenous immunoglobulin G. However, the recurrence rate is high.

Data from 80 patients with ITP, AIHA and ES who were refractory to corticosteroids/relapsed and were treated with tacrolimus from January 2018 to January 2019 in Peking Union Medical Colleague Hospital were reviewed retrospectively.

There were 24 males and 56 females, with a median age of 43 (14-81) years, including 66 with ITP, 11 with AIHA and 3 with ES. The median disease duration before tacrolimus was 16 (2-432) months. The complete response (CR) rates were 30.3%, 63.6% and 0%; the overall response (OR) rates were 63.6%, 72.7% and 66.7% for ITP, AIHA and ES, respectively; and the median time to response was 3 (2-10) months. In a median of 18 (10-24) months of follow-up time, 21.4% of ITP patients relapsed at a median time of 7 months. No relapse was found for patients with AIHA and ES. Side effects occurred in 16.3% of patients, including elevated creatinine (N = 3, 3.8%), gastrointestinal reactions (N = 3, 3.8%), and pulmonary infection (N = 2, 2.5%), and resulted in 3 patients stopping tacrolimus. The OR rate was found to be related with age (P = 0.01) but not with sex (P = 0.62), the duration of disease (P = 0.66), tacrolimus concentration (P = 0.99) or disease type (P = 0.84).

Tacrolimus can achieve a durable response with mild side effects in patients with steroid-refractory/relapsed autoimmune cytopenias. Patients with younger age had a better response.