Long-Term Experience with Luspatercept in Relapsed/Refractory Myelodysplastic Neoplasms: A Chinese Real-World Study

Luspatercept has been shown to be efficacious for patients with relapsed or refractory lower-risk myelodysplastic neoplasms (LR-MDS) in both clinical trials and real-world studies. Nevertheless, long-term follow-up data in real-world settings remain scarce, particularly in Asia.

Data from patients diagnosed with relapsed or refractory LR-MDS who had been treated with luspatercept at our center between June 2022 and May 2024 were retrospectively collected.

In total, 60 patients were included in this study (63.4% males). The median duration of luspatercept exposure was 9 (range 3-25) months, and the median follow-up time was 15 (range 3-26) months. The hematologic improvement-erythroid (HI-E) rate was 46.7%, 51.0%, 48.6%, and 43.3% at the 3rd, 6th, and 12th months, and at the end of follow-up, respectively. Among patients who were transfusion-dependent prior to luspatercept, 48.3%, 38.7%, and 25.8% achieved transfusion independence for 8, 12, and 16 weeks or longer at the 6th month. Over time, patients treated with luspatercept had a significant increase in hemoglobin level compared with that of the baseline from the 1st month to the end of follow-up (all P < 0.05). At the end of follow-up, 5 of 32 (15.6%) patients who had response had experienced a relapse, 1 patient (1.7%) had progressed to higher-risk myelodysplastic neoplasms (MDS), and 2 patients (3.3%) had progressed to acute myeloid leukemia. Three patients (5.0%) died of pulmonary infection. Serum erythropoietin (EPO) ≤ 500 IU/l at baseline was the only independent predictive factor for HI-E at the 3rd month (P = 0.007).

Luspatercept is proved efficacious and well tolerated in relapsed/refractory LR-MDS and appears to be beneficial in reducing disease progression and prolonging survival.