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Luspatercept effective for myelofibrosis-associated anaemia

Presented by
Prof. Aaron Gerds, Cleveland Clinic Taussig Cancer Institute, USA
Conference
ASH 2019
Prof. Aaron Gerds (Cleveland Clinic Taussig Cancer Institute, USA) presented the preliminary results of an ongoing, open-label, phase 2 trial, which evaluated whether anaemia resulting from myelofibrosis (MF) could be effectively treated with erythroid maturation agent luspatercept [1].

In this ongoing trial, patients (n=74) with MF and anaemia received luspatercept at 21-day intervals. Based on their red blood cell transfusion-dependent status at baseline, patients were allocated to 4 cohorts, also taking into account whether or not they had been receiving treatment with ruxolitinib at the start of the study.

Cohort 1 (n=20) included non-transfusion dependent (NTD) participants who had not received ruxolitinib treatment, and Cohort 2 (n=21) included transfusion-dependent (TD) participants, also without ruxolitinib. For patients who had received ruxolitinib, Cohort 3A (n=14) were NTD, and Cohort 3B patients (n=19) were TD. The primary endpoint was a ≥33% reduction in transfusion burden, with a ≥2-fold reduction in red blood cell units required during weeks 13-24 when compared with the initial 12-week baseline period.

Of the patients who received luspatercept, 21.4% reached the primary endpoint as opposed to only 4.5% who had received placebo (95% CI 10.4-23.6; P<0.0001). In the intent-to-treat analysis, the percentages of NTD patients who showed a consistent haemoglobin improvement of ≥1.5 g/dL above baseline at each evaluation for 12 consecutive weeks were 10% in Cohort 1 and 21% in Cohort 3A. Of patients who were initially TD, 32% in Cohort 3B demonstrated transfusion independence for ≥12 consecutive weeks as compared with 10% of Cohort 2. A mean haemoglobin increase of ≥1.5 g/dL was reported for 57% of Cohort 3A participants and 15% of Cohort 1 participants. A >50% reduction in transfusion burden was experienced by 38% of Cohort 2 patients and 53% of Cohort 3B patients.

Grade 3 or 4 treatment-related adverse events were observed in 5% of patients, but no treatment-related deaths occurred. In conclusion, luspatercept was safe and modestly effective at treating anaemia associated with MF.

1. Gerds A, et al. Abstract 557, ASH 2019, 7-10 December, Orlando, USA.





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