Patient-Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non-Transfusion-Dependent β-Thalassemia Treated in the BEYOND Trial.

What was studied

This study looked at patient-reported outcomes (PROs) with luspatercept. The patients had non-transfusion-dependent β-thalassemia (NTDT). Data came from the BEYOND trial. It was double-blind and randomized. Patients also came from a long-term follow-up (LTFU) study. In BEYOND, patients got luspatercept or placebo every 3 weeks for 48 weeks or more. Tools were NTDT-PRO, FACIT-F for fatigue and SF-36. Mixed-effects models estimated changes from baseline.

What they found

144 patients from BEYOND had usable PRO data. 95 got luspatercept and 49 got placebo. 58 came from LTFU. They had all received luspatercept. Luspatercept improved tiredness/weakness, shortness of breath, fatigue and vitality scores versus placebo. This held through double-blind treatment. It was generally kept through week 96. Other FACIT-F and SF-36 domains improved or held. In LTFU patients, fatigue and vitality scores improved from baseline. This came within 12 weeks of starting treatment. It lasted for up to 5 years.

What it means for patients

The authors say luspatercept offers rapid and durable benefits. They say it improves symptoms tied to anemia, plus quality of life, in NTDT.

Why it is not certain yet

LTFU had only luspatercept patients, 58 in all. It had no placebo group. The abstract gives no numbers for the score changes. PRO data came from usable cases only.

Source

Patient-Reported Outcomes With Luspatercept Through 5 Years of Treatment in Patients With Non-Transfusion-Dependent β-Thalassemia Treated in the BEYOND Trial.

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