For patients with anaemia associated with myelodysplastic syndrome, when an epoetin is no longer an option, luspatercept has been evaluated in a single placebo-controlled trial. A 5q deletion was not present in the neoplastic cells in any of these patients. In this trial, a minority of patients from the luspatercept group were able to dispense with transfusions in the short term compared to those in the placebo group. However, there was no reduction in the main symptoms of anaemia, and the level of evidence provided by these data is low due to methodological flaws in the trial. Luspatercept has numerous adverse effects, including: fatigue, dyspnoea, infections, gastrointestinal disorders, hypersensitivity reactions, kidney damage (sometimes fatal), liver damage (sometimes severe), tachyarrhythmias, and possibly cancer in the longterm. Red blood cell transfusions, or lenalidomide in patients with 5q deletion, are better options, despite their limitations.
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