Clinical Benefits of Luspatercept Dose Titration: Real-World Evidence

Case: SF3B1+ MDS-RS: IPSS-M-Guided 1L Luspatercept

Clinical Presentation:

70-year-old man diagnosed 6 months ago with LR-MDS with multilineage dysplasia - moderate anemia (Hb 11.2) and thrombocytosis (PLT 500,000 μl)

• SF3B1 mutation positive
• Non-del(5q)
• No family history of cancer or significant genotoxic agent exposure
• IPSS-R: Low
• IPSS-M: SF3B1α mutation [SF3B1, ASXL1 and TET2 mutations]

Current Visit Clinical Workup and Diagnosis:

• Serum EPO - 250m U/L, Ring sideroblast (RS) agnostic (either positive or negative)
• Hgb: 8.2 g/dL.
• WBC and ANC: WNL
• PLT: 450,000 μl

He complains of increasing fatigue over the past 1-2 months. He normally plays 2-3 rounds of golf a week. Lately he has only been playing a full round once a week and maybe 9 holes on another day.

Initial Treatment(s):

• The patient was started on luspatercept at the starting dose [1.0 mg/kg] as 1L therapy.
• After 24 weeks, the patient has not received any transfusion and his Hgb is now 10.7 g/dL.