The RACE trial (phase III RCT) compared standard IST (hATG + cyclosporine A) alone (arm A, n=101) versus standard IST plus eltrombopag 150 mg/day from day +14 (arm B, n=96) as front-line therapy in 197 treatment-naive severe aplastic anemia patients, with a median follow-up of 23.2 months.
Adding eltrombopag significantly improved 2-year complete response (62.4% vs. 35.3%, p<0.001), disease-free survival (56% vs. 37%, adjusted HR 0.49, p<0.001), and event-free survival (48% vs. 32%, adjusted HR 0.54, p<0.001). Overall survival trended better (91% vs. 86%, p=0.081). Clonal evolution was rare (1 vs. 2 patients), and evolution to clinical PNH was lower in arm B (1% vs. 8%, p=0.041).
Overall survival difference did not reach statistical significance (p=0.081), limiting conclusions on survival benefit. Eltrombopag was started at day +14, so the schedule used may not generalize to other dosing strategies. The study was not powered to detect rare clonal evolution events.
Adding eltrombopag to hATG + cyclosporine as front-line therapy for severe aplastic anemia roughly doubles complete response rates and meaningfully improves disease-free and event-free survival at 2 years without increasing risk of secondary myeloid malignancies. Clinicians can use this combination with confidence in treatment-naive patients.
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