Matched related transplantation versus immunosuppressive therapy plus eltrombopag for first-line treatment of severe aplastic anemia: a multicenter, prospective study

This study prospectively compared the efficacy and safety between matched related donor-hematopoietic stem cell transplantation (MRD-HSCT) (n = 108) and immunosuppressive therapy (IST) plus eltrombopag (EPAG) (IST + EPAG) (n = 104) to determine whether MRD-HSCT was still superior as a front-line treatment for patients with severe aplastic anemia (SAA). Compared with IST + EPAG group, patients in the MRD-HSCT achieved faster transfusion independence, absolute neutrophil count >= 1.0 x 10(9)/L (P < 0.05), as well as high percentage of normal blood routine at 6-month (86.5% vs. 23.7%, P < 0.001). In the MRD-HSCT and IST + EPAG groups, 3-year overall survival (OS) was 84.2 +/- 3.5% and 89.7 +/- 3.1% (P = 0.164), whereas 3-year failure-free survival (FFS) was 81.4 +/- 4.0% and 59.1 +/- 4.9% (P = 0.002), respectively. Subgroup analysis indicated that the FFS of the MRD-HSCT was superior to that of the IST + EPAG among patients aged < 40 years old (81.0 +/- 4.6% vs. 63.7 +/- 6.5%, P = 0.033), and among patients with vSAA (86.1 +/- 5.9% vs. 54.9 +/- 7.9%, P = 0.003), while the 3-year OS of the IST + EPAG was higher than that of the MRD-HSCT among the patient aged >= 40 years old (100.0 +/- 0.0% vs. 77.8 +/- 9.8%, P = 0.036). Multivariate analysis showed that first-line MRD-HSCT treatment was associated favorably with normal blood results at 6-month and FFS (P < 0.05). These outcomes suggest that MRD-HSCT remains the preferred first-line option for SAA patients aged < 40 years old or with vSAA even in the era of EPAG.

Automated summary

This study compared the efficacy and safety of matched related donor-hematopoietic stem cell transplantation (MRD-HSCT) with immunosuppressive therapy plus eltrombopag (IST + EPAG) for severe aplastic anemia (SAA) patients. The results showed that MRD-HSCT achieved faster transfusion independence and better outcomes, making it the preferred first-line treatment option for younger patients or those with vSAA.