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

Abstract

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 × 109/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.

Unrelated donor hematopoietic stem cell transplantation compared to immunosuppressive therapy plus eltrombopag as first-line treatment for adults with severe aplastic anemia

“…As in previous study of MSD-HSCT versus IST + EPAG [ 12 ], the hematologic response rate and speed of URD-HSCT were higher and faster than those of IST + EPAG, and URD-HSCT and IST + EPAG yielded similar OS rates in the present study. However, the improved OS in the first-line IST + EPAG group may be partly derived from the fact that patients did not respond to initial IST + EPAG and subsequently underwent salvage transplantation [ 12 ]. Even so, our data still indicate that FFS and HRQoL in the URD-HSCT group are superior to those in the IST + EPAG group.…”

Efficacy and safety of immunosuppressive therapy combined with eltrombopag for severe aplastic anemia: a systematic review and meta-analysis

“…Severe Aplastic anemia (SAA) has acute onset, rapid progression, and high mortality. The treatment mainly includes hematopoietic stem cell transplantation (HSCT) and immunosuppressive therapy (IST) [ 3 , 4 ]. Because HSCT is affected by age, donor restrictions, and other reasons, IST has become a first-line therapy for more patients who are not suitable for HSCT [ 5 ].…”