重组人血小板生成素对非重型再生障碍性贫血免疫抑制治疗疗效的影响

OBJECTIVE: To compare the efficacy of cyclosporin A (CsA) alone and CsA combined with recombined human thrombopoietin (rhTPO) in patients with non-severe aplastic anemia (NSAA). METHODS: Data from 83 patients with NSAA between August 2014 and February 2019 were collected retrospectively. The study population included 35 men and 48 women, with a median age of 45 years (14–85 years). Among them, 57 had been treated with CsA + rhTPO, TPO was administered at 15 000 U QD for 7 days, once a month for 3 months, and the other 26 patients with compatible baseline characters were treated with CsA alone. All the enrolled patients had been treated with CsA for at least 6 months and were followed up for at least 1 year. The efficacy and outcome were compared between the two groups. RESULTS: Total 23 men and 34 women, with a median age of 46 years (14–85 years) were treated with CsA + rhTPO. The median duration of CsA treatment was 17 (8–28) months, and the patients were followed up for a median of 27 (12–45) months. Total 12 men and 14 women, with a median age of 40 years (20–64) were treated with CsA alone. The median duration of CsA treatment was 19 months (9–30 months), and the median follow-up duration was 29 months (16–66 months). There was no significant difference in the baseline characteristics of the two groups (P>0.05). There was no significant difference in the CR and OR rates of the two groups at 1, 3, 6, 12, and 24 months of treatment (P>0.05). The change in the platelet level for the CsA + rhTPO treated group after 1 month[8 (−12–86) ×10(9)/L vs. 3 (16–57) ×10(9)/L, P=0.029), 3 months[24 (−6–102) ×10(9)/L vs. 7 (−9–76) ×10(9)/L, P=0.006], and 6 months[33.5 (−4–123) ×10(9)/L vs. 12.5 (−14–109) ×10(9)/L, P=0.048] of treatment was higher than that in the CsA alone group, while no significant difference was found between the two groups at other time points. There was no significant difference in the change in the megakaryocyte level between the two groups[3 (0–4) vs. 2 (0–5), z=−0.868, P=0.385] after 6 months of treatment. Apart from 10.5% (6/57) of the patients in the CsA + rhTPO treated group who reported soreness at the injection site, there was no other significant difference between the two groups in terms of adverse effects. During the follow-up period, there were two cases of increasing paroxysmal nocturnal hemoglobinuria clone to over 10%, one in the CsA + rhTPO treated group, the other in the CsA alone group; and there was one case of progression to SAA in the CsA + rhTPO treated group; while no case of death or thromboembolic event (TEE), fibrosis or reticulin proliferation, progression to myelodysplastic syndrome (MDS), or acute myeloid leukemia was observed in either group. There was one case of progression to SAA in the CsA + rhTPO treated group but none in the CsA alone group. CONCLUSION: Compared to CsA alone, CsA + rhTPO treatment can accelerate the recovery of the platelet level with acceptable adverse effects.