骨髓增生异常综合征-难治性贫血伴原始细胞增多患者应用重组人血小板生成素对地西他滨联合预激方案治疗效果的影响

Effect of recombinant human thrombopoietin on myelodysplastic syndrome- refractory anemia with excess blasts treated by desitabine combined with preexcitation therapy

目的探讨骨髓增生异常综合征-难治性贫血伴原始细胞增多(myelodysplastic syndrome-refractory anemia with excess blasts, MDS-RAEB)患者应用地西他滨+预激方案+重组人血小板生成素(recombinant human thrombopoietin, rhTPO)治疗的临床疗效,及对血小板计数的影响。方法 MDS-RAEB患者65例,随机分为观察组33例和对照组32例,观察组给予地西他滨+预激方案+rhTPO治疗,对照组给予地西他滨+预激方案治疗。比较2组血小板计数恢复至25×10^9/L、50×10^9/L、75×10^9/L、100×10^9/L的时间;治疗3个疗程时记录2组血小板悬液输注量,评定临床疗效;随访观察急性髓细胞性白血病(acute myeloid leukemia, AML)转化率及无进展生存时间。结果观察组血小板恢复至25×10^9/L、50×10^9/L、75×10^9/L、100×10^9/L的时间[(7.13±1.38)、(12.58±2.71)、(14.21±2.06)、(18.65±3.11) d]均较对照组[(9.72±1.41)、(16.35±2.84)、(19.71±1.50)、(22.83±2.27)d]短(P<0.05);治疗3个疗程时,观察组血小板悬液输注量[(5.28±0.52)份]较对照组[(8.51±0.56)份]少(P<0.05),总有效率(81.8%)与对照组(78.1%)比较差异无统计学意义(P>0.05);截至末次随访,观察组AML转化率(12.12%)、中位无进展生存时间(16.3个月)与对照组(9.38%、17.0个月)比较差异无统计学意义(P>0.05)。结论 rhTPO能促进应用地西他滨联合预激方案治疗的MDS-RAEB患者血小板计数的恢复,且不影响临床疗效,不增加AML转化率。

Objective To investigate the clinical effect of desitabine + preexcitation regimen + recombinant human thrombopoietin(rhTPO) on myelodysplastic syndrome-refractory anemia with excess blasts(MDS-RAEB) and influence on platelet count. Methods Sixty-five patients with MDS-RAEB were randomly divided into observation group(n=33) and control group(n=32), treated with desitabine + preexcitation regimen + rhTPO, and desitabine + preexcitation regimen, respectively. The recovery time of platelet to 25×10^9/L, 50×10^9/L, 75×10^9/L and 100×10^9/L was recorded in two groups. The volume of platelet suspension and the clinical effect were compared between two groups after 3 therapeutic courses. The transformation rate of acute myeloid leukemia and progression free survival were recorded. Results The recovery time of platelet to 25×10^9/L, 50×10^9/L, 75×10^9/L and 100×10^9/L was shorter in observation group((7.13±1.38),(12.58±2.71),(14.21±2.06),(18.65±3.11) d) than that in control group((9.72±1.41),(16.35±2.84),(19.71±1.50),(22.83±2.27) d)(P<0.05). The volume of platelet suspension was less in observation group(5.28±0.52) than that in control group(8.51±0.56)(P<0.05) after 3 courses.There were no significant differences in overall response rate,transformation rate of acute myeloid leukemia and progression free survival between observation group(81.8%,12.12%,16.3 months)and control group(78.1%,9.38%,17.0 months)(P>0.05).Conclusion rhTPO can promote the recovery of platelet count in patients with MDS-RAEB treated with desitabine combined with preexcitation regimen,neither affecting the clinical effect,nor increasing the transformation rate of acute myeloid leukemia.