Blood:聚乙二醇化-rIFN-α2a治疗HU耐药性/难治性的ET/PV患者的预后

2019-09-16 MedSci MedSci原创

既往研究表明用重组干扰素-α(rIFN-α)治疗血小板增多(ET)和红细胞增多(PV)的患者,可获得较高的缓解率。为进一步明确rIFN-α的作用,Yacoub等人开展试验,评估以聚乙二醇化-rIFN-α2a(PEG)治疗既往采用羟基脲(HU)治疗过的ET/PV患者预后。本研究是一项国际性的、多中心的2期试验,评估PEG诱导HU耐药性/难治性高危型ET/PV患者获得完全和部分造血缓解(CR和PR)的

既往研究表明用重组干扰素-α(rIFN-α)治疗血小板增多(ET)和红细胞增多(PV)的患者,可获得较高的缓解率。为进一步明确rIFN-α的作用,Yacoub等人开展试验,评估以聚乙二醇化-rIFN-α2a(PEG)治疗既往采用羟基脲(HU)治疗过的ET/PV患者预后。

本研究是一项国际性的、多中心的2期试验,评估PEG诱导HU耐药性/难治性高危型ET/PV患者获得完全和部分造血缓解(CR和PR)的能力。

本研究共招募了65位ET患者和50位PV患者。12个月时,ET患者的总体缓解率ORR(CR/PR)为69.2%(43.1%/26.2%),PV患者的ORR为60%(22%/28.0%)。与无CALR突变的个体相比,携带CALR突变的ET患者的CR率较高(56.5% vs 28.0%)。获得CR的患者与获得PR或无缓解(NR)的患者相比,JAK2 V617F突变等位基因频率(VAF)的绝对值分别降低了-6%(范围-84%-47%)vs +4%(范围-18%--56%)。

治疗与显著的不良事件发生率(AE)相关,大多数是可控的,而由于AE导致的PEG停药仅发生在13.9%的受试者中。治疗相关的显著副反应事件(AE),大部分可控,仅13.9%的个体因AEs而中断PEG治疗。

综上所述,PEG可有效治疗HU耐药性/难治性的ET/PV患者。

原始出处:

Abdulraheem Yacoub, et al.Pegylated Interferon Alfa-2a for Polycythemia Vera or Essential Thrombocythemia Resistant or Intolerant to Hydroxyurea.Blood 2019 :blood.2019000428; doi: https://doi.org/10.1182/blood.2019000428

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    2020-03-03 weiz
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    2019-09-18 bugit
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    2019-09-18 freve