Hasselbalch Hans Carl, Silver Richard T
Department of Hematology, Roskilde Hospital, University of Copenhagen, Roskilde, Denmark.
Expert Rev Hematol. 2015 Aug;8(4):439-45. doi: 10.1586/17474086.2015.1045409. Epub 2015 May 21.
Recently, it was concluded that the optimal therapy for essential thrombocythemia and polycythemia vera, either recombinant interferon alpha (rIFNα) or hydroxyurea can only be determined by the completion of a randomized clinical trial. We present our recommendations for the use of rIFNα for those patients who are not candidates for the randomized trial. We argue for rethinking the approach whether we should continue to wait for the results from a randomized trial before recommending treatment with rIFNα for those unable and unwilling to enter these trials. The interferon story shows that clinical experience may be an alternative path to follow when making treatment decisions and recommendations in orphan diseases.
最近得出的结论是，原发性血小板增多症和真性红细胞增多症的最佳治疗方法，无论是重组干扰素α（rIFNα）还是羟基脲，都只能通过完成一项随机临床试验来确定。对于那些不适合参加随机试验的患者，我们给出了使用rIFNα的建议。我们主张重新思考这种方法，即对于那些无法且不愿意参加这些试验的患者，在推荐使用rIFNα进行治疗之前，我们是否应该继续等待随机试验的结果。干扰素的情况表明，在对罕见病做出治疗决策和建议时，临床经验可能是另一条可供遵循的途径。
