Weill Cornell Medical College, Department of Medicine, Division of Hematology and Medical Oncology, New York, NY 10021, USA.
Haematologica. 2012 Apr;97(4):538-42. doi: 10.3324/haematol.2011.053348. Epub 2011 Nov 18.
Although reduction in the JAK2(V617F) allele burden (%V617F) has been suggested as a criterion for defining disease response to cytoreductive therapy in polycythemia vera, its value as a response monitor is unclear. The purpose of this study is to determine whether a reduction in %V617F in polycythemia vera is a prerequisite to achieving hematologic remission in response to cytoreductive therapy.
We compared the clinical and hematologic responses to change in %V617F (molecular response) in 73 patients with polycythemia vera treated with either interferon (rIFNα-2b: 28, Peg-rIFNα-2a: 18) or non-interferon drugs (n=27), which included hydroxyurea (n=8), imatinib (n=12), dasatinib (n=5), busulfan (n=1), and radioactive phosphorus (n=1). Hematologic response evaluation employed Polycythemia Vera Study Group criteria, and molecular response evaluation, European Leukemia Net criteria.
Of the 46 treated with interferon, 41 (89.1%) had a hematologic response, whereas only 7 (15.2%) had a partial molecular response. Of the 27 who received non-interferon treatments, 16 (59.3%) had a hematologic response, but only 2 (7.4%) had a molecular response. Median duration of follow up was 2.8 years. Statistical agreement between hematologic response and molecular response was poor in all treatment groups.
Generally, hematologic response was not accompanied by molecular response. Therefore, a quantitative change in %V617F is not required for clinical response in patients with polycythemia vera.
虽然 JAK2(V617F)等位基因负荷（%V617F）的降低已被认为是真性红细胞增多症对细胞减少治疗有反应的一个标准，但它作为反应监测的价值尚不清楚。本研究的目的是确定真性红细胞增多症患者中%V617F 的降低是否是对细胞减少治疗有反应达到血液学缓解的前提。
我们比较了 73 例接受干扰素（rIFNα-2b：28 例， Peg-rIFNα-2a：18 例）或非干扰素药物（n=27）治疗的真性红细胞增多症患者的临床和血液学反应与%V617F（分子反应）的变化，非干扰素药物包括羟基脲（n=8）、伊马替尼（n=12）、达沙替尼（n=5）、白消安（n=1）和放射性磷（n=1）。血液学反应评估采用真性红细胞增多症研究组标准，分子反应评估采用欧洲白血病网络标准。
在接受干扰素治疗的 46 例患者中，41 例（89.1%）有血液学反应，而仅有 7 例（15.2%）有部分分子反应。在接受非干扰素治疗的 27 例患者中，16 例（59.3%）有血液学反应，但仅有 2 例（7.4%）有分子反应。中位随访时间为 2.8 年。在所有治疗组中，血液学反应与分子反应的统计学一致性均较差。
一般来说，血液学反应并不伴有分子反应。因此，真性红细胞增多症患者的临床反应不需要定量改变%V617F。
