Zhao Xin, Zhang Li, Jing Liping, Zhou Kang, Li Yuan, Peng Guangxin, Ye Lei, Li Yang, Li Jianping, Fan Huihui, Song Lin, Yang Wenrui, Zhang Fengkui
Department of Anemia Therapeutic Center, Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences and Peking Union Medical College, No. 288 Nanjing Road, Heping District, Tianjin, 300020, People's Republic of China.
Ann Hematol. 2015 Jul;94(7):1105-10. doi: 10.1007/s00277-015-2348-5. Epub 2015 Mar 19.
Whether paroxysmal nocturnal hemoglobinuria (PNH) clone in aplastic anemia (AA) is a prognostic factor to immunosuppressive therapy is a subject of debate. We evaluated hematological responses to immunosuppressive therapy (IST) in severe AA (SAA) patients with or without the presence of a PNH clone. In 97 SAA patients who received first-line IST between January and December 2011, 24 (24.7 %) had a PNH clone prior to treatment, with a median clone size of 7.82 % (range 1.19-45.46 %). The response rates to IST for patients with or without a PNH clone were 66.7 and 50.7 % (P < 0.172), 79.2 and 57.5 % (P < 0.057), and 79.2 and 67.1 % (P < 0.264) at 3, 6, and 12 months, respectively. Combined rate of complete and good partial responses differed between patients with or without a PNH clone: insignificantly at 3 months (41.7 vs. 21.9 %, P < 0.058), but significantly at 6 (66.7 vs. 31.5 %, P < 0.002) and 12 (75.0 vs. 46.6 %, P < 0.015) months. Multivariate analysis revealed that a pretreatment neutrophil count of >0.2 × 10(9)/L is indicative of a better response, while the presence of a PNH clone is predictive to a higher combined rate of complete and good partial responses. This study demonstrated that the presence of a PNH clone could predict a better hematological response instead of a higher response rate in patients with SAA.
再生障碍性贫血（AA）患者中阵发性睡眠性血红蛋白尿（PNH）克隆是否为免疫抑制治疗的预后因素是一个有争议的话题。我们评估了有或无PNH克隆的重型再生障碍性贫血（SAA）患者对免疫抑制治疗（IST）的血液学反应。在2011年1月至12月接受一线IST治疗的97例SAA患者中，24例（24.7%）在治疗前存在PNH克隆，克隆大小中位数为7.82%（范围1.19 - 45.46%）。有或无PNH克隆患者在3、6和12个月时对IST的反应率分别为66.7%和50.7%（P < 0.172）、79.2%和57.5%（P < 0.057）以及79.2%和67.1%（P < 0.264）。完全缓解和部分缓解良好的联合率在有或无PNH克隆的患者中有所不同：3个月时无显著差异（41.7%对21.9%，P < 0.058），但6个月时差异显著（66.7%对31.5%，P < 0.002），12个月时差异也显著（75.0%对46.6%，P < 0.015）。多因素分析显示，治疗前中性粒细胞计数>0.2×10⁹/L提示反应较好，而PNH克隆的存在预示完全缓解和部分缓解良好的联合率较高。本研究表明，PNH克隆的存在可预测SAA患者有更好的血液学反应，而非更高的反应率。
