摘要
目的分析伴阵发性睡眠性血红蛋白尿症（PNH）克隆的骨髓增生异常综合征（MDS）的临床特点及其对疗效和预后的影响。方法回顾性分析2004年10月至2012年6月诊断明确的31例伴PNH克隆的MDS患者临床资料，分析其临床特点及PNH克隆大小对疗效和生存的影响。结果①31例患者均行染色体核型分析，未见分裂象者1例，正常核型23例（76．7％），异常核型7例（23．3％）。其中＋8者3例，-Y2例，del（7q）1例，xp＋1例。根据IPSS积分系统，低危组1例，中危-1组27例，中危-2组2例，高危组1例，治疗有效组与无效或稳定组间感染发生率、中性粒细胞绝对计数（ANC）、网织红细胞（Ret）计数及IPSS积分差异均有统计学意义（P值分别为0．049、0．006、0．031及0．043）。②免疫抑制治疗（IST）后完全缓解4例（12．9％），显效及有效者17例（54．8％），总体有效率67．7％。31例患者中位随访19（3～59）个月，无一例转化为急性白血病，3年及5年总体生存（OS）率分别为82．7％与55．1％。③单因素分析显示，年龄／〉45岁、感染及ANC〈0．5×10^9几为影响患者长期生存的高危因素（P值分别为0．050、0．031及0．026）。④单因素及COX多因素分析均未发现PNH克隆大小与疗效和长期生存的相关性（P=0．393）。结论伴PNH克隆的MDS异常染色体发生概率相对较低，IST疗效良好，较少转化为急性白血病，且PNH克隆大小对疗效及生存无明显影响。
Objective To analyze the clinical characteristics and risk factors on responses and survival of myelodysplastic syndromes （MDS） patients with paroxysmal nocturnal hemoglobinuria （PNH） clones. Methods The clinical data of 31 MDS cases with PNH clones from October 2004 to June 2012 were retrospectively analyzed to reveal the influence of PNH clone size on responses and survival. Results （1）The chromosome karyotypes were analyzed in all patients, 23 patients with normal karyotype, 7 patients with abnotmal karyotype [ including 3 patients with ＋ 8, 2 - Y, 1 del（Tq） and 1 Xp ＋ I and 1 patient with no mito- sis. 1 patient belonged to low-risk, 27 intermediate-1 risk, 2 intermediate-2 risk and 1 high-risk groups, re- spectively, according to IPSS. There were significantly statistical differences between responders and nonresponders in terms of infection, ANC, Reticulocyte count and IPSS （P values were 0. 049, 0. 006, 0.031 and 0. 043, respectively）. （2）The overall responsive rate was 67.7% , no patients progressed to acute leukemia （AL） during median follow-up of 19 months after immnnosuppressive therapy （IST）. The 3-year and 5-year overall survival rates were 82.7% and 55.1% , respectively. （3）According to univariate analysis, age, infec- tion and ANC had significant influence on survival （P values were 0. 050, 0. 031 and 0. 026, respectively）. （4）The PNH clone size had no significant influence on survival through univariate and COX analyses （P = 0. 393 ）. Conclusion MDS patients with PNH clone had less cytogenetic abnormalities, higher probability of response to IST and lower probability of progression to AL; Furthermore, the PNH clone size had no significant influence on response and survival.
出处
《中华血液学杂志》
CAS
CSCD
北大核心
2013年第3期242-246,共5页
Chinese Journal of Hematology
基金
卫生行业科研专项基金（201202017）
国家科技重大专项基金（2011ZX09302-007-4）
关键词
骨髓增生异常综合征
血红蛋白尿
阵发性
GPI锚链蛋白
预后
Myelodysplastic syndrome
Hemoglobinuria, paroxysmal
Glycosylphosphatidylinositol-anchored proteins
Prognosis
