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[环孢素A治疗骨髓增生异常综合征患者的疗效]

[Efficiency of cyclosporin A therapy in patients with myelodysplastic syndrome].

作者信息

Kokhno A V, Parovichnikova E N, Mikhaĭlova E A, Ustinova E N, Kaplanskaia I B, Dvirnyk V N, Ol'shanskaia Iu V, Domracheva E V, Savchenko V G

出版信息

Ter Arkh. 2010;82(8):48-53.

PMID:20873246
Abstract

AIM

To evaluate the efficacy of cyclosporin A (CsA) in patients with myelodysplastic syndromes (MDS) and to identify determinants of a response to this therapy.

SUBJECTS AND METHODS

The efficacy of CsA was evaluated in 52 patients (30 men and 22 women aged 16 to 74 years) with MDS. Thirty-two patients were given CsA as first-line therapy; 20 patients took the agent after prior therapy. CsA was used in a daily oral dose of 5 mg/kg. Its efficacy was evaluated following 3, 6, and 12 months. Actuarial survival was determined by the Kaplan-Meier method.

RESULTS

The efficacy of CsA used as first- and second-line therapy was 56 and 55%, respectively; complete remissions were achieved in 19 and 20% of cases. Baseline refractory anemia (RA) transformed to RA with excess blasts (RAEB) in 31% of cases; baseline RAEB did to acute myeloid leukemia in 34%. Overall survival was significantly associated with bone marrow (BM) blast cell percentage (< 5% or > 5%; p = 0.0009), BM cellularity (hypoplasia and focal hypoplasia of hematopoiesis or BM hyperplasia; p = 0.03), focal polyclonal lymphoid infiltration in the BM (p = 0.01) and karyotype anomalies (low, moderate, and high risks; p = 0.001).

CONCLUSION

CsA is the drug of choice in treating patients with MDS, including RA, RA with ringed sideroblasts, refractory cytopenia with multilineage dysplasia, with hypoplasia of hematopoiesis, with nodular polyclonal lymphoid infiltration in the BM, a normal karyotype or changes corresponding to a low or moderate IPSS risk.

摘要

目的

评估环孢素A(CsA)对骨髓增生异常综合征(MDS)患者的疗效,并确定该治疗反应的决定因素。

对象与方法

对52例(30例男性和22例女性,年龄16至74岁)MDS患者评估CsA的疗效。32例患者接受CsA作为一线治疗;20例患者在先前治疗后使用该药物。CsA的口服剂量为每日5mg/kg。在3、6和12个月后评估其疗效。采用Kaplan-Meier法确定精算生存率。

结果

CsA作为一线和二线治疗的疗效分别为56%和55%;19%和20%的病例实现了完全缓解。31%的病例中,基线难治性贫血(RA)转变为伴有过多原始细胞的RA(RAEB);34%的基线RAEB转变为急性髓系白血病。总生存期与骨髓(BM)原始细胞百分比(<5%或>5%;p=0.0009)、BM细胞密度(造血发育不全和局灶性造血发育不全或BM增生;p=0.03)、BM局灶性多克隆淋巴细胞浸润(p=0.01)和核型异常(低、中、高风险;p=0.001)显著相关。

结论

CsA是治疗MDS患者的首选药物,这些患者包括RA、伴有环形铁粒幼细胞的RA、多系发育异常的难治性血细胞减少症、造血发育不全、BM有结节性多克隆淋巴细胞浸润、核型正常或对应低或中度国际预后评分系统(IPSS)风险的改变。

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