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初诊骨髓增生异常综合征成年患者的自发缓解:一种可能发生的情况。

Spontaneous remission in adult patients with de novo myelodysplastic syndrome: a possible event.

作者信息

Petti M C, Latagliata R, Breccia M, Alimena G, Spadea A, D'Andrea M, Mancini M, Aloe Spiriti M A, Mandelli F

机构信息

Dipartimento di Biotecnologie Cellulari ed Ematologia, University La Sapienza, via Benevento 6, 00161 Rome, Italy.

出版信息

Haematologica. 2001 Dec;86(12):1277-80.

Abstract

BACKGROUND AND OBJECTIVES

Spontaneous remission (SR) in de novo myelodysplastic syndromes (MDS) is a rare event, which has been so far described only in children with monosomy 7. The phenomenon is extremely heterogeneous, perhaps depending on different pathogeneses of the disease.

DESIGN AND METHODS

We retrospectively evaluated the outcome of 564 consecutive adult patients with primary MDS diagnosed at our Institution in a 12-year period. SR was defined as an unexpected improvement lasting more than 1 year without concomitant treatments other than vitamins or low-dose steroids (in patients with platelets < 50 x 10(9)/L).

RESULTS

Nine cases of SR were observed in 3 males and 6 females (median age 38.7 years). At diagnosis, all patients had Hb levels < 10 g/dL and 8/9 required packed red cell transfusions. The median time from diagnosis to SR was 18 months (range 4-46) and all patients had normalization of peripheral blood parameters: in 2 out of 3 patients with karyotypic abnormalities at onset, a cytogenetic remission was documented. The median duration of SR was 56 months; 5 patients are still in SR and 4 patients have relapsed (1 as MDS and 3 as acute myeloid leukemia).

INTERPRETATION AND CONCLUSIONS

SR is a rare (less than 2% in our experience) but possible event also in adult MDS patients. It should be kept in mind in the evaluation of experimental treatments for MDS in which very low rates of complete responses are expected.

摘要

背景与目的

初诊骨髓增生异常综合征(MDS)中的自发缓解(SR)是一种罕见事件,迄今为止仅在7号染色体单体的儿童中有所描述。该现象极为异质性,可能取决于疾病的不同发病机制。

设计与方法

我们回顾性评估了在12年期间于我院确诊的564例连续性成年原发性MDS患者的结局。SR定义为未经维生素或低剂量类固醇(血小板<50×10⁹/L的患者)以外的其他治疗而持续超过1年的意外改善。

结果

观察到9例SR,其中男性3例,女性6例(中位年龄38.7岁)。诊断时,所有患者血红蛋白水平<10g/dL,9例中有8例需要输注浓缩红细胞。从诊断到SR的中位时间为18个月(范围4 - 46个月),所有患者外周血参数均恢复正常:3例起病时伴有核型异常的患者中有2例记录到细胞遗传学缓解。SR的中位持续时间为56个月;5例患者仍处于SR状态,4例患者复发(1例为MDS,3例为急性髓系白血病)。

解读与结论

SR在成年MDS患者中是一种罕见(根据我们的经验少于2%)但可能发生的事件。在评估预期完全缓解率极低的MDS实验性治疗时应予以考虑。

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