Long- term outcome of thalidomide and cyclosporine in patients with IPSS low/intermediate- 1 myelodysplastic syndromes

• Published in: Zhōnghuá xuèyèxué zázhì Vol. 36; no. 11; pp. 942 - 946
• Main Authors: Wang, Jingya, Qin, Tiejun, Xu, Zefeng, Zhang, Yue, Zhang, Hongli, Fang, Liwei, Pan, Lijuan, Hu, Naibo, Qu, Shiqiang, Li, Bing, Xiao, Zhijian
• Format: Journal Article
• Language: Chinese
• Published: China 01.11.2015
• Subjects: Adaptor Proteins, Signal Transducing; Anemia; Blood Platelets; Blood Transfusion; Bone Marrow; China; Cyclosporine - therapeutic use; Erythrocytes; Humans; Myelodysplastic Syndromes - classification; Myelodysplastic Syndromes - drug therapy; Neutrophils; Peptide Hydrolases - metabolism; Remission Induction; Retrospective Studies; Thalidomide - therapeutic use; Treatment Outcome; Ubiquitin-Protein Ligases; China
• ISSN: 0253-2727
• DOI: 10.3760/cma.j.issn.0253-2727.2015.11.011

To investigate the long- term outcome of cyclosporin A (CsA) combined with thalidomide regime for Chinese patients with IPSS low/intermediate- 1 myelodysplastic syndromes (MDS) without del（5q）and the predictive variables which could impact the response to the therapy. Seventy-six MDS patients who were treated with these drugs at a single institute in China were retrospectively analyzed. The polymorphism of cereblon gene, rs1672753, was detected in patients of this cohort by PCR and direct sequencing. A total of 53% of patients showed hematological improvement（HI）to the therapy. Thirty-one patients（31/73, 43%）achieved erythrocyte response（HI-E）; 15 patients（15/50, 30%）achieved neutrophil response（HI-N); 18 patients（18/58, 31%）achieved platelet response（HI-P). Twenty-seven of the 50 patients（46%）who were dependent on red blood cell transfusion achieved HI- E and became independent of transfusion. The median duration of response among the responders was 22 months (range, 1- 131 + months). Bone marrow blasts ≤2%