Bonifazi F, de Vivo A, Rosti G, Guilhot F, Guilhot J, Trabacchi E, Hehlmann R, Hochhaus A, Shepherd P C, Steegmann J L, Kluin-Nelemans H C, Thaler J, Simonsson B, Louwagie A, Reiffers J, Mahon F X, Montefusco E, Alimena G, Hasford J, Richards S, Saglio G, Testoni N, Martinelli G, Tura S, Baccarani M
Blood. 2001 Nov 15;98(10):3074-81. doi: 10.1182/blood.v98.10.3074.
Achieving a complete cytogenetic response (CCgR) is a major target in the treatment of chronic myeloid leukemia (CML) with interferon-alpha (IFN-alpha), but CCgRs are rare. The mean CCgR rate is 13%, in a range of 5% to 33%. A collaborative study of 9 European Union countries has led to the collection of data on 317 patients who were first seen between 1983 and 1997 and achieved CCgRs with IFN-alpha alone or in combination with hydroxyurea. The median time to first CCgR was 19 months (95% CI, 17-21; range, 3-84 months). At last contact, 212 patients were still alive and in continuous CCgR; 105 patients had lost CCgR, but 53% of them were still alive and in chronic phase. IFN-alpha treatment was discontinued permanently in 23 cases for response loss, in 36 cases for chronic toxicity (15 are still in unmaintained continuous CCgR), and in 8 cases because it was believed that treatment was no longer necessary (7 of these 8 patients are still in unmaintained continuous CCgR). The 10-year survival rate from first CCgR is 72% (95% CI, 62%-82%) and is related to the risk profile. High-risk patients lost CCgR more frequently and more rapidly and none survived more than 10 years. Low-risk patients survived much longer (10-year survival probability 89% for Sokal low risk and 81% for Euro low risk). These data point out that a substantial long-term survival in CCgRs is restricted mainly to low-risk and possibly intermediate-risk patients and occurs significantly less often in high-risk patients.
实现完全细胞遗传学缓解（CCgR）是使用α干扰素（IFN-α）治疗慢性髓性白血病（CML）的主要目标，但CCgR很少见。平均CCgR率为13%，范围在5%至33%之间。一项对9个欧盟国家的合作研究收集了317例患者的数据，这些患者于1983年至1997年间首次就诊，单独使用IFN-α或与羟基脲联合使用后实现了CCgR。首次达到CCgR的中位时间为19个月（95%CI，17 - 21；范围，3 - 84个月）。在最后一次随访时，212例患者仍存活且处于持续CCgR状态；105例患者失去了CCgR，但其中53%仍存活且处于慢性期。23例因缓解丧失而永久停用IFN-α治疗，36例因慢性毒性（15例仍处于未维持的持续CCgR状态），8例因认为不再需要治疗（这8例患者中有7例仍处于未维持的持续CCgR状态）。从首次CCgR开始计算的10年生存率为72%（95%CI，62% - 82%），且与风险特征相关。高危患者更频繁、更快速地失去CCgR，且无一人存活超过10年。低危患者存活时间长得多（索卡尔低危患者10年生存概率为89%，欧洲低危患者为81%）。这些数据表明，CCgR患者的长期生存主要局限于低危以及可能的中危患者，高危患者中这种情况明显较少发生。
