Myelodysplastic syndromes (MDS) treated with DNMTI therapy have responses according to the 2006 IWG response criteria. CR responses have had the strongest association with OS. Recently, CR with partial hematologic recovery (CRh; i.e. blasts <5%, ANC > 500, platelets > 50) has been evaluated in AML, but its relevance is unknown in MDS. We identified adult patients with MDS treated with DNMTIs. We assessed best overall response to therapy according to IWG 2006 criteria, and subsequently identified patients meeting CRh criteria from the subgroup with SD or mCR. We evaluated duration of therapy and overall survival according to response. We identified 311 patients with MDS who received treatment between 2007 and 2018. The median age at the time of therapy was 69 years (range 23–91). Median follow up was 60 months. According to IWG 2006, responses included CR (n = 43, 14%), PR (n = 2, 1%), mCR (n = 57, 18%), SD (n = 149, 48%) and PD (n = 60, 19%). 79 patients (25%) achieved HI. A total of 62 patients (20%) met CRh criteria leading to reclassification of mCR (now n = 26, 8%) or SD (now n = 118, 38%). Patients achieving CR had similar time on therapy (median 8.1mo) compared to CRh (median 6mo, HR 1.4, 95% CI 0.9–2.0), and longer than other responses (p < 0.001). OS varied according to response; median OS was similar between CR (23.3mo) and CRh (25mo, HR 1.28 [0.79–2.08]), which was longer than those with mCR (17.2mo, HR 1.71 [0.96–3.05]), SD (16.3mo, HR 1.61 [1.04–2.48]), and PD (8.7mo, HR 3.04 [1.91–4.83]) (p < 0.001). OS associations with CR/CRh were confirmed in multivariable analysis accounting for allogeneic transplant. MDS patients who achieve a CRh response had similar survival and duration on therapy as patients who achieve CR response and superior to other IWG responses. These data support further evaluation of CRh into future response criteria and clinical trials.
接受DNMTI治疗的骨髓增生异常综合征（MDS）根据2006年IWG疗效标准评估治疗反应。完全缓解（CR）与总生存期（OS）的相关性最强。近期，在急性髓系白血病（AML）中已对伴有部分血液学恢复的完全缓解（CRh；即原始细胞<5%、中性粒细胞绝对计数>500、血小板>50）进行了评估，但其在MDS中的意义尚不明确。我们纳入了接受DNMTIs治疗的成人MDS患者，根据IWG 2006标准评估最佳总体治疗反应，并从疾病稳定（SD）或骨髓完全缓解（mCR）亚组中筛选出符合CRh标准的患者。我们依据不同治疗反应评估了治疗持续时间和总生存期。共纳入2007年至2018年间接受治疗的311例MDS患者，治疗中位年龄69岁（范围23-91岁），中位随访时间60个月。根据IWG 2006标准，治疗反应包括：CR（43例，14%）、部分缓解（PR）（2例，1%）、mCR（57例，18%）、SD（149例，48%）和疾病进展（PD）（60例，19%）。79例患者（25%）达到血液学改善（HI）。共有62例患者（20%）符合CRh标准，导致原mCR（现为26例，8%）和SD（现为118例，38%）患者重新分类。获得CR的患者治疗持续时间（中位8.1个月）与CRh患者（中位6个月，HR 1.4，95% CI 0.9–2.0）相似，且长于其他治疗反应（p<0.001）。不同反应组的总生存期存在差异：CR组（中位23.3个月）与CRh组（中位25个月，HR 1.28 [0.79–2.08]）中位OS相近，且均长于mCR组（17.2个月，HR 1.71 [0.96–3.05]）、SD组（16.3个月，HR 1.61 [1.04–2.48]）和PD组（8.7个月，HR 3.04 [1.91–4.83]）（p<0.001）。在校正异基因移植因素的多变量分析中，CR/CRh与OS的相关性得到确认。达到CRh反应的MDS患者，其生存期和治疗持续时间与达到CR反应者相似，且优于其他IWG标准定义的反应。这些数据支持将CRh纳入未来疗效标准和临床试验中进行进一步评估。
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