在第三阶段口服JAK抑制剂治疗控制骨髓纤维化症临床试验COMFORT-I和COMFORT-II的综合分析中，随机分组接受口服瑞秋利珠单抗或在安慰剂或最佳治疗方案 (BAT) 转换后接受口服瑞秋利珠单抗的中风险-2或高风险骨髓纤维化成年患者的总生存率得到了改善。这项后期分析对疾病持续时间 (自诊断起≤12个月或>12个月) 进行了相关疾病结果的检查，在研究中包括了525名患者（瑞秋利珠单抗：≤12个月，n = 84；>12个月，n = 216；安慰剂/BAT：≤12个月，n = 66；>12个月，n = 159），他们的年龄中位数为65.0-70.0岁。较早开始瑞秋利珠单抗治疗的患者中，出现的血小板减少和贫血事件较少。在第24周和第48周，较早开始瑞秋利珠单抗治疗的患者脾脏体积反应 (SVR) 更高 (在第24周为47.6%，与在第48周的32.9% 相比，p = .0610)；在与安慰剂/BAT组的治疗相比，SVR也更高 (在第48周为44.0%，与在第24周的26.9%相比，p = .0149)。通过一项多元分析，控制混杂因素后发现，短疾病持续时间的患者与长持续时间的患者相比，脾脏体积减少更明显 (p = .022)。在第240周，较早开始瑞秋利珠单抗治疗的患者的总生存率明显提高 (63% [95% CI，51%-73%] vs. 57% [95% CI，49%-64%]，危险比为1.53；95% CI，1.01-2.31；p = .0430)。无论疾病持续时间如何，接受瑞秋利珠单抗治疗的患者的总生存率均较接受安慰剂/BAT 的患者更长。这些发现表明，对于中风险-2和高风险骨髓纤维化成年患者，早期开始瑞秋利珠单抗治疗可能会改善临床结果，包括更少的细胞减少事件，持久的SVR和延长的总生存率。骨髓纤维化患者通常不像普通人那样长寿，他们可能还伴随有脾脏增大和疲劳等困难的症状。两项大型临床试验表明，接受瑞秋利珠单抗治疗的患者的生存状况更佳，症状也较接受安慰剂或其他标准治疗的患者有所改善。在此研究中，检查了早期开始瑞秋利珠单抗治疗 (即在诊断后的一年内) 是否比延迟治疗更有益。接受瑞秋利珠单抗治疗的患者中，早期治疗的患者比延迟治疗的患者更长寿，疾病症状减少。© 2023 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society.

In a pooled analysis of the phase 3 Controlled Myelofibrosis Study With Oral JAK Inhibitor Treatment I (COMFORT-I) and COMFORT-II clinical trials, adult patients with intermediate-2 or high-risk myelofibrosis who received oral ruxolitinib at randomization or after crossover from placebo or best available therapy (BAT) had improved overall survival (OS).This post hoc analysis of pooled COMFORT data examined relevant disease outcomes based on the disease duration (≤12 or >12 months from diagnosis) before ruxolitinib initiation.The analysis included 525 patients (ruxolitinib: ≤12 months, n = 84; >12 months, n = 216; placebo/BAT: ≤12 months, n = 66; >12 months, n = 159); the median age was 65.0-70.0 years. Fewer thrombocytopenia and anemia events were observed among patients who initiated ruxolitinib treatment earlier. At Weeks 24 and 48, the spleen volume response (SVR) was higher for patients who initiated ruxolitinib earlier (47.6% vs. 32.9% at Week 24, p = .0610; 44.0% vs. 26.9% at Week 48, p = .0149). In a multivariable analysis of factors associated with spleen volume reduction, a logistic regression model that controlled for confounding factors found that a significantly greater binary reduction was observed among patients with shorter versus longer disease duration (p = .022). At Week 240, OS was significantly improved among patients who initiated ruxolitinib earlier (63% [95% CI, 51%-73%] vs. 57% [95% CI, 49%-64%]; hazard ratio, 1.53; 95% CI, 1.01-2.31; p = .0430). Regardless of disease duration, a longer OS was observed for patients who received ruxolitinib versus those who received placebo/BAT.These findings suggest that earlier ruxolitinib initiation for adult patients with intermediate-2 and high-risk myelofibrosis may improve clinical outcomes, including fewer cytopenia events, durable SVR, and prolonged OS.Patients with myelofibrosis, a bone marrow cancer, often do not live as long as the general population. These patients may also have an enlarged spleen and difficult symptoms such as fatigue. Two large clinical trials showed that patients treated with the drug ruxolitinib lived longer and had improved symptoms compared to those treated with placebo or other standard treatments. Here it was examined whether starting treatment with ruxolitinib earlier (i.e., within a year of diagnosis) provided benefits versus delaying treatment. Patients who received ruxolitinib within a year of diagnosis lived longer and experienced fewer disease symptoms than those whose treatment was delayed.© 2023 The Authors. Cancer published by Wiley Periodicals LLC on behalf of American Cancer Society.