Transfusion-related cost offsets and time burden in patients with myelofibrosis on momelotinib vs. danazol from MOMENTUM

<b>Aim:</b> To estimate projected US-based cost and time burden for patients with myelofibrosis and anemia treated with momelotinib compared with danazol. <b>Methods:</b> Cost and time burden were calculated based on the transfusion status of patients in the MOMENTUM trial and estimates extracted from previous studies. <b>Results:</b> Reductions in transfusion associated with momelotinib are projected to result in cost and time savings compared with danazol in transfusion-dependent and transfusion-independent/requiring patients with myelofibrosis, respectively: annual medical costs ($53,143 and $46,455 per person), outpatient transfusion costs ($42,021 and $8,370 per person) and annual time savings (173 and 35 h per person). <b>Conclusion:</b> Fewer transfusions with momelotinib are projected to result in cost and time savings in patients with myelofibrosis and anemia compared with danazol. <b>Estimated cost &amp; time savings in patients with the blood cancer myelofibrosis</b> Myelofibrosis is a rare blood cancer often associated with bone marrow damage, too few of some types of blood cells and symptoms including tiredness, night sweating, itching and feelings of fullness and pain because of increased spleen size. Patients with anemia (too few red blood cells) may require regular blood transfusions and this is one sign that myelofibrosis is getting worse. MOMENTUM was a Phase III clinical trial showing that the drug momelotinib was safe and effective in patients with myelofibrosis who were previously treated with a type of drug called a JAK inhibitor. In particular, the trial showed that momelotinib reduced the need for transfusions compared with danazol, another drug typically used to treat patients with anemia. Based on this transfusion information from MOMENTUM and other publicly available information about estimated medical costs and patients' time spent in receiving transfusions, the analysis described here shows that a reduction in the number of transfusions with momelotinib compared with danazol is estimated to lead to cost savings as well as reduced patient time spent in transfusion-related travel, preparing and waiting for transfusions and receiving and recovering from transfusions. Reductions in transfusions associated with momelotinib are projected to result in savings in medical and transfusion-related costs and time burden relative to danazol in patients with myelofibrosis and anemia. Momelotinib is a JAK1, JAK2 and ACVR1 inhibitor that targets both the complex drivers of iron-restricted anemia and chronic inflammation in MF, thereby improving constitutional symptoms and splenomegaly while also maintaining or improving Hb levels across the continuum of JAK inhibitor–naive and previously JAK inhibitor–treated patients. Transfusion dependency and moderate-to-severe anemia are critical negative prognostic factors in MF and lead to significantly higher rates of HCRU and costs in affected patients compared with non-TD patients. MOMENTUM is a randomized, double-blind study comparing momelotinib and danazol in symptomatic patients with MF and anemia who previously received JAK inhibitor therapy. Projected differences in cost and time burden associated with momelotinib relative to danazol were based on patient transfusion data from the MOMENTUM trial and cost estimates from studies using the IBM MarketScan Commercial Database and Medicare fee-for-service database as well as time burden estimates from the 2022 Knoth et al. study. Reductions in transfusions associated with momelotinib are projected to result in savings in medical and transfusion-related costs and patient time savings relative to danazol in patients with baseline TD and TI/TR status, respectively, using a terminal TI rate: annual medical costs ($53,143 and $46,455 per person), outpatient transfusion costs ($42,021 and $8,370 per person) and time savings (173 and 35 h per person). Projected savings in total annual medical costs with momelotinib relative to danazol were also shown using a rolling TI rate in patients with baseline TD and TI/TR status ($23,780 and $39,305). Projected savings in transfusion-related costs and patient time savings with momelotinib relative to danazol were also observed for patients aged ≥65 years. The results of this analysis demonstrate that momelotinib not only addresses a high unmet need in patients with MF-associated anemia but may also provide medical cost and time savings for patients by reducing their transfusion dependency.

<b>研究目的：</b> 本研究旨在对比达那唑（danazol），评估美国骨髓纤维化（myelofibrosis）合并贫血患者接受莫美替尼（momelotinib）治疗时的预计医疗成本与时间负担。
<b>研究方法：</b> 医疗成本与时间负担的计算基于MOMENTUM试验（MOMENTUM trial）中患者的输血状态，以及既往研究提取的相关估算数据。
<b>研究结果：</b> 相较于达那唑，莫美替尼可减少输血需求，预计分别在输血依赖（transfusion-dependent, TD）与非输血依赖/需输血（transfusion-independent/requiring, TI/TR）的骨髓纤维化患者中实现成本与时间节约：年医疗成本分别为每人53143美元与46455美元，门诊输血成本分别为每人42021美元与8370美元，年时间节约分别为每人173小时与35小时。
<b>研究结论：</b> 相较于达那唑，莫美替尼可减少输血需求，预计能为骨髓纤维化合并贫血患者带来医疗成本与时间节约。
<b>血液癌症骨髓纤维化患者的预计成本与时间节约</b>
骨髓纤维化（myelofibrosis）是一种罕见血液癌症，常伴随骨髓损伤、部分血细胞数量减少，以及因脾脏增大引发的疲劳、夜间盗汗、瘙痒、饱胀感与疼痛等症状。贫血（anemia，即红细胞数量不足）患者可能需要定期输血，而这也是骨髓纤维化病情恶化的标志之一。
MOMENTUM试验（MOMENTUM trial）是一项Ⅲ期临床试验，结果显示莫美替尼（momelotinib）在既往接受过JAK抑制剂（JAK inhibitor）治疗的骨髓纤维化患者中具有安全性与有效性。具体而言，相较于常用于治疗贫血的达那唑（danazol），莫美替尼可降低患者的输血需求。
基于MOMENTUM试验的输血相关数据，以及现有公开的医疗成本估算与患者输血相关耗时数据，本分析显示，与达那唑相比，莫美替尼可减少输血次数，预计能够节约医疗成本，同时减少患者在输血相关的出行、准备、等待、接受输血及术后恢复上花费的时间。
莫美替尼是一种JAK1、JAK2与ACVR1抑制剂，可同时靶向骨髓纤维化（MF）中铁限制性贫血与慢性炎症的复杂致病机制，从而改善全身症状与脾肿大（splenomegaly），同时在既往未接受过JAK抑制剂治疗及既往接受过JAK抑制剂治疗的患者群体中，维持或提升血红蛋白（Hb）水平。
输血依赖与中重度贫血是骨髓纤维化的关键不良预后因素，与非输血依赖患者相比，受影响患者的健康护理资源使用（HCRU）率与医疗成本显著更高。
MOMENTUM试验是一项随机双盲研究，对比了莫美替尼与达那唑在既往接受过JAK抑制剂治疗的有症状骨髓纤维化合并贫血患者中的疗效。
本次分析中，莫美替尼相较于达那唑的预计成本与时间负担差异，基于MOMENTUM试验的患者输血数据，以及使用IBM MarketScan商业数据库（IBM MarketScan Commercial Database）与医疗保险按服务收费数据库（Medicare fee-for-service database）得出的成本估算，同时结合2022年Knoth等人研究中的时间负担估算数据。
针对基线为输血依赖与非输血依赖/需输血状态的患者，若采用终末非输血依赖率计算，莫美替尼相关的输血减少预计可带来医疗与输血相关成本节约及患者时间节约：年医疗成本分别为每人53143美元与46455美元，门诊输血成本分别为每人42021美元与8370美元，时间节约分别为每人173小时与35小时。
若采用滚动非输血依赖率计算，莫美替尼相较于达那唑的预计年总医疗成本节约在基线输血依赖与非输血依赖/需输血患者中分别为每人23780美元与39305美元。
针对年龄≥65岁的患者，莫美替尼相较于达那唑也可实现输血相关成本节约与患者时间节约。
本分析结果表明，莫美替尼不仅可满足骨髓纤维化相关性贫血患者未被满足的高医疗需求，还可通过降低患者的输血依赖程度，为其节约医疗成本与时间。