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. 2024 Jul 29;20(30):2259–2270. doi: 10.1080/14796694.2024.2368450

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

Lucia Masarova a,*, Srdan Verstovsek a, Tom Liu b, Sumati Rao b, Gautam Sajeev c, Mirko Fillbrunn c, Ryan Simpson c, Weilong Li c, Joseph Yang b, Yvette Le Lorier b,‡, Boris Gorsh b,‡, James Signorovitch c
PMCID: PMC11508939  PMID: 39072442

Abstract

Aim: To estimate projected US-based cost and time burden for patients with myelofibrosis and anemia treated with momelotinib compared with danazol.

Methods: Cost and time burden were calculated based on the transfusion status of patients in the MOMENTUM trial and estimates extracted from previous studies.

Results: 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).

Conclusion: Fewer transfusions with momelotinib are projected to result in cost and time savings in patients with myelofibrosis and anemia compared with danazol.

Keywords: : ACVR1, anemia, cost, danazol, JAK inhibitor, momelotinib, myelofibrosis, time burden, transfusion

Plain Language Summary

Estimated cost & time savings in patients with the blood cancer myelofibrosis

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.

Tweetable Abstract

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.

Article highlights.

  • 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.

1. Background

Myelofibrosis (MF) is a chronic, progressive, Philadelphia chromosome–negative myeloproliferative neoplasm (MPN) characterized by anemia, splenomegaly, debilitating symptoms and bone marrow fibrosis [1]. MPNs are rare, with MF estimated to have a worldwide annual incidence rate of 0.47 to 1.98 per 100,000 and a prevalence ranging from 1.76 to 4.05 per 100,000 [2,3]. While MF is often a primary disease, it may also occur post polycythemia vera (PV) or post essential thrombocythemia (ET) [1]. Primary MF is more aggressive and has a poorer prognosis than PV or ET [4], as disease progression with MF can include bone marrow failure, transformation to acute myeloid leukemia and early mortality [5]. While MF can occur at any age, it is most frequently diagnosed in older patients, with a median age of diagnosis between 65 and 70 years [6,7].

Among patients with MF, one-third have anemia and one-quarter have transfusion dependent (TD) anemia by the time of MF diagnosis [8,9]. Over time, most patients with MF develop anemia, which is associated with poor prognosis [9–11]. Any grade of anemia adversely affects the survival of patients with MF, and severe anemia is associated with a >1.5-fold increase in the risk of death compared with moderate anemia [12]. According to a retrospective analysis of 1109 patients with primary MF, median survival was 4.9, 3.4 and 2.1 years for patients with mild (hemoglobin [Hb] ≥10 g/dl but below the sex-adjusted lower limit of normal), moderate (Hb between 8 g/dl and <10 g/dl) and severe (Hb <8 g/dl or TD) MF-associated anemia, respectively [12]. While the emergence of JAK inhibitors has provided substantial benefit for splenomegaly and constitutional symptoms, and may improve survival [13], anemia remains a challenge in MF management. Among currently approved JAK inhibitor therapies for MF, ruxolitinib and fedratinib are intrinsically myelosuppressive and can cause or worsen anemia and exacerbate transfusion dependency [8,14].

Red blood cell (RBC) transfusions are the main treatment option for worsening or severe anemia in patients with MF. However, transfusion dependence is associated with poor survival and may add to the economic burden on patients, their caregivers and the health system via healthcare resource utilization (HCRU) and costs associated with transfusions and managing transfusion complications [15–18]. In a retrospective analysis, transfusion dependence imposed a high cost burden on healthcare systems and patients, with annual total medical costs up to nine-times higher for TD patients than for non-TD patients ($255,200 vs. $27,800) [16]. Other approaches to managing MF-associated anemia include erythropoiesis-stimulating agents, corticosteroids, androgens such as danazol, immunomodulatory drugs and splenectomy [8,19,20]. These strategies have shown modest and transient clinical benefit, but none directly target key mechanistic contributors to MF-associated anemia [21,22].

Momelotinib is an oral inhibitor that targets JAK1, JAK2 and ACVR1 [23,24]. While inhibition of JAK-STAT signaling in MF blocks the inflammatory cytokines and clonal proliferation that lead to extramedullary hematopoiesis and splenomegaly, ACVR1 inhibition increases iron availability, thereby mitigating iron-restricted anemia associated with MF [14,25]. Based on three Phase III trials, momelotinib improved splenomegaly, disease-related symptoms and anemia in patients with JAK inhibitor–naive or –experienced MF [26–28]. In the Phase III MOMENTUM trial (NCT04173494) of JAK inhibitor–experienced, symptomatic patients with MF and anemia, momelotinib demonstrated reduced transfusion burden and achieved durable transfusion independence compared with danazol, a commonly used treatment specifically for MF-associated anemia [20,28]. Given the dual JAK-STAT signaling and ACVR1 inhibitory activities of momelotinib and reported clinical trial efficacy, safety and observed effects on MF-associated anemia and transfusion dependence, there is a potential for cost savings and less resource utilization with momelotinib relative to other treatments. The objective of this study was to estimate the projected differences in total medical costs, transfusion-related costs and patient time burden associated with momelotinib vs. danazol from a US perspective.

2. Materials & methods

2.1. Data sources

To address the study objective, projected differences in cost and time burden associated with momelotinib and danazol were determined based on transfusion independence data from the MOMENTUM trial and cost and time burden data from the published literature.

MOMENTUM is an international, double-blind, randomized, controlled, Phase III study that enrolled patients at 107 sites across 21 countries worldwide [28]. The patient population for MOMENTUM was previously described [28]. Briefly, eligible patients were ≥18 years with a confirmed diagnosis of intermediate-1–, intermediate-2–, or high-risk primary or secondary (post-PV or post-ET) MF who were symptomatic, were anemic, had baseline splenomegaly and were previously treated with an approved JAK inhibitor. Patients were randomly assigned (2:1) to receive momelotinib (200 mg orally once per day) plus danazol placebo (i.e., the momelotinib group) or danazol (300 mg orally twice per day) plus momelotinib placebo (i.e., the danazol group) [28]. Data on clinical efficacy of momelotinib relative to danazol were extracted or calculated from the MOMENTUM trial for use in calculations of projected cost and time burden differences between these groups.

A targeted literature search was conducted to obtain US-based estimates of costs and time burden for patients with MF. Cost estimates were extracted from two separate studies of patients with MF, one a study of adults using the IBM MarketScan Commercial Database and the other a study of adults aged ≥65 years using the Medicare fee-for-service database [16,17]. Overall costs of care were extracted from the IBM MarketScan Claims study, which summarized medical, pharmacy and transfusion-specific costs of adult patients with MF [16]. Cost estimates were reported as annual costs per person in USD, stratified by baseline transfusion status (TD and transfusion independent/requiring [TI/TR]) (Supplementary Table S1 for definition of transfusion status and Supplementary Table S2 for reported costs). Similarly, the Medicare fee-for-service study provided cost-of-care estimates among patients with MF aged ≥65 years, stratified by baseline transfusion status (TI, TR and TD) (Supplementary Tables S1 & S2). While diagnoses of iron overload were captured in the health claims analyses, specific costs related to iron chelation therapy were not available in these data sources.

Time burden estimates were from a cross-sectional web-based survey from the Cooley's Anemia Foundation (the 2022 Knoth et al. study [29]), which provided estimates for a patient's mean time spent for one RBC transfusion procedure in patients with β-thalassemia and included the following time components: preparation, waiting room, waiting for blood, RBC transfusion procedure and recovery and total round trip travel time (Supplementary Table S3) [29].

2.2. Analyses of transfusion status & rates in MOMENTUM

Data on clinical efficacy of momelotinib relative to danazol were extracted or calculated from the MOMENTUM trial for use in subsequent calculations of projected cost and time burden differences between groups. Individual patient data from MOMENTUM were used to determine the proportion of patients who were TI, TR and TD in the momelotinib and danazol trial arms at baseline and at week 24 of the study based on the transfusion status criteria of the MOMENTUM trial (Supplementary Table S1) and the average rate of transfusions in each arm. This TI rate at week 24 was based on the prespecified terminal 12-week definition of no transfusions and all Hb levels ≥8 g/dl in the last 12 weeks before week 24. Analyses were repeated in the subset of patients aged 65 years and older. Estimates included only patients with known transfusion status at week 24 in the base case calculations of projected cost and time burden differences. A separate analysis also evaluated patients based on a rolling TI rate defined as no transfusions and all Hb levels ≥8 g/dl during any 12-week period through week 24 (Supplementary Table S1). Sensitivity analyses under different assumptions about missing transfusion status were also explored. All analyses assumed a sustained effect of treatment on transfusion status.

2.3. Calculating projected differences in cost & time burden

Projected medical costs for momelotinib and danazol, stratified by baseline transfusion status, were calculated by multiplying the cost estimates from the IBM MarketScan study with the proportion of patients with TD or TI/TR status in each group at week 24 in the MOMENTUM trial. Projected cost differences were calculated as the difference between these projected costs in the momelotinib and danazol groups (Supplementary Figure S1). Cost estimates for momelotinib and danazol among patients aged ≥65 years were calculated by multiplying the cost estimates from the Medicare study by the proportion of patients aged ≥65 years with TD, TI or TR status in each group at week 24 in the MOMENTUM trial, and projected cost differences were calculated as the differences between these group-specific estimates. The proportions of patients with TD or TI/TR status were reported in the Clinical Study Report for the overall population and calculated using individual patient-level data for patients aged ≥65 years.

Projected outpatient transfusion costs for momelotinib and danazol were calculated by multiplying cost estimates per outpatient transfusion visit extracted from the literature with rates of transfusion visits for each group in the MOMENTUM trial. Costs per transfusion visit were reported only in the IBM MarketScan Commercial Database study; cost estimates from this study were therefore also used in calculations for patients aged ≥65 years. Rates of transfusion visits for momelotinib and danazol patients were reported in the Clinical Study Report for the overall population and calculated using individual patient-level data for patients aged ≥65 years. Rates of RBC transfusion visits observed over the 24-week trial period in each group were rescaled to reflect annualized rates (Supplementary Figure S1).

Projected transfusion-related time burden for momelotinib and danazol was calculated by multiplying the estimated time spent on average per transfusion visit from the Knoth et al. study [29] with the average number of transfusion visits per patient per year within the momelotinib and danazol arms from the MOMENTUM trial, respectively. Projected time savings were calculated as the difference between the projected time burden estimates for momelotinib and danazol. (Supplementary Figure S1).

2.4. Sensitivity analysis

Sensitivity analyses were performed to assess the robustness of the base case estimates based on two methods for imputing unknown transfusion status at week 24 in MOMENTUM. Unknown transfusion status was imputed either as TI/TR in both trial arms or as TD in both trial arms. These analyses assumed that patients were either TI/TR vs. TD without the potential for becoming disease-free (i.e., having no treatment cost). Projected cost and time burden differences were then recalculated under these assumptions.

3. Results

3.1. Transfusion status & rates

At week 24, 143 patients were analyzed (momelotinib arm, n = 102; danazol arm, n = 41) in the MOMENTUM trial. At baseline, 45.1% of patients in the momelotinib arm were TD, and 54.9% of patients were TI/TR; in the danazol arm, 39.0% were TD and 61.0% were TI/TR (Supplementary Table S4A). Among patients who were TD at baseline, 60.9% in the momelotinib arm were TI/TR at the end of the 24-week trial period and 39.1% remained TD. In the danazol arm, 37.5% were TI/TR and 62.5% remained TD. At week 24, 109 patients aged ≥65 years in the MOMENTUM trial were analyzed (momelotinib arm, n = 76; danazol arm, n = 33) (Supplementary Table S4B). In this subpopulation, at baseline, 47.4% of patients in the momelotinib arm were TD and 52.6% were TI/TR; in the danazol arm, 36.4% were TD and 63.6% were TI/TR (Supplementary Table S4B). Among patients aged ≥65 years who were TD at baseline, 63.9% in the momelotinib arm were TI/TR at the end of the 24-week trial period and 36.1% remained TD; in the danazol arm, 33.3% were TI/TR and 66.7% remained TD.

Patients in the momelotinib arm had reduced rates of transfusion visits compared with patients in the danazol arm among those who were TD at baseline (mean, 15.26 vs. 26.34 visits/year) and among those who were TI/TR at baseline (mean, 5.00 vs. 7.21 visits/year) (Supplementary Table S5A). Similarly, patients aged ≥65 years in the momelotinib arm had reduced rates of transfusion visits compared with patients in the danazol arm among those who were TD at baseline (mean, 15.45 vs. 27.34 visits/year) and those who were TI/TR at baseline (mean, 5.81 vs. 6.10 visits/year) (Supplementary Table S5B).

3.2. Projected differences in medical costs

Reduced rates of transfusion dependence resulted in lower projected costs for momelotinib relative to danazol. Stratified by TD and TI/TR status at baseline and using a terminal TI rate, the projected average annual total medical cost savings with momelotinib vs. danazol were $53,143 per patient with baseline TD status ($116,772 vs. $169,915) and $46,455 per patient with baseline TI/TR status ($35,910 vs. $82,365) (Figure 1A). Sensitivity analyses, assuming patients with unknown transfusion status at week 24 were either all TD or all TI/TR, showed that total annual medical service cost savings with momelotinib in patients with baseline TD status ranged from $1,911 to $60,938 per person (Supplementary Table S6). Based on a rolling TI rate, the projected average annual total medical cost savings with momelotinib vs. danazol were $23,780 per patient with baseline TD status ($204,656 vs. $228,437) and $39,305 per patient with baseline TI/TR status ($105,852 vs. $145,157) (Supplementary Figure S2).

Figure 1.

Figure 1.

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Projected medical costs based on transfusion status associated with momelotinib vs. danazol. Projected total annual medical costs in $USD/per person based on transfusion status associated with momelotinib vs. danazol based on the IBM MarketScan Commercial Database (A) and Medicare database (B).

†The IBM MarketScan Commercial Database categorized patients with MF into two groups (TI/TR and TD). Refer to Supplementary Table S1. Costs are stratified by transfusion status at baseline. Refer to Supplementary Table S4. Bars represent costs, which were aggregated across patients with known TI/TR or TD status at week 24.

‡Differences in cost burden are calculated as cost burden for momelotinib minus cost burden for danazol. Differences <0 indicate cost savings for momelotinib relative to danazol; differences >0 indicate cost savings for danazol relative to momelotinib.

§The Medicare fee-for-service database categorized patients with MF into three groups (TI, TR and TD). Refer to Supplementary Table S1. Each bar includes costs for patients with a status of TD or TI/TR at week 24; costs are stratified by transfusion status at baseline. The sample size of patients in the MOMENTUM trial who were aged ≥65 years was 155. The numbers of individuals in this subpopulation in the momelotinib and danazol arms were 101 and 54, respectively. Refer to Supplementary Table S4.

MF: Myelofibrosis; TD: Transfusion dependent; TI: Transfusion independent; TR: Transfusion requiring; USD: US dollar.

Among patients aged ≥65 years with baseline TD status, the projected average annual cost of care was similar in patients treated with momelotinib vs. danazol ($125,625 vs. $124,618), with a $1,007 savings with danazol (Figure 1B). In patients with baseline TI/TR status, the projected average annual cost of care showed savings of $6,482 per patient-year ($100,296 vs. $106,778) with momelotinib compared with danazol (Figure 1B).

3.3. Projected differences in outpatient transfusion cost

Reductions in transfusions were associated with savings in projected outpatient transfusion cost with momelotinib relative to danazol. Among patients who were TD at baseline, there was a projected outpatient transfusion cost savings of $42,021 per patient for momelotinib vs. danazol (Figure 2A). Among patients who were TI/TR at baseline, there was a projected outpatient transfusion cost savings of $8,370 per patient for momelotinib vs. danazol (Figure 2A).

Figure 2.

Figure 2.

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Projected annual outpatient visit cost per patient based on transfusion status associated with momelotinib vs. danazol. Projected annual outpatient visit cost in $USD/per person based on transfusion status associated with momelotinib vs. danazol based on the IBM MarketScan Commercial Database (A) and Medicare database (B).

†Costs of care from the IBM MarketScan Commercial Database and the Medicare fee-for-service database determined the average cost per person per visit for an outpatient transfusion among those treated for MF. Refer to Supplementary Table S2. Costs are stratified by transfusion status at baseline. Refer to Supplementary Table S4. Bars represent costs, which were aggregated across patients with known TI/TR or TD status at week 24.

‡The mean number of RBC transfusion visits was calculated using individual patient-level data from the MOMENTUM trial. RBC transfusions utilized included those received by trial patients and recorded in the GSK raw transfusion data within the 24-week trial period (inclusive). Under the assumption that an RBC transfusion represented a single transfusion visit, RBC transfusion visits were stratified by treatment arm and baseline transfusion status such that the mean RBC visits could be calculated for each subgroup. Dimensional analysis was subsequently used to adjust the time parameter from per 24 weeks to per year. Refer to Supplementary Table S5.

§Differences in cost burden are calculated as cost burden for momelotinib minus cost burden for danazol. Differences <0 indicate cost savings for momelotinib relative to danazol; differences >0 indicate cost savings for danazol relative to momelotinib.

MF: Myelofibrosis; RBC: Red blood cell; TD: Transfusion dependent; TI: Transfusion independent; TR: Transfusion requiring; USD: US dollar.

Similarly, among patients aged ≥65 years who were TD at baseline, projected outpatient transfusion cost savings were $45,060 per patient with momelotinib vs. danazol (Figure 2B). Among patients aged ≥65 years who were TI/TR at baseline, projected outpatient transfusion cost savings were $1,107 per patient for momelotinib vs. danazol (Figure 2B).

3.4. Projected differences in transfusion-related patient time burden

Reduced rates of transfusions associated with momelotinib also corresponded to reduced transfusion-related time burden for patients. Among patients who were TD at baseline, the projected average annual savings in transfusion visit time associated with momelotinib vs. danazol totaled 173 h per patient (238 vs. 411 h) (Figure 3A). Projected savings with momelotinib vs. danazol in patients with baseline TD status included 68 h for preparation and waiting time, 82 h for transfusion procedure and recovery and 24 h for travel time (Supplementary Table S7). Among patients who were TI/TR at baseline, the projected average annual savings in transfusion visit time associated with momelotinib vs. danazol totaled 34 h per patient (78 vs. 113 h) (Figure 3A).

Figure 3.

Figure 3.

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Projected transfusion-related time burden for patients based on transfusion status associated with momelotinib vs. danazol. Projected transfusion-related time burden for patients in h/person-year associated with momelotinib vs. danazol based on the IBM MarketScan Commercial Database (A) and Medicare database (B).

†Costs of care were extracted from the 2022 Knoth et al. study. This source provided estimates for mean time spent for one RBCT procedure stratified by time spent during procedure preparations, waiting room, waiting for blood, RBCT procedure and recovery and round-trip travel time. Refer to Supplementary Table S3. Time burdens are stratified by transfusion status at baseline. Refer to Supplementary Table S4. Bars represent time burdens, which were aggregated across patients with known TI/TR or TD status at week 24.

‡Differences in time burden are calculated as time burden for momelotinib minus time burden for danazol. Differences <0 indicate time savings for momelotinib relative to danazol; differences >0 indicate time savings for danazol relative to momelotinib.

§Preparation and waiting included time in preparation, waiting room and waiting for blood. Refer to Supplementary Table S7.

The sample size of patients in the MOMENTUM trial who were aged ≥65 years was 155. The numbers of individuals in this subpopulation in the momelotinib and danazol arms were 101 and 54, respectively. Refer to Supplementary Table S4.

RBCT: Red blood cell transfusion; TD: Transfusion dependent; TI: Transfusion independent; TR: Transfusion requiring; USD: US dollar.

Among patients aged ≥65 years who were TD at baseline, projected average annual savings in transfusion visit time associated with momelotinib vs. danazol totaled 186 h per patient (241 vs. 427 h) (Figure 3B). Projected savings with momelotinib vs. danazol in patients with baseline TD status included 73 h for preparation and waiting time, 88 h for transfusion procedure and recovery and 25 h for travel time (Supplementary Table S7). Among patients who were TI/TR at baseline, the projected average annual savings in transfusion visit time associated with momelotinib vs. danazol totaled 5 h per patient (91 vs. 95 h) (Figure 3B).

4. Discussion

This analysis showed that reduction in transfusions associated with momelotinib are projected to result in savings in medical and transfusion-related costs and patient time burden relative to danazol in TD and TI/TR patients with MF in the US.

Projected cost and time burden offsets estimated for momelotinib were larger for TD patients owing to the larger reductions in transfusions relative to danazol in this population compared with the TI/TR patients. Projected savings in total annual medical costs with momelotinib compared with danazol were shown using both terminal TI rates and rolling TI rates. A potential for cost savings with momelotinib in TD patients is particularly relevant because TD patients experience a higher burden than TI/TR patients. For example, a retrospective analysis of the Medicare fee-for-service database estimated total costs (medical and pharmacy) were approximately 1.8-times greater for patients who were TD vs. TI and for patients with severe vs. mild anemia, demonstrating that achieving or maintaining transfusion independence and maintaining Hb levels in patients diagnosed with MF can minimize healthcare costs and reduce the financial burden for patients and healthcare systems [17]. Similarly, a retrospective analysis using the IBM MarketScan Database estimated that total medical costs were approximately nine-times higher for patients who were TD vs. TI [16]. While we observed some differences in the magnitude of projected cost and time burdens when restricting to patients aged ≥65 years, our findings of potential offsets with momelotinib with respect to transfusion-related costs and time burden were consistent with the broader adult population. Insights on the cost of care for older patients are particularly important because MF occurs most frequently in this population (median age at diagnosis for primary MF, 65–70 years), with advanced age correlated with a worse prognosis in MF [7,30]. Notably, patients who require transfusions in the first year of diagnosis were older than patients who did not require transfusions, with median ages of 68 (range, 47–85) vs. 57 (range, 28–87) years, respectively [31]. The differences in the magnitude of projected cost and time offset estimates for the broader population and the patients aged ≥65 years may be partially attributed to differences in characteristics of patients in the Medicare fee-for-service database vs. the IBM MarketScan Database. More generally, differences in costs paid by Medicare vs. costs paid by private insurers captured in the MarketScan Database likely also contribute to the observed differences, particularly when considering overall medical costs. Importantly, the projected decrease in transfusion time burden with momelotinib observed in both the overall population and patients aged ≥65 years may contribute to improving patients' quality of life. In patients with MF, transfusion dependence was shown to have detrimental effects on multiple aspects of quality of life [32]. In a post hoc analysis of the SIMPLIFY-1 and SIMPLIFY-2 studies, greater improvement across quality of life domains were reported in patients who were TD at baseline but became TI compared with those who remained TD [32]. In addition, reducing transfusion burden, and thereby travel time, may be especially beneficial for patients in limited-resource settings including rural areas with fewer transfusion facilities [33].

Limitations of this study include the use of extrapolated data, which provides projections but may not reflect the actual cost and time savings with momelotinib compared with danazol. An additional limitation may be the differences in the definitions of transfusion status between data sources. However, for this study, transfusion criteria were expected to be functionally similar because all the studies defined TI status as 0 RBC transfusions and TD status as approximately 0.5 transfusions per week. Furthermore, an estimate on the costs of transfusion visits was not available in the Medicare study and was approximated by the estimate from the IBM MarketScan study. There were also differences in the patient populations and time frame in the MOMENTUM trial compared with the IBM MarketScan Database, Medicare database and the 2022 Knoth et al. study. While MOMENTUM included JAK inhibitor–experienced, symptomatic patients with anemia, both the IBM MarketScan and Medicare databases included both JAK inhibitor–experienced and –naive patients [16,17,28]. Time burden for transfusion was estimated using the 2022 Knoth et al. study in patients with transfusion-dependent β-thalassemia, as estimates for patients with MF were not available [29]. Compared with MOMENTUM, this study also had differences in patient characteristics including age and race [29]. However, patients with β-thalassemia, similar to those with MF, have reduced RBC production, which often necessitates RBC transfusions [34]. Time frames of follow-up also varied across the underlying data sources (i.e., MOMENTUM assessed transfusion status at week 24, the IBM MarketScan analysis included a 2–calendar year study period with continuous enrollment for each patient and the 2022 Knoth et al. study reported transfusion time burden over a 6-month period); therefore, transfusion rates, time burden and costs were annualized in our analysis [16,28,29]. Population sizes also differed and for patients aged ≥65 years were especially small in the cohort of TD patients treated with danazol (n = 12). Finally, early study discontinuation in this population with advanced, symptomatic MF and anemia resulted in unknown or missing transfusion status at week 24. However, sensitivity analyses were performed to account for these missing data and showed broadly similar results. Based on these limitations, interpretation of these results should focus on the overall trend demonstrating transfusion-related cost and patient time savings with momelotinib compared with danazol rather than the numeric cost values, which may not be generalizable for all circumstances.

Although transfusion dependency and anemia are critical negative prognostic factors in MF and lead to greater HCRU, cost and time burden, treatment options for TD patients appear to have minimal impact on the underlying cause of MF-associated anemia [12,16,18,35,36]. While transfusions rapidly increase Hb levels, they do not provide long-term clinical benefits alone and are associated with lower survival, reduced quality of life and complications including iron overload [8,37–39]. Transfusions also increase the burden on healthcare systems because they require infrastructure, logistics, human resources and financial capital to ensure blood availability, processing and delivery [40].

Moreover, transfusion dependence and subsequently greater HCRU and costs may result from myelosuppressive JAK inhibitors such as ruxolitinib and fedratinib, which have been shown to exacerbate anemia [41,42]. In addition, disease-associated or treatment-exacerbated anemia may lead to JAK inhibitor dose adjustments, interruptions or discontinuation, which may limit treatment efficacy and are associated with poor survival [14,42–46]. A retrospective real-world study of 1191 patients with MF in the US enrolled in the Medicare Advantage health plan revealed that significant HCRU and costs remained in patients treated with ruxolitinib, and major drivers of costs included inpatient hospitalizations and pharmacy costs [47]. While this study did not analyze patients with TD vs. TI status or identify what proportion of costs were due to transfusions, mean all-cause and MF-related total medical costs were increased from 6 months before MF diagnosis to 6 months after MF diagnosis (all cause, $24,216 to $48,966 and MF related, $16,502 to $39,383) [47]. Beyond JAK inhibitors, common supportive treatments for MF-related anemia, including danazol, immunomodulatory drugs and erythropoiesis-stimulating agents, have shown inadequate efficacy, durability and tolerability [8,19,21,22,35,48,49].

In contrast to these myelosuppressive JAK inhibitors and supportive treatments, momelotinib decreases aberrant inflammation and facilitates normalized Hb levels, addressing a high unmet need in patients with MF-associated anemia, effects driven at least in part by its inhibition of ACVR1 [14]. In the Phase III MOMENTUM trial, momelotinib not only demonstrated clinically significant improvement in MF-associated symptoms, anemia measures and spleen response, but also had higher transfusion independence at week 24 compared with danazol (30% vs. 20%; p = .0116, noninferior) [28]. Transfusion independence rate from baseline to week 24 increased by 17% in the momelotinib group vs. 5% in the danazol group [28]. This analysis using both the IBM MarketScan Commercial Database and the Medicare fee-for-service database demonstrates that reductions in transfusions due to treatment with momelotinib may translate into meaningful cost offsets and time savings for patients and the healthcare system.

5. Conclusion

While this study focuses on comparing the projected cost and time burden for patients treated with momelotinib and danazol in the MOMENTUM trial, additional analyses will assess the economic and humanistic costs associated with momelotinib relative to ruxolitinib in JAK inhibitor–naive patients with MF as well as relative to best available therapies in JAK inhibitor–experienced patients using corresponding analyses from the SIMPLIFY-1 and SIMPLIFY-2 trials. While acquisition costs for momelotinib were not yet available at the time of the present analysis, budget impact and cost effectiveness models that incorporate this information complement the results described here and may help determine whether reduced transfusion-related costs and supportive care costs associated with anemia and other comorbidities can offset drug costs [50,51]. As the present analysis was limited to the 24-week randomized period of MOMENTUM, future real-world analyses may be required to evaluate long-term effects beyond this time point. Analyses of real-world data may also be considered to assess patient costs in specific treatment settings. Given that initial regulatory approval of momelotinib was in the US, the present analysis is focused on projected cost and time savings from a US perspective; additional analyses to determine impacts in other markets, which are likely to vary, may be warranted based on further regulatory decisions. Finally, future work estimating the time burden and resource utilization associated with MF-related transfusions from the provider and patient's quality of life perspectives will be important to further describe the potential impact of momelotinib.

Supplementary Material

Supplementary Materials
IFON_A_2368450_SM0001.zip (163.2KB, zip)

Acknowledgments

This study was funded by GSK. Writing support was provided by J Chu (Nucleus Global, an Inizio Company), in accordance with the International Committee of Medical Journal Editors and Good Publication Practice guidelines, and was supported by GSK. We also thank the patients and families who participated in the trial and all study investigators and site staff.

Funding Statement

Funding was provided by GSK.

Supplemental material

Supplemental data for this article can be accessed at https://doi.org/10.1080/14796694.2024.2368450

Author contributions

All authors were involved in the content, writing and revising of this research article.

Financial disclosure

Funding was provided by GSK. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Competing interests disclosure

L Masarova reports advisory board participation with Cogent, GSK, MorphoSys, and PharmaEssentia. S Verstovsek received consulting fees from GSK. T Liu is a current employee of GSK. S Rao is a current employee of GSK, has stock/stock options and received support for attending meetings and/or travel from GSK. G Sajeev is an employee of Analysis Group, Inc., which received consulting fees from GSK for this research. M Fillbrunn is an employee of Analysis Group, Inc., which received consulting fees from GSK for this research. R Simpson is an employee of Analysis Group, Inc., which received consulting fees from GSK for this research. W Li is an employee of Analysis Group, Inc., which received consulting fees from GSK for this research. J Yang is a former employee of GSK. Y Le Lorier was a former GSK employee. B Gorsh was a former GSK employee and has stock/stock options and received support for attending meetings and/or travel from GSK and Daiichi-Sankyo. J Signorovitch is an employee of Analysis Group, Inc., which received consulting fees from GSK for this research. The authors have no other competing interests or relevant affiliations with any organization or entity with the subject matter or materials discussed in the manuscript apart from those disclosed.

Writing disclosure

Editorial support was provided by Nucleus Global and funded by GSK plc (Philadelphia, PA). Neither GSK plc nor Nucleus Global influenced the content of this manuscript.

Ethical conduct of research

The MOMENTUM study was conducted in accordance with the Declaration of Helsinki and the International Council for Harmonisation guidelines on Good Clinical Practice. The institutional review board or independent ethics committee at each study site approved the protocol. A full list of the ethics committees can be found in the associated Supplementary Material. All participants provided written informed consent.

Data availability statement

The authors certify that this manuscript reports the secondary analysis of clinical trial data that have been shared with them, and that the use of these shared data is in accordance with the terms (if any) agreed upon their receipt. The source of these data is GSK plc (MOMENTUM; ClinicalTrials.gov: NCT04173494).

Previous presentation

Presented in abstract form at: European Hematology Association 2023 Congress, June 8–11, 2023, Frankfurt, Germany [52].

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Associated Data

This section collects any data citations, data availability statements, or supplementary materials included in this article.

Supplementary Materials

Supplementary Materials
IFON_A_2368450_SM0001.zip (163.2KB, zip)

Data Availability Statement

The authors certify that this manuscript reports the secondary analysis of clinical trial data that have been shared with them, and that the use of these shared data is in accordance with the terms (if any) agreed upon their receipt. The source of these data is GSK plc (MOMENTUM; ClinicalTrials.gov: NCT04173494).

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