Integrating Clinical Evidence and Cost Considerations in Myelofibrosis Care

Introduction

Myelofibrosis (MF) is a type of myeloproliferative neoplasm (MPN) that involves overproduction of platelet-producing cells (megakaryocytes) and other stem cells, leading to an accumulation of scar tissue in the bone marrow. This scar tissue, called fibrosis, impedes the bone marrow from producing other blood cells normally.¹ MF can be classified as either primary or secondary. Primary MF (PMF) develops independently, whereas secondary MF develops from the prior MPNs of essential thrombocythemia (ET) and polycythemia vera (PV). Between 10% to 20% of all MF cases are classified as secondary.¹

Epidemiology and Economic Impact

It is difficult to fully quantify the incidence of MF due to the overlapping nature of secondary MF with other MPN diagnoses. According to a 2022 analysis of epidemiologic data from 2002-2016, the incidence of primary MF is approximately 0.44 new cases per 100,000 people per year (95% CI, 0.43-0.45 new cases/100,000 people/year), with higher incidences observed in patients 65 years or older (1.80 new cases; 95% CI, 1.73-1.87 new cases/100,000 people/year) compared to younger patients (0.16 new cases/100,000 people/year; 95% CI, 0.15-0.17 new cases/100,000 people/year).² In total, there are approximately 13,000 individuals in the US currently with living with MF.³ The 5-year relative survival rate of patients with primary MF is estimated to be 35% to 46%.⁴

MF can present with a wide variety of clinical manifestations, including constitutional symptoms such as fatigue, fever or night sweats. Severe anemia, an enlarged spleen, bone pain and bleeding are other symptoms associated with MF.⁵ The formation of blood outside of the bone marrow within the liver and spleen is caused by fibrosis; this is the main cause of the observed enlargement of the spleen.⁵ Other possible complications include portal hypertension, pulmonary hypertension, cord compression, diffuse extremity pain and, in 20% of patients, progression to acute myeloid leukemia (AML).⁵

In addition to their clinical symptoms, patients with MF experience increased healthcare resource use and financial burden compared with patients without MF. A retrospective analysis of claims data showed that total annual direct health costs were higher for patients with MF compared with matched comparators ($77,993 vs. $10,537, respectively; p-value < 0.001).⁶ A diagnosis of MF was also associated with a significantly higher incidence of use of short-term disability (11% vs. 2.9%; p-value < 0.001) and higher total indirect costs associated with both short-term disability ($697 vs. $257; p-value < 0.05) and long-term disability ($553 vs. $72; p-value < 0.05) when compared to a matched comparator group.⁶ The results of a systematic literature review of 23 studies, including eight US-based studies, revealed that total medical healthcare costs ranged from $21,000 to $66,000 per patient for the US-based studies.⁷

Anemia, its severity and the related transfusion support needed for treatment are major factors in the economic burden associated with MF. A large retrospective cohort study using Medicaid fee-for-service data assessed the healthcare resource utilization and cost burden of MF based on patients’ transfusion status and anemia severity; transfusion dependence due to severe anemia was associated with healthcare costs that were 1.8 times higher, on average, than needed for patients who were transfusion independent ($14,655 vs. $8,191).⁸

Current Therapeutic Landscape and Management Goals

Risk Stratification in MF

Diagnosis of PMF is based on the 2022 World Health Organization and International Consensus Classification criteria; all three major criteria must be met, and one of the minor criteria must be met in two consecutive observations.⁹ Once a diagnosis of MF is confirmed, risk stratification is essential to guide treatment decisions and is based on validated prognostic scoring systems that incorporate clinical, hematologic and genetic variables. The Dynamic International Prognostic Scoring System (DIPSS) developed by the International Working Group on Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) assigns risk based on five adverse features: age above 65 years, constitutional symptoms, hemoglobin level less than 10 grams per deciliter (g/dL), leukocyte counts above 25 × 10⁹ per liter (/L), and circulating blasts of at least 1%. Risk categories are defined as follows: low risk (score of 0), intermediate-1 (score of 1 or 2), intermediate-2 (score of 3 or 4) and high risk (score of 5 or 6).¹⁰ The median survival associated with these risk categories was not reached for the low-risk group but was 14.2 years in the intermediate-1 group, 4 years in the intermediate-2 group and 1.5 years in the high-risk group.¹⁰

More recently, mutations were incorporated into two new prognostic models: MIPSS70 and MIPSS70-Plus. MIPSS70 was developed from the results of a multivariate analysis involving 805 patients 70 years or younger with MPF; independent predictors of decreased overall survival (OS) were associated with a hemoglobin level less than 10 g/dL, leukocyte level greater than 25 × 10⁹/L, circulating blasts of at least 2%, a bone marrow fibrosis grade above MF-2, constitutional symptoms, the absence of CALR type-1 mutation and the presence of two or more high molecular risk (HMR) mutations (ASXL1, EZH2, SRSF2 and IDH1/2).¹¹ MIPSS70 stratified patients into low-, intermediate- and high-risk categories with median OS of 27.7 years, 5.6 years and 2.1 years, respectively.¹¹ MIPSS70-Plus and MIPSS70-Plus version 2.0 were developed to include cytogenetic risk-categories (very-high risk, unfavorable and favorable), an additional HMR mutation (U2AF1 Q157) and sex- and severity-adjusted hemoglobin thresholds.¹² These models stratified patients into five risk groups from very-low risk to very-high risk and found 10-year OS rates ranging from 86% in the group having very-low risk to less than 3% in the group having very-high risk.¹²

The NCCN MPN guidelines recommend the use of MIPSS70 or MIPSS70-Plus version 2.0 for risk stratification of PMF, but they allow for the use of DIPSS if recent karyotyping or molecular testing is not available.⁹ For secondary MF derived from ET or PV, the NCCN recommends the Myelofibrosis Secondary to PV and ET - Prognostic Model (MYSEC-PM).⁹ The MYSEC-PM stratifies patients with post-ET and post-PV MF into four risk groups based on age, hemoglobin level, circulating blasts, CALR mutation status, platelet count and constitutional symptoms.¹³

Treatment Strategies

Selection of treatment should be based on risk stratification, the presence of symptoms and disease stage. The goals of treatment are to reduce bone marrow fibrosis, improve cytopenias and symptom burden, restore transfusion independence and/or prevent progression to AML.⁹ Assessment of symptom burden is one of the initial steps in assessing the need for treatment initiation or change. The Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score is a patient survey recommended to assess symptom burden at baseline and throughout the disease course.⁹

According to the most recent guidelines, patients at lower risk (defined by the NCCN as a MIPSS70-Plus score ≤ 3) who are asymptomatic can be considered for a clinical trial or can be monitored for signs of disease progression every 3 to 6 months.⁹ Treatment options for symptomatic patients at lower risk include hydroxyurea (if cytoreduction would be symptomatically beneficial), peginterferon alfa-2a and JAK inhibitors.⁹ Evaluation for allogeneic hematopoietic stem cell transplant (HSCT) is recommended for all patients with higher-risk MF; transplant is recommended for all those who meet eligibility criteria.⁹ For patients not eligible for a transplant who are at higher risk (MIPSS70-Plus > 3), the treatment options are JAK inhibitors or enrollment in a clinical trial.⁹

Factors Influencing Treatment Selection

The only currently available treatment modality shown to prolong survival and potentially cure MF is allogeneic HSCT. However, transplanted-related mortality and severe morbidity can be expected in at least 30% of patients, necessitating a patient-specific assessment of the risks of undergoing transplant versus the risks of not undergoing transplant, informed by risk stratification based on current disease status.⁵

The choice of JAK inhibitor is individualized to patient-specific factors. The NCCN recommends the JAK1/2 inhibitor ruxolitinib (Jakafi; Incyte) and the JAK2 inhibitor fedratinib (Inrebic; Bristol Myers Squibb) for patients with platelet counts above 50 × 10⁹/L; it also recommends pacritinib (Vonjo; Sobi) as the standard of choice for patients who have platelet counts below 50 × 10⁹/L due to its specific approval in that patient population.⁹ The most recently approved JAK inhibitor, momelotinib (Ojjaara; GSK),¹⁴ shows activity against three of the most common symptoms experienced by patients with MF: anemia, splenomegaly and constitutional symptoms. A phase 3 study including 195 patients with MF and anemia (hemoglobin < 10 g/dL) who were exposed to JAK inhibition and who were at high/intermediate risk were assigned to receive daily doses of either momelotinib, 200 milligrams (mg), or danazol, 600 mg; momelotinib provided a significant benefit in transfusion-independence rate, spleen volume reduction and corresponding reductions in symptom score rates.¹⁵ Information regarding the FDA-approved JAK inhibitors is summarized in the Table.^16-25

Momelotinib has an apparent benefit among the JAK inhibitors in improving anemia, but its overall cost-benefit to the healthcare system needs to also be considered. In a cost-comparison model evaluating patients with MF and anemia who were JAK inhibitor-naïve, ruxolitinib was associated with lower total costs compared to momelotinib over 6 months, 1 year and 2 years. Although momelotinib demonstrated higher transfusion independence rates that led to reduced transfusion costs, these savings were outweighed by higher pharmacy costs. Specifically, total per-patient costs with ruxolitinib were $134,824 at 6 months, $265,355 at 1 year and $502,145 at 2 years compared to $181,212, $349,594 and $646,684, respectively, for momelotinib. This resulted in cost savings with ruxolitinib of $46,388, $84,239 and $144,539 at each time point. Despite higher survival linked to use of ruxolitinib (leading to longer drug exposure and increased pharmacy costs), the lower wholesale acquisition cost of ruxolitinib led to consistent savings.²⁶ Of note, the model did not factor in clinical benefits (e.g., symptom response rate, spleen response rate and secondary benefits of lower transfusion needs) due to the lack of accurate data in total cost of care related to these outcomes.²⁶

Quality of Life and Economic Impact on Patients

Quality of life (QOL) is a critical component of care for patients with MF, as symptom burden can impact daily functioning and overall well-being. In a large, international, cross-sectional study of 1,416 patients with MPNs, the relationship between symptom burden and QOL was assessed using the a validated symptom assessment tool (e.g., MPN-Symptoms Assessment Form) and a validated QOL scoring tool (e.g., the Quality of Life Questionnaire-C30).²⁷ Fatigue, inactivity and depression were the symptoms most strongly associated with reduced QOL among patients with MF surveyed, although correlations were only moderate.²⁷ Patient-level symptom severity that was based on each individual’s worst symptoms modeled QOL better than group-level symptom averages. QOL was more accurately predicted by the number of symptoms above certain severity thresholds than by the symptom prevalence alone.²⁷ These findings highlight the importance of individualized symptom assessments of QOL and suggest that symptom response criteria could be refined to focus on each patient’s most severe symptoms.

Treatment-related shifts in QOL can be impactful to patients. Post-hoc analyses from the phase 3 COMFORT-2 study (NCT00934544) showed the impact of ruxolitinib on disease-related symptoms and health-related QOL (HRQOL) in patients with MF. During the follow-up period of 48 weeks, HRQOL, physical functioning, appetite loss and role functioning symptoms improved from baseline for ruxolitinib-treated patients when compared to those treated with best available therapy.²⁸

Financial toxicity refers to the economic burden that cancer treatment places on patients and their families.²⁹ In an analysis of survey data of 369 patients with MPN (MF diagnosis, 85 patients), a notable proportion of patients reported that their disease led to reduced work hours (38%), discontinued employment (35%) and medical disability (33%).³⁰ In the MF group, the reported mean percentage household income losses in patients with reduced work hours, discontinued employment and medical disability were 16%, 18% and 28%, respectively, compared to those patients who did not report any effect on their employment.³⁰ A screening option clinicians can use to evaluate financial toxicity in patients with cancer is the Comprehensive Score for Financial Toxicity tool, an 11-question, validated, patient-reported assessment. During validation, financial toxicity correlated with employment status, race, household income, psychological distress and the number of inpatient admissions.²⁹ When assessed with HRQOL measures, financial toxicity correlated with worse HRQOL.²⁹

Strategies for Improving Value

One possible strategy for improving value in cancer treatment is the use of value-based frameworks (VBFs), which represents a clinical strategy for improving outcomes while managing costs. VBFs measure and compare the value of treatment benefits with costs and align reimbursement with quality, efficiency and patient outcomes.³¹ These models support evidence-based decision-making and help to align physicians, patients and payers around treatments that deliver value. In a survey of 28 cancer center sites, 58% of respondents indicated that VBFs were used at their sites, with a higher proportion noted in community cancer centers (78%) than academic centers (39%). The most common type of VBF used at all sites was the NCCN Evidence Blocks (85%), followed by the American Society of Clinical Oncology Value Framework (31%).³¹ The top reported motivator for using VBFs was the production of real-world data.³¹ The most common reported reason for not using VBFs (given by 47% of respondents) was a lack of accessible patient-specific outcomes.³¹

Another type of oncology care payment model is the clinical care pathway (CCP), which provides structured, evidence-informed recommendations to support prescribers in selecting high-value, appropriate therapies. By standardizing care delivery, CCPs aim to reduce variation and improve patient outcomes while also managing oncology costs.³¹ In the previously described survey, 76% of respondents indicated that CCPs were used at their practice sites, with a higher proportion used in community cancer centers (87%) than in academic centers (67%). The most commonly used CCP was the NCCN clinical practice guidelines (79%). CCP use was reported to be driven quite evenly by providers, institutions and payers at 47%, 44% and 38%, respectively. Among the 11 respondents whose sites did not use a CCP, the most common reason (cited by 64% of respondents) was a lack of consensus on pathway choices.³¹

The alternative payment model (APM) is another possible strategy for improving value in cancer treatment. APMs are playing an increasingly prominent role in oncology care as payers seek to align reimbursement with value-based principles. These models are designed to incentivize providers to deliver high-quality, cost-efficient care by moving away from traditional fee-for-service structures. Some examples of these include accountable care organizations, patient-centered medical homes and the oncology care model.³¹ Among responders, 67% indicated they had implemented an APM.The largest barrier to implementation (reported by 27% of respondents working at sites with APMs) was that innovations in therapies and services had to be adopted at the site’s own financial risk. The most impactful cost-saving measure reported with the use of APMs was reduced drug spending (noted by 33% of respondents), while the most frequently reported reason for those that had not implemented an APM was administrative complexity (reported by 53% of respondents).³¹

Emerging Therapies

Numerous trials intended to improve the treatment of MF are currently underway or recently completed.

MANIFEST-2 (NCT04603495) is a phase 3 trial evaluating the addition of pelabresib, an investigational BET inhibitor, to ruxolitinib versus ruxolitinib and placebo in patients with primary and secondary MF who are naïve to JAK inhibition.³² The primary end point of at least 35% reduction in spleen volume by week 24 was observed in a significantly larger proportion of patients in the pelabresib combination group versus the placebo group (65.9% vs. 35.2%; p-value < 0.001).³² Thrombocytopenia and anemia were the most common treatment-emergent side effects reported in the pelabresib group; these occurred in 52.8% of the pelabresib-ruxolitinib group (≥ grade 3, 13.2%) versus 37.4% in the placebo-ruxolitinib group (≥ grade 3, 6.1%) and 44.8% in the pelabresib-ruxolitinib group (≥ grade 3, 23.1%) versus 55.1% in the placebo-ruxolitinib group (≥ grade 3, 36.5%), respectively.³²

The POIESIS trial (NCT06479135) is an ongoing global, randomized, double-blind, placebo-controlled, phase 3 study evaluating the efficacy and safety of navtemadlin, a potent MDM2 inhibitor, as an add-on for patients naïve to JAK inhibitors who started ruxolitinib and had a suboptimal response.³³ This study design is meant to mimic how MF treatment is treated in routine clinical practice — that is, treating with add-on therapy when necessary. The study involves a run-in period using ruxolitinib monotherapy. Patients having a suboptimal response after the run-in period were then randomly assigned to receive navtemadlin or placebo as an add-on.³³

The IMpactMF phase 3 trial (NCT04576156) is evaluating imetelstat, a telomerase inhibitor, versus best available therapy in patients with MF (DIPSS ≥ intermediate-2) that is refractory to JAK inhibition and who show active symptoms with measurable splenomegaly.³⁴ Of note, patients included in this study had to have an adequate trial of treatment with a JAK inhibitor; the option of using a JAK inhibitor is not included in the best available therapy arm.³⁴ The primary end point is OS.

Upcoming trials are promising in the landscape of MF treatment, but a lack of consensus on the definition of disease medication and the need for more inclusive clinical trial designs remain key barriers.³⁵ Broad collaboration among stakeholders will be essential to develop personalized, effective and safe treatments that can meaningfully improve outcomes for patients with MF.³⁵

Conclusion

MF is a complex and costly disease that has a lasting impact on the HRQOL of affected patients. Close collaboration among all stakeholders in the healthcare system, pharmaceutical industry and patient community is needed to provide the most beneficial and cost-effective care to patients with MF. Strategies that alleviate economic burden on both the healthcare system and patients will be key in achieving the best possible outcomes for patients and their families.

Acknowledgement
Medical writing support was provided by Sebastian Cerdeña, PharmD.

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