Treatment Patterns, Health Care Resource Utilization, and Cost in Patients with Myelofibrosis in the United States

Abstract

Background This study analyses treatment patterns, health care resource utilization (HCRU), and costs in patients with myelofibrosis (MF) and a subgroup treated with ruxolitinib (RUX). Materials and Methods Treatment patterns, all-cause and MF-related HCRU, and costs were analyzed in adults with MF with continuous enrollment in a commercial or the Medicare Advantage health plan in the pre-index period, defined as the 12 months immediately prior to the index date (date of primary or secondary MF diagnosis), and the post-index period, defined as ≥6 months following the index date. In a subgroup analysis, outcomes were analyzed in patients treated with optimal RUX (OPT RUX, ≥30 mg) and suboptimal RUX (SUB RUX, <30 mg) in the pre-index RUX period, defined as the 3 months immediately prior to the index RUX date (first date for an RUX claim), and the post-index RUX period, defined as ≥6 months following the index RUX date. Results Of 2830 patients with an MF diagnosis, 1191 met eligibility requirements. The median age of patients was 72 years, 54% were male, and comorbidities were frequent. Sixty percent of patients received ≥1 line of therapy (LOT), of which 46% (n = 331) had ≥2 LOTs during the post-index MF period. Costs increased considerably 6-month pre-index to 6-month post-index (all-cause: cause ($24,216 to $48,966) and MF-related ($16,502 to $39,383), driven by inpatient stays and pharmacy costs. In the subgroup analysis, patients treated with RUX (n = 495) experienced significant disease burden and high costs, regardless of dose. A shorter duration of therapy and a higher rate of discontinuation were observed in patients treated with SUB RUX (n = 191) versus OPT RUX (n = 304). Conclusion These findings suggest a significant disease and economic impacts associated with MF patients that persists with RUX therapy, highlighting the need for additional therapeutic options for MF.

Physician-Reported Perspectives on Myasthenia Gravis in the United States: A Real-World Survey

“…Continued disease burden and high healthcare cost is, unfortunately, a familiar issue in the US. Real world data on treatment patterns and HCRU show high burden and high cost for a wide range of diseases [ 33 – 35 ], and while the development of new MG treatment options is therefore of benefit, they are unlikely to fully resolve the issues identified in our study.…”

Momelotinib in Myelofibrosis Patients With Thrombocytopenia: Post Hoc Analysis From Three Randomized Phase 3 Trials

“…Cytopenias are common in patients with MF, and patients with moderate or severe thrombocytopenia tend to have greater symptom burden, higher rates of anemia and transfusion dependence, and worse prognosis than MF patients with higher platelet counts 5–7 . Thrombocytopenia and anemia events are frequent in patients with MF receiving ruxolitinib and may require treatment dose reductions and discontinuation, which limit efficacy and survival 13 , 14 , 35 . Currently, ruxolitinib and fedratinib are not approved for MF patients with severe thrombocytopenia, 8 , 19 whereas pacritinib approval is limited to patients with platelets <50 × 10 9 /L in the United States 22 .…”

“…[5][6][7] Thrombocytopenia and anemia events are frequent in patients with MF receiving ruxolitinib and may require treatment dose reductions and discontinuation, which limit efficacy and survival. 13,14,35 Currently, ruxolitinib and fedratinib are not approved for MF patients with severe thrombocytopenia, 8,19 whereas pacritinib approval is limited to patients with platelets <50 × 10 9 /L in the United States. 22 Accelerated approval of pacritinib was based on spleen response in a limited number of patients, and no confirmed symptom benefits are depicted in its label.…”

“…Worsening thrombocytopenia commonly results in dose reductions to as little as 25% of the standard dose 11 . In the real world, approximately one‐third of patients initiate ruxolitinib at a lower dose than recommended 12–14 and an estimated 41%–64% of patients discontinue ruxolitinib within 3 years, 14 , 15 most often due to treatment‐related cytopenias 12 , 15 . Rapid increases in spleen size and symptoms have been observed with ruxolitinib dose reductions, 16 and ruxolitinib discontinuation is associated with poor survival, particularly for patients with thrombocytopenia 12 .…”

Cost of Treatment for Myelofibrosis Patients in Ministry of Health Hospitals in Sarawak, Malaysia

“…A study from Canada of 1,031 MF patients in the Ontario provincial health database reported treatment cost of C$25,863 per patient per year, with hospitalisation and medication accounting for 66.5% and 18.6% of treatment costs, respectively (Bankar et al, 2020). Similarly, an analysis of US data reported MF-related medical costs of US$38,383 in the first 6 months post-MF diagnosis, of which inpatient stay and pharmacy costs contributed to 45.5% and 32.2% of total costs (Copher et al, 2022). However, it should be noted that hospitalisation costs in our study was likely to be underestimated as we did not include the cost of ICU or procedures during admission.…”