Economic burden of disease progression among multiple myeloma patients who have received transplant and at least one line of therapy in the US

Rafael Fonseca,Sumeet Panjabi,Emre Yucel,Thomas Delea,Jacqueline Buchanan,May Hagiwara

doi:10.1038/s41408-021-00431-5

Rafael Fonseca, Sumeet Panjabi + Show 4 more

Open Access

https://doi.org/10.1038/s41408-021-00431-5

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Abstract

Effects of disease progression on healthcare resource utilization (HRU) and costs among multiple myeloma (MM) patients with ≥1 line of therapy (LOT) who received their first stem cell transplant (SCT) within 1 year of initial MM diagnosis were estimated using a large US claims database. Disease progression was defined as advancement to the next LOT, bone metastasis, hypercalcemia, soft tissue plasmacytoma, skeletal related events, acute kidney disease, or death within 12 months of LOT initiation. Annual HRU and costs in the first three LOTs (L1–L3) were compared for patients with versus without disease progression using inverse probability of treatment weighting to adjust for differences between groups in baseline characteristics. In all LOTs, mean annual hospitalizations and healthcare costs were greater for patients with versus without progression. Total incremental annual costs among patients with versus without progression in L1–L3 were $18,359, $87,055, and $71,917, respectively, among LOTs initiated between 2006 and 2018. In LOTs initiated between 2013 and 2018, the figures were $46,024, $100,329, and $101,942 in L1–L3, respectively. The economic burden of disease progression is substantial in this population of MM patients who underwent SCT and received systemic anti-myeloma therapy.

Highlights

Introduction MultipleMyeloma (MM) is a rare hematologic malignancy of the plasma cells that is associated with a variety of complications, including—but not limited to—anemia, neutropenia, thrombocytopenia, bone loss and fractures, and kidney disease
The objective of this study was to investigate if the results found among transplant ineligible MM patients in Hagiwara[13] can be found among MM patients with receipt of transplant using the same approach as much as possible except for the algorithm for identifying line of therapy (LOT): i.e., to compare and quantify healthcare resource utilization (HRU) and healthcare costs in the US between MM patients with versus without progression who have received at least one LOT including a stem cell transplant using inverse probability of treatment weights (IPTW) to adjust for differences between the two groups[14]
Vorinostat is not indicated for MM, it was included in our study since its use among MM patients has been investigated in clinical studies[16]

Summary

Introduction

Myeloma (MM) is a rare hematologic malignancy of the plasma cells that is associated with a variety of complications, including—but not limited to—anemia, neutropenia, thrombocytopenia, bone loss and fractures, and kidney disease. In the US, ~32,270 new cases of MM are diagnosed, and ~12,830 persons die from the disease each year. Five-year survival for patients diagnosed with MM is ~54%1. Current treatment guidelines by the National Comprehensive Cancer Network (NCCN) and the International Myeloma Working Group recommend initiating therapy when patients become symptomatic or are identified. NCCN Guidelines recommend numerous treatment options including PIs, IMiDs, and mAbs[2]. Despite the multitude of options, the likelihood and duration of response decrease with each line of Fonseca et al Blood Cancer Journal (2021)11:35 therapy (LOT) and the disease is fatal for many patients[2]

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