Because of the rising health care costs in the United States, there has been a focus on value-based care and improving the cost-effectiveness of surgical procedures. Patient-reported outcome measures (PROMs) can not only give physicians and health care providers immediate feedback on the well-being of the patients but also be used to assess health and determine outcomes for surgical research purposes. Recently, PROMs have become a prominent tool to assess the cost-effectiveness of spine surgery by calculating the improvement in quality-adjusted life years (QALY). The cost of a procedure per QALY gained is an essential metric to determine cost-effectiveness in universal health care systems. Common patient-reported outcome questionnaires to calculate QALY include the EuroQol-5 dimensions, the SF-36, and the SF-12. On the basis of the health-related quality of life outcomes, the cost-effectiveness of various spine surgeries can be determined, such as cervical fusions, lumbar fusions, microdiscectomies. As the United States attempts to reduce costs and emphasize value-based care, PROMs may serve a critical role in spine surgery moving forward. In addition, PROM-driven QALYs may be used to analyze novel spine surgical techniques for value-based improvements.
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Background: Perivascular epithelioid cell neoplasms (PEComas) are a diverse family of mesenchymal tumors with myomelanocytic differentiation that disproportionately affect women and can be associated with tuberous sclerosis (TS). Although mTOR inhibition is widely used as first-line treatment, it is unclear what genomic alterations exist in these tumors and how they influence the response to therapy. Methods: This was a multicenter study conducted at five sites within the US. The data were collected from 1 January 2004 to 31 January 2021. We conducted a retrospective analysis to identify PEComa patients with next-generation sequencing (NGS) data and compared outcomes based on mutations. Results: No significant differences in survival were identified between TSC-1 and TSC-2 mutated PEComa or TSC-1/-2 versus other mutations. No significant difference was seen in progression-free survival (PFS) after first-line therapy between mTOR inhibition versus other systemic therapies. Conclusions: We were unable to detect differences in survival based on genomic alterations or PFS between mTOR inhibition versus other systemic therapies. Future studies should seek to identify other drivers of TSC-1/-2 silencing that could predict response to mTOR inhibition.
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