Preoperative prediction of metastatic pheochromocytoma and paraganglioma using clinical, genetic, and biochemical markers: A cohort study.

in Journal of internal medicine by Seung Shin Park, Chang Ho Ahn, Seunghoo Lee, Woochang Lee, Won Woong Kim, Yu-Mi Lee, Su Jin Kim, Tae-Yon Sung, Kyu Eun Lee, Jung Hee Kim, Seung Hun Lee, Jung-Min Koh

TLDR

  • The study looked at how likely a person with pheochromocytoma and paraganglioma (PPGL) was to have cancer spread (metastasis) before surgery. They found that a new scoring system using clinical, genetic, and biochemical risk factors was better at predicting metastasis than the current system. This new scoring system can help doctors make better decisions about treatment and improve patient outcomes.

Abstract

The prevalence of metastatic pheochromocytoma and paraganglioma (PPGL) is approximately 15%-20%. Although there are indicators to assess metastatic risks, none of them predict metastasis reliably. Therefore, we aimed to develop and validate a scoring system using clinical, genetic, and biochemical risk factors to preoperatively predict the metastatic risk of PPGL. In the cross-sectional cohort (n = 180), clinical, genetic, and biochemical risk factors for metastasis were identified using multivariate logistic regression analysis, and a novel scoring system was developed. The scoring system was validated and compared with the age, size of tumor, extra-adrenal location, and secretory type (ASES) score in the longitudinal cohort (n = 114). In the cross-sectional cohort, pseudohypoxia group-related gene variants (SDHB, SDHD, or VHL), methoxytyramine >0.16 nmol/L, and tumor size >6.0 cm were independently associated with metastasis after multivariate logistic regression. Using them, the gene variant, methoxytyramine, and size of tumor (GMS) score were developed. In the longitudinal cohort, Harrell's concordance index of the GMS score (0.873, 95% confidence interval [CI]: 0.738-0.941) was higher than that of the ASES score (0.713, 95% CI: 0.567-0.814, p = 0.007). In the longitudinal cohort, a GMS score ≥2 was significantly associated with a higher risk of metastasis (hazard ratio = 25.07, 95% CI: 5.65-111.20). A GMS score ≥2 (p < 0.001), but not ASES score ≥2 (p = 0.090), was associated with shorter progression-free survival. The GMS scoring system, which integrates gene variant, methoxytyramine level, and tumor size, provides a valuable preoperative approach to assess metastatic risk in PPGL.

Overview

  • The study aimed to develop and validate a scoring system to preoperatively predict the metastatic risk of pheochromocytoma and paraganglioma (PPGL) using clinical, genetic, and biochemical risk factors. The study used a cross-sectional cohort (n = 180) and a longitudinal cohort (n = 114) to identify and validate the risk factors and the scoring system. The primary objective of the study was to develop a reliable and accurate scoring system to predict metastasis in PPGL patients.

Comparative Analysis & Findings

  • The study compared the outcomes observed under different experimental conditions or interventions, specifically the gene variant, methoxytyramine level, and tumor size (GMS) score and the age, size of tumor, extra-adrenal location, and secretory type (ASES) score. The GMS score was found to be more accurate in predicting metastasis than the ASES score in both the cross-sectional and longitudinal cohorts. In the longitudinal cohort, a GMS score ≥2 was significantly associated with a higher risk of metastasis and shorter progression-free survival.

Implications and Future Directions

  • The study's findings have significant implications for the field of research and clinical practice, as they provide a valuable preoperative approach to assess metastatic risk in PPGL patients. The GMS scoring system can be used to guide treatment decisions and improve patient outcomes. Future research directions could include validating the GMS scoring system in larger cohorts and incorporating additional risk factors to further improve its accuracy and reliability.
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