Prognostic Factor Analysis and Nomogram Construction of Primary Retroperitoneal Liposarcoma: A Review of 10 Years of Treatment Experience in a Single Asian Cohort of 211 Cases

ObjectiveThis study intended to retrospectively analyze the data of patients with primary retroperitoneal liposarcoma in a single Asian large-volume sarcoma center and to establish nomograms focused on PRLPS for predicting progression-free survival (PFS) and overall survival (OS).MethodsA total of 211 patients treated surgically for primary, non-metastatic retroperitoneal liposarcoma during 2009–2021 were identified, and clinicopathologic variables were analyzed. PFS and OS nomograms were built based on variables selected by multivariable analysis. The discriminative and predictive ability of the nomogram was assessed by concordance index and calibration curve.ResultsThe median follow-up time was 25 months. A total of 117 (56%) were well-differentiated, 78 (37%) were dedifferentiated, 13 (6%) were myxoid, and 3 (1%) were pleomorphic morphology. Compared to the western population cohort reported by the Memorial Sloan-Kettering Cancer Center, the median age of patients in this cohort was younger (57 vs. 63 years), the tumor burden was lower (20 vs. 26 cm), and the proportion of patients with R0 or R1 resection was higher (97% vs. 81%). The 5-year PFS rate was 49%, and factors independently associated with PFS were symptoms at visit, preoperative needle biopsy, histologic subtypes, and postoperative hospital stay. The 5-year OS rate was 72%. American Society of Anesthesiologists Physical Status and Clavien-Dindo classification were independently associated with OS. The concordance indexes for PFS and OS nomograms were 0.702 and 0.757, respectively. The calibration plots were excellent.ConclusionsThe proposed nomogram provided a favorable reference for the treatment of primary retroperitoneal liposarcoma patients.

Download Full-text

mTOR inhibitor therapy for metastatic sarcomatoid clear cell renal cell carcinoma (ccRCC).

Journal of Clinical Oncology ◽

10.1200/jco.2013.31.6_suppl.450 ◽

2013 ◽

Vol 31 (6_suppl) ◽

pp. 450-450 ◽

Cited By ~ 1

Author(s):

Diogo Assed Bastos ◽

Martin Henner Voss ◽

Christoph Karlo ◽

Albulena Ajeti ◽

Sujata Patil ◽

...

Keyword(s):

Mtor Inhibitor ◽

Progression Free Survival ◽

Mtor Inhibitors ◽

Risk Groups ◽

Median Number ◽

Tumor Burden ◽

Inhibitor Therapy ◽

Cancer Center ◽

Cell Renal Cell Carcinoma ◽

Metastatic Sites

450 Background: Sarcomatoid variant describes a histomorphologic pattern with presence of spindle-cell features that can be seen across all subtypes of RCC. It is associated with an aggressive phenotype and poor prognosis. Outcome to mTOR inhibitor therapy was accessed in patients (pts) with ccRCC with sarcomatoid features. Methods: Patients with metastatic sarcomatoid ccRCC treated with mTOR inhibitors at our center were retrospectively identified. Demographic characteristics and treatment outcome were assessed. Overall response rate (ORR) was determined by formal response criteria (RECIST 1.1). Progression-free survival (PFS) and overall survival (OS) were calculated using the Kaplan-Meier method. Results: 29 pts were treated with temsirolimus (18) or everolimus (11). Median age was 57 years (53 to 61) and there were 21 male pts (72%). Median number of metastatic sites was 3 (1 to 7) and 18 pts (62%) had ≥ 3 metastatic sites. Distribution between Memorial Sloan-Kettering Cancer Center risk groups was 14% favorable, 72% intermediate, and 14% poor-risk disease. Median number of prior therapies was 1 (0 to 5) and 79% of pts received mTOR inhibitor as ≥ 2nd-line of therapy. Three patients (10.3%) achieved a partial response (PR), 11 (38%) stable disease (SD), and 15 (51.7%) had progressive disease (PD). Tumor shrinkage was observed in 9 pts (31%) and the mean change in tumor burden was +24% (-45% to +100%). Median PFS and OS were 3.4 months (CI: 1.4-5.3) and 8.3 months (CI: 4.8- 17), respectively. Conclusions: OS was poor in this heterogeneous group of pts. Future studies to characterize this variant at a molecular level are warranted and might identify predictive markers for clinical outcome in patients treated with mTOR inhibitors. [Table: see text]

Download Full-text

Radiation and Its Impact on Local Recurrence in Extremity and Trunk Well-Differentiated Liposarcomas

The American Surgeon ◽

10.1177/000313481908500126 ◽

2019 ◽

Vol 85 (1) ◽

pp. 52-58

Author(s):

Annabelle L. Fonseca ◽

Christina L. Roland ◽

Janice N. Cormier ◽

Keila E. Torres ◽

Kelly H. Hunt ◽

...

Keyword(s):

Soft Tissue ◽

Recurrent Disease ◽

Multivariable Analysis ◽

Retrospective Chart Review ◽

Cancer Center ◽

Free Standing ◽

Time To Recurrence ◽

Increased Risk ◽

Well Differentiated ◽

Difficult Area

Patients with well-differentiated liposarcomas (WDLPS) of the extremity and trunk are treated primarily with surgical resection, with radiation used for a number of anecdotal reasons, including large size and positive margins. In this study, we evaluate the appropriate role for radiation in these tumors. A retrospective chart review of patients with extremity and trunk soft tissue liposarcomas referred to a free-standing cancer center from January 1995 to December 2011 was performed. One hundred eighty-three patients with extremity and trunk soft tissue WDLPS were identified: 61 per cent were female, median age was 60 years (range, 19–84 years) and 2 per cent had a focal area of dedifferentiation, margin status was positive in 57 per cent. Fourteen per cent of patients received radiation. Fifty patients developed recurrent disease; 28 per cent of these received radiation. Median time to recurrence was 18 years (range, 0.7–22 years). Of the 50 patients who recurred, 14 (28%) received radiation. Radiation was associated with decreased second recurrence when administered for recurrent disease (P = 0.03). On multivariable analysis, tumor size ≤ 10 cm (P = 0.014) and anatomically difficult area of resection (P = 0.008) were predictive of increased risk of recurrence. Older age (P = 0.02), dedifferentiated liposarcomas (P < 0.001), and difficult area of resection (P = 0.02) were associated with the administration of radiotherapy. Administration of radiation therapy was not associated with decreased time to recurrence in WDLPS overall; however, it should be considered in patients with recurrent disease.

Download Full-text

Association of percentage of tumor burden removed with debulking nephrectomy and progression-free survival (PFS) in metastatic renal cell carcinoma (mRCC) patients (Pts) treated with VEGF-targeted therapy

Journal of Clinical Oncology ◽

10.1200/jco.2009.27.15_suppl.5095 ◽

2009 ◽

Vol 27 (15_suppl) ◽

pp. 5095-5095 ◽

Cited By ~ 1

Author(s):

J. Barbastefano ◽

J. A. Garcia ◽

P. Elson ◽

L. S. Wood ◽

B. S. Lane ◽

...

Keyword(s):

Targeted Therapy ◽

Systemic Therapy ◽

Primary Tumor ◽

Multivariable Analysis ◽

Cleveland Clinic ◽

Progression Free Survival ◽

Standard Of Care ◽

Tumor Burden ◽

Urologic Oncology ◽

Start Date

5095 Background: Debulking nephrectomy is a standard of care in mRCC, although data in patients subsequently treated with targeted therapy is lacking. The objective of this study was to determine if fractional percentage of tumor volume (FPTV) removed with debulking nephrectomy is associated with PFS on subsequent VEGF-targeted therapy. Methods: The Cleveland Clinic Urologic Oncology database from 2005–2008 was retrospectively reviewed to identify mRCC patients who had undergone debulking nephrectomy followed by VEGF-targeted therapy, defined as treatment with sunitinib, sorafenib, bevacizumab or sunitinib + bevacizumab. FPTV was determined by the diameter of the primary tumor divided by the total tumor burden (per RECIST criteria) by investigator re-review of imaging studies. PFS was defined from the start date of systemic therapy to disease progression per RECIST criteria. Results: Seventy-five Pts were identified; 76% male, median age 60 years (range, 34–84), 95% clear cell histology and 69% ECOG PS 0. Pts received treatment with bevacizumab (28 pts), sunitinib (23), sorafenib (20), or sunitinib + bevacizumab (4). Lung (76%) and lymph node (48%) were the predominant sites of metastasis. The median diameter of the primary tumor was 9.3 cm (range, 3.3–21 cm). The median FPTV removed was 95% (range, 80–99%). The median PFS was 12 months. In univariable analysis, the FPTV removed was associated with prolonged PFS (p < 0.001), as were low nuclear grade (p = 0.009), longer interval from diagnosis to treatment (p = 0.05), normal hemoglobin (p = 0.02), number of metastatic sites (p = 0.05), and lack of lung (p = 0.05) and brain (p = 0.05) metastasis. In multivariable analysis, the FPTV removed, as well as the interval from diagnosis to treatment (p = 0.03), were found to be independent predictors of PFS (< 0.001). Conclusions: Improved PFS on targeted systemic therapy is significantly associated with a greater percentage of tumor burden removed at debulking nephrectomy. No significant financial relationships to disclose.

Download Full-text

The efficacy of capecitabine and temozolomide for the treatment of metastatic neuroendocrine tumors: Memorial Sloan-Kettering Cancer Center experience.

Journal of Clinical Oncology ◽

10.1200/jco.2012.30.4_suppl.363 ◽

2012 ◽

Vol 30 (4_suppl) ◽

pp. 363-363 ◽

Cited By ~ 2

Author(s):

Alex Ganetsky ◽

Nelly G. Adel ◽

Kinh Gian Do ◽

Diane Lauren Reidy

Keyword(s):

Progression Free Survival ◽

Tumor Grade ◽

Tumor Burden ◽

Cancer Center ◽

Low Grade ◽

High Grade ◽

First Line ◽

First Line Therapy ◽

Tumor Expression ◽

Line Setting

363 Background: Emerging literature has suggested the benefit of capecitabine/temozolomide (C/T) therapy in metastatic pancreatic NETs (pNETs) as first line therapy. We conducted a retrospective analysis of the efficacy of (C/T) therapy in all patients with metastatic NETs treated at MSKCC. Methods: Using the electronic pharmacy database, we included all patients’ ≥ 18 years of age who received C/T combination therapy for pNETs between 1/2003-10/2010. Primary endpoint was the overall response rate (ORR). Secondary endpoints included progression-free survival (PFS) and overall survival (OS). Response rates were evaluated by a radiologist using CT scans and per RECIST 1.1. MGMT tumor expression was conducted to correlate with response. Results: Twenty patients (mean age 64, 35% female) were identified. There were 16 (80%) pNETs (1 functional, 15 nonfunctional), 2 (10%) carcinoid, 1 high grade biliary (5%) and 1 (5%) gastric neuroendocrine carcinoma. Eight tumors were low grade (1 carcinoid, 7 pNET), 8 intermediate grade (7 pNET, 1 carcinoid), and 4 high grade (2 pNET, 1 stomach, 1 biliary). Twelve (60%) received C/T in the first-line setting and 8 in the relapsed setting. Six (30%) had a partial response and 7 (35%) had stable disease. There were no complete responses ( Table 1 ). With a median follow-up of approximately 3 years, the PFS was 16.4 months. Four pNET patients had unresectable disease at presentation and 2/4 were resected and rendered free of disease after C/T therapy. There were no high grade responders. Liver tumor burden (0%, <10%, 10-50%, >50%), number of prior treatments, and tumor grade did not predict response. Grade 3-4 events potentially related to C/T included neutropenia (1/20, 5%), nausea (3/20 15%), diarrhea (1/20, 5%), and fatigue (3/20, 15%). Conclusions: Combination C/T for the treatment of pNETs is an effective regimen for well differentiated NETs irrespective of tumor burden and prior treatment. No responses were seen in our carcinoid patients. MGMT expression will be presented at the meeting. [Table: see text]

Download Full-text

Efficacy and Toxicity Analysis of Capecitabine and Temozolomide in Neuroendocrine Neoplasms

Journal of the National Comprehensive Cancer Network ◽

10.6004/jnccn.2021.7017 ◽

2021 ◽

pp. 1-8

Author(s):

Taymeyah Al-Toubah ◽

Eleonora Pelle ◽

Tiffany Valone ◽

Mintallah Haider ◽

Jonathan R. Strosberg

Keyword(s):

Opportunistic Infections ◽

Progression Free Survival ◽

Pancreatic Tumors ◽

Prolonged Treatment ◽

Cancer Center ◽

Neuroendocrine Neoplasms ◽

Significant Activity ◽

Large Patient ◽

Toxicity Analysis ◽

Well Differentiated

Background: The capecitabine/temozolomide (CAPTEM) regimen has significant activity in advanced neuroendocrine tumors (NETs). Questions exist regarding activity in pancreatic versus nonpancreatic NETs, risk of opportunistic infections, long-term myelotoxicity, and safety of prolonged treatment duration. Analysis of large patient cohorts is needed for the evaluation of rare toxicities and assessment of risk factors. Methods: We conducted a retrospective study of all patients with advanced NETs seen at Moffitt Cancer Center between January 2008 and June 2019 who received treatment with CAPTEM. Results: A total of 462 patients were eligible. The objective radiographic response rate was 46%, and the disease control rate was 81%. Median progression-free survival (PFS) was 18 months (95% CI, 14.0–21.9 months) and median overall survival was 51 months (95% CI, 42.8–59.2 months): 62 months in well-differentiated NETs versus 14 months in poorly differentiated neuroendocrine carcinomas (P<.0001). Patients with primary pancreatic tumors had the highest partial response rates and longest median PFS. Incidences of grade 4 thrombocytopenia and neutropenia were 7% and 3%, respectively, and substantially higher in women than men (P=.02 and P=.004, respectively). Only 1 case (0.2%) of suspected Pneumocystis pneumonia (PCP) was observed in a patient receiving corticosteroids. Three patients developed myelodysplastic disease, all of whom had received prior peptide receptor radiotherapy (PRRT). There were no acute treatment-related deaths; 1 patient died 2 months after a thrombocytopenic bleed. Conclusions: The CAPTEM regimen is exceptionally safe. Efficacy is particularly robust in well-differentiated pancreatic NETs. Severe myelotoxicity is rare; the risk of grade 4 cytopenias is significantly increased in women, and therefore sex-based dosing should be considered. There were no cases of myelodysplastic syndromes, except among patients who had received PRRT, a known risk factor. The risk of PCP is negligible.

Download Full-text

Salvage therapies for radiation-relapsed IDH-mutant astrocytoma and 1p/19q codeleted oligodendroglioma

Neuro-Oncology Advances ◽

10.1093/noajnl/vdab081 ◽

2021 ◽

Author(s):

Sirui Ma ◽

Soumon Rudra ◽

Jian L Campian ◽

Milan G Chheda ◽

Tanner M Johanns ◽

...

Keyword(s):

Cox Regression ◽

Salvage Surgery ◽

Multivariable Analysis ◽

Progression Free Survival ◽

Cancer Center ◽

First Line ◽

Free Survival ◽

Cox Regression Analysis ◽

Kaplan Meier ◽

Tertiary Cancer Center

Abstract Background Optimal management for recurrent IDH-mutant glioma after radiation therapy (RT) is not well-defined. This study assesses practice patterns for managing recurrent IDH-mutant astrocytoma (Astro) and 1p/19q codeleted oligodendroglioma (Oligo) after RT and surveys their clinical outcomes after different salvage approaches. Methods Ninety-four recurrent Astro or Oligo patients after RT who received salvage systemic therapy (SST) between 2001 and 2019 at a tertiary cancer center were retrospectively analyzed. SST was defined as either alkylating chemotherapy (AC) or non-alkylating therapy (non-AC). Overall survival (OS) and progression-free survival (PFS) were calculated using the Kaplan-Meier method from the start of SST. Multivariable analysis (MVA) was conducted using Cox regression analysis. Results Recurrent Oligo (n=35) had significantly higher PFS (median: 3.1 vs 0.8 years, respectively, P = 0.002) and OS (median: 6.3 vs 1.5 years, respectively, P < 0.001) than Astro (n=59). Overall, 90% of recurrences were local. Eight-three percent received AC as the first-line SST; 50% received salvage surgery before SST; approximately 50% with local failure >2 years after prior RT received reirradiation. On MVA, non-AC was associated with worse OS for both Oligo and Astro; salvage surgery was associated with improved PFS and OS for Astro; early reirradiation was associated with improved PFS for Astro. Conclusions Recurrent radiation-relapsed IDH-mutant gliomas represent a heterogeneous group with variable treatment approaches. Surgery, AC, and reirradiation remain the mainstay of salvage options for retreatment.

Download Full-text

Surgical Diagnosis and Treatment of Primary Retroperitoneal Liposarcoma

Frontiers in Surgery ◽

10.3389/fsurg.2021.672669 ◽

2021 ◽

Vol 8 ◽

Author(s):

Jie Chen ◽

Ying Hang ◽

Qi Gao ◽

Xinyu Huang

Keyword(s):

Overall Survival ◽

Prognostic Factor ◽

Recurrence Rate ◽

Univariate Analysis ◽

Surgical Margin ◽

Progression Free Survival ◽

Tumor Stage ◽

Free Survival ◽

Retroperitoneal Liposarcoma ◽

Primary Retroperitoneal

Background: Primary retroperitoneal liposarcoma (PRPLS) is the most common soft tissue sarcoma of the retroperitoneum with high recurrence rate and short overall survival (OS).Methods: A retrospective review of 51 patients with PRPLS, treated between September 1, 2009 and November 30, 2020, was conducted to evaluate clinical outcomes of PRPLS resection. Patient demographics, histopathologic subtypes, overall survival (OS), progression-free survival (PFS), disease recurrence rate, and tumor stage were reviewed and analyzed. Univariate analysis was done to identify factors potentially affecting OS and PFS of PRPLS patients. Multivariate Cox proportional hazards analysis was used to evaluate the impact of various clinicopathological factors on OS and PFS of PRPLS patients.Results: Fifty-one PRPLS patients (28 Males, 23 Females; mean age 56.25 years) were evaluated. There was no significant effect of age, gender, contiguous organ resection, degree of differentiation and tumor size on the OS and PFS of the patients. Univariate analysis showed that negative surgical margin and early tumor stage significantly correlated with OS and PFS (all P < 0.001). Multivariate analysis showed that tumor stage [hazard ratio (HR) = 1.177, P = 0.001] was an independent predictors of poor progression-free survival, and surgical margins [HR = 4.0674 P = 0.038] and tumor stage [HR = 1.167 P = 0.001] were identified as independent predictors of poor overall survival.Conclusion: Negative surgical margin is a prognostic factor of OS, and can prolong the postoperative survival time of PRPLS patients. Tumor stage is a prognostic factor for OS and PFS, and can influence the survival of PRPLS patients. Earlier tumor stages of PRPLS are associated with significantly better outcomes.

Download Full-text

Association of tumor burden characteristics with outcomes in patients (pts) with metastatic renal cell carcinoma (mRCC) treated with sunitinib

Journal of Clinical Oncology ◽

10.1200/jco.2009.27.15_suppl.5043 ◽

2009 ◽

Vol 27 (15_suppl) ◽

pp. 5043-5043

Author(s):

A. R. Golshayan ◽

P. Elson ◽

L. S. Wood ◽

J. A. Garcia ◽

R. Dreicer ◽

...

Keyword(s):

Overall Survival ◽

Lymph Nodes ◽

Disease Progression ◽

Multivariable Analysis ◽

Progression Free Survival ◽

Tumor Location ◽

Tumor Burden ◽

Curative Therapy ◽

Prior Systemic Therapy ◽

The Impact

5043 Background: An important goal of non-curative therapy for mRCC is tumor burden (TB) control. However, the impact of tumor burden characteristics on clinical outcome has not been studied in mRCC pts treated with VEGF-targeted therapy. Methods: Pts with clear-cell mRCC treated with sunitinib from June 1, 2004, to October 5, 2007, were retrospectively identified. CT scan images were re-reviewed from baseline, at the time of maximal tumor burden shrinkage (TS), at time of disease progression and at time of last assessment prior to death. TB and percent TS were measured per RECIST criteria. Results: Sixty-nine pts were identified. The majority (54%) were favorable risk based on CCF TKI risk group classification. All pts underwent prior nephrectomy and 77% had received prior systemic therapy. Sites of metastases included: lung (87%), mediastinal lymph nodes (52%), retroperitoneal lymph nodes (36%), adrenal (29%), bone (38%), liver (22%), pancreas (14%), kidney (7%), and brain (6%). There were a median of 8 metastatic deposits across all organs (range, 1–20). Median TB at start of therapy was 14.0 cm (range, 3.0–42.2 cm). Overall response rate was 52% and 87% had some degree of TS. Median progression-free survival (PFS) and overall survival (OS) were 13.5 months and 30.9 months, respectively. In multivariable analysis, disease confined to above the diaphragm (p = 0.03) and total TB <13cm (p = 0.09) prior to sunitinib were independent positive predictors of PFS. Total number of metastases <10 (p < 0.001) and tumor volume above the diaphragm <6.5 cm (p = 0.05) were independent positive predictors of OS. Increased TS while on sunitinib was also prognostic for OS (p < 0.001). Fifty-nine pts (86%) have progressed. At time of disease progression (PD), tumor location and pattern of progression were not associated with OS. However, total TB (p = 0.003) and total number of metastatic deposits (≤12 vs. >12, p < 0.001) were significant predictors of OS from PD. At the time of last assessment prior to death, median TB was 23.9 cm, significantly higher (p < 0.001) than in pts still alive (median TB 14.4 cm). Conclusions: Tumor burden shrinkage and tumor burden at time of disease progression are associated with overall survival in pts with mRCC treated with sunitinib. [Table: see text]

Download Full-text

A clinical score for neuroendocrine tumor patients under consideration for Lu-177-DOTATATE therapy

Endocrine Related Cancer ◽

10.1530/erc-20-0482 ◽

2021 ◽

Vol 28 (3) ◽

pp. 203-212

Author(s):

Satya Das ◽

Liping Du ◽

Aimee Schad ◽

Shikha Jain ◽

Aaron Jessop ◽

...

Keyword(s):

Cox Regression ◽

Multivariable Analysis ◽

Progression Free Survival ◽

Tumor Grade ◽

Clinical Score ◽

Cancer Center ◽

Tumor Type ◽

Cox Regression Analysis ◽

Point Increase ◽

Systemic Treatments

We developed a clinical score (CS) at Vanderbilt Ingram Cancer Center (VICC) that we hoped would predict outcomes for patients with progressive well-differentiated neuroendocrine tumors (NETs) receiving therapy with Lutetium-177 (177Lu)-DOTATATE. Patients under consideration for 177Lu-DOTATATE between March 1, 2016 and March 17, 2020 at VICC were assigned a CS prospectively. The CS included 5 categories: available treatments for tumor type outside of 177Lu-DOTATATE, prior systemic treatments, patient symptoms, tumor burden in critical organs and presence of peritoneal carcinomatosis. The primary outcome of the analysis was progression-free survival (PFS). To evaluate the effect of the CS on PFS, a multivariable Cox regression analysis was performed adjusting for tumor grade, primary tumor location, and the interaction between 177Lu-DOTATATE doses received (zero, 1–2, 3–4) and CS. A total of 91 patients and 31 patients received 3–4 doses and zero doses of 177Lu-DOTATATE, respectively. On multivariable analysis, in patients treated with 3–4 doses of 177Lu-DOTATATE, for each 1-point increase in CS, the estimated hazard ratio (HR) for PFS was 2.0 (95% CI 1.61–2.48). On multivariable analysis, in patients who received zero doses of 177Lu-DOTATATE, for each 1-point increase in CS, the estimated HR for PFS was 1.22 (95% CI 0.91–1.65). Among patients treated with 3–4 doses of 177Lu-DOTATATE, those with lower CS experienced improved PFS with the treatment compared to patients with higher CS. This PFS difference, based upon CS, was not observed in patients who did not receive 177Lu-DOTATATE, suggesting the predictive utility of the score.

Download Full-text

The Role of Serum 5-HIAA as a Predictor of Progression and an Alternative to 24-h Urine 5-HIAA in Well-Differentiated Neuroendocrine Neoplasms

Biology ◽

10.3390/biology10020076 ◽

2021 ◽

Vol 10 (2) ◽

pp. 76

Author(s):

Maria Wedin ◽

Sagar Mehta ◽

Jenny Angerås-Kraftling ◽

Göran Wallin ◽

Kosmas Daskalakis

Keyword(s):

Liver Tumor ◽

Disease Surveillance ◽

Tumor Burden ◽

Neuroendocrine Neoplasms ◽

Mri Imaging ◽

Exact Test ◽

Recist 1.1 ◽

Tumor Involvement ◽

Well Differentiated ◽

Disease Stages

Our aim was to investigate the clinical utility of serum 5HIAA for disease surveillance and diagnostic purposes in a cohort of patients with well-differentiated neuroendocrine neoplasms (WD-NENs). Forty-eight patients with WD-NENs and concurrent serum and urinary 5HIAA testing, as well as CT/MRI imaging, were included. Analysis of matching-pairs did not reveal any association between RECIST 1.1 responses and changes in serum 5HIAA levels (p = 0.673). In addition, no correlation was evident between RECIST 1.1 responses and >10%, >25% or >50% changes in serum 5HIAA levels (Fisher’s exact test p = 0.380, p > 0.999, and p > 0.999, respectively). The presence of liver metastases and extensive liver tumor involvement were associated with higher serum 5HIAA levels (p = 0.045 and p = 0.041, respectively). We also confirmed a strong linear correlation between the measurements of serum and urine 5HIAA (n = 24, r = 0.791, p < 0.0001). The concordance rate of serum and urinary 5HIAA positivity at standardized laboratory cut-offs was 75%. In patients with normal renal function tests, the concordance between the two methods was as high as 89%, and a sensitivity and specificity of 80% and 88.9%, respectively, was evident (Cohen’s kappa coefficient = 0.685). In conclusion, serum 5HIAA performs well compared to urinary testing for diagnostic purposes, mainly in advanced disease stages, and corresponds well to liver tumor burden. However, it is not adequate to predict tumor progression.

Download Full-text