Increasing use of neoadjuvant chemotherapy (NAC) in muscle-invasive bladder cancer (MIBC): Prognostic impact of non-standard of care (SOC) regimens.

2019 ◽  
Vol 37 (15_suppl) ◽  
pp. 4532-4532 ◽  
Author(s):  
Yaw A. Nyame ◽  
Sarah K Holt ◽  
Brian Winters ◽  
Sarah P. Psutka ◽  
Atreya Dash ◽  
...  
Keyword(s):  
Bladder Cancer ◽  
Logistic Regression ◽  
Cox Regression ◽  
Multivariable Analysis ◽  
Population Based ◽  
Competing Risk ◽  
Oncologic Outcomes ◽  
Prognostic Impact ◽  
Cox Regression Analysis ◽  
The Impact

4532 Background: Cisplatin-based NAC can prolong overall survival (OS) in patients (pts) with MIBC. Utilization of NAC has increased to about 20% of pts with MIBC over the last decade. We evaluated NAC utilization with and without SOC cisplatin-based combination regimens and oncologic outcomes using registry data. Methods: This is a population-based analysis of linked SEER-Medicare data (2004-2011). We identified 4534 pts with MIBC (cT2-4N0-1) undergoing radical cystectomy (RC). Based on pharmacy records data, pts were stratified into 3 groups: SOC, non-SOC, and immediate cystectomy (IC). We used descriptive statistics to compare groups, and multivariate logistic regression to define factors associated with receiving SOC NAC. Competing risk bladder cancer-specific mortality (BCSM) incidence curves were generated and KM analysis was used to assess OS from time of RC. The impact of NAC on OS was evaluated with Cox regression analysis. Results: 694 (15.3%) pts received NAC, increasing from 11% in 2004 to 24.8% in 2011, with 345 (50%) receiving non-SOC, e.g. gemcitabine/carboplatin (49.3%), gemcitabine alone (21.2%), carboplatin alone (14.8%), cisplatin alone (8.4%), and methotrexate/vinblastine/ adriamycin/carboplatin (0.8%). On logistic regression, increasing age (OR 0.91, 95%CI 0.88 – 0.94, p < 0.0001), Hispanic/Latin ethnicity (OR 0.49, 95%CI 0.22 – 1.10, p = 0.08), and ≥moderate renal dysfunction (OR 0.20, 95%CI 0.08 – 0.51, p < 0.001) were associated with lower odds of SOC NAC. Non-SOC NAC was associated with higher BCSM (competing risk) and lower OS (KM) vs. IC and SOC NAC. On multivariable analysis, non-SOC NAC was associated with higher risk of BCSM (HR 1.35, 95%CI 1.06 – 1.72, p = 0.01) and lower OS (HR 1.38, 95%CI 1.11 – 1.70, p = 0.003) vs. SOC NAC. Conclusions: About 50% of pts receiving NAC were not treated with SOC regimens. Non-SOC NAC was associated with higher bladder cancer death risk. This stresses the role of SOC NAC ideally in a multidisciplinary expert setting, as well as the need for timely RC and neoadjuvant clinical trials, including cisplatin-ineligible pts.

The impact of histological variants on bladder cancer survival: A population-based analysis.

2016 ◽  
Vol 34 (2_suppl) ◽  
pp. 458-458 ◽  
Author(s):  
Francisco Gelpi-Hammerschmidt ◽  
Dayron Rodriguez ◽  
Ilker Tinay ◽  
Christopher Brian Allard ◽  
Michael Blute ◽  
...  
Keyword(s):  
Bladder Cancer ◽  
Large Population ◽  
Multivariable Analysis ◽  
Population Based ◽  
Cox Proportional Hazards ◽  
Prognostic Impact ◽  
Histological Variants ◽  
Urothelial Tumors ◽  
The Impact ◽  
Disease Specific

458 Background: We evaluated the clinical and prognostic impact of bladder cancer histologic variants (BCHV) using a large population-based cancer database. Methods: Using the Surveillance, Epidemiology, and End Results database (SEER), we identified bladder cancer patients from 2001-2012, and categorized them according to histological differentiation. 5 year disease-specific survival (DSS) was calculated using the Kaplan-Meier method. Cox proportional hazards regression model was used to predict association with disease-specific mortality (DSM). In addition, we fitted multivariate logistic regression models to predict the impact of histological variants on muscle-invasive status (MI), nodal involvement (NI), and metastatic disease (MD). Results: The cohort included 175,544 urothelial (96.3%) and 6,714 non-urothelial (3.7%) cancers. The latter were divided into: 2,382 squamous cell carcinoma, 1,648 adenocarcinoma, 888 small cell, 912 sarcomatoid, 292 signet-ring cell, 314 neuroendocrine and 278 micropapillary bladder tumors. Urothelial cancers overall had the best 5-year DSS. Of the non-urothelial variants, micropapillary and squamous had the best and worst DSS respectively (p < 0.001). On multivariable analysis predicting DSM, micropapillary and squamous variants had the best and worst prognosis respectively (HR 0.79, p = 0.102 and HR 2.63, p < 0.001), compared to urothelial tumors. On multivariable analysis predicting MI, NI, and MD: squamous (OR 22.76, p < 0.001), micropapillary (OR 3.17, p < 0.001) and adenocarcinoma (OR 4.14, p < 0.001), had higher likelihood respectively, compared to urothelial tumors. Conclusions: Despite accounting for a minority of bladder cancers, BCHV are associated with worst outcomes. It is essential to recognize the potential implications of these variants when deciding treatment. Additional studies are warranted to better characterize the clinical impact of these variants.

The difference in prognostic value of tumour-infiltrating T cells according to adjuvant chemotherapy in radiochemonaïve gastroesophageal cancer.

2017 ◽  
Vol 35 (7_suppl) ◽  
pp. 41-41
Author(s):  
Maria Christina Svensson ◽  
Jonna Berntsson ◽  
Charlotta Hedner ◽  
David Borg ◽  
Bjorn Nodin ◽  
...  
Keyword(s):  
T Cells ◽  
Adjuvant Chemotherapy ◽  
Gastric Adenocarcinoma ◽  
Prognostic Value ◽  
Cox Regression ◽  
Multivariable Analysis ◽  
Prognostic Impact ◽  
Prognostic Information ◽  
Cox Regression Analysis ◽  
The Impact

41 Background: Several studies have demonstrated a beneficial prognostic impact of tumour-infiltrating lymphocytes (TILs) in oesophageal and gastric adenocarcinoma, but whether this association differs according to adjuvant chemotherapy has not been reported. Herein, we examined the prognostic impact of different TIL subsets according to adjuvant chemotherapy in these cancer types. Methods: Immunohistochemistry was applied to assess the density of T cells (CD3+, CD8+, FoxP3+) and natural killer (NK)/T cells (CD56+) in radiochemonaïve tumors from a consecutive cohort of 174 patients with resected oesophageal or gastric adenocarcinoma. Cox proportional hazard’s modelling was applied to examine the impact of the investigated markers on time to recurrence (TTR) and overall survival (OS). Results: Dense infiltration of CD3+, CD8+, and FoxP3+ cells were all associated with a significantly prolonged TTR in univariable Cox regression analysis (hazard ratio[HR] = 0.57; 95% confidence interval [CI] 0.37-0.89; HR = 0.31, 95% CI 0.13-0.77, and HR = 0.44, 95% CI 0.28-0.68). This association remained significant for CD8+ and FoxP3+ cells in multivariable analysis, adjusted for age, tumour location, TNM stage, differentiation grade, resection margin and adjuvant chemotherapy ( HR = 0.30, 95% CI 0.11-0.77, and HR = 0.52, 95% CI 0.32-0.84), and borderline significant for CD3+ cells (HR = 0.63, 95% CI 0.39-1.02). Similar associations were observed for all markers in relation to OS. Notably, analysis in strata according to adjuvant chemotherapy revealed that none of the investigated lymphocyte subsets carried prognostic information in patients having received treatment (n = 13; 7.5%), with a significant treatment interaction between CD3+ and CD8+ cells and adjuvant chemotherapy (pinteraction0.035 and 0.021). Conclusions: These results confirm the prognostic value of a high density of TILs in gastroesophageal adenocarcinoma, but also indicate that adjuvant chemotherapy is only beneficial in patients with tumors displaying a low density of TILs. These results are of potential clinical relevance and need to be confirmed in additional, preferentially randomized, studies.

NLRP3 is a Novel Prognostic Biomarker and Correlates With Immune Infiltrates in Lung Adenocarcinoma and Skin Cutaneous Melanoma

2021 ◽  
Author(s):  
Chenxi Yuan ◽  
Qingwei Wang ◽  
Xueting Dai ◽  
Yipeng Song ◽  
Jinming Yu
Keyword(s):  
Logistic Regression ◽  
Regression Analysis ◽  
Lung Adenocarcinoma ◽  
Immune Cells ◽  
Cutaneous Melanoma ◽  
Cox Regression ◽  
Cox Regression Analysis ◽  
Potential Biomarker ◽  
The Impact ◽  
Nlrp3 Expression

Abstract Background: Lung adenocarcinoma (LUAD) and skin cutaneous melanoma (SKCM) are common tumors around the world. However, the prognosis in advanced patients is poor. Because NLRP3 was not extensively studied in cancers, so that we aimed to identify the impact of NLRP3 on LUAD and SKCM through bioinformatics analyses. Methods: TCGA and TIMER database were utilized in this study. We compared the expression of NLRP3 in different cancers and evaluated its influence on survival of LUAD and SKCM patients. The correlations between clinical information and NLRP3 expression were analyzed using logistic regression. Clinicopathologic characteristics associated with overall survival in were analyzed by Cox regression. In addition, we explored the correlation between NLRP3 and immune infiltrates. GSEA and co-expressed gene with NLRP3 were also done in this study. Results: NLRP3 expressed disparately in tumor tissues and normal tissues. Cox regression analysis indicated that up-regulated NLRP3 was an independent prognostic factor for good prognosis in LUAD and SKCM. Logistic regression analysis showed increased NLRP3 expression was significantly correlated with favorable clinicopathologic parameters such as no lymph node invasion and no distant metastasis. Specifically, a positive correlation between increased NLRP3 expression and immune infiltrating level of various immune cells was observed. Conclusion: Together with all these findings, increased NLRP3 expression correlates with favorable prognosis and increased proportion of immune cells in LUAD and SKCM. These conclusions indicate that NLRP3 can serve as a potential biomarker for evaluating prognosis and immune infiltration level.

Prognostic Nomogram Incorporating Immunohistochemical Results for the Overall Survival of Patients with Bladder Cancer.

2020 ◽  
Author(s):  
Tianwei Wang ◽  
Yunyan Wang
Keyword(s):  
Risk Factors ◽  
Overall Survival ◽  
Bladder Cancer ◽  
Risk Model ◽  
Cox Regression ◽  
Validation Cohort ◽  
Multivariable Analysis ◽  
Clinical Information ◽  
Internal Validation ◽  
Cox Regression Analysis

Abstract Objectives: In this study, we want to combine GATA3, VEGF, EGFR and Ki67 with clinical information to develop and validate a prognostic nomogram for bladder cancer.Methods: A total of 188 patients with clinical information and immunohistochemistry were enrolled in this study, from 1996 to 2018. Univariable and multivariable cox regression analysis was applied to identify risk factors for nomogram of overall survival (OS). The calibration of the nomogram was performed and the Area Under Curve (AUC) was calculated to assess the performance of the nomogram. Internal validation was performed with the validation cohort., the calibration curve and the AUC were calculated simultaneously.Results: Univariable and multivariable analysis showed that age (HR: 2.229; 95% CI: 1.162-4.274; P=0.016), histology (HR: 0.320; 95% CI: 0.136-0.751; P=0.009), GATA3 (HR: 0.348; 95% CI: 0.171-0.709; P=0.004), VEGF (HR: 2.295; 95% CI: 1.225-4.301; P=0.010) and grade (HR: 4.938; 95% CI: 1.339-18.207; P=0.016) remained as independent risk factors for OS. The age, histology, grade, GATA3 and VEGF were included to build the nomogram. The accuracy of the risk model was further verified with the C-index. The C-index were 0.65 (95% CI, 0.58-0.72) and 0.58 (95% CI, 0.46-0.70) in the training and validation cohort respectively. Conclusions: A combination of clinical variables with immunohistochemical results based nomogram would predict the overall survival of patients with bladder cancer.

scholarly journals Risk factors and survival outcomes of metachronous contralateral upper tract urothelial carcinoma

2020 ◽  
Vol 10 (1) ◽  
Author(s):  
Kan Wu ◽  
Jiayu Liang ◽  
Yiping Lu
Keyword(s):  
Risk Factors ◽  
Bladder Cancer ◽  
Regression Model ◽  
Urothelial Carcinoma ◽  
Large Population ◽  
Multivariable Analysis ◽  
Population Based ◽  
Upper Tract Urothelial Carcinoma ◽  
Competing Risk ◽  
Upper Tract

Abstract Because population-based risk estimates for metachronous contralateral UTUC are lacking. In this study, we aimed to evaluate the risk and survival of metachronous contralateral upper tract urothelial carcinoma (UTUC) on a large population-based level. A total of 23,075 patients were identified from the Surveillance, Epidemiology, and End Results database (1973–2015), 144 (0.6%) patients developed metachronous contralateral UTUC (median of 32 months after diagnosis). The cumulative incidence at 10, 20, and 30 years of follow-up was 1.1%, 1.6%, and 2.6%, respectively. We applied Fine and Gray’s competing risk regression model to determine the risk factors of a new contralateral, metachronous UTUC. The competing risk regression model demonstrated that older age (hazard ratio [HR] 0.75; 95% CI 0.67–0.85) and larger tumor size (HR 0.61; 95% CI 0.39–0.97) were associated with a significantly decreased risk of metachronous contralateral UTUC. However, bladder cancer presence was an independent risk factor for the development of contralateral tumors (HR 2.42; 95% CI 1.73–3.37). In addition, we demonstrated developing contralateral UTUC was not associated with poor prognosis by using Kaplan–Meier and multivariable analysis. Our findings suggest that metachronous contralateral UTUC is comparatively rare, and has not impact on survival. Importantly, patients with younger age, small tumours, and the presence of bladder cancer were more likely to develop a contralateral tumor, which may provide a rationale for lifelong surveillance in high-risk patients.

P0211GLOMERULAR HYPERFILTRATION AND CANCER: A NATIONWIDE POPULATION-BASED STUDY

2020 ◽  
Vol 35 (Supplement_3) ◽  
Author(s):  
Yaerim Kim ◽  
Jeongsoo Yoon ◽  
Jin Hyuk Paek ◽  
Woo Yeong Park ◽  
Kyubok Jin ◽  
...  
Keyword(s):  
National Health ◽  
Malignant Disease ◽  
Cox Regression ◽  
Asian Population ◽  
Population Based ◽  
Glomerular Hyperfiltration ◽  
Outcome Variable ◽  
Cox Regression Analysis ◽  
Increased Risk ◽  
The Impact

Abstract Background and Aims Glomerular hyperfiltration is associated with all-cause mortality. Herein, we evaluated the association between glomerular hyperfiltration and the development of malignant disease, the most common cause of death, in an Asian population. Method We retrospectively reviewed the National Health Insurance Service database of Korea for people who received national health screenings from 2012 to 2013. Glomerular hyperfiltration was defined as the 95th percentile and greater after adjusting for age and sex. We performed a multivariate Cox regression analysis using glomerular hyperfiltration at the first health screening as the exposure variable and cancer development as the outcome variable to evaluate the impact of glomerular hyperfiltration on the development of malignant disease. Results A total of 1,953,123 examinations who followed-up for 4.9 years were included in this study. Among the 8 different site-specific malignant disease categories, digestive organs and female genital organs showed a significant associations between glomerular hyperfiltration and malignancy. The population with glomerular hyperfiltration showed an increased risk for stomach cancer (adjusted hazard ratio [aHR], 1.27), colorectal cancer (aHR, 1.23), and liver or intrahepatic malignancy (aHR, 1.40). In addition, the risk for uterine and ovarian cancer was significantly increased in the population with glomerular hyperfiltration (aHR, 1.36). Conclusion Glomerular hyperfiltration was associated with an increased risk for the development of malignant diseases in specific organs, such as the stomach, colorectum, uterus, and ovary. Glomerular hyperfiltration needs to be considered a significant sign of the need to evaluate the possibility of hidden adverse health conditions, including malignancies.

Prognostic Significance of Angiogenesis and Ki-67, p53, and p21 Expression: A Population-Based Endometrial Carcinoma Study

1999 ◽  
Vol 17 (5) ◽  
pp. 1382-1382 ◽  
Author(s):  
Helga B. Salvesen ◽  
Ole Erik Iversen ◽  
Lars A. Akslen
Keyword(s):  
High Risk ◽  
Endometrial Carcinoma ◽  
Cox Regression ◽  
Figo Stage ◽  
Population Based ◽  
Prognostic Impact ◽  
Histologic Type ◽  
Ki 67 ◽  
Cox Regression Analysis

PURPOSE: For endometrial carcinoma patients, there is a need for improved identification of high-risk groups that may benefit from postoperative adjuvant therapy. We therefore studied the prognostic impact of markers for cell proliferation, cell-cycle regulation, and angiogenesis among endometrial carcinoma patients in a population-based setting. PATIENTS AND METHODS: All patients diagnosed with endometrial carcinoma between 1981 and 1985 in Hordaland County, Norway, were studied. The median follow-up for the survivors was 11.5 years (range, 8 to 15 years), with no patient lost because of insufficient follow-up information. Paraffin-embedded tumor tissue, available in 96% of the cases (n = 142), was studied immunohistochemically for microvessel density (MVD) and expression of Ki-67, p53, and p21 proteins. We used the hot spot method for calculation of MVD, and expression of Ki-67 and p21 protein, because this approach may increase the probability of detecting small aggressive clones of possible prognostic relevance. The importance of these tumor markers was investigated in univariate survival analyses and Cox regression analysis. RESULTS: The majority of traditional clinicopathologic variables was significantly associated with the tumor biomarkers. Age, International Federation of Gynecology and Obstetrics (FIGO) stage, histologic type, histologic grade, MVD, as well as Ki-67, p53, and p21 protein expression, all significantly influenced survival in univariate analyses (P ≤ .05). In the Cox regression analysis, age, FIGO stage, MVD, Ki-67 expression, and p53 expression were the only variables with independent prognostic impact (P ≤ .05), whereas histologic type, histologic grade, and p21 expression had no independent influence. A group of high-risk patients with more than one unfavorable marker was identified. CONCLUSION: In addition to age and FIGO stage, MVD, Ki-67, and p53 protein expression showed an independent prognostic impact. Thus, information derived from routine histologic specimens identified a subgroup of high-risk endometrial carcinoma patients in this population-based study.

Impact of putative chemopreventative agents on prostate cancer diagnosis.

2019 ◽  
Vol 37 (7_suppl) ◽  
pp. 40-40
Author(s):  
Hanan Goldberg ◽  
Faizan Moshin ◽  
Zachary William Abraham Klaassen ◽  
Thenappan Chandrasekar ◽  
Christopher Wallis ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Cox Regression ◽  
Large Population ◽  
Multivariable Analysis ◽  
Population Based ◽  
Cumulative Exposure ◽  
Population Based Study ◽  
Diagnosis Rate ◽  
The Impact

40 Background: Prostate cancer (PC) is the most common non-cutaneous cancer in Canadian men and the third most common cause of cancer death in Canada. Several studies have shown that use of commonly prescribed medications, including those used for diabetes and hypercholesterolemia, is associated with improved survival in various malignancies, including PC. There has not been any large population-based study, examining the effects of these and other commonly prescribed medications, on the rate of PC diagnosis, over a 20 years follow-up period. Methods: A retrospective population-based study using data from the institute of clinical evaluative sciences, including all male patients aged 65 and above in Ontario who have had a negative first prostate biopsy between 1994 and 2016. We assessed the impact of commonly prescribed medications on PC diagnosis. The medications included Statins (hydrophilic and hydrophobic), diabetes drugs (metformin, insulins, sulfonylureas, and thizolidinedions), proton pump inhibitors, 5 alpha reductase inhibitors, and alpha blockers. Time dependent Cox regression proportional hazards models were performed determine predictors of PC diagnosis. Medication exposure was time varying and modeled as “ever” vs. “never” use or as cumulative exposure for 6 months of usage. A priori variables included in the model included age, ADG comorbidity score, rurality index, index year, and all medications. Results: A total of 51,415 men were analyzed over a mean (SD) follow-up time of 8.06 (5.44) years. Overall, 10,466 patients (20.4%) were diagnosed with PC, 16,726 (32.5%) had died, and 1,460 (2.8%) patients died of PC. On multivariable analysis increasing age and rurality index were associated with higher PC diagnosis rate, while a more recent index year, and usage of hydrophilic statins was associated with a lower diagnosis rate in both “ever” vs. “never” and cumulative models (HR 0.832, 95% CI 0.732-0.946, p = 0.005, HR 0.973 95% CI 0.951-0.995, p = 0.016, respectively). Conclusions: Hydrophilic statins are associated with a clinically significant lower PC diagnosis. To our knowledge this is the first study demonstrating a clear advantage of one group of statins (hydrophilic) over another (hydrophobic) in PC prevention.

Dissecting outcomes of patients (pts) with

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. 5043-5043
Author(s):  
Praful Ravi ◽  
Gregory Russell Pond ◽  
Leonidas Nikolaos Diamantopoulos ◽  
Rohit K. Jain ◽  
William Paul Skelton ◽  
...  
Keyword(s):  
Urinary Tract ◽  
Cox Regression ◽  
Pathologic Complete Response ◽  
Multivariable Analysis ◽  
Pathologic Response ◽  
Cox Regression Analysis ◽  
Risk Of Recurrence ◽  
Split Dose ◽  
Female Sex ◽  
The Impact

5043 Background: Pathologic complete response (pCR) after NAC for MIBC is strongly correlated with long-term overall survival. However, there are sparse data on the risk of recurrence based on depth of pathologic response (pT0, pTa, pTis, pT1), and the differential impact of clinicopathologic factors and NAC regimen on recurrence. Methods: Baseline data on all pts with cT2-4N0-1 MIBC receiving NAC and who achieved < ypT2N0 disease at radical cystectomy (RC) from 9 international centers were obtained. The key outcome was time to recurrence (TTR) – defined as the time to any recurrence in the urinary tract or regional/distant metastasis, with death (in the absence of recurrence) considered a competing risk. Cox regression analysis was used to analyze the impact of clinical factors on recurrence. Results: A total of 506 pts were available. Median age was 66 years (range 33-86) and 78% (n = 396) were male; median follow-up after RC was 2.6 years. The majority of patients had pure urothelial histology (n = 371, 73%), and baseline stage was cT2N0 (n = 368, 73%), cT3-4N0 (n = 95, 19%) and TanyN1 (n = 43, 9%). NAC regimens were gemcitabine-cisplatin (GC, n = 296, 59%), dose-dense methotrexate-vinblastine-doxorubicin-cisplatin (ddMVAC, n = 141, 28%), split-dose GC (n = 29, 6%), MVAC (n = 29, 6%) and non-cisplatin based regimens (n = 11, 2%). At RC, 304 patients (60%) had ypT0N0 disease, 32 (6%) had ypTaN0, 107 (21%) had ypTisN0 and 63 (13%) had ypT1N0. Overall, 43 patients (8%) recurred with a median TTR of 56 weeks (range 7-251); 5-year freedom from recurrence was 87% (95% CI 83-91). The majority (n = 38) recurred outside the urinary tract. On multivariable analysis, ypTa (HR = 3.36 [1.24-9.11]) and ypT1 (HR = 2.88 [1.33-6.22], p = 0.013) disease at RC were predictors of shorter TTR, while female sex was associated with longer TTR (HR = 0.52 [0.27-0.98], p = 0.043). The type of NAC was not predictive of TTR (GC vs. other, HR = 1.49 [0.75-2.97], p = 0.26). Conclusions: To our knowledge, this is the largest study to quantify the risk of recurrence in pts achieving pathologic response after NAC and RC for MIBC. 8% of patients undergoing NAC and achieving < ypT2N0 at RC recurred. Residual ypTa and ypT1 disease conferred a significantly higher risk of recurrence, while ypTis did not; female sex was associated with a lower risk of recurrence. Importantly, the type of cisplatin-based NAC regimen used was not an independent predictor of recurrence.

scholarly journals Clinical Efficacy of Ruxolitinib in Patients with Myelofibrosis: A Nationwide Population-Based Study in Korea

2021 ◽  
Vol 10 (20) ◽  
pp. 4774
Author(s):  
Byung-Hyun Lee ◽  
Hyemi Moon ◽  
Jae-Eun Chae ◽  
Ka-Won Kang ◽  
Byung-Soo Kim ◽  
...  
Keyword(s):  
Propensity Scores ◽  
Cox Regression ◽  
Multivariable Analysis ◽  
Previous History ◽  
Population Based ◽  
Population Based Study ◽  
Cox Regression Analysis ◽  
History Of ◽  
Korean National Health Insurance ◽  
Insurance Database

Previous studies have reported the survival benefit after ruxolitinib treatment in patients with myelofibrosis (MF). However, population-based data of its efficacy are limited. We analyzed the effects of ruxolitinib in MF patients with data from the Korean National Health Insurance Database. In total, 1199 patients diagnosed with MF from January 2011 to December 2017 were identified, of which 731 were included in this study. Patients who received ruxolitinib (n = 224) were matched with those who did not receive the drug (n = 507) using the 1:1 greedy algorithm. Propensity scores were formulated using five variables: age, sex, previous history of arterial/venous thrombosis, and red blood cell (RBC) or platelet (PLT) transfusion dependence at the time of diagnosis. Cox regression analysis for overall survival (OS) revealed that ruxolitinib treatment (hazard ratio (HR), 0.67; p = 0.017) was significantly related to superior survival. In the multivariable analysis for OS, older age (HR, 1.07; p < 0.001), male sex (HR, 1.94; p = 0.021), and RBC (HR, 3.72; p < 0.001) or PLT (HR, 9.58; p = 0.001) transfusion dependence were significantly associated with poor survival, although type of MF did not significantly affect survival. Considering evidence supporting these results remains weak, further studies on the efficacy of ruxolitinib in other populations are needed.

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