scholarly journals Sorafenib for Desmoid Tumors. A Cost Analysis: Too Much for Too Little?

2021 ◽  
Author(s):  
Michael S. Johns ◽  
William Merritt III ◽  
Lori Rhodes ◽  
Candice N. Ford ◽  
Mark Thompson ◽  
...  
Keyword(s):  
Cost Analysis ◽  
Absolute Risk ◽  
Financial Burden ◽  
Objective Response ◽  
Evaluation Criteria ◽  
Progression Free Survival ◽  
Absolute Risk Reduction ◽  
Number Needed To Treat ◽  
Desmoid Tumors ◽  
Radiologic Progression

Abstract Background: A recent randomized trial showed that sorafenib increased progression free survival (PFS) in patients with desmoid tumors despite many patients experiencing stable disease or spontaneous regression without treatment. Using these trial data, we completed a cost analysis of sorafenib efficacy through two years of treatment. Methods: 2019 Medicare Part D rates for sorafenib were used (dose 400 mg/day, cost $309/day). Yearly costs per progression and objective response were calculated. Radiologic progression and response were defined using Response Evaluation Criteria in Solid Tumors (RECIST). Patients with disease progression were separately analyzed in two groups: both clinical and radiologic (CAR), and radiologic alone. Results: 84 previously randomized patients were analyzed (placebo: 35, sorafenib: 49). After 1 year, sorafenib was associated with 43% absolute risk reduction (ARR) of CAR progression and number-needed-to-treat (NNT) of 2.3 patients/year, costing $259,406. After 2 years, ARR was 48% and NNT of 2.1 patients/year, costing $473,697. When assessing only patients with RECIST defined radiologic progression, sorafenib patients had ARR of 13.9% with NNT 7.2 and estimated costs of $812,052 at one year. Two-year ARR was 17.5% with NNT 5.7 and estimated costs $1,285,052. Sorafenib patients experienced improved RECIST partial response rates at 1 and 2 years of 14.7% and 14.3%, with NNT 6.8 and 6.9, and costs of $766,938 and $1,556,433; respectively. Conclusion: For the treatment of desmoid tumors, Sorafenib led to improved PFS, but at a substantial cost per patient. Beneficial RECIST outcomes were less likely and at higher cost. Patients should be informed of possible benefits of treatment versus potential financial burden.

scholarly journals OP0240 EPIDEMIOLOGY, PREDICTORS OF MORTALITY AND ROLE OF PROPHYLAXIS FOR PNEUMOCYSTITIS JIROVECI PNEUMONIA AMONG RHEUMATIC PATIENTS: A TERRITORY-WIDE STUDY

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 147-147
Author(s):  
C. W. S. Chan ◽  
H. Y. Chung ◽  
W. Y. Yeung ◽  
C. S. Lau ◽  
P. H. LI
Keyword(s):  
Lupus Erythematosus ◽  
Absolute Risk ◽  
Absolute Risk Reduction ◽  
Number Needed To Treat ◽  
Healthcare Database ◽  
Pneumocystis Jiroveci ◽  
Predictors Of Mortality ◽  
Prednisolone Dose ◽  
Rheumatic Patients

Background:Pneumocystis jiroveci pneumonia (PJP) is an opportunistic infection affecting immunocompromised individuals. Due to its high mortality, PJP prophylaxis is commonly recommended for many immunocompromising conditions. However, evidence regarding the burden and role of prophylaxis in PJP among rheumatic patients remains limited. There is lack of consensus for when and for whom to initiate prophylaxis. Delineating the epidemiology, predictors of mortality and efficacy of prophylaxis in PJP among rheumatic patients is urgently needed.Objectives:To delineate the epidemiology of PJP, identify predictors of mortality and evaluate the usefulness of prophylaxis in rheumatology patients.Methods:We performed a big-data cohort study based on the territory-wide healthcare database of the Hong Kong Hospital Authority. All patients with a diagnosis of anti-neutrophil cytoplasmic antibody-associated vasculitis (AAV), immune-mediated myositis (IMM), rheumatoid arthritis (RA), systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or spondyloarthritis (SpA) between 2015-2019 were included. PJP were identified based on physician diagnosis and/or positive microbiological results from deep respiratory tract specimens. Prophylaxis was defined as prescription of a prophylactic dose of co-trimoxazole for at least 2 weeks and/or inhaled pentamidine. Prevalence of PJP, prophylaxis and mortality among rheumatic patients were calculated. Demographics, blood parameters and immunosuppressants use was also collected for multivariate analysis. Number needed to treat (NNT) analysis was performed based on absolute risk reduction of PJP in patients with and without prior PJP prophylaxis.Results:A total of 21,587 unique rheumatic patients were analysed (54% RA, 25% SLE, 13% SpA, 5% IMM, 2% AAV and 1% SSc). Between 2015-2019, 1141 (5.3%) patients were prescribed PJP prophylaxis and 48 (0.2%) developed PJP. None of those patients who developed PJP had received prophylaxis prior to infection. The risk of PJP was highest among SSc (1.8%), AAV (1.4%) and IMM (0.7%) patients, with NNT of SSc 36, AAV 48 and IMM 114. Within these disease entities, the majority of PJP occurred at prednisolone dose of 15mg/day (P15) or above (100% in SSc and IIM, 66.7% in AAV). Overall, PJP was associated with a mortality-rate of 39.6%. Glucocorticoid dose (daily prednisolone dose equivalent 29.1±23.5mg vs 11.4±7.2mg, P<0.01) and lymphopenia (0.44x109/L vs 0.90x109/L, P= 0.04) at PJP diagnosis were associated with PJP mortality in rheumatic patients.Conclusion:PJP is an uncommon but important infection in rheumatic patients associated with significant mortality. PJP prophylaxis is effective and should be considered in patients with SSc, AAV and IMM, especially in those receiving a steroid dose above P15.Disclosure of Interests:None declared

scholarly journals Cabozantinib As Salvage Therapy for Patients With Tyrosine Kinase Inhibitor–Refractory Differentiated Thyroid Cancer: Results of a Multicenter Phase II International Thyroid Oncology Group Trial

2017 ◽  
Vol 35 (29) ◽  
pp. 3315-3321 ◽  
Author(s):  
Maria E. Cabanillas ◽  
Jonas A. de Souza ◽  
Susan Geyer ◽  
Lori J. Wirth ◽  
Michael E. Menefee ◽  
...  
Keyword(s):  
Thyroid Cancer ◽  
Targeted Therapy ◽  
Phase Ii ◽  
Differentiated Thyroid Cancer ◽  
Kinase Inhibitor ◽  
Objective Response ◽  
Evaluation Criteria ◽  
Growth Factor Receptor ◽  
Progression Free Survival ◽  
Multikinase Inhibitor

Purpose Sorafenib and lenvatinib are oral multikinase inhibitors targeting vascular endothelial growth factor receptor (VEGFR) and approved for radioiodine (RAI)-refractory differentiated thyroid cancer (DTC). However, there are no approved second- or third-line therapies. MET is implicated in resistance to VEGFR inhibitors. Cabozantinib is an oral multikinase inhibitor targeting MET in addition to VEGFR and is approved for medullary thyroid cancer. In a phase I study of cabozantinib, five of eight patients with DTC previously treated with a VEGFR-targeted therapy had an objective response to cabozantinib. Patients and Methods Patients with RAI-refractory disease with Response Evaluation Criteria in Solid Tumor (RECIST) measurable disease and evidence of progression on prior VEGFR-targeted therapy were enrolled in this single-arm phase II study. The cabozantinib starting dose was 60 mg/day orally but could be escalated to 80 mg if the patient did not experience a response. Patients underwent tumor assessment according to RECIST v1.1 every 8 weeks. In this study, if at least five of 25 response-evaluable patients had an objective response, cabozantinib would be considered a promising agent in this patient population. Results Twenty-five patients were enrolled. The median age was 64 years, and 64% of patients were men. Twenty-one patients had received only one prior VEGFR-targeted therapy (sorafenib, pazopanib, or cediranib), and four patients had received two such therapies. The most common treatment-related adverse events were fatigue, weight loss, diarrhea, palmar-plantar erythrodysesthesia, and hypertension. One drug-related death was noted. Of the 25 patients, 10 (40%) had a partial response, 13 (52%) had stable disease, and two (8%) had nonevaluable disease. The median progression-free survival and overall survival were 12.7 months and 34.7 months, respectively. Conclusion Cabozantinib demonstrated clinically significant, durable objective response activity in patients with RAI-refractory DTC who experienced disease progression while taking prior VEGFR-targeted therapy.

Prevalence of underlying malignancies in complex cysts of the breast

2021 ◽  
pp. 1-7
Author(s):  
J.C.W.L. Gerets ◽  
M. Kool ◽  
P.C.G. Simons ◽  
F. Aarts ◽  
F.J. Vogelaar
Keyword(s):  
Healthcare Costs ◽  
Disease Burden ◽  
Watchful Waiting ◽  
Absolute Risk ◽  
Absolute Risk Reduction ◽  
Patient Characteristics ◽  
Number Needed To Treat ◽  
Single Center ◽  
Underlying Malignancy

INTRODUCTION: The management of complex cysts of the breast is an ongoing topic of discussion. The aim of this study was to determine the prevalence of underlying malignancy in radiologically diagnosed complex cysts, and to assess whether watchful waiting could be the preferred method to safely manage complex cysts of the breast. SUBJECTS AND METHODS: A single-center retrospective study was performed between May 2003 and November 2019 in the VieCuri Medical Centre. Women with a radiologically diagnosed complex cyst of the breast were included. Prevalence of underlying malignancy was calculated, as were absolute risk reduction and number needed to treat in order to diagnose malignancy. In addition, patient characteristics were compared to determine characteristics associated with malignancy. RESULTS: Of 78 radiologically diagnosed complex cysts of the breast, five (6,4%) were found to be malignant. The number needed to treat was calculated at 12,8 (absolute riks reduction 0,078). Age (P = 0,003) was associated with malignancy. CONCLUSION: Complex cysts of the breast could be managed more conservatively. Patient characteristics can be used to assess the eligibility for radiological follow-up. This, in turn, would lead to a lower NNT and possibly a decrease in disease burden and healthcare costs.

scholarly journals Effect of Using a Safety Checklist on Patient Complications after Surgery

2014 ◽  
Vol 120 (6) ◽  
pp. 1380-1389 ◽  
Author(s):  
Brigid M. Gillespie ◽  
Wendy Chaboyer ◽  
Lukman Thalib ◽  
Melinda John ◽  
Nicole Fairweather ◽  
...  
Keyword(s):  
Absolute Risk ◽  
Absolute Risk Reduction ◽  
Cochrane Library ◽  
Number Needed To Treat ◽  
Complication Rates ◽  
Random Effects Models ◽  
Fixed And Random Effects ◽  
Before And After ◽  
Meta Analyses ◽  
The Cochrane Library

Abstract Background: Previous before-and-after studies indicate that the use of safety checklists in surgery reduces complication rates in patients. Methods: A systematic review of studies was undertaken using MEDLINE, CINAHL, Proquest, and the Cochrane Library to identify studies that evaluated the effects of checklist use in surgery on complication rates. Study quality was assessed using the Methodological Index for Nonrandomized Studies. The pooled risk ratio (RR) was estimated using both fixed and random effects models. For each outcome, the number needed to treat (NNT) and the absolute risk reduction (ARR) were also computed. Results: Of the 207 intervention studies identified, 7 representing 37,339 patients were included in meta-analyses, and all were cohort studies. Results indicated that the use of checklists in surgery compared with standard practice led to a reduction in any complication (RR, 0.63; 95% CI, 0.58 to 0.72; P &lt; 0.0001; ARR, 3.7%; NNT, 27) and wound infection (RR, 0.54; 95% CI, 0.40 to 0.72; P = 0.0001; ARR, 2.9%; NNT, 34) and also reduction in blood loss (RR, 0.56; 95% CI, 0.45 to 0.70; P = 0.0001; ARR, 3.8%; NNT, 33). There were no significant reductions in mortality (RR, 0.79; 95% CI, 0.57 to 1.11; P = 0.191; ARR, 0.44%; NNT, 229), pneumonia (RR, 1.03; 95% CI, 0.73 to 1.4; P = 0.857; ARR, 0.04%; NNT, 2,512), or unplanned return to operating room (RR, 0.75; 95% CI, 0.56 to 1.02; P = 0.068; ARR, 0.52%; NNT, 192). Conclusion: Notwithstanding the lack of randomized controlled trials, synthesis of the existing body of evidence suggests a relationship between checklist use in surgery and fewer postoperative complications.

Comparing the clinical efficacies of anti-PD-1 antibody monotherapy and anti-PD-1 and anti-CTLA-4 combination therapy as first-line immunotherapy in Japanese advanced acral melanoma: A retrospective, multicenter study (JAMP-neo study).

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 9542-9542
Author(s):  
Yasuhiro Nakamura ◽  
Yukiko Kiniwa ◽  
Hiroshi Kato ◽  
Osamu Yamasaki ◽  
Takeo Maekawa ◽  
...  
Keyword(s):  
Combination Therapy ◽  
Clinical Efficacy ◽  
Objective Response ◽  
Evaluation Criteria ◽  
Progression Free Survival ◽  
First Line ◽  
Acral Melanoma ◽  
Kaplan Meier ◽  
Melanoma Patients ◽  
Clinical Records

9542 Background: Anti-PD-1 antibody monotherapy (PD1) has been commonly used for patients with advanced acral melanoma (AM). However, recent studies have demonstrated the limited clinical efficacy of PD1 in AM compared to non-acral cutaneous melanoma, particularly in nail apparatus melanoma. Although advanced AM patients are strong candidates for first-line anti-PD-1 and anti-CTLA-4 combination therapy (PD1+CTLA4), data on the clinical efficacy of PD1+CTLA4 in AM are lacking. Thus, we aimed to compare the clinical efficacies of PD1+CTLA4 and PD1 in Japanese advanced AM patients. Methods: We retrospectively reviewed the clinical records of advanced AM patients treated with PD1+CTLA4 or PD1 as first-line immunotherapy at 23 Japanese institutions. Clinical response was assessed using the Response Evaluation Criteria in Solid Tumors (RECIST) criteria. Survival was estimated using Kaplan-Meier analysis. Toxicity was assessed according to CTCAE 4.0. Results: A total of 192 patients (median age, 72 years) with advanced AM (palm and sole melanoma, 135; nail apparatus melanoma, 57) were included in the study. PD1+CTLA4 and PD1 were used as first-line immunotherapy in 39 and 153 patients, respectively. The baseline demographics and characteristics were similar between the PD1+CTLA4 and PD1 groups, except for age (median age 67.3 vs. 73.2; P = 0.005). The objective response rate (ORR) in PD1+CTLA4 was significantly higher than that of the PD1 group (38.5% vs. 16.3%; P = 0.047). The median progression-free survival (PFS) and overall survival (OS) in the PD1+CTLA4 group tended to be longer than those of the PD1 group, but the differences were not significant (median PFS 7.3 months vs. 4.5 months; P = 0.19, median OS 43.6 months vs. 18.2 months; P = 0.19). In the subgroup analysis of the palm and sole melanoma cohorts, no significant differences in ORR, PFS, and OS were observed between the PD1+CTLA4 and PD1 groups (ORR 31% vs. 20.8%; P = 0.67, median PFS 5.3 months vs. 5.9 months; P = 0.87, median OS not reached vs. 22.3 months; P = 0.66). Meanwhile, the nail apparatus melanoma cohort in the PD1+CTLA4 group exhibited significantly higher ORR, and longer PFS and OS than the PD1 group (ORR 60% vs 6.1%; P < 0.001; median PFS 19.6 months vs 3.8 months; P = 0.008, median OS 43.6 months vs 13.5 months; P = 0.049). Due to immune-related adverse events in all cohorts, the treatment cessation rate was higher in the PD1+CTLA4 group than the PD1 group (59% vs. 11.8%). Conclusions: PD1+CTLA4 was clinically more efficacious than PD 1 in advanced AM patients. Notably, advanced nail apparatus melanoma patients were strong candidates for first-line PD1+CTLA4.

scholarly journals LO066: H1-antihistamine administration is associated with a lower likelihood of progression to anaphylaxis among emergency department patients with allergic reactions

CJEM ◽  
2016 ◽  
Vol 18 (S1) ◽  
pp. S53-S53
Author(s):  
T. Kawano ◽  
B.E. Grunau ◽  
K. Gibo ◽  
F.X. Scheuermeyer ◽  
R. Stenstrom
Keyword(s):  
Absolute Risk ◽  
Absolute Risk Reduction ◽  
Number Needed To Treat ◽  
Allergic Reactions ◽  
Severe Anaphylaxis ◽  
Number Of Patients ◽  
Lower Likelihood ◽  
H1 Antihistamines ◽  
Secondary Outcomes ◽  
Propensity Score Adjustment

Introduction: H1-antihistamines are often used to treat allergic reactions, however, the influence of H1-antihistamines on progression to anaphylaxis remains unclear. Among patients initially presenting with allergic reactions, we investigated whether H1-antihistamines were associated with a lower proportion of patients progressing to anaphylaxis during observation. Methods: This was a retrospective cohort study conducted at two urban EDs from 2007 to 2012. We included adult patients with allergy and excluded those who met criteria of anaphylaxis at first evaluation by medical professionals and/or received antihistamines before the evaluation. Primary outcomes of interest were the number of patients who developed anaphylaxis during observation at ED and/or transportation by EMS. Secondary outcomes were the number of biphasic reactions and severe anaphylaxis (defined as sBP<90; SpO2<92%; and/or confusion, collapse, loss of conscious, or incontinence). Logistic regression was performed comparing primary and secondary outcomes between H1-antihistamine treated and non-treated groups with propensity score adjustment of the baseline covariates. Number needed to treat (NNT) was calculated by adjusted absolute risk reduction of H1-antihistamine compared to non H1-antihistamine use on primary outcome. Results: This study included 1717 patients with allergic reactions, of whom 1228 were treated with H1-antihistamines. In the H1-antihistamine group 1.0% and 0.2% developed anaphylaxis and severe anaphylaxis, respectively; in the non-H1-antihistamine group 2.6% and 0.6% developed anaphylaxis and severe anaphylaxis, respectively. There were no biphasic reactions (0%, 95% confidence interval [CI] 0 to 0.17%). Administration of H1-antihistamines was associated with a lower incidence of subsequent anaphylaxis (adjusted odds ratio [OR] 0.23, 95% CI 0.10 to 0.53; NNT to benefit 49.1, 95% CI 41.6 to 83.3). There were no significant associations between H1-histamines administration and secondary outcomes. Conclusion: Among ED patient with allergic reactions, H1-antihistamine administration was associated with a lower likelihood of progression to anaphylaxis. These findings suggest that H1-antihistamines should be administered early in the care of patients with allergic reactions.

scholarly journals Phase II California Cancer Consortium Trial of Gemcitabine-Eribulin Combination in Cisplatin-Ineligible Patients With Metastatic Urothelial Carcinoma: Final Report (NCI-9653)

2019 ◽  
Vol 37 (29) ◽  
pp. 2682-2688 ◽  
Author(s):  
Sarmad Sadeghi ◽  
Susan G. Groshen ◽  
Denice D. Tsao-Wei ◽  
Rahul Parikh ◽  
Amir Mortazavi ◽  
...  
Keyword(s):  
Urothelial Carcinoma ◽  
Phase Ii ◽  
Performance Status ◽  
Objective Response ◽  
Evaluation Criteria ◽  
Progression Free Survival ◽  
Median Number ◽  
Final Report ◽  
Curative Surgery ◽  
Metastatic Urothelial Carcinoma

PURPOSE Patients with metastatic urothelial carcinoma are often ineligible for cisplatin-based treatments. A National Cancer Institute Cancer Therapy Evaluation Program–sponsored trial assessed the tolerability and efficacy of a gemcitabine-eribulin combination in this population. METHODS Patients with treatment-naïve advanced or recurrent metastatic urothelial carcinoma of the bladder, ureter, or urethra not amenable to curative surgery and not candidates for cisplatin-based therapy were eligible. Cisplatin ineligibility was defined as creatinine clearance less than 60 mL/min (but ≥ 30 mL/min), grade 2 neuropathy, or grade 2 hearing loss. Treatment was gemcitabine 1,000 mg/m2 intravenously followed by eribulin 1.4 mg/m2, both on days 1 and 8, repeated in 21-day cycles until progression or unacceptable toxicity. A Simon two-stage phase II trial design was used to distinguish between Response Evaluation Criteria in Solid Tumors, version 1.1 objective response rates of 20% versus 50%. RESULTS Between June 2015 and March 2017, 24 eligible patients with a median age of 73 years (range, 62 to 88 years) underwent therapy. Performance status of 0, 1, or 2 was seen in 11, 11, and two patients, respectively. Sites of disease included: lymph nodes, 16; lungs, nine; liver, seven; bladder, five; bones, two. Median number of cycles received was four (range, one to 16). Of 24 patients, 12 were confirmed responders; the observed objective response rate was 50% (95% CI, 29% to 71%). Median overall survival was 11.9 months (95% CI, 5.6 to 20.4 months), and median progression-free survival was 5.3 months (95% CI, 4.5 to 6.7 months). The most common treatment-related any-grade toxicities were fatigue (83% of patients), neutropenia (79%), anemia (63%), alopecia (50%), elevated AST (50%), and constipation, nausea, and thrombocytopenia (42% each). CONCLUSION Gemcitabine-eribulin treatment response and survival for cisplatin-ineligible patients compare favorably to other regimens. Additional research is needed.

scholarly journals Early Tumor Shrinkage as a Predictive Factor for Outcomes in Hepatocellular Carcinoma Patients Treated with Lenvatinib: A Multicenter Analysis

Cancers ◽  
2020 ◽  
Vol 12 (3) ◽  
pp. 754 ◽  
Author(s):  
Aya Takahashi ◽  
Michihisa Moriguchi ◽  
Yuya Seko ◽  
Toshihide Shima ◽  
Yasuhide Mitsumoto ◽  
...  
Keyword(s):  
Hepatocellular Carcinoma ◽  
Characteristic Curve ◽  
Objective Response ◽  
Evaluation Criteria ◽  
Treatment Decision ◽  
Progression Free Survival ◽  
Tumor Shrinkage ◽  
Discrimination Ability ◽  
Early Tumor Shrinkage ◽  
Early Tumor

We investigated the association between early tumor shrinkage (ETS) and treatment outcome in patients with hepatocellular carcinoma treated with lenvatinib (LEN). A retrospective analysis was performed in 104 patients. ETS was defined as tumor shrinkage at the first evaluation in the sum of target lesions’ longest diameters from baseline according to the Response Evaluation Criteria in Solid Tumors (RECIST). The median overall survival (OS) was not reached, whereas the median progression-free survival (PFS) was 5.0 months. The receiver operating characteristic curve analysis in differentiating long-term responders (PFS ≥ 5.0 months) from short-term responders (PFS < 5.0 months) revealed an ETS cut-off value of 10%. ETS ≥ 10% was significantly correlated with better PFS and OS compared with ETS < 10%. Additionally, ETS ≥ 10% showed a better discrimination ability on prognosis compared with modified RECIST-based objective response at the first evaluation. Multivariate analysis confirmed ETS ≥ 10% as an independent predictor of better OS, as well as a Child–Pugh score of 5 and macrovascular invasion. In conclusion, ETS ≥ 10% was strongly associated with outcome in patients treated with LEN. This biomarker could allow earlier assessment of the treatment response and guide treatment decision-making for HCC.

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