Real-world outcomes of different lines and sequences of treatment in BRAF-positive advanced melanoma patients.

The objective of this study is to compare efficacy with different treatment sequences and lines of treatment among BRAF V600 mutated (BRAF+) advanced melanoma patients with immunotherapies (IO) and targeted therapies (TT) using real-world data. This was a retrospective cohort study using the Novartis BRAF+ meLanoma patients ObsErvational database, the harmonized customized data from Flatiron and ConcertAI. The study included BRAF+ advanced unresectable melanoma patients treated with first-line (1L) IO or TT between 1 January 2014 and 31 May 2020. Patient characteristics and treatment patterns were described. Kaplan-Meier curves and propensity score-adjusted Cox models were used for analyzing progression-free survival (PFS) and overall survival (OS). A total of 1961 patients were included, of which, 57.2% received IO and 42.8% received TT on 1L therapy. Overall, 603 patients initiated a 2L therapy: 56.2% IO and 43.8% TT. Regardless of treatment sequence, patients progressed at a relatively similar rate with no significant difference between groups (median PFS: 12.9 months for 1L TT/2L IO and 13.1 months for 1L IO/2L TT; HR, 0.84; P = 0.137). The 2-year OS rate was also similar with 1L TT/2L IO and 1L IO/2L TT (78% vs. 80%; HR, 1.09; P = 0.730). PFS was worse on 2L therapy compared with 1L (median 4.7 vs. 6.5 months). Efficacy on 2L therapy was poor compared with 1L. Among patients who received 2L therapy, regardless of treatment sequences, outcomes were comparable between 1L TT/2L IO and 1L IO/2L TT in this study that reflects real-world experiences beyond clinical trial selective eligibility criteria.

Risk tolerance in adjuvant and metastatic melanoma settings: a patient perspective study using the threshold technique.

Background: Adverse events (e.g., pyrexia) may affect treatment patterns and adherence. This study explored pyrexia risk tolerance among melanoma patients when treatment benefit is unknown versus known. Materials & methods: US respondents with stage III (n = 100) or stage III unresectable/stage IV melanoma (n = 125) chose between hypothetical melanoma treatments, defined by reoccurrence/progression-free survival and pyrexia risk, one resembling standard-of-care and one resembling dabrafenib + trametinib. Respondents chose first when efficacy was unknown and then when efficacy was known; pyrexia risk was varied systematically to define maximum acceptable risk. Results: Maximum acceptable risk of pyrexia was statistically significantly higher when efficacy was known versus unknown in stage III patients (85 vs 34%) and stage III unresectable/stage IV patients (66 vs 57%). Conclusion: Patients accepted higher levels of pyrexia risk when they understood treatment benefit.

Survival, costs, and health care resource use by line of therapy in US Medicare patients with newly diagnosed glioblastoma: a retrospective observational study.

BACKGROUND: Glioblastoma (GBM) is associated with poor prognosis, large morbidity burden, and limited treatment options. This analysis evaluated real-world treatment patterns, overall survival, resource use, and costs among Medicare patients with GBM. METHODS: This retrospective observational study evaluated Medicare patients age 66 years or older with newly diagnosed GBM using the Surveillance, Epidemiology, and End Results (SEER)-Medicare linked data from 2007 through 2013. Patients were followed from diagnosis to death or end of follow-up. An algorithm defined treatment patterns as lines of therapy (LOTs). The Kaplan-Meier method was used to estimate overall survival for the full sample as well as by LOT, surgical resection, Charlson Comorbidity Index (CCI), tumor size, and age. Resource use and costs during the follow-up period were reported in terms of total and per-patient-per-month (PPPM) estimates. RESULTS: A total of 4308 patients with GBM were identified (median age, 74 years; CCI of 0, 52%). The most commonly used first LOT was temozolomide (82%), whereas chemotherapy + bevacizumab was most prevalent for second-line (42%) and third-line (58%) therapy. The median overall survival was 5.9 months for resected patients and 3 months for unresected patients, with considerable heterogeneity depending on patient characteristics. A great proportion of patients had claims for an ICU admission (86.2%), skilled nursing facility (76.9%), and home health (56.0%) in the postdiagnosis period. The cumulative mean cost was $95 377 per patient and $18 053 PPPM, mostly attributed to hospitalizations. CONCLUSIONS: Limited treatment options, poor survival, and economic burden emphasize the need for novel interventions to improve care for Medicare patients with GBM.

Real-world use of extracorporeal photopheresis for patients with cutaneous T-cell lymphoma in the United States: 2010-2015.

Introduction: The National Comprehensive Cancer Network and the European Organization for Research and Treatment of Cancer recommend extracorporeal photopheresis (ECP) as systemic therapy for cutaneous T-cell lymphoma (CTCL).Objective: To investigate real-world use of ECP in CTCL patients in the US.Methods: Data from the Truven MarketScan® database (2010-2015) were used to create a cohort of CTCL patients receiving systemic treatment. Multivariable regressions were performed to compare health care resource utilization between ECP and propensity score-matched non-ECP patients.Results: Of the 1106 eligible patients, 117 (10.6%) received ECP, with an average treatment duration of 13.6 months. Psoriasis, organ transplant, graft versus host disease, and scleroderma were the most common comorbidities. ECP was used as monotherapy in 76 patients (65.0%) and combination in 41 patients (35.0%), mostly with interferon and/or a retinoid. Higher Charlson Comorbidity Index (2.6 vs 2.2, p < .05), rates of organ transplant (49.6% vs 7.8%, p < .001), and graft vs host disease (41.9% vs 3.4%, p < .001) were observed in ECP versus non-ECP patients. Post-matching analyses showed that ECP patients had shorter all-cause inpatient stay (6.67 vs 11.80 days, p = .001).Conclusions: Approximately 1 out of 10 CTCL patients receiving systemic treatment were on ECP treatment in the US. Post-matching analysis showed ECP was associated with a shorter hospital stay.

Psychometric Evaluation of the Chinese Breast Cancer Prevention Trial Symptom Scale.

BACKGROUND: Women with breast cancer experience a wide spectrum of symptoms after diagnosis and treatment. Symptoms experienced by this specific population might not be fully assessed using available traditional Chinese-language symptom measures. OBJECTIVES: The aim of this study was to examine the latent constructs and psychometric properties of the Chinese Breast Cancer Prevention Trial (C-BCPT) Symptom Scale. METHODS: Two hundred women with breast cancer were recruited in Taiwan. Psychometric properties, including construct validity, internal consistency, and test-retest reliability, of the C-BCPT Symptom Scale were tested after translating the original instrument. RESULTS: A 21-item C-BCPT Symptom Scale, with 7 extracted factors accounting for 72.26% of the total variance, resulted from an exploratory factor analysis. Construct validity was confirmed by significant correlations between scores on the C-BCPT Symptom Scale and the Taiwan-version Short Form-36 Health Survey (r = -0.49 to -0.53)/Greene Climacteric Scale (r = 0.81). Reliability coefficients for the overall scale/6 extracted factors (Cronbach's α = 0.72-0.88) and test-retest reliability (intraclass correlation coefficients = 0.77-0.94) of the translated instrument were satisfactory, whereas 1 reliability coefficient for 1 extracted factor was inadequate (Cronbach's α = 0.57). CONCLUSION: An interpretable structure with preliminary acceptable psychometric properties of the C-BCPT Symptom Scale was obtained; the C-BCPT can help traditional Chinese-speaking healthcare professionals perform adequate assessments of the symptoms experienced by women with breast cancer. IMPLICATIONS FOR PRACTICE: The C-BCPT Symptom Scale can be used in clinical practice and research to assess symptoms experienced by this specific population or effects of related interventions.

Direct and indirect costs among patients with binge-eating disorder in the United States.

OBJECTIVE: To quantify the economic burden of binge-eating disorder (BED) in terms of work productivity loss, healthcare resource utilization, and healthcare costs. METHODS: Respondents of the US National Health and Wellness Survey 2013 were invited to participate in a follow-up internet survey to identify adults with BED using DSM-5 criteria. Work productivity loss, healthcare resource utilization, and direct and indirect costs were assessed for BED respondents and matched non-BED respondents using generalized linear models or two-part models as appropriate. RESULTS: A total of 1,720 people were included in our analysis (N = 344 with BED; N= 1,376 without BED). BED respondents had higher levels of activity impairment than non-BED respondents (41.29% vs. 23.18%, p < .001). Employed BED respondents (N = 178) had a greater level of work impairment than employed non-BED respondents (N = 686) (36.83% vs. 14.41%, p = .009). Higher healthcare resource utilization in the past 6 months among BED respondents was reported than matched non-BED respondents: numbers of surgeries (0.23 vs. 0.13, p = .021), ER visits (0.26 vs. 0.15, p = .016), and physician visits (6.09 vs. 4.56, p = .002). BED respondents reported higher total direct costs than matched non-BED respondents ($20,194 vs. $14,465, p = .005). The indirect costs among employed BED respondents were also higher than those without BED ($19,327 vs. $9,032, p < .001). DISCUSSION: Individuals with BED reported significantly greater economic burden with respect to work productivity loss, level of healthcare resource utilization, and costs compared to non-BED respondents. © 2016 Wiley Periodicals, Inc.(Int J Eat Disord 2017; 50:523-532).

Treatment Patterns of Anti-Vascular Endothelial Growth Factor and Laser Therapy Among Patients with Diabetic Macular Edema.

BACKGROUND: Diabetic macular edema (DME) is a form of diabetic retinopathy caused by continued leakage from retinal blood vessels. The use of anti-vascular endothelial growth factor (VEGF) injections has gained in popularity in the treatment of DME due to satisfactory efficacy, while laser photocoagulation is still the first-line therapy. Examining anti-VEGF treatment patterns may improve understanding of real-world medication-taking behaviors.   OBJECTIVES: To (a) compare demographic and clinical characteristics and treatment patterns of anti-VEGF (bevacizumab, ranibizumab, and pegaptanib) and laser therapies among DME patients and (b) determine predictors of switching and anti-VEGF therapy initiation.  METHODS: A retrospective cohort analysis was conducted with Texas Medicaid medical and prescription claims (January 1, 2008-December 31, 2012) for patients who were aged 18-63 years, continuously enrolled 1 year pre- and post-index, diagnosed with DME and treated with anti-VEGF or laser therapies. Treatment patterns included treatment frequency and switching between anti-VEGF and laser therapies. Logistic regression and multinomial analysis were used to determine factors associated with switching and initiation of anti-VEGF therapy, while controlling for demographic and clinical characteristics. RESULTS: DME patients (N = 2,201) were aged 54.7 (SD ± 7.9) years; 63.1% were female; 59.1% were Hispanic; and 10.3% were visually impaired. CCI mean score was 6.5 (SD ± 3.1), and patients were on 2.6 (SD ± 3.3) unique prescription medications. Anti-VEGF users had significantly (P less than 0.0001) fewer prescriptions compared with laser users (1.9 [SD ± 3.1) vs. 2.8 [SD ± 3.3], respectively). Laser was the most commonly used (84.9%) therapy from 2009 to 2011; however, anti-VEGF use increased from 11.7% in 2009 to 21.8% in 2011 (P less than 0.0001). Patients received 1.5 (SD ± 0.7) laser surgeries compared with 1.7 (SD ± 1.1) anti-VEGF injections per eye annually. Switching from laser to anti-VEGF injections was 9.7%, while switching from anti-VEGF injections to laser surgery was 42.2%. Patients who switched from anti-VEGF injections to laser surgery were more likely to be Hispanic (OR = 1.415, 95% CI = 1.037-1.930); male (OR = 1.341, 95% CI = 1.053-1.709); have fewer prescriptions (OR = 0.944, 95% CI = 0.905-0.985); and less likely to have no visual impairment (OR = 0.641, 95% CI = 0.449-0.915). Multinomial regression results showed anti-VEGF users were more likely to remain on the same therapy if they had more prescriptions (OR = 1.094, 95% CI = 1.029-1.172) or were female (OR = 1.441, 95% CI = 1.024-2.041). Anti-VEGF initiators had fewer prescriptions (OR = 0.917, 95% CI = 0.868-0.947) and initiated in 2011 vs. 2009 (OR = 2.363, 95% CI = 1.777-3.141).    CONCLUSIONS: Although anti-VEGF use is increasing, laser use is still more prevalent. Over 40% of patients who initiated on anti-VEGF injections switched to laser surgery. Additional research should be conducted to determine factors associated with this high rate of switching.