Summary Background Hepatorenal syndrome and acute kidney injury are common complications of decompensated cirrhosis, and terlipressin is recommended as first‐line vasoconstrictor therapy. However, data on its use outside of clinical trials are lacking. Aims To assess practice patterns and outcomes around vasoconstrictor use for hepatorenal syndrome in UK hospitals. Methods This was a multicentre chart review study. Data were extracted from medical records of patients diagnosed with hepatorenal syndrome and treated by vasoconstrictor drugs between January 2013 and December 2017 at 26 hospitals in the United Kingdom. The primary outcome was improvement of kidney function, defined as complete response (serum creatinine improved to ≤1.5 mg/dL), partial response (serum creatinine reduction of ≥20% but >1.5 mg/dL) and overall response (complete or partial response). Other outcomes included need for dialysis, mortality, liver transplantation and adverse events. Results Of the 225 patients included in the analysis, 203 (90%) were treated with terlipressin (median duration, 6 days; range: 2‐24 days). Mean (±standard deviation) serum creatinine at vasopressor initiation was 3.25 ± 1.64 mg/dL. Terlipressin overall response rate was 73%. Overall response was higher in patients with mild acute kidney injury (baseline serum creatinine <2.25 mg/dL), compared to those with moderate (serum creatinine ≥2.25 mg/dL and <3.5 mg/dL) or severe (serum creatinine ≥3.5 mg/dL). Ninety‐day survival was 86% for all patients (93% for overall responders vs 66% for treatment nonresponders, P < 0.0001). Conclusion Terlipressin is the most commonly prescribed vasoconstrictor for patients with hepatorenal syndrome in the United Kingdom. Treatment with terlipressin in patients with less severe acute kidney injury (serum creatinine <2.25 mg/dL) was associated with higher treatment responses, and 90‐day survival.
The 21-gene test is a validated multi-gene diagnostic test that predicts chemotherapy (CT) benefit in oestrogen receptor positive (ER+), lymph node-negative (N0) breast cancer (BC) patients (pts). Ireland was the first public health care system to reimburse this test in Europe. Study objectives were to assess the impact of this test on decision-making and to analyse the economic impact of testing. Between October 2011 and February 2013, a national, retrospective, cross-sectional observational study of ER+, N0 BC pts tested with the 21-gene test was conducted. Surveyed breast medical oncologists, provided the assumption for the decision impact analysis that grade (G) 1 pts would not have received CT before testing and G2/3 pts would have received CT before testing. Descriptive statistical analyses were performed. 592 pts were identified; Low, intermediate and high recurrence score were identified in 53, 36 and 10 % pts, respectively. 384 (70 %) pts had G2, 129 (22 %) G3 and 76 (13 %) G1 tumours. Post testing, 345 pts (59 %) experienced a change in CT decision; 339 changed to hormone therapy alone and 6 advised to receive CT. 172 (30 %) pts received CT, 12 (3.9 %) of pts with low scores, 108 (50.9 %) of intermediate risk and 50 (90.9 %) of pts with high risk scores. Net reduction in CT use was 58 % and net savings achieved were €793,565. Since public reimbursement, the introduction of the 21-gene test has resulted in a significant reduction in chemotherapy administration and cost savings for the Irish public healthcare system.
Objectives The treatment paradigm in newly diagnosed multiple myeloma (NDMM) is evolving toward individualized, risk‐directed, and longer duration of therapy (DOT). The objective of this study was to describe treatment patterns and outcomes in non‐transplant NDMM in four European countries. Methods This retrospective chart review included adults with NDMM diagnosed between January 1, 2012, and December 31, 2013 (early cohort), or April 1, 2016, and March 31, 2017 (recent cohort). Results Among 836 patients, molecular testing was performed in 21% and 35% patients of early vs recent cohorts; proteasome inhibitor (PI)/alkylator combinations were the principal first‐line (1 L) therapy (39% vs 43%). Use of immunomodulatory drug (IMID)/alkylator combinations declined from early to recent cohort (26% vs 13%) but IMID (7% vs 16%) use increased. Few patients (5%) received 1 L maintenance therapy. Two‐thirds of patients were treated with a fixed duration intent, with a median 7‐month 1 L DOT and progression‐free survival (PFS) of 32.8 months in the early cohort. Both 1 L DOT and PFS were longer with oral compared to injectable regimens. Conclusions Although frontline treatment patterns changed significantly, 1 L DOT is short. The uptake of molecular testing and 1 L maintenance is low. These results highlight areas of unmet need in NDMM.
Purpose: Advanced non-small-cell lung cancer (aNSCLC; stage IIIB/IV) presents a substantial clinical burden to society; reliable estimates of its economic burden are lacking. Therefore, this study aimed to quantify real-world health care resource utilization (HCRU) and costs of patients with squamous (SQ) and non-SQ (NSQ) aNSCLC who received two or more lines of treatment (2L+) in Europe, and to describe cost-predictors. Methods: The LENS (Leading the Evaluation of Non-squamous and Squamous NSCLC) retrospective chart review study collected data from 2L+ patients with aNSCLC diagnosed between 07/2009 and 08/2011 (wave 1) or 07/2010 and 09/2012 (wave 2) in France, Germany, Italy, Spain, England, the Netherlands, and Sweden. Patients were followed from diagnosis through most recent visit/death. A weighted average of countryspecific unit costs (2018 Euro) was applied to systemic anti-cancer therapy usage and HCRU (hospital/emergency department visit, surgery, radiotherapy, ancillary care, biomarker testing) to determine the total cost from aNSCLC diagnosis to death. Generalized linear models (gamma distribution, log link) were used to assess clinical and demographic predictors. Results: Of 973 2L+ aNSCLC patients, median overall survival (OS) was 1.5 years from advanced diagnosis (range: 0.2-5.3; median OS: 1.4 [SQ], 1.6 [NSQ]), 79.0% died during follow-up. Weighted mean total per-patient costs were €21,273, ranging from €17,761 (England) to €30,854 (Sweden), and €15,446 (SQ) to €26,477 (NSQ). Systemic drug costs comprised 77.4% of total costs. Insurance status, presence of epidermal growth factor receptor (EGFR) mutation, SQ histology, age, alcohol abuse, and year of diagnosis were significant predictors for lower total costs per patient-month, Eastern Cooperative Oncology Group performance status (ECOG PS) ≥1 and country for higher costs. Conclusion: In the era pre-immunotherapy, HCRU and costs were substantial in aNSCLC 2L+ patients, with most of the costs accrued prior to start of 2L. NSQ patients incurred significantly higher total costs than SQ patients in all participating countries.
Objectives: To evaluate treatment sequences and associated costs among patients with relapsed/refractory multiple myeloma (RRMM). MethOds: Patients with RRMM between January 2007 and September 2013 were identified from US MarketScan databases. Outcomes included treatment regimen per line (L; 2-4L), sequences of treatment regimens, and cost per line and of progression (2L vs > 2L). All-cause and MM-specific monthly costs captured inpatient, outpatient, emergency department, and drug-related costs, adjusted for censoring. Results: 4449 MM patients initiated a 2L regimen, of whom 38% (n= 1696) progressed to 3L and 15% (n= 689) progressed to 4L. Median follow-up was 14 months (range 1-83). The most frequent 2L regimens were lenalidomide (18%), lenalidomide-dexamethasone (12%), bortezomib-dexamethasone (12%), bortezomib-lenalidomide-dexamethasone (10%), and bortezomib (7%). Of patients whose prior therapy included an immunomodulatory drug (IMiD) (n= 1752), bortezomib-dexamethasone was the most common 2L regimen (17%); for those with no prior IMiD exposure (n= 2697), lenalidomide was the most common 2L (23%). Median time to start of 3L was 5.8 months. Among the 27% of lenalidomide 2L patients who progressed to 3L, 64% had bortezomib-based regimens in 3L. Among the 47% of bortezomib-dexamethasone 2L patients who progressed to 3L, 63% had lenalidomide-based regimens in 3L. MM-specific costs were highest for all patients during the first year of 2L: $122,960 versus $74,573 (12-24 mths), $68,940 (24-36 mths), $59,327 (36+ mths). Drug-related costs accounted for 39-62%, and were dependent on regimen and treatment line. During 0-36 mths, MM-specific costs after progression from 2L were higher by $122,823 compared with costs incurred on 2L. cOnclusiOns: In this study of RRMM patients, 38% progressed to subsequent treatment lines, with variability in 2L regimens and treatment sequences. Highest costs were incurred during the first 12 months of follow-up. The cost of managing progressive disease in RRMM is high, as observed by the difference in costs before and after progression. PCN119 TreaTmeNT STraTegieS, healTh Care reSourCe uSe aNd CoSTS of aggreSSive hiSTologiCal TyPeS of NoN-hodgkiN lymPhomaS iN The Slovak rePubliC. reSulTS from The CroSS-SeCTioNal Survey iN The haemaTology-oNCologiCal CeNTerS
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