Real-world comparisons of biologic treatment outcomes for ulcerative colitis (UC) or Crohn's disease (CD) patients are limited. We sought to evaluate the real-world effectiveness of vedolizumab (VDZ) ...and anti-tumor necrosis factor alpha (anti-TNFα) in UC and CD patients in Germany.
A retrospective chart review (15 sites) investigated UC and CD patients who were biologic-treatment naïve (biologic-naïve) or had received no more than one prior anti-TNFα before initiating treatment with VDZ or anti-TNFα between 15 July 2014 and 20 October 2015. Kaplan-Meier analyses assessed time to first chart-documented clinical remission (CR) and symptom resolution (UC: rectal bleeding RB, stool frequency SF; CD: abdominal pain AP, liquid stools LS) and outcome duration.
A total of 133 UC (76 VDZ; 57 anti-TNFα) and 174 CD (69 VDZ; 105 anti-TNFα) patients were included. By Week 26, estimated cumulative rates of patients achieving CR or symptom resolution with VDZ vs anti-TNFα treatment were for UC: CR, 53.7% vs 31.7%; RB, 66.8% vs 55.8%; and SF, 59.8% vs 50.7%, respectively; and for CD: CR, 14.4% vs 32.8%; AP, 62.5% vs 56.0%; and LS, 29.9% vs 50.3%, respectively. Outcomes were sustained similarly between treatments, except RB (VDZ vs anti-TNFα: median 38.1 vs 15.1 weeks, P = 0.03). Treatment-related adverse events occurred in 5.3% vs 7.0% (UC) and 8.7% vs 19.0% (CD) of VDZ vs anti-TNFα patients, respectively.
Although there were differences in CR, symptom resolution, and safety profiles, real-world data support both VDZ and anti-TNFα as effective treatment options in UC and CD.
Purpose
The purpose is to describe management of adverse events of special interest across tyrosine kinase inhibitors approved for metastatic renal cell carcinoma.
Methods
We conducted a ...retrospective chart review in metastatic renal cell carcinoma patients initiating tyrosine kinase inhibitor monotherapy between 15 November 2010 and 15 November 2013, and experiencing ≥ 1 adverse events of special interest (diarrhea, fatigue, hand-foot syndrome, hypertension, or stomatitis/mucositis) within 3 months of initiation. Demographics, medical history, treatment regimens, and adverse events of special interest management data for 3.5 months postonset were collected.
Results
In 220 charts from 27 centers, tyrosine kinase inhibitors prescribed included sunitinib (55%), pazopanib (27%), axitinib (9%), and sorafenib (8%). During the study period, patients experienced 376 adverse events of special interest (13% serious). Fatigue was most common (62% of patients), followed by hypertension (37%), diarrhea (30%), stomatitis/mucositis (29%), and hand-foot syndrome (12%). Over half (56%) the adverse events of special interest were resolved or resolving. Treatment discontinuation due to adverse events of special interest occurred in 15% of patients. Prophylaxis was rarely provided (8%), whereas 59% of patients received adverse events of special interest treatment (pharmacologic (55%) and/or nonpharmacologic (7%)), 27% received tyrosine kinase inhibitor dose management, 23% received both adverse events of special interest treatment and dose management, and 31% received neither. Hypertension was the most treated (72% of all events), and fatigue was the least treated (9%); only 4% of patients received pharmacologic treatment for fatigue.
Conclusions
Most adverse events of special interest were nonserious and more than half of the patients received pharmacologic and/or nonpharmacologic treatment. Fatigue was the most common yet least frequently treated adverse event of special interest, and few patients received prophylaxis or nonpharmacologic treatment. More emphasis on treatment and prophylaxis options for bothersome adverse events is warranted.
Introduction
Patients with type 2 diabetes mellitus (T2DM) who fail to meet glycaemic control are at increased risk of diabetes complications. For patients who cannot maintain glycaemic control with ...oral medication, one recommended option is to add an injectable glucagon-like peptide-1 receptor agonist (GLP-1 RA) to their treatment regimen. The purpose of this study was to examine time to treatment intensification with GLP-1 RAs, including the duration of time that patients did not maintain glycaemic control with oral medication.
Methods
This was a medical record review conducted in the UK via a physician survey. Patients eligible to have their records reviewed were required to be ≥ 18 years of age, have a confirmed T2DM diagnosis, and have initiated GLP-1 RA treatment for T2DM in the past 6 months. All glycated haemoglobin (HbA1c) values within 5 years prior to GLP-1 RA initiation were collected.
Results
A total of 113 physicians contributed data for 1096 patients (mean age at the time of GLP-1 RA initiation was 54.9 years, 55.4% were male, and 71.4% were White). Median time from T2DM diagnosis to GLP-1 RA initiation was 6.1 years. Median consecutive time patients taking oral regimens were not under glycaemic control (HbA1c > 7.0%) prior to GLP-1 RA initiation was 13.5 months. Patients treated by general practitioners (GPs) had a significantly longer duration of time with insufficient glycaemic control prior to GLP-1 RA initiation compared with patients treated by diabetes specialists (median time for specialists was 11.0 months vs. 17.0 months for GPs;
p =
0.038).
Conclusions
Results suggest that treatment intensification is often delayed despite consistently poor glycaemic control for more than 12 months, contrary to treatment guideline recommendations. Findings from this study highlight that some T2DM patients may benefit from more rapid treatment intensification, which could improve glycaemic control and reduce the risk for many short- and long-term health complications.
Abstract
Background and Aims
This study aimed to compare real-world clinical effectiveness and safety of vedolizumab, an α4β7-integrin inhibitor, and anti-tumour necrosis factor-α anti-TNFα agents in ...biologic-naïve ulcerative colitis UC and Crohn’s disease CD patients.
Methods
This was a 24-month retrospective medical chart study in adult UC and CD patients treated with vedolizumab or anti-TNFα in Canada, Greece and the USA. Inverse probability weighting was used to account for differences between groups. Primary outcomes were cumulative rates of clinical effectiveness clinical response, clinical remission, mucosal healing and incidence rates of serious adverse events SAEs and serious infections SIs. Secondary outcomes included cumulative rates of treatment persistence patients who did not discontinue index treatment during follow-up and dose escalation and incidence rates of disease exacerbations and disease-related surgeries. Adjusted analyses were performed using inverse probability weighting.
Results
A total of 1095 patients 604 UC, 491 CD were included. By 24 months, rates of clinical effectiveness were similar between groups, but incidence rates of SAEs (hazard ratio HR = 0.42 0.28–0.62) and SIs (HR = 0.40 0.19–0.85) were significantly lower in vedolizumab vs anti-TNFα patients. Rates of treatment persistence p < 0.01 by 24 months were higher in vedolizumab patients with UC. Incidence rates of disease exacerbations were lower in vedolizumab patients with UC (HR = 0.58 0.45–0.76). Other outcomes did not significantly differ between groups.
Conclusion
In this real-world setting, first-line biologic therapy in biologic-naïve patients with UC and CD demonstrated that vedolizumab and anti-TNFα treatments were equally effective at controlling disease symptoms, but vedolizumab has a more favourable safety profile.
Real-world data (RWD) generated during the pre-approval phase could be supplementary to primary clinical trial outcomes; however, as we discuss here, a data collection framework is needed to ensure ...the validity and applicability of these data.
This study provides real-world insight into patient profile, clinical effectiveness and health-related quality of life among patients with advanced gastric/gastroesophageal junction (GEJ) ...adenocarcinoma treated with nivolumab.
Data were collected from medical records of patients with advanced GEJ adenocarcinoma treated with nivolumab in a UK Early Access to Medicines Scheme and from the patient-reported EuroQoL five dimensions questionnaire.
Evaluable patients (n = 113; median age 62 years) were predominantly male (76.1%), White (87.4%) and with GEJ adenocarcinoma (61.9%). Median follow-up was 2.8 months. The 6-month progression-free survival and overall survival were 31.6 and 56.7%, respectively. Mean EuroQoL five dimensions questionnaire index utility scores at baseline, 8, 16 and 24 weeks were 0.795, 0.831, 0.870 and 0.793, respectively.
Progression-free survival was consistent with trial results and health-related quality of life remained stable over time.
determining whether a colonoscopy is performed for screening or nonscreening purposes can facilitate clinical practice and research. However, there is no simple method to determine the colonoscopy ...indication using patient medical files or health administrative databases.
to determine patient-endoscopist agreement on the colonoscopy indication.
a cross-sectional study was conducted among staff endoscopists and their patients at seven university-affiliated hospitals in Montreal, Quebec. The study participants were 50 to 75 years of age, they were able to understand English or French, and were about to undergo colonoscopy. Self- (endoscopist) and interviewer-administered (patient) questionnaires ascertained information that permitted classification of the colonoscopy indication. Patient colonoscopy indication was defined as the following: perceived screening (routine screening, family history, age); perceived nonscreening (follow-up); medical history that implied nonscreening; and a combination of the three preceding indications. Agreement between patient and endoscopist indications was measured using concordance and Kappa statistic.
in total, 702 patients and 38 endoscopists participated. The three most common reasons for undergoing colonoscopy were routine screening⁄regular check-up (33.8%), follow-up to a previous problem (30.2%) and other problem (24.6%). Concordance (range 0.79 to 0.85) and Kappa (range 0.58 to 0.70) were highest for perceived nonscreening colonoscopy. Recent large bowel symptoms accounted for 120 occurrences of disagreement in which the patient perceived a nonscreening colonoscopy while the endoscopist perceived a screening colonoscopy.
patient self-report may be an acceptable means for rapidly assessing whether a colonoscopy is performed for screening or nonscreening purposes. Delivery of patient-centred care may help patients and endoscopists reach a shared understanding of the reason for colonoscopy.
In the UK, there are limited data sources for evaluating real-world research questions related to oncology therapy. We conducted a pilot study to investigate the feasibility of extracting data ...directly from a chemotherapy prescription platform (ChemoCare
) utilized in standard care.
Concordance was compared with data extracted manually for patients with advanced melanoma as part of a concurrent chart review (gold-standard) using Cohen's kappa and the intraclass correlation coefficient.
There was high concordance between data automatically extracted from the prescription platform and chart review data.
This pilot can be used as a framework for future studies using direct, automated extraction from prescription platforms.
In clinical trials, dabrafenib plus trametinib improved overall survival (OS) compared with single-agent BRAF inhibitors (BRAFi) in patients with BRAF V600-mutant unresectable or metastatic melanoma. ...We investigated dabrafenib plus trametinib therapy in a compassionate-use setting Named Patient Program (NPP); DESCRIBE II. A retrospective chart review of patients with BRAF V600-mutated unresectable stage III/IV melanoma receiving dabrafenib plus trametinib as compassionate use was conducted. Treatment patterns and duration, clinical outcomes, and tolerability were evaluated. Of 271 patients, 92.6% had stage IV melanoma, including 36.5% with brain metastases. Overall, 162 patients (59.8%) were BRAFi naive and 171 (63.1%) received first-line dabrafenib plus trametinib. Among BRAFi-naive patients, the overall response rate (ORR) was 67.3%, median OS (mOS) was 20.0 months, and median progression-free survival (mPFS) was 7.5 months. In BRAFi-naive patients with known brain metastases (n = 62), ORR was 61.3%, mOS was 15.5 months, and mPFS was 6.2 months. Eighty-four patients received BRAFi monotherapy for >30 days and switched to dabrafenib plus trametinib prior to progression. Of these 84 patients, 63 had known disease status at the time of switch, and 22 improved with the combination therapy. No new safety signals were identified, and dabrafenib plus trametinib was well tolerated. Dabrafenib plus trametinib showed substantial clinical activity in NPP patients with BRAF V600-mutated unresectable or metastatic melanoma. Analysis of treatment patterns demonstrated the effectiveness of the combination in patients with brain metastases and across lines of therapy with a well tolerated and manageable safety profile.
A named patient program (NPP) was designed to provide patients with advanced soft-tissue sarcoma (aSTS) access to pazopanib, a multitargeted tyrosine kinase inhibitor. The SPIRE study was a ...retrospective chart review of participating patients.
Eligibility criteria for the NPP and SPIRE mirrored those of the pivotal phase-III study, PALETTE, which compared pazopanib with placebo in patients ≥18 years with aSTS and whose disease had progressed during or following prior chemotherapy or were otherwise unsuitable for chemotherapy. Outcomes of interest included treatment patterns, treatment duration, relative dose intensity, progression-free survival (PFS), overall survival (OS), clinical benefit rate, adverse events (AEs) and reasons for treatment discontinuation.
A total of 211 patients were enrolled (median age 56 years; 60% female). Most patients received pazopanib in second- and third-line therapy (28.0% and 28.4%, respectively), followed by fourth line (19.0%) and ≥ fifth line (18.5%). The median duration of pazopanib treatment was 3.1 months (95% CI: 2.8-3.8), with a mean daily dose of 715 mg equating to 92% of recommended dose. Median OS was 11.1 months and clinical benefit rate was 46%. There was evidence of some clinical benefit across most histological subtypes. At study end, 40% of patients were alive and of these, 18% remained on pazopanib. Thirteen percent (13%) of patients discontinued pazopanib due to AEs.
The SPIRE study demonstrated activity of pazopanib in heavily pretreated aSTS patients in a compassionate use setting. No new safety concerns were noted. Reassuringly, the relative dose intensity of pazopanib was 92%.
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