scholarly journals Real-world results of definitive chemoradiation in carcinoma oesophagus: can SCOPE1 results be replicated outside trial setting?

2021 ◽  
Vol 15 ◽  
Author(s):  
Tapesh Bhattacharyya ◽  
Vishnu Harilal ◽  
Rohit Sashidharan ◽  
Indranil Mallick ◽  
Moses Arunsingh ◽  
...  
Keyword(s):  
Real World ◽  
Definitive Chemoradiation ◽  
Carcinoma Oesophagus ◽  
Trial Setting

scholarly journals Real-World Use of Enasidenib in Relapsed or Refractory Acute Myeloid Leukemia Is Associated with Reduced Risk of Disease Progression and Death

Blood ◽  
2020 ◽  
Vol 136 (Supplement 1) ◽  
pp. 18-18
Author(s):  
Andrew J Klink ◽  
Ronda Copher ◽  
Russell L. Knoth ◽  
Landon Marshall ◽  
Ying Hou ◽  
...  
Keyword(s):  
Acute Myeloid Leukemia ◽  
Cohort Study ◽  
Real World ◽  
Myeloid Leukemia ◽  
Patient Characteristics ◽  
Response To Therapy ◽  
Phase 3 ◽  
Ed Visits ◽  
Trial Setting ◽  
Acute Myeloid

Introduction: Since 2017, several new therapies were approved by the United States (US) Food & Drug Administration (FDA) for the treatment of acute myeloid leukemia (AML), many of which target specific gene mutations. Enasidenib, a once-daily oral tablet, was approved by the FDA in August 2017 for patients with relapsed or refractory (RR) AML with an isocitrate dehydrogenase-2 (IDH2) mutation, which occurs in 8-19% of patients with AML. While efficacy was established in a phase 3 trial that demonstrated a complete remission (CR)/CR with partial hematologic recovery (CRh) rate of 23% and a median remission duration of 8.2 months, data outside the trial setting are lacking. This study aimed to assess real-world outcomes (i.e. response to therapy, survival, and rates of emergency department [ED] visits and hospitalizations) among a large cohort of patients with RR AML with IDH2 mutations treated with enasidenib, or other systemic therapies, in the first-line (1L) RR setting. Methods: A retrospective, observational cohort study of patients with RR AML and IDH2 mutations treated with enasidenib between January 2018 and June 2019, or with another approved RR AML therapy (including cytotoxic chemotherapy, hypomethylating agent, targeted therapy, e.g. venetoclax) between January 2016 and July 2017 as 1L RR therapy, was conducted. Data were collected from patient medical records abstracted by 20 community oncologists across all geographic areas in the USA (30% Northeast, 20% Midwest, 25% South, 25% West). Patient characteristics, treatment patterns, physician-reported response to 1L RR therapy, ED visits, hospitalizations, and date of death were collected. Descriptive statistics were used to summarize patient characteristics and outcomes, and univariate comparisons were made by chi-square and t-tests. Median (95% confidence interval [CI]) progression-free survival (PFS) and overall survival (OS) from 1L RR therapy initiation were estimated by the Kaplan-Meier method, and adjusted risk (hazard ratio [HR] and 95% CI) of progression and death were estimated by multivariable Cox proportional hazard models to control for potential confounders. Results: Of the 124 patients treated with enasidenib and 76 control patients treated with another (non-enasidenib) 1L RR therapy for RR IDH2-mutated AML, 52% and 55% were male (P = 0.62), and median age at 1L RR therapy was 68 and 63 years (P = 0.04), respectively (Table). The proportion of patients with European Cooperative Oncology Group performance status (ECOG PS) 2+ (52% vs 53%) and poor cytogenetic risk (29% vs 29%) were similar among enasidenib and other 1L RR therapy patients, respectively (both P > 0.05). Approximately 23% of 1L RR enasidenib patients were refractory to induction therapy versus 40% of other 1L RR therapy patients (P = 0.02). CR/partial response/morphologic leukemia-free state rate was higher among patients treated with enasidenib versus other 1L RR therapies (77% vs 52%; P < 0.01). After a median follow-up of 9 and 6 months from 1L RR treatment among enasidenib and other 1L RR therapy patients, respectively, median PFS was 8.4 and 4.8 months (adjusted HR = 0.36, 95% CI 0.23-0.57; P < 0.01), and median OS was 11.0 and 6.4 months (adjusted HR = 0.37, 95% CI 0.22-0.60; P < 0.01), respectively (Figure). While rates of ED visits were similar across treatment groups (22% 1L RR enasidenib vs 18% other 1L RR therapy, P = 0.59), fewer 1L RR enasidenib patients were hospitalized during 1L RR therapy versus other 1L RR therapies (14% vs 46%, P < 0.01). Conclusions: Results from this large real-world cohort study confirm the effectiveness of 1L RR enasidenib among patients with IDH2-mutated AML outside the trial setting and are similar to the pivotal phase 3 trial results. Superior response rates, PFS, and OS were observed in enasidenib-treated patients compared with other commonly used 1L RR therapies. Additionally, rates of hospitalizations were significantly lower among those treated with 1L RR enasidenib versus other 1L RR therapies. Further research is warranted to understand how and when physicians test for mutations and apply targeted therapies and how practicing physicians assess response to contemporary RR AML therapies in real-world clinical practice. Disclosures Klink: Cardinal Health: Current Employment, Current equity holder in publicly-traded company. Copher:Bristol Myers Squibb: Current Employment. Knoth:Bristol Myers Squibb: Current Employment. Marshall:Cardinal Health: Current Employment. Hou:Cardinal Health: Current Employment. Gajra:Cardinal Health: Current Employment. Tibes:Celgene/BMS: Consultancy, Honoraria.

scholarly journals Eltrombopag in the management of aplastic anaemia: real-world experience in a non-trial setting

Hematology ◽  
2018 ◽  
Vol 23 (7) ◽  
pp. 399-404 ◽  
Author(s):  
Yu-Yan Hwang ◽  
Harinder Gill ◽  
Thomas S.Y. Chan ◽  
Garret M.K. Leung ◽  
Carol Y.M. Cheung ◽  
...  
Keyword(s):  
Aplastic Anaemia ◽  
Real World ◽  
Trial Setting

Persistence of ibrutinib-associated hypertension in CLL pts treated in a real-world experience.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. 7525-7525 ◽  
Author(s):  
Lisa M. Gashonia ◽  
Joseph R. Carver ◽  
Rupal O'Quinn ◽  
Suparna Clasen ◽  
Mitchell E. Hughes ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Real World ◽  
Retrospective Cohort ◽  
Management Strategies ◽  
Clinical Factors ◽  
Time Points ◽  
World Series ◽  
Aggressive Management ◽  
Trial Setting

7525 Background: Cardiovascular (CV) complications associated with ibrutinib (Ibr) include hypertension (HTN) and atrial fibrillation (AFIB) (incidence 26% and 9%, OBrien, ASH 2016). Unlike clinical trials, Ibr toxicities are the most common reasons for its discontinuation in clinical practice. The incidence of HTN in pts treated with Ibr outside of clinical trial setting and its impact on outcomes is unknown. Methods: Retrospective, cohort study of Ibr-treated CLL pts to estimate HTN incidence. Baseline CLL characteristics and co-morbidities were recorded. Blood pressure (BP) measurements were recorded prior to Ibr and sequentially following exposure at specific time points. CV meds were reviewed during a 12 mo follow-up period. The association between Ibr exposure and BP was tested. Results: 153 consecutive CLL pts treated with Ibr at a dose of 420 mg/day were identified. Med age was 57 yr (range: 34-87), relapsed CLL (69%), follow-up 14.5 mo. CV pre-Ibr characteristics included: smoking hx (49%), HTN (42%), hyperlipidemia (39%), diabetes (17%), CAD (12%), AFIB (6.8%). Proportion of pts on ≥1 anti-HTN med increased from 44% pre-Ibr (20% ≥ 2) to 57% during Ibr (30% ≥ 2). Med pre-Ibr BP was 127/70 mmHg (range 90-182/48-95mmHg). At 1, 3, 6, 9, 12 mo, med BPs were 137/73, 141/75, 143/76, 140/75, 142/77 (7 mo to peak BP). There was a significant association between Ibr exposure and increased BP (p<.01). New HTN was observed in 40% of pts and 36% HTN pts had BP increased above baseline (med baseline 135/70 vs peak 161/80). Incidence of new AFIB was 8.1%. In UV analyses, predictive clinical factors for HTN were not identified. Pre-Ibr HTN (OR 3.0, p .05), CAD (OR 4.3, p .03), prior AFIB event (OR 10.8, p.001), hyperlipidemia (OR 3.4, p.05) were associated with post-Ibr AFIB. Conclusions: In the largest real-world series focused on BP in Ibr treated pts, we demonstrate a clear association between Ibr and HTN. Nearly 40% of pts developed HTN within 12 mo of Ibr exposure (vs. 26% in clinical trials over 5 yr). Despite aggressive management (multiple agents), Ibr associated HTN was persistent. These data underscore the critical need for monitoring and management strategies for HTN and follow-up data on future CV events.

Regorafinib outcomes from the population-based South Australian mCRC registry (SAmCRCR).

2020 ◽  
Vol 38 (15_suppl) ◽  
pp. e19344-e19344
Author(s):  
Timothy Jay Price ◽  
Cynthia Piantadosi ◽  
Amanda Rose Townsend ◽  
Rob Padbury ◽  
Amitesh Chandra Roy ◽  
...  
Keyword(s):  
Median Survival ◽  
Real World ◽  
Potential Benefit ◽  
Randomized Trials ◽  
Stage Iv ◽  
Population Based ◽  
Kaplan Meier ◽  
Trial Setting

e19344 Background: Regorafinib has been shown to improve survival over BSC alone in large randomized trials. The median survival in the CORRECT study was 6.4 months and 12 month survival 24.3%. In Australia regulators have limited the use of regorafenib and patients are required to self-fund. Understanding the potential benefit of regorafenib in a real world non trial setting would be informative for consumers when considering self funding. Methods: The SAmCRCR collects all mCRC patients prospectively. The Registry was analysed for patients who had received regorafinib between February 2006 and December 2018. Survival was analysed using Kaplan Meier method. Results: Only 53 patients have received regorafenib therapy since February 2006. The median age was 66 (range 34-82). 66% were male, 66% had stage IV at diagnosis, 53% had liver only, 13% liver and lung and 6% lung only involvement. 75% were left sided. K/RAS was available in 35/53 patients, 49% WT. BRAF in 8/53, 25% MT. MSI measured in 14, and MSI-H 21%. Prior lines of treatment received: one 4%, two 9%, three 23%, four 26%, > four 37%. Prior biological use: bevacizumab 72%, anti-EGFR 100% (for RAS WT). Median survival from diagnosis was 3.3 years (95% CI 2.8-3.8 years). From start of regorafinib median survival was 7.1 months (95% CI 4.8-9.4 months) and12 month survival 28%. Conclusions: The outcome for those patients who do access regorafinib is in keeping with that reported from large randomized trials and thus clinicians can quote these outcomes when discussing access to regorafinib in the community.

Real-world analysis of prostate-specific antigen (PSA) outcomes among patients with metastatic castration-resistant prostate cancer (mCRPC) treated with enzalutamide (ENZA).

2020 ◽  
Vol 38 (6_suppl) ◽  
pp. 33-33
Author(s):  
Stephen J. Freedland ◽  
Neil M. Schultz ◽  
Anna D. Coutinho ◽  
Rupali Fuldeore ◽  
Nancy Hedlund ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Clinical Trial ◽  
Median Time ◽  
Real World ◽  
Index Date ◽  
Clinical Trial Setting ◽  
Psa Response ◽  
Psa Progression ◽  
Trial Setting ◽  
Baseline Psa

33 Background: ENZA showed efficacy in chemotherapy-naïve men with mCRPC in a clinical trial setting (PREVAIL). We report real-world outcomes with ENZA for this population in a urology practice setting. Methods: This retrospective cohort study included men with prostate cancer newly initiating ENZA in the IntrinsiQ Specialty Solutions™ urology electronic medical records database between September 1, 2014 and February 28, 2018. Due to the approved indication in the study time frame, prescription of ENZA (first claim date = index date) was used as a proxy for mCRPC. Patients with evidence of prior chemotherapy and/or abiraterone were excluded. PSA value closest to index date (±30 days) was used as baseline. Men were followed until the earliest of: discontinuing ENZA, leaving practice, death, or study end. Best PSA response (largest decline or smallest increase in absence of decline from baseline), PSA declines of ≥50% and ≥90%, undetectable PSA, and time to PSA progression were analyzed. Results: We identified 931 eligible men. Most (>95%) were ≥60 years old; hypertension (54.6%) and diabetes (17.0%) were the most common comorbidities. Median (interquartile range [IQR]) baseline PSA was 9.0 (2, 37) ng/dL. Median (IQR) follow-up time was 12.5 (7.6, 19.4) months, during which a median (IQR) of 4 (3, 6) PSA tests were observed. Median time between two adjacent PSA tests was 2.0 months. A ≥50% and ≥90% PSA decline was observed in 55.0% and 23.8%, respectively. Best PSA response was a median (IQR) PSA decline of 58% (-89%, 1%), with 14.2% reaching an undetectable PSA value. Median time to PSA progression was 18.5 months (95% confidence interval 15.6, 23.7). Conclusions: This real-world study supports the effectiveness of ENZA in patients with mCRPC. The median PSA at treatment was much lower than PREVAIL, potentially explaining the longer time to PSA progression vs. PREVAIL. However, a lower proportion had PSA declines of ≥50% and ≥90% vs. PREVAIL, which may be attributed to more frequent PSA monitoring within a clinical trial setting and thus more opportunity to capture the true best PSA response.

scholarly journals Real-World Outcomes of Advanced Soft Tissue Sarcoma Patients Treated with Pazopanib

2021 ◽  
Vol 6 (1) ◽  
Author(s):  
Fawaz A ◽  
◽  
Shim I ◽  
Tilley D ◽  
Kelaney MR ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Overall Survival ◽  
Soft Tissue ◽  
Soft Tissue Sarcoma ◽  
Real World ◽  
Systemic Therapy ◽  
Kinase Inhibitor ◽  
Advanced Soft Tissue Sarcoma ◽  
Clinical Trial Setting ◽  
Trial Setting

Background: Pazopanib is an oral multitarget tyrosine kinase inhibitor that is currently approved for the treatment of select subtypes of advanced Soft Tissue Sarcoma (STS) in patients who have progressed on prior anthracyclinebased chemotherapy regimens. In this study, we examine data from multiple centers to assess the efficacy of pazopanib in practice outside of a clinical trial setting. Methods: A retrospective chart analysis was conducted for pre-treated, advanced soft tissue sarcoma patients who began treatment with pazopanib in Alberta, Canada and Cairo, Egypt (2012-2018). Results: In total, 39 predominantly male (56.4%) patients received pazopanib. The median age was 51, 67% of whom had an ECOG of one or less. The predominant sarcoma subtype was leiomyosarcoma (30.8%), and all patients had received at least one prior line of systemic therapy. Thirtytwo of the 39 patients (82%) were initially given the full dose of 800mg with a median time on treatment of 116 days. Seven of the 39 (18%) patients required a dose reduction while on treatment. A majority (94.9%) of patients ultimately discontinued pazopanib treatment for reasons including death (21.6%), disease progression (62.2%), and toxicity (16.4%). The median progression-free and overall survival for these patients was 4.1 months (95%CI, 3.6-4.5) and 8.4 months (95% CI, 4.3-12.5), respectively. Conclusion: Pazopanib is an efficient and generally well-tolerated oral systemic therapy for the treatment of advanced, pre-treated, non-adipocytic soft tissue sarcoma. These results show the efficacy of pazoponib outside of a clinical trial setting.

scholarly journals Somatic cancer genetics in the UK: real-world data from phase I of the Cancer Research UK Stratified Medicine Programme

ESMO Open ◽  
2018 ◽  
Vol 3 (6) ◽  
pp. e000408 ◽  
Author(s):  
Colin R Lindsay ◽  
Emily C Shaw ◽  
Fiona Blackhall ◽  
Kevin G Blyth ◽  
James D Brenton ◽  
...  
Keyword(s):  
Lung Cancer ◽  
Cancer Research ◽  
Phase I ◽  
Real World ◽  
Molecular Pathology ◽  
Cancer Genetics ◽  
Genetic Alterations ◽  
Low Grade ◽  
Stratified Medicine ◽  
Trial Setting

IntroductionPhase I of the Cancer Research UK Stratified Medicine Programme (SMP1) was designed to roll out molecular pathology testing nationwide at the point of cancer diagnosis, as well as facilitate an infrastructure where surplus cancer tissue could be used for research. It offered a non-trial setting to examine common UK cancer genetics in a real-world context.MethodsA total of 26 sites in England, Wales and Scotland, recruited samples from 7814 patients for genetic examination between 2011 and 2013. Tumour types involved were breast, colorectal, lung, prostate, ovarian cancer and malignant melanoma. Centralised molecular testing of surplus material from resections or biopsies of primary/metastatic tissue was performed, with samples examined for 3–5 genetic alterations deemed to be of key interest in site-specific cancers by the National Cancer Research Institute Clinical Study groups.Results10 754 patients (98% of those approached) consented to participate, from which 7814 tumour samples were genetically analysed. In total, 53% had at least one genetic aberration detected. From 1885 patients with lung cancer, KRAS mutation was noted to be highly prevalent in adenocarcinoma (37%). In breast cancer (1873 patients), there was a striking contrast in TP53 mutation incidence between patients with ductal cancer (27.3%) and lobular cancer (3.4%). Vast inter-tumour heterogeneity of colorectal cancer (1550 patients) was observed, including myriad double and triple combinations of genetic aberrations. Significant losses of important clinical information included smoking status in lung cancer and loss of distinction between low-grade and high-grade serous ovarian cancers.ConclusionNationwide molecular pathology testing in a non-trial setting is feasible. The experience with SMP1 has been used to inform ongoing CRUK flagship programmes such as the CRUK National Lung MATRIX trial and TRACERx.

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