bluuebunny/crossref_metadata_2025_split huggingface dataset:
Multidisciplinary tumor boards: A prospective study of the impact on patient management in a community-based Brazilian cancer center.
Authors: Costa Marcos , Teixeira Carlos Henrique , Peixoto Renata , Jorge Waldec , Ades Felipe , Kann Ariel Galapo , Caponero Ricardo , Tzu Cheng , Ricardo Eliza Dalsasso , Mutão Taciana , Machado Larissa , Pimenta Jefferson , Santos Marcelo 
Published: May 2020

e19261 Background: MDTB have emerged as a valuable forum to address questions related to patient management. There is a general data lack of its overall benefit by attending physicians. However, few reports describe numerical impact on patient care of this tailor-made and shared model of medical decision. Methods: We describe, in this prospectively-collected study, data from a Cancer Center (HAOC) regarding multiple weekly, 1h-long discussions, as part of MDTB (GU, neuro-oncology GI, thorax, HN, breast, gyneco, melanoma/sarcoma and palliative care). Only newly-diagnosed or on-treatment challenging cancer cases were included. Attendees (onco, surgeons, RT, paths and radiologists) pooled their expertise to warrant quality and maximize resources. The primary endpoint was change in the medical planning. In our institution, further adherence to MDTB recommendations are left totally at physician discretion. Results: From Oct/17 to Feb/19, 413 cases were discussed (60% female), mean of 2.9 cases/MDTB, but GI (3.8), thorax (3.7) and breast (2.94) were above the median. Mean was 13.4 doctors/MDTB - more in breast (16.5), GI (15.8) and uro (15.1). Mean of oncologist/MDTB in general was 6.1, but 8.1 in GI, 7.8 thorax, breast 6.8 and 6.7 uro. Mean of surgeons/MDTB in general was 3.5, but 6.2 in breast, 5.8 uro and 5.5 GI. 100% of all had at least 1 oncologist and uro/GI/breast had at least 1 surgeon in 100% of them. In 50% (uro), 44% (neuro), 23.5% (breast), 6.4% (thorax) and 3.7% (GI) had at least 1 physician of 5 major areas. Oncologists engaged with more cases: 80.4% (thorax), 70.6% (uro), 59.6% (GI), 56.5% (HN). Prostate (38.2%), metastasis (neuro, 28.9%), colorectal (18.2%), lung adenoCA (43.5%), mouth (30.4%), ductal carcinoma (35.8%) were the more frequently discussed per system. In 25.7%, MDTB changed original medical planning. By site: GI (35,6%), thorax (24,7%), breast (22,6%), neuro (21,7%), uro (17,7%) and HN (17,4%). Oncologists were responsible more in thorax (73,9%) and less in breast (33%) and surgeons more in breast (50%) and less in GI (33%). Adherence to NCCN guidelines was total. Finally, but not measurable, a sizeable number of cases requires significant weekly time-effort. Conclusions: This study confirms MDTB leading role in cancer care, highlighting the importance of teamwork for more precise patient care. We point out that it led to substantial practice-changing in our institution, reinforcing its importance in a scenario which doctors are confronted with increasing complexities in patient management.
Cancer types applicable to efficacy assessment using real-world data analyzed from PMDA-approved data.
Authors: Kato Yuka , Hirase Chikara , Hatogai Ken 
Published: May 2020

e19272 Background: Clinical development of anti-cancer agents has been accelerated due to emergence of new therapies. Although randomized comparative trials are the gold standard for the evaluation of clinical utility, it is often impossible to conduct such trials. At ASCO 2019, 89 real-world data (RWD) studies were presented. These studies highlighted the increasing awareness of the value of RWD. We here investigated for which cancer types RWD could be applicable to efficacy assessment based on the previous reviews of Pharmaceuticals and Medical Devices Agency, Japan (PMDA). Methods: Review reports and Summaries of submitted documents publicly available on the website of PMDA and research articles of trials included in the new drug applications of anti-cancer agents approved in Japan between 2006 and 2019 were reviewed. Results: Among 209 anticancer-agents approved for a new indication based on clinical trial (CT) data, 42 agents and 167 agents were approved based on results of single-arm CTs and those of comparative trials, respectively. The numbers of agents for hematological malignancies and solid tumors were nearly same. Among agents approved based on single-arm CT results, most of them (85.7%) were designated for orphan drug (OD), and 1/3 of them were specified for a biomarker (BM), such as ALK fusion gene in non-small cell lung cancer (35.7%). Among 42 single-arm CT-based approvals, a threshold for efficacy endpoint was set at least in one pivotal trial in 30 items based on the following references: (i) results of CTs for the same population (11), (ii) no standard therapy (10), (iii) clinical trial plans of other agents for the same population (7), (iv) others such as values defined by the investigators’ discretion (14). Regardless of BM, only 1/4 had a pre-defined threshold for efficacy evaluation in the pivotal trial based on the results of CTs for the same population. Among 15 BM-related agents, most of them (93.3%) had been designated for OD. Meanwhile, among the agents without biomarker and OD, the setting was (ii) or (iv). Conclusions: RWD could be applicable for efficacy evaluation of BM-related agents with OD designation, agents for BM-unrelated tumor types with no established standard treatment, and agents for those in which drug development has not been advanced enough to set a threshold for efficacy evaluation.
What are the challenges for building real world comparative cohorts in oncology?
Authors: Jeswani Nikita , McDonell Amanda , MacDougall Finlay , Hodge Jeff Paul 
Published: May 2020

e19285 Background: There has been a proliferation of single arm trials in oncology being presented to regulators. An external comparator using real world data (RWD) can help establish context to trial results and direct comparison to the treatment arm by mirroring the inclusion/exclusion criteria for the trial and examining trial outcomes in an RWD external comparator cohort. RWD has successfully supported some regulatory submissions, although there have been failures as well, with comparability of patients and endpoints under scrutiny. Methods: IQVIA has partnered with life sciences companies on > 20 external comparator projects from 2017-present using a mix of data acquisition strategies (e.g., chart review, database extraction) and methods (e.g., real-world cohort vs. real-world benchmark, propensity matching vs. inverse probability treatment weighting). We collated, reviewed, and synthesised the challenges and risk mitigations documented throughout study execution in aggressive and indolent lymphomas, multiple myeloma, Merkel cell carcinoma, and synovial sarcoma. Results: Patient characteristics used to assess trial eligibility (e.g., biomarker expression, International Prognostic Index, ECOG status) and outcomes (e.g., response rate) are not often captured in a structured format in RWD or recorded in routine clinical practice. As a result, there is a greater reliance on chart reviews today over database extractions to fulfil external comparator requirements, though in some diseases, databases have proven to be a faster and more cost-effective solution. Conclusions: To improve the success of external comparator studies, trial design should be informed by existing RWD so that relevant real-world endpoints and outcome evaluation criteria are included alongside RCT standards. Advances are needed to facilitate RWD capture that matches trial data more closely. This could include bespoke data collaborations, biobank linkage, and natural language processing to drive study execution in databases in the future.
Do assumptions in health economic evaluations hamper drug uptake?
Authors: Franchini Fanny , Degeling Koen , Pereira-Salgado Amanda , Tran Ben , Au-Yeung George , McArthur Grant A. , Lee Belinda , Solomon Benjamin J. , Gibbs Peter , IJzerman Maarten J. 
Published: May 2020

e19289 Background: Health economic studies of anti-cancer systemic treatments typically make strong assumptions regarding the number of drug lines received after initial therapy. This may have a substantial effect on health economic outcomes and impact drug reimbursement and uptake in practice. This study aims to quantify the real-world systemic treatment patterns in 4 metastatic cancers using clinical registries to explore whether health economic assumptions are justifiable. Methods: Data from 4,431 metastatic cancer patients were extracted from Australian clinical registries: colorectal (COL, n = 3087), non-small cell lung (LUN, n = 705), pancreatic (PAN, n = 459), and melanoma (MEL, n = 180). A set of criteria was defined to consistently identify drug lines across the cancer-specific registries. These criteria were based on the type of drug (biological agent or chemotherapy), switches in chemotherapy regimen (whether a chemotherapy agent was added or removed) and the timing of such changes. Consequently, the identified drug lines provide a more detailed view on treatment patterns compared to clinical treatment lines (defined by disease progression), the latter typically including multiple drug lines. Censoring in the number of drug lines received due to ongoing treatment was accounted for in all analyses. Results: Most patients started treatment after diagnosis: 77% (COL), 89% (LUN), 56% (PAN) and 79% (MEL). For COL, LUN, PAN and MEL respectively, the proportion of patients starting a 2nd drug line was 51%, 60%, 19% and 24%, whereas 28%, 35%, 6% and 8% of patients started a 3rd drug line. In all cancers, patients were most likely to receive only a single drug line, i.e. the mode number of drug lines was 1. For all patients, the median number of drug lines were COL: 2, LUN: 2, PAN: 1, MEL: 1. For treated patients only, the median (range) number of drug lines received was COL: 2 (1-9), LUN: 2 (1-8), PAN: 1 (1-5), MEL: 1 (1-5). Conclusions: We show that patients are most likely to receive a single drug line. Considering our findings, most health economic analyses likely overestimate the intensity of drug treatment in metastatic disease, thereby underestimating the impact of initial treatment relative to downstream treatments. This is likely to bias estimates of total treatment cost, cost-effectiveness and budget impact, which will hamper the uptake of novel anti-cancer agents and may lead to suboptimal decisions regarding treatment strategies.
Real-world outcomes of patients with metastatic breast cancer (BC) treated with osteoclast inhibitors (OIs).
Authors: Karimi Yasmin , Purington Natasha , Liu Mina , Kurian Allison W. , Sledge George W. , Blayney Douglas W. 
Published: May 2020

e19314 Background: Patients with metastatic BC have a high incidence of bone metastasis with associated morbidity and mortality. OIs including denosumab and zoledronic acid reduce the rate of skeletal-related events (SREs). Since denosumab was approved in 2011, there have been no analyses about the comparative effectiveness and safety of denosumab versus zoledronic acid. Methods: Patients with BC treated with three or more doses of either denosumab or zoledronic acid after 2011 were identified in Stanford’s BC research database, Oncoshare. SREs were defined as a composite measure of spinal cord compression, pathologic fracture, surgery or radiation to bone. Unadjusted Cox proportional hazard models were fit to time to: first SRE, subsequent SRE, overall survival (OS) and BC-specific survival. Results: 515 patients were identified: 388 and 127 treated with denosumab and zoledronic acid per oncologist’s discretion, respectively. Groups were well balanced for age at diagnosis, race/ethnicity, tumor grade, prior chemotherapy use and median time from first OI therapy to last follow-up. More patients with triple-negative BC (TNBC) received zoledronic acid. Median follow-up time from first OI dose was 28 months. 42% of patients experienced at least one SRE and there were no differences in incidence of first or subsequent SREs between groups (Table). Median OS was 91 months with no statistically significant difference between groups. Median BC-specific survival was not reached (78% of patients survived), however, patients in the zoledronic acid group had over twice the risk of BC-specific mortality, a difference that remained significant after adjustment for TNBC status. There was no difference in adverse events including osteonecrosis of the jaw, vertebral fracture or hypocalcemia. Conclusions: In a contemporary, real-world care setting, we observed no difference in the incidence of SREs or OS between metastatic BC patients treated with denosumab or zoledronic acid. [Table: see text]
Preferences for research participation among individuals with Lynch syndrome: Findings from the HEROIC Registry.
Authors: Dubin Robin Beth , Burton-Chase Allison 
Published: May 2020

e19317 Background: AliveAndKickn, whose mission is to improve the lives of individuals and families affected by Lynch Syndrome (LS) through research and education, maintains the HEROIC Registry. It is the first-of-its-kind, patient-centric, genetic database that enables individuals to take an active role in furthering LS research. Methods: The HEROIC Registry allows patients to contribute medical information and their experiences living with LS and its associated cancers to help researchers develop new treatments, understand the various genetic mutations, and conduct further studies. It also asks individuals about their preferences for participating in different types of research studies. Results: The HEROIC Registry was launched in February of 2016. Potential participants were notified about the availability of the Registry in multiple ways, including social media and patient conferences. As of January 2020, 269 individuals have added their data to the Registry. Of those, 217 have a known LS mutation and were on average 42.5 years old at the time of diagnosis. 38% report having had a diagnosis of colorectal cancer with an average diagnosis age of 43; 23% of female respondents report having had endometrial cancer with an average diagnosis age of 48. 37% are cancer-unaffected. For interest in participating in future research, 69% are somewhat or very likely or definitely want to participate in research that involves a LS treatment, with rates ranging from 62-80% based on the delivery method of the treatment. For non-treatment studies, the percentage of individuals that are somewhat or very likely or definitely want to participate in research that involves community meetings, hospital stays, a medical procedure, wearing a device, or providing information by phone are text are 76%, 46%, 52%, 83%, and 78%, respectively. The percentage of individuals willing to donate a blood sample used to study their DNA is 95%. Conclusions: The HEROIC Registry provides a unique opportunity for health care providers and researchers to partner directly with a patient-advocacy organization for the purpose of improving LS care. It also has the benefit of including a diverse set of patients who are being seen in a variety of health care settings, which can aid in exploring research questions outside of a single institution. However, individuals show clear preferences for the types of research they are likely to participate in, a fact that should be taken into consideration when researchers are planning new studies in this population.
Outpatient cancer rehabilitation to improve patient reported and objective measures of function.
Authors: Pergolotti Mackenzi , Covington Kelley Renee , Lightner Ashley N , Bertram Jessica , Thess Melissa , Sharp Julia L , Spraker Matthew B , Williams Grant Richard , Manning Peter 
Published: May 2020

e19325 Background: Adults with cancer often struggle with persistent symptoms that negatively impact quality of life (QoL), physical and social function. Cancer rehabilitation delivered by a trained physical and/or occupational therapist (PT/OT) is recommended. However, evidence is lacking regarding the impact of community-based PT/OT on patients' functioning. Methods: We assessed adults (≥ 18 years) who completed cancer-specific visits in routine care at two outpatient rehabilitation clinics. Demographic and clinical data was patient-reported. Patient-Reported Outcomes Measurement Information System (PROMIS) measures included: Global Physical Health (GPH) and Mental Health (GMH) scale (10 item); Physical Function (PF; 4-item), and Ability to Participate in Social Roles and Activities (SRA; 4-item). Hand grip strength (HGS) and the Timed Up and Go (TUG) were administered by PT/OT. We compared outcomes collected during the first and final therapy evaluations using paired samples t-test and calculated effect size using Cohen’s d. We used independent t-test to compare mean pre-post change between patients on active treatment versus post-treatment. Results: Patients (N= 185) were predominantly female (75%), 41-64 years old (57%), and majority receiving cancer treatment (58%). Most (N =115, 62%) completed at least one follow-up evaluation over 6 weeks. From pre to post therapy, a significant (p<.05; d = 0 .35-.21) effect was observed for GPH, SRA, HGS, and TUG. There was no difference in the average improvement between active and post-treatment groups. Conclusions: Community-based cancer-specific PT/OT has a significant impact on the well-being and functioning of adults with cancer. Evidence supports the utilization of community-based, cancer-specific PT/OT throughout the cancer-care continuum. [Table: see text]
Is scalp cooling system effective in chemotherapy induced alopecia? A real-world experience in Asian patients.
Authors: Kate Shruti , Patil Roshan , Dhande Sonal Chandrakant , Shaikh Dina , Vyavahare Rohini , Joseph Sheela , Ramesh Yasam Venkata , Nagarkar Rajnish Vasant 
Published: May 2020

e19334 Background: Chemotherapy induced alopecia(CIA) is the most common and afflicting chemotherapy side effect. Data on the use of scalp cooling (SC) to prevent CIA is limited and debatable due to lack of experience. This study analyzed the effectiveness and tolerability of SC in clinical practice. Methods: We prospectively analysed 100 consecutive patients who opted for Paxman scalp cooling. The assessment of alopecia was done by WHO grading system. The extent of alopecia was analyzed at baseline, during each session, and 4 weeks from the last SC session. Results: A total of 100 (94 females, 6 males) patients were enrolled. Amongst them, 69% had breast cancer, 18% had ovarian cancer and 26% had various other cancers. Majority of patients were treated with a curative intent (69%) and had a good performance status (ECOG PS 0-1). Overall 40% patients received anthracyclines, 45% received taxanes, 9% received both and 6% patient received other chemotherapeutic agents. Hair preservation rate was in 31% (Grade 0 alopecia 12%, Grade 1 alopecia 19%). After a single SC session, 30% of patients discontinued due to grade 2 alopecia. On cox regression analysis, onset of grade 2 alopecia on SC was significantly longer in patients on taxanes compared to anthracyclines (1.357 HR, 0.98-1.87 CI, p= .05). One out of 100 patients developed scalp metastases. Majority of the patients experienced mild side effects [chills (30%), headache (12%)]. One out of 100 patients discontinued SC due to intolerable side effects. None of the other predetermined factors (age, menopausal status, ECOG performance status, body mass index), were found to delay the onset of grade 2 alopecia amongst the male patients, hair preservation rate was 100% . Conclusions: Scalp cooling is very effective for hair preservation for some chemotherapy patients. In our study, we found that it is more effective in reducing alopecia in taxane chemotherapy. Scalp metastasis and SC discontinuation were observed to be very rare.
Bevacizumab for the treatment of brain radionecrosis in cancer patients.
Authors: Raez Luis E. , Khan Alina , Botero Ana , Domingo Gelenis Calzadilla , Izquierdo Paola , Castellon Ignacio , Below Jameshia 
Published: May 2020

e19352 Background: Brain radionecrosis (BRN) occurs as a result of radiation therapy for brain tumors or metastatic brain lesions. BRN is characterized by an increase in permeability and disruption of the blood-brain barrier (BBB). There are currently no standard treatment options for BRN. While the mechanism of BRN is unknown, it is hypothesized that there is an inflammatory reaction of the local tissue to radiation which results in a continuous process involving endothelial cell dysfunction. This leads to tissue hypoxia and increased vascular endothelial growth factor (VEGF) which in turn causes capillary leakage, progressive BBB dysfunction, and cerebral edema. Bevacizumab (BEV), a humanized monoclonal antibody with action against VEGF, has recently been used in some studies for the treatment BRN. BEV essentially blocks VEGF from reaching its targets on the endothelium, thus making it an ideal treatment modality for BRN. Methods: The primary purpose of this study is to assess the effectiveness and safety of BEV for the treatment of BRN. Fifteen patients (pts), 14 diagnosed with lung cancer and one with breast cancer, that had BRN and treated with BEV between January 2014 and November 2019 were identified from Memorial Cancer Institute's database. A retrospective chart review analyzing pts's age, sex, BEV dose, dosing frequency, number of treatments received, medication-related adverse effects, and clinical benefit was conducted. Brain imaging pre-treatment and after four cycles of therapy were compared to assess the efficacy of BEV. Pts who exhibited clinical benefit (complete response (CR), partial response (PR), or stable disease (SD)) received an additional four cycles of treatment. Results: The median age was 65 years (y) (49-78y) and 10/15 (67%) of pts were female. Clinical benefit was achieved in 13/15 (87%) of pts. The most frequent dosing regimen administered was 10 mg/kg every two weeks and the median number of cycles given was eight cycles (1-12). Treatment with BEV was well tolerated with eight pts (53%) experiencing BEV-related Grade 2 or less adverse effects (AE), including hypertension (27%), proteinuria (13%), thrombocytopenia (7%), and mild nose bleeds (7%). There were no Grades 3-5 AE. Conclusions: This study demonstrates that there is a clinical benefit when administering BEV for the treatment of BRN. BEV was well tolerated and has an acceptable safety profile.