bluuebunny/crossref_metadata_2025_split huggingface dataset:
Patient perceptions of palliative care in the process of allogeneic hematopoietic cell transplantation.
Authors: Melody Megan Elizabeth , Bruning Chimere , Mack Rachel , Parrott Kimberley , Taylor Richard , Foran James M. , Roy Vivek , Ayala Ernesto , Kharfan-Dabaja Mohamed , Murthy Hemant S. , Kilpatrick Molly , Robinson Maisha 
Published: June 2021

e24082 Background: Allogeneic hematopoietic cell transplantation (allo-HCT) is associated with significant short- and long-term sequelae that affect patients’ overall quality of life (QoL) and their physical and psychological well-being. Studies have shown improvement in patient QoL and physician satisfaction with palliative care involvement in the allo-HCT process. However, patient and physician perceptions regarding palliative care often impact timely referral. We conducted a prospective study to examine patient perceptions of palliative care both before and after palliative care consultation during evaluation for allo-HCT. Methods: This is a prospective, single-center study, of patients > 18 years of age, who were referred for a palliative care consultation as part of standard evaluation for allo-HCT. Patients were administered a pre- and post-visit internally derived questionnaire to assess their understanding and comfort level with palliative care. The primary objective of this study was to examine patient perceptions of palliative care consultation during evaluation for allo-HCT. Primary end point was the change in patient pre- and post- questionnaire responses. Secondary endpoint was the completion of advance care planning documentation pre- and post- consultation. Results: We enrolled 32 patients (male = 14) undergoing evaluation for allo-HCT with a primary diagnosis of AML (n = 8), NHL (n = 8), MDS (n = 7), ALL (n = 2), and other (n = 7). Following consultation with palliative care there was a statistically significant increase in patient understanding of the reason for the appointment, p = 0.0006. There was also a significant increase in patient’s knowledge of palliative care, with only 28% describing their knowledge as “good” or “excellent” prior to consultation and 79.3% after consultation, p < 0.0001. There was an observed positive, non-statistically significant, trend in patient comfort level with palliative medicine with only 20.7% (n = 6) describing their comfort as “high” or “very high” prior to consultation and 51.7% (n = 15) after consultation, p = 0.60. There was a higher rate of completion of advance care planning documentation following consultation with palliative medicine 56.3% vs 71.4%, p = 0.042. Conclusions: Palliative care consultation prior to allo-HCT increases a patient’s understanding of the role of palliative medicine as part of their allo-HCT and improves advance care planning. These findings need to be validated in a larger, multicenter, patient cohort.
Patient-reported outcomes in cancer patients admitted during the COVID 19 pandemic in a tertiary Spanish hospital.
Authors: Velasco Durantez Veronica , Rodríguez González Adán , Solis-Hernandez Maria Pilar , Iglesias Gomez Clara , Revuelta Alfonso , Jiménez-Fonseca Paula , Mihic Góngora Luka , Contreras Toledo Debora Corina , Valcárcel González Sena , Fernandez Arrojo Sara , Gómez David , del Río Fernández Jorge , Izquierdo Marta , Alvarez-Fernandez Carlos , Villanueva Noemí , Esteban Emilio ,   Onco HUCA
Published: June 2021

e24098 Background: Patient reported outcomes measures (PROMs) represent a tool to objectively assess the health of cancer patients. PROMs may complement oncological evaluations by adding patients’ perspectives on their care priorities. Aim: to address more accurately the management of tumor related symptoms or drug related toxicities to bring he therapies more accurately to bring patients the best quality of life possible. Methods: Data was collected from our electronic registry after proper authorization and corresponding anonymization. Patients were systematically asked for symptoms as fever, diarrhea, dyspnea, vomiting and nausea. These symptoms were graded according to their severity using the Common Toxicity Criteria v5.0. Results: 49 patients admitted to Medical Oncology Hospitalization were included from July of 2020 to January 2021, 80% in advanced disease. Median age 63yo, 32% above 70 yo. Baseline data showed that 27% patients (19% stage IV) had fever at admission, decreasing at 48h to 4%. Patients who had vomiting at admission were 33% (28% stage IV) median grade 2 becoming 20% and grade 1 at 48h. Nausea was present in 49% patients (35% stage IV) at the time of admission with a median grade 1.5, and it decreased to 16% at 48h with a median grade 2. Diarrhea was reported in 12% patients (6% stage IV) median grade 2 at baseline and it was reduced to 10% median grade 1 at 48h. The median severity of diarrhea at admission was 2 and only 1 at 48h. At admission, 33% (all stage IV) of patients presented dyspnea with a median grade 3, while at 48h it was present in 26% of patients and reduced to median grade 2. Conclusions: By systematically measuring symptoms, patients achieved better control of diarrhea, dyspnea, vomiting and nausea after admission. It should be noted that nausea was the variable that decreased the most at 48h, followed by fever, vomiting, dyspnea and diarrhea. All patients with stage IV disease had dyspnea and most of them had nausea and vomiting. These results reflect that these symptoms are more usual in patients with advanced disease compared to those with localized disease. PROMS also help us educate patients by teaching them how to manage treatments, thus improving therapeutic adherence.
Comparing the clinical impact of pancreatic cyst surveillance programs: A trial of the ECOG-ACRIN cancer research group (EA2185).
Authors: Weinberg David , Steingrimsson Jon , Zeh Herbert , Carlos Ruth , O'Dwyer Peter J. 
Published: June 2021

TPS10608 Background: The optimal surveillance strategy for pancreatic cysts, which occur in up to 20% of the adult population, is ill defined. Although risk of malignant degeneration of these cysts is low, pancreatic cancer mortality remains high. Two cyst surveillance guidelines, one proposed by an international consensus group (Fukuoka) and the other by the American Gastroenterological Association (AGA), are accepted standards. Both rely on radiographic and endoscopic ultrasonographic imaging. They differ in indications for, and intervals between, imaging tests, with the Fukuoka guideline advocating more intensive imaging. Clinical guidelines that provide discordant recommendations may undercut the quality and/or value of care, and have implications for societal health care costs. The primary objective of this prospective trial is to compare the clinical effectiveness and associated resource utilization of the Fukuoka and AGA guidelines for pancreatic cyst surveillance. Secondary objectives include a comparison of resource utilization and patient reported outcomes. We are also collecting and banking radiomics data and biospecimens to identify novel methods that might improve cancer risk stratification. Methods: 4606 asymptomatic patients with newly identified pancreatic cysts ≥1cm in diameter are being randomized 1:1 to high intensity (Fukuoka) or low intensity (AGA) surveillance. The primary endpoint is a composite of any pancreatic cancer without surgery, unresectable pancreatic cancer or cancer > T1a, N0 at surgery, and benign disease at surgery. This sample size will provide 90% power to identify a 30% relative difference in the primary outcome at 5 years between the two study arms. Study duration is 8 years in total, allowing for 2 years of cohort enrollment, 5 years of prospective follow-up, and six months reserved for study initiation and close out. Study participants must be ≥ 50 years and ≤ 75 years with an ECOG Performance Status 0-1 at baseline. Participants must have received a CT or MRI within 6 months of registration that identifies a new ≥1 cm pancreatic cyst. Patients with a prior diagnosis of a pancreatic cyst, pancreatic malignancy or a history of pancreatic resection are not eligible. Additional exclusion criteria include a history of acute or chronic pancreatitis, a family history of pancreatic adenocarcinoma in 1 or more first degree relatives, imaging findings or clinical signs that would prompt immediate surgical consideration (enhancing mural nodule, solid component in cyst, pancreatic duct > 10mm, cyst causing obstructive jaundice), a comorbid illness that precludes pancreatic cyst resection, pregnancy or current participation in an established surveillance program. As of February 4, 2021, thirty three (33) participants have been enrolled from two hundred (200) potential sites. Clinical trial information: NCT04239573 .
HER2CLIMB-04: Phase 2 open label trial of tucatinib plus trastuzumab deruxtecan in patients with HER2+ unresectable locally advanced or metastatic breast cancer with and without brain metastases (trial in progress).
Authors: Krop Ian E. , Ramos Jorge , Zhang Chiyu , Hamilton Erika P. 
Published: June 2021

TPS1097 Background: Tucatinib (TUC) is an oral, reversible, small molecule tyrosine kinase inhibitor highly selective for human epidermal growth factor receptor 2 (HER2) with minimal inhibition of human epidermal growth factor receptor (EGFR). In the pivotal, randomized HER2CLIMB trial (NCT02614794), the combination of TUC + trastuzumab (T) + capecitabine (C) demonstrated statistically significant and clinically meaningful improvements in progression free survival (PFS), overall survival (OS), and PFS in patients (pts) with brain metastases (BM), compared to T + C alone. These data supported regulatory approvals in the US and internationally for TUC in combination with T + C in pts with HER2+ metastatic breast cancer (MBC). Trastuzumab deruxtecan (T-DXd) is an antibody-drug conjugate of trastuzumab and a topoisomerase I inhibitor payload approved in the US for treatment of HER2+ MBC in pts who have received 2 or more prior anti-HER2 regimens in the metastatic setting. Approval was based on data from the single arm Destiny-Breast01 trial (NCT03248492) where treatment with T-DXd resulted in a confirmed objective response rate (cORR) of 61.4% (95% CI: 54.0, 68.5) in pts with HER2+ MBC who had prior ado-trastuzumab emtansine treatment. Despite these advances, HER2+ MBC remains incurable, and pts will eventually progress on currently available therapies. Combining TUC and T-DXd may result in further improvement on the efficacy seen with either agent alone. Methods: HER2CLIMB-04 (NCT04539938) is a single arm, open-label phase 2 trial evaluating safety and antitumor activity of TUC + T-DXd in pts with HER2+ unresectable locally-advanced or MBC who have received 2 or more prior HER2-based regimens in the metastatic setting. Pts with BM, including active BM, may be enrolled. Ten pts will be enrolled in the safety lead-in portion of the trial and followed for at least 1 cycle. If safety of the combination is acceptable, the trial will continue until approximately 60 response-evaluable pts have been enrolled, approximately evenly distributed between pts with and without BM. The primary endpoint is cORR by investigator (INV) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. Secondary endpoints are PFS, duration of response (DOR), and disease control rate (DCR) by INV per RECIST v1.1, OS, and safety. Exploratory endpoints will include cORR, PFS, DOR, DCR by independent central review per RECIST v1.1, pharmacokinetic analyses, biomarker analyses, and changes in patient-reported outcomes using the European Quality of Life 5-Dimension 5-Level instrument. Enrollment began in late 2020 in the United States. Clinical trial information: NCT04539938 .
acelERA Breast Cancer (BC): Phase II study evaluating efficacy and safety of giredestrant (GDC-9545) versus physician’s choice of endocrine monotherapy in patients (pts) with estrogen receptor-positive, HER2-negative (ER+/HER2-) locally advanced or metastatic breast cancer (LA/mBC).
Authors: Martin Miguel , Lim Elgene , Chavez Mac Gregor Mariana , Shivhare Mahesh , Ross Graham , Patre Monika , Roncoroni Laura , Louka Maria , Sohn Joohyuk 
Published: June 2021

TPS1100 Background: The standard-of-care therapy for ER+ BC typically involves modulation of estrogen synthesis and/or ER activity. Despite disease progression with standard treatments, growth and survival of the majority of tumors are thought to remain dependent on ER signaling; therefore, pts with ER+ BC can still respond to second- or third-line endocrine treatment (ET) after progression on prior therapy (Di Leo 2010; Baselga 2012). ESR1 mutations may drive estrogen-independent transcription and proliferation leading to resistance. The highly potent, nonsteroidal oral selective ER degrader, giredestrant, achieves robust ER occupancy and is active regardless of ESR1 mutation status. Phase I data have shown that giredestrant is well tolerated and active both as a single agent and in combination with the cyclin-dependent kinase 4/6 inhibitor (CDK4/6i), palbociclib (Lim 2020). Single-agent giredestrant has also shown encouraging antitumor activity in pts previously treated with fulvestrant and/or a CDK4/6i. Methods: acelERA BC (NCT04576455) is a randomized, open-label, multicenter phase II study evaluating the efficacy and safety of giredestrant vs. physician’s choice of ET (fulvestrant or aromatase inhibitor) in males, or postmenopausal or pre/perimenopausal females with ER+/HER2– LA/mBC who have received 1–2 prior lines of systemic therapy in the LA or mBC settings, at least one of which must be ET. Randomization: 1:1 to receive giredestrant (30 mg PO QD on Days 1–28 of each 28-day cycle) or physician’s choice of ET per local guidelines. Men and pre-/perimenopausal women will receive a luteinizing hormone-releasing hormone agonist. Eligibility: ≥18 years, ECOG PS 0–1, histologically or cytologically confirmed diagnosis of LA (recurrent or progressed) or metastatic adenocarcinoma of the breast, measurable disease (per modified RECIST v1.1) or evaluable bone lesions, and ER+/HER2– tumors (locally assessed). Primary endpoint: progression-free survival (PFS; investigator-assessed per RECIST v1.1). Secondary endpoints: overall survival, objective response rate, duration of response, clinical benefit rate, PFS in pts with baseline ESR1 mutations, and quality of life. Safety, pharmacokinetics, biomarkers, and health status utility will also be assessed. Stratification: site of disease (visceral vs. nonvisceral), prior treatment with CDK4/6i (yes vs. no), and prior treatment with fulvestrant (yes vs. no). PFS will be compared using a stratified log-rank test; median PFS, using Kaplan–Meier analyses. Recruitment for the global enrollment phase is ongoing, the first patient was enrolled November 27, 2020. Clinical trial information: NCT04576455 .
NBE-002: A novel anthracycline-based antibody-drug conjugate (ADC) targeting ROR1 for the treatment of advanced solid tumors—A phase 1/2 clinical trial.
Authors: Tolcher Anthony W. , Meric-Bernstam Funda , McKean Meredith , Beerli Roger R , Waldmeier Lorenz , Gebleux Rémy , Hellmann Ina , Chrom Pawel , Grawunder Ulf 
Published: June 2021

TPS1108 Background: The receptor tyrosine kinase-like orphan receptor 1 (ROR1) is highly expressed during embryonic development, but is minimally present or absent on post-partum healthy tissues. ROR1 is expressed in a variety of hematological and solid tumors and is associated with aggressive cancer phenotype and poor clinical outcomes. NBE-002 is an ADC targeting ROR1, obtained by site-specific, enzymatic conjugation of the anthracycline-derivative PNU-159682, modified with a non-cleavable linker to a humanized recombinant IgG1 monoclonal antibody, based on a novel anti-human ROR1 monoclonal antibody XBR1-402 (Peng et al. (2017) J. Mol. Biol. 429: 2954-73). Direct anti-tumor activity of NBE-002 was evaluated in immunodeficient, ROR1 expression-low/-intermediate/-high PDX models of several carcinoma and sarcoma subtypes. The most pronounced anti-tumor effect was achieved in triple-negative breast cancer (TNBC), at doses as low as 0.033 mg/kg, suggesting a best-in-class therapeutic index in light of the high tolerability in preclinical toxicology models. Administration in a fully immune competent setting (EMT6/ROR1 orthotopic breast cancer model) led to a strong anti-tumor response and a long-lasting anti-tumor immune protection dependent on CD8 T cells. Methods: NBE-002-01 (NCT04441099) is a first-in-human, open-label, multi-center, phase (Ph) 1/2 study of NBE-002 in adult patients with advanced solid tumors. Ph 1 of the study consists of a Dose Escalation Cohort (DEC), utilizing an accelerated titration design, followed by a traditional 3+3 design, and an optional Safety Expansion Cohort (SEC). Ph 2 will include two parallel Expansion Cohorts (EC), enrolling patients with advanced TNBC (EC1) or other solid tumors (EC2), with Simon’s two-stage design. Key eligibility criteria include Eastern Cooperative Oncology Group performance status of 0-2 (Ph 1) or 0-1 (Ph 2), adequate organ function defined as: hemoglobin ≥9.0 g/dL, neutrophils ≥1500 /µL, platelets ≥100000/µL, aspartate and alanine aminotransferases ≤2.5x the upper limit of normal (ULN), total bilirubin ≤1.5x ULN, creatinine ≤1.5x ULN, left ventricular ejection fraction ≥50%. The primary objectives are to assess safety and tolerability and to establish the recommended dose for further development (Ph 1), and to evaluate anti-tumor activity of (Ph 2). Secondary and exploratory objectives include characterization of immunogenicity as well as pharmacokinetic and pharmacodynamic profiles. NBE-002 is given intravenously once every three weeks until disease progression, unacceptable toxicity, withdrawal of consent, or other protocol-specific criteria are met. Ph 1 dose escalation was initiated on 17 JULY 2020 and is still recruiting in the US. Ph 2 is planned to be initiated in 2022. Clinical trial information: NCT04441099 .
A phase II clinical trial of talazoparib monotherapy for PALB2 mutation-associated advanced breast cancer.
Authors: Gruber Joshua James , Gross Wyatt , McMillan Alex , Ford James M. , Telli Melinda L. 
Published: June 2021

TPS1109 Background: PALB2 (Partner and Localizer of BRCA2) plays a critical role in the maintenance of genomic integrity through its role in the Fanconi anemia and homologous recombination DNA repair pathways. Our recently reported phase II clinical trial (Gruber, et al. JCO 2019) showed that talazoparib monotherapy was associated with single agent activity in germline PALB2 (gPALB2) mutation-associated advanced breast cancers. Of 5 patients with a germline PALB2 mutation, all 5 had reduction in target lesions > 20% with 3 of 5 achieving a RECIST 1.1 response. All patients had visceral metastases and were heavily pretreated; one response occurred in a patient with 8 prior lines of therapy for metastatic breast cancer. Clinical activity of the PARP inhibitor olaparib was also demonstrated in breast cancer patients with gPALB2 mutations in the Olaparib Expanded trial (TBCRC 048). Thus, we believe there is significant value in generating additional PARP inhibitor monotherapy data in subjects with advanced PALB2 mutation-associated breast cancer. We propose this phase II trial to better estimate the rate of RECIST 1.1 objective response in this patient population. Methods: This is a single-arm, two-stage, non-randomized study to assess the objective response rate (ORR) of talazoparib monotherapy in patients with PALB2 mutation-associated advanced breast cancer. Eligible subjects will be adults with inoperable locally advanced or metastatic breast cancer with a germline or somatic PALB2 mutation with 0-3 prior therapies for advanced disease. Eligible patients must have a deleterious or suspected deleterious mutation in PALB2 on a CLIA approved commercial germline or next generation sequencing tumor assay. Study treatment is talazoparib 1 mg PO daily. In stage 1, ORR will be assessed after 15 patients; if at least 2 responses are observed then an additional 15 patients will be enrolled; if less than 2 responses are observed the study will be terminated. The primary objective is to evaluate whether talazoparib monotherapy can induce a 30% ORR in subjects with advanced breast cancer associated with a PALB2 mutation. Response will be assessed by RECIST 1.1. Secondary objectives include safety, PFS, clinical benefit rate, and patient-reported quality-of-life. Exploratory endpoints will assess for the presence of PALB2 loss-of-heterozygosity, biallelic mutations, correlation of ctDNA profile with treatment response and the correlation of germline versus somatic mutations with response rate. This trial is currently activated and enrolling at Stanford Cancer Center and it is expected that 4 additional sites will be added. Conclusion: This trial will evaluate gPALB2 as a biomarker for PARP inhibitor monotherapy in advanced or metastatic HER2- breast cancer. Clinical trial information: pending .
REGOSTA: A randomized, placebo-controlled, double-blinded, multicenter study evaluating the efficacy and safety of regorafenib (REGO) as maintenance therapy after first-line treatment in patients (pts) with osteosarcoma (OS) and non-osteosarcomas (non-OS) of bone (non-Ewing, non-chondrosarcomas and non-chordomas).
Authors: Duffaud Florence , Chabaud Sylvie , Gautier Julien , Ferlay Celine , Vizoso Séraphine , Brahmi Mehdi , Benezech Sarah , Dufresne Armelle , Marec-Berard Perrine , Ray-Coquard Isabelle Laure , Kalbacher Elsa , Collard Olivier , Penel Nicolas , Rios Maria , Bompas Emmanuelle , Chevreau Christine , Mir Olivier , Boudou-Rouquette Pascaline , Blay Jean-Yves , Piperno-Neumann Sophie ,   French Sarcoma Group
Published: June 2021

TPS11576 Background: Pts with OS and non-OS of bone are treated with a multimodal sequence therapy of neoadjuvant chemotherapy (CT), surgery and adjuvant CT, followed by a close surveillance until recurrence. At recurrence, the prognosis remains poor with objective response rates of 3-29%, and a median Progression-Free Survival (PFS) of less than 4 months in OS. There is a clinical need to reduce the risk of recurrence after the initial treatment sequence. The REGOBONE study reported a significant clinical benefit of regorafenib compared to placebo in patients with relapsed OS (median PFS: 16.4 versus 4.1 weeks). Methods: This multicenter trial is ongoing to study the efficacy and safety of maintenance REGO in pts > = 16 years, with complete remission after initial treatment sequence of their bone sarcoma. 168 pts will be randomly allocated in a 1:1 ratio to receive either oral REGO or its matching placebo (control arm) at a daily dose of 120mg, continuously and for a maximum of 12 months. Randomization will be stratified according to the following risk factors: metastases (mets) at diagnosis and/or poor response to neoadjuvant CT versus no mets at diagnosis and good response to neoadjuvant CT. The primary objective is to compare the efficacy (Relapse-Free Survival) between the 2 arms. The expected 3-year RFS rates are 55% in the control arm and 74.6% in the REGO arm (HR = 0.5). 66 events will provide 80% power to show significant improvement in RFS, using a 2-sided log-rank test at a 5% level. Secondary endpoints include Time to Treatment Failure, Overall Survival, Quality of Life, safety profile, and compliance to treatment. Radiological endpoints will be evaluated using the RECIST 1.1. Translational objectives will be to identify predictive biomarkers for efficacy of REGO as maintenance therapy using liquid biopsies. As of Feb 1st, 2021, 3 patients have been randomized. 15 sites of the French Sarcoma Group will participate. Clinical trial information: NCT04055220.