tbuckley/cabot-search huggingface dataset:
Stage I findings of a two-stage phase II study to assess the efficacy, safety, and tolerability of barasertib (AZD1152) compared with low-dose cytosine arabinoside (LDAC) in elderly patients (pts) with acute myeloid leukemia (AML).
Authors: Giovanni Martinelli, Hagop Kantarjian, Elias Jabbour, Alfonso Quintas-Cardama, Kiyoshi Ando, Jacques-Olivier Bay, Andrew Wei, Stefanie Gröpper, Kate Owen, Laura Pike, Nicola Schmitt, Paul Stockman, Aristoteles Giagounidis
Journal: Journal of Clinical Oncology (2012)

6527 Background: Barasertib is the pro-drug to barasertib-hQPA, a selective Aurora B kinase inhibitor with preliminary anti-AML activity in a Phase I/II study (Löwenberg et al. Blood 2011;118:6030). Methods: AML pts aged ≥60 y considered unsuitable for intensive chemotherapy were randomized 2:1 to open-label barasertib 1200 mg (7-day iv infusion) or LDAC 20 mg (sc twice daily for 10 days) in 28-day cycles (NCT00952588). The primary endpoint was improved objective complete response rate (OCRR: CR + CRi [Cheson criteria, but requiring CRi confirmation ≥21 days after first appearance, with partial recovery of platelets and neutrophils]). Secondary endpoints included duration of response (DoR), overall survival (OS) and safety. Results: 74 pts (barasertib, 48; LDAC, 26) received treatment and all completed ≥1 cycle. A significant improvement in OCRR was observed with barasertib (35.4% [17/48] vs 11.5% [3/26]; difference, 23.9% [95% CI, 2.7-39.9]; P&lt;0.05); barasertib responses were seen in all cytogenetic risk groups and appeared to be durable (median DoR [range], 82 [28-321] days; vs LDAC 30-85 days). Although not formally sized to compare OS data, a trend favoring barasertib was observed (HR=0.88, 95% CI, 0.49-1.58; P=0.663; median OS, 8.2 vs 4.5 mo). Stomatitis and febrile neutropenia were the most common adverse events (AEs) in the barasertib arm with higher incidences vs LDAC (71% vs 15%; 67% vs 19%, respectively). Grade ≥3 AEs with a greater incidence in the barasertib arm were febrile neutropenia (50% vs 19%), stomatitis (29% vs 0%) and pneumonia (25% vs 8%); grade ≥3 infection rates were also higher with barasertib (40% vs 23%). For both arms, there were similar discontinuation rates due to AEs (barasertib 8.3% vs LDAC 7.7%), deaths due to AEs (12.5% vs 11.5%) and 30-day mortality (12.5% vs 15.4%). Cumulative toxicities were not observed with barasertib treatment. Conclusions: In this population with poor prognosis using standard chemotherapy, barasertib showed a significant improvement in OCRR vs LDAC, and a safety profile that was manageable and consistent with previous studies.
Characterization of the p.A1461D Mutation Causing Von Willebrand's Disease Type 2B with Severe Thrombocytopenia, Circulating Giant Platelets and Defective α -Granule Secretion
Authors: Florian Langer, Tobias Obser, Florian Oyen, Katharina Holstein, Brigitte Spath, Andreas Greinacher, Ulrich Budde, Carsten Bokemeyer, Reinhard Schneppenheim
Journal: Blood (2012)
Patterns of distribution of giant congenital melanocytic nevi (GCMN): The 6B rule
Authors: Vanessa P. Martins da Silva, Ashfaq Marghoob, Ramon Pigem, Cristina Carrera, Paula Aguilera, Joan A. Puig-Butillé, Susana Puig, Josep Malvehy
Journal: Journal of the American Academy of Dermatology (2017)

Garment-related terms have been used to describe the pattern of distribution of giant congenital melanocytic nevi (GCMN).We sought to describe patterns of distribution of GCMN and propose a classification scheme.Photographic records of patients with GCMN from the Hospital Clinic of Barcelona were analyzed and a classification based on observed GCMN distribution patterns was created. The classification was independently applied by 8 observers to cases found in the literature. The interobserver agreement was assessed.Among 22 patients we observed 6 repeatable patterns of distribution of GCMN, which we termed the "6B": bolero (involving the upper aspect of the back, including the neck), back (on the back, without involvement of the buttocks or shoulders), bathing trunk (involving the genital region and buttocks), breast/belly (isolated to the chest or abdomen without involvement of bolero or bathing trunk distributions), body extremity (isolated to extremity), and body (both bolero and bathing trunk involvement). Our literature search found 113 cases of GCMN, which we were able to classify into 1 of the 6B patterns with an overall kappa of 0.89.Some patterns occur infrequently with a dearth of images available for analysis.The anatomic distribution of GCMN occurs in 6 recognizable and repeatable patterns.
Elevated plasma endoglin (CD105) predicts decreased response and survival in metastatic breast cancer patients
Authors: M. N. Vo, K. Leitzel, S. M. Ali, L. Demers, M. Wilson, D. Evans, A. Lipton
Journal: Journal of Clinical Oncology (2006)

10091 Background: Endoglin (CD105) is a proliferation-associated surface protein expressed by human vascular endothelial cells. It is a co-receptor for transforming growth factor (TGF) -β1 and TGF-β3, and plays a major role in angiogenesis. Upregulation of endoglin has been reported in endothelial cells of breast and colorectal cancers, and elevated levels of serum endoglin have been associated with tumor metastasis in breast cancer patients. Materials and Methods: Pretreatment EDTA plasma from 224 metastatic breast cancer patients enrolled in a phase III 2nd-line hormone therapy trial and 50 healthy post-menopausal female controls were assayed for soluble endoglin using a double-antibody sandwich ELISA from R&amp;D Systems (Minneapolis, MN). Results: In the post-menopausal female control group (n=50), the mean ± SD for plasma endoglin levels were 5.00 ± 1.84 ng/ml, with a range of 2.38–9.83 ng/ml. The upper limit of normal was defined as the mean + 2 SD (8.7 ng/ml) for plasma endoglin. In the breast cancer patient group, the mean ± SD for plasma endoglin was 6.40 ± 2.23 ng/ml, with a range of 3.00–19.79 ng/ml. Elevated plasma endoglin levels were detected in 26 of 224 patients (11.6%). Patients with elevated vs. normal plasma endoglin levels had a reduced clinical benefit rate (CR +PR+Stable)(15% vs. 42%)(p=0.01) to hormone therapy. Time to progression (TTP) was shorter for patients with elevated vs. normal plasma endoglin levels, but did not reach statistical significance (p=0.2). Patients with elevated plasma endoglin had decreased overall survival (median 645 days vs. 947 days)(p=0.005). Another plasma angiogenic marker, soluble Tie-2 receptor, did not significantly correlate with clinical outcome. A weak correlation existed between plasma endoglin and Tie-2 (r =.54, p&lt;0.00001). There was no correlation between plasma endoglin levels and the presence of bone metastasis vs. other sites of metastases. Conclusions: Elevated pretreatment plasma endoglin levels predicted for decreased clinical benefit rate to 2nd-line hormone therapy and a shorter overall survival in metastatic breast cancer patients. Circulating endoglin levels deserve further study for monitoring anti-angiogenic therapy. [Table: see text]
Developing a predictive model for cancer clinical trial accrual.
Authors: Wendy R. Tate, Lee D. Cranmer
Journal: Journal of Clinical Oncology (2014)

6557 Background: With increasing cost to conduct clinical trials, it is imperative to select trials rationally for local activation. We sought to create a predictive model with an outcome of anticipated accrual that can be used when considering a prospective clinical trial. Methods: This retrospective cohort study used 5.8 years of registry data to predict clinical trial accrual at our center. A negative binomial regression model was employed using variables known pre-study. These were abstracted from the OnCore clinical trial management system (Forte Systems, Madison, WI) and clinicaltrials.gov. Statistical significance was set a priori to 0.05. Normality and collinearity of independent variables and model fit were assessed. Variables modified an effect if the beta coefficient of the other independent variable changed by >10%. Accrual was predicted for the studies used to build the model. Results: The model included 207 trials with complete information. Mean accrual was 7.3 per trial (±18.4) and 55 (26.6%) trials did not succeed in accruing subjects locally. In univariate analysis, use of an investigational drug, local disease management team (DMT), number of national sites, use of local IRB, number of total months open nationally, months of accrual already completed, and overall proposed national enrollment were significantly associated with accrual. In multivariate analysis, DMT, proposed national enrollment, number of sites, use of local IRB, number of total months open, and the number of months already opened were significantly and independently associated with accrual. The full model was statistically significant (P<0.001), demonstrating these variables’ contribution to protocol accrual. Accrual was predicted at 94% of the actual value and maintained predictive value at multiple cutoff values. Conclusions: We identified key factors, both nationally and locally, associated with the accrual of subjects to a therapeutic clinical trial at our site. This model can aid in deciding whether a study is likely to accrue a desirable number of subjects. We plan to prospectively validate the model. If valid, this model would provide a quick and valuable metric in assessing trial success as well as planning resource allocation and estimating costs.
IMPACT OF ATRIAL FIBRILLATION ON OUTCOMES IN PATIENTS HOSPITALIZED WITH ST- SEGMENT ELEVATION MYOCARDIAL INFARCTION
Authors: Lohit Garg, Sahil Agrawal, Manyoo Agarwal, Aakash Garg, Amitoj Singh, Nirmanmoh Bhatia, Mahek Shah, Brijesh Patel, Sudip Nanda, David Cox
Journal: Journal of the American College of Cardiology (2017)
Characterizing the Effects of NN1731 and rFVIIa In Severe Hemophilia Patients with a Poor Laboratory Response to In Vivo Dosing of rFVIIa 90 μ g/Kg
Authors: Donald F Brophy, Erika J Martin, John Christian Barrett, Jan G Kuhn, Melinda Nolte, Ulla Hedner, Mirella Ezban
Journal: Blood (2010)
DELIVERABILITY AND SAFETY OF A NOVEL NON-INVASIVE, ULTRASOUND-BASED RENAL NERVE ABLATION SYSTEM IN A LARGE PRECLINICAL MODEL VIA HISTOPATHOLOGICAL EVALUATION
Authors: Armando Tellez, Michael Gertner, Krista N. Dillon, Don Christopher, Dane Brady, Serge Rousselle
Journal: Journal of the American College of Cardiology (2017)
Measuring Postconcussive Activity Levels of Patients—Reply
Authors: Douglas J. Wiebe, Michael L. Nance, Christina L. Master
Journal: JAMA Pediatrics (2017)

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T Cell Transcriptomes from Paroxysmal Nocturnal Hemoglobinuria Patients Reveal Novel Signaling Pathways
Authors: Kohei Hosokawa, Sachiko Kajigaya, Keyvan Keyvanfar, Qiao Wangmin, Yanling Xie, Olga Rios, Barbara Weinstein, Maria del Pilar Fernandez Ibanez, Danielle M. Townsley, Xingmin Feng, Neal S. Young
Journal: Blood (2016)
CARACTERÍSTICAS DIFERENCIALES EN CUANTO A DIFICULTAD, ÉXITO Y COMPLICACIONES ENTRE LA CPRE BILIAR Y PANCREÁTICA
Authors: JJ Vila, A Cebrián, E Sainza, J Urman, I Fernández-Urién, D Ruiz-Clavijo, B González De La Higuera, C Saldaña, J Jiménez-Pérez
Journal: Endoscopy (2015)

Introducción: La CPRE por indicación biliar (CPREb) es más frecuente en nuestro medio en parte debido a la percepción de dificultad y riesgo de la CPRE pancreática (CPREp).
Use of C-terminal deletion mutation of CD20 molecule to predict CD20 expression and time to progression after rituximab in non- Hodgkin lymphoma
Authors: Y. Terui, Y. Mishima, M. Yokoyama, Y. Mishima, K. Takeuchi, Y. Ito, S. Takahashi, K. Hatake
Journal: Journal of Clinical Oncology (2007)

8088 Background: Rituximab was the first chimeric antibody that is most frequently used for CD20-posistive B-cell lymphomas and gives better response and prognosis. However, resistance to rituximab is one of the important issues to be clarified in the increasing number of monoclonal antibody therapy, and we experienced the case whose lymphoma cells never expressed CD20 at the relapsed phase during rithuximab therapy. We investigated whether CD20 mutation is related to expression level of CD20, relapse or resistance to rituximab therapy, and prognosis. Methods: We analyzed 50 patients with fresh or relapsed/resistant B- cell lymphomas and DNA sequencing analysis of CD20 products from genomic PCR and RT-PCR was performed. CD20 mutants were subcloned by TA cloning, and tested CD20 expression after transfection to K562 cells. Results: Four types of CD20 mutations were found in 11 of 50 NHL patients (22.0%), which include C- terminal deletion (8.0%) extracellular domain (2.0%) transmembrane domain (2.0%) and early termination (10.0%). The group of C-terminal deletion mutations significantly showed lower CD20 expression (3.26, 95%CI = 0.09 to 6.89) than non-mutation (30.8, 95%CI = 22.4 to 39.2) (p 0.05). Although there was no significant difference between the groups with non-mutation and C-terminal deletion mutation in CR rate (49% versus 25%, Fisher's exact test; p = 0.6137), time to progression after rituximab therapy in C-terminal deletion mutation (7 months, 95%CI = 0 to 18 months) was significantly shorter than that in non-mutation (31 months, 95% CI = 18 to 44 months) by log-rank test (p = 0.0481). Non-mutation and early termination groups did not show any significant differences in TTP and CD20 expression. Conclusion: The important mutations of CD20 gene related to shorter duration to progression disease after rituximab therapy were discovered. Especially, point mutations bearing during rituximab therapy should be examined at progression disease after partial remission. No significant financial relationships to disclose.
Phase II Results of Ara-C and Idarubicin in Combination with the Selective Inhibitor of Nuclear Export (SINE) Compound Selinexor (KPT-330) in Patients with Relapsed or Refractory AML
Authors: Walter Fiedler, Michael Heuser, Jörg Chromik, Felicitas Thol, Carsten Bokemeyer, Susann Theile, Isabell Lebkuechner, Anne L. Kranich
Journal: Blood (2016)
The Combination of Bortezomib and Lenalidomide (VR) Consolidation Post-ASCT, in the Absence of Dexamethasone and Bisphosphonates, Improves Response Rates and Bone Metabolism in Newly Diagnosed Patients with Multiple Myeloma
Authors: Evangelos Terpos, Efstathios Kastritis, Dimitrios Christoulas, Evangelos Eleutherakis-Papaiakovou, Maria Gavriatopoulou, Despoina Kalapanida, Marios Bakogeorgos, Maria Roussou, Meletios A. Dimopoulos
Journal: Blood (2014)
PREDICTORS FOR 2-YEAR CARDIOVASCULAR EVENTS AFTER ROTABLATION IN SEVERE CALCIFIED LESION
Authors: Hisao Otsuki, Kentaro Jujo, Kazuki Tanaka, Hiroyuki Arashi, Masashi Nakao, Junichi Yamaguchi, Nobuhisa Hagiwara
Journal: Journal of the American College of Cardiology (2017)
PREVENTION OF CONTRAST-INDUCED NEPHROPATHY FOR COMPUTED TOMOGRAPHY (CT)-SCANNING IN TAVR WORK-UP: ONE-HOUR HYDRATION WITH SODIUM BICARBONATE IS NOT INFERIOR TO 24-HOUR SALINE HYDRATION
Authors: Martijn Stefan van Mourik, Floortje Van Kesteren, Esther M.A. Wiegerinck, R. Niels Planken, Jan Baan, Maartje Vis
Journal: Journal of the American College of Cardiology (2017)
Efficacy of Eculizumab in Gemcitabine-Induced Thrombotic Microangiopathy: Experience of the French Thrombotic Microangiopathies Reference Centre
Authors: Maximilien Grall, Francois Provôt, Jean-Philippe Coindre, Claire Pouteil-Noble, Dominique Guerrot, Ygal Benhamou, Dominique Bordessoule, Agnes Veyradier, Paul Coppo, Steven Grange
Journal: Blood (2016)
Successful Abolition of Prophylactic Cranial Irradiation in Children with Non-T Acute Lymphoblastic Leukemia (ALL) in the Japan Association of Childhood Leukemia Study (JACLS) ALL-02 Trial
Authors: Daiichiro Hasegawa, Junichi Hara, Souichi Suenobu, Yoshihiro Takahashi, Atsushi Sato, Nobuhiro Suzuki, Shinichiro Nishimura, Keiko Yumura-Yagi, Hirohide Kawasaki, Hiroki Hori, Akiko M. Saito, Koji Kato, Akihiro Iguchi, Tooru Kudo, Tatsutoshi Nakahata, Megumi Oda, Keizo Horibe
Journal: Blood (2011)
Pharmacogenetic investigation of pyrexia in patients treated with dabrafenib alone or in combination with trametinib from five melanoma studies.
Authors: Diptee A Kulkarni, Kijoung Song, Linda P Briley, Karen S King, Christine Ellen Dabrowski, Heather Lynn Del Buono, Bijoyesh Mookerjee, Jeffrey J. Legos, Colin F. Spraggs
Journal: Journal of Clinical Oncology (2015)

e20026 Background: Dabrafenib (D) and trametinib (T) single agents have superior efficacy compared with chemotherapy in metastatic melanoma with BRAF V600E/K mutation, and their use in combination (DT) delays development of resistance compared with D alone. Pyrexia is one of the most common adverse events in patients exposed to D (25–35%) with higher incidence (55–65%) seen in patients treated with DT. However, pyrexia is not associated with baseline clinical characteristics or efficacy, and the mechanism for development of pyrexia is not known. This exploratory pharmacogenetic study aimed to identify germline genetic variants associated with pyrexia by meta-analysis of melanoma patients from five clinical studies. Methods: Blood DNA samples from 1,006 melanoma patients treated with D or DT from clinical studies, BREAK-2, BREAK-MB, BREAK-3, COMBI-d and COMBI-v, were genotyped for genome-wide single nucleotide polymorphisms (SNPs). A total of 6 million SNPs and 50 Human Leucocyte Antigen (HLA) alleles (imputed using the genome-wide SNP data) were tested for association with pyrexia using case-control status. Pyrexia cases (n = 218) were defined as patients developing grade ≥ 2 pyrexia (according to NCI CTCAE, version 4.0) on treatment with D or DT. Controls (n = 361) were defined as patients with no pyrexia, despite sufficient duration of exposure to D or DT (duration by which > 90% cases had an event of pyrexia). This analysis had 80% power to detect associations of common SNPs or HLA alleles (minor allele frequency ≥ 5%) with pyrexia with odds ratios > 5.8 and > 3.2, respectively. Results: No SNP or HLAallele was significantly associated with pyrexia at the pre-specified multiple testing thresholds (p < 5.0e-08 and p < 0.001, respectively) in this analysis. Conclusions: This analysis of > 1,000 trial patients treated with either D or DT was adequately powered to detect relatively large genetic effects and suggests such an association is unlikely. The lack of HLA association is not consistent with drug hypersensitivity as a mechanism for pyrexia. Much larger sample sizes would be needed to detect any small to moderate genetic effects that may exist.
Health-Related Quality of Life in Patients with Advanced-Stage Follicular Lymphoma Receiving Consolidation with 90Y-Ibritumomab Tiuxetan (Zevalin®) of First Remission: Results from the Randomized Phase 3 First-Line Indolent Trial (FIT).
Authors: Kathleen Gondek, Sonalee Shah, Angelika Bischof-Delaloye, Ama Rohatiner, Gilles Salles, Barbara Putz, Michael Kunz, Anton Hagenbeek
Journal: Blood (2007)
Long-Term Follow-up of Patients with Corticosteroid-Refractory Graft-Versus-Host Disease Treated with Ruxolitinib
Authors: Robert Zeiser, Andreas Burchert, Claudia Lengerke, Mareike Verbeek, Kristina Maas-Bauer, Stephan Metzelder, Silvia Spoerl, Markus Ditschkowski, Matyas Ecsedi, Katja Sockel, Francis Ayuk, Ajib Salem, Flore Sicre de Fontbrune, Il-Kang Na, Penter Livius, Udo Holtick, Dominik Wolf, Esther Schuler, Everett Meyer, Petya Apostolova, Hartmut Bertz, Reinhard Marks, Michael Lübbert, Ralph Wäsch, Christof Scheid, Friedrich Stölzel, Rainer Ordemann, Gesine Bug, Guido Kobbe, Omid Shah, Robert S. Negrin, Mats Brune, Alexandros Spyridonidis, Annette Schmitt-Graeff, Walter J.F.M. van der Velden, Gerwin Huls, Stephan Mielke, Goetz Ulrich Grigoleit, Jurgen Kuball, Ryan P Flynn, Gabriele Ihorst, Jing Du, Bruce R. Blazar, Renate Arnold, Nicolaus Kröger, Jakob R. Passweg, Joerg Halter, Gérard Socié, Dietrich W. Beelen, Christian Peschel, Andreas Neubauer, Jürgen Finke, Justus Duyster, Nikolas von Bubnoff
Journal: Blood (2016)
SHORT-TERM INTRAVENOUS ILOPROST TREATMENT IMPROVES CLINICAL STATUS AND NEUROHUMORAL MEASURES IN CLINICAL WORSENING EPISODES OF PRECAPILLARY PULMONARY HYPERTENSION REGARDLESS OF THE BACKGROUND TREATMENTS: INSIGHTS FROM EUPHRATES STUDY
Authors: Cihangir Kaymaz, Ibrahim Halil Tanboga, Ozgur Y. Akbal, Hacer Ceren Tokgoz, Fatih Yilmaz, Aykun Hakgor, Nertila Poci, Canan Elif Yildiz, Sevim Turkday, Tugba Aktemur, Durmus Demir, Seda Tanyeri, Nihal Ozdemir
Journal: Journal of the American College of Cardiology (2017)