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Multivariate analysis of patients with acute myeloid leukaemia (AML) at day 28 in clinical or pathologic complete remission showed that DNMT3A R882 mutations were associated with an increased risk of minimal residue disease (MRD) at the time of complete remission. DNMT3A R882 mutations may therefore be important in chemoresistance in AML. A novel genetic mouse model was used to study the role of DNMT3A R882 mutations in AML pathogenesis and chemoresistance. Presented by Olga Guryanova, MD, PhD, Memorial Sloan Kettering Cancer Center, New York, NY, at the 20th Congress of the European Hematology Association (EHA) European Medical Journal Website: http://emjreviews.com/…

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The randomised, controlled phase 2 SORAML trial showed that the addition of the tyrosine kinase inhibitor, sorafenib, to chemotherapy improved event-free survival and relapse-free survival in younger patients with acute myeloid leukaemia (AML). Presented by Gerhard Ehninger, MD, Technical University Dresden, Dresden, Germany, at the 20th Congress of the European Hematology Association (EHA) European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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The identification of eight new Growth Factor Independence 1B (GFI1B) variants in families with bleeding and platelet disorders may result in the identification of therapeutic targets for these bleeding disorders. Half of the patients had thrombocytopaenia. Several patients showed similar platelet abnormalities as the earlier reported patients with the GFI1B defect, such as decreased ?-granule numbers and/or platelet CD34 expression. The GFI1B protein is known to regulate gene expression. Almost all GFI1B variants were still active, while one had lost this function in transcription repression assays. If the new GFI1B variants are disease-causing, this would indicate a distinct molecular mechanism…

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The phase 3 PERSIST-1 study showed that pacritinib, a selective inhibitor of Janus Kinase 2 (JAK2) and Fms-Like Tyrosine Kinase-3 (FLT3), is more effective than the best available therapy (BAT) at improving disease symptoms and quality of life (QoL) for patients with myelofibrosis. Patient-reported outcomes (PROs), which have become important for approval of new therapies, was also measured to determine whether pacritinib reduced patients symptom burden and helped them feel better. Presented by Ruben Mesa, MD, FACP, Mayo Clinic, Scottsdale, AZ, at the 20th Congress of the European Hematology Association (EHA) European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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The European Treatment Outcome Study (EUTOS) investigated the treatment and treatment outcome of patients with chronic myeloid leukaemia (CML). Data of 2904 newly diagnosed CML patients in 20 countries or prespecified regions of Europe were recorded in a web-based registry. 94% of patients were diagnosed in early stage of disease and received treatment with imatinib according to the European LeukemiaNet (ELN) recommendations. Newer treatment options were used by patients in clinical trials or with chromosomal abnormalities. Sub-standard treatments were used rarely, but mainly in older patients. Patient survival probabilities were similar to clinical trial results after adjusting to risk profile.…

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In a phase 1b, open-label, dose-escalation, multicentre study of patients with relapsed or refractory chronic lymphocytic leukaemia (CLL), the combination of the BCL-2 inhibitor, venetoclax (ABT-199/GDC-0199), and monoclonal anti-CD20 antibody, rituximab, showed a tolerable safety profile and induced deep and durable responses, with 41% of patients achieving complete response (CR). Presented by Andrew Roberts, MB BS, PhD, FRCPA, FRACP, Royal Melbourne Hospital, Melbourne, Australia, at the 20th Congress of the European Hematology Association (EHA) European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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ELOQUENT-2, a phase 3, randomised, open-label study of lenalidomide and dexamethasone with or without elotuzumab in patients with relapsed or refractory multiple myeloma, met its primary endpoints of progression-free survival and overall response rate. The two-year progression-free survival rate was 41% in the elotuzumab group versus 27% in the control group. The overall response rate in the elotuzumab group was 79% versus 66% in the control group. Presented by Meletios Dimopoulos, MD, National and Kapodistrian University of Athens School of Medicine, Athens, Greece, at the 20th Congress of the European Hematology Association (EHA) European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews…

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At the 2015 American Society of Clinical Oncology (ASCO) Annual Meeting, James Larkin, FRCP, PhD, Consultant Medical Oncologist at The Royal Marsden, London, UK, discusses the phase 3 CheckMate 067 trial. The results of this trial demonstrate a superior progression-free survival of nivolumab (a PD-1 checkpoint inhibitor) and ipilimumab (a CTLA-4 checkpoint inhibitor) regimen or nivolumab monotherapy versus ipilimumab monotherapy in previously untreated advanced melanoma. European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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In the phase 3 NCCTG N0574 (Alliance) trial, patients with 1 to 3 small brain metastases were randomised to receive stereotactic radiosurgery (SRS) alone or SRS followed by whole brain radiation therapy (WBRT). The primary endpoint of the trial was to determine the impact of SRS alone or with SRS combined with WBRT on cognitive progression. At 3 months, cognitive decline in the combination arm was 91.7% compared with 63.5% in the SRS-alone arm (P = .0007). Presented by Jan Buckner, MD, Mayo Clinic, Rochester, MN, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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A phase 3, randomised controlled trial showed that elective neck dissection improved overall survival by 12.5% and reduced risk of death by 36% when compared with a wait-and-watch approach for therapeutic neck dissection in early, node-negative, oral squamous cell carcinoma. Presented by Anil K. D’Cruz, MS, DNB, Tata Memorial Hospital, Mumbai, India, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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Data from the Childhood Cancer Survivor Study (CCSS), a retrospective cohort study of children diagnosed with cancer from 19701999 at 31 participating institutions, suggests the strategy of modifying therapy to reduce the occurrence of late effects, and promotion of early detection, is significantly reducing the observed late mortality in this population. Presented by Gregory T. Armstrong, MD, MSCE, St. Jude Children’s Research Hospital, Memphis, TN, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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The randomised, double-blind, phase 3 CheckMate 067 trial showed that nivolumab alone or nivolumab plus ipilimumab significantly improved progression-free survival (PFS) in patients with metastatic melanoma compared with ipilimumab alone. The median PFS in the group treated with the combination therapy was 11.5 months compared with 6.9 months in the nivolumab monotherapy group and 2.9 months in the ipilimumab monotherapy group. Presented by Jedd D. Wolchok, MD, PhD, Memorial Sloan Kettering Cancer Center, New York, NY, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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A phase II trial suggests that anti-CD38 antibody daratumumab is effective as a standalone therapy for heavily treated multiple myeloma. In a group of 106 patients whose disease worsened after at least three prior treatments, nearly one in three responded to daratumumab, and disease progression was delayed by 3.7 months, on average. The estimated one-year overall survival rate was 65%. Presented by Saad Zafar Usmani, MD, Levine Cancer Institute, Carolinas Healthcare System, Charlotte, NC, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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Interim analysis of a phase III study finds that adding the new anti-CD20 monoclonal antibody obinutuzumab to standard bendamustine chemotherapy significantly delays progression of indolent non-Hodgkin lymphoma (NHL). Among patients for whom rituximab therapy no longer provided benefit, the average duration of remission was 29.2 months after receiving the new combination vs. 14 months after bendamustine alone. The trial was stopped early based on these encouraging results. Presented by Laurie Helen Sehn, MDCM, MPH, BC Cancer Agency, Vancouver, Canada, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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Findings from the PERSIST-1 study of patients with myelofibrosis suggest that pacritinib is significantly more effective than best available therapy (BAT), which includes a range of off-label treatments. At 24 weeks of treatment, 19.1% of patients on the pacritinib arm experienced spleen shrinkage, compared to only 4.7% of patients on the BAT arm. Presented by Ruben A. Mesa, MD, Mayo Clinic Cancer Center, Scottsdale, AZ, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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An interim analysis of a large phase III study suggests that the combination of ibrutinib and bendamustine/rituximab (BR) improves outcomes for patients with chronic-lymphocytic leukemia (CLL) that worsened despite prior therapy. At a median follow-up of 17 months, patients who received ibrutinib and BR had an 80% lower risk of disease progression or death than those who received placebo and BR. Based on this striking benefit, patients were permitted to cross over from the placebo group to receive ibrutinib. Presented by Asher Alban Akmal Chanan-Khan, MD, Mayo Clinic, Jacksonville, FL, at ASCO 2015 European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews…

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This was my discussion at the ASCO Annual Meeting June 1, 2015 with Dr. Petros Grivas, medical oncologist specializing in prostate and bladder cancers at the Cleveland Clinic. He discusses his work in translating genomic profiling to the clinic through clinical trials. http://my.clevelandclinic.org/staff_directory/staff_display?doctorid=18967

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Findings from a phase I/II study suggest that nivolumab is safe and effective in advanced liver cancer. Based on the results of the phase I part of the study, eight (19%) of the 42 evaluable patients responded to the anti-PD-1 antibody with tumour reduction beyond 30%. More importantly, the responses have been durable and surpassed 12 months in four patients. The overall survival rate at 12 months was 62%. Presented by Anthony B. El-Khoueiry, MD, University of Southern California Norris, Comprehensive Cancer Center, Los Angeles, CA at ASCO 2015, Chicago, IL European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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Findings from a randomised phase III study indicate that PD-1 immunotherapy is an effective treatment option for patients with non-squamous, non-small cell lung cancer (NSCLC). Among patients with advanced disease that worsened after receiving platinum-based chemotherapy, those treated with nivolumab lived on average three months longer than those treated with docetaxel chemotherapy. Presented at ASCO 2015, Chicago, IL by Professor Luis Paz-Ares, MD, PhD, Hospital Universitario 12 de Octubre, Madrid, Spain European Medical Journal Website: http://emjreviews.com/ Twitter: https://twitter.com/EMJReviews Facebook: https://www.facebook.com/emjreviews

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