A 2-stage Phase II study evaluating the safety and efficacy of combination pomalidomide and low dose dexamethasone therapy in patients with relapsed myeloma previously treated wtih lenalidomide maintenance post Autologous Stem Cell Transplant (LEOPARD follow-on study)

开始日期2015-06-08

申办/合作机构

Alfred Health

[+1]

ACTRN12616000777493 / Completed临床2期

Safety and tolerability of lenalidomide consolidation post allogeneic stem cell transplant for patients with high risk multiple myeloma failing to achieve stringent Complete Response

开始日期2015-03-10

申办/合作机构

Alfred Health

[+1]

100 项与 Celgene Pty Ltd. 相关的临床结果

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2019-05-01·Research in Social and Administrative Pharmacy3区 · 医学

Consumer opinions on adverse events associated with medical devices

3区 · 医学

Article

作者: Fois, Romano ; McGarry, Theresa ; Tong, Vivien ; Aslani, Parisa ; Spagnardi, Sarah ; Cook, Jane ; Chen, Timothy F ; Moore, Carter ; Whitelock, Karen ; Hamrosi, Kim ; Peters, Rodney

INTRODUCTIONPost-market surveillance of medical devices relies on compulsory and voluntary reports. Although direct consumer reporting of medical device-related adverse events (AEs) is available in Australia, the proportion of consumer reports has remained low. Limited qualitative research has previously explored consumer insights on AEs associated with medical devices and in particular, AE reporting.OBJECTIVETo explore consumer opinions on AEs related to medical devices, and their knowledge of, experiences with, and views on, the reporting of medical device-related AEs.METHODSFocus groups (n = 4; total of 29 participants) were conducted in metropolitan Sydney, Australia. Focus group discussions of approximately 1.5 h in length centred on consumers' understanding of AEs, opinions on AEs and their previous experiences, views on medical device benefits and harms, and actions taken (or potential actions) in response to AEs. With participant consent, discussions were audio-recorded, transcribed verbatim, and thematically analysed.RESULTSParticipants regarded medical device-related side effects to be unexpected AEs associated with their use. Where there was a clear need for the medical device itself, potential improvement in quality of life took precedence over potential harms. Most participants had not experienced negative issues with their medical device(s). There was poor awareness among participants of an existing direct consumer AE reporting system for medical devices. Despite this, the value of reporting was acknowledged. Severity of the AE was a key motivator for potential AE reporting.CONCLUSIONSFurther efforts are necessary to improve consumer awareness of available AE reporting systems to better support post-market surveillance and safe medical device use.

2016-02-01·Advances in Therapy3区 · 医学

The Effectiveness of an Educational Brochure as a Risk Minimization Activity to Communicate Important Rare Adverse Events to Health-Care Professionals

3区 · 医学

Article

作者: Di Vito-Smith, Michelle ; Bester, Nicolette ; McGarry, Theresa ; Riffkin, Michael ; Bwire, Robert ; Pilot, Richard ; Kaehler, Stefan

INTRODUCTIONEducational brochures are an important tool for communicating risk to health-care professionals. It is important to evaluate the impact of any risk minimization tool to understand the effectiveness of the strategy. The objective of this study was to assess the effectiveness (i.e., respondents' awareness and understanding of the communication) of a targeted educational brochure distributed to health-care professionals (HCPs) as a risk minimization strategy for the communication of new rare and important adverse events (AEs).METHODSA prospective, non-interventional, online survey was performed following distribution of a specifically designed brochure highlighting new and important adverse events to a targeted HCP population, consisting of known users of the target medicine, as represented by a commercial database. Predefined multiple-choice survey questions assessed overall HCP awareness of the brochure and understanding and retention of information in those HCPs who reported receiving the brochure.RESULTSThe educational brochure was sent to a total of 565 HCPs; 121 (21.4%) responded to the survey. The majority of respondents (95.0%) had previously prescribed or dispensed the target medicine. In all, 88 (72.7%) respondents said they had received the educational brochure, of whom 95.5% stated they had at least scanned the main points. More participants who had received the brochure (86.4% to 96.6%) answered the five individual survey questions correctly compared with those who did not (51.5% to 97.0%); this was significant for four out of five questions (P ≤ 0.005). Significantly more HCPs who received the brochure achieved the predefined pass rate (at least four of five questions answered correctly) compared with HCPs who did not receive the brochure (93.2% vs 57.6%, respectively; P = 0.000003).CONCLUSIONSDistribution of targeted educational brochures may be an effective risk minimization strategy to raise HCP awareness of new rare and important AEs; educational brochures may also be an effective channel for sharing information on how these AEs can be best managed and on the importance and means of reporting AEs.FUNDINGCelgene Pty Ltd, Melbourne, Australia.

2015-12-03·Blood

An Integrative Genomics Approach Uncovers the Basis of Resistance to AZA Therapy in MDS and CMML

作者: Wilson, Susan R. ; Lynch, Kevin ; Woll, Petter S. ; Thoms, Julie A.I. ; Huang, Yizhou ; Mufti, Ghulam J ; Kulasekararaj, Austin G. ; Hellstrom-Lindberg, Eva ; Pimanda, John E. ; Papaemmanuil, Elli ; Chandrakanthan, Vashe ; Pellagatti, Andrea ; Kumari, Ashu ; Campbell, Peter J ; Galbraith, Sally ; Wong, Jason W.H. ; Mackenzie, Karen Lee ; Tursky, Melinda L. ; Tobiasson, Magnus ; Verma, Arjun ; Knezevic, Kathy ; Beck, Dominik ; Jacobsen, Sten Eirik ; Lindeman, Robert ; Richards, Laura A. ; Boultwood, Jacqueline ; Unnikrishnan, Ashwin

AbstractBackground:Myelodysplastic Syndrome (MDS) and Chronic Myelomonocytic Leukaemia (CMML) are haematological disorders that develop in haematopoietic stem or progenitor cells (HSPCs) and are characterised by ineffective haematopoiesis. 5'-Azacitidine (AZA), a DNA demethylating agent, is the primary drug for the treatment of high-risk MDS and CMML and response is associated with improved survival benefits. However, only half of treated patients will ever respond to AZA and the molecular basis for poor response is currently unknown. There are few alternative therapies for the non-responders. Additionally, AZA response is rarely sustained and a substantial fraction of responders will eventually relapse. The in vivo effect of AZA therapy on dysplastic cells in responders is unclear and there are no predictive markers for impending relapse in responders.Methods:To address these fundamental questions, we enrolled 18 high-risk MDS and CMML patients on a compassionate access program for AZA in Australia. Bone marrow was collected at seven different points - before treatment; through 6 cycles of treatment; and at up to two years after initiation - and we isolated high-purity CD34+ HSPCs (Figure A). 10 patients had a complete response while 8 were poorer responders. We performed RNA-seq to query the transcriptomes (and validated by Fluidigm-based PCR) and deduced the clonal evolution in the bone marrow in response to AZA therapy (by whole exome-sequencing, followed by targeted capture resequencing, and genotyping of individual CFU colonies). Our findings were validated in an independent cohort of 57 patients. We used flow cytometry to develop a clinically relevant prognostic assay for AZA resistance and developed a novel stromal co-culture based functional drug testing platform to rationally discover combinational drug therapies to overcome AZA resistance.Results:We hypothesised that primary AZA resistance would be driven by pre-existing molecular differences between responders and non-responders. Analysis of the pre-treatment RNA-seq data strikingly revealed the differential expression of 1148 genes between responders and non-responders (Figure B). Pathway analyses of these genes indicated that cell cycle and DNA damage response pathways were relatively up-regulated in responders compared to non-responders, indicating that HSPCs of non-responders are more quiescent compared to responders (Figure C). We validated these gene expression differences in independent patient cohorts from the U.K. and Sweden (n=57; 27 responders, 30 non-responders). We then adapted a flow cytometry based assay, amenable to prospective use in a clinical diagnostic setting, to directly detect the increased quiescence of CD34+ CD38+ haematopoietic progenitors in unsorted bone marrows of non-responders across all cohorts (Figure D). Finally, to reverse the quiescence of progenitor cells of non-responders, and make them more susceptive to AZA, we leveraged our RNA-seq discoveries to target pathways that were relatively up-regulated in non-responders. Using a stromal co-culture drug testing platform that we developed, we discovered that inhibiting integrin-linked signalling combinatorially with AZA improved the functionality of dysplastic cells (Figure E). Additionally, dysplastic cells were particularly sensitive to the inhibition of the mTOR pathway.To trace the fate of dysplastic cells as patients undergo AZA therapy, and thereby understand the basis of eventual relapse in responders, we performed whole exome sequencing of all patients (Figure F). Using the mutations as "molecular barcodes", we deduced the clonal architecture in each individual and observed the clonal evolution that occurred in response to AZA treatment. Combined with genotyping of CFU colonies grown in vitro, we have discovered that clonal haematopoiesis originating from resistant multipotent cells bearing mutations persists even upon complete response and forms the basis for eventual relapse (Figure G).Conclusions:Our findings, across independent cohorts and relevant to both MDS and CMML, have immediate clinical utility not simply to prospectively identify AZA non-responders but also by suggesting combinatorial therapies that could improve response. Finally, elucidating the in vivo effects of AZA therapy lay the foundation for developing more durable treatments.Figure 1. Figure 1.DisclosuresKulasekararaj: Alexion: Consultancy. Lynch:Celgene: Employment, Equity Ownership. Campbell:14M genomics: Other: Co-founder and consultant.

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