24 research outputs found

    Building a Healthcare Alliance for Resourceful Medicine Offensive Against Neoplasms in hematologY added value framework for hematologic malignancies: a comparative analysis of existing tools

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    OBJECTIVES: The Innovative Medicines Initiative-funded, multistakeholders project Healthcare Alliance for Resourceful Medicine Offensive Against Neoplasms in hematologY (HARMONY) created a task force involving patient organizations, medical associations, pharmaceutical companies, and health technology assessment/regulator agencies' representatives to evaluate the suitability of previously established value frameworks (VFs) for assessing the clinical and societal impact of new interventions for hematologic malignancies (HMs). METHODS: Since the HARMONY stakeholders identified the inclusion of patients' points of view on evaluating VFs as a priority, surveys were conducted with the patient organizations active in HMs and part of the HARMONY network, together with key opinion leaders, pharmaceutical companies, and regulators, to establish which outcomes were important for each HM. Next, to evaluate VFs against the sources of information taken into account (randomized clinical trials, registries, real-world data), structured questionnaires were created and filled by HARMONY health professionals to specify preferred data sources per malignancy. Finally, a framework evaluation module was built to analyze existing clinical VFs (American Society of Clinical Oncology, European Society of Medical Oncology, Magnitude of Clinical Benefit Scale, Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen, Institute for Clinical and Economic Review, National Comprehensive Cancer Network Evidence Blocks, and patient-perspective VF). RESULTS: The comparative analysis describes challenges and opportunities for the use of each framework in the context of HMs and drafts possible lines of action for creating or integrating a more specific, patient-focused clinical VF for HMs. CONCLUSIONS: None of the frameworks meets the HARMONY goals for a tool that applies to HMs and assesses in a transparent, reproducible, and systematic way the therapeutic value of innovative health technologies versus available alternatives, taking a patient-centered approach and using real-world evidence

    Transition pathways for young people with complex disabilities: exploring the economic consequences

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    Section five of "The RITE transition the only way forward". The RITE project was a four-year joint venture between the ACE Centre Oxford and the DARE Foundation

    Expression profiles of representative genes in selected GO categories.

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    <p>(A) Carbohydrate metabolic process; (B) Lipid metabolic process; (C) Proteolysis; (D) Amino acid metabolic process; and (E) Oxidation-reduction process. Significantly regulated genes –in at least one of the time points [immediately after packing (0 h), and at 48 and 72 h during transport] compared to the values prior to transport (basal) - are displayed in the heat maps. Columns and rows in the heat map indicate time points and genes, respectively. Color scale represents fold changes of gene expression. Genes belonging to more than one GO category is shown only once under a particular GO term. *indicates significant differences (P<0.05) at a particular time point compared to the expression of the respective genes prior to transport (basal).</p

    Validation of microarray results by quantitative real time PCR.

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    <p>Correlation plots indicating the relationship between qPCR results (fold change; Y- axis)) of six selected genes and the corresponding data from microarray analysis (X- axis). Fold changes of genes immediately after packing (0 h), and at 48 and 72 h during transport compared to the values prior to transport (basal) are displayed in the figure. Note that the fold changes for <i>scd</i> are 1/10<sup>th</sup> of the actual changes.</p

    Postovulatory maternal transcriptome in Atlantic salmon and its relation to developmental potential of embryos

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    Background Early development of an oviparous organism is based on maternally stocked structural, nutritional and regulatory components. These components influence the future developmental potential of an embryo, which is referred to as egg quality. Until zygotic genome activation, translational activity in a fish early embryo is limited to parentally inherited transcripts only. In this study, we asked whether egg transcriptome is associated with egg quality in Atlantic salmon (Salmo salar), which is capable of storing ovulated eggs in its abdominal cavity for a long time before spawning. Results We analyzed messenger RNA (mRNA) and micro RNA (miRNA) transcriptomes throughout the post-ovulatory egg retention period in batches of eggs from two quality groups, good and poor, classified based on the future developmental performance. We identified 28,551 protein-coding genes and 125 microRNA families, with 200 mRNAs and 5 miRNAs showing differential abundance between egg quality groups and/or among postovulatory ages. Transcriptome dynamics during the egg retention period was different in the two egg quality groups. We identified only a single gene, hepcidin-1, as a potential marker for Atlantic salmon egg quality evaluation. Conclusion The overlapping effect of post-ovulatory age on intrinsic egg developmental competence makes the quantification of egg quality difficult when based on transcripts abundance only

    Venn diagrams indicating the differentially expressed genes in the zebrafish liver during the transport process.

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    <p>The number of upregulated (A) and downregulated (B) genes immediately after packing (0 h) and at 48 and 72 h during transport compared to the values prior to transport (basal) are marked inside each circle. The genes which have ≥2 fold change in at least one of the pair wise comparisons (between the time points) and significantly altered (P<0.05) compared to basal values are included for each time point.</p

    Expression profiles of representative genes in selected GO categories.

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    <p>(A) Response to stress; (B) Response to chemical stimulus; and (C) Cellular homeostasis. Significantly regulated genes – in at least one of the time points [immediately after packing (0 h), and at 48 and 72 h during transport] compared to the values prior to transport (basal) - are displayed in the heat maps. Columns and rows in the heat map indicate time points and genes, respectively. Color scale represents fold changes of gene expression. Genes belonging to more than one GO category is shown only once under a particular GO term. *indicates significant differences (P<0.05) at a particular time point compared to the expression of the respective genes before transport (basal).</p
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