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Work Packages

MDS-RIGHT consists of seven work packages that address different aspects of MDS diagnosis and treatment. In each work package different research institutes are working together to address different themes. The individual work packages are described below.

Myelodysplastic syndrome
Myelodysplastic syndrome

Work Package 1: Health care interventions (HCI)

In this work package (WP) current health care interventions will be evaluated, using both established core outcome sets (COS) and COS that are newly developed within this WP. Health care use and associated health care costs will be quantified and evaluated. National policies regarding pharmacological treatment options for MDS will be evaluated as to their cost-effectiveness. Data from WP2, 3 and 4 will be used to stimulate personalised medicine, e.g., to identify which health care intervention works best for which patient, based on clinical and (epi)genetic information. The outcomes of this WP should lead to better clinical and regulatory decisions, which should lead to reduction of overtreatment (and therefore more efficient use of health care resources), effective patient management, evidence based therapy use and healthier ageing.

Partners in this work package

  • University of York
  • Groupe Francophone des Myélodysplasies
  • Fundación Instituto de Estudios de Ciencias de la Salud de Castilla y León

Deliverables

  • D1.1: Interim core dataset for model development and report on detailed statistical analysis plan.
  • D1.2: Preliminary analyses of clinical and cost effectiveness data based on D1.1.
  • D1.3: Final dataset incorporating data generated from WP2, WP3 and WP4.
  • D1.4: Updated models of D1.2 and generation of new core outcome sets incorporating data generated from WP2, WP3 and WP4.
  • D1.5: Final analyses of models and data from D1.3 to produce output to feed into WP5 and WP6.
  • D1.6: Final report of WP1 and preparation of manuscripts.

Work Package 2: Diagnostics and bioinformatics

The improvement of diagnostic procedures is an important driver of this WP. Up to now bone marrow biopsies are still required to diagnose MDS. This procedure is invasive and demanding. Besides that, current techniques of BM examination are not always diagnostic of MDS in subgroups of patients. The application of less invasive new molecular methods using peripheral blood and flow cytometry within the elderly AoE population will be explored and compared to bone marrow biopsies. In addition, genetic research will be performed. Genome-wide methylation patterns of 500 patients will be studied and related to genetic mutational analyses. Also, a large set of clinical, biological and quality of life data will be integrated, to assess the added diagnostic, prognostic and predictive value of the individual techniques used in WP2. The aim of WP2 is to lead to new and improved diagnostic methods and to individualized therapeutic choices.

Deliverables

  • D2.1: Periodic reports on mutational sequencing and epigenetic profiling in various cohorts.
  • D2.2: Periodic reports on immunophenotyping in various cohorts.
  • D2.3: Reports on ms data comparing 100 blood samples with 100 paired bone marrow samples.
  • D2.4: Periodic report on bioinformatic integration of data.

Work Package 3: Health-related quality of life issues in elderly patients with anaemia

Health related quality of life (HRQoL) is an important indicator of treatment efficacy. To correctly assess HRQoL, a new questionnaire (QUALMS-1) will be validated and patient compliance to complete these type of questionnaires will be improved by introducing electronic and patient friendly versions. Items and domains from HRQoL instruments considered relevant by patients will be identified and combined with existing core outcome sets (COS; WP1) to create new COS. Predictors for low HRQoL will be identified. HRQoL will be used to evaluate the effect of different treatment regimes on the different and newly identified subtypes (WP2) of MDS. Overall, the measures of HRQoL will be used for effect measurement of health care interventions and to evaluate the cost-effectiveness of interventions stratified for subgroups.

Deliverables

  • D3.1: A specific HRQoL questionnaire for MDS patients (QUALMS-1) validated in a large population of European MDS patients.
  • D3.2: A new treatment outcome set including patient reported, disease specific outcomes.
  • D3.3: Report of new predictors and prediction functions for impaired HRQoL.
  • D3.4: A list of HRQoL adapted treatments for each biologic MDS-subtype.
  • D3.5: Causal diagram with a description of direct and indirect effect estimates for different health-care interventions on HRQoL.
  • D3.6: Report on the newly defined burden of MDS on HRQoL by comparison with non-MDS matched reference cohorts.
  • D3.7: A set of measures and prediction functions of HRQoL.

Work Package 4: Outcome prediction

The aim of this work package is to improve the tailoring of interventions to the individual patient. Therefore, new and better treatment-outcome prediction models will be developed and used. Molecular defects with prognostic value identified in WP2 will be integrated in these models. Statistical techniques will be used to identify new biological subgroups of MDS with specific biological pathways that might be good candidates for the development of targeted therapies. Data from WP2 will be used to identify if molecular defects associated with MDS exist in elderly patients with unexplained anaemia. If this is the case, this will enable better diagnosis of anaemia and use of therapeutic clinical interventions proven to be effective in MDS treatment.

Deliverables

  • D4.1: Report describing prognostic impact of newly defined individual genetic defects and groups of associated genetic defects on response to the existing health care interventions (HCI) (together with WP1).
  • D4.2: List of newly identified genetic defects which support development of novel drugs.
  • D4.3: Report of early treatment response indicators supporting a targeted use of existing HCI.
  • D4.4: Report of new specific genetic signatures which support minimal diagnostic criteria recognizing MDS in older individuals with anaemia, and guiding appropriate treatment.
  • D4.5: Report on newly generated, high-quality health economic evidence of benefit arising from implementation of personalised MDS care strategies.

Work Package 5: Guideline development

Existing guidelines are mainly based on expert opinions and retrospective observational data in selected patient groups. The first step in this WP will be to improve the existing European guidelines published in 2013 by the European LeukemiaNet (ELN) in close co-operation with both European and non-European MDS-experts. In parallel, new and evidence-based guidelines will be developed, based on newly identified prognostic subgroups within MDS-RIGHT. The modified existing guidelines will be compared with the MDS-RIGHT based guidelines and discussed with relevant experts. Interactive web-based guidelines and interactive guideline algorithms will be developed to allow professional health care providers to enter individual patient data and obtain advice for individual patients based on existing and newly defined dynamic prognostic response parameters. The European MDS competence network that will be developed in WP6, will serve as a platform for providing up-to-date, evidence-based information and regulatory guidance to health care providers.

Deliverables

  • D5.1: Approved European and international guidelines uploaded at new ELN-linked website.
  • D5.2: Yearly electronic updates of the evidence-based therapeutic guidelines based on the ongoing analyses of MDS-RIGHT (WP1-4) and consensus within the Steering Committee.
  • D5.3: Report of socio-economical aspects of the elderly low risk MDS population.
  • D5.4: Scientific report describing the flexible electronic web-based system with permanently updated evidence-based guidelines for MDS treatment.
  • D5.5: Report on the endorsement of the new evidence-based guidelines for MDS patients in all institutes of the MDS-RIGHT consortium and for MDS patients outside the MDS-RIGHT institutions.
  • D5.6: Updated patient handbooks of the national MDS patient groups containing relevant information on the MDS-RIGHT guidelines.
  • D5.7: Panel methodology and annual panel meetings.

Work package 6: Dissemination, exploitation and communication

All new findings on health care interventions, HRQoL, new diagnostic tools, new treatment-outcome prediction models and new guidelines have to find their way to the health care providers treating MDS-patients. This is the ultimate goal of WP6, in which a European MDS competence network and a platform/website for communication with stakeholders will be developed. The MDS competence network will closely collaborate with the national MDS patient groups in Europe and the international MDS Foundation. The stakeholder network will serve as an easily accessible information source for both patients and professionals, as a platform for discussion and as a lobby organisation for MDS-patients towards other stakeholders. Stakeholders and end-users will be continuously involved in WP6 to increase acceptance and compliance with treatment. The organisation of workshops and conferences, participation in exhibitions, scientific publications and PR activities are also an important aspect of this WP.

Deliverables

  • D6.1: Report of dissemination plan.
  • D6.2: Discussion platforms and a common website for stakeholder interaction.
  • D6.3: Completed catalogue of all MDS caregiver cooperative groups patient support groups and existing documents (written, website) in participating countries.
  • D6.4: Yearly MDS Stakeholders Newsletters.
  • D6.5: Report on the participation in international patient support group meetings.
  • D6.6: Report on two meetings with physicians (professional stakeholders) to inform about state-of-the-art in the field of MDS.
  • D6.7: Yearly updates of MDS website. D6.8: Promotion of educational and scientific meetings, pocket cards, posters, flyers, exhibition booth for meetings.

Work Package 7: Project management and consortium

This WP mainly consists of managing, monitoring and coordinating the different WPs and other aspects of MDS-RIGHT. Besides this, the organisation of project and project related meetings and the reporting to, and liaising with, the European Commission (EC) are important parts of WP7.

Deliverables

  • D7.1: To manage and monitor a large prospective, longitudinal, observational European-wide study on current health care interventions for elderly patients with lower-risk MDS.
  • D7.2: To manage implementation of new technological diagnostic methodologies.
  • D7.3: To manage implementation of new COS in the established HCI.
  • D7.4: To monitor, track and control deviations due to progress, costs, financial and scheduling changes.
  • D7.5: To ensure that the required reporting is prepared and delivered in a timely manner according to quality assurance standards and homogeneity.