The paediatric transplantation (of solid organs and hematopoietic progenitors) has increased survival in children with end-stage disease, becoming one of the main treatment options for this population. However, there are significant challenges associated with optimising the effects of these interventions:
i)The mechanisms associated with tolerance to graft rejection;
ii) The pre-transplant -and surgical techniques and care procedures; ii) post-transplantation care;
iii) The specificities associated with this population group and type of disease;
iv) The social and economic impact of these surgeries on patients, their families and health systems.
Since these factors are key to the success of the process and a common link in various types of transplantations, there is a need to implement and standardise recent improvements in this field for routine use, directing them towards prevention, improvement of the surgical procedures themselves, treatment of chronicity and the appearance of secondary diseases associated with transplantation.
As a result of these activities, the paediatric transplantation network will achieve to increase life expectancy and quality of life of paediatric patients requiring a solid organ or hematopoietic progenitor transplantation and their families, and will provide added value,
European Reference Networks (ERNs) are virtual networks involving healthcare providers across Europe. They aim to tackle complex or rare diseases and conditions that require highly specialised treatment and a concentration of knowledge and resources for the purpose of improving access to diagnosis, treatment and the provision of high-quality healthcare for patients no matter where they are in Europe.
TransplantChild is an ERN for Paediatric Transplantation, both Solid Organ Transplantation (SOT) and Hematopoietic Stem Cell Transplantation (HSCT) which are low-prevalence and complex conditions that require highly specialized expertise and resources
Paediatric transplantation (PT), both solid organ (SOT) and hematopoietic stem cell transplantation (HSCT), is the only curative procedure for several end stage rare diseases. Advances in paediatric surgical procedures allow transplantation of virtually all organs and short term success is achieved, among other factors, due to the efficacy of current immunosuppresive regimens.
Transplanted individuals shift their primary disease to a chronic condition of immunosuppression to avoid rejection, requiring a proper monitoring and handling of post-transplant complications.
ERN TransplantChild aims:
• To improve life expectancy and quality of life for EU paediatric patients requiring transplantation, by:
o Ensuring their access through the network to the best possible advice on diagnostic, care practices and support procedures, considering a cross-cutting approach.
o Developing and joining efforts within the Network&stakeholders for integrative, innovative and better procedures,information, training, knowledge and expertise.
• To become a network of excellence for patients / families based on their health outcomes achieved throughout the life of the transplanted patient. Is based on innovative monitoring techniques and evidence-based treatments. Has highly specialized multidisciplinary teams committed to improving the quality of life of patients and their families, research, and dissemination of knowledge.
The cross-cutting approach will allow:
– The identification of common topics to all types of transplants such as clinical, personal and socio-economic issues;
– The improvement of the transplant quality of life of children and their families by:
a. Preventive practices to anticipate and minimize patients risks;
b. Treatment standardisation;
c. Harmonisation of clinical best practices;
d. Care humanisation;
e. Improving chronicity approach and the treatment of secondary diseases related to PT;
f. The provision of psycho-social support in different phases of patients’ life;
g. The provision of grounds for patients’ empowerment.
– The research and innovation in clinical areas such surgical procedures, pharmacological toxicity, immunosuppression and its long -term consequences, tolerance and rejection, GVHD, infections, innovative biological and cellular therapies, monitoring for personalised-precision medicine, etc.
– An improvement to obtain relevant data that will support innovation due to the higher amount of transplant cases where common complications are the focus. This view is specially needed in PT as compared to adult transplantation in which the number of patients per transplanted organ is higher.
– The establishment of synergies from knowledge and results from different types of transplants (e.g. knowledge in HSCT is being applied in SOT).
– The integration and share of knowledge and clinical expertise among all clinical teams and support departments.
– The training of the professionals that belong to the different health care levels.
– Social awareness.
As established in the Strategic approach of the Network, the following strategic areas have been approved in order to ensure the achievement of the mission and vision of the Network and to fulfil the requirements of the Delegated and Implementing Decision (2014/286/EU and 2014/287/EU):
- To improve patient healthcare;
- To harmonise clinical best practices;
- To harmonise research and innovation;
- Spread knowledge;
- Education and training;
- Network organization, quality and safety.
A systematic for Strategic areas deployment, monitoring and follow up was needed in order to ensure the objectives as established in this Strategic Approach of the Network. This systematic is aimed at defining the required concrete and concise activities, obtaining reliable information about their progress and facilitating their monitoring and control by the Board of the Network (BoN). Progress results shall allow the updating of the Strategic Approach when facing new scenarios.
Thus, the Board of the Network (BoN) has developed and approved the ERN TransplantChild Multiannual Work Plan for the following 5 years and an Annual Work Plan including all the actions to be developed grouped in six interrelated Work Packages or Action Plans (WP).
These WP specify activities, timeframe and deadlines, related indicators, dissemination actions and means to monitor and follow-up the achievement of planned results allowing the earliest identification of any arising issue.
The Executive Operating Committee (EOC) through six Working Groups (WG) coordinates the development of these WP. Each of them is constituted by:
- A Technical Director, appointed by the BoN.
- A designated member of the EOC as Coordinator of the WG.
- HCP members.
The Technical Director and the Coordinator of the corresponding WG are responsible for the coordination, monitoring and control of its activities, results, outcomes and deliverables.
The EOC is responsible for the Action Plans monitoring and control activities during its periodic meetings. In addition, the BoN review the Action Plans progress and approval the proposals during its periodic meetings (at least three times a year).
Approved actions and deliverables are disseminated to all HCP members in accordance with the provisions of the Communication Plan, the Action Plans and the SOP for Sharing Knowledge.
The activities carried out were focused on:
- Addressing the process of managing patient cases from HCPs members of the Network;
- The identification of key aspects for the joint achievement of the Network objectives;
- The development of actions for the joint achievement of the Network strategic objectives: specific objectives
- The proper performance and improvement of the Network by the development of organizational structures, methodologies and tools.
According to the established WP, the main outputs are:
- The Network initial map developed and the Healthcare committee rules established and disseminated to the HCPs members of the Network.
- A SOP for the functioning of the Healthcare committee and the Advisory group has been developed.
- The operational activities for the use of the CPMS tool have also been disseminated
- A List of formal clinical guidelines centred on the different aspects of transplant activities is available within the network.
- A List of Clinical Practice Guidelines (CGP) and new CPG disseminated between HCP&MS involved into the Network.
- Improvement of the the Network research potential and opportunities by collaborating and participating in different European programmes and initiatives.
- Explicit Critical Knowledge: Dissemination activities performed (website updates, participation in workshops, documents, etc.)
- Means to be used for knowledge management.
- Different cooperation /communication/dissemination strategies has been established with external stakeholders.
- Main stakeholders groups were identified.
- Up to 20 Patients’ Associations (Tx related) were also contacted and involved within the Network.
- An Education&Training Plan stated and disseminated.
- The methodology for managing E&T activities is established.
According to the established WP, the main outcomes are:
- The first approach to the Network map was developed and published in the Network website.
100% of the HCP&MS involved into the Network know the mapping on Tx in order to facilitate the access of HCP-Professionals -patient cases to information and experience of TransplantChild
- The methodology to manage patient cases through the CPMS tool was established.
100% of the HCP involved into the Network know how to manage patient cases.
- Different User Manuals have been provided by the EC to support the work of the ERNs and these documents and information have been disseminated to the members of the Network via email.
- Prioritized CPGs to be developed within the Network was carried out during the reporting period. The first CPG will be focused on the relevant aspects of immunosuppressive strategies and monitoring in paediatric transplantation.
- A SOP was developed by the WG for the constitution and definition of responsibilities for the CPG subgroups needed for the CPG development. This SOP was disseminated to the HCPs members of the Network and further discussed in the BoN meetings.
- Research main needs werwidentified.
- 100% of the HCP involved into the Network know research projects and research gaps.
- The research WG activities were focused on strengthening the research position of the Network (see section 1.3 of this report for further details).
- The Coordinating centre of the Network has carried out visits to the rest of the HCPs members of the Network focusing on raising awareness and spreading information about the Network.
- In addition, members have participated in workshops in different external forums (e.g. RD-Action initiatives such as the workshop on ´How can ERNs generate, appraise and utilise clinical practice guidelines, to enhance the impact of consensus guidelines in national health systems? ´, workshops related to the CPMS tool organized by the EC and workshops with the EJP, EURORDIS, the ERN Coordinator Group). Several posters related to the Network have also been presented in different forums: the Transplantation Society (TTS) and the European Bone Marrow Transplantation (EBMT).
- 1 document (addressed to professionals) related to patient care shared into the Network.
- The Network has set up different communication and collaboration strategies with several stakeholders: Orphanet, Organización Nacional de Trasplantes, EMTB, RD-Action, EURORDIS, EJP, IRDIC, RD Connect and ESPHGAN.
- Up to 20 Patients’ Associations from different MS included in the Network has been involved during the reporting period.
- 3 new education and training activities performed (webinar, face-to-face course, educational workshop, etc.).
- The criteria for the integration of new members in the Network were reviewed establishing mandatory and recommended requirements during the reporting period.
100% of HCP fulfil the stated mandatory requirements.
The activities developed by the Network and the results obtained during its first year of operation have a direct impact on the following target groups:
- HCPs and their multi-professional teams:
Giving HCP members and their teams access to a much larger pool of expertise, through the HCC and Advisory Groups.
Ensuring that professional teams within the Network are expert enough for providing advice services as well as its specific outcomes show the use of best practices in transplantation.
Providing professionals from MS involved within the Network with access to clinical excellence and support at national, regional and local levels of healthcare;
Developing and making available for professionals the best-harmonized alternatives to implement immunosuppression and monitoring strategies, mechanisms for the prevention of infectious disease, ensuring an adequate transition to adulthood.
Promoting the development of collaborative and harmonized research projects based on the Network priorities.
Increasing the capacity
ERN TransplantChild have a Strategic Communication Plan. During the first year of the Network, communication and dissemination actions have been mainly focused on strengthening the visibility of the project, raising awareness and involvement of HCPs and their professional teams as well as patients’ organizations. Some examples are:
- Newsletters: Regular publications including the events, forecast, etc.
- Profiles in Twitter
- Design of promotional material: flyer, videos, etc.
- Participation in worshops and congress related to Paediatric TransplantChild
Other dissemination and communication activities addressed to external stakeholders have been developed to spread the transversal approach of the Network as well as to increase their interest in the Network goals.
TransplantChild developed their own website (www.transplantchild.com ) as a key tool of the network's external and internal communication. This Website includes information for r professionals, patients and families, actions, etc.
The dissemination activities have achieved:
- The improvement of the visibility of the project, spreading information and knowledge about the Network.
- The increase in the awareness of the relevant stakeholders.
- The provision of the basis and references for carrying out future projects or studies.
- The facilitation of future cooperation with other Networks, knowledge hubs, associations, consortiums and scientific societies.