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Recommendations

Prompt diagnosis and treatment of TTP are crucial to prevent significant morbidity and mortality.xxxv However, several barriers to optimal care, including delays in diagnosis, lack of awareness and knowledge about TTP among healthcare providers, and limited access to specialised care centres with expertise in managing TTP, can hinder the management of the disease. In this roadmap, the experts involved in the Expert Policy Forum on TTP explored these barriers in-depth and discuss potential solutions to improve the management of TTP and ultimately enhance patient outcomes.

Patient empowerment and engagement

Improve TTP patient wellbeing by putting in place multidisciplinary and holistic teams by national public health systems. This multidisciplinary team should include at least haematology, neurology, nephrology, psychology, and intensive care services. Paediatric and obstetrics and gynaecology services should be part of this team in case the patient is a child or a woman.

Goal: Multidisciplinary and holistic teams are in place for rare diseases, including TTP, are integrated in national health systems.
Empower patients with TTP by providing them with appropriate information and improving their understanding of the disease and its management. The patient community should be supported with key volunteers (patients, healthcare professionals) that would act as a disease spokesperson, having the right knowledge and information about TTP, to whom patients can turn for information, advice, and support.

Goal: TTP patients are empowered thanks to the creation of communication channels and activities that provide accurate information and improve their understanding of the disease and management.
Recognise the key role medical societies and patient associations play during the TTP patient journey, providing them the necessary tools and resources to work together to advance research, education, and advocacy efforts related to TTP.

Goal: Medical societies and patient associations leading role in the TTP patient journey is recognised by providing them the necessary tools and resources to work together to advance research, education, and advocacy efforts related to TTP.

Disease awareness and education

Create opportunities for HCPs to further develop their knowledge of rare diseases, including TTP, as well as create or expand network of specialists, i.e., the European Reference Networks, to support referrals, education of both specialists and non-specialist HCPs, and knowledge transfer amongst the healthcare community.

Goal: Healthcare professionals have further developed their knowledge of rare diseases, including TTP, as well as expanded network of specialists.
Raise awareness amongst HCPs about triggering conditions and medication for TTP, including the management of these in the dedicated guidelines and recommendations for management and care of TTP.

Goal: Specialists and non specialist healthcare professionals have a better understanding of triggering conditions and medication for TTP.
Leverage and actively support the international TTP Awareness Day in September to raise awareness of TTP among the general population.

Goal: The international TTP Awareness Day in September is established as a key date to raise awareness.

Patient-centric health systems

Develop, review, and regularly update clinical guidelines for management and care of iTTP (immune-mediated TTP) and cTTP (congetinal TTP) to ensure key developments are taken into account, as well as ensure experts are aligned on definitions and support the approach to TTP as a chronic disease.

Goal: Clinical guidelines for management and care of iTTP and cTTP exist and ensure experts are aligned on definitions and support the approach to TTP as a chronic disease.
Create, implement, and support access to referral and expert centres to ensure TTP patient optimal care, by engaging and supporting EU-wide initiatives, such as the European Reference Networks. Referral centres would not only ensure patient care, but would also support investigation and access to clinical trials, advancing research for TTP.

Goal: Referral and expert centres to ensure TTP patient optimal care have become the norm across Europe, Canada and Israel.
Ensure that regular monitoring of ADAMTS13 is included in their guidelines for disease management and care of TTP. Moreover, national health system planners and decision-makers must guarantee appropriate funding to support widespread access to diagnostic and monitoring equipment.

Goal: Regular monitoring of ADAMTS13 is included in the TTP patient journey.
Promote and leverage the current initiatives at international level, such as the European Reference Networks, to improve participation in data registries to support research and improve patient care.

Goal: Data registries participation improves through the promotion and leveraging of initiatives at international level.