About the Expert Policy Forum on TTP

The Expert Policy Forum on TTP aims to identify gaps and unmet medical needs in the care and management of TTP, as well as provide recommendations to address them.

The Expert Policy Forum on TTP was set up in September 2022, bringing together five specialist haematologists and seven patient organisation representatives from Austria, Belgium, Bulgaria, Canada, Germany, Romania, Spain, Sweden, Israel, and the United Kingdom.

The experts involved in the forum at the point of publication are:

Bruce Morton

Answering TTP, Canada

Bruce Morton is an engineer and an MBA professional who lives in Ottawa, Canada. He works in the public key cryptography industry with a team which issues digital certificates to provide user trust for browsers and operating systems. Bruce is a family man and dedicates his time to his spouse and sons. He has been an iTTP patient since January 2012. In 2022, he has had a total of six TTP occurrences, but has survived them all and still has a positive look on life.

Gergana Sandeva

Answering TTP, Canada

Gergana Sandeva was diagnosed with TTP in January 2007, months prior to her first day of medical school at the University of Toronto. This diagnosis changed her life, as she was unable to start medical school and chose a career in clinical research. She completed her post graduate certifications as Clinical Research Associate and Clinical Research Professional and works as a Research Coordinator at Pharma Medica Research Inc.

Ioana Todorova-Yanakieva

Bulgarian Association Moschcowitz Syndrome, Bulgaria

Ioana Todorova-Yanakieva is a passionate TTP patients’ supporter. After diagnosis, Ms Todorova-Yanakieva experienced three TTP episodes and faced significant TTP treatmentrelated challenges, which motivated the establishment of the Bulgarian non-profit patient association. She works as a Clinical Research Professional, with two master’s degrees and extensive experience in healthcare. Her mission is to help patients get proper, affordable, and quality care (prevention, diagnosis, and treatment).

Rainer Stähler

DHG e.V., Germany

Rainer Stähler is Process and Transformation Manager for a German software company, living in Ingelheim, Germany. He was diagnosed with severe haemophilia A and had to use a wheelchair after being prescribed the wrong treatment. After treatment with Factor VIII at seven years of age, Mr Stähler learned to walk again and became a competitive swimmer. He is chairman of the board of the German Haemophilia Organization (DHG e.V.) and is married with three kids and a grandkid.

Dr Oleg Pikovsky

Soroka University Medical Center, Israel

Dr Pikovsky is a trained Clinical Haematologist and a Director of the Blood Bank and Pheresis unit at Soroka University Medical Centre in Israel. He is responsible for the hospital’s transfusion and plasmapheresis services and specialises in various malignant and non-malignant haematological disorders, participating in different research projects. His primary interests are in transfusion medicine and coagulation.

Professor Marie Scully MBE

University College London Hospitals, United Kingdom

Prof. Marie Scully is a Consultant Haematologist at University College London Hospitals (UCLH), currently acting as the clinical lead for haemostasis and thrombosis, having spent ten years as the clinical lead for blood transfusion. She runs specialist ITP and TTP clinics and works on obstetric haematology, as part of a team that specialises in treating varied and complex thrombosis, acquired and inherited bleeding disorders.

Dr Paul Knöbl

Medical University of Vienna, Austria

Dr Paul Knöbl works as a Senior Physician in the Department of Medicine 1, Division of Haematology and Blood Coagulation, at the Medical University of Vienna as a specialist in internal medicine, haemato-oncology and critical care medicine. His scientific work focuses on blood coagulation, platelet disorders, diabetes, haematology, and sepsis. He has published more than 200 scientific articles, book chapters and reviews, and has edited books on acquired haemophilia and blood coagulation in critical illness.

Rozalina Lapadatu

APAA, Romania

Rozalina Lapadatu is an activist, a professional advocating for social change and civic responsibility, and the founder of APAA (Association of Patients with Autoimmune Diseases). With more than 20 years’ experience in pharma, governmental and private health, CSR, and NGO work she has unique expertise and a different point of view. She is a Stanford University trainer for “Self-management in arthritis and fibromyalgia course” in Romania and European Patient’s Academy on Therapeutic Innovations Alumni.

Kevin Claes

TTP Community, Belgium

Kevin Claes was diagnosed with TTP in 2011 and has had four TTP occurrences, from which he has recovered. Mr Claes works at Hansen Industrial Transmissions, is a trade union representative, and a member of the TTP Community in Belgium. The patient association is working to provide TTP patients and their families with the support and accurate information they need, strengthen the contact between patients, their relatives, medical professionals, research teams, promote awareness of the disease, and support research.

Dr Javier de la Rubia

University Hospital La Fe, Spain

Dr Javier de la Rubia is the Head of the Department of Haematology at the University Hospital La Fe and Professor of Haematology at the Universidad Católica in Valencia, Spain. He is a member of the advisory board of the Spanish Group of Apheresis, and member of the European Haematology Association, and the International Working Group on TTP. His main research interests involve management of patients with multiple myeloma, and the study and management of patients with thrombotic microangiopathy. He has published over 200 peer-reviewed journal articles, several book chapters and more than 600 abstracts at national and international meetings.

Jo McIntyre

TTPNetwork, UK

Jo McIntyre is a TTP patient who established the not-for-profit patient organisation TTPNetwork, in 1998. Since her diagnosis, Ms McIntyre has experienced four episodes of TTP, and one threatened relapse. In addition to being the Founding Trustee of TTPNetwork, Ms McIntyre works in the domestic abuse sector.

Dr Fredrik Celsing

Karolinska Hospital, Sweden

Dr Fredrik Celsing is a Specialist in Haematology and Internal Medicine, having worked over 32 years in the Haematology Department at Karolinska Hospital in Stockholm, Sweden. His career and work have special focus in acute leukaemia, TTP, and CNS lymphomas.

Testimonials

Listen to patients and healthcare professionals from Austria, Belgium, Bulgaria, Canada, Germany, Romania, Spain, Sweden, Israel, and the United Kingdom as they tell their stories about TTP:

Patient registries are a very important tool for us physicians and national registries should be implemented in all countries. However, to be relevant, registries need to be updated regularly, which can translate into an additional burden for healthcare professionals.

Dr Javier de la Rubia

University Hospital La Fe, Spain

In England, the existence of guidelines developed by the NHS and the set-up of 10 specialised Referral centres have substantially improved TTP patients’ journey. This provides the centres with the best tools and mechanisms to take care of patients in a holistic way.

Professor Marie Scully MBE

University College London Hospitals, United Kingdom

We need more patient and healthcare professionals’ knowledge of what TTP is and related symptoms. Patients should be empowered and have more awareness of their disease and possible symptoms, in order to monitor them and to react as quickly as possible when an episode starts. Early diagnosis is key to provide patients with the adequate treatment as soon as possible. For that, ADAMTS13 levels and other related markers should not only be tested when patients are presenting symptoms, but on a regular basis to ensure patients monitoring and early diagnosis in case there’s a new episode.

Gergana Sandeva

AnsweringTTP, Canada

I have had a recurrence of TTP five times. Before the first three recurrences we only tested for platelets. This test only indicated that I did or didn’t have TTP, but gave no warning. Before the fourth time, we also tested for ADAMTS13, but it took many weeks to get the result back. Before my sixth time, not only did we test, but we also had a plan in the event that ADAMTS13 activity levels dropped. We almost avoided TTP, but I think we reacted too late. All patients are different, so we keep on learning.

Bruce Morton

AnsweringTTP, Canada

I personally had my first TTP episode triggered by a pregnancy. It was manifested by a heavy bleeding, which is kind of common during pregnancy and often considered a Disseminated Intravascular Coagulation (DIC syndrome). I had to undergo an abortion because of that. The proper diagnosis has been neglected as no one ever suspected the bleeding was of a hematologic origin and was considered pregnancy related with the focus being more on the pregnancy rather than the cause of the low thrombocyte count. Three years after the first episode, I was following hormone replacement therapy (HRT) to prevent the growth of an ovarian cyst. This HRT has provoked a very severe life-threatening relapse. In other words, both episodes have been linked to gynaecological triggers. Gynaecologists need to understand the disease and be aware HRT might cause serious hematologic conditions and that pregnancy itself might provoke a TTP episode in a certain subset of women. Despite the fact TTP is a rare disease, awareness is crucial as it might impact not only womens’ lives but also the proper development of the fetus and the birth of a healthy child.

Ioana Todorova-Yanakieva

Bulgarian Association Moschcowitz Syndrome, Bulgaria

For TTP, as for all rare blood disorders, the lack of knowledge about the disease is what leads to long pathways of diagnosis. Developing guidelines would help to have a framework, where the main diagnosis tests and main symptoms are outlined. In this way, even a general practitioner would know how to better help his patient.

Gergana Sandeva

AnsweringTTP, Canada

Developing education programmes to raise awareness of TTP among healthcare professionals is an absolute priority, focusing on general practitioners, neurologists and emergency units, as these are the first practitioners patients will see. Education of healthcare professionals is key to ensure optimal care and early diagnosis of TTP.

Dr Paul Knöbl

Medical University of Vienna, Austria

Multidisciplinary teams should become the norm when treating TTP patients. The team in my centre includes haematologists, laboratory specialists, cardiologists, and members of the intensive care unit. The team also collaborates closely with gynaecologists when the patient is a woman. Communication and close coordination between the team are key, which is why we have also put in place an internal procedure to be followed as soon as TTP is diagnosed, to ensure the patient is taken care holistically.

Dr Javier de la Rubia

University Hospital La Fe, Spain

In my centre, our primary care physicians have been trained in TTP early recognition and awareness, which has helped in early diagnosis of the disease, which is crucial for patients.

Dr Oleg Pikovsky

Soroka University Medical Centre, Israel

The role of patient organisations is essential when a patient receives a TTP diagnosis. Information about peer led support groups should be provided to patients at the earliest opportunity. This will enable the patient and their loved ones to make contact and receive support from the patient group and their community at the time they need it most. In other words, it should be part of the basic standard of care to refer patients to advocacy and support groups.

Jo McIntyre

TTPNetwork, United Kingdom

Information is key for patients. In Germany we have regional representatives who have the right knowledge and information about blood disorders. This is crucial for patients as they can turn to their regional representatives to ask questions about their disease or to simply keep in contact, which helped quite a lot during the COVID-19 pandemic.

Rainer Stähler

DHG e.V., Germany

Being diagnosed with TTP is lifechanging, as it impacts patient’s lifestyle, quality of life and even your professional life. Most TTP patients cannot simply go back to their previous job and need to adapt to their new reality. This also has an impact of patients’ mental health as they feel they don’t contribute to society the same way.

Gergana Sandeva

AnsweringTTP, Canada

TTP has a different face for each patient, but the common element is the impact on patients’ everyday life. This can be tiredness, confusion, concentration loss, headaches or poor mental health. In addition, patients can also live with secondary effects from the medication they take for these associated symptoms. In my case, I’ve a knee protheses following cortisone intake.

Kevin Claes

TTP Community, Belgium