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LUPUS WORLD ENGAGED! Session 1 - Engagement

Friday September 4th, 2015 – Hofburg Congress Centre – LUPUS WORLD ENGAGED!

Anne Charlet, Secretary of LUPUS EUROPE opened the session at 09:00 welcoming all attendants for this First Patient-focused meeting and opening the first session, focused on “Engagement”.

Experience from EULAR task force on Recommendations, by Francesca Machiori:

Trained by Eular as a Patient Partner in Research (PPR), Francesca participated in the project “Recommendations for the management of family planning, assisted reproduction, gestation, delivery and menopause in patients with Systemic Lupus Erythematosus and Antiphospholipid Syndrome”. This work was done between July 2014 and June 2015. The team, composed exclusively of volunteers among rheumatologists, immunologists, obstetricians, nurses and patients, from all over Europe. The result is that we now will have (soon to be published) evidence-based recommendations developed by expert consensus on family planning, assisted reproduction, pregnancy and menopause for SLE and APS patients.
The key recommended principles are (a) that doctors should embrace, not warn against pregnancy, after consideration of individual risks, and (b) that decisions regarding family planning are a patient decision under counselling of health professionals. Discussions about family planning should take place so that patients are properly informed, taking into account that fertility decreases with age.
The health care professional’s advice should cover contraceptive measures, assisted procreation, and methods to preserve fertility. With regards to fertility, a special focus in the discussions went about assessment of disease activity and therapies to prevent/manage SLE flares during pregnancy. Another discussion considered menopause, including hormone replacement therapy, guidelines for screening on cancer based on SLE specific risk factors, and the recommendation to take HPV immunization.
Francesca’s perspective from participating in this project is that we are not too old to learn. At EULAR we are part of a network (PARE network of national and European patient organizations for RMD) and trained to handle these projects: As patients, we have a valuable opinion and experience. We might not fully understand everything immediately, but people, including doctors, are there to answer questions, and it is absolutely fine to ask! But most importantly, as we represent patients, our opinion is important and listened to.
Because we represent patients, part of our role is also to disseminate what is said, ensuring “translation” for the general public, so that all people with lupus can understand what concerns them. This includes having a translation in local language. One of the roles of the PRP should be to translate into his/her own language.
From a methodology point of view, everyone from the very broad and diverse team was invited to share all his questions in writing. There were so many questions… and in the end, everyone was also asked if they agreed with the outcome, so the team really remained united.

Experience from International Study Groups initiatives on T2T and DORIS, by Kirsten Lerstrøm
Kirsten started by sharing her experience as part of the Treat to Target (T2T) study group. The first meeting considered the complexity of measurement of disease activity, and discussed whether we should or not go with a project of treat to target. The team concluded that it should be pursued because research shows better outcomes for patients when there is a specified treatment target: research has already resulted in successfully applying T2T principles for Diabetes, Hypertension and RA. The T2T project started with 18 people in May 2012, but over time the team grew truly multidisciplinary to 36 people including patients. The meetings used the Delphi method: you start from formulating research questions to be validated by literature and research, then people vote on what they think is most important, and the team moves forward with those topics.
While T2T goals can be multiple (control of disease activity, reduction of damage or quality of life), all T2T plans should be carried out according to basic principles: (a) Shared decision (patient and physician), (b) focus on Long term survival, prevention of organ damage, optimizing quality of Life, (c) the approach should be Multidisciplinary, taking the whole person into account, and (d) Regular long term monitoring and review of therapy, as opposed to only managing flares.
But at some point, the team arrived at the complicated question of defining Remission. For this purpose, another team was assembled in 2014, DORIS (Definition Of Remission In SLE), to develop potential definitions of remission for use in practice and clinical trials. This definition could also impact health policy decisions, so it is important to carefully define when it is that your disease activity is so low that you can be considered in remission, because the treatment applied effectively controls activity. Two main strands of a definition have been made: “off treatment”, or “R-off” (perhaps allowing chronic, basic treatment) or “on treatment” or “R-on” (allowing treatment other than hydroxychloroquine and steroids and at what minimum).
An important question is whether you can have remission “on treatment”. Usually remission implies that you are off treatment, but for lupus patients, it was judged relevant to also define a remission on treatment as less than 3% of Lupus patients will go off treatment for more than 3 years.
Various definitions are tested using existing data sets from all over the world, with the first results shared in August, but more work is required. The next step is to test the 16 definition elements following the two strands R-on/R-off.
In this project, the patient representative focus is to make sure that the definition is actually understandable and operable, not just technically, but also to make sure patients understand it and that it can be used in T2T.
Kirsten’s opinion is that remission requires “off treatment”, even if indeed remission does not mean you are cured. This is because many medications still have side effects (so a remission “on treatment” would carry those), difficulty in distinguishing this particular condition with reactions to various treatments of other symptoms) and because being in remission would potentially have administrative impacts (reimbursement, …). Voices in the room suggested that if the treatment is acceptable with limited side effects, then remission is a lower disease activity that results in a proper social/quality of life. But quality of life is not chosen as a measure in this context.

Experience from the Innovative Medicines Initiative (IMI), by Blanca Rubio
Blanca participated in the Ethics Committee of IMI, in the scope of the PRECISEADS project. PRECISESADS is a European collaborative project within the Innovative Medicines Initiative (IMI). It involves 23 research centres and 5 companies from 12 European countries, and benefits from huge EU funding, with a total budget of 22M Euros over 6 years. The objective is to understand the origin of autoimmune diseases, and why people with a same diagnosis (like “lupus”), respond to some medications and others not. This understanding would drive a better map of treatment for patients. The option selected is to classify diseases according to biomarkers or other indicators. The scope is not just lupus, but also other autoimmune diseases, where today there is little sharing across disease specialty centres.
There is growing evidence that many of these conditions may be incorrectly classified. Data collected on the molecular causes of their disease and clinical symptoms of 2 500 people with autoimmune diseases, will be used to build a new classification of Systemic Autoimmune diseases based on meaningful clinical biomarkers, that will allow doctors to offer patients more personalised treatments at an earlier stage.
Similarly, while new biological treatments are being developed for SLE, it is not always clear which lupus patient will respond to which treatment, leading to problems in determining the usefulness of drugs in clinical trials, possibly because the results are good for some patients, but have no effect on others, blurring the statistics. For many years, no drug development has come to maturity. We need new medication, and this new classification could really help.
The ethics committee in which Blanca participates originates from an EU requirement. It contains as a minimum a lawyer, a social worker and a patient representative. They operate independently of the project, and control / analyze the personal aspects of the project, such as the data collected, the implication of children, patients, their consent to the research, data protection, and all ethical aspects of the project. A first meeting took place in Feb 2015, and the next meeting in November will review data collected so far.

Training – Key to Patient Partner in Research (PPR), by Francesca Marchiori
Francesca started with a quote, “Nothing about us without us. Involving patients in research projects improves both the methodology and outcomes of the research, and offers invaluable additional insights. It is essential to ensure that high quality research brings real benefits for patients and their daily lives.”
Research is done for patients. Without them there is no reason to do research. The objective of EULAR is also to move them from the passive role of being co-opted in a research as guinea pigs, to a more active role in all phases, and for all research types.
To be effective, the Patient Partner in Research (PPR), needs to be equipped as equal to the researchers. This implies that patients gain in personal skills such as communication (including language skills), motivation, being constructive, etc… They need support from experts who already know the methods, but also from all patients, as the PRP must speak for all the patients, not just for his/her self. His/her experience is important but should be integrated with the interaction and the listening to other patients and patients’ organizations.
Eupati training seeks to have 100 trained people for all diseases. It is interesting to have general training to enhance patient participation (project organization, Delphi method, …), but also specific health related training (on clinical trials, health technology assessment, …regulation & reimbursements, organization of health care). A number of organizations make that training available (including PARE, EUPATI, EURORDIS).
But for lupus, additional training is needed because your lupus is different from another’s: understanding its complexity, complication, and also its impact on social lives and various treatment options is very important. In selecting the appropriate training, one must be careful to confirm the reliability of the information (not everything on internet is true), as well as the depth of expertise required: Patients don’t need to become research specialists, but need to understand the medical research language.
Francesca then shared her thought that similar training is also important not only for patients, but also care givers, nurses, doctors (GP) so that they can include lupus thinking even when just dealing with a “normal cold”
Finally, Francesca insists that PRP need to install two-way communication: On the one side, sharing experience & outcomes of research, on the other side, getting information to better play the PRP role. This requires new efforts to be made, which could include surveys, social network usage, meetings with patients, or groups facilitating interactions among PRP. Many people are involved in projects that could share their thoughts more frequently.