The ERA-EDTA WGIKD (Working Group on Inherited Kidney Disorders) has just published recommendations for the use of Tolvaptan in ADPKD.

Recommendations for the use of tolvaptan in autosomal dominant polycystic kidney disease: a position statement on behalf of the ERA-EDTA Working Groups on Inherited Kidney Disorders and European Renal Best Practice


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European Journal of Human Genetics

The article is signed by RD Connect researchers on the basis of the work done so far by various committees where FEDERG is active. The RD Connect paper on informed consent, which FEDERG contributed to via the Rome workshop and paper drafts has now been published. It’s open access and so available for all to read:

Improving the informed consent process in international collaborative rare disease research: effective consent for effective research.’ is now published online in EJHG http://www.nature.com/doifinder/10.1038/ejhg.2016.2 in our Advance Online Publication (AOP) service.

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HEIDELBERG 12th January 2016 – First Meeting of the European Reference Network for Rare Renal Disease (EuRenNet)
Twenty participants in this first informal meeting of members of the future European reference network for rare kidney disease, from nine countries. The meeting was chaired by Pr. Franz Schaeffer Heidelberg, and was sponsored by the Working Group on Inherited Kidney Diseases of the ERA-EDTA, coordinated by Pr. Olivier Devuyst, Chairman of the FEDERG Scientific Council.

Patients are heavily involved in the preparatory work for future network. FEDERG and three representatives of national associations, federation members, were present at the meeting.

The objective of this meeting was to take stock of the application process, according to the latest instructions issued from the Commission, in particular the “Toolbox” put online on 23rd December 2015.

The meeting has highlighted the challenges that the teams will face for the national accreditation of their reference centers. Much work will have to be done by each center candidate to provide documentation for obtaining highest score on the evaluation criteria of reference centers (Health care providers), which are severe according to the professionals.

The project is then a bet on the future, since there are no European funds to support the network at this stage of the process and because those who have already done a great job for organizing their national reference networks are solicited more, with benefits in other countries.

Although this noble task of solidarity is perceived by everyone as heavy, the participants have formally pronounced for rapid work to enable the network to be ready when the call will be launched in a few weeks.

FEDERG presented the patient organizations perspectives: patient’s organisations are stakeholders for the rare renal ERN, and motivated for its development. They see it as an opportunity to expand access to good health care to all patients in Europe. This is a strong solidarity objective. The principle is that the expertise goes as close to the patient no matter the level of organization of the country in which the patient lives. FEDERG also sees the ERN as a process that will enhance organization of patients across Europe, and yield to greater mobilization for registries and trials with more patients.

In summary: “Better care for all and then better research”

View of the Participants at the meeting

IMG_6206 (1)

FEDERG representatives, de gauche à droite : Daniel Renault (Chair), Claudia Sproedt (Vice-Chair),
Marjolein Storm (NVN), Marjolein Bos (Cystinosis Netherlands & NVN).

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KDIGO Controversies Conference on Complement-Mediated Kidney Diseases
Barcelona, November 19-21, 2015

KDIGO guides are the best known among professionals (doctors and researchers) worldwide. The
organization KDIGO born in USA, invites doctors and researchers, and the most prestigious experts in
each renal disease.

At this conference, we ASHUA have been fortunate to participate with 2 people, and representing
FEDERG and all of European aHUS patients. Due our agenda, Mireya (Secretary of ASHUA) was all
time participating all time on the Treatment Strategies Group, while I was on the second day.

For two full days, doctors and researchers, discussed the questions, detailed below, made by the
other professionals involved in KDIGO worldwide. The findings of the whole debate, we shall know
once drafted the new guidelines for the management of aHUS.

As an organization and representatives of aHUS patients, we have known as health professionals,
their interest in improving the lives of patients, their dedication … but above all, their willingness to
work with us the patients.

We were able to chat and exchange points of views with many Doctors, worldwide, although in the
case of aHUS, perhaps research and good management of aHUS, are concentrated in Europe and

We highlight several conversations … with Dr. Goodship, which have made possible apart from our
attendance at this Conference, he paid much attention to our comments and concerns,
encouraging us to continue to participate in future KDIGO conferences, representing all kidney
diseases, but also sharing all KDIGO jobs with all patients, in all countries.

Due to technical and medical concepts spoken, I do not feel appropriate to summarize here two full
days of debate among professionals, we shall have it soon, when editing the new guide. Although
all comments between doctors and researchers may I highlight the following:

  • Dr. Ardisino (Italy). A case of cold in a patient with Eculizumab, DO NOT stop the medicine,
    perhaps even increase the dosage.
  • Dr. Ruggenenti (Italy). When a liver transplant is performed at an aHUS patient, Eculizumab is
    strongly recommended during and after transplant until the new liver produces all new
    proteins (especially factor H) in sufficient quantity to avoid new onset aHUS in the following
    days transplantation.
  • Dr. Loirat (France), Dr. Ariceta (Spain) and Dr. Appel (USA). When a debut of aHUS,
    immediately suspected it is aHUS, administer Eculizumab, not loose time, especially in
  • Dr. Noris (Italy). Genetic analyses help to understand the origin of the aHUS. May it is time to
    start to customized treatments depending of each aHUS patient, but due we don’t know
    much about that, we should take care and learn step by step. We must be careful with the
  • Dr. Blasco (Spain). Patients … associations are the key to request and pressure administrations
    to create reference hospitals and further enhance the investigation, which should be
    centralized and organized.
  • Dr. Van de Kar (Netherlands). She presented the new video made between aHUS patients
    and doctors (coordinated by the association NVN), to raise awareness throughout the
    population, but also to the administration and doctors. We have shown our Personal ASHUA
    bracelet, and she liked a lot and paid special interest in it as she sees many advantages for
    all patients.
  • Dr. Fadi Fakhouri (France). Eculizumab discontinue??? There are cases is completely
    discouraged as the factor H, but for other cases is highly recommended to continue
    treatment for at least one year, and from there continued again between 6 or 12 months
    once the renal function has stabilized. Once discontinued, the patient should be monitored
    very strictly.
  • Dr. Carla Nester (USA). PNH patients take many more years with Eculizumab, so in some cases,
    data can be compared with aHUS cases and learn.
  • Dr. Véronique Frémeaux-Bacchi (France). The visibility of patients is essential, she liked very
    much our ASHUA red bracelet and the campaign we are doing with celebrities.


Group 5. Treatment Strategies, breakout group questions.

  1. What is the optimal duration for eculizumab treatment in aHUS patients? What is the evidence
    that justifies life-long therapy especially in adults?
  2. What is the evidence for optimal dose and dose interval for eculizumab and are there
  3. What is the optimal treatment for patients with aHUS due to anti-FH antibodies?
  4. How do we stratify C3G patients for novel agents and clinical trials?
  5. What should the current approach to treatment of C3G be?
  6. Kidney transplantation in aHUS and C3G
    • What is the best time frame for kidney transplantation after the onset of end-stage
      renal disease (ESRD) in aHUS or C3G patients treated with anti- cellular therapy and/or
    • What is the risk of recurrence?
    • What is the best strategy in order to prevent aHUS (C3G) recurrence after kidney
      transplantation: preventive vs. pre-emptive strategies?
    • Should living kidney donors be used (preferentially?); living related?
  7. How should eculizumab treatment be monitored: CH50, AP50, free eculizumab or total
    eculizumab, in vitro EC tests?
  8. What is the optimal treatment for rapidly progressive or bad prognosis histology C3G?
  9. What is the role for immunosuppression in the treatment of C3G
  10. What is the role of eculizumab in the treatment of C3G? a. In what subgroups might it be


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WELCOME to NephcEurope !

FEDERG is pleased to welcome NephcEurope as new member of our federation. Having NephcEurope with us is a great help in particular for representing the patients in the coming elaboration of the European Reference Network on Rare Renal Diseases. This adds the Nephrotic Syndrome, the most frequent rare renal disease, into the overall coverage of pathologies of FEDERG.

FEDERG and NephcEurope know each other for quite a while. Together we are the voices of the patients in various committees within EURenOmics and RD-Connect projects. Our joint expertise and efforts have contributed into depth to new developments in these projects.

NephcEurope is an umbrella organization dedicated to all aspects of care and cure for all forms of primary Nephrotic Syndrome (both genetic and also non-genetic) covering MCNS, FSGS, MPGN, membranous nephropathy and IGA Nephropathy. The organization is the international liaison between European national organizations.

NephcEurope has special attention to representation specifically in the European field on the higher EU level whereas the national patient organizations are covering national entities. We are well represented in all large kidney projects as EurenOmics (SAB and PAC), RD Connect (PEC), EurostAM a science project of Eurotransplant and Safepedrug on the development of pediatric medication. For more information see the website: www.nephceurope.eu


photo: Marieke van Meel, Chair of NephcEurope, (on the left) taken at the EurenOmics
launching meeting in 2012 Sitges, together with Pr. Loirat both members of the ethical committee. 

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ERN, the move is starting!

The European Reference Networks, ERN, the move is starting!

“ERN” an acronym you need to get familiar with!

EURORDIS organized in Paris on the 27-29th October, the annual meeting of the Council of European Federations (CEF), the meeting was very well attended this year because of the focus on the establishment of the European Reference Network for Rare diseases. Daniel Renault Chair of FEDERG participated to the meeting.

Recall that Eurordis has played a key role in the EUCERD committee, (the European Committee of Rare Diseases) that have paved the way to ERN.

The situations for the ERN pre-set themes are much diverse. There are thematic for which the patients organizations are scattered (for 10 themes of ERN there is, for each theme, between 20 and 100 associations members of EURORDIS) with a similar divided situation for the clinicians.

Fortunately the theme for which we are concerned “rare renal diseases” (RRD) is in a better situation on both sides. Only 10 renal associations in EURORDIS (out of 610), half of them are already member of FEDERG. Thus FEDERG is somehow legitimate to represent the patients in the ERN project on RRD.

EURORDIS will make efforts in the coming weeks to get all the groups organized into Patient Advocacy Group, that will be done for ERN but also looking beyond. Again Rare Renal is already almost done.
Officially the fist call is to be launched before March ends with 3 months for making the proposal. EURORDIS urges us to speed up the preparation steps.

For Rare Renal, a core team of nephrologists and geneticians together with FEDERG will start the preparatory works mid November. A partner meeting is to be held 12th January 2016 in Heidelberg at the invitation of Franz Schaeffer.

Photo 1 Yann Le Cam Director of EURORDIS presenting the ERN process and how patients should be organised.

Photo 2 The CEF audiences with Matt Johnson, Scientific Director of EURORDIS, presenting the process of submission and validation of the ERN projects.

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FEDERG present at the 48th Scientific Meeting of the European Society of Paediatric Nephrologists (ESPN) from the 3 to 5th September 2015 in Brussels.

The organizers offered a boot during the 3 days of the Congress. Thanks to AIRG-Belgium the logistic for FEDERG went smooth. The last day of the Congress FEDERG hold a Members Meeting in the premises of the Congress.

This has been an opportunity to make our presence known of the European nephrologists and of some pharmaceutical companies that were in the Hall of exposition.

The members meeting addresses several issues related to FEDERG mandate and management. The last meeting was held in June 2013, and this was an opportunity for the members to express how they see FEDERG in future. An important issue discussed has been the on-going initiative on the creation of European Reference Networks for Rare Renal Diseases.

On the scientific side of the congress more than 400 contributions, the Nephrotic syndrome was the focus of several sessions, this is a frequent paediatric disease. For the renal genetic diseases, a significant number of contributions on aHUS. Also noteworthy a joint session ESPN and ERA-EDTA (Adult) on APKD and the new possibilities for treatment. See full program at http://www.espn-2015.org/program.php

Photo on the booth (left to right): Michel Schenkel, Daniel Renault, Marjolein Bos, Tess Harris and Marjolein Storm.

Members meeting (left to right): Daniel Renault Michel Schenkel, Bruno Woitrin, Tess Harris, Marjolein Bos and Marjolein Storm.

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FEDERG is on its way!

The launching meeting of the FEDERG’s initiative – Federation of European patient groups affected by Renal Genetic Diseases (RGD) – was held at the initiative of AIRG-France on May 26, 2012 as a dedicated meeting within the 49th ERA-EDTA Congress held in Paris, Palais des Congrès.

The launching meeting was supported by ERA-EDTA (European Renal Association and European Dialysis and Transplantation Association), by ESPN (European Society of Pediatric Nephrologists), by EURORDIS, and by AIRG-France.



The meeting brought together 10 nephrologists, some had made the trip especially for the meeting FEDERG, and 10 representatives of associations of renal genetic diseases.

The meeting has been an opportunity for all participants of the launching, to express and share unanimously the desire to see the emergence of a European federation. The willingness to join forces is present, and is ready to concretize itself and expand to others.

The first part of the meeting chaired by Professor Yves Pirson, Chairman of the Scientific AIRG Belgium, focused on the perspective of nephrologists about the importance of considering the genetic renal diseases in a European context .The speakers were Professor Dominique Chauveau, Chairman of the Scientific Committee, AIRG-France, Prof. Oliver Gross, Nephrologist Göttingen, also representing the German group of patients on Alport syndrome, Dr Elena Levchenko of Leuven, representing pediatric nephrologists and the society ESPN, and  Prof. Olivier Devuyst Zurich, Head of the Working Group of the ERA-EDTA on Inherited kidney disorders.

Each speaker was able to propose and develop strong arguments that advocate for an efficient organization at the European level of all patient groups and associations with a wide scope of diseases (renal and multi-organs) and not restricted to a single pathology.

The second part of the meeting, chaired by Daniel Renault, President AIRG France, was dedicated to identify the motivational elements of the associations represented and initialize the following preparations in the construction of the federation. Associations were represented either directly by the members present (some excused because of health problem) or by nephrologists attending the conference: – 8 countries of the EU-27 were represented: Germany – Belgium – Bulgaria – Spain-France – Ireland – Netherlands – United Kingdom; – 2 outside countries: Switzerland and Morocco as well as two international federations.

In the short time of two hours, the meeting allowed everyone to know each other and to outline areas of work and mobilization for the future. The collaboration between nephrologists and patient groups is recognized by everyone as the key to high efficiency. At the same time, it is recognized that it is hard to get patients mobilized in some contexts; therefore external support might be required. This should be one of the roles of FEDERG to support the emergence of local associations in national contexts unfavorable!

Beyond targeting a good collaboration between nephrologists and patient groups, the improvement of the “quality of life” for those who suffer from genetic kidney disease, has been emphasized by many as a priority. This should also be one of the focuses of a European federation.

Full reports of the meeting by clicking our AIRG-France website (see link below) and on the FEDERG web platform where you can directly contribute to and monitor the progresses of the federation.


One of the main recommendations of the meeting is to quickly enlarge the basis of FEDERG to more associations, covering more pathologies as well as more European countries.


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