aHUS Diagnosis & Treatment – KDIGO Controversy Conference


The KDIGO Controversies Conference on Complement-Mediated Kidney Disease was held November 19-21 2015 in Barcelona Spain.   KDIGO’s mission is to improve global outcomes for kidney disease, to include this Barcelona conference which centered on the complement-mediated kidney diseases C3 glomerulopathy (C3G) and atypical hemolytic uremic syndrome (aHUS).  “The objective of this KDIGO conference was to gather a global panel of multi-disciplinary clinical and scientific expertise to identify key issues relevant to the optimal management of complement-mediated kidney diseases. The goal was to define the renal pathology of C3G and aHUS; describe the clinical phenotype and evaluation of C3G and aHUS; characterize the genetic and acquired drivers of these two diseases; and determine best practice treatment and clinical trial strategies for C3G and aHUS.”  On 17 December 2016, KDIGO released the Barcelone conference report “Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference.”
KDIGO co-chairs Dr. Richard Smith (University of Iowa, USA) and Dr. Tim Goodship (Newcastle University, UK) opened the conference with a robust agenda for the global panel of aHUS and C3G researchers and clinicians, as well as representatives of the aHUS Alliance and two DDD patient organizations.  Pharmaceutical companies Achillion, Alexion, Akari Therapeutics, and Omeros were present at this Barcelona conference as “industry attendees” rather than participants in discussions, and as such did not attend conference components such as breakout sessions. (The list of conference participants from countries to include Italy, France, the UK, Germany, Sweden, the Netherlands, Turkey, India, China, and the UK, is available by clicking here.)
C3 Glomerulopathy began to appear in the literature in 2013 as a term to indicate complement-mediated disease that includes Dense Deposit Disease and a similar sub-type C3GN, in addition to atypical HUS.   Information and discussion was grouped at the KDIGO conference by topic areas, each guided by key questions.  Aptly named a “controversies” conference, clinicians and researchers from around the world met to vigorously discuss gaps in knowledge as well as weigh diverse thoughts and opinions in areas such diagnosis, treatment strategies, genetic testing, and disease drivers.  Included were these aHUS-related content groupings from the KDIGO Controversies Conference on Complement-Mediated Kidney Disease:
Renal pathology:  Terry Cook, presenter.  Pathology of aHUS
Clinical Phenotype and Assessment:  David Kavanagh, presenter.  What are the challenges in short-term and long-term patient management?
Genetic Drivers of Disease aHUS:  Marina Noris, presenter.   What can we learn from genetics? Is genetic testing clinically relevant?
Acquired Drivers of Disease:  Véronique Frémeaux-Bacchi, presenter.  aHUS and autoantibodies: Their role in disease and their impact on patient management.
Treatment Strategies:  Fadi Fakhouri, presenter.   aHUS: How should patients be followed long term? Are there options to lifelong Eculizumab?
As the KDIGO website noted prior to the Barcelona meeting in November 2015: “The conference also aims to summarize outstanding knowledge gaps and propose a research agenda to resolve standing controversial issues. It is hoped that the deliberations from this conference will inform clinicians of the evidence base for present treatment options and help pave the way for future studies in this area.” The just released KDIGO conference report “Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference.” does exactly that, providing a guidebook for all who treat aHUS patients and others with complement mediated kidney disease.
The recommendations and conclusions within this KDIGO global document are likely to greatly impact the entire continuum of aHUS information and practices, to include such diverse areas as future physician education, genetic testing, diagnosis of aHUS, and standards of care for treatment of patients with atypical HUS. At the same time  it is clear that much remains to be known, and this KDIGO document clearly indicates potential research directions as well as  differing opinions that still remain until advancement and investigation yield more evidence.  We extend our gratitude to KDIGO, and all Barcelona conference participants, as the aHUS Alliance recognizes with appreciation this international and cross-discipline collaboration of clinical and scientific experts.
* featured photo is of Dr Richard Smith, Linda Burke and Prof. Tim Goodship
Note:  The aHUS Alliance knows that aHUS patients and their families, as so often is the case with rare disease patients, seek to keep current on  information regarding their diagnosis, treatment, and medical care.  Below are links that highlight  information of high interest to the aHUS community, continuing the aHUS Alliance goal to connect efforts, groups, research, best practice information, and resources for all with an interest in atypical HUS.
Supplemental Material from KDIGO – Highlights for aHUS Patients & Families
From the main document, “Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference.”
Targeted Supplemental Material of high interest to aHUS patients, caregivers, and families:
*Other Organs and Body Systems are affected by aHUS –  Heart, Lungs, Eyes, Skin, GI, Neurology & Other Issues.  Supplementary Table 1. Extrarenal manifestations reported in aHUS, DDD and C3GN
*Lab Tests & Medical Studies that may be ordered by specialists, or within Patient Medical Records.  Supplementary Table 2. Investigations recommended for TMA
*Dosing, Intervals, and Discontinuation of Eculizumab.  Information includes specific dosage amounts and time intervals when starting (Induction) and continuing eculizumab (Maintenance) for varied patient ages and situations.  On stopping use, “Desire to discontinue complement blockade No consensus exists regarding tapering of dose.”  Supplementary Table 9. Eculizumab dosing in aHUS based on dosing goal.   
KDIGO Controversies Conference
Conference Details:  COMPLEMENT-MEDIATED KIDNEY DISEASES    Barcelona, Spain   November 19-21, 2015  (Click link for Scope of Work and Discussion Questions)
KDIGO Controversies Conference on Complement-Mediated Kidney Disease:  Conference Report & Conclusions:   “Atypical hemolytic uremic syndrome and C3 glomerulopathy: conclusions from a “Kidney Disease: Improving Global Outcomes” (KDIGO) Controversies Conference.”  Goodship, T et al.  17 Dec 2016   Courtest of ISN.  DOI: http://dx.doi.org/10.1016/j.kint.2016.10.005  Also at: http://www.kidney-international.org/article/S0085-2538(16)30604-4/fulltext
aHUS Research
aHUS Alliance list of Publications, Info Centre:   http://ow.ly/dG6s307dS6O
NCBI Pub.Med List for aHUS:  https://www.ncbi.nlm.nih.gov/pubmed/?term=aHUS
Resources from the aHUS Alliance
2016 aHUS Fact Sheets
aHUS Facts- a Brief Look     One Double-Sided Page, to Print & Share at:  http://ow.ly/UetJ304bKcS
aHUS Key Facts & Information     Detailed, Full Version with Links and Cited Research at:  http://ow.ly/dttf304bJGZ
2016 aHUS Global Poll
Poll RESULTS: Information and Links, along with Poll Graphs  http://ow.ly/1DA7303FoJx
2016 Poll – Questions:   http://ow.ly/ASFH307dSZg
2016 White Papers
Access to aHUS Treatment    http://ow.ly/RYHQ306DY3f
aHUS and Dialysis Insights  http://ow.ly/rExu306DV8C
KDIGO – aHUS Alliance at the Barcelona Conference – 2 Blogs
Global aHUS Experts Meet in Barcelona at KDIGO Conference on Kidney Disease
KDIGO Conference in Barcelona – What’s the News?   http://ow.ly/6jN4307dSyl

Leave a Reply