RESEARCH THAT MATTERS TO aHUS PATIENTS IN KIDNEY WEEK 2018

Next week the American Society of Nephrology hosts its Kidney Week 2018 Conference, during 23-28 October in San Diego California. Attended by clinicians ,industry and patient organisations from all over the world , the conference is a key learning opportunity about all matters kidney.

Throughout the week the results of thousands of kidney research projects will be presented and talked about. Among them there are 40 research studies relating directly to aHUS. These are listed below and catergorised by the research topics which aHUS Patients have told the alliance matter to them and which have become the aHUS Patients Research Agenda.

The top three topics studies were Genetic Predisposition and Triggering factors (12) , Diagnosis (8) and Pregnancy (7)

If the visitor has not enough time to look at all the aHUS research results on this visit to the website it is recommended that the following three  studies are seen at least:

The safety of eculizumab for aHUS

The economics of eculizumab

The discontinuation of eculizumab

Otherwise if aHUS is your research interest then the alliance provides links to all aHUS studies appearing at ASN Kidney Week 2018, just click on the study title to see the full abstract. (Article about the aHUS Patients Research Agenda)

 

1.Is there a diagnosis sweet spot which can be found before a developing thrombotic microangiopathy turns into a catastrophic episode of aHUS?

Platelet Count and Serum Creatinine in the Differential Diagnosis of TMA: An Analysis from the CESAR Study

Hypertension or Complement-Associated Thrombotic Microangiopathy Diagnostic Implications and Response to Eculizumab

Clinical Outcomes of Renal Biopsy-Proven Thrombotic Microangiopathy

Diacylglycerol Kinase Epsilon (DGKE) Deficiency in Endothelial Cell Results in Marked Abnormalities in Phosphoinositides

(STEC-HUS) in Adults: An Underrecognized Cause of Thrombotic Microangiopathy

A Case Imitating Thrombotic Microangiopathy

Atypical Hemolytic Uremic Syndrome Overlapping with Antiphospholipid Antibody Syndrome: A Case Report with Four Years Follow Up

Postpartum Ruptured Sub-Capsular Liver Hematoma with Dialysis-Dependent AKI and Intracranial Bleed with Delayed Recovery: Is It HELLP or aHUS?

 

2. Is there an optimal way in which a complement inhibitor can be delivered to suit an individual’s need?

Suboptimal Dosing of Initial Eculizumab Therapy in aHUS?

 

3. Can a clinically effective therapy be developed that is affordable for all aHUS patients?

Dutch Initiative to Improve Therapy and Economize Healthcare in Atypical HUS

Ideal Clinical Outcome and Potential Cost Savings with Individualized Eculizumab Dosing using Bayesian Modeling in Atypical Hemolytic Uremic Syndrome

CH50 Result Variability by Methodology in Eculizumab-Treated Patients with Atypical Hemolytic Uremic Syndrome

Role of Eculizumab in Dialysis Dependent AKI Secondary to Atypical Hemolytic Uremic Syndrome

 

4. Can a complement inhibitor be stopped safely when not needed by some aHUS patients and what makes them different?

Thrombotic Microangiopathy Frequency in Patients with Atypical HUS: Discontinuing vs Remaining on Eculizumab Treatment

 

5. Are there long-term studies of outcomes of those in remission whether treated by a complement inhibitor or not?

Coexisting Variations in Complement Regulatory Genes Increase Risk of Relapse in Anti-Factor H (FH) Antibodies Associated Atypical Hemolytic Uremic Syndrome (aHUS)

 

6. Is there a significant difference in outcome between having a complement inhibitor before or after a kidney transplant?

Outcomes of Kidney Transplant Patients with Atypical Hemolytic Uremic Syndrome Treated with Eculizumab: A Meta-Analysis

Deceased Donor Kidney Transplantation in Atypical Hemolytic Uremic Syndrome Without Preemptive Eculizumab Use

 

7. Can the side effects of treatment using a complement inhibitor be distinguished from those temporary and permanent ongoing ailments which follow initial onset?

 

Eculizumab Safety: 5-Year Experience from the Global aHUS Registry

Candida albicans Peritonitis with Urgent Start Peritoneal Dialysis in a Patient with aHUS Treated with Eculizumab

 

8. Does the anxiety and self-esteem of aHUS patients vary significantly between treatment types and what can be done reduce and boost them respectively?

9. How does living with aHUS impact on education and work?

10. Do aHUS families have all the correct information to make informed family planning decisions?

Postpartum Associated Atypical Hemolytic Uremic Syndrome

Concomitant Atypical Hemolytic Uremic Syndrome and Lupus Nephritis in a Pregnant Woman

Individualized Eculizumab Every 3 Months Maintains Successful Remission In Pregnancy-Associated Hemolytic Uremic Syndrome with Hybrid CFHR1/CFH Gene: Case Report and Review of Literatures

Altered Function of TRPC6 May Contribute to Pregnancy-Associated aHUS

Role of Eculizumab in Postpartum Atypical Hemolytic Syndrome in a Kidney Transplant Recipient

Typical Atypical Hemolytic Uremic Syndrome: A Pregnancy-Induced Story

Placental Passage of Eculizumab and Complement Blockade in a Newborn

 

11. When it comes to genetic testing of aHUS family members what is best – to know or not to know -and what can be done with the knowledge?

 

12. How many aHUS patients are there in my local area, my country and the world and how do they differ?

Safety and Effectiveness of Eculizumab for Adult Patients with Atypical Hemolytic-Uremic Syndrome in Japan: Interim Analysis of Post-Marketing Surveillance

 

13. Are the predisposing genetic and triggering factors of aHUS fully catalogued and understood and will it help to know how variable are the risks of these between individuals?

Yield of Atypical Hemolytic Uremic Syndrome (aHUS) Genetic Susceptibility Panel Testing

Whole-Exome Sequencings Reveal Additional Extracellular Matrix Gene Mutations in Atypical Hemolytic Uremic Syndrome

Additional Genes Associated with Atypical Hemolytic Uremic Syndrome

Functional Consequences of Complement Activation on Vascular Endothelial Cells – Results of a Pilot RNA Seq Study

Elucidating the Role of Neutrophil Extracellular Traps in Atypical Hemolytic Uremic Syndrome

Atypical Hemolytic Uremic Syndrome (HUS): DGKE and Other Mutations in West Virginia Children’s Hospital

Eculizumab Responsive Atypical Hemolytic Uremic Syndrome Triggered by a Multisystem Lupus Flare

A Rare Case of Clostridium Difficile Causing Atypical Hemolytic Uremic Syndrome

Enteric Fever Causing Hemolytic Uremic Syndrome

Lessons from Comprehensive Analysis of a Factor I (FI) Variant in Atypical Hemolytic Uremic Syndrome (aHUS)

Scleroderma Renal Crisis – A Genetic Complementopathy?

Complement Factor I Gene Mutation with Normal Factor I Activity in a Child with ESRD Secondary to Atypical HUS

 

14. Is it more cost effective, as well as clinically effective, for the management of an aHUS patient’s treatment to undertake genetic testing?

15. Will there be a cure eventually?