Research that matters to aHUS patients at ASH

The American Society of Haematology hosts its annual conference in San Diego from 30th November to 4th December 2018 .

A search through the programme of events reveals the following studies of relevance to aHUS patients .

Ravulizumab studies on PNH patients are included as this drug is due to replace eculizumab for aHUS too  if trials are successful. Also given the haematological nature of the conference the topic of TMA in stem cell bone marrow transplants as a trigger of secondary aHUS is well featured. The studies are catergorised by the research topics included in the Interim aHUS Patients’ Research Agenda,

 

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

3565 Economic Benefit of Early Inhospital Diagnosis and Treatment Initiation of Eculizumab in aHUS

3748 An Alternative Pathway Specific Flow Cytometric Assay to Detect Complement Activation in Atypical Hemolytic Uremic Syndrome (aHUS)

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

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

625 Results from a Phase 3, Multicenter, Non-Inferiority Study of Ravulizumab (ALXN1210) Versus Eculizumab in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria Currently Treated with EculizumabClinically Relevant Abstract

2330 A Prospective Analysis of Breakthrough Hemolysis in 2 Phase 3 Randomized Studies of Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria

626 Ravulizumab (ALXN1210) Versus Eculizumab in Adults with Paroxysmal Nocturnal Hemoglobinuria: Pharmacokinetics and Pharmacodynamics Observed in Two Phase 3 Randomized, Multicenter Studies

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

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

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

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

3754 Chronic Kidney Disease, Hypertension and Cardiovascular Sequelae during Long Term Follow up of Adults with Atypical Hemolytic Uremic Syndrome

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

How does living with aHUS impact on education and work?

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

4810 Thrombotic Microangiopathies Associated with Pregnancy: Diagnostic and Therapeutic Challenges

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?

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

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?

2323 Current Status and Optimal Management of Eculizumab Poor-Responders Due to C5 Polymorphisms

3383 Largest Reported Series of Adults Who Received Treatment with Eculizumab (ECU) for Hematopoietic Cell Transplantation-Associated Thrombotic Microangiopathy (TA-TMA). a Single Center Experience

3401 Pre-Transplant Genetic Susceptibility in Adult Allogeneic Hematopoietic Cell Transplant Recipients: Incidence and Clinical Relevance in Transplant-Associated Thrombotic Microangiopathy

2090 Transplant Associated Thrombotic Microangiopathy (TA-TMA): Lessons Learned from a Large Pediatric Center

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

Will there be a cure eventually?