Closing the aHUS knowledge gap a bit more

A key outcome from conferences like Kidney Week in Chicago ( American Society of Nephrologists.) is the sharing of knowledge from thousands of bits of research which together help close the knowledge gap on renal disease.
A quick search of the Kidney Week programme revealed thirty one topics relating to aHUS ,the full list can be seen by clicking here.
The aHUS alliance has been working on “agenda for aHUS research” to assess priorities from a patient perspective and an abstract of the early draft of a manuscript about this agenda features as one the abstracts to be included in the Supplement of JASN publication which accompanies Kidney Week.
Taking some of the aHUS research priorities identified , it can be seen how some of the abstract posters/presentations have gone some way to address understanding and knowledge gaps in these topics.
What are the outcomes of a transplant without eculizumab and what non-kidney damage is likely from any resulting aHUS onset?
This is partially addressed by  the Andrew Siedleki  group  conclusion “This retrospective analysis shows that fewer patients with aHUS who received Ecu(lizumab)  prior to  KTx( Kidney Transplant) compared with after KTx required dialysis or had TMA. This suggests initiating Ecu pre-transplant may be associated with better renal outcomes after KTx.” click here for full abstract
Jan A J G  van den Brand’s group  added more evidence about the cost effectiveness of eculizumab supported transplants acroos the spectrum and found “kidney transplantation was more cost-effective than dialysis to treat ESRD due to aHUS. However, Eculizumab upon recurrence was more cost-effective than kidney transplantation. Gain in QALYs in the lifelong eculizumab strategy was offset by extremely high costs. Therefore, eculizumab upon recurrence of aHUS was more acceptable. Click here for full abstract
Another group led by Gianluigi Ardissino offered more insight into the benefit of prophylactic eculizimab  saying “Our experience favours the prophylactic use of ECU(lizumab) in pts undergoing KTx with a history of aHUS. We recommend complete characterization (as to disease etiology) pre-KTx and that maintenance Rx (treatment) is continued lifelong. Click here for full abstract
What are the barriers to diagnosis, and how can they be overcome?
Jan Hofmann suggests one of speeding up diagnosis and concludes “with development of improved genetic testing (ie, increased speed and sensitivity), aHUS genetic assays may represent a “real-time” diagnostic tool enabling more rapid assessment of complex TMA cases.” click here for full abstract
How transient is aHUS due to. pregnancy and is there a role for prophylactic eculizumab in some cases? 
Dominico Santoro’s group found something more about aHUS onset with a complement factor H significant variant not in the terminal end normally associated with aHUS  in their conclusion that  “our case show the identification of a new gene mutations coding for complement factor H (CFH). Moreover, it emphasizes the importance of a early diagnosis of aHUS for a prompt start of therapy with eculizumab in order to avoid dialysis and induce a rapid renal recovery. Future studies will help to understand how long such therapy needs to be prolonged.” click here for full abstract
Can the degree of kidney function recovery be predicted by the time between aHUS onset and diagnosis/treatment?
Salem Almaani and team found some answers to this and concluded “the delayed administration of eculizumab is associated with an increased risk of poor renal outcomes in aHUS. Initiation of terminal complement blockade within 10 days of clinical presentation is essential for long-term preservation of renal function in patients with aHUS.”  Click here for the full abstract
Gianluigi Ardissino’s et al  found similar conclusions in their study but also that some eculizumab  late is better than none at all  stating that” early Rx ( treatment)  provides better renal outcome as to response rate, need for acute RRT, final renal function and time to reach the nadir of sCr. The better outcome turns into lower costs for the possibility of early discontinuation of ECU, for which a good residual renal function is essential. We stress that ECU Rx can be useful even if started late, we encourage to treat Pts as long as signs of ongoing TMA are present, regardless of disease duration.” Click here for full abstract
Is it possible to ensure the effectiveness of eculizumab in the body?
Kioa L Wijnsma’s group looked at withdrawal of eculizumab when not needed and found that ” a restrictive eculizumab regimen in aHUS is safe and effective. Future studies should focus on finding reliable predictors of disease recurrence”  Click here for full abstract
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