What’s new in aHUS research? Quite a bit, if you consider that over four dozen publications have just been added to our aHUS-specific ‘Virtual Library’.
There’s a reason we’re so committed to tracking and sharing the latest in aHUS research and clinical updates– we’re also aHUS families ourselves (patients and caregivers). As with our past ‘research roundups’, there’s a widespread range of topics published which are of high interest in the aHUS community.
We’ve accumulated and sorted almost 1,600 aHUS-specific publications in 21 categories for people who wish to explore a specific topic such as transplants, pregnancy, or extra renal involvement- the impact on organs such as the heart, brain, or GI tract. (FMI on our aHUS Virtual Library,see below)
Here’s just a small selection of these recently released publications.
Complement Inhibitors – Reasons for Vigilance
Complement inhibitor therapies have transformed care for people living with conditions like aHUS—but they work by intentionally “dialing down” part of the immune system called the complement pathway. That pathway normally helps the body recognize and quickly clear certain bacteria. When it’s blocked, the body becomes more vulnerable to infections—especially from what are called encapsulated bacteria. These bacteria (such as Neisseria meningitidis, Streptococcus pneumoniae, and Haemophilus influenzae type b) have an outer “coating” that makes them harder for the immune system to detect and fight off without complement working properly. As a result, infections can develop quickly and, in some cases, become life-threatening if not treated right away.
For both prescribers and patients, understanding this risk is not about causing alarm—it’s about staying one step ahead. Even with recommended vaccinations, the risk for serious issues such as meningitis is reduced but not eliminated, so awareness matters. Patients and family caregivers should remain to act quickly if something doesn’t seem right—such as sudden fever, severe headache, neck stiffness, unusual fatigue, confusion, or flu-like symptoms that escalate quickly. Clinicians play a key role in reinforcing this vigilance, ensuring preventive steps are in place, and encouraging patients to trust their instincts. When everyone understands the “why” behind these precautions, it creates a shared safety net—where early recognition and prompt care can make all the difference.
Al Jurdi A, Kotton CN, Lafayette R. Modern challenges in infection prevention: encapsulated organisms in the era of novel complement inhibitors. Kidney Int. 2026 Mar;109(3):484-488. doi: 10.1016/j.kint.2025.12.014.
Java A, Subramanian A, Nester C. Practical Considerations for Infection Prevention with the Clinical Use of Complement Inhibitors. Clin J Am Soc Nephrol. 2026 Feb 6. doi: 10.2215/CJN.0000001020.
Subramanian AK, Java A, Gupta SK, Gupta S, Bomback AS. Strategies to Mitigate Infection Risk in Patients Receiving Complement Inhibitor Therapy. Clin Infect Dis. 2026 Feb 18:ciag081. doi: 10.1093/cid/ciag081.
Vujović A, Schaefer F, Sellier-Leclerc AL, Parolin M, Pérez-Beltrán V, Hofstetter J, Boyer O, Mancuso MC, Habbig S, Levart TK; ERKNet TMA Working Group and Other Collaborators. Antimeningococcal Protection in Patients Receiving Terminal Complement Inhibitors. Kidney Int Rep. 2025 Nov 25;11(2):103700. doi: 10.1016/j.ekir.2025.11.025.
aHUS Care: the Need for a Multidisciplinary Approach
There’s a reason the term uremic appears in the full name of this very rare disease: ‘atypical Hemolytic Uremic Syndrome’ – and that’s because kidney damage occurs with aHUS activity. Atypical HUS also can impact the functioning of other organs (kidneys, brain, heart, GI system, or any location in the body) so patients can experience symptoms that overlap with other diseases, making diagnosis and treatment complex. That’s why medical professionals in nephrology and hematology need to be alert and open to conferring with other specialists to more fully understand aHUS patient needs and disease management..
Just as importantly, coordinated teamwork leads to better, more personalized treatment. Complex conditions can change quickly and involve multiple systems at once, so having a collaborative team working with a multidisciplinary approach leads to better patient outcomes rather than fragmented treatment.
We’ve just updated our research scrolls with several more publications focused on ‘extra renal manifestations’ (as aHUS affects other than on kidneys), centered on new topics ranging from pancreatitis to skin. Check out Table 2 within the Zhou et al article below for how many aHUS cases being treated with ravulizumab have reported damage occurring to various parts of the body (System Organ Class, or SOC). Since there are multiple articles on heart (cardiac) impact, one of five on this topic is included here, as well as what arguably may be the most unexpected article – aHUS and missing molars.
Zhou Y, Wu Y, Su Y, Meng Z. Analysis of adverse drug reactions associated with ravulizumab: a retrospective pharmacovigilance study utilizing the FAERS database. Front Immunol. 2026 Feb 25;17:1736692. doi: 10.3389/fimmu.2026.1736692.
Tsiamis N, Kole C, Stefanou E, Tountas C, Theodoros H, Liapis G, Kitsiou A. Early recognition of atypical hemolytic uremic syndrome to prevent irreversible kidney injury: cardiac failure and refractory hypertension as critical clues in young patients. CEN Case Rep. 2026 Mar 24;15(2):62. doi: 10.1007/s13730-026-01100-x.
Šimunović L, Špiljak B, Kovač AZ, Vranić L, Negovetic Vranic D. Bilateral root absence of permanent first molars: literature review and case report. Eur J Paediatr Dent. 2026 Mar 4;27(1):31-36. doi: 10.23804/ejpd.2025.2333.
The Future of aHUS Treatment
If you’ve been following our website articles on the theme of aHUS horizon-scanning to determine ‘What’s Next’, it won’t be surprising to see these recent articles highlighted. Our ‘virtual library’ provides a vital snapshot of advancements in aHUS knowledge, since information and research still remains scattered regarding key words and variants in aHUS classification (how it’s grouped with other medical conditions) and nomenclature (agreement on disease name and abbreviations).
What’s the prognosis for aHUS patients long-term? What about the length of time ‘on treatment’, and what’s involved in stopping a complement inhibitor like eculizumab or ravulizumab? Have any new publications been added to the Drug Discovery/Research scroll of the virtual library?
Researchers from the UK in 2025 led with results from the SETS HUS study focused on safely stopping eculizumab. The topic of discontinuing treatment was the focus of the earlier Dutch CureIHUS study, with its 2024 follow-up publication on living with aHUS (Bouwmeester et al). As pointed out in the SETS HUS companion publication by Bryant et al (Sept 2025), various factors come into play. In late 2025, Lecouturier and Sheerin dug deeper to study which factors influenced decisions for aHUS patients to withdraw from eculizumab therapy. Continuing on this very important topic continues to be explored, within the samples noted here.
Whether it’s considerations for managing this rare disease long-term, how to conduct patient monitoring, risk of relapse or potential new treatments, you can find these and more in our virtual library’s scrolling lists titled Discontinuing Treatment/Relapse and Drug Discovery/Research.
Hockman A, Anuskiewicz S, Brennan E, Chowdhury SR, Coltoff A, Poston JN, Greenberg C, Djulbegovic B. Efficacy of eculizumab discontinuation in atypical hemolytic uremic syndrome: a systematic review and meta-analysis. Blood Adv. 2025 Dec 9;9(23):6096-6107. doi: 10.1182/bloodadvances.2025017004.
Afshar-Kharghan V. Long-term outcome and management of complement-mediated thrombotic microangiopathy/aHUS. Hematology Am Soc Hematol Educ Program. 2025 Dec 5;2025(1):147-153. doi: 10.1182/hematology.2025000700C.
Macciò L, Russo E, Costigliolo F, Battaglia Y, Viazzi F, Esposito P. New Anticomplement Drugs in Nephrology – Mechanism and Indication. Kidney Blood Press Res. 2026 Apr 10:1-19. doi: 10.1159/000551965.
A ‘Virtual Library’ of aHUS-specific Research – What is that?
Often publications with advancements and insights into this very rare disease are scattered across medical journals related to more than nephrology and hematology – atypical HUS information can be found under varied terms and across multiple specialties. That’s where the aHUS Alliance Global Action team comes in, since as aHUS patients and family caregivers ourselves we have a vested interest in staying up-to-date with current research. It’s likely that other families affected by aHUS also wish to focus on a particular topic, so to aid navigation of our ‘Research and Publications’ page we’ve not only categorized entries but also have added links to jump to each Scroll (topic section) to speed your search.
Research articles about atypical HUS are listed within our website on topic-specific lists within our Info Centre, where you can see a list for “New Research” which are then also listed by category. Use the blue ‘jump links’ at the top of the page to go directly to a specific topic category, which are: New Research, Critical Care, Diagnosis, Treatment, Discontinuing Treatment/Relapse, Thrombotic Microangiopathy (TMA), Extra Renal (Effects on Organs other than Kidneys), Pregnancy, Transplants, Genetics, Complement, Secondary aHUS, Triggers, Research in Specific Nations, Case Studies, Patient Registries: Publications, Drug Discovery/Research, Summary Articles/Literature Reviews, Consensus Documents/Guidelines, and Varied Topics.
Visit our ‘virtual library’ of almost 1,600 publications specific to aHUS
by clicking this link: Atypical HUS Research & Publications
Keeping Pace with Advancements: Are you ‘aHUS Aware’?
The aHUS Alliance Global Action team encourages researchers to release their studies and publications in full text versions, with Open Access to facilitate sharing knowledge. Additionally, we welcome and applaud those individuals and groups who include “Plain Language Versions” as part of their publication’s Supplementary Materials.
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