Atypical hemolytic uremic syndrome
Conditions
Interventions
Observation group:None
Sponsors
Beijing Children's Hospital Affiliated to Capital Medical University Baoding Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. patients of any age with aHUS diagnosed by a medical professional at any age (first episode or recurrence). 2. Evidence of TMA, including thrombocytopenia, evidence of haemolysis, and renal dysfunction, should be documented over a 1-week period based on the following laboratory findings: (1) platelet count =1.5 × upper limit of normal (ULN), haemoglobin = ULN in adult patients (>= 18 years of age) or >= 97.5% of the upper limit of normal (ULN) for the age group in which they are enrolled in paediatric patients (patients with acute kidney injury requiring dialysis are also eligible). 3. Male and/or female. 4. able to provide signed informed consent or consent as described in Appendix 6.2.1, which includes compliance with the requirements and limitations outlined in the Intelligent Consent Form (ICF) and in this protocol.
Exclusion criteria
Exclusion criteria: 1.Patients diagnosed with HUS+ due to infection with Shiga toxin-producing Escherichia coli (STEC). 2.Patients with a clear diagnosis of TTP who exhibit ADAMTS13 activity of less than 10%. 3.A written informed consent form cannot be provided. 4.The researchers believe that any medical or psychological conditions the patient may have could potentially increase their risk of participating in the study or interfere with the study’s outcome.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Survival rate without incident; an incident is defined as ESRD or death.; | — |
Secondary
| Measure | Time frame |
|---|---|
| Descriptive data on serious adverse events (SAEs) during the treatment period 2.;Proteinuria status at different time points.;death;Time to achieve complete remission in TMA.;Urine albumin/creatinine ratio at different time points.;Descriptive data on incidents (cases of severe infections (aspergillus infections and infections caused by Neisseria meningitidis and other encapsulated bacteria), pregnancy, lactation, and follow-up;Observed values of glomerular filtration rate (eGFR) and changes from baseline.;TMA relapse.;The stages of chronic kidney disease (CKD) evaluated by the researchers were categorized as improved, stable (no change), or deteriorating compared to the baseline.;Expression of plasma C3, C4, CH50, complement factor H, |, soluble C5b-9 (sC5b-9), and CD46 at different time points.;Observed values for hematological parameters (platelets, LDH, hemoglobin), along with changes from baseline.;During the observation period, TMA showed complete remission, as evidenced by the normalization of hematological parameters (platelet count and LDH) and a 25% improvement in serum creatinine compared;Patients with normal platelet counts, normal upper limits of lactate dehydrogenase levels, serum creatinine 5% compared to baseline), glomerular filtr;The state of dialysis needs at different time points.;ESRD;Complete remission of TMA at different time points.; | — |
Countries
China
Contacts
Public ContactHui Wang
Beijing Childrens Hospital,Capital Medical University
Outcome results
None listed