Atypical Hemolytic Uremic Syndrome
Conditions
Keywords
aHUS, Atypical Hemolytic Uremic Syndrome, Ravulizumab, Observational
Brief summary
This multicenter, observational cohort study uses retrospective collection of past medical history and prospective follow-up to capture longitudinal data on the management and clinical outcomes of patients with atypical hemolytic uremic syndrome (aHUS) treated with ravulizumab as part of routine clinical practice under Poland's National Drug Program (NDP).
Interventions
DRUGRavulizumab
Ultomiris
Sponsors
AstraZeneca
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients of all ages diagnosed with atypical hemolytic uremic syndrome (aHUS) who received treatment with ravulizumab under the National Drug Program (NDP) in Poland. * Patients who are willing to participate in the study and have provided informed consent by signing the informed consent form (ICF).
Exclusion criteria
* Individuals who intend to participate in a clinical trial for atypical hemolytic uremic syndrome (aHUS) on or after the date of their first ravulizumab infusion through the National Drug Program. * Patients with cognitive impairments, those who are unwilling to participate, or those facing language barriers that hinder adequate comprehension or cooperation.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve) | Up to 24 months | In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS |
| Proportion of patients attaining/maintaining. Complete TMA Response during observation (switched) | Up to 24 months | In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to Complete TMA Response | Up to 24 months | Time from initiation of ravulizumab to TMA Response: continuous variable (days; specify when complete response criteria are met) |
| Proportion of dialysis-free patients | Up to 24 months | In order to achieve the secondary objectives, the following variables will be estimated |
| Complete TMA response | Up to 24 months | In order to achieve the secondary objectives, the following variables will be estimated: * Platelet count (≥150 x 109/L) * Lactate dehydrogenase (LDH) levels (≤ULN) * Serum creatinine (≤ULN for age or an improvement \> 25% compared to baseline), |
| Proportion of patients with lab results normalization during observation | Up to 24 months | Laboratory Parameters: * platelet count (≥150 x 109/L)\*, * lactate dehydrogenase (LDH) levels (≤ULN), * serum creatinine (≤ULN for age or an improvement \> 25% compared to baseline), * serum creatinine improvement of \>50% compared to baseline * estimated glomerular filtration rate (eGFR) ≥ 60 mL/min/1.73 m2, * increase in haemoglobin of ≥ 20 g/L |
| Change from baseline in CKD stage, as evaluated by the physician over time | Up to 24 months | In order to achieve the secondary objectives, the following variables will be estimated: CKD stage 1 (rather theoretical at baseline, possible following successful treatment): eGFR ≥90 ml/min./1.73m2 CKD stage 2 (rather theoretical at baseline, possible following successful treatment): eGFR 60 - 90 ml/min./1.73m2 CKD stage 3a: eGFR 45 - 59 ml/min./1.73m2 CKD stage 3b: eGFR 30 - 44 ml/min./1.73m2 CKD stage 4: eGFR 15 - 29 ml/min./1.73m2 CKD stage 5: eGFR\< 15 ml/min./1.73m2 CKD stage 5D: need for dialysis independent from eGFR value |
| Change from baseline in proteinuria status over time | Up to 24 months | In order to achieve the secondary objectives, the following variables will be estimated: At least one of the following numbers describing the highest proteinuria and/or albuminuria: * urine protein-to-creatinine ratio \[mg/g\] (preferred) * urine protein loss \[mg/24 hours\] * urine protein concentration \[mg/dl\] * urine albumin-to-creatinine ratio \[mg/g\] (preferred) * urine albumin loss \[mg/24 hours\] Urine protein reduction to ≤ 500 mg/g (500 mg/24 hours) Urine protein reduction by ≥50% from baseline Time to urine protein reduction to ≤ 500 mg/g (500 mg/24 hours) Time to urine protein reduction by ≥50% from baseline |
| Change from baseline in the Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue; adults) and Pediatric Functional Assessment of Chronic Illness Therapy - Fatigue (Peds FACIT-F; pediatric patients) score overtime (naïve) | Up to 24 months | FACIT-Fatigue - scoring ranges from 0 (highest fatigue) to 52 (lowest fatigue), where higher scores indicate lower levels of fatigue. Peds FACIT-F - scoring ranges from 0 (highest fatigue) to 52 (lowest fatigue), where higher scores indicate lower levels of fatigue. |
| Change form baseline in EQ- 5D-5L (adults) and EQ-5D-Y- 5L (paediatric patients) score overtime (naïve) | Up to 24 months | EQ-5D-5L/EQ-5D-Y-5L consists of 2 pages: EQ-5D descriptive system and EQ visual analogue scale (EQ VAS). EQ-5D/EQ-5D-Y comprises 5 dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems, and extreme problems. EQ VAS - scale rating from 0 (worst imaginable health state) to 100 (best imaginable health state) |
Countries
Poland
Contacts
CONTACTAstraZeneca Clinical Study Information Center Study Information Center
Outcome results
None listed