Ravulizumab Outcomes in Polish Patients With aHUS

Browse trials for

• Atypical Hemolytic Uremic Syndrome 42

Keywords

• aHUS
• Atypical Hemolytic Uremic Syndrome
• Ravulizumab
• Observational

Study population

Groups and cohorts

• Prospective cohort

  Group of patients naive to complement inhibitors

  Drug: Ravulizumab

• Retrospective cohort

  Group of patients who transitioned from other complement inhibitors to ravulizumab.

  Drug: Ravulizumab

Interventions

• DrugRavulizumab

  Ultomiris

Primary outcomes

1. Proportion of patient attaining Complete Thrombotic Microangiopathy (TMA) Response during observation (naïve)

   In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS

   Time frame: Up to 24 months

2. Proportion of patients attaining/maintaining. Complete TMA Response during observation (switched)

   In order to achieve the primary objectives, the following variables will be estimated: To assess ravulizumab primary treatment outcome in Polish patients with aHUS

   Time frame: Up to 24 months

Secondary outcomes

1. Time to Complete TMA Response

   Time from initiation of ravulizumab to TMA Response: continuous variable (days; specify when complete response criteria are met)

   Time frame: Up to 24 months

2. Proportion of dialysis-free patients

   In order to achieve the secondary objectives, the following variables will be estimated

   Time frame: Up to 24 months

3. Complete TMA response

   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),

   Time frame: Up to 24 months

4. Proportion of patients with lab results normalization during observation

   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

   Time frame: Up to 24 months

5. Change from baseline in CKD stage, as evaluated by the physician over time

   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

   Time frame: Up to 24 months

6. Change from baseline in proteinuria status over time

   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

   Time frame: Up to 24 months

7. 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)

   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.

   Time frame: Up to 24 months

8. Change form baseline in EQ- 5D-5L (adults) and EQ-5D-Y- 5L (paediatric patients) score overtime (naïve)

   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)

   Time frame: Up to 24 months

Individual participant data

Plan to share: Yes — Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.