A Retrospective Study to Evaluate the Clinical Outcome According to Treatment in aHUS Patients in South Korea

Handok Inc.97 enrolled

Overview

Primary objective \- To determine the clinical prognosis by identifying the disease course and severity by treatment with or without eculizumab in patients with aHUS in Korea. Secondary objectives * To determine the clinical prognosis of the disease in all patients with aHUS in Korea. * To determine the clinical prognosis of the disease in patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea. * To determine the treatment responses by treatment options in patients with aHUS in Korea. * To identify risk factors that affect mortality in all patients with aHUS in Korea. * To investigate the recurrence and clinical prognosis in patients with aHUS in Korea when eculizumab is discontinued

As a retrospective, non-interventional, multi-center study

Study Type

OBSERVATIONAL

Enrollment

Conditions

aHUS

Eligibility

Sex: ALL

Medical Language ↔ Plain English

Inclusion Criteria: * Patients who are clinically diagnosed with the aHUS Exclusion Criteria: * Patients who are diagnosed with Shiga toxin-producing E. coli hemolytic-uremic syndrome (STEC-HUS). * Patients with less than 10% in a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13 (ADAMTS-13) activity test

Locations (1)

Handok

Seoul, South Korea

Outcomes

Primary Outcomes

Clinical prognosis (Renal survival) by treatment with or without eculizumab

Renal survival (end-stage renal disease \[ESRD\]-free survival)

Time frame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, or date of ESRD diagnosis, whichever came first

Clinical prognosis (Overall survival) by treatment with or without eculizumab

Overall survival (OS)

Time frame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first

Secondary Outcomes

Clinical prognosis (Renal survival) of all aHUS patients in Korea

Renal survival (ESRD-free survival)

Time frame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, or date of ESRD diagnosis, whichever came first

Clinical prognosis (Overall survival) of all aHUS patients in Korea

Overall survival (OS)

Time frame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first

Clinical prognosis (Renal survival) of patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea

Renal survival (ESRD-free survival)

Time frame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, or date of ESRD diagnosis, whichever came first

Clinical prognosis (Overall survival) of patients diagnosed with aHUS prior to the initiation of eculizumab reimbursement system in Korea

Overall survival (OS)

Time frame: from the first onset date of TMA diagnosed as aHUS to the last follow up (within March 31, 2022), death, whichever came first

Response rates of each treatment, compared to prior to start treatment

Complete response of TMA

Time frame: 3rd and 6th months