In:
Nephrology Dialysis Transplantation, Oxford University Press (OUP), Vol. 35, No. Supplement_3 ( 2020-06-01)
Abstract:
Practice patterns of Eculizumab use in patients with thrombotic microangiopathies (TMA) and C3-glomerulopathy (C3G) are not well described. Method We used the “Vienna TMA cohort” and the hospital pharmacy database at the Medical University of Vienna to identify adult patients with a history of eculizumab use between 2012 and 2019. We describe clinical characteristics, details of eculizumab use, and outcomes of patients with complement gene variant mediated TMA (cTMA), secondary TMA (sTMA) and C3G. Results As of December 2019, 212 individuals were enrolled in the Vienna TMA cohort comprising 51 cTMA, 144 sTMA, and 17 TTP patients. We included also our cohort of 14 patients with C3G for this analysis. 47 patients (22 TMA and 2 C3G, 23 other indications, i.e. paroxysmal nocturnal haemoglobinuria) received at least one dose of eculizumab at the Medical University of Vienna (Figure 1). Table 1 indicates demographic and clinical details of 15 cTMA (29.4% of all cTMA), 7 sTMA (4.9% of all sTMA) and 2 C3G (14.3% of all C3G) patients treated with eculizumab. 60% of cTMA patients showed a rare complement gene variant, while sTMA was ruled out in the remaining 40%. Causes of sTMA were bone marrow transplantation (BMT) (n=2), malignant hypertension, malignoma, systemic lupus erythematodes, antiphospholipid syndrome and lung transplantation (each n=1). One sTMA patient, a BMT recipient, had a donor with a thrombomodulin gene variant. Patients with cTMA had a greater delay from first diagnosis to treatment with eculizumab than the other groups and received maintenance therapy for a longer period of time. More female patients received eculizumab as compared to male patients. Chronic kidney disease stage improved in 60% and 43% of cTMA and sTMA patients, respectively. TMA relapses did not occur during administration of eculizumab. The 2 patients with C3G didn’t respond to eculizumab in our center. Eculizumab therapy was stopped in 66% of patients with cTMA and in all patients presenting with sTMA or C3G. In general, eculizumab was well tolerated and we did not observe life threatening infections of our patients. Three adverse drug reactions included exanthema, liver injury, and hypertensive emergency. Two patients died during therapy with eculizumab (1 cTMA, 1 C3G,) and two after cessation of eculizumab therapy (1 cTMA, 1 sTMA) resulting in a mortality of 16.7%. Conclusion Improvement of CKD stage was achieved in 60% of patients with cTMA and in 43% of patients with sTMA. In our patients with C3G, eculizumab did not improve kidney function. In general, therapy with eculizumab was well tolerated.
Type of Medium:
Online Resource
ISSN:
0931-0509
,
1460-2385
DOI:
10.1093/ndt/gfaa144.P0179
Language:
English
Publisher:
Oxford University Press (OUP)
Publication Date:
2020
detail.hit.zdb_id:
1465709-0
