Outcomes of patients with complement-mediated thrombotic microangiopathy

Abstract

Purpose: Complement-mediated thrombotic microangiopathy (CM-TMA) is a rare, progressive and lifethreatening type of thrombotic microangiopathy (TMA) characterized by microangiopathic hemolytic anemia, thrombocytopenia and associated acute kidney disease (AKI) caused by dysregulation of the alternative complement pathway. The aim of this study was to retrospectively analyze the clinical features, follow-up, treatment and mortality of patients with CM-TMA. Materials and methods: This was a retrospective study evaluating 13 patients diagnosed with CM-TMA who were followed retrospectively from 2024. Data were collected through a comprehensive review of electronic medical records of patients diagnosed with CM-TMA and receiving Eculizumab in the Department of Hematology and Nephrology. Results: Thirteen patients with a mean age at diagnosis of 36.0±17.8 years were included. Age at disease onset ranged from 17 to 66 years. Only 3 (23.1%) patients were over 50 years of age. All patients were female. The mean follow-up period was 78.6±34.6 months. After an increase in GFR with eculizumab treatment, 76.9% of patients were weaned from dialysis. Conclusion: CM-TMA was found to be predominant in young women. Eculizumab treatment provided significant improvements in clinical and laboratory values of the patients.

Keywords

• CM-TMA
• aHUS
• C5 inhibitorleri
• eculizumab
• complement-mediated thrombotic microangiopathy

Kompleman aracılı trombotik mikroanjiyopatili hastaların sonuçları

Öz

Amaç: Kompleman aracılı trombotik mikroanjiyopatili (CM-TMA), alternatif kompleman yolunun düzensizliğinden kaynaklanan mikroanjiyopatik hemolitik anemi, trombositopeni ve eşlik edebilen akut böbrek hastalığı (ABH) ile karakterize nadir, ilerleyici ve yaşamı tehdit eden bir trombotik mikroanjiyopati (TMA) türüdür. Bu çalışmada retrospektif olarak izlenilen CM-TMA hastaların klinik özellikleri, takibi tedavisi ve mortalitesinin incelemesi amaçlanmıştır. Gereç ve yöntem: Bu çalışma, 2024 yılından itibaren geriye dönük olarak izlenilen 13 CM-TMA tanılı hastaları değerlendiren retrospektif bir çalışma olarak yapıldı. Veriler, Hematoloji ve Nefroloji Bölümü'nde CM-TMA tanısı konulan ve Eculizumab alan hastaların elektronik tıbbi kayıtlarının kapsamlı bir incelemesiyle toplanmıştır. Bulgular: Tanı anındaki yaş ortalamaları 36,0±17,8 olan 13 hasta alındı. Hastalık başlangıç yaşı 17 ile 66 arasında değişmekteydi. 50 yaş üzerinde olan sadece 3 (%23,1) hasta vardı. Hastaların tümü kadındı. Hastaların ortalama takip süresi 78,6±34,6 aydı. Eculizumab tedavisi ile GFR de artma sonrası hastaların %76,9 diyalizden çıkarılarak iyileşti. Sonuç: CM-TMA genç kadınlarda baskın olduğu görüldü. Eculizumab tedavisi ile hastaların klinik ve laboratuvar değerlerinde önemli düzelmeler sağladı.

Anahtar Kelimeler

• CM-TMA
• aHUS
• C5 inhibitörleri
• eculizumab
• kompleman aracılı trombotik mikroanjiyopati

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