Kompleman aracılı trombotik mikroanjiyopatili hastaların sonuçları

Yıl 2025, , 151 - 155, 01.01.2025

Davut Akın

https://doi.org/10.31362/patd.1579336

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

Outcomes of patients with complement-mediated thrombotic microangiopathy

Öz

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.

Anahtar Kelimeler

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

Kaynakça

• George JN, Nester CM. Syndromes of thrombotic microangiopathy. N Engl J Med. 2014;371 (7):654-666. doi:10.1056/NEJMra1312353
• Raina R, Grewal MK, Radhakrishnan Y, et al. Optimal management of atypical hemolytic uremic disease: challenges and solutions. Int J Nephrol Renovasc Dis. 2019;12:183-204. Published 2019 Sep 4. doi:10.2147/IJNRD.S215370
• Fakhouri F, Zuber J, Frémeaux-Bacchi V, Loirat C. Haemolytic uraemic syndrome [published correction appears in Lancet. 2017 Aug 12;390(10095):648. doi: 10.1016/S0140-6736(17)31055-3]. Lancet. 2017;390(10095):681-696. doi:10.1016/S0140-6736(17)30062-4
• Loirat C, Fakhouri F, Ariceta G, et al. An international consensus approach to the management of atypical hemolytic uremic syndrome in children. Pediatr Nephrol. 2016;31(1):15-39. doi:10.1007/s00467-015-3076-8
• Canpolat N. Hemolitik üremik sendrom. Türk Pediatr Ars. 2015 Haziran; 50(2):73–82.
• Yan K, Desai K, Gullapalli L, Druyts E, Balijepalli C. Epidemiology of atypical hemolytic uremic syndrome: a systematic literature review. Clin Epidemiol. 2020;12:295-305. Published 2020 Mar 12. doi:10.2147/CLEP.S245642
• Sperati CJ, Moliterno AR. Thrombotic microangiopathy: focus on atypical hemolytic uremic syndrome. Hematol Oncol Clin North Am. 2015;29(3):541-559. doi:10.1016/j.hoc.2015.02.002
• Amisha F, Konda M, Malik P, Fugere B, Roy AM, Sasapu A. Clinical, laboratory, and genetic profiles and outcomes of patients with aHUS and restrictive use of eculizumab: a single-center experience. J Hematol Oncol Pharm. 2023;13(5):256-263
• Koehl B, Boyer O, Biebuyck-Gougé N, et al. Neurological involvement in a child with atypical hemolytic uremic syndrome. Pediatr Nephrol. 2010;25(12):2539-2542. doi:10.1007/s00467-010-1606-y
• Brocklebank V, Walsh PR, Smith-Jackson K, et al. Atypical hemolytic uremic syndrome in the era of terminal complement inhibition: an observational cohort study. Blood. 2023;142(16):1371-1386. doi:10.1182/blood.2022018833
• Conkar S, Mir S, Berdeli A. Genetics and outcome of atypical hemolytic-uremic syndrome in Turkish children: a retrospective study between 2010 and 2017, a single-center experience. Iran J Kidney Dis. 2019;13(5):316-321.
• Baskin E, Gulleroglu K, Kantar A, Bayrakci U, Ozkaya O. Success of eculizumab in the treatment of atypical hemolytic uremic syndrome. Pediatr Nephrol. 2015;30(5):783-789. doi:10.1007/s00467-014-3003-4
• Štolbová Š, Bezdíčka M, Seeman T, et al. Molecular basis and outcomes of atypical haemolytic uraemic syndrome in Czech children. [published correction appears in Eur J Pediatr. 2022 Apr;181(4):1781. doi: 10.1007/s00431-021-04342-2]. Eur J Pediatr. 2020;179(11):1739-1750. doi: 10.1007/s00431-020-03666-9
• Kara MA, Kilic.BD. Retrospective evaluation of patients with diagnosis of atypical hemolytic uremic syndrome: Single center experience. Ann Med Res. 2022;29(12):1425-431. doi: 10.5455/annalsmedres.2022.07.221
• Bouwman HB, Guchelaar HJ. The efficacy and safety of eculizumab in patients and the role of C5 polymorphisms. Drug Discov Today. 2024;29(9):104134. doi:10.1016/j.drudis.2024.104134