Tıp Fakültesi / Faculty of Medicine

Permanent URI for this collectionhttps://hdl.handle.net/11727/1403

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    Evolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in turkey: a multicenter retrospective analysis
    (2021) Karadag, Fatma Keklik; Yenerel, Mustafa Nuri; Yilmaz, Mehmet; Uskudar, Hava; Ozkocaman, Vildan; Tuglular, Tulin Firatli; Erdem, Fuat; Unal, Ali; Ayyildiz, Orhan; Ozet, Gulsum; Comert, Melda; Kaya, Emin; Ayer, Mesut; Salim, Ozan; Guvenc, Birol; Ozdogu, Hakan; Mehtap, Ozgur; Sonmez, Mehmet; Guler, Nil; Hacioglu, Sibel; Aydogdu, Ismet; Bektas, Ozlen; Toprak, Selami Kocak; Kaynar, Lale; Yagci, Munci; Aksu, Salih; Tombak, Anil; Karakus, Volkan; Yavasoglu, Irfan; Onec, Birgul; Ozcan, Mehmet Ali; Undar, Levent; Ali, Ridvan; Ilhan, Osman; Saydam, Guray; Sahin, Fahri; 34322292
    Paroxysmal nocturnal hemoglobinuria (PNH) is a rare X-linked genetic disorder. On the contrary to its name, it is a multisystemic disease and various symptoms other than hemoglobinuria could be occurred. It could be life threatening especially because of thromboembolic events. In the last decade, a terminal complement inhibition with eculizumab approved with promising results for PNH patients. We conducted this study to evaluate the long term experience of eculizumab therapy from Turkey for the first time. Our cohort included 138 patients with PNH treated with eculizumab between January 2008 and December 2018 at 28 centers in Turkey. Laboratory and clinical findings at the time of diagnosis and after eculizumab therapy were recorded retrospectively. The median age was 39 (range 18-84) years and median granulocyte PNH clone size was 74% (range 3.06-99.84%) at the time of diagnosis. PNH with bone marrow failure syndrome was detected in 49 patients and the rest of 89 patients had classical PNH. Overall 45 patients (32.6%) had a history of any prior thrombotic event before eculizumab therapy and only 2 thrombotic events were reported during the study period. Most common symptoms are fatigue (75.3%), hemoglobinuria (18.1%), abdominal pain (15.2%) and dysphagia (7.9%). Although PNH is commonly related with coombs negativity, we detected coombs positivity in 2.17% of patients. Seven months after the therapy, increased hemoglobin level was seen and remarkably improvement of lactate dehydrogenase level during the treatment was occurred. In addition to previous studies, our real life data support that eculizumab is well tolerated with no serious adverse events and improves the PNH related findings.
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    Transplantation in pediatric aHUS within the era of eculizumab therapy
    (2020) Ozcakar, Zeynep Birsin; Ozaltin, Fatih; Gulhan, Bora; Comak, Elif; Parmaksiz, Gonul; Baskin, Esra; Topaloglu, Rezan; Kasap Demir, Belde; Canpolat, Nur; Yuruk Yildirim, Zeynep; Demircioglu Kilic, Beltinge; Yuksel, Selcuk; Soylemezoglu, Oguz; 0000-0003-4361-8508; 33217100; B-5785-2018
    aHUS is caused by the over-activation and dysregulation of the alternative complement pathway. Data regarding outcomes of pediatric aHUS patients after kidney transplantation are still very scarce. Accordingly, the aim of this study was to describe the clinical findings and outcomes of pediatric aHUS patients after renal transplantation. This is a retrospective, multicenter study including 12 patients from the national registry system. Among the 12 patients, eight had received prophylactic eculizumab and none of those patients (except one) had experienced aHUS recurrence during a median follow-up period of 58.5 (min-max, 4-94) months. Although eculizumab had been started on the day before transplantation in one of them, aHUS recurrence occurred during the transplantation procedure. Eculizumab had been stopped in only one patient who had no complement gene mutation after 35 months of therapy, and recurrence had not been observed during the 19 months of follow-up. In three patients, maintenance doses had been spaced out without any recurrence. One additional patient with anti-CFH antibody received only two doses of eculizumab for transplantation and had been followed for 46 months without aHUS recurrence. The remaining three patients had not received anti-C5 therapy and none of those patients experienced aHUS recurrence during a median follow-up period of 21 (min-max, 9-42) months. Prophylactic eculizumab is a safe and effective treatment for the prevention of aHUS recurrence. Eculizumab interval prolongation, discontinuation, and transplantation without eculizumab prophylaxis can be tried in selected patients with close follow-up.