Evolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in turkey: a multicenter retrospective analysis
Özet
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.
Atıf için künye
KEKLİK KARADAĞ, F., Yenerel, M. N., YILMAZ, M., ÜSKÜDAR TEKE, H., ÖZKOCAMAN, V., Tuglular, T. F., Erdem, F., ÜNAL, A., Ayyildiz, O., Ozet, G., Comert, M., KAYA, E., Ayer, M., SALİM, O., GÜVENÇ, B., Ozdogu, H., MEHTAP, Ö., SÖNMEZ, M., Guler, N., … ŞAHİN, F. (2021). Evolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in turkey: a multicenter retrospective analysis. AMERICAN JOURNAL OF BLOOD RESEARCH, 11(3), 279–285.
BibTeX
@article{keklikkaradag2021-228535,
author = {KEKLİK KARADAĞ, FATMA and Yenerel, Mustafa Nuri and YILMAZ, MEHMET and ÜSKÜDAR TEKE, HAVA and ÖZKOCAMAN, VİLDAN and Tuglular, Tulin Firatli and Erdem, Fuat and ÜNAL, ALİ and Ayyildiz, Orhan and Ozet, Gulsum and Comert, Melda and KAYA, EMİN and Ayer, Mesut and SALİM, OZAN and GÜVENÇ, BİROL and Ozdogu, Hakan and MEHTAP, ÖZGÜR and SÖNMEZ, MEHMET and Guler, Nil and Hacioglu, Sibel and AYDOĞDU, İSMET and BEKTAŞ, ÖZLEN and TOPRAK, SELAMİ KOÇAK and Kaynar, Lale and Yagci, Munci and AKSU, SALİH and TOMBAK, ANIL and Karakus, Volkan and YAVAŞOĞLU, İRFAN and ÖNEÇ, BİRGÜL and ÖZCAN, MEHMET ALİ and ÜNDAR, LEVENT and ALİ, RIDVAN and İLHAN, OSMAN and SAYDAM, GÜRAY and ŞAHİN, FAHRİ},
title = {Evolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in turkey: a multicenter retrospective analysis},
journal = {AMERICAN JOURNAL OF BLOOD RESEARCH},
year = {2021},
volume = {11},
number = {3},
pages = {279--285},
issn = {2160-1992},
url = {https://akademik.atauni.edu.tr/yayinlar/228535-evolution-of-clinical-characteristics-of-patients-with-paroxysmal-nocturnal}
}