Evolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in turkey: a multicenter retrospective analysis

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dc.authoridKAYA, Emin/0000-0001-8605-8497
dc.authoridComert, Melda/0000-0002-7798-4349
dc.authoridÖneç, Birgül/0000-0003-2824-1044
dc.authoridYagci, Munci/0000-0002-0458-2920
dc.authoridGuvenc, Birol/0000-0001-7641-5673
dc.authoridGuler, Nil/0000-0003-0604-6475
dc.authoridYILMAZ, Mehmet/0000-0002-5710-5263
dc.authorwosidKAYA, Emin/W-2951-2017
dc.authorwosidilhan, osman/ABC-8182-2021
dc.authorwosidComert, Melda/ABH-5764-2020
dc.authorwosidAyyildiz, Orhan/AEO-2377-2022
dc.authorwosid, aliunal/GLQ-6094-2022
dc.authorwosidaydın kaynar, lale/GLU-6163-2022
dc.authorwosidSaydam, Guray/W-3827-2017
dc.contributor.authorKaradag, Fatma Keklik
dc.contributor.authorYenerel, Mustafa Nuri
dc.contributor.authorYilmaz, Mehmet
dc.contributor.authorUskudar, Hava
dc.contributor.authorOzkocaman, Vildan
dc.contributor.authorTuglular, Tulin Firatli
dc.contributor.authorErdem, Fuat
dc.date.accessioned2024-08-04T20:58:46Z
dc.date.available2024-08-04T20:58:46Z
dc.date.issued2021
dc.departmentİnönü Üniversitesien_US
dc.description.abstractParoxysmal 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.en_US
dc.identifier.endpage285en_US
dc.identifier.issn2160-1992
dc.identifier.issue3en_US
dc.identifier.pmid34322292en_US
dc.identifier.startpage279en_US
dc.identifier.urihttps://hdl.handle.net/11616/103156
dc.identifier.volume11en_US
dc.identifier.wosWOS:000672609100010en_US
dc.identifier.wosqualityN/Aen_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherE-Century Publishing Corpen_US
dc.relation.ispartofAmerican Journal of Blood Researchen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.subjectPNHen_US
dc.subjecteculizumaben_US
dc.subjectLDHen_US
dc.subjecthemolysisen_US
dc.subjectcoombs testen_US
dc.titleEvolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in turkey: a multicenter retrospective analysisen_US
dc.typeArticleen_US

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