Publication:
Evolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in Turkey: A multicenter retrospective analysis.

dc.contributor.authorKaradag, Fatma Keklik
dc.contributor.authorYenerel, Mustafa Nuri
dc.contributor.authorYilmaz, Mehmet
dc.contributor.authorUskudar, Hava
dc.contributor.authorTuglular, Tulin Firatli
dc.contributor.authorErdem, Fuat
dc.contributor.authorUnal, Ali
dc.contributor.authorAyyildiz, Orhan
dc.contributor.authorOzet, Gulsum
dc.contributor.authorComert, Melda
dc.contributor.authorKaya, Emin
dc.contributor.authorAyer, Mesut
dc.contributor.authorSalim, Ozan
dc.contributor.authorGuvenc, Birol
dc.contributor.authorOzdogu, Hakan
dc.contributor.authorMehtap, Ozgur
dc.contributor.authorSonmez, Mehmet
dc.contributor.authorGuler, Nil
dc.contributor.authorHacioglu, Sibel
dc.contributor.authorAydogdu, Ismet
dc.contributor.authorBektas, Ozlen
dc.contributor.authorToprak, Selami Kocak
dc.contributor.authorKaynar, Lale
dc.contributor.authorYagci, Munci
dc.contributor.authorAksu, Salih
dc.contributor.authorTombak, Anil
dc.contributor.authorKarakus, Volkan
dc.contributor.authorYavasoglu, Irfan
dc.contributor.authorOnec, Birgul
dc.contributor.authorOzcan, Mehmet Ali
dc.contributor.authorUndar, Levent
dc.contributor.authorIlhan, Osman
dc.contributor.authorSaydam, Guray
dc.contributor.authorSahin, Fahri
dc.contributor.buuauthorOzkocaman, Vildan
dc.contributor.buuauthorÖZKOCAMAN, VİLDAN
dc.contributor.buuauthorAli, Ridvan
dc.contributor.buuauthorALİ, RIDVAN
dc.contributor.departmentTıp Fakültesi
dc.contributor.orcid0000-0003-2687-9167
dc.contributor.orcid0000-0002-7798-4349
dc.contributor.orcid0000-0001-8605-8497
dc.contributor.orcid0000-0003-1977-0104
dc.contributor.orcid0000-0002-8902-1283
dc.contributor.orcid0000-0002-2176-4371
dc.contributor.orcid0000-0003-0604-6475
dc.contributor.orcid0000-0003-0757-9206
dc.contributor.orcid0000-0001-7717-5827
dc.contributor.orcid0000-0002-7195-1845
dc.contributor.orcid0000-0001-9178-2850
dc.contributor.orcid0000-0003-2824-1044
dc.contributor.orcid0000-0001-7842-9702
dc.contributor.researcheridAAA-2012-2021
dc.contributor.researcheridS-4300-2019
dc.contributor.researcheridGLU-6163-2022
dc.contributor.researcheridHRC-6282-2023
dc.contributor.researcheridABH-5764-2020
dc.contributor.researcheridW-2951-2017
dc.contributor.researcheridABE-4485-2021
dc.contributor.researcheridITT-2117-2023
dc.contributor.researcheridIZQ-0529-2023
dc.contributor.researcheridW-3827-2017
dc.contributor.researcheridGLQ-6094-2022
dc.contributor.researcheridHKM-4739-2023
dc.contributor.researcheridABC-8182-2021
dc.date.accessioned2024-07-01T06:34:50Z
dc.date.available2024-07-01T06:34:50Z
dc.date.issued2021-01-01
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.
dc.identifier.endpage285
dc.identifier.issn2160-1992
dc.identifier.issue3
dc.identifier.startpage279
dc.identifier.urihttps://hdl.handle.net/11452/42617
dc.identifier.volume11
dc.identifier.wos000672609100010
dc.indexed.wosWOS.ESCI
dc.language.isoen
dc.publisherE-century Publishing Corp
dc.relation.journalAmerican Journal Of Blood Research
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi
dc.rightsinfo:eu-repo/semantics/closedAccess
dc.subjectComplement inhibitor eculizumab
dc.subjectDisease burden
dc.subjectManagement
dc.subjectThrombosis
dc.subjectDiagnosis
dc.subjectHemolysis
dc.subjectPnh
dc.subjectEculizumab
dc.subjectLdh
dc.subjectHemolysis
dc.subjectCoombs test
dc.subjectScience & technology
dc.subjectLife sciences & biomedicine
dc.subjectMedicine, research & experimental
dc.subjectResearch & experimental medicine
dc.titleEvolution of clinical characteristics of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in Turkey: A multicenter retrospective analysis.
dc.typeArticle
dspace.entity.typePublication
local.contributor.departmentTıp Fakültesi
relation.isAuthorOfPublication516d4442-e436-45a6-87b9-741a9e213d31
relation.isAuthorOfPublication578cac50-ce4d-4818-8e99-2634ef340753
relation.isAuthorOfPublication.latestForDiscovery516d4442-e436-45a6-87b9-741a9e213d31

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