Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience

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dc.authoridAladag, Elifcan/0000-0002-1206-9908
dc.authoridKaynar, Leylagul/0000-0002-2035-9462
dc.authoridCengiz Seval, Guldane/0000-0001-9433-2054
dc.authoridKAYA, Emin/0000-0001-8605-8497
dc.authoridcivriz bozdağ, sinem/0000-0001-8359-7794;
dc.authorwosidAladag, Elifcan/ABA-4185-2020
dc.authorwosidKaynar, Leylagul/F-6991-2013
dc.authorwosidCengiz Seval, Guldane/GXV-6155-2022
dc.authorwosidKAYA, Emin/W-2951-2017
dc.authorwosidcivriz bozdağ, sinem/AAQ-4752-2020
dc.authorwosidSoyer, Nur/JYO-9281-2024
dc.contributor.authorYilmaz, Fergun
dc.contributor.authorSoyer, Nur
dc.contributor.authorSeval, Guldane Cengiz
dc.contributor.authorBozdag, Sinem Civriz
dc.contributor.authorTopcuoglu, Pervin
dc.contributor.authorUnal, Ali
dc.contributor.authorKaynar, Leylagul
dc.date.accessioned2024-08-04T20:10:08Z
dc.date.available2024-08-04T20:10:08Z
dc.date.issued2021
dc.departmentİnönü Üniversitesien_US
dc.description.abstractObjective: Although inhibition of the complement system at different steps is a promising therapy modality in patients with paroxysmal nocturnal hemoglobinuria (PNH), allogeneic hematopoietic stem cell transplantation (HCT) is still the only curative therapy, especially for patients with intractable hemolysis or bone marrow failure. The aim of this study is to evaluate the outcomes of allogeneic HCT in PNH patients with aplastic anemia (PNH-AA) or without. Materials and Methods: Thirty-five PNH/PNH-AA patients who were treated with allogeneic HCT in 10 transplantation centers in Turkey were retrospectively analyzed. Results: Sixteen (45.7%) and 19 (54.3%) patients were diagnosed with classical PNH and PNH-AA, respectively. The median age of the patients was 32 (18-51) years. The 2-year overall survival (OS) rate and rate of graft-versus-host disease-free, failure-free survival (GFFS) was 81.2% and 78.1%, respectively. The 2-year OS in cases of classical PNH and PNH-AA was 81.3% and 79.9%, respectively (p=0.87), and 2-year GFFS in cases of PNH and PNH-AA was 79% and 76% (p=0.977), without statistical significance. The OS and GFFS rates also did not differ between transplantations with matched sibling donors (MSDs) and matched unrelated donors (MUDs). Conclusion: Allogeneic HCT with MSDs or MUDS is a good option for selected patients with classical PNH and PNH-AA. In particular, patients with debilitating and refractory hemolysis and patients with bone marrow failure might form an excellent group of candidates for allogeneic HCT.en_US
dc.identifier.doi10.4274/tjh.galenos.2021.2021.0105
dc.identifier.endpage203en_US
dc.identifier.issn1300-7777
dc.identifier.issn1308-5263
dc.identifier.issue3en_US
dc.identifier.pmid34057336en_US
dc.identifier.scopus2-s2.0-85114022751en_US
dc.identifier.scopusqualityQ3en_US
dc.identifier.startpage195en_US
dc.identifier.trdizinid508985en_US
dc.identifier.urihttps://doi.org/10.4274/tjh.galenos.2021.2021.0105
dc.identifier.urihttps://search.trdizin.gov.tr/yayin/detay/508985
dc.identifier.urihttps://hdl.handle.net/11616/92608
dc.identifier.volume38en_US
dc.identifier.wosWOS:000688501100004en_US
dc.identifier.wosqualityQ4en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakTR-Dizinen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherGalenos Yayinciliken_US
dc.relation.ispartofTurkish Journal of Hematologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/openAccessen_US
dc.subjectParoxysmal nocturnal hemoglobinuriaen_US
dc.subjectTransplantationen_US
dc.subjectAllogeneic stem cell transplantationen_US
dc.subjectAplastic anemiaen_US
dc.titleHematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experienceen_US
dc.typeArticleen_US

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