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Yılmaz F. et al: Stem Cell Transplantation in PNH PatientsRESEARCH ARTICLEDOI: 10.4274/tjh.galenos.2021.2021.0105Turk J Hematol 2021;38:195-203Hematopoietic Stem Cell Transplantation for Patients withParoxysmal Nocturnal Hemoglobinuria with or without AplasticAnemia: A Multicenter Turkish ExperienceAplastik Aneminin Eşlik Ettiği ve Etmediği Paroksismal Noktürnal HemoglobinüriHastalarında Hematopoetik Kök Hücre Nakli Deneyimi: Çok Merkezli Türkiye DeneyimiFergün Yılmaz 1, *, Nur Soyer 2, *, Güldane Cengiz Seval 3 , Sinem Civriz Bozdağ 3, *, Pervin Topçuoğlu 3 , Ali Ünal 4, *,Leylagül Kaynar 4 , Gökhan Özgür 5, *, Gülsan Sucak 5 , Hakan Göker 6 , Mustafa Velet 6 , Hakan Özdoğu 7 ,Mehmet Yılmaz 8, *, Emin Kaya 9, *, Ozan Salim 10, *, Burak Deveci 11 , İhsan Karadoğan 11 , Güray Saydam 2, *,Fahri Şahin 2, *, Filiz Vural 2, *1Marmara University Faculty of Medicine, Department of Hematology, İstanbul, Turkey2Ege University Faculty of Medicine, Department of Hematology, İzmir, Turkey3Ankara University Faculty of Medicine, Department of Hematology, Ankara, Turkey4Erciyes University Faculty of Medicine, Department of Hematology, Ankara, Turkey5Medical Park Bahçeşehir Hospital, Clinic of Hematology and Transplantation, İstanbul, Turkey6Hacettepe University Faculty of Medicine, Department of Hematology, Ankara, Turkey7Başkent University Faculty of Medicine, Adana Bone Marrow Transplantation Center, Department of Hematology, Adana, Turkey8SANKO University Faculty of Medicine, Department of Hematology, Gaziantep, Turkey9İnönü University Faculty of Medicine, Department of Hematology, Malatya, Turkey10Akdeniz University Faculty of Medicine, Department of Hematology, Antalya, Turkey11İstanbul Gelişim University, Medstar Antalya Hospital Bone Marrow Transplantation Center, Department of Hematology, Antalya, Turkey*PESG, PNH, and Education Study GroupAbstractObjective: Although inhibition of the complement system at differentsteps is a promising therapy modality in patients with paroxysmalnocturnal hemoglobinuria (PNH), allogeneic hematopoietic stem celltransplantation (HCT) is still the only curative therapy, especially forpatients with intractable hemolysis or bone marrow failure. The aimof this study is to evaluate the outcomes of allogeneic HCT in PNHpatients with aplastic anemia (PNH-AA) or without.Materials and Methods: Thirty-five PNH/PNH-AA patients who weretreated with allogeneic HCT in 10 transplantation centers in Turkeywere retrospectively analyzed.Results: Sixteen (45.7%) and 19 (54.3%) patients were diagnosedwith classical PNH and PNH-AA, respectively. The median age of thepatients was 32 (18-51) years. The 2-year overall survival (OS) rate andrate of graft-versus-host disease-free, failure-free survival (GFFS) was81.2% and 78.1%, respectively. The 2-year OS in cases of classical PNHand PNH-AA was 81.3% and 79.9%, respectively (p=0.87), and 2-yearGFFS in cases of PNH and PNH-AA was 79% and 76% (p=0.977),ÖzAmaç: Paroksismal noktürnal hemoglobunüri (PNH) hastalarınıntedavisinde kompleman sisteminin değişik basamaklardaki inhibisyonuumut vadedici bir tedavi yöntemidir. Ancak hastalığın belirti vebulgularını kontrol etmekte etkin bir tedavi yöntemi olsa da küratif birtedavi seçeneği değildir. Özellikle kompleman inhibisyonuna rağmendevam eden hemolizi, replasman ihtiyacı ve kemik iliği yetmezliğiile birlikteliği olan hasta grubunda halen tek küratif tedavi seçeneğiallojeneik kök hücre naklidir (KHN). Bu çalışmamızda aplastik anemiile birlikteliği olan (PNH-AA) ve olmayan PNH hastalarında yapılmışallojeneik KHN’nin sonuçlarını değerlendirmeyi amaçladık.Gereç ve Yöntemler: On ayrı merkezde PNH/PNH-AA tanısı olupallojeneik KHN yapılan toplam 35 hasta retrospektif olarak tarandı.Bulgular: On altı (%45,7) PNH ve 19 (%54,3) PNH-AA hastasıçalışmaya dahil edildi. Hastaların ortanca yaşı 32 (18-51) idi.Hastalardaki sağkalım analizlerine baktığımızda; 2 yıllık genel sağkalım(GS) ve graft versus host hastalıksız ve graft yetmezliksiz sağkalım(GFFS) oranları sırasıyla %81,2 ve %78,1 idi. İki yıllık sağkalım PNH©Copyright 2021 by Turkish Society of HematologyTurkish Journal of Hematology, Published by Galenos Publishing HouseAddress for Correspondence/Yazışma Adresi: Fergün Yılmaz, M.D., Marmara University Faculty of Medicine,Department of Hematology, İstanbul, TurkeyPhone : +90 532 783 90 18E-mail : fergunaydin@hotmail.com ORCID: orcid.org/0000-0001-5118-6894Received/Geliş tarihi: February 5, 2021Accepted/Kabul tarihi: May 31, 2021195
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