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

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

Öz:

Objective: 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

Anahtar Kelime:

Aplastik Aneminin Eşlik Ettiği ve Etmediği Paroksismal Noktürnal Hemoglobinüri Hastalarında Hematopoetik Kök Hücre Nakli Deneyimi: Çok Merkezli Türkiye Deneyimi

Öz:

Amaç: Paroksismal noktürnal hemoglobunüri (PNH) hastalarının tedavisinde kompleman sisteminin değişik basamaklardaki inhibisyonu umut vadedici bir tedavi yöntemidir. Ancak hastalığın belirti ve bulgularını kontrol etmekte etkin bir tedavi yöntemi olsa da küratif bir tedavi seçeneği değildir. Özellikle kompleman inhibisyonuna rağmen devam eden hemolizi, replasman ihtiyacı ve kemik iliği yetmezliği ile birlikteliği olan hasta grubunda halen tek küratif tedavi seçeneği allojeneik kök hücre naklidir (KHN). Bu çalışmamızda aplastik anemi ile birlikteliği olan (PNH-AA) ve olmayan PNH hastalarında yapılmış allojeneik KHN’nin sonuçlarını değerlendirmeyi amaçladık. Gereç ve Yöntemler: On ayrı merkezde PNH/PNH-AA tanısı olup allojeneik KHN yapılan toplam 35 hasta retrospektif olarak tarandı. Bulgular: On altı (%45,7) PNH ve 19 (%54,3) PNH-AA hastası çalışmaya 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 PNve PNH-AA hastalarında sırasıyla %81,3 ve %79,9 olarak hesaplandı ve gruplar arasında istatistiksel bir fark tespit edilmedi (p=0,87). Tam uyumlu akraba donör ve tam uyumlu akraba dışı donör alt grupları karşılaştırıldığında 2 yıllık OS ve GFFS oranlarında istatistiksel fark gözlenmedi. Sonuç: Seçilmiş PNH ve PNH-AA hasta grubunda akraba veya akraba dışı tam uyumlu donör ile yapılan allojeneik KHN uygun bir seçenektir. Özellikle refrakter hemolizi ve replasman ihtiyacı devam eden ve kemik iliği yetmezliği bulguları olan hasta grubunda allojeneik KHN bir tedavi seçeneği olarak değerlendirilmelidir.

Anahtar Kelime:

Belge Türü: Makale Makale Türü: Araştırma Makalesi Erişim Türü: Erişime Açık

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APA	YILMAZ F, SOYER N, Cengiz Seval G, BOZDAĞ S, TOPÇUOĞLU P, ÜNAL A, Kaynar L, Ozgur G, SUCAK G, GOKER A, Velet M, ÖZDOĞU H, YILMAZ M, KAYA E, SALİM O, deveci B, KARADOĞAN İ, SAYDAM G (2021). Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. , 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
Chicago	YILMAZ Fergün,SOYER Nur,Cengiz Seval Guldane,BOZDAĞ Sinem CİVRİZ,TOPÇUOĞLU Pervin,ÜNAL Ali,Kaynar Leylagul,Ozgur Gokhan,SUCAK Gülsan,GOKER ALI HAKAN,Velet Mustafa,ÖZDOĞU Hakan,YILMAZ MEHMET,KAYA Emin,SALİM Ozan,deveci BURAK,KARADOĞAN İhsan,SAYDAM Güray Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. (2021): 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
MLA	YILMAZ Fergün,SOYER Nur,Cengiz Seval Guldane,BOZDAĞ Sinem CİVRİZ,TOPÇUOĞLU Pervin,ÜNAL Ali,Kaynar Leylagul,Ozgur Gokhan,SUCAK Gülsan,GOKER ALI HAKAN,Velet Mustafa,ÖZDOĞU Hakan,YILMAZ MEHMET,KAYA Emin,SALİM Ozan,deveci BURAK,KARADOĞAN İhsan,SAYDAM Güray Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. , 2021, ss.195 - 203. 10.4274/tjh.galenos.2021.2021.0105
AMA	YILMAZ F,SOYER N,Cengiz Seval G,BOZDAĞ S,TOPÇUOĞLU P,ÜNAL A,Kaynar L,Ozgur G,SUCAK G,GOKER A,Velet M,ÖZDOĞU H,YILMAZ M,KAYA E,SALİM O,deveci B,KARADOĞAN İ,SAYDAM G Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. . 2021; 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
Vancouver	YILMAZ F,SOYER N,Cengiz Seval G,BOZDAĞ S,TOPÇUOĞLU P,ÜNAL A,Kaynar L,Ozgur G,SUCAK G,GOKER A,Velet M,ÖZDOĞU H,YILMAZ M,KAYA E,SALİM O,deveci B,KARADOĞAN İ,SAYDAM G Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. . 2021; 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
IEEE	YILMAZ F,SOYER N,Cengiz Seval G,BOZDAĞ S,TOPÇUOĞLU P,ÜNAL A,Kaynar L,Ozgur G,SUCAK G,GOKER A,Velet M,ÖZDOĞU H,YILMAZ M,KAYA E,SALİM O,deveci B,KARADOĞAN İ,SAYDAM G "Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience." , ss.195 - 203, 2021. 10.4274/tjh.galenos.2021.2021.0105
ISNAD	YILMAZ, Fergün vd. "Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience". (2021), 195-203. https://doi.org/10.4274/tjh.galenos.2021.2021.0105

APA	YILMAZ F, SOYER N, Cengiz Seval G, BOZDAĞ S, TOPÇUOĞLU P, ÜNAL A, Kaynar L, Ozgur G, SUCAK G, GOKER A, Velet M, ÖZDOĞU H, YILMAZ M, KAYA E, SALİM O, deveci B, KARADOĞAN İ, SAYDAM G (2021). Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. Turkish Journal of Hematology, 38(3), 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
Chicago	YILMAZ Fergün,SOYER Nur,Cengiz Seval Guldane,BOZDAĞ Sinem CİVRİZ,TOPÇUOĞLU Pervin,ÜNAL Ali,Kaynar Leylagul,Ozgur Gokhan,SUCAK Gülsan,GOKER ALI HAKAN,Velet Mustafa,ÖZDOĞU Hakan,YILMAZ MEHMET,KAYA Emin,SALİM Ozan,deveci BURAK,KARADOĞAN İhsan,SAYDAM Güray Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. Turkish Journal of Hematology 38, no.3 (2021): 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
MLA	YILMAZ Fergün,SOYER Nur,Cengiz Seval Guldane,BOZDAĞ Sinem CİVRİZ,TOPÇUOĞLU Pervin,ÜNAL Ali,Kaynar Leylagul,Ozgur Gokhan,SUCAK Gülsan,GOKER ALI HAKAN,Velet Mustafa,ÖZDOĞU Hakan,YILMAZ MEHMET,KAYA Emin,SALİM Ozan,deveci BURAK,KARADOĞAN İhsan,SAYDAM Güray Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. Turkish Journal of Hematology, vol.38, no.3, 2021, ss.195 - 203. 10.4274/tjh.galenos.2021.2021.0105
AMA	YILMAZ F,SOYER N,Cengiz Seval G,BOZDAĞ S,TOPÇUOĞLU P,ÜNAL A,Kaynar L,Ozgur G,SUCAK G,GOKER A,Velet M,ÖZDOĞU H,YILMAZ M,KAYA E,SALİM O,deveci B,KARADOĞAN İ,SAYDAM G Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. Turkish Journal of Hematology. 2021; 38(3): 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
Vancouver	YILMAZ F,SOYER N,Cengiz Seval G,BOZDAĞ S,TOPÇUOĞLU P,ÜNAL A,Kaynar L,Ozgur G,SUCAK G,GOKER A,Velet M,ÖZDOĞU H,YILMAZ M,KAYA E,SALİM O,deveci B,KARADOĞAN İ,SAYDAM G Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience. Turkish Journal of Hematology. 2021; 38(3): 195 - 203. 10.4274/tjh.galenos.2021.2021.0105
IEEE	YILMAZ F,SOYER N,Cengiz Seval G,BOZDAĞ S,TOPÇUOĞLU P,ÜNAL A,Kaynar L,Ozgur G,SUCAK G,GOKER A,Velet M,ÖZDOĞU H,YILMAZ M,KAYA E,SALİM O,deveci B,KARADOĞAN İ,SAYDAM G "Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience." Turkish Journal of Hematology, 38, ss.195 - 203, 2021. 10.4274/tjh.galenos.2021.2021.0105
ISNAD	YILMAZ, Fergün vd. "Hematopoietic Stem Cell Transplantation for Patients with Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic Anemia: A Multicenter Turkish Experience". Turkish Journal of Hematology 38/3 (2021), 195-203. https://doi.org/10.4274/tjh.galenos.2021.2021.0105