TY  - JOUR
TI  - Hematopoietic Stem Cell Transplantation for Patients with
Paroxysmal Nocturnal Hemoglobinuria with or without Aplastic
Anemia: A Multicenter Turkish Experience
AB  - 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
AU  - SALİM, Ozan
AU  - TOPÇUOĞLU, Pervin
AU  - ÖZDOĞU, Hakan
AU  - SUCAK, Gülsan
AU  - Velet, Mustafa
AU  - KARADOĞAN, İhsan
AU  - deveci, BURAK
AU  - SOYER, Nur
AU  - KAYA, Emin
AU  - ÜNAL, Ali
AU  - GOKER, ALI HAKAN
AU  - Kaynar, Leylagul
AU  - BOZDAĞ, Sinem CİVRİZ
AU  - YILMAZ, Fergün
AU  - Cengiz Seval, Guldane
AU  - Ozgur, Gokhan
AU  - YILMAZ, MEHMET
AU  - SAYDAM, Güray
DO  - 10.4274/tjh.galenos.2021.2021.0105
PY  - 2021
JO  - Turkish Journal of Hematology
VL  - 38
IS  - 3
SN  - 1300-7777
SP  - 195
EP  - 203
DB  - TRDizin
UR  - http://search/yayin/detay/508985
ER  -