Turk J Haematol. 2021 May 31. doi: 10.4274/tjh.galenos.2021.2021.0105. Online ahead of print.
ABSTRACT
OBJECTIVE: Although inhibition of complement system at different steps is a promising therapy modality in PNH (paroxysmal paroxysmal nocturnal hemoglobinuria) patients, allogeneic hematopoetic 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 or without aplastic anemia ( PNH-AA).
MATERIAL AND METHODS: 35 PNH / PNH-AA patients who were treated with allegeneic HCT in ten transplantation centers in Turkey were retrospectively analyzed.
RESULTS: 16 (45.7 %) and 19 (54.3 %) patients were diagnosed as classical PNH and PNH / AA respectively. The median age of the patients was 32 ( 18-51) 2-year overall survival (OS) and GVHD-free, failure-free survival (GFFS) was 81.2 % and 78.1 % , respectively. 2 year OS in classical PNH and PNH /AA was 81.3 % and % 79.9 (p =0,87), respectively and 2 year GFFS in PNH and PNH/AA was 79% and 76% (0,977) without statistical significance. OS and GFFS rates did not differ between transplantations with matched sibling donor (MSD) and matched unrelated donor (MUD), neither.
CONCLUSION: Allo HCT with MSD or MUD is a good option in selected patients with classical PNH and PNH / AA. Especially, patients with debilitating and refractory hemolysis and patients with bone marrow failure might form the excellent group for allo – HCT.
PMID:34057336 | DOI:10.4274/tjh.galenos.2021.2021.0105
