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Outcomes of allogeneic stem cell transplantation in patients with paroxysmal nocturnal hemoglobinuria with or without aplastic anemia
Author(s) -
Lee SungEun,
Park Sung Soo,
Jeon YoungWoo,
Yoon JaeHo,
Cho ByungSik,
Eom KiSung,
Kim YooJin,
Lee Seok,
Min ChangKi,
Kim HeeJe,
Cho SeokGoo,
Kim DongWook,
Min WooSung,
Lee Jong Wook
Publication year - 2017
Publication title -
european journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.904
H-Index - 84
eISSN - 1600-0609
pISSN - 0902-4441
DOI - 10.1111/ejh.12922
Subject(s) - paroxysmal nocturnal hemoglobinuria , medicine , aplastic anemia , transplantation , bone marrow failure , gastroenterology , stem cell , hematopoietic stem cell transplantation , hemoglobinuria , population , surgery , anemia , bone marrow , haematopoiesis , environmental health , biology , genetics
Objective The aim of this study was to evaluate the long‐term outcomes of allogeneic stem cell transplantation ( SCT ) in patients with paroxysmal nocturnal hemoglobinuria ( PNH ) with or without aplastic anemia ( AA ). Method A total of 33 patients with PNH clones who underwent allogeneic SCT were analyzed. Results After a median follow‐up of 57 months (range, 6.0‐151.3), the 5‐year estimated overall survival rate was 87.9±5.7%. Four patients died of transplant‐related mortality ( TRM ). With the exception of one patient with early TRM , 32 patients were engrafted. Two patients who had developed delayed GF received a second transplant and recovered. The cumulative incidences of acute graft‐vs‐host disease ( GVHD ) (≥grade II ) and chronic GVHD (≥moderate) were 27.3±7.9% and 18.7±7.0%, respectively. Twenty‐one patients receiving SCT with reduced‐intensity conditioning ( RIC ) had available follow‐up data for PNH cell population for the first 6 months post‐transplant. Analysis of these data revealed that the PNH clones disappeared within approximately 2 months. Conclusion RIC regimen was sufficient to eradicate PNH clones with sustained donor‐type engraftment after allogeneic SCT . Therefore, application of allogeneic SCT with RIC should be considered in patients with PNH , in accordance with the severity of the underlying bone marrow failure.