Efficacy and safety of haploidentical hematopoietic stem cell transplantation for 17 patients with paroxysmal nocturnal hemamoglobinuria / 中华血液学杂志
Chinese Journal of Hematology
;
(12): 904-907, 2018.
Article
in Chinese
| WPRIM
| ID: wpr-1011885
ABSTRACT
Objective:
To explore the efficacy and safety of haploidentical hematopoietic stem cell transplantation (Haplo-HSCT) for paroxysmal nocturnal hemoglobinuria (PNH).Methods:
A total of 17 patients with PNH who received Haplo-HSCT from January 2013 to September 2017 were analyzed retrospectively.Results:
Of them, 4 patients had de novo PNH, 13 patients had aplastic anemia-PNH syndrome (AA-PNH). All patients received modified busulfan and Cytoxan (BuCy)-based regimens combined with anti-thymocyte globulin (ATG). Granulocyte colony-stimulating factor-mobilized bone marrow and peripheral blood stem cells were transplanted as graft. Prophylaxis for graft-versus-host disease (GVHD) was ciclosporin A+ mycophenolate mofetil (MMF)+short-term methotrexate (MTX). All patients were engrafted successfully. The median time of neutrophils to 0.5×10(9)/L and platelets to 20×10(9)/L was 12(10-15) days and 14(11-45) days, respectively. All of the 17 patients achieved full donor chimerism at 30 d after Haplo-HSCT. Seven patients developed grade Ⅱ-Ⅳ acute GVHD, and 4 chronic GVHD. Median follow-up time was 27.1 (8.6-60.4) months. Of the 17 patients, 15 survived and 2 died of severe pulmonary infection and transplant associated thrombotic microangiopathy. Three-year overall survival was (77.8±15.2)%.Conclusion:
Haplo-HSCT may be effective and safe for PNH patients who did not have matched donor.
Full text:
Available
Index:
WPRIM (Western Pacific)
Main subject:
Hemoglobins
/
Retrospective Studies
/
Hematopoietic Stem Cell Transplantation
/
Transplantation Conditioning
/
Graft vs Host Disease
/
Anemia, Aplastic
Limits:
Humans
Language:
Chinese
Journal:
Chinese Journal of Hematology
Year:
2018
Type:
Article
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