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Successful engraftment of haploidentical bone marrow with post‐transplantation cyclophosphamide in patients with aplastic anemia
Author(s) -
Dang Brian Norman,
De Oliveira Satiro,
Gray Ashley,
Bowles LaVette,
Moore Theodore Bruce
Publication year - 2020
Publication title -
pediatric transplantation
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 0.457
H-Index - 69
eISSN - 1399-3046
pISSN - 1397-3142
DOI - 10.1111/petr.13652
Subject(s) - medicine , aplastic anemia , fludarabine , cyclophosphamide , total body irradiation , transplantation , hematopoietic stem cell transplantation , stem cell , bone marrow failure , bone marrow , anemia , haematopoiesis , surgery , gastroenterology , chemotherapy , biology , genetics
Patients with severe aplastic anemia (SAA) may benefit from hematopoietic stem cell transplantation, but many of them lack a matched donor. Haploidentical transplantation is increasingly utilized for the treatment of nonmalignant disease where patients lack a matched donor. We report patients with aplastic anemia who experienced successful engraftments of haploidentical stem cells with post‐transplantation cyclophosphamide (PTCy). Case series and review of the literature. We present two cases of pediatric patients with severe aplastic anemia who experienced successful engraftment of haploidentical related bone marrow. Both patients received conditioning consisting of rabbit ATG, cyclophosphamide, fludarabine, and total body irradiation pretransplant, with PTCy. The conditioning regimen was well tolerated by both patients, and they achieved full donor engraftment and were weaned off all immunosuppressants. Haploidentical stem cell transplantation in patients with severe aplastic anemia may be an effective alternative when fully matched donors are not available. PTCy can facilitate successful engraftment and therefore expand the pool of eligible donors for patients with aplastic anemia.