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Development of hemolytic paroxysmal nocturnal hemoglobinuria without graft loss following hematopoietic stem cell transplantation for acquired aplastic anemia
Author(s) -
Oved Joseph H.,
Stanley Natasha,
Babushok Daria V.,
Huang Yanping,
Duke Jamie L.,
Monos Dimitrios S.,
Teachey David T.,
Olson Timothy S.
Publication year - 2019
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.13393
Subject(s) - paroxysmal nocturnal hemoglobinuria , medicine , hematopoietic stem cell transplantation , immunology , aplastic anemia , bone marrow failure , hemoglobinuria , disease , stem cell , hemolytic anemia , haematopoiesis , bone marrow , hemolysis , biology , genetics
PNH is the most common clonal hematopoietic disorder arising in patients with aAA. PNH is caused by mutations in PIGA, a gene that encodes the catalytic subunit of an enzyme involved in the biosynthesis of GPI anchors, transmembrane glycolipids required for cell surface expression of many proteins. PNH clones likely arise as immune escape mechanisms in aAA by preventing CD1D‐restricted T‐cell recognition of GPI anchors and GPI‐linked autoantigens. Though many patients with aAA treated with IST will develop subclinical PNH clones, only a subset will develop PNH disease, characterized by increased thrombosis, intravascular hemolysis, and potential for severe organ dysfunction. In contrast to IST, allogeneic HSCT for patients with aAA is thought to cure bone marrow aplasia and prevent hematopoietic clonal evolution to PNH. Herein, we present a phenomenon of host‐derived PNH disease arising in a patient with aAA many years following MSD‐BMT, highlighting the importance of monitoring for this clonal disease in aAA patients with stable mixed donor/recipient chimerism after HSCT. We also provide a literature review for similar occurrences of PNH arising after HSCT.