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Successful reduced‐intensity conditioning hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia in two children
Author(s) -
Andolina Jeffrey R.,
Reinish Ariel L.,
Akhtar Razia,
Noronha Suzie,
Shand Jessica C.,
Girvin Angela,
Korones David N.,
Bruckner Lauren B.,
Mullen Craig A.,
Curran Kevin J.,
Boulad Farid
Publication year - 2018
Publication title -
pediatric blood and cancer
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.116
H-Index - 105
eISSN - 1545-5017
pISSN - 1545-5009
DOI - 10.1002/pbc.27218
Subject(s) - paroxysmal nocturnal hemoglobinuria , medicine , pancytopenia , aplastic anemia , hematopoietic stem cell transplantation , bone marrow failure , hemoglobinuria , stem cell , savior sibling , transplantation , anemia , bone marrow , haematopoiesis , biology , genetics
Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare cause of bone marrow failure in children. We report two children who presented with pancytopenia, and were diagnosed with PNH with severe aplastic anemia. Both children underwent upfront, successful hematopoietic stem cell transplantation with reduced‐intensity conditioning. One patient had a syngeneic donor, and one patient had a 10/10 matched unrelated donor. Neither patient developed graft versus host disease, infections, or recurrent PNH. Reduced‐intensity conditioning hematopoietic stem cell transplantation is a reasonable therapy for PNH with marrow failure in children.