Successful reduced‐intensity conditioning hematopoietic stem cell transplantation for paroxysmal nocturnal hemoglobinuria with aplastic anemia in two children |
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| Authors: | Jeffrey R. Andolina Ariel L. Reinish Razia Akhtar Suzie Noronha Jessica C. Shand Angela Girvin David N. Korones Lauren B. Bruckner Craig A. Mullen Kevin J. Curran Farid Boulad |
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| Affiliation: | 1. Department of Pediatrics, Golisano Children's Hospital, University of Rochester Medical Center, Rochester, New York;2. Department of Pediatrics, Mouth Sinai, New York City, New York;3. Department of Pediatrics, Memorial Sloan Kettering Cancer Center, New York City, New York |
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| Abstract: | 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. |
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| Keywords: | paroxysmal nocturnal hemoglobinuria pediatric stem cell transplantation |
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