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Unusual complications after bone marrow transplantation for dyskeratosis congenita
Author(s) -
Rocha Vanderson,
Devergie Gnès,
SociÉ Gerard,
Ribaud Patricia,
EspÉrou Hélène,
Parquet Nathalie,
Gluckman Eliane
Publication year - 1998
Publication title -
british journal of haematology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.907
H-Index - 186
eISSN - 1365-2141
pISSN - 0007-1048
DOI - 10.1046/j.1365-2141.1998.00949.x
Subject(s) - dyskeratosis congenita , medicine , evans syndrome , bone marrow failure , microangiopathy , pancytopenia , bone marrow , bone marrow aplasia , aspergillosis , gastroenterology , pathology , surgery , immunology , stem cell , haematopoiesis , anemia , genetics , dna , telomere , autoimmune hemolytic anemia , biology , diabetes mellitus , endocrinology
Dyskeratosis congenita (DC) is a rare inherited disorder often associated with aplastic anaemia. We report the cases of five boys transplanted with an HLA‐identical related donor for severe aplastic anaemia (SAA) associated to DC; in all cases successful engraftment was observed. Three patients died 2–8 years after bone marrow transplantation (BMT) with signs of endothelial cell damage syndrome (kidney microangiopathy and liver veno‐occlusive disease). Another boy died 1 year after BMT from Evans syndrome and invasive aspergillosis. One boy currently presents anaemia, polyarthritis of unknown origin, pulmonary fibrosis and gut malabsorption 7.5 years after BMT. SAA associated with DC can be successfully treated by allogeneic BMT. However, these early and late complications observed are very unusual after BMT and probably reflect the association of transplanted‐related factors, evolution of the underlying disease, and increased sensitivity of endothelial cells. Modified conditioning approaches, advances in supportive care and surveillance of these unusual complications offer the possibility of improved outcome for these patients.