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Stem cell transplantation for children with Glanzmann thrombasthenia
Author(s) -
Connor P.,
Khair K.,
Liesner R.,
Amrolia P.,
Veys P.,
Ancliff P.,
Mathias M.
Publication year - 2008
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 - 10.1111/j.1365-2141.2007.06890.x
Subject(s) - medicine , thrombasthenia , glanzmann's thrombasthenia , transplantation , stem cell , platelet , sibling , immunology , biology , platelet aggregation , genetics , psychology , developmental psychology
Summary Glanzmann thrombasthenia (GT) is a rare autosomal recessive platelet function disorder. Stem cell transplantation (SCT) is curative, but it is only indicated in selected patients with a severe clinical phenotype or who develop anti‐platelet antibodies. SCT have previously been limited to full intensity myeloablative conditioning regimens. This study details the successful outcome of SCT in five consecutive patients with GT, three of whom received reduced intensity conditioning (RIC) with stem cells from non‐sibling donors. This is the first time RIC SCT has been reported in GT, and offers the possibility of curative therapy with reduced late effects.