Liječnički vjesnik, Vol. 140 No. 3-4, 2018.
Review article
https://doi.org/10.26800/LV-140-3-4-12
HEMATOPOIETIC STEM CELL TRANSPLANTATION IN A CHILD WITH FANCONI ANEMIA – CASE REPORT
Toni Matić
Zrinko Šalek
Radovan Vrhovac
Nadira Duraković
Ružica Lasan-Trčić
Sandra Bašić-Kinda
Dino Dujmović
Ernest Bilić
Abstract
Fanconi anemia (FA) is a rare inherited disorder associated with congenital abnormalities, progressive bone marrow failure and a predisposition for hematological and nonhematological malignant disease. Bone marrow failure initially presents with thrombocytopenia, erythrocyte macrocytosis, followed by granulocytopenia and anemia. Allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative option for the hemato-logical diseases of FA. We present a boy with pancytopenia as a first manifestation of the FA at the age of 5. The boy is the first FA patient in our country treated with HSCT. The bone marrow from a matched unrelated donor was transplanted. Conditioning regimen we used is based on GEFA03 protocol which includes fludara-bine, cylophosphamide and busulfan together with alemtuzumab and mycophenolate mofetil as GVHD prophy-laxis.
Keywords
Fanconi anemia – therapy; Hematopoetic stem cell transplantation – methods; Transplantation conditioning – methods; Graft vs host reaction – prevention and control; Transplantation, homologous; Unrelated donors; Treatment outcome
Hrčak ID:
201097
URI
Publication date:
4.6.2018.
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