RESULTS
Patient characteristics and clinical outcomes after HSCT are described
in Table 1. Although the center is located in the state of Paraná, most
patients (59%) were from other Brazilian states, spanning up to 3 400
kilometers.
The cohort consisted of malignant and non-malignant diseases (BM failure
syndromes, inborn errors of innate immunity and inborn errors of
metabolism). Diagnoses of malignant diseases were acute lymphoblastic
leukemia (n=37), acute myeloblastic leukemia (n=10), acute biphenotypic
leukemia (n=4), myelodysplastic syndrome (n=2), juvenile myelomonocytic
leukemia (n=1) and non-Hodgkin’s Lymphoma (n=1). Bone marrow failure
syndromes were acquired severe aplastic anemia (n=14), Fanconi anemia
(n=11), Blackfan Diamond anemia (n=7) and dyskeratosis congenita (n=2).
Inborn errors of innate immunity included severe combined
immunodeficiency (n=9), Wiskott Aldrich syndrome (n=5), hemophagocytic
lymphohistiocytosis (n=2), CD40L deficiency (n=1) and Griscelli syndrome
(n=1). Adrenoleukodystrophy (n=3) and mucopolysaccharidosis type I (n=1)
represented the inborn errors of metabolism.