Pediatria pre prax 1/2016

Stem cell transplantation in pediatric patients with bone marrow failure

Bone marrow failure syndrome is a group of inherited or acquired disorders affecting the hematopoietic stem cells that may manifest as single cytopenia (e. g., erythroid, myeloid, or megakaryocytic) or as pancytopenia. The purpose of our study was to analyze certain laboratory parameters in patients with bone marrow failure at diagnosis of acquired aplastic anemia (AA) or myelodysplastic syndrome (MDS) and to estimate the overall survival probability in patients who underwent stem cell transplantation between January 2000 and December 2014 at the Bone Marrow Transplantation Unit of the University Children’s Hospital in Bratislava. We analyzed a cohort of 48 patients with median age at diagnosis of 11 years. Evaluation of the blood count revealed pancytopenia in 93,62 % and bicytopenia in 6,38 % of patients. According to mean corpuscular volume majority of patient had normocytic anemia. AA was diagnosed in 27 patients of whom 74 % suffered from very severe AA (VSAA) with absolute neutrophil count, (ANC) < 0,2 x 10 9/l. MDS was diagnosed in 21 patients of whom 9 had refractory cytopenia of childhood (MDS/RCC) and 12 suffered from advanced MDS (juvenile myelomonocytic leukemia, refractory anemia with excess of blasts and acute myeloblastic leukemia that evolved from MDS). The overall survival probability (OS) of the whole cohort was 75 %, OS of AA and MDS patients was 85 % and 59 % respectively.

Keywords: aplastic anemia, myelodysplastic syndrome, pancytopenia, bone marrow hypocelularity, bone marrow transplantation, overall survival probability