Variables related to chronic immune thrombocytopenia: experience from a single center and comparison to a meta-analysis.

Classically, several variables have been related to the disease course of chronic primary immune thrombocytopenia (cITP), though to date, there is no consensus on their clinical relevance. In a recent systematic review, a meta-analysis was made and confirmed the existence of certain cITP-related variables that may be related to prognosis in pediatric patients. We retrospectively analyzed a cohort of patients diagnosed with ITP, identified prognostic variables, and compared our results to the variables described by the authors. A multivariate study revealed that older age at diagnosis and higher platelet count were the only independent variables related to cITP. Children up to age 4 years and those with lower platelet counts (below 20 × 109/L) were at lower risk for cITP.Conclusion: We therefore concluded that only age and platelet count at diagnosis are independent variables that should be considered when evaluating the risk of developing cITP. What is Known: • Around 20% of patients with immune thrombocytopenia progress to chronic disease as determined by a sustained platelet count below 100×109/L for more than 12 months. • A number of variables potentially related to the development of cITP are being studied, such as age, sex, cell count, and previous treatment. What is New: • This is a new group of patients diagnosed with ITP in which the platelet count and age at diagnosis are the only independent variables closely related to cITP. • In this new series, we could not confirm other variables previously related to cITP such as total leukocyte count or the absence of treatment at diagnosis.

Hematopoietic transplantation for bone marrow failure syndromes and thalassemia.

Several genetic diseases, generally considered as congenital diseases, are characterized by bone marrow failure during early childhood. Hematopoietic stem cell transplantation is the only curative treatment for syndromes involving bone marrow failure and thalassemia. In this slate-of-the-art review, we wish to focus on the results of hematopoietic transplantation in treating some of these diseases, with a special emphasis on congenital bone marrow failure and thalassemia. The results of this procedure have improved over the previous years, mainly when performed by experienced teams. New conditioning regimes based on fludarabine and the use of HLA-identical donors have been related with better survivals. In the previous years, donors other than HLA-identical siblings have been increasingly used in patients not responding to conventional measures, but this approach needs to be evaluated in larger studies.

[Acute leukemia in patients with Down syndrome]. / Leucemia aguda en pacientes con síndrome de Down.

BACKGROUND: Children with Down syndrome (DS) have a higher risk of acute leukemia than the remaining pediatric population. A favorable outcome of acute myeloid leukemia (AML) has recently been described in these patients whereas the prognosis of acute lymphoblastic leukemias (ALL) is similar to that in other children. The main cause of morbidity and mortality in children with Down syndrome are complications related to chemotherapy, leading to numerous modifications in treatment protocols. OBJECTIVES: To characterize acute leukemias in children with Down syndrome in our center and determine their clinical outcome. METHODS AND RESULTS: Between 1990 and 2002, 214 children were diagnosed with acute leukemia at the Niño Jesus Hospital (40 with AML and 174 with ALL). Of these, eight children (3.8 %) had Down syndrome. AML (2/40) represented 5 % of myeloid leukemias and ALL (6/174) represented 3.4 % of lymphoblastic leukemias. The most frequent complication was hematologic toxicity due to chemotherapy, causing a high incidence of infections: pneumonia (5/8) and bacteriemia (5/8). In all patients, these complications led to treatment interruption or dose reduction. Two children died from treatment-related toxicity. Of these, one with AML developed fulminant sepsis due to Candida infection and the other, diagnosed with high risk ALL, died from multiorgan failure after high doses of methotrexate and ARA-C. CONCLUSIONS: Patients with Down syndrome diagnosed with acute leukemia show a higher incidence of treatment-related complications, which affects their prognosis. Consequently, individualized treatment of these children in qualified units is essential.