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Afibrinogenemia is a rare blood clotting disorder with an estimated prevalence of 1: 1,000,000. It is an autosomal recessive disease caused by mutations in one of the three genes encoding the three polypeptide chains of fibrinogen located on the long arm of chromosome 4. Spontaneous bleeding, bleeding after minor trauma, and excessive bleeding during interventional procedures are the main symptoms. Replacement therapy is the mainstay of management of bleeding episodes in these patients, with plasma-derived fibrinogen concentrate being the drug of choice. Cryoprecipitate and fresh frozen plasma are alternative treatments that should only be used when fibrinogen concentrate is not available. Secondary preventive treatment can be considered after life-threatening bleeding, but primary preventive treatment is currently not recommended. We also discuss alternative treatment options and management of surgery, pregnancy, and thrombosis in these patients. New tests to identify at-risk patients and the development of safer replacement therapies will improve the treatment of afibrinogenemia in the future.

Acute leukemia is the most common childhood malignancy, accounting for nearly 35% of all childhood cancers. Acute myeloid leukemia (AML) accounts for 15-20% of childhood acute leukemias. The majority of AML cases are de novo, but a minority may present as secondary malignancies. AML is a highly heterogeneous disease, the diagnosis of which involves morphology, immunophenotyping, cytochemistry, and diagnostic analyzes involving leukemic blasts derived from peripheral blood or bone marrow exhibiting cytogenic and molecular characteristics. Includes combinations. By identifying recurrent genetic mutations, it is now possible to improve individual prognosis and guide treatment management. Pediatric acute myeloid leukemia (AML) is a heterogeneous disease that requires a multifaceted therapeutic approach. Although the outcomes of low-risk AML have improved significantly over the past decades, high-risk AML continues to be associated with poor prognosis. Recent advances in molecular diagnostics, risk stratification, and supportive care have helped improve outcomes in childhood AML.