β-Thalassemia, originally named Cooley anemia, is an inherited blood disease. Various types of thalassemia are inherited anemias caused by mutations at the globin gene loci on chromosomes 16 and 11,…
Hydroxyurea therapy offers promise for ameliorating the clinical course of children with sickle cell disease (SCD). Hydroxyurea is a prototypic therapeutic option; it can be administered with minimal side effects,…
This article describes recent clinical and research advances in hemophilia therapy. Different prophylactic regimens for the management of severe hemophilia are described along with the use of adjuvant treatment options…
Arterial ischemic stroke (AIS) is a rare disorder in children. Research suggests that risk factors, outcomes, and presentation are different from those of adult stroke. In particular, prothrombotic abnormalities and…
With improved pediatric survival from serious underlying illnesses, greater use of invasive vascular procedures and devices, and a growing awareness that vascular events occur among the young, venous thromboembolism (VTE)…
Childhood cancer survivors are at increased risk of serious morbidity, premature mortality, and diminished health status. Proactive and anticipatory risk-based health care of survivors and healthy lifestyles can reduce these…
The past 25 years have seen an increase in our understanding of immunology and further expansion in the clinical use of immunotherapeutic modalities. How immunotherapy will be integrated with chemotherapy,…
Central nervous system (CNS) tumors comprise 15% to 20% of all malignancies occurring in childhood and adolescence. They may present in a myriad of ways, often delaying diagnosis. Symptoms and…
Neuroblastoma, a neoplasm of the sympathetic nervous system, is the second most common extracranial malignant tumor of childhood and the most common solid tumor of infancy. Neuroblastoma is a heterogeneous…
Acute myeloid leukemia (AML) is a heterogeneous group of leukemias that result from clonal transformation of hematopoietic precursors through the acquisition of chromosomal rearrangements and multiple gene mutations. As a…
