Recent advances in the diagnosis and treatment of pediatric acquired aplastic anemia

Acquired aplastic anemia aplastic anemia: (ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most… (AA) in children is a rare bone marrow failure bone marrow failure: A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be… that requires several special considerations for its diagnosis and treatment compared with that in adults. The most common issue is the differential diagnosis with refractory refractory: Not responsive to treatment or cure. For example, refractory anemia is a low red blood cell count that doesn't respond to standard treatments. cytopenia cytopenia: (sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count. of childhood and inherited bone marrow failure syndromes, which is crucial for making decisions on the appropriate treatment for pediatric AA. In addition to detailed morphological evaluation, a comprehensive diagnostic work-up that includes genetic analysis using next-generation sequencing will play an increasingly important role in identifying the underlying etiology etiology: The cause or origin of a disease. of pediatric AA. When discussing treatment strategies for children with acquired AA, the long-term sequelae and level of hematopoietic recovery that affect daily or school life should also be considered, although the overall survival rate has reached 90% after immunosuppressive therapy immunosuppressive therapy: Immunosuppressive drug therapy lowers your body's immune response. This prevents your immune system from attacking your bone marrow, allowing bone marrow stem cells to grow, which raises blood counts. For older patients with acquired aplastic anemia, immunosuppressive drug therapy is the… or hematopoietic cell transplantation (HCT). Recent advances in HCT for pediatric patients with acquired AA have been remarkable, with the successful use of upfront bone marrow bone marrow: The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow. transplantation from a matched unrelated donor unrelated donor: A donor that is not a sibling or other familial relation of the patient (recipient). , unrelated cord blood transplantation or haploidentical HCT as salvage treatment, and fludarabine fludarabine: The U.S. Food and Drug Administration (FDA) approved fludarabine for treating chronic lymphocytic leukemia (CCL). Researchers are studying fludarabine in combination with other medicines for treating bone marrow failure. /melphalan-based conditioning regimens. This review discusses current clinical practices in the diagnosis and treatment of acquired AA in children based on the latest data.