Immunosuppressive therapy for severe aplastic anemia in children under the age of 3 years yields a high response rate: a North American collaborative study

Distinguishing acquired (idiopathic/immune) severe 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… (SAA) from inherited 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… syndromes (IBMFS) is critical since only acquired cases respond to 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… (IST), the standard treatment in cases without a matched sibling donor. Differentiating SAA from IBMFS is particularly challenging in children younger than 3 years, in whom inherited disorders are more prevalent. No age threshold exists to guide IST use in this population. Thus, we evaluated IST outcomes in children aged.