Immunosuppressive therapy for pediatric aplastic anemia: a North American Pediatric Aplastic Anemia Consortium study

Quality of response 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… and long-term outcomes for pediatric 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… remain incompletely characterized. Contemporary evidence to inform treatment of relapsed or 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. severe aplastic anemia are also limited for pediatric patients. The clinical features and outcomes for 314 children treated from 2002-2014 with immunosuppressive therapy for acquired severe aplastic anemia were analyzed retrospectively from 25 institutions in the North American Pediatric Aplastic Anemia Consortium. The majority of subjects (n=264) received horse anti-thymocyte globulin plus cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. with 61 months median follow up. Following horse anti-thymocyte globulin/cyclosporine, 71.2% (95% CI: 65.3,76.6) achieved an objective response. In contrast to adult studies, the quality of response attained in pediatric patients was high, with 59.8% (95% CI: 53.7,65.8) reaching a complete response and 68.2% (95% CI: 62.2,73.8) attaining at least a very good partial response with a platelet platelet: The smallest type of blood cell. Platelets help the blood to clot and stop bleeding. Also called a thrombocyte. count ≥50,000/μL. At 5 years post-horse anti-thymocyte globulin/cyclosporine, overall survival was 93% (95% CI: 89,96), but event free survival without subsequent treatment was only 64% (95% CI: 57,69) without a plateau. Twelve of 171 evaluable patients (7%) acquired clonal abnormalities after diagnosis with a median time of 25.2 months (range 4.3-71 months) post-treatment. Myelodysplastic syndrome or leukemia developed in 6/314 (1.9%). For relapsed/refractory disease, treatment with a hematopoietic stem cell transplant had a superior event-free survival compared to second immunosuppressive therapy treatment in a multivariate analysis (HR=0.19, 95% CI:0.08, 0.47, p=0.0003). This study highlights the need for improved therapies leading to sustained high quality remission for children with severe aplastic anemia.