Eltrombopag plus cyclosporine A for moderate aplastic anemia (EMAA): a placebo-controlled, double-blind, phase 3 trial

Eltrombopag Eltrombopag: What are the possible side effects of eltrombopag (Promacta)? Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat. Stop using eltrombopag and call your doctor at once if you have: … combined with horse antithymocyte globulin antithymocyte globulin: ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH. and cyclosporine cyclosporine: Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure. A (CSA) is the standard of care for patients with 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… who are not eligible for stem cell transplantation. No consensus exists on the optimal treatment of moderate aplastic anemia (MAA). The "Eltrombopag for MAA" (EMAA) trial is an investigator-initiated, prospective, randomized, placebo placebo: A placebo is an inactive pill, liquid, or powder that has no treatment value. -controlled, double-blind, multicenter, phase III trial and compared eltrombopag+CSA (n=41) with placebo+CSA (n=44) as a first-line therapy for MAA patients with clinically significant cytopenia cytopenia: (sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count. . In patients with evaluable response at week 24 (n=75), the overall response rate (ORR), including partial response (PR) and complete response (CR), was significantly higher in the eltrombopag+CSA arm (71.4%; 95% confidence interval [CI], 54.8%-83.8%) than in the placebo+CSA arm (42.5%; 95% CI, 28.5%-57.8%) (p=0.011, Fisher's exact test; primary endpoint). After response assessment at week 24 and unblinding, patients in the placebo arm without CR were switched to receive eltrombopag in addition to continued CSA. This group achieved an ORR of 73.5% by week 48 (p=0.009, McNemar test, comparing response at 24 vs. 48 weeks). Relapse rate and failure-free survival did not differ significantly among the arms. Eltrombopag was well tolerated without new safety concerns. Adding eltrombopag to CSA for first-line treatment of MAA significantly improves trilineage hematologic response at week 24 and addition of eltrombopag in patients without CR after 24 weeks of single-agent CSA treatment also significantly improves the ORR. The trial was registered at www.clinicaltrials.gov as #NCT02773225 and at European Union Drug Regulating Authorities Clincial Trials as EudraCT2014-000174-19.