Therapies | Aplastic Anemia and MDS International Foundation (AAMDSIF) Return to top.

Therapies

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There are many therapies and approaches doctors use to treat bone marrow failure disease patients. Some treatments are used for several different diseases. Others are used only for aplastic anemia, MDS or PNH. Every person's condition is unique, and each situation is different. That's why your health care team must look carefully at your specific case before recommending what's right for you.

MDS Drug Therapy

There are four medicines approved in the U.S. and European Union to treat MDS. Azacitidine (Vidaza®) and decitabine (Dacogen®) are approved to treat both low- and high-risk patients with all sub-types of MDS. Lenalidomide (Revlimid®) is approved for transfusion-dependent MDS patients with isolated del(5q) and with a low or intermediate-1 risk IPSS score. Luspatercept-aamt (REBLOZYL®) is approved to treat adult MDS patients with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T), who have been failed an erythropoiesis stimulating agent (ESA) or are unlikely to respond to an ESA, and are requiring transfusions of 2 or more red blood cell (RBC) units over 8 weeks. INQOVI® (previously known as ASTX727) is a prescribed oral tablet combination of decitabine and cedazuridine approved by the US Food and Drug Administration for adult patients with myelodysplastic syndromes (MDS) and patients with chronic myelomonocytic leukemia (CMML) including the following: • previously treated and untreated, de novo and secondary MDS with the following French-American-British subtypes (refractory anemia, refractory anemia with ringed sideroblasts, refractory anemia with excess blasts, and CMML) and• intermediate-1, intermediate-2, and high-risk International Prognostic Scoring System groups.

Pegcetacoplan (EMPAVELI®)

EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI®is given by infusion under the skin (subcutaneously) into your stomach (abdomen), back of upper arms, hips, or thighs using an infusion pump.

 

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