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.
Blood clots are very dangerous. If you have a blood clot, you need to get help right away. Patients with PNH are at an increased risk of getting serious blood clots. Here are some ways blood clots are treated.
A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some patients with low blood counts.
If you have MDS and have a high-risk or intermediate-2 risk IPSS score, you have a higher risk of developing Acute Myelogenous Leukemia (AML). To prevent this from happening, your doctor may prescribe intensive chemotherapy in order to kill bone marrow cells that have an abnormal size, shape, or look.
Eculizumab (Soliris ®) was the first drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA) to treat PNH. It works by making your complement system less active and reduces hemolysis Soliris® is approved for the treatment of patients with PNH in nearly 50 countries worldwide.
There are several medications 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. Ivosidenib (TIBSOVO®) is approved in the US to treat treat adult patients with myelodysplastic syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH1) mutation either when the disease has relapsed or has not improved after previous treatments.
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.
Ravulizumab-cwvz (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade. On June 7, 2021, the U.S. FDA approved the expanded use of ULTOMIRIS® to include children (one month of age and older) and adolescents with PNH.