MDS Drugs and Treatments
More drugs and treatments are available to treat bone marrow failure disease than ever before. Researchers constantly look for ways to enhance drugs already on the market and develop new ones with fewer side effects. Some drugs treat only one type of the disease, while others may be used to treat more than one type. Check with your doctor to find out which drugs are right for you and why.
Heparin
Brand name: Calciparine, Liquaemin
Heparin
Heparin:
Heparin is in a class of medications called anticoagulants ('blood thinners'). It works by decreasing the clotting ability of the blood. Heparin is also used to stop the growth of clots that have already formed in the blood vessels, but it cannot be used to decrease the size of clots that have…
is in a class of medications called anticoagulants ('blood thinners'). It works by decreasing the clotting ability of the blood. Heparin is also used to stop the growth of clots that have already formed in the blood vessels, but it cannot be used to decrease the size of clots that have already formed.
Hydroxyurea
Brand name: Hydrea
Hydroxyurea
Hydroxyurea:
Hydroxyurea is in a class of medications called antimetabolites. Hydroxyurea treats cancer by slowing or stopping the growth of cancer cells in your body. It can be used alone or with other medications to treat a certain types of leukemias.
is in a class of medications called antimetabolites. Hydroxyurea treats cancer by slowing or stopping the growth of cancer cells in your body. It can be used alone or with other medications to treat a certain types of leukemias.
Imetelstat
Brand name: RYTELORYTELO (imetelstat) is an oligonucleotide telomerase inhibitor used to treat adults with IPSS-R low to intermediate-1
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at…
with transfusion dependent
anemia
anemia:
(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.
requiring four or more
red blood cell
red blood cell:
The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC.
units over 8 weeks who have not responded/lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs).
Iron Chelation
Iron chelation therapy
Iron chelation therapy:
(kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S. - deferasirox, an oral iron chelator,…
is the main treatment used when you have a condition called
iron overload
iron overload:
A condition that occurs when too much iron accumulates in the body. Bone marrow failure disease patients who need regular red blood cell transfusions are at risk for iron overload. Organ damage can occur if iron overload is not treated.
. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of
red blood cell
red blood cell:
The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC.
transfusions.
Lenalidomide
Brand name: RevlimidAn immunomodulatory and anti-angiogenic agent, this drug slows the growth of blood vessels feeding abnormal cells and kills abnormal cells in the
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
.
Luspatercept-aamt
Brand name: REBLOZYL®First erythroid maturation agent (EMA) approved by the FDA for the treatment of
anemia
anemia:
(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.
in adult patients with
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at…
(MDS) 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
red blood cell:
The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC.
(RBC) units over 8 weeks.
Prednisone
Brand name: Rayos, Sterapred, Deltasone
Prednisone
Prednisone:
Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own.
How does it work?
For PNH, it may be used to decrease…
a man-made version of a
hormone
hormone:
A part of the endocrine system that serves as the body's chemical messengers. Hormones move through the bloodstream to transfer information and instruction from one set of cells to another.
produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own.
Romiplostim
Brand name: NPlate
Romiplostim
Romiplostim:
Romiplostim is in a class of medications called thrombopoietin receptor agonists. It works by causing the cells in the bone marrow to produce more platelets. Romiplostim injection is used to increase the number of platelets enough to lower the risk of bleeding, but it is not used to increase the…
is in a class of medications called thrombopoietin receptor agonists. It works by causing the cells in the
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
to produce more platelets. Romiplostim injection is used to increase the number of platelets enough to lower the risk of bleeding, but it is not used to increase the number of platelets to a normal level.
Sabatolimab
Sabatolimab
Sabatolimab:
Sabatolimab (MBG453) is a novel immuno-myeloid therapy that binds to TIM-3 on immune cells, facilitating antileukemic immune activation and phagocytic killing of leukemic cells. Sabatolimab also binds to TIM-3 on leukemic cells, potentially impeding self-renewal of LSCs via inhibition of TIM3…
(MBG453) is a novel immuno-myeloid therapy that binds to TIM-3 on immune cells, facilitating antileukemic immune activation and phagocytic killing of leukemic cells. Sabatolimab also binds to TIM-3 on leukemic cells, potentially impeding self-renewal of LSCs via inhibition of TIM3-galectin-9.
Thalidomide
Brand name: Thalomid
Thalidomide
Thalidomide:
Currently it is approved by the U.S. Food and Drug Administration (FDA) for use in treating a skin disease associated with leprosy. Thalidomide is being studied, either as a single agent or in combination with other medicines, for treating of myelodysplastic syndromes (MDS). These clinical trials…
is an old drug, though it has not been widely available since the 1960s, when it was found to cause birth defects.