PNH 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.
Deferiprone
Brand name: Ferriprox
Deferiprone
Deferiprone:
Deferiprone is in a class of medications called iron chelators. It works by attaching to iron in the body so that it can be excreted (removed from the body).
Deferiprone comes in a pill form and is taken by mouth. Deferiprone is approved for use in Europe. Researchers in the U.S. are studying…
is in a class of medications called iron chelators. It works by attaching to iron in the body so that it can be excreted (removed from the body).
Deferoxamine
Brand name: Desferal
Deferoxamine
Deferoxamine:
Deferoxamine is an iron-binding agent that belongs to a class of drugs known as heavy metal antagonists. It works by helping the kidneys and gallbladder get rid of the extra iron.
is an iron-binding agent that belongs to a class of drugs known as heavy metal antagonists. It works by helping the kidneys and gallbladder get rid of the extra iron.
Eculizumab
Brand name: Soliris
Eculizumab
Eculizumab:
Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks.
…
(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
complement system:
A group of proteins that move freely in the bloodstream. These proteins support (complement) the work of white blood cells by fighting infections.
less active and reduces
hemolysis
hemolysis:
(hi-MOL-uh-suss) The destruction of red blood cells.
Soliris® is approved for the treatment of patients with PNH in nearly 50 countries worldwide.
Enoxaparin
Brand name: Lovenox
Enoxaparin
Enoxaparin:
Enoxaparin is in a class of medications called low molecular weight heparins. It works by stopping the formation of substances that cause clots. It is also used in combination with warfarin to treat blood clots in the leg.
is in a class of medications called low molecular weight heparins. It works by stopping the formation of substances that cause clots. It is also used in combination with
warfarin
warfarin:
It is used to prevent blood clots from forming. Warfarin is a tablet that is taken by mouth.
to treat blood clots in the leg.
Filgrastim
Brand name: G-CSF, NeupogenA man-made version of a naturally occurring substance in the body that promotes
white blood cell
white blood cell:
Cells in the body that fight disease and infection by attacking and killing germs. There are several types of white blood cells including neutrophils, eosinophils, basophils, lymphocytes and monocytes. Each type of cell fights a different kind of germ. Also called WBC, leukocyte.
growth in the
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
.
Fludarabine
Brand name: FludaraAn antineoplastic agent, this medicine works by slowing or stopping the growth of cancer cells.
Folate and Folic Acid
Folate
Folate:
(FOE-late) A B-vitamin that is found in fresh or lightly cooked green vegetables. It helps the bone marrow make normal blood cells. Most people get enough folate in their diet. Doctors may have people with paroxysmal nocturnal hemaglobinuria (PNH) take a man-made form of folate called folic acid.
is a B-vitamin that is found in fresh or lightly cooked green vegetables. It helps your
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
make normal blood cells. When your bone marrow has to make more cells, it needs a larger supply of
folic acid
folic acid:
See folate.
. Most people get enough folate in their diet. But if you have PNH, it's a good idea to take 1 mg each day of a man-made form of folate called folic acid.
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.
Iptacopan
Brand name: FabhaltaFABHALTA, a complement factor B inhibitor, is the first oral medication approved to treat adults with
paroxysmal nocturnal hemoglobinuria
paroxysmal nocturnal hemoglobinuria:
(par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A…
(PNH). It was approved by the U.S. Food and Drug Administration in December 2023. Fabhalta is taken twice a day in a capsule form.
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.