Imagine finding bruises that seem to randomly appear on your body, or having your skin erupt in dozens of tiny red spots. When you have immune thrombocytopenia, or ITP, your immune system attacks your platelets – the cells in your bloodstream that are responsible for clotting.1 A low platelet count makes it hard for the body to stop bleeding – leading to sometimes intense fatigue and causing the tell-tale purple bruises and tiny red spots on the skin that are hallmarks of the disease.1 In some cases, the bleeding can be life threatening.1
The exact cause of ITP is still unknown, although some cases have been linked to certain foods, medications or other diseases that cause low platelet counts.2 Women are 2 to 3 times more likely than men to develop the chronic form of this rare and potentially serious blood disorder.1 Acute ITP typically lasts less than 6 months and primarily affects children.1 Adults more commonly suffer from chronic ITP, when the condition lasts 6 months or longer.1
Many patients live in fear of uncontrolled bleeding or may feel embarrassed by the appearance of the red spots.3 Because a low platelet count can be a symptom of other blood disorders, doctors must first rule out all other causes before diagnosing a patient with ITP.1 Treatment depends on how severe the symptoms are and may include making certain lifestyle changes, like avoiding overexertion.4
Symptoms of ITP:
- Unexplained bruises
- Tiny red or purple dots on your skin
- Bleeding too easily from your gums, nose or cuts
- Bleeding that is hard to stop
- Blood in your stool or urine
- A heavier or unusual period flow (females)
To diagnose ITP you more than likely will have a routine complete blood test. The blood test will show the number of red blood cells, white blood cells, and platelets in your blood. In ITP, the red and white blood cell counts are normal, but the platelet count is low.
"It is my honor to be partnering with Novartis to speak about this rare blood disease," said Ivy P. Altomare, MD, hematologist in Durham, North Carolina. "For patients with rare diseases is not rare to them. They are living with it their entire lives. I think the more information people have is really better. We are fortunate to have many effective therapies and treatments for this disease. The goal is to get the platelet count into a safe and healthy range. If there is active bleeding we want to treat the bleeding. If there is a risk of bleeding we want to eliminate that risk so patients have less concern that they're going to get in trouble and they can participate in the activities that they want to so they can live a full life."
The FDA has granted approval to Novartis for the Breakthrough Therapy designation to Promacta® (eltrombopag) for use in combination with standard immunosuppressive therapy for the treatment of patients with severe aplastic anemia (SAA) as a first-line therapy. Promacta, which is marketed as Revolade® in most countries outside the US, is already approved as a second-line therapy in the refractory setting in SAA. Promacta is also approved for adults and children with chronic immune thrombocytopenia (ITP), for patients who are refractory to other treatments.
About Ivy P. Altomare, MD
Ivy P. Altomare, MD, is a hematologist in Durham, North Carolina and affiliated with multiple hospitals in the area, including Duke University Hospital and Duke Regional Hospital. Board certified in hematology, medical oncology and internal medicine, Dr. Altomare is active in ongoing clinical research those areas. She has been the lead author on recent publications related to immune thrombocytopenia (ITP), costs of cancer care, and biomarkers in oncology, among other research topics. To date, she has published more than 20 clinical studies or retrospective analyses related to hematology and oncology. She completed her medical degree at the University of Medicine and Dentistry of New Jersey and her residency and fellowship at Mount Sinai Medical Center in New York City.
About Promacta® (eltrombopag)
Eltrombopag, marketed as Promacta® in the United States and Revolade® in countries outside the US, is approved in more than 100 countries worldwide for the treatment of thrombocytopenia in adult patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an inadequate response or are intolerant to other treatments, approved in over 45 countries worldwide for the treatment of patients with severe aplastic anemia (SAA) who are refractory to other treatments, and also approved in more than 50 countries for the treatment of thrombocytopenia in patients with chronic hepatitis C to allow them to initiate and maintain interferon-based therapy. Eltrombopag is approved in the US and in the European Union for the treatment of thrombocytopenia in pediatric patients 1 year and older with chronic immune (idiopathic) thrombocytopenia (ITP) who have had an insufficient response to corticosteroids and immunoglobulins.
Important Safety Information for Promacta® (eltrombopag)
Promacta can cause serious side effects, including liver problems, abnormal liver function tests, high platelet counts and higher risk for blood clots, and new or worsened cataracts (a clouding of the lens in the eye).
Promacta is not for treatment of people with a precancerous condition called myelodysplastic syndromes (MDS). If you have MDS and receive Promacta, your MDS condition may worsen and become AML. If MDS worsens to become AML, you may die sooner from AML.
For patients who have chronic hepatitis C virus and take Promacta with interferon and ribavirin treatment, Promacta may increase the risk of liver problems. Patients should tell a healthcare provider right away if they have any of these signs and symptoms of liver problems including yellowing of the skin or the whites of the eyes (jaundice), unusual darkening of the urine, unusual tiredness, right upper stomach area pain, confusion, swelling of the stomach area (abdomen).
A healthcare provider will order blood tests to check the liver before starting Promacta and during Promacta treatment. In some cases, treatment with Promacta may need to be stopped due to changes in liver function tests.
The risk of getting a blood clot is increased if the platelet count is too high during treatment with Promacta. The risk of getting a blood clot may also be increased during treatment with Promacta if platelet counts are normal or low. Some forms of blood clots, such as clots that travel to the lungs or that cause heart attacks or strokes can cause severe problems or death. A healthcare provider will check blood platelet counts, and change the dose of Promacta or stop Promacta, if platelet counts get too high. Patients should tell a healthcare provider right away if they have signs and symptoms of a blood clot in the leg, such as swelling, pain, or tenderness in the leg.
People with chronic liver disease may be at risk for a type of blood clot in the stomach area. Patients should tell a healthcare provider right away if they have stomach area pain that may be a symptom of this type of blood clot.
New or worsened cataracts have happened in people taking Promacta. A healthcare provider will check the patient's eyes before and during treatment with Promacta. Patients should tell a healthcare provider about any changes in eyesight while taking Promacta.
Patients should tell a healthcare provider about all the medicines they take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Promacta may affect the way certain medicines work. Certain medicines may keep Promacta from working correctly. Patients should take Promacta at least 2 hours before or 4 hours after taking products such as antacids used to treat stomach ulcers or heartburn and multivitamins or products that contain iron, calcium, aluminum, magnesium, selenium, and zinc, which may be found in mineral supplements. Patients should ask a healthcare provider if they are not sure if the medicine is one that is listed above.
Patients should avoid situations and medications that may increase the risk of bleeding while taking Promacta.
The most common side effects of Promacta when used to treat chronic ITP in adults are: nausea; diarrhea; upper respiratory tract infection (symptoms may include runny nose, stuffy nose, and sneezing); vomiting; muscle aches; urinary tract infection (symptoms may include frequent or urgent need to urinate, low fever in some people, pain or burning with urination); pain or swelling (inflammation) in the throat or mouth (oropharyngeal pain and pharyngitis); abnormal liver function tests; back pain; flu-like symptoms (influenza), including fever, headache, tiredness, cough, sore throat, and body aches; skin tingling, itching, or burning; and rash.
The most common side effects of Promacta in children 1 year and older when used to treat chronic ITP are: upper respiratory tract infections (symptoms may include runny nose, stuffy nose, and sneezing); pain or swelling (inflammation) in the nose and throat (nasopharyngitis); cough; diarrhea; pyrexia; runny, stuffy nose (rhinitis); stomach (abdominal) pain; pain or swelling (inflammation) in the throat or mouth; toothache; abnormal liver function tests; rash; runny nose (rhinorrhea).
The most common side effects when Promacta is used in combination with other medicines to treat chronic HCV are: low red blood cell count (anemia); fever; tiredness; headache; nausea; diarrhea; decreased appetite; flu-like symptoms (influenza), including fever, headache, tiredness, cough, sore throat, and body aches; feeling weak; trouble sleeping; cough; itching; chills; muscle aches; hair loss; and swelling in the ankles, feet, and legs.
The most common side effects of Promacta when used to treat severe aplastic anemia are: nausea, feeling tired, cough, diarrhea, headache, pain in arms, legs, hands or feet, shortness of breath, fever, dizziness, pain in nose or throat, abdominal pain, bruising, muscle spasms, abnormal liver function tests, joint pain, and runny nose. Laboratory tests may show abnormal changes to the cells in bone marrow.
Please see full Prescribing Information, including Boxed WARNING and Medication Guide, for Promacta.
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2016, the Group achieved net sales of USD 48.5 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 121,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit http://www.novartis.com.
Courtesy: Novartis Pharmaceuticals Corporation
- National Heart, Lung and Blood Institute (2018). Immune Thrombocytopenia
- Platelet Support Disorders Association (2018). About ITP: Causes.
- Mathias, Susan D., et al. "Impact of chronic Immune Thrombocytopenic Purpura (ITP) on health-related quality of life: a conceptual model starting with the patient perspective." Health and quality of life outcomes 6.1 (2008): 1.
- ITP Foundation (2018). How is ITP treated?
Source : https://patch.com/michigan/farmington-mi/unraveling-mystery-around-rare-blood-disease1958