Eltrombopag studied in Idiopathic Thrombocytopenic Purpura

November 28, 2007

NEW YORK (Nov. 29, 2007) -- There are estimated to be between 50,000 -- 100,000 individuals in the U.S. diagnosed with chronic Immune Thrombocytopenic Purpura (ITP), an autoimmune disease that dramatically reduces the number of platelets in their blood -- causing bruises, nosebleeds and, sometimes, life-threatening brain hemorrhages. Now, the results of an international multicenter clinical research study led by NewYork-Presbyterian Hospital/Weill Cornell Medical Center demonstrate that an investigational oral platelet growth factor called eltrombopag successfully increased platelet counts and decreased bleeding in patients with the condition.

Published in today's New England Journal of Medicine and representing the largest ever clinical trial of its kind for ITP patients, the Phase 2 study found that eltrombopag doses of 50 and 75 mg daily elevated platelet counts to a safe level (= 50,000 per cubic millimeter) in more than 75 percent of chronic ITP patients within two weeks -- compared to 11 percent of patients receiving placebo. As long as the therapy was administered, platelet counts continued to rise or remain increased, and bleeding symptoms decreased. Incidence and severity of adverse effects was similar in the placebo and eltrombopag treated groups.

"These findings represent an important step in the development of a new treatment option for those living with chronic ITP. The fact that eltrombopag elevated platelet counts in this study within one week could be very useful to chronic ITP patients in need of short-term treatment," says the study's principal investigator, Dr. James Bussel, director of the platelet research and treatment program at the Phyllis and David Komansky Center for Children's Health at NewYork-Presbyterian Hospital/Weill Cornell Medical Center and professor of pediatrics in obstetrics and gynecology and in medicine at Weill Cornell Medical College.

The study represents the second major clinical study of a platelet growth factor for ITP. In October 2006, Dr. Bussel was the lead author of publication reporting the results of a Phase I, II study of AMG 531, also in the New England Journal of Medicine.

Current and future studies will evaluate the safety and efficacy of eltrombopag as a long-term treatment for ITP, and its efficacy and safety in populations like the four million Americans with hepatitis C or patients receiving myelosuppressive chemotherapy.
-end-
Co-authors included Weill Cornell's Dr. Bethan Psaila, and physician-scientists from Prince of Wales Hospital in Hong Kong; George Cancer Specialists in Tucker, Ga.; Hematology Research Center in Moscow, Russian Federation; Hôpital La Rabta in Tuhis, Tunisia; Akademia Medyczna in Bialystok, Poland; GlaxoSmithKline in Collegeville, Pa. and Greenford, U.K.; and Barts & The London School of Medicine, London.

The study was financially supported by GlaxoSmithKline. Dr. Bussel is an Advisory Board Member for GlaxoSmithKline and has received research grant support, lecture fees, and consulting fees from GlaxoSmithKline and reports equity ownership in GlaxoSmithKline.

Immune thrombocytopenic purpura (ITP) is an autoimmune disease in which anti-platelet antibodies accelerate destruction of platelets. ITP patients commonly have platelet counts of less than 30,000 per cubic millimeter, compared to normal platelet counts of between 150,000 and 440,000. ITP affects women of child bearing age at 2-to-3 times the rate of men.

For more information, patients may call (866) NYP-NEWS.

NewYork-Presbyterian Hospital/Weill Cornell Medical Center

NewYork-Presbyterian Hospital/Weill Cornell Medical Center, located in New York City, is one of the leading academic medical centers in the world, comprising the teaching hospital NewYork-Presbyterian and Weill Cornell Medical College, the medical school of Cornell University. NewYork-Presbyterian/Weill Cornell provides state-of-the-art inpatient, ambulatory and preventive care in all areas of medicine, and is committed to excellence in patient care, education, research and community service. Weill Cornell physician-scientists have been responsible for many medical advances -- from the development of the Pap test for cervical cancer to the synthesis of penicillin, the first successful embryo-biopsy pregnancy and birth in the U.S., the first clinical trial for gene therapy for Parkinson's disease, the first indication of bone marrow's critical role in tumor growth, and, most recently, the world's first successful use of deep brain stimulation to treat a minimally-conscious brain-injured patient. NewYork-Presbyterian, which is ranked sixth on the U.S.News & World Report's list of top hospitals, also comprises NewYork-Presbyterian Hospital/Columbia University Medical Center, Morgan Stanley Children's Hospital of NewYork-Presbyterian, NewYork-Presbyterian Hospital/Westchester Division and NewYork-Presbyterian Hospital/The Allen Pavilion. Weill Cornell Medical College is the first U.S. medical college to offer a medical degree oversees and maintains a strong global presence in Austria, Brazil, Haiti, Tanzania, Turkey and Qatar. For more information, visit www.nyp.org and www.med.cornell.edu.

NewYork-Presbyterian/Weill Cornell Medical Center

Related Autoimmune Disease Articles from Brightsurf:

New insights into a potential target for autoimmune disease
With insights into a molecular pathway that regulates the activity of Tregs, a type of T cell involved in immunosuppression, research by the University of Pennsylvania's George Hajishengallis and colleagues opens up possibly new avenues for treating inflammatory and autoimmune diseases.

Research pinpoints rogue cells at root of autoimmune disease
Breakthrough cellular genomics technology has allowed Garvan and UNSW Sydney researchers to reveal genetic mutations causing rogue behaviour in the cells that cause autoimmune disease.

University of Alberta researchers discover new biomarker for rare autoimmune disease
University of Alberta researchers have identified a unique biological marker that can be used to identify the presence of the rare autoimmune disease myasthenia gravis, predict the course of the disease and identify new, personalized treatments.

Scientists discover autoimmune disease associated with testicular cancer
Using advanced technology, scientists at Chan Zuckerberg (CZ) Biohub, Mayo Clinic and University of California, San Francisco (UCSF), have discovered an autoimmune disease that appears to affect men with testicular cancer.

New proof that narcolepsy is an autoimmune disease
Researchers from the University of Copenhagen have discovered autoreactive cells in persons suffering from narcolepsy.

Novel treatments offer new hope for patients with autoimmune disease
Researchers at University of Utah Health have developed a new approach that targets the misfunctioning immune cells while leaving normal immune cells in place.

Undiplomatic immunity: Mutation causing arterial autoimmune disease revealed
Takayasu arteritis is an autoimmune disease resulting in chronic aortic inflammation leading to aneurysm or aortic regurgitation.

Immune checkpoints could be key to treating autoimmune disease myasthenia gravis
The PD-1 immune checkpoint is essential for self-recognition by the immune system, with disruption of the pathway associated with several autoimmune disorders.

Link between autoimmune, heart disease explained in mice
Autoimmune diseases such as psoriasis, lupus and rheumatoid arthritis more than double the risk of cardiovascular disease.

Organs are not just bystanders, may be active participants in fighting autoimmune disease
Findings from mouse study suggest organs affected by autoimmune disease suppress immune cells using methods similar to those used by cancer cells to evade detection.

Read More: Autoimmune Disease News and Autoimmune Disease Current Events
Brightsurf.com is a participant in the Amazon Services LLC Associates Program, an affiliate advertising program designed to provide a means for sites to earn advertising fees by advertising and linking to Amazon.com.