Navigation Links
NIH study in NEJM shows Novartis drug eltrombopag as first-line therapy with standard treatment improves responses in severe aplastic anemia

EAST HANOVER, N.J., April 19, 2017 /PRNewswire/ -- Novartis today announced the publication of a study conducted by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH) demonstrating that 58% of patients with treatment-naïve severe aplastic anemia (SAA) achieved complete response at six months when treated with eltrombopag at the initiation of and concurrently with standard immunosuppressive treatment1. The study evaluated three sequential treatment groups, or cohorts. Cohort 3 added eltrombopag at the initiation of immunosuppressive therapy and showed a higher complete response rate than cohorts 1 and 2, where eltrombopag was initiated on day 14. The data is published in the latest issue of The New England Journal of Medicine.

SAA is a rare and serious blood disorder in which a patient's bone marrow fails to make enough red blood cells, white blood cells and platelets2. As a result, people living with SAA may experience debilitating symptoms and complications, such as fatigue, trouble breathing, recurring infections and abnormal bruising or bleeding that can limit their daily activities2. The current standard of care includes immunosuppressive therapy (IST) or hematopoietic stem cell transplantation. However, one-quarter to one-third of patients will not respond to IST and 30-40% of responders will relapse, causing symptoms to return3.

"Our research in NEJM shows that eltrombopag plus standard immunosuppressive therapy appeared to increase the overall response rate and substantially increase the frequency, speed and robustness of hematologic recovery in patients with SAA compared to historical controls," said the study's lead author, Danielle Townsley, MD, researcher in the NHLBI.

In the NIH study, the primary efficacy endpoint of complete response rate with eltrombopag plus standard immunosuppressive treatment at six months exceeded the historic rate (10%) across all three treatment cohorts (cohorts differed in length of eltrombopag administration; dose adjusted by age)1. Patients in cohort 1 received eltrombopag from day 14 to six months and achieved a complete response rate of 33%. The complete response was lowest in cohort 2 (26%), in which eltrombopag exposure was shortest (day 14 to three months). Furthermore, overall increases in platelet and neutrophil blood level counts were higher in comparison to the historic cohort, which is a key treatment goal for SAA1,4. The overall survival rate at a median follow-up of two years was 97% (95% CI, 94-100%) for all cohorts1.

"We are committed to improving the care of people living with serious conditions over the long term, particularly those with few options and great unmet need," said Vasant Narasimhan, Global Head, Drug Development and Chief Medical Officer, Novartis. "Eltrombopag is the  only thrombopoietin receptor agonist to be used in the second-line treatment of SAA, and these results from the NIH study now show its potential as a first-line treatment, which we look forward to discussing with health authorities."

The study also looked at clonal evolution, which is a major complication of SAA (with potential for development of myelodysplastic syndrome and acute myeloid leukemia)1. As of May 25, 2016, the addition of eltrombopag did not increase the rate of clonal evolution and was not higher compared to historical data1,5,6,7. Clonal cytogenetic evolution occurred in 7 patients at 2 years (95% CI, 1-14%)1.

The safety profile was consistent with the known safety profile of eltrombopag. Eltrombopag was briefly discontinued during the first two weeks in 7 patients who experienced transient liver enzyme elevations. Two severe adverse events, grade 2-3 cutaneous eruptions, were attributed to eltrombopag and required discontinuation of the drug. Adverse events not attributed to eltrombopag were due to neutropenic infections and known toxicities from immunosuppressive therapy7. One death occurred on study in a non-responding patient with thymoma three months following treatment, due to paraneoplastic encephalopathy1.

NIH Study Design
The Phase I-II, non-randomized study is being conducted by the National Heart, Lung and Blood Institute through a Cooperative Research and Development Agreement (CRADA) with Novartis Pharmaceuticals Corporation. The primary analysis included 92 patients with treatment-naïve severe aplastic anemia in three treatment cohorts, and nearly 80% of patients were over the age of 18. Eltrombopag was administered at 150 mg daily for patients 12 years or older, 75 mg daily for those 6 to 11 years, and 2.5 mg/kg/day for children 2 to 5 years. Duration of treatment with eltrombopag varied per cohort (cohort 1: day 14 to six months; cohort 2: day 14 to three months; cohort 3: day one to six months). ATG and cyclosporine were administered as standard immunosuppression therapy1.

The study's primary efficacy endpoint was hematologic complete response at six months defined by absolute neutrophil count =1,000/µl, hemoglobin =10 gm/dL, and platelets =100,000/µl. Secondary endpoints included partial and overall hematologic responses at three months, six months, and yearly; survival; self-reported health outcomes; relapse, paroxysmal nocturnal hemoglobinuria (PNH) and clonal evolution as defined by a new clonal cytogenetic abnormality or characteristic dysplastic or leukemic changes in marrow consistent with myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML)1.

About Eltrombopag
Eltrombopag, marketed as Promacta® in the US 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, in over 45 countries worldwide for the treatment of patients with severe aplastic anemia (SAA) who are refractory to other treatments, and 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 United States 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, immunoglobulins, or splenectomy.

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).

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®.

The foregoing release contains forward-looking statements that can be identified by words such as "will," "committed," "over the long term," "potential," "look forward," or similar terms, or by express or implied discussions regarding potential new indications or labeling for eltrombopag, or regarding potential future revenues from eltrombopag. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that eltrombopag will be submitted or approved for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that eltrombopag will be commercially successful in the future. In particular, management's expectations regarding eltrombopag could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing pressures; safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Located in East Hanover, NJ Novartis Pharmaceuticals Corporation is an affiliate of Novartis which 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 118,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit

Novartis is on Twitter. Sign up to follow @Novartis at and @NovartisCancer at
For Novartis multimedia content, please visit 
For questions about the site or required registration, please contact


  1. Townsley, D et al. Eltrombopag added to standard immunosuppression for aplastic anemia. The New England Journal of Medicine. 2017;376(16):1540-1550.
  2. Aplastic Anemia. U.S. National Institutes of Health website. U.S. National Institutes of Health. Web. 16 Feb 2017.
  3. Townsley DM, Desmond R, Dunbar CE, et al. Pathophysiology and management of thrombocytopenia in bone marrow failure: possible clinical applications of TPO receptor agonists in aplastic anemia and myelodysplastic syndromes. Int J Hematology. 2013;98(1):48-55.
  4. Your Guide to Understanding Aplastic Anemia. Aplastic Anemia & MDS International Foundation. Web. 17 Feb 2017.
  5. Kulasekararaj AG et al. Somatic mutations identify a subgroup of aplastic anemia patients who progress to myelodysplastic syndrome. Blood. 2014.124:2698-2704.
  6. Scheinberg P, Young NS. How I treat acquired aplastic anemia. Blood. 2012;120:1185-96.
  7. Townsley, D et al. Myeloid neoplasm gene somatic mutations in patients with severe aplastic anemia treated with eltrombopag and standard immunosuppression. Blood. 2016;128:727.

Novartis Media Relations
Central media line: +41 61 324 2200

To view the original version on PR Newswire, visit:

SOURCE Novartis
Copyright©2017 PR Newswire.
All rights reserved

Related medicine technology :

1. Abide Therapeutics Announces Dosing of First Patient In Phase 1b Study of ABX-1431 in Tourette Syndrome and Collaboration with Tourette Association of America
2. CASI Pharmaceuticals Reports On Enrollment Status Of ENMD-2076 Phase 2 Study In Triple-Negative Breast Cancer
3. Colitis Study Shows Curcumin More Powerful When Combined with Turmeric Essential Oil
4. Kaiser Permanente Study Tests New Way to Reduce Vaccine Hesitancy
5. Medivir Announces Positive Data From the Phase II Study of Remetinostat in Patients With Early-stage Cutaneous T-cell Lymphoma
6. MGB Biopharma Closes $1 million Financing Round to Finance Manufacturing of MGB-BP-3 for Phase II Study in Clostridium difficile-Associated Disease (CDAD)
7. HLT Announces First Patient Enrollments in a Feasibility Study of a New Transcatheter Aortic Valve Replacement System
8. Retrospective Study Confirms Bovine Carotid Artery Biologic Graft Outperforms Expanded Polytetrafluoroethylene for Hemodialysis Access
9. Akcea Initiates Phase 2 Study of AKCEA-APO(a)-L Rx in Patients with High Lp(a) and Cardiovascular Disease
10. Center Point Clinical Services Studies Show That Pharmacist-led Interventions Increase Clinical Trial Patient Medication Adherence and Compliance and Provide Robust Study Data
11. Study Finds Surfacide UV-C Light Effective as an Adjunct to Infection Control in a Pediatric Burn Hospital
Post Your Comments:
(Date:9/27/2017)... MIAMI , Sept. 27, 2017  Commended for their ... recent notable awards. Ranked as number one in the South ... ninth time in Inc. 5000 yearly list, the national specialty ... CEO, Armando Bardisa will soon be honored by ... Set to receive his award ...
(Date:9/23/2017)... Pa. , Sept. 22, 2017 Janssen ... a complete response letter from the U.S. Food and ... seeking approval of sirukumab for the treatment of moderately ... letter indicates additional clinical data are needed to further ... moderately to severely active RA. ...
(Date:9/19/2017)... Ky. , Sept. 19, 2017   ZirMed Inc ... predictive analytics, today announced that it has been ranked #1 ... Black Book™ Rankings 2017 User Survey. ZirMed was ... solution for large hospitals and medical centers over 200 beds ... Black Book,s healthcare technology user survey history. ...
Breaking Medicine Technology:
(Date:10/13/2017)... ... October 13, 2017 , ... The ... to promote standards of excellence for the field of eating disorders, announces the ... – 25, 2018 in Orlando, Florida at the Omni Resort at ChampionsGate. ...
(Date:10/13/2017)... ... October 13, 2017 , ... ... ‘17, and Jennifer Huggins, PharmD ’17, along with clinical associate professor Janice ... of cardiovascular diseases during the 15th Annual Women’s Health Conference. The SIU ...
(Date:10/13/2017)... , ... October 13, 2017 , ... Apple Rehab ... services, staged a mock evacuation of the facility as part of a disaster drill ... Department, Echo Hose EMS and Shelton City Emergency Manager, as well as the ...
(Date:10/13/2017)... ... 13, 2017 , ... The Visiting Nurse Association (VNA) of Somerset Hills is ... vendors and unique items from across the nation, this holiday-themed event will raise funds ... by the VNA. The boutique will be open Saturday, November 4 (10:00 a.m. ...
(Date:10/13/2017)... ... ... Global Healthcare Management’s 4th Annual Kids Fun Run brought out many kids this ... by Global Healthcare Management’s CEO, Jon Letko, is aimed at getting kids excited about ... ages; it is a non-competitive, non-timed event, which is all about having fun and ...
Breaking Medicine News(10 mins):