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PDSA E-News: July 26, 2017




Promising New Approach to Treating ITP with ARGX-113

ARGX-113There is a new approach to treating ITP that holds promise. A biotechnology company in the Netherlands and Belgium, argenx, has developed a new treatment, ARGX-113, that claims to remove recycled IgG antibodies from circulation. ARGX-113 is the Fc-portion of an antibody. It is designed to block recycling of the IgG antibodies, which results in their removal from circulation. When these particular antibodies are removed, more platelets should remain in the blood stream.

The company has completed a Phase I clinical trial for ARGX-113 and has begun enrolling patients in the Phase II trial. The double-blind, placebo-controlled Phase II trial will enroll about 36 ITP patients who have platelet counts under 30,000. The patients will receive dosing with ARGX-113 in addition to their current standard of care, steroids, immunomodulatory, or TPO treatments. Primary trial endpoints will be safety and tolerability. Secondary endpoints will be the effect on platelet count and use of rescue treatment. Development work on ARGX-113 was done in cooperation with researchers at the University of Texas and Texas A & M University. The initiation of the Phase II trial is considered an important milestone for the company. CMO Nicolas Leupin, said, “We believe ARGX-113 could be a breakthrough therapy in an indication like ITP where current therapies do not adequately address symptoms or achieve remissions.”

“argenx Announces First Patient Dosed in Phase II Proof-of-Concept Study of ARGX-113 for the Treatment of Primary Immune Thrombocytopenia.” Press release from argenx, March 31, 2017.


Treatment with TPO Successful for ITP Patient with Heart Condition


It is not uncommon for patients with Chronic Lymphocytic Leukemia (CLL), a type of cancer that affects white blood cells, to develop low blood cell numbers from autoimmune issues, especially ITP. A recent case study published by two physicians in Madrid detailed the successful treatment course for a patient with ITP incident to CLL and with congestive heart failure. Blood thinners were initially suggested to resolve the cardiovascular issues and to reduce the risk of a blood clot, but was not possible because of the patient’s low platelet count from ITP.

To raise the platelet count, the patient was administered chemotherapy (Bendamustine) and rituximab, prednisone, and IVIg, but was unresponsive to all these. The patient was then treated with 50 mg of the TPO Promacta® daily for a year. This resulted in a platelet increase to 120,000, which permitted the use of anticoagulant therapy for the patient’s heart. The patient maintained a platelet count of 80,000 after one year of treatment with Promacta. Although this patient’s chief health problem was leukemia, this case illustrates that TPO agents can be safely given to ITP patients who have vascular issues for extended periods of time, both elevating platelet counts and protecting their cardiovascular and immune systems.

Garbutt, T. “Long-Term Use of TPO Receptor Agonist Safe in CLL-Associated Immune Thrombocytopenia.” Oncology Nurse Advisor, 6/28/2017.





Cost of Care: How Much Does an Emergency Hospital Visit Cost for ITP Patients?

hospital emergency sign

Treating bleeding events in the US can be expensive for both insurers and patients. Researchers from Amgen, Inc., a pharmaceutical company based in Thousand Oaks, CA and the manufacturer of the ITP treatment Nplate®, wanted to know what the cost of treating bleeding-related episodes (BREs) is for ITP patients in the US. The study used Medicare benefits and commercial claims databases to approximate the cost of these events, based on a combination of out-of-pocket costs for patients and reimbursement from insurance.

Out of the 6,551 patients studied, 14,115 bleeding events were identified. These patients were on average 55 years old and 62% were women. The amount for each bleeding event was $6,022, where an episode cost an average of $45,114 if admitted to the hospital and $2,150 if treated as an outpatient. Costs were slightly higher when patients had undergone splenectomy. On average, the cost of a hospital visit for a bleeding event was lower when just rescue therapy was required ($2,787) in comparison to treatment of bleeding alone ($10,396). Cost for BREs that included both bleeding and rescue therapy was $11,065. It is important to emphasize that these costs reflect the total reimbursement cost to physicians, and that an ITP patient with insurance should not be paying these high costs out-of-pocket to their doctor’s office.
Lin J, et al. “Cost of Bleeding-related Episodes in Adult Patients with Primary Immune Thrombocytopenia: A Population-based Retrospective Cohort Study of

Administrative Claims Data for Commercial Payers in the United States.” Clinical Therapeutics, March 2017.





During a Health Crisis Having a Positive Outlook Improves Health


According to recent research studies, positive thinking and maintaining an optimistic outlook can do more than raise your spirits. In fact, they may improve your health and lead to a longer life. Research shows that what happens in the brain influences what occurs in the body. In fact, during a health crisis, developing positive emotions can boost the immune system and decrease depression. An internist who survived 15 years with cancer offered a number of ways to set the stage for happiness including: surrounding yourself with people who help lift your spirit, doing something nice for someone else, keeping a daily gratitude journal, and watching uplifting and funny movies.

New research shows that people can learn skills to help them experience more positive emotions when they are faced with severe, life-threatening illness. These skills include: savoring an event and logging it in a journal, starting a daily gratitude journal, listing your personal strengths, setting an attainable goal and noting your progress, reporting a fairly minor stress and listing ways to reevaluate the event in a positive way, noticing and practicing small daily acts of kindness, and practicing mindfulness by focusing on here and now rather than the past or future. In a study with 159 patients, those who were trained in the eight skills and encouraged to use at least three of them were found to hold higher levels of positive feelings and fewer negative thoughts connected with their illness. It also improved patients’ quality of life, fostered adherence to taking medications, lead to healthy behaviors, and helped build increased social support.

Jane E. Brody, “A Positive Outlook May be Good for Your Health.” Personal Health, The New York Times, July 3, 2017.





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