PDSA E-News: September 26, 2016




Bleeding Disorder Drug Fostamatinib Clears First of Two Key Trials

Rigel logo Pharmaceutical company, Rigel announced positive results from the first of two studies in the FIT 1 Phase 3 program for fostamatinib in chronic ITP. Fostamatinib is being studied to treat chronic immune thrombocytopenia (ITP) a platelet disorder in which the immune system attacks and destroys the body's own blood platelets. Fostamatinib is an oral inhibitor of Spleen Tyrosine Kinase (SYK), which is a key player in the platelet destruction that occurs in ITP. By uniquely addressing the underlying autoimmune cause of ITP fostamatinib may inhibit platelet destruction. Dr. James Bussel, of Cornell University and who serves on PDSA’s Medical Advisors Board, was Principal Investigator on the study.

About 18 percent of the 76 adult chronic ITP patients who were treated with fostamatinib achieved a stable platelet response (defined as a platelet count of 50,000 or higher in at least four of the six visits between weeks 14 and 24) in the study, compared with none on placebo. Patients typically saw increases in platelet counts to a level above 50,000/µL within the initial weeks of treatment. There were no new or unexpected safety signals, compared to previous experience with the drug. More patients experienced drug-related adverse events in the fostamatinib group compared to the placebo group, but frequency of serious adverse events was similar between both groups. Rigel said they are encouraged by these data that demonstrate potential benefit of fostamatinib for chronic ITP patients who need new treatment options.

Barber, J. “Rigel's fostamatinib meets primary endpoint of Phase III immune thrombocytopenia trial.” FirstWord Pharma, Aug. 30, 2016.


Health Canada Issues New Safety Alert for Revolade®

Revolade logoRecent findings from Canada showed the ITP treatment Revolade® (eltrombopag), in some patients, may cause serious damage to the liver and might lead to more serious illness.  Health Canada issued an alert for eltrombopag (Revolade® in Canada/called Promacta® in the US) on August 25, 2016.  The alert announced that Revolade® was reported to have caused severe liver toxicity in five Canadian patients, including two chronic ITP patients, in clinical trials and post-marketing. Fortunately, in all five cases the liver abnormalities resolved following discontinuation of Revolade®.  Patients with liver problems are encouraged to talk with their doctor before taking Revolade®, as this can increase the risk of liver injury.

The new information about Revolade® was issued to Canadian healthcare professionals (hematologists, pharmacists); pharmacy, nursing, and medical associations; hospitals, and patient groups.  The alert stated that elevation of liver laboratory values typically occurred within the first three (3) months of initiation of treatment.  The alert recommended that healthcare professionals should measure serum alanine aminotransferase (ALT), aspartate aminotransferase (AST), and bilirubin before starting patients on Revolade® treatment; then every two weeks during the dose adjustment period, and then monthly following establishment of a stable dose.  They stated Revolade® should be discontinued if ALT levels increase greater than or equal to 3X the upper limit of normal (ULN) in patients who have normal liver function; or increase greater than 3X baseline or 5X the ULN in patients who already had elevation  of transaminases prior to starting treatment.  Health Canada, in collaboration with Novartis Pharmaceuticals Canada Inc. has updated the Canadian Product Monograph for Revolade® to reflect this new safety information.  Health Canada is communicating this new information to healthcare professionals using the Recalls and Safety Alerts Database, which is on the Healthy Canadians Web site.

“REVOLADE (eltrombopag) – Risk of Severe Hepatotoxicity.”  Recalls & alerts - Healthy Canadians, Government of Canada, August 25, 2016.


FDA Recommends all US Blood Donations be Screened for Zika Virus

mosquitoWith the rapid spread of Zika virus (ZIKV) from Brazil and other regions in South America to cases recently found in the United States, researchers, physicians, and public health officials alike are scrambling to prevent future transmission of the virus from occurring.  As a result, the US Food and Drug Administration (FDA) has mandated that all US blood donations should be screened for the Zika virus, even in states where an active virus has not yet been discovered.  Using nucleic acid analysis, donations can easily be tested for the infection prior to blood transfusion.  Alternatively, blood banks can decontaminate blood and plasma through pathogen reduction technology.  These scientifically based approaches are significantly safer and more accurate than current blood bank guidelines to exclude donors who may have been exposed to certain viruses.  Currently they simply ask via questionnaire where donors have vacationed or whether they have been exposed to specific environmental factors.

Of particular interest to ITP patients who receive IVIg, the Plasma Protein Therapeutics Association (PPTA) released a statement earlier in September, indicating that because of PPTA’s effective process of producing IVIg from donated plasma (a process which extensively kills any viral products), it is not necessary for PPTA to defer donation of blood plasma, mitigating the risk of contracting ZIKV from manufactured IVIg.  Across the United States each year 14 million units of blood are donated from 7 million unique donors.  According to the New York Times, “every day, as many as 36,000 units of red blood cells are given to patients, along with 7,000 units of platelets and 10,000 units of plasma.  Consistent screening of the blood supply is an enormous task.”  However, the US Centers for Disease Control (CDC) has confirmed over 11,500 cases of ZIKV in the United States thus far, demonstrating a crucial necessity for blood banks to increase oversight and vigilance in testing for potential infections.

Plasma Protein Therapeutics Association. “Zika Virus and Plasma Protein Therapies.”  Plasma Protein Therapeutics Association Web site. 9/8/2016.

Saint Louis, Catherine. “All Donated Blood in U.S. Should Be Tested for Zika, F.D.A. Says” The New York Times. 8/26/2016.



Unscreened Blood Donation Lead to Zika-infected Platelet Transfusions

blood donationA New England Journal of Medicine (NEJM) case study reported that transmission of the Zika virus (ZIKV) is possible through platelet donation.  Earlier this year, a 54-year-old woman with myelofibrosis and a 14-year-old girl with acute myeloid leukemia received platelet transfusions from one blood donor who was pre-symptomatic at the time of blood donation.  After the recipients had received their transfusions, the donor contacted the blood bank immediately to report a rash, headache, and joint pain, which are some common symptoms of ZIKV.  Viral infection was confirmed by serological analysis.

The donor and both transfusion recipients live in Brazil, where the Zika virus has had the greatest incidence and prevalence rate, as well as the most devastating impact.  Thankfully, the women infected did not experience any symptoms related to ZIKV infection; however, the cases reported demonstrate evidence for ZIKV transmission through platelet transfusion.

As ITP patients receive platelet transfusions when their platelet counts are low, this reported finding is of particular concern to the ITP community.  Moving forward, guidelines for blood screening by the US Food and Drug Administration (FDA) and the US Centers for Disease Control (CDC) plan to decrease the risk of similar infection occurring in the US.

Motta, Lara J.F.  “Evidence for Transmission of Zika Virus by Platelet Transfusion.” The New England Journal of Medicine. 8/17/2016.



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