New Publication Answers Questions About the SIPPET Study

Inhibitors are the most troubling complication of hemophilia A treatment today. In this week’s blog, I share with you a new publication concerning the SIPPET study, which sheds light on factor VIII products and inhibitors.
New Publication Answers Questions About the SIPPET Study


An article written by noted hematologist Dr. Flora Peyvandi and colleagues was recently published in the medical journal Haemophilia. This article directly addresses and answers several questions that were raised about the 2016 publication of the Survey of Inhibitors in Plasma-Product Exposed Toddlers, or SIPPET study.1

With this new article, SIPPET: methodology, analysis and generalizability, the authors respond to the 17 most common questions associated with the design, methodology, and results of the SIPPET study, including1:
  • Is the inhibitor risk higher in SIPPET than in previous reports?
  • Could differences in treatments between countries have affected the results?
  • Could the results have been affected by the way the study was randomized?
  • Do the SIPPET results also apply to other recombinant factor VIII (rFVIII) products
    beyond the 1st and 2nd generation products used in the study?
  • Is there a difference in inhibitor risk between the different brands within the plasmaderived and recombinant groups?
Original SIPPET Study
The SIPPET study, conducted by Dr. Peyvandi and colleagues, was the first randomized trial to compare the incidence of inhibitors in plasma-derived factor VIII (pdFVIII/VWF) products and rFVIII products in previously untreated patients (PUPs).2

Results from this landmark study showed that there was an 87% higher rate of inhibitor development in patients who received rFVIII compared with patients who received pdFVIII containing von Willebrand factor (VWF).2

Based on the results of the SIPPET study, the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) now recommends that pdFVIII/VWF be considered as one of several treatment options in PUPs.3
Visit www.inhibitorinfo.com to Learn More About Inhibitors
Inhibitors are the most serious and challenging hemophilia A treatment complications. All patients with hemophilia A are at risk for developing inhibitors, regardless of age and disease severity.
Inhibitorinfo.com is a comprehensive website that provides important information and resources about inhibitors and the risk of inhibitors. There is a discussion guide patients can download and use to talk with their hematologists about inhibitors. Visitors can also read about the results of the SIPPET study and watch leading hematologists talk about its implications.
When visitors sign up for updates at inhibitorinfo.com, they will receive access to the full SIPPET study, as well as updates about hemophilia, inhibitors, and the latest clinical data.
References: 1. Peyvandi F, Mannucci PM, Palla R, Rosendaal FR. SIPPET: methodology, analysis
and generalizability [published online ahead of print March 17, 2017]. Haemophilia. doi:
10.1111/hae.13203. 2. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII
and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374(21):2054-2064. 3. National
Hemophilia Foundation. MASAC Update on SIPPET. National Hemophilia Foundation website.
https://www.hemophilia.org/Newsroom/NHF-Community-News/MASAC-Update-on-SIPPET.
Published March 9, 2016. Accessed April 27, 2017.
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BN/A8/0517/0275

Plasma Awareness Week

This week marks the first ever International Plasma Awareness Week, an opportunity to recognzie the importance of plasma in our lives, and the need to donate blood to provide the life-saving liquid to those in need. It’s especially important to many with hemophilia throughout the world as donated blood donations can be fractionated into factor VIII and IX for many hemophilia patients who use plasma therapy. 

You learned this in high school science class but here it is again: Plasma is the clear, straw-colored liquid portion of blood that remains after red blood cells, white blood cells, platelets and other cellular components are removed. It is the single largest component of human blood, comprising about 55 percent, and contains water, salts, enzymes, antibodies and other proteins. It is a clear, straw colored liquid that is 90% water and serves as a transporting medium for cells and a variety of substances vital to the human body.
In the US, we often think only of recombinant factor when we think about treating hemophilia, but plasma therapies remain the mainstay for many with hemophilia, those with inhibitors and undergoing ITI, and those with von Willebrand disease patients. 
In addition, plasma protein therapies are used to in emergency and surgical medicine.Plasma protein therapies are not interchangeable and have been defined by regulators as sole-source biologic products because no generics or substitutions exist. In addition, their biological nature demands storage and handling requirements by specialty distributors that ensure their safety.

Source plasma is plasma that is collected exclusively for further manufacturing through a process called plasmapherisis. Recovered plasma is collected through whole blood donation which has been separated into its cellular components.

Safety and quality of plasma protein therapies is the top priority of the plasma protein therapeutics industry. Both collectors and manufacturers adhere to strict regulatory policies and have instituted Good Manufacturing Practices in every step of plasma collection and manufacturing processes.

Plasma-derived therapies depend on the generosity and commitment of healthy donors. Source plasma is collected in over 450 specialized donor centers in the U.S., Canada, Germany, Austria and the Czech Republic. Source plasma collection in the U.S. is regulated by the Food and Drug Administration and by the European Medicines Agency and national regulatory authorities in Europe. Additionally, 436 plasma collection centers are also certified by the International Quality Plasma Program (IQPP), a rigorous, voluntary program that goes beyond regulatory requirements to help ensure donor safety and further improve the quality of plasma used to manufacture therapies.
This is a good week to doante blood then! If you can make the time, please visit your local blood donation center and give the gift of life. 
See www.donatingplasma.org and www.pptaglobal.org  for more information.


International Plasma Awareness Week is sponsored by the Plasma Protein Therapeutics Association and its member companies.

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