Treating Your Hemophilia A and von Willebrand Disease With ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human])

 

This is a paid public announcement from Grifols and does not constitute an endorsement of products or services. When you click on the links in this blog entry, you will be directed to the Grifols website. LA Kelley Communications always advises you to be a savvy consumer when contacting any company; do not reveal identifying information against your will.

 

Treating Your Hemophilia A and von Willebrand Disease With ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) A diagnosis of hemophilia A can be an overwhelming experience, not only for patients, but for their families as well. The best way to combat the feeling of helplessness that often follows a diagnosis is by becoming empowered with information. Grifols, the manufacturer of ALPHANATE—the #1 prescribed plasma-derived factor VIII/von Willebrand factor treatment—has just launched a new patient website. ALPHANATE.com provides experienced and newly diagnosed patients and their caregivers with an understanding of hemophilia A and von Willebrand disease (VWD) along with tools for living with these bleeding disorders.

Living With Hemophilia A
                                                      ALPHANATE.com answers some of the most common hemophilia A questions in one convenient location. On the new site, patients and caregivers can learn exactly what hemophilia A is; why a plasma-derived FVIII could be a treatment option; and details about inhibitors, the greatest challenge in hemophilia A treatment.1,2 ALPHANATE.com also outlines how patients can identify, manage, and prevent bleeds. In other words, ALPHANATE.com is not just about providing patients and caregivers with information, it’s about helping people with hemophilia A or VWD.

 
Patient
Support & Resources
All patients should have access to proper medications, resources, and strong support. With this commitment to care in mind, Grifols offers Factors for Health, a comprehensive patient support and assistance program. The Factors for Health program offers:
  • $0 Copay Assistance Program, which may cover out-of-pocket expenses not
    covered or partially covered by insurance
  • Free Trial Program, for eligible patients new to ALPHANATE
  • Benefits investigation and support services
  • Patient Assistance Program
  • Care Coordination, helping patients gain access to and remain on
    ALPHANATE
Other resources on thenew ALPHANATE.com include a Doctor Discussion Guide to prepare you to get the most out of your conversations with your doctor and a blog with tips for living with and managing a bleeding disorder.
 
ALPHANATE
ALPHANATE is an FDA-approved human plasma-derived treatment for adult and pediatric patients with hemophilia A or VWD. The manufacturing process for ALPHANATE is specifically designed to preserve FVIII and VWF together, the way they are naturally found in the body.3
 
Since product launch in 1997, there has never been a confirmed case of virus or prion transmission with ALPHANATE. Grifols is committed to safety before, during, and after ALPHANATE manufacturing. In fact, ALPHANATE is manufactured using a 10-steps-to-safety process, with full traceability from donor to patient.
 
Because ALPHANATE is made from human plasma, it may carry a risk of transmitting infectious agents, eg, viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, despite
steps designed to reduce this risk.
To meet the needs of a variety of patients, ALPHANATE is offered in a range of convenient vial sizes with low diluent volume.3
Visit Plasma City on the new ALPHANATE.com to see how others with hemophilia A and VWD have been building foundations for success with ALPHANATE for 20 years. And don’t forget to sign up to stay up to date on what’s happening in Plasma City.
 
 
 
Indications

ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) is indicated for:

  • Control and prevention of bleeding episodes and perioperative management in adult and
    pediatric patients with factor VIII (FVIII) deficiency due to hemophilia A
  • Surgical and/or invasive procedures in adult and pediatric patients with von Willebrand
    disease (VWD) in whom desmopressin (DDAVP) is either ineffective or
    contraindicated. It is not indicated for patients with severe VWD (type 3)
    undergoing major surgery
 
Important Safety Information
 
ALPHANATE is contraindicated in patients who have manifested life-threatening immediate hypersensitivity
reactions, including anaphylaxis, to the product or its components. Anaphylaxis and severe hypersensitivity reactions are possible with ALPHANATE. Discontinue use of ALPHANATE if hypersensitivity symptoms occur, and initiate appropriate
treatment. Development of procoagulant activity-neutralizing antibodies (inhibitors) has been detected in patients
receiving FVIII-containing products. Carefully monitor patients treated with AHF products for the development of FVIII inhibitors by appropriate clinical observations and laboratory tests.
 
Thromboembolic events have been reported with AHF/VWF complex (human) in VWD patients, especially in the
setting of known risk factors. Intravascular hemolysis may occur with infusion of large doses of AHF/VWF complex (human).
 
Rapid administration of a FVIII concentrate may result in vasomotor reactions.
 
Because ALPHANATE is made from human plasma, it may carry a risk of transmitting infectious agents, eg, viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent, and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent, despite steps
designed to reduce this risk.
 
Monitor for development of FVIII and VWF inhibitors. Perform appropriate assays to determine if FVIII and/or VWF inhibitor(s) are present if bleeding is not controlled with expected dose of ALPHANATE.
 
The most frequent adverse drug reactions reported with ALPHANATE in >1% of infusions were pruritus, headache, back
pain, paresthesia, respiratory distress, facial edema, pain, rash, and chills.
 
 
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
 
References: 1. Kempton CL, White GCII. Blood. 2009;113(1):11-17.  2. Leissinger CA. Am J Hematol. 2004;77:187-193. 3. ALPHANATE® (antihemophilic factor/von Willebrand factor complex [human]) Prescribing Information. Grifols
 
BN/A8/0717/0277

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.
This is a paid public announcement from Grifols and does not constitute an endorsement of
products or services. When you click on the links in this blog entry, you will be directed to
the Grifols website. LA Kelley Communications always advises you to be a savvy consumer
when contacting any company; do not reveal identifying information against your will.
BN/A8/0517/0275

Name Game, Game Change?

I’m thinking “game” this weekend because the Patriots are in the Super Bowl, again, and I will need to watch it like the rest of Boston. I only watched my first football game two years ago when, surprise surprise, the Patriots were in the Super Bowl. Every sports fan knows the Patriots, I am told. It’s easy to cheer for your home teams when the names (Red Sox, Patriots, Bruins, Celtics) endure for decades. It’s harder when it’s the drug companies that make your factor concentrate. There’s a lot of game changing going on lately.

The news was announced just three days ago: Biogen, maker of Eloctate and Alprolix, has spun off its hemophilia division, which has now become an entirely separate and new company called Bioverativ.

Biogen’s two revolutionary products, the first ones with a longer half-life, were game changers. Now there are a few more choices for longer half-life products, but these were the first and were rolled out with much fanfare. Then boom! Three years later, Biogen doesn’t want them anymore.

This is just a reminder to us patients that this is a marketplace, and we are consumers. And companies need to make business decisions—hence the “game.” As consumers, it’s up to us to understand how the game is played, and who are the players. The names have been changing, more rapidly than I can keep up with in the specialty pharmacy arena, and those are truly game changers.

In the factor concentrate manufacturing arena, we had two name changes just in the last few months: Biogen to Bioverativ, and Baxalta to Shire. But this has been happening for years in our community, so here’s a review. It’s worth knowing the players—of which YOU, the consumer, are the most important!

Remember Alpha? Those of you who use Alphanate or Alphanine may wonder why these drugs are called that when sold by the Spanish company Grifols. Simple: Grifols bought Alpha Therapeutics hemophilia therapies years ago and simply kept the drugs’ names the same. (It’s hard to change a drug’s name.) In 2011, Grifols also bought Talecris, making it the third-largest global manufacturer of plasma-derived therapies. Oh, and Talecris? It was a spin-off from Bayer, which didn’t want to keep plasma-therapies anymore. Bayer’s plasma-product Koate-DVI went to Talecris, and Bayer kept Kogenate FS. If you look at the Koate-DVI packaging, you’ll still see the Bayer primary color line around the box!

Baxter Healthcare produced factor concentrates like Recombinate, Advate, Hemofil M and FEIBA. It spun off its hemophilia division, which became an independent company called Baxalta. That didn’t last long. Shire, an Irish pharmaceutical company, liked what it saw and scooped it up. All the former Baxter/Baxalta products now belong to Shire.

The biggest name changer is CSL Behring. I knew it in 1987 as Armour Pharmaceutical. Then in 1996, Armour and Behringwerke (a Geman company) formed a joint venture known as Centeon. Things happen fast: in 1999, Centeon became Aventis Behring. Why? Armour’s parent companies (Rhone-Polenc Rorer and Hoechst) merged to become Aventis. Meanwhile, CSL (an Australian plasma therapies manufacturer) acquired ZLB Blood Transfusion Services. In 2004, CSL acquired Aventis Behring, to form ZLB Behring, later called CSL Behring.

(There’s a comprehensive timeline of this interesting company here.)

Genetics Institute: anyone remember that? They developed BeneFIX and ReFacto (no longer on the market). It evolved into Wyeth, and then was bought by pharma giant Pfizer Inc.

Bayer is one that seems to have stayed the same, but it’s had name changes too. Bayer bought Cutter Labs in 1978 and Miles Labs in 1979. In 1995, they all became Bayer. I think Novo Nordisk (Denmark) has stayed the same… so far!
And some companies dropped out altogether, like the American Red Cross.  And new ones entered, like Octapharma (Switzerland) and Kedrion (Italy), tapped to distribute Koate DVI for Grifols. And Aptevo Therapeutics… oh, which was owned by Cangene (Canada) first, then Cangene was bought by Emergent Biosolutions in 2014! And they all originated from Inspirational Biopharmaceuticals, which in 2013 sold all its product rights to them, and dropped out of the game.

More passes than Tom Brady!

Whew! It’s amazing tracking the history of just the name changes: but we also need to know products. We are tracking who makes what on our website Hemophilia Factor Chart by Brand, available as a download. We are updating it all the time… a necessity to keep track of this ever-changing game, and business.

Share Your Story!

“After nourishment, shelter and companionship, stories are the thing we need most in the world.” 
As a writer, I tend to agree! Whether we are imparting facts or weaving a tale, we seem to learn best when we hear it all as a story. Stories are especially important for families dealing with bleeding disorders. We all want to hear the Introduction (Beginning), the Conflict and the Climax (Middle), and the Outcome (End). And we want to hear lots of stories because our own story will mirror one someday.
Grifols, manufacturers of plasma-derived clotting factors, is offering a chance for you to tell your story, to help other families who have hemophilia or von Willebrand Disease. 
Visit www.mybleedingdisorder.com to share your experience and provide inspiration and motivation to others seeking ways to better manage their bleeding disorder.
Your story may be featured on the newly launched consumer website , and may also be selected to be shown in the Grifols booth at the National Hemophilia Foundation conference in Dallas, Texas in August!
As I always advise, please read the privacy notice on the site before sharing.
This is a paid public awareness notice from Griflols.

Great Book I Just Read

Anthem by Ayn Rand [Kindle]

If you want to know what the legendary Ayn Rand, who grew up in Stalinist Russia, believes in a nutshell, read this novella. In this futuristic story, people’s names are replaced by genres and numbers (Liberty 5-3000), humanity has all individuality stripped away, and a collective society emerges. The word “I” is banished, even when referring to oneself. The narrator knows he is different but is not sure how or why. He knows he is not meant to be a street sweeper, as the Elders dictate to him. He has no parents, no family–no one does. His life is controlled by Elders from beginning to the day he dies. He refers to himself as “we,” and is raised to serve the common needs of all. Until one day he discovers an underground place where electricity works–apparently a subway or city from before the end of days. Excited, he shows the Elders but is shunned and shamed. He decides to return to the place, try to make sense of what has happened to mankind and why such technology is being ignored, to improve the lot of the common worker. In the process, he discovers himself, love, his convictions and a future as an individual. A very short read, with powerful and rich ideas about individualism to think about.  Five/five stars.

Inhibitor Meeting in UK


Grifols, a Spanish based company that manufacturers Alphanate and Alphanine, held a one day educational conference on inhibitors for nurses in London yesterday. I was pleased to start the day with a talk on inhibitors in America: how the main struggle is finding a way to afford the treatment that is already available, given mounting pressure from payers and limited lifetime caps. Professor Alessandro Gringeri, of the University of Milan, gave an excellent comparison of ITT protocols around the world. He reminded us that ITT has been with us for 31 years, but since the first attempts, different protocols have developed. He reviewed the specifics of the Bonn, Malmo and Van Creveld Klinick protocols, and how responses to different ITT are either patient related or treatment related. ITT does not work with all patients, particularly those with mild hemophilia.

Dr. Sylvia von Mackensen is a psychologist at the University Hospital Hamburg-Eppendorf, Hamburg, and gave a presentation on Quality of Life Studies: measurements, outcomes and expectations. This is an important subject for inhibitor patients, who suffer a great loss of QoL when by passing agents or ITT are not always effective.

My colleague Becky Berkowitz, RN from the HTC in Las Vegas, Nevada, spoke about nursing in the US. Becky has a unique position working in an enormous state with a small population, and a lean HTC staff: her efforts to visit the native Americans and advocate for their treatment held the audience’s rapt attention.

The rest of the afternoon held presentations by Dr. Mauricio Alvarez-Reyes of Grifols, nurse Allyson Hague of Manchester Children’s Hospital, nurse Kate Khair of Great Ormond Street Hospital and Vicky Vidler, nurse consultant of Sheffield Children’s Hospital. It was a wonderful event, full of information and diverse speakers.

After the program, I took a tour of Great Ormond Street Hospital with Kate Khair, Virginia Kraus (Grifols), Becky Berkowitz, and Mark McDonnell, UK Manager (Grifols). It’s a major HTC, with over 450 pediatric patients, with a beautiful ward. I had always heard of this famous hospital and it was a pleasure to see it from inside.

This has been productive week: I was also able to visit on Tuesday the Haemophilia Society, which I believe was the first hemophilia society in the world. Chris James is the relatively new executive director, and he graciously allotted me 3 hours to tell me about their programs and population. We also spoke about sharing my company’s free resources with his members. They have excellent programs and in many ways are one step ahead of many other organizations in terms of programs for inhibitor patients.

Last of all, I saw on Monday an historic site: the Broad Street Pump, the epicenter of the devastating 1854 cholera epidemic of London. This important landmark represents the beginning of modern public health. It was here that a contaminated well led to hundreds dying within one week, and caused English physician Dr. John Snow to buck conventional medical thinking, which thought it was miasma–bad air– that caused the outbreak (this was before germ theory). By using a scientific method and putting himself at great risk, Snow interviewed an entire neighborhood, noting where deaths occurred: the outcome was a map which revolved around the pump and well. After a struggle to convince authorities that water was responsible, Snow had the handle removed. The epidemic was over and a new theory on germs was born. This one pump represents all modern day efforts to provide clean drinking water to overcrowded cities worldwide. It was simply amazing to actually see the pump, and be grateful for the dedicated efforts of this remarkable British physician. (Read The Ghost Map for an excellent recounting of this story)

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