Novo Nordisk

Factor XIII Patients Get New Treatment

Laurie Kelley January 13, 2014

This is going to be quite a year with potentially new products.

Here’s one to start the year off right!

The FDA has approved Novo Nordisk’s Tretten® (coagulation factor XIII A-subunit [recombinant]) for routine prophylaxis of bleeding in people with congenital factor XIII (FXIII) A-subunit deficiency. This is one of
the rarest inherited bleeding disorders, with an incidence of 1 in 1 million to 1 in 5 million.

FXIII is composed of two subunits, A and B, with genes on two different chromosomes. FXIII deficiency is usually caused by a deficiency of the A-subunit. In the absence of FXIII, loosely formed clots are developed, leading to bleeding complications similar to those in severe hemophilia A. Tretten is the only recombinant treatment for the disorder

Why does this matter? Patients with congenital FXIII A-subunit deficiency have a lifelong susceptibility to bleeding, including intracranial hemorrhage (spontaneous bleeding into the brain), which could be life-threatening if untreated.

For more info: TRETTEN-US.com

Great Book I Just Read
Rebecca by Daphne du Maurier [Kindle]

Rebecca is a gothic romance/psychological thriller, much along the lines of Jane Eyre, that skillfully transports the reader
into the early 1900s: a young bride (never named) narrates the tale, about coming to the famed estate of Manderley, as the new bride of middle-aged Maxim de Winter, whose first wife drowned in a mysterious boating accident only a year ago. Rich in character, thought and detail, transfixed on the lush countryside, landscaped grounds and endless ocean as the story progresses. The mansion seems haunted by the first wife—Rebecca—and every act, every word by everyone, including Max, seems to scream how missed the charming and stunning Rebecca is, how hopelessly inadequate the unsophisticated, young new bride is, until the bride considers ending her life. But then …Things are not at all what they appear. Through subtle hints and plot twists, the real story emerges bit by bit to an amazing and gripping ending. The Hitchcock movie by the same name is also fantastic. Four/five stars.

HERO: One who is revered for sacrifice, skills, dedication…

Laurie Kelley December 16, 2013

I’m in Italy this week on some business, where I am surrounded by Italian heroes, both mythical and real. While here I received notice of some videos you might like to see. These are the latest videos filmed at the US Hemophilia Experiences, Results, and Opportunities (HERO) Summit for Solutions, of which I took part. Some of our top opinion leaders from our community were invited together by Novo Nordisk to brainstorm ways to implement the findings of the HERO study, supported by Novo Nordisk, the largest psychosocial study on hemophilia ever done.Please feel free to view!

The HERO Initiative: Improving Understanding of the Burden of Hemophilia on Families focuses on our discussion in Workshop 2 around how hemophilia can affect the entire family in different ways, including emotionally, financially, and professionally. This video includes the following US HERO Summit participants:

·        Mary-Jane Frey, Nurse Coordinator, Children’s Hospital of Michigan
·        Chris Guelcher, Nurse Practitioner, Children’s National Medical Center
·        Dana Kuhn, PhD, President and Founder, Patient Services, Inc.
·        Dawn Rotellini, Director of Chapter Development and Training, National Hemophilia Foundation
·        Michelle Witkop, Bleeding Disorders Nurse Practitioner, Northern Regional Bleeding Disorder Center

The HERO Initiative: Working to Understand and Improve Pain Management in Hemophilia focuses on our discussion in Workshop 3 around the impact of pain on the daily lives of people with hemophilia and approaches to better evaluate and manage this common complication. The following US HERO Summit participants appear in this video:

·        Mary-Jane Frey, Nurse Coordinator, Children’s Hospital of Michigan
·        Neil Frick, Vice President for Research and Medical Information, National Hemophilia Foundation
·        Chris Guelcher, Nurse Practitioner, Children’s National Medical Center
·        Michelle Witkop, Bleeding Disorders Nurse Practitioner, Northern Regional Bleeding Disorder Center

To view all the videos, visit the Novo Nordisk YouTube Channel , and please feel free to share them with your friends, family, and organization members!

Great Book I Just Read
The Boys on the Boat by Daniel Jay Brown

Incredible true story of underdogs who go to the Olympics: the University of Washington’s 1936 eight-oar crew, their amazing and youthful coxswain, their aloof but dedicated coach and a legendary boat builder team together for an epic quest for an Olympic gold medal during the 1936 Olympics in Nazi Germany. The story revolves particularly around one boy, Joe Rantz, abandoned entirely by his family during the depression, who has to survive on his own, put himself through college and discovers his calling rowing crew. He seeks to fill a hole in his life, and learns to trust. He and his team became national heroes at a time when Germany sought to dominate sports, and eventually the world. It’s a lesson in human spirit, indomitable will, teamwork, leadership, strategy, family, optimism and hope. Along the lines of Seabiscuit and Unbroken, both of which happened at the same time as this story. Fantastic writing and story! Five/five stars.

A Camp to Call Their Own

Laurie Kelley January 7, 2013

By Janet Brewer

The bleeding
disorder community is small, comparatively speaking. Smaller still is the community of families and individuals affected by an inhibitor. But what we lack in size, we make up in our voices.

The hemophilia community has advocated for better care, better products and better programs to support our daily needs. As a result, we have arguably the best treatment model in the world. In the last 20 to 30 years, hemophilia has realized a standard of care that involves routine prophylaxis. This 2-3 day per week regiment enables most affected by hemophilia to lead a relatively “normal” life. Annual chapter meetings, national education conferences offer a plethora
of information specific to those living with hemophilia. Week long summer camp programs where self-infusion is taught and opportunities to meet peers in an environment that promotes leadership are a right of passage for our community.

Yet the smaller subset of individuals and families affected by an inhibitor continues to struggle. An inhibitor diagnosis changes the entire landscape of hemophilia treatment. Routine prophylaxis becomes a thing of the past until the inhibitor is tolerized. Immune tolerance treatment (ITT) can take years of daily, sometimes twice daily infusions and even then, not every child becomes tolerized. There is no single factor product that works to stop bleeding consistently. Bleeds can take days, even weeks to stop. Hospitalizations are frequent and family life is turned upside. Attending a weeklong hemophilia summer camp often becomes a wish. When attendance is possible, there is a high probability that there won’t be anyone else there with an inhibitor. Participating
in many of the activities that other blood brothers can do with prophylaxis is impossible.
As one camper with an inhibitor states, “How I envied them and wished I could
do what they could do.”

Jane Cavanaugh Smith and Janet Brewer conceived the idea for Inhibitor Family Camp. As long standing members of the hemophilia community and mothers of sons with an inhibitor, they endeavored to bring families affected by an inhibitor together in an activity filled, intimate environment and where better than camp? The very nature of a camp setting provides opportunities to try things for the first time, stretch ones limits and create peer relationships.

In 2010, Comprehensive Health Education Services (CHES) sponsored the very first Inhibitor Family Camp at Victory Junction in North Carolina. For the first time, children with an inhibitor and their families had a camp to call their own. Supported by an educational grant from Novo Nordisk, its goal then and still is to bring together families who continue to struggle with the challenge
of an active inhibitor. Families of a child whose inhibitor has tolerized are welcome if space allows. With camp now in its third year, CHES has partnered with Serious Fun camps (founded by Paul Newman), The Painted Turtle in California and Victory Junction in North Carolina to bring two Inhibitor Family Camp programs to the inhibitor community. In response to participant requests, this year’s camp program will offer three nights of education, activities and fun!

Inhibitor Family Camp is limited to 25 families per camp and slots fill up quickly. The Painted
Turtle session will be held Friday April 19 to Monday April 22, 2013 in Lake Hughes, California. Registration opens on January 4, 2013. The Victory Junction session will be held Thursday October 17-Sunday October 20, 2013 in Randleman, NC. Registration will open on July 1, 2013. Each of these programs are offered totally free of charge to eligible families.

Families response to what Inhibitor Family Camp means to them include:

“I loved all the activities. Lots of things to do. The boys had a wonderful time. I have no
complaints except we didn’t want to leave! Thank you!”

“Hanging with the other kids and connecting with them through similar experiences.”

“This program has been fully instrumental to my family in helping us deal with hemophilia and inhibitors.”

For additional information,
please call Comprehensive Health Education Services at 781-878-8561.

Click below to view Comprehensive Health Education
Services’  Inhibitor Family Camp Brochure http://attachment.benchmarkemail.com/c51644/2013_Inhibitor_Family_Camp.pdf

iPhone App Helps in Diagnosis

Laurie Kelley February 20, 2011

Here is the latest in very cool applications for the iPhone. I don’t think I have yet seen one that helps in the diagnosis; I believe this is the first. This is from Novo Nordisk. This app does hematologic testing, including prothrombin time (PT) and activated partial thromboplastin time (aPTT) tests. This resource has been developed to help you diagnose coagulation disorders. Here, you have access to the following features:

Lab Value Analyzer: Input the patient’s coags, and the clinician receives a list of possible diagnoses
Diagnostic Algorithm: this walks a clinician through step by step to narrow the possibilities of the diagnosis
Acquired Hemophilia

Check it out at http://www.coagsuncomplicated.com/Home.aspx

Great Book I Just Read
Annapurna: A Woman’s Place by Arlene Blum

Annapurna is the 10th highest peak, and renowned for avalanches. It takes extreme mental toughness and physical endurance. Of the 4 successful ascents of Annapurna only 2 men had reached the top each time. Arlene Blum became the first woman to ever summit it, and led an all woman team. She was met with skepticism and ridicule, and also great enthusiasm. This book details the preparation and the journey, and her struggles as a leader. It’s a great book about leadership. I had my own doubts reading it, as Arlene seems so insecure. But she is candid, and in the end, her leadership really shines. “My scientific training contributes to a natural tendency to see all sides of an issue. I tend to agonize about the full range of possibilities, and even after I’ve made a decision, to worry interminably about whether it was the right one.” Sadly, two women climbers on the team died when they chose to strike out on their own, against the wishes of the others to summit after Arlene.

Arlene Blum has played a groundbreaking role in US women’s mountaineering. She led the first women’s team up Mt. McKinley; was the first American woman to attempt Mt. Everest; and has played a leading role in more than 20 mountain expeditions worldwide. Three stars.

World’s First Inhibitor Book Published

Laurie Kelley November 1, 2010

Three years in the making, and it’s finally here–Managing Your Child’s Inhibitor, the world’s first book on inhibitors. At 279 pages, this is a comprehensive look at everything parents and patients need to know about so many aspects of inhibitors. From ITI to pain management, from insurance to attending school, this book weaves together advice from over 40 families with hemophilia, and includes the latest research and knowledge to give you a guidebook through every stage of inhibitor management.

The book is authored by me and my friend and colleague Paul Clement, who also has a son with hemophilia. The idea for the book was born during the Novo Nordisk Consumer Council meeting of 2005, after I heard the anguish, the suffering and saw the tears of so may families with inhibitors. I couldn’t believe what I was hearing. It was as though inhibitors were a separate disorder. I didn’t know people with hemophilia still suffered like that in the US.

As you may know, an inhibitor is an antibody the blood makes, directed against factor VIII or IX. The body doesn’t recognize the infused factor as belonging to the body–it sees it as a foreign invader and mounts an attack. The body produces antibodies which latch on and neutralize the infused factor. It renders it useless, and the blood continues to bleed. Very scary.

I learned so much creating this book: about pain management, different protocols, and the different products. But most of all, the incredible strength of people with inhibitors and their families. They are truly heroic people. I hope the book is a testament to their courage, and also helps give guidance to families new to inhibitors, so they can learn from their peers, and find the best route to elimination or at least management of the inhibitor.

My thanks to Novo Nordisk, which gave us an unrestricted grant to research write and publish this book. Novo Nordisk is the manufacturer of NovoSeven® RT, a bypassing agent used in the treatment of acute bleeds in patients with hemophilia and inhibitors.

You can order the inhibitor book via our website at https://www.kelleycom.com/books.html