December 2015

“All I want for Christmas…”

Jagatman’s amputation

“All I want for Christmas…” It isn’t two front teeth. (Did you know that Nat King Cole does a version of that song?) 

It’s a leg.

With all the shopping we do at Christmas, imagine wishing only for a leg. When I visited Nepal in September, I reported on one of our “boys,” Jagatman. He’s only 26, same age as my daughter Tara. Unlike her, his life is so uncertain and frail. He has hemophilia; he lives in a country that purchases no factor. 

Jagatman at work

And he lost his leg due to an untreated bleed. Through Project SHARE, we provided factor for the surgery to remove his leg, to save his life. And he’s so grateful to us. Sigh, the fates must be angry. The April 25 earthquake ripped apart his brick home. I stood before the rubble of what once was the home of the Rajbchak family. 

But he has a mobile phone repair shop (Save One Life helped get him started with funding) and I saw him eagerly at work. He’s good! Industrious, focused and determined to succeed. It must have taken a lot of courage to ask me to help him purchase a leg. He’s outgrown his current prosthetic; it hurts him. Given the high tolerance for pain these boys have, it must re

ally hurt him. He earns only about $500 a month from the shop, and must pay rent. The leg he showed me in a colorful brochure costs almost $4,000. A fortune. A king’s ransom.

With brother Monsoon,
who also has hemophilia

But I promised this handsome young man that by Christmas, we’d buy him that leg. What better gift?

And last week we made good. The money was wired and he will soon have his leg. Despite a chronic disorder, limited access to medicine and poverty, Jagatman is beating the odds. His attitude is astounding; his determination like Rocky’s. His focus like a laser beam. 

And he’ll never know that the gift he’s given me is so much more than anything I can ever give him. 

Merry Christmas to our friends in Nepal, and those with bleeding disorders everywhere!

Great Book I Just Read

Life [Kindle]
Keith Richards

Best rock bio I have ever read. Told by the iconic guitarist of the Rolling Stones, Richards almost seems as surprised by his life’s journey as you are bound to be after reading this. Meticoulously detailed, Richards shares his humble beginnings, his teen encounter with life long rock partner Mick Jagger, their rocky road to fame, the drugs, the alcohol… but what makes this bio so different is first, the Stones are one of the greatest rock bands in the world, from the early 60s and still playing! Second, Richards delves into the music: his love of American blues, what he wrote, why, how he got ideas, the chords, the notes, the guitars. This is what I have been wanting to know: how do these musicians create? He’s a voracious reader, loves his library, and lives in Connecticut, a few hours from me. He’s a survivor for sure, and what a story he has to tell. Five/five stars!

It’s Just a Phase: Clinical Trials of New Products

We’ve witnessed several new products come to market in the past few months; it’s pretty astounding, given that years went by without any new products. You’ve probably heard about others that are in clinical trials. What does that mean, exactly?
The FDA requires that new drugs and treatments be tested for safety, and also to demonstrate that they effectively treat the disorder or disease for which they are being developed. Drugs always start with a
phase I clinical trial, and then may move to phase II and phase III trials. If a drug is found unsafe or ineffective at any stage, then the clinical trial will be ended, and research on the drug will be either modified or abandoned.
Phase I: After a new drug or treatment is initially tested on animals, researchers test it on a small group of people (20–80) to evaluate safety, determine the safe dosage range, and identify side effects.
Phase II: If a drug or treatment appears safe at the end of the phase I trial, researchers give it to a larger group of people (100–300) to evaluate its effectiveness in patients, and to further assess safety.
Phase III: At this stage, the drug or treatment is given to an even larger group of people (1,000–3,000) to confirm effectiveness over a broad range of patients, monitor side effects, compare to commonly used treatments, and collect information to promote safe use. For rare disorders such as hemophilia, it’s almost impossible to enroll 1,000 patients for a clinical trial. In these cases, the FDA will approve
phase II or III trials with much smaller populations.
Phase IV: Post-marketing studies provide more information, including the drug or treatment’s risks, benefits, and best use.

Clinical trials for new bleeding disorder products can take place only when patients volunteer to be in them. This may seem risky, trying a new product that hasn’t been FDA-approved yet. Yet you can participate in a clinical trial, to help research, to take advantage of a new product that is needed, or simply for free factor. You’ll need to speak with your HTC about joining but k
now as much as possible about the clinical trial you are interested in, 

and feel comfortable asking your healthcare team 

about it. Use these questions as a guide:

• What is the purpose of the trial?

• Who is going to be in the trial?
• Why do researchers believe the experimental treatment being tested may be effective? 
• Has this treatment been tested before?

• What kinds of tests and experimental treatments are involved?

• How do the possible risks, side effects, and benefits compare with my current treatment?
• How might this trial affect my daily life?
• How long will the trial last?
• Will hospitalization be required?
• Who will pay for the experimental treatment?
• Will I be reimbursed for other expenses?
• What type of long-term follow-up care is part of this trial?
• How will I know that the experimental treatment is working?
• What happens if I am harmed by the trial?
• Who will be in charge of my care?
Patients who volunteer to be in clinical trials are truly heroes and trailblazers. Perhaps you will be one of them someday!

Adapted from

Great Book I Just Read

Deep Down Dark: The Untold Stories of 33 Men Buried in a Chilean Mine, and the Miracle That Set Them Free [Kindle]

Hector Tobar

Astounding survival story of the 33 Chilean miners trapped over 1,000 feet underground for 69 days. Tobar skillfully introduces each man, his home life, aspirations, character flaws… all which would impact each’s reaction and perspective during their ungodly entombment. As in many crises, the positional leader does not always become the situational leader. This is as much a story of survival as it is leadership. Their time underground is gut-wretchedly and vividly portrayed. It seems a true miracle that they survived. The efforts to help the men spread internationally, and forever changed the Chilean mine industry. Five/five stars. (The movie “The 33” is good but not nearly as good as the book)

Pulse on the Road in Baton Rouge

Zoraida and Kelly before the presentation

Just a 90 minute drive northwest from New Orleans, the “Big Easy,” Pulse on the Road brought a sense of urgency to the 80 attendees in Baton Rouge, state capital of Louisiana, of our latest presentation on insurance. Over a warm lunch of chicken, potatoes and greens, we provided the history of insurance reform in the US, particularly as it affects people with chronic disorders like hemophilia and von Willebrand disease, and how payers continue trying to cut costs… and why.
Laurie Kelly and attendee
Shawn Whelan
As we did two weeks ago, I opened with the brief history of escalating drug and medical costs, showing how imperative it was that insurance companies find ways to cut costs. Michelle Rice, vice president of public policy and stakeholder relations at NHF and recognized insurance “guru” of our community, then educated about how payers would go about doing this. Restricted networks, higher co-pays, specialty tiers, prior authorizations–sound familiar? And each of these methods at best will impact your bottom line and at worst could impact your access to certain therapies you might need.
Speakers Michelle Rice (NHF), Kelly
Lynn Gonzales and Laurie Kelley
Finally we closed with an emotive and inspirational story by the indomitable Kelly Lynn Gonzales of Nevada, a woman with von Willebrand disease who fought tenaciously for her daughter’s right to treatment–and won! Not only the treatment for her daughter, but treatment for all with VWD in Nevada… and tremendous respect from the medical and insurance community.

Said Carl, an attendee with hemophilia, “I’ve heard and read lots of testimonials from people and their problems with insurance, doctors and hospitals. But these ladies really opened my heart on how some people are really neglected.”

Donnie Akers, former board member of Hemophilia of America, told us that this was one of the best presentations he has seen in the last few years. Thanks to all who made this a success, including the Louisiana Hemophilia Foundation, and Baxalta, for sponsoring this event.That concludes our year of Pulse on the Road! 
Laurie and Jan Hamilton,
founder of HFA
After the event, Zoraida and I drove to New Orleans to enjoy the sights, sounds and tastes of one of our greatest American cities. Admiring the Spanish wrought-iron balconies and old architecture, the “voodoo” shops, the jazz music pouring out of every street corner bar and the smells of Cajun cooking, New Orleans is like no other place in this country! We capped our day with a “Haunted Houses” tour in the dark. New Orleans has a rich history of hauntings, and the stories vary from when pirates roamed the streets, to Nicholas Cage’s purchase of a $5 million house, not knowing its gruesome history! He soon sold it.
We end the year with a pep talk! Read your insurance policy annually, document your insurance inquiries and treatments, watch for hidden costs, and put aside an emergency fund for unexpected escalating costs and to help pay for hospitalizations. Be vigilant!
See you next year! 

Voodoo Shop!
Louisiana Riverboat

Andrew Jackson Statue

Most famous haunted hotel: The
Andrew Jackson!

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