Uncategorized

Ambassador for bleeding disorders

Laurie Kelley November 3, 2019

Excerpted with permission from an on-line article by Jojo G. Silvestre

World Hemophilia Day [April 17] is an international day of observance of which not many people seem to be aware. I am one person who may have a better understanding of bleeding disorders, a unique yet quietly widespread human malady only because my dear friend, Andrea Hernandez Trinidad, president of Hemophillia Advocates-Philippines, internationally-recognized as the face of hemophilia in the Philippines.

I met Andrea in the early years of the new millennium. A former newspaperwoman whose stories appeared on page one of a top daily, she shifted to public relations and, not unexpectedly, was very warm and friendly. It turned out it was not for convenience and business purposes that she had a ready smile. This was one lady, I soon realized, who was sincere in her dealings with others and whose friendliness is not dictated by the so-called exigencies of her profession.

Andrea, from day one of our friendship, had been one socially-confident, articulate and smart gal who dressed up properly (her style could be powerful at one time, sporty the next, or glamorous when the occasion called for it), laughed with gusto and listened attentively. Ours has been a two-way traffic, although I must say I’ve been more on the receiving end (ha ha ha) because, well, she has so much to give and I just don’t mean the “pasalubong” (gifts from travels) that she showers me with whether she needs me to edit something or not.

Managing bleeding disorders is expensive. But the scarcity of medicines — the factors — is something that truly surprised me. That even if you have the capacity to purchase these medicines, there are times you can’t find enough supply.

Although this may be late for World Hemophilia Day itself, I am focusing on her and her special cause — the promotion of awareness of bleeding disorders so that those who suffer from them would be given suitable and timely support which can be best institutionalized through legislation.

According to the website of the World Federation of Hemophilia (WFH), the organization, this year, “is focusing on reaching out and identifying new members of the bleeding disorders community. The WHD 2019 campaign will feature examples of WFH outreach initiatives such as organizing regional workshops, raising awareness of bleeding through media coverage or training health professionals and collecting data. It will combine these examples with submissions from our community about outreach efforts in their own regions or ideas about what would be helpful for future initiatives.”

Andrea comes from a family of public servants in the Visayas. Her maternal great grandfather, Jose Lopez-Vito, was a Supreme Court Justice. On the other hand, her paternal great grandfather, Vicente Vergara, was the first Mayor of Guihulngan, a locality in central Negros.

Her own father, Jesus Trinidad, was a long-term councilor and currently, two of her siblings and few cousins hold different positions in government.

Andrea and her five siblings were practically raised by their mother, Sofia, single-handedly, because their father was a martial law detainee in the 1970s and had to leave his family to work elsewhere after his incarceration.

Andrea remembers seeing her mom having bad monthly periods. Many times she would be confined to bed. She recalls, “In 1988, her bleeding became so bad that doctors wanted to run a biopsy on the lump on her uterus. She didn’t survive the supposed simple operation.

“My mother literally bled to death while undergoing biopsy operation simply because the condition was still unknown in the Philippines at that time,” recalls Andrea.

If it was devastating to the whole family, Andrea would later find out that she had inherited the disease. No wonder that as a teenager, she had a bleeding disorder.

She also experienced multiple bleeds every time she was pregnant, so all her three children were born prematurely. But it was not until her youngest child, Star, was born that she finally suspected that a bleeding disorder runs in her family.

Next week, Part 2: A Star is Born!

From: https://tribune.net.ph/index.php/2019/04/30/ambassador-for-bleeding-disorders/

Hemophilia and Vampires!

Laurie Kelley October 27, 2019

It’s Halloween week!

The holiday is a festive one, in which children and adults dress up in traditional costumes like ghosts, Frankenstein, pumpkins, devils and vampires, and nontraditional like pirates, doctors, celebrities and political figures.

I recently watched a holiday classic, The Nightmare Before Christmas, and am reading the classic Dracula, by Bram Stoker—a masterpiece of creativity and writing! If you haven’t read it, please do!

And as vampires are associated with blood, our contributing writer Richard Atwood has investigated literature about hemophilia involving vampires!

Vampire Blood Bank is a book about Lorenzo Zilch, called Larry, a male emergency room nurse at Samaritan Hospital in New York City. Larry narrates his involvement with Aaron E. Newman, a Jewish person with hemophilia who, while walking through Central Park on Thanksgiving night, is bitten by the first ever Jewish vampire!

Aaron turns as a vampire. Yet he has hemophilia. And Aaron abhors the sight of blood, so he cannot bite others! Larry and Aaron then open the Eleventh Hour Blood Bank to meet their needs, though not without others wanting a piece of the action.

Sound interesting? Richard writes that this farcical novel has no redeeming value other than laughter, “which is the best medicine.” The full title is The Saga of a Jewish Hemophiliac Who Can’t Bite So He Opens a Vampire Blood Bank. Almost every page contains a joke or gag designed for mature audiences. Aaron takes treatment for his hemophilia, and for his thirst for blood, from the stockpile at the Eleventh Hour Blood Bank.

He treats himself with four bags of whole blood per day. This treatment is enough to cover his prostate surgery. Aaron finds that being a vampire improves his blood clotting, and even helps his usual symptoms of hemophilia to recede. The commercialization and the profitability of the blood bank become evident for other vampires and for others in the blood business!

The author, Harry Zelenko, grows orchids in New York City in addition to having a writing career. Well, this crazy little novel can’t compare to Dracula, but then, what can? Read and enjoy them both! Happy Halloween!

Know Your Factor Choices

Laurie Kelley October 20, 2019

It’s truly a new era in bleeding disorders with new products, new therapies and new indications. Octapharma’s wilate just got approval for indication in adults and adolescents with hemophilia A; previously it was indicated only for VWD. With this news, I realized that we should remind everyone to know what product they use, where it comes from, and why they are using it.

Who chose your product? Was it your hematologist? Did you have any input? You should, and this means bearing the responsibility to read about products, as questions about them and be part of the decision making process.

Back in 1990, when Raising a Child with Hemophilia was first published, we were the first ones to describe parents and patients as consumers, and to treat products like any other commercial product you would buy. We provided the question to ask your doctor, and we explained the different manufacturing processes, and the difference between safety and purity. And we urged parents not to be passive in the treatment process but to help make medical decisions.

It’s more important now than ever. We have a dazzling choice of products, and novel therapies like Hemlibra. Maybe there’s no reason for you to switch products, but you might want to get up to speed on what’s available. Please note: all US FDA-approved products are considered safe and efficacious.

We’ve been writing about products and product choice for decades. I hope you read up on them and be prepared! To help you, we created a chart of products that you can download. Take it to the HTC with you and discuss with your hematologist about choice. You can download it right from our home page or go here.

We also have a chart for VWD products too!

We did our part. Now do yours; know your product, manufacturer, and why you use it. Keep learning… more therapy choices are coming!

A Personal Approach to Healthcare

admin October 13, 2019

Ever hear of personalized healthcare (PHC)? PHC means tailoring a treatment regimen specific to an individual patient. It acknowledges that every patient is different, with different physiology, biology, reactions to medicine, and lifestyle. A treatment regimen that works for one patient may not work for another with the exact same ailment.

The goal of PHC is to improve your quality of life as a patient with a specific disease or disorder, by taking into
account your individual needs and lifestyle, and then tailoring a treatment plan. How does that happen? It’s a collaborative process involving you and your medical team. Your HTC medical team knows all the treatment options, but you know yourself, your body, and your lifestyle. To develop your own PHC, you’ll need to share with the team every aspect of your past health, current health, needs, and desires. Only then can you all explore treatment options that best suit you.

Your Personalized Healthcare Team

The best place to get help with hemophilia and all bleeding disorders is at a hemophilia treatment center (HTC). These are centers of excellence that specialize in diagnosing, treating, and monitoring bleeding disorders. There are over 140 HTCs throughout the US. They follow the model of comprehensive healthcare, which means that they’re not just treating bleeding episodes, but all aspects of living with a bleeding disorder. From orthopedics to psychosocial needs to genetics, the HTC team knows how a bleeding disorder can impact your life.

By using comprehensive healthcare, isn’t your HTC team already implementing PHC?

Maybe not. First, not all HTCs are created equal. Some may lack certain experts you need, such as a pediatric hematology department or a geneticist. And some may have strong beliefs, different from yours, about when—or whether—to start prophylaxis (prophy), or about whether you should try new products.

Parents and patients need to become partners in PHC. Are they ready for this collaboration? Parents of newly
diagnosed children may be too shocked at first, and not even know what questions to ask. Older patients may be overlooked for PHC because they’ve been on the same treatment plan for a long time, their blood work is good, and they don’t complain or ask questions.

It’s important for you to be ready to partner with your HTC and let the team know your needs. But even if you’re comfortable standing up and being heard, what will you say? What will you ask?

1. CHOOSE THE RIGHT TREATMENT

One of the first decisions you need to make about your PHC is which treatment to use. All products licensed in the US are considered safe and effective, but they’re not all the same. How do you find one that’s best for you?

You know that new products are entering the market. You can choose between plasma-derived (made from human blood) and recombinant (made from animal cells containing human genes). Within the recombinant products, there are categories: first, second, or third generation. And there are novel therapies, that are not even factor! So begin by asking your hematologist for opinions on all products.

Your decision about treatment may come down to lifestyle, when selecting a particular brand for prophy. Perhaps the brand you’re using now, three times a week, works well for you or your child. Does a product with an extended half-life offer fewer infusions, saving veins from wear and tear? Or would you benefit from a new, subcutaneous product? Some products might not be available through your insurance. Talk through these choices with your HTC.

2. BLEEDING PATTERNS

Perhaps nothing is more personal than your individual bleeding pattern. People with hemophilia bleed differently, in different places, from different causes. Once your child begins getting bleeds, notice his bleeding pattern, if any. This is where you can really help your HTC team personalize your treatment. The information you share can help prevent a target joint from starting, or can compel the HTC team to put your child on prophy.

You may hear severity levels described like this: Children with severe hemophilia will bleed from trauma, or spontaneously, with no known trauma. Children with moderate hemophilia may bleed on average once a month, with known trauma. Children with mild hemophilia may bleed only after dental extractions and surgery. But what’s the reality? Some children with severe hemophilia bleed only monthly; some with moderate bleed every week, with no known trauma. Your child’s bleeding is unique!

And know his personal symptoms of a bleed: Tingling? Hot? Swollen? Your HTC staff can help you identify symptoms, so you can infuse more quickly at the first sign of a bleed.

3. ESTABLISH A TREATMENT REGIMEN

Personalized healthcare really shines when it’s time to devise a treatment plan. You can use the new, subcutaneous novel therapy. Or you have two options for infusing factor: on-demand (also called episodic) or prophylaxis. On-demand means infusing at the first sign of a bleed. Prophy is the scheduled infusing of factor. It’s designed to keep factor levels in the bloodstream high enough—greater than 1%—to prevent most spontaneous bleeds.

Prophy is the recommended therapy for children with hemophilia in countries like the US with ready access to clotting factor.

And what about your schedule for prophy? This is about as personal as PHC gets! Your HTC team will offer a schedule based on your child’s needs: your family lifestyle, activity level, perhaps pharmacokinetics (PK) data to determine how quickly factor is cleared from the blood after an infusion, and any breakthrough bleeding that might happen.

4. DISCUSS PERSONALIZED MEDICINE

Personalized healthcare is not the same as personalized medicine, a common term. Personalized medicine often refers to using a person’s genetic profile—genomes and specific genetic markers—to guide therapy for cancer and other diseases and disorders, including hemophilia. But PHC includes personalized medicine, and it’s worth discussing with your HTC team because more and more, the hemophilia community is focusing on personalized medicine.

National Hemophilia Foundation (NHF) recognized the importance of this genetic research and launched My Life, Our Future (MLOF). Through this program, you can get a blood test that enables you to learn more about the specific genetic mutation that caused your child’s hemophilia.

So PHC uses personalized medicine to examine your genetic makeup, help predict which medical disorders or diseases your child is most at risk for, and suggest which treatments will be safe and effective (or not) for him. This is particularly important for complications like the risk of getting inhibitors.

We’ve shown you examples of treatment and lifestyle areas to focus on, potential needs to address, and questions to ask your HTC team to design the best life possible with your bleeding disorder. Your HTC team will become one of your most important partners.

NHF’s 71st Bleeding Disorder Conference

Laurie Kelley October 7, 2019

I just returned from Anaheim, California, where the 71st bleeding disorder conference was held. From Thursday till Saturday night, this unique community gathered to share stories, learn from one another, hear experts speak on a variety of subjects. I’ve been attending since 1992, and I think I’ve only missed one, in a year when I gave birth to my third child. This year, however, was quite special. It marks the end of an era: NHF CEO Val Bias is stepping down as leader.

I always feel funny using the term CEO with Val, because he is more than just that title to this community. He has been a friend, camp counselor, mentor and activist. He always has a ready smile, and a demeanor that makes you feel he has all the time in the world to chat with you.

I didn’t always perceive him that way. I first saw Val when in 1992 I attended my first NHF annual meeting in Atlanta. This was at the height of tensions between the consumer advocacy groups and pharmaceutical companies, during the HIV crisis. One group of activists even dressed as grim reapers, poured red paint on themselves to represent spilled blood, and walked among the booths at the exhibit hall, banging a drum. It was chilling, and everyone was on edge. Many pharma reps left, especially after they had received death threats. The exhibit hall became deserted.

Kim Phelan, executive director of the Coalition for Hemophilia B congratulates Val Bias

I attended one session, a town hall, where people could speak up and bring their complaints and air their views. At the head of the room was a podium and table, at which sat representatives from our community: Bill Sparrow, Rachel Warner, and a few others whose names I can’t recall. In the front row, an angry Michael Rosenberg, who had hemophilia and HIV, and who headed an advocacy group that was seeking justice for the HIV infection of factor. I didn’t know all the issues at that time but watched as Michael eventually was on his feet, yelling at the speakers on the podium, and the room erupted! The community was angry, hurt… poisoned. Children and adults had died. The tension was incredible.

Out of this pandemonium came a voice from the podium asking everyone to calm down and to sit down. It commanded the room, and everyone listened. People stopped shouting; people sat down. I was amazed and wondered, who is this guy, who can bring order to chaos and calm people with a commanding voice and apparently, reputation?

That was my very first impression of Val, a leader in a time of crisis. He has played many roles in our community, including helping to bring the Ricky Ray bill into law, which provided compensation to those with hemophilia who were infected with HIV and hepatitis C. He’s been a fighter on the front line for blood safety, and more recently, a fighter to keep access to all therapies in the face of insurance barriers.

The opening night of NHF last week was a tribute to Val. A beautiful video, with testimonies from former NHF president Mark Skinner and current NHF COO Dawn Rotellini, shared his contributions for the past 11 years as CEO and his contributions to the community overall. It’s a life well lived. It always struck me that while Val too was infected with HIV and hepatitis C (his first wife died shortly after being infected by him), Val never seemed to harbor bitterness. He only sought justice.

We will miss his leadership. But we hope that he will stay visible in the community, to share his warm smile, hearty laugh, wisdom and friendship. He leaves a legacy, and very hard shoes to fill.