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When to Suspect an Inhibitor

Laurie Kelley June 16, 2019

Let’s face it: an inhibitor is a major complication of hemophilia. It develops when the body’s immune system does not recognize infused factor as a normal part of blood. Instead, the body thinks that factor is a foreign invader, like a virus or germ, and it develops antibodies to attack the factor and make it harmless—and useless. So despite an infusion of factor, your child continues to bleed. Prolonged bleeding, even after a factor infusion, is the most common sign that your child may have an inhibitor. But other symptoms may also tip you off.

Unresolved bleeds

You may suspect an inhibitor the hard way: when factor no longer works well to stop bleeds. If you’re new to hemophilia, this may be difficult to judge—how long should it take for a bleed to stop after an infusion? It may not immediately dawn on you that the infused factor isn’t working properly, especially if your child is already well into a muscle or joint bleed. It’s common for parents to think that they simply need to give their child more frequent infusions, or give a higher dose per infusion. If you think your child’s bleed is not resolving normally, or wonder whether you should dose higher or more frequently, please call your hemophilia treatment center (HTC).

Increased bruising

Bruising in young children with severe hemophilia is common. But if your child is on prophylaxis and you notice increased bruising, this may be a sign of an inhibitor.

Routine clinic visit

A blood test at your child’s HTC comprehensive clinic visit can identify an inhibitor. Low-level inhibitors are often diagnosed in this way. It’s wise to have a child with hemophilia tested for inhibitors routinely. Learning that he has an inhibitor prior to surgery or a major bleed allows parents to have a plan in place and the correct treatment on hand.

Breakthrough bleeds while on prophylaxis

Prophylaxis is the scheduled infusion of factor to help prevent bleeding. Many children with hemophilia on prophylaxis receive factor two to three times a week, enough to allow circulating factor to prevent most spontaneous bleeds and abnormal bruising. When a child on prophy starts bruising or bleeding more often than usual, an inhibitor may be inactivating some of the factor, lowering his factor level and increasing his risk of bleeding.

Bleeding after surgery

Any kind of surgery on a child with hemophilia requires careful planning and monitoring of factor levels, and any child who continues to bleed following surgery, even with adequate factor, should be immediately tested for an inhibitor. Ideally, all children with hemophilia should be tested for an inhibitor before any surgery. If you see any kind of bleeding following surgery, call your HTC immediately.

Reaction following infusion

An allergic reaction is a response by the immune system to environmental contaminants such as pollen, animal dander, or food. It can also happen after an infusion of factor. Symptoms may include sneezing; itching; hives; rapid swelling of the skin, neck or face; wheezing; faintness; fast heart rate; low blood pressure. Allergic reactions are especially worrisome with hemophilia B. An allergic reaction after a factor IX infusion is sometimes the first sign that an inhibitor to factor IX has developed. A whopping 45% of people with hemophilia B and inhibitors also develop allergic reactions at about the same time that they develop inhibitors.

Don’t downplay allergic reactions. They may start out mild but then increase in severity after repeated exposure to products containing factor IX, often to a serious, life-threatening allergic reaction called anaphylaxis. If your child has severe hemophilia B, his first 20 infusions of factor IX concentrate should be done in a hospital or clinic with expertise in treating severe allergic reactions.

Following a major bleed or emergency

Whenever your child receives large amounts of factor—in response to a major bleed or during and after surgery—he may be at higher risk of developing an inhibitor. Experts aren’t sure if large amounts of factor stimulate inhibitor development, or if the body is more susceptible to inhibitor development during a medical crisis because the immune system is on high alert. But whatever the reasons, be aware that the risk of developing an inhibitor is slightly higher during an illness or surgery.

Later in life

If a person with hemophilia develops an inhibitor, it’s usually while he’s a child, almost always before exposure day 100. And he probably has severe hemophilia. But in rare cases, an inhibitor develops in a teen or an adult—usually in people with mild or moderate hemophilia, and usually after intensive exposure to factor during and after surgery or traumatic injury.

Inhibitors are scary to contemplate. Even when your child passes exposure day 100, don’t be lulled into thinking that he may never develop one. You can always request an inhibitor test from your HTC. Never try to diagnose on your own, or change your child’s dosing regimen on your own.

In your favor? A great array of educational resources, listed in this issue, and a wonderful team of medical experts. Don’t be afraid! Be proactive, just as you were when you learned about hemophilia. Inhibitors are one obstacle to overcome on your family’s hemophilia journey.

The Joy of Giving

Laurie Kelley June 9, 2019

“Fundraising is a systematic, coordinated effort inviting people to experience the joy of giving.” Aly Sterling

Last weekend, June 1, I was in New Orleans at the Hope for Hemophilia spring leadership weekend and gala, at the invitation of its president, Jonathan James, who has hemophilia. Hope for Hemophilia is a nonprofit that does in the US what we at Save One Life do overseas: provide direct financial aid for families with bleeding disorders who need assistance. While we have never worked together formally, I sometimes get requests for help here in the US: a plea for rent to be paid for the month, or a heating bill, car payment, co-pay. Even college tuition. I typically will forward the request to Hope first, or maybe HFA’s Helping Hands program, but if the person has already tapped out there, we will try to help them.

There is such joy in giving. Not so much like Christmas gifts or graduation gifts, but a gift that helps a person get ahead in life. A gift with no strings attached, often to a stranger.

Save One Life does this; Hope for Hemophilia does this. And this time, Hope did it to me. Gave me a gift, no strings attached.

Jonathan had not prepared me for the gala, though I had heard it was the most elegant event in hemophilia in the country. Black tie and gowns, everyone was dressed for a ball, and the theme was masquerade. Many people bought charming masquerade masks to cover just their faces, á la Phantom of the Opera.

Doug and I walked arm in arm up a huge flight of stairs to the ballroom, which had such a high ceiling, it felt like a canopy of stars at night. Each of the many tables had huge candelabras; everything was donned in purple, giving it a mysterious, Houdini feel. We sat with Jonathan’s parents, who were charming, and another family, who were founding members of Hope.

The gift Hope for Hemophilia gave to me that night was not just their Legacy Award, lovely in itself, but a beautiful video, narrated and sincere, which chronicled my story from the birth of my son, to my books, to my work overseas. I was stunned. No one had ever paid this type of tribute to me before. I don’t even have time to organize my own photos; how did they do this? How did they have the time, when they had an entire gala, with over one hundred guests, dancers, music, silent auction…

(See the video here)

It made me realize two things: 1) We truly honor whatever we spend time on. Hope for Hemophilia recognized the time and energy I’ve sent the past 30 years in the community, and I was honored with this amazing video. 2) Giving without any expectation of return. I’ve never donated to Hope for Hemophilia, as I have my own nonprofit to fund. And I give away funding to those in need directly. Jonathan James gave me an award and a video as a gift, pure and simple. And it was so profound. I was very humbled by this gesture.

In my acceptance speech, I touched upon this theme. We are put on this earth, I believe, to give without expectation of return on investment. We give because it’s the right thing to do. Because another human being is in need. And what we get in return whether it is immediate or somewhere down the road, or maybe even in the next life, is worth so much more than the gift!

That very week, on Tuesday, a handsome, fit young man of 20 showed up in the Save One Life office. Owen is from Kenya and he wanted to meet Save One Life, since we operate in Kenya. In speaking with him, I found out he also wanted to thank me; 18 years before, he was a two-year-old, in the public hospital, bleeding. There was no factor in Kenya. His doctor, who I know, made a request to us, and we shipped the factor . He was infused, healed and was sent home. Now he is in my home state, studying to be a physical therapist so he can one day work with hemophilia patients.

He came to thank me; I was the one who had sent him that factor, 18 years ago. Amazing!

And his gift to me? He will join our board of directors to represent Africa.

Thanks to Jonathan James, Porter Snow and everyone at Hope for Hemophilia for their generous programs, their sincerity in wanting to make the world a better place, and their goodwill and positivity. Thank you for an amazing weekend. I’m pleased to know we share the same values and outlook, and desire to help, and I would love to work with them in the future.

Please be sure to give, without expectations, and experience the pure joy of it!

See the video here.

When Donating Factor

Laurie Kelley June 2, 2019

“I only want to help…”

It’s disturbing when we see the photo of an impoverished African child with hemophilia who is in pain or has chronic joint damage.

And we feel helpless when we get a Facebook request for factor from a desperate young man with hemophilia in Asia. As patients, we can feel their pain. As parents of children with bleeding disorders, we want to alleviate their suffering. Yet in our desire to do good, we may end up doing something not so good. We may send factor, on our own, to a place we’ve never visited, to people we don’t know. But won’t our generosity help them? What could be wrong with that?

Plenty.

In our efforts to help, we may make several serious mistakes.

Here’s what to look for if you receive an international request for help.

Laurie Kelley with family in
Pondicherry, India

Is the request for real?

This is your first question. Just because someone says he has hemophilia and needs factor, this doesn’t mean he actually does. At Project SHARE, we do extensive background checks with the local physicians and hemophilia organization (if there is one) to verify the need. And even if the request is valid, don’t forget that English is not the first language in most developing countries; it’s easy to get request mix-ups, dosage errors, even incorrect diagnoses.

SHARE keeps reference files on every one of the hundreds we have helped. We must be sure we have valid and accurate information. If you are approached, always realize that there may be missing information, so ask questions. We always ask for diagnosis, physician’s name and contact info, whether the patient knows how to self-infuse, and how far he lives from an HTC, for starters.

Should a private citizen ship factor?

Factor is a biological drug that requires careful shipping and handling. It’s expensive to ship and must travel via international carrier, such as FedEx. Factor can’t be left on trucks, on planes, or in warehouses. And addresses can be tricky overseas!

In India, for example, one of my favorite addresses is “Next to Camel Lot, behind Cinema.”

Are you familiar with the country’s customs laws? Who will pay the duties, or the tax on the shipment? It may even be illegal for you to ship these products; do you know the penalties for shipping a prescription drug over international borders? If you are employed by a hemophilia organization or HTC, are you putting that entity at risk? Whether you’re a private citizen or employed by a healthcare agency, if you are unsure about duties, penalties, and costs, don’t take the risk—don’t ship.

Getting an infusion of Project SHARE factor in
Vijayawda, India

What if the person is asking to come to the States for treatment?

Just say no. Most medical problems related to bleeding disorders can be handled locally, in the country’s capital. India and Pakistan, for example, have some of the best hematologists on earth. The biggest obstacle to care is often not the lack of doctors or expertise, but the lack of factor. At Project SHARE, we’ve found that many requests to come to the States are not for treatment but for job opportunities, college, or the chance to join relatives, either legally or illegally. Hemophilia is a ticket to get in. Be careful!

“It’s an emergency!”

Emergencies are hard to fix. Often, by the time Project SHARE ships factor, the emergency has passed—for better or worse. Declaring an emergency is sometimes a way for a patient, HTC, or hemophilia organization to acquire donated factor; whether it will be used for the emergency remains to be seen. We must assess the nature of the emergency to see whether a shipment will even help. If it’s surgery that can wait five days, then we can ship. Once, we saw a devastating photo of a baby in ICU with a head bleed…and we knew no factor shipment was going to help. We declined.

Check locally first.

All requests should first be vetted through the local and/or national hemophilia organization in a country. India has more than 65 chapters, so local is best. By contrast, the Dominican Republic has only one national organization. You can hop online and check the World Federation of Hemophilia’s website (www.wfh.org) to get the name and email of a national organization. But with over 100 member countries, this can be time-consuming and may not produce all the info you need.

Not speaking the patient’s local language may make it hard to communicate! At Project SHARE, we’ve found that we can resolve a lot of questions by informing the local organizations, whose staff often speak English, about requests that are coming from their members directly to the United States. Our goal is never primarily to “rescue” a patient, but to turn the request back to the national organization, to allow it a chance to help, and to strengthen its ability to solve problems.

Social media like Facebook creates connections between the developed and developing world. At SHARE we’ve seen many requests for factor and medical help come through Facebook to nice—but often inexperienced—hemophilia families and organizations who want to help. Americans are generous but aren’t always familiar with the international world. Be careful. Don’t risk losing a shipment of factor. Don’t weaken the local or national groups by training patients to come to you first instead of to the national organizations. Don’t be duped by people who end up getting the lion’s share of donated factor because they know how to use Facebook and email—people who pump out simultaneous requests to many developed countries and organizations.

What can you do that will really help?

Contact Project SHARE. We have been working with more than 65 countries for 17 years, and we’ve shipped over 144 million IU of factor. We’ve seen and heard just about every scenario, and we can make decisions about factor that will give help where it’s most needed, strengthen the national or local hemophilia organizations, and ensure that factor goes to patients with bleeding disorders. Consider us your US experts on hemophilia international aid. Like you, we want to help. Let us help you give help, effectively and efficiently.

Bayer Teams Up with WFH to Provide Aid

Laurie Kelley May 26, 2019

Anyone who knows me, knows I care deeply for patients with bleeding disorders in developing countries. I’ve been trying since 1996 to create ways to get factor concentrates to them, from simply shipping it through Project SHARE, to helping start patient nonprofits in other countries in order to ship more factor.

So whenever I hear of stories like the one I’m about to share, I get very excited, and deeply grateful. I know so many children whose lives have been forever changed by simply getting factor. It’s a miracle drug, and needed by so many.

Bayer announced last week that it is entering into a five-year partnership with the World Federation of Hemophilia (WFH) to bring education, training, and Bayer’s full portfolio of recombinant factor VIII (rFVIII) treatments to healthcare professionals in more than 60 countries where access to care is limited. Yes!

As part of the agreement, Bayer will provide financial assistance to support the training and education of healthcare professionals on the accurate and safe administration of treatments. WFH has local partner organizations through which they distribute the donation and manage logistics.

The expanded WFH Humanitarian Aid Program seeks to improve the lack of access to care and treatment by providing much needed support for people with inherited bleeding disorders in developing countries. As we all know all too well, providing patients with a more predictable and sustainable flow of humanitarian aid donations is critical, and the WFH Humanitarian Aid Program makes it possible for patients to receive consistent and reliable access to treatment and care.

In particular, Bayer will donate 50 million IU of factor VIII treatment in 2019. Factor VIII treatment donation will support up to 5,000 people living with hemophilia A in countries with insufficient access to care.

I’ve been to over 30 developing countries, some of them up to four times, and know many patients. I am thrilled to hear this news; it’s bold, it’s needed and it’s time. People with hemophilia continue to die. We can prevent that, and give them a chance at life, which they so deserve. Kudos, Bayer!

Footnote: In 1996, a rep from Bayer approached me to create and run a leadership program in developing countries to encourage parents and patients to start their own nonprofits and advocate for change. Project SHARE grew out of this program, and we continue today independently to provide leadership training, financial assistance and product.

Basil Rathbone Hemophilia Celebrity Spokesperson

Laurie Kelley May 19, 2019

By Richard J. Atwood

originally published in PEN, February 2019

Who would you pick as a celebrity spokesperson for our bleeding disorder community?

Celebrity status automatically captures the public’s attention. And our community wants to be noticed—for public awareness, for outreach, and just for the pleasure of being recognized. Soon after its founding in 1948, National Hemophilia Foundation (NHF) chose actor Basil Rathbone as its celebrity spokesperson. From the beginning, NHF wanted to be recognized. But why Rathbone?

Basil Rathbone (1892–1967) may be familiar to you, especially if you enjoy older movies. At one time, Rathbone was very well known, most famously as Sherlock Holmes. He appeared in 83 films from 1921 to 1967. Plus, he performed in the theater, on the radio, and for live television. Born in South Africa and raised in England, Rathbone began his career in 1913 as a classically trained Shakespearean stage actor. He saw combat in Europe during World War I.

In 1921, Rathbone debuted in his first silent film. He then moved to America. Rathbone kept his English citizenship, and he took frequent trips to London. Eventually, Rathbone wanted to return to his first love—acting onstage. In 1947 he left Hollywood for New York City, where NHF had its offices. So it made sense for NHF to ask Rathbone to be a celebrity spokesperson.

In the early 1950s, Rathbone was at the height of his acting career on stage, screen, and radio. He could sing and dance, and was a skilled swordsman. He did paid advertising for cigarettes, liquor, insurance, and diet food. Amazingly, he played 52 roles in 23 plays by Shakespeare. Over seven years starting in 1939, Rathbone starred in the role of detective Sherlock Holmes in 16 films and 275 radio episodes. Rathbone was rewarded with two Academy Award nominations. His popularity would peak again later in the 1960s, when his Sherlock Holmes films were broadcast on television.

Despite his wide range of roles on stage and screen, Rathbone is mostly remembered, regrettably, as a type-cast villain. His villainous roles include Mr. Murdstone in David Copperfield; the Marquis St. Evrémonde in A Tale of Two Cities; Captain Esteban Pasquale in The Mark of Zorro; and probably his most famous role, Sir Guy of Gisbourne in The Adventures of Robin Hood.

By coincidence, Rathbone performed in two movies that included hemophilia. He portrayed Prime Minister Disraeli in the 1961 television production of Laurence Housman’s Victoria Regina on NBC’s Hallmark Hall of Fame program. Julie Harris portrayed Queen Victoria, a known hemophilia carrier. Unfortunately, Housman’s play, originally staged in 1936, includes some inaccuracies about hemophilia genetics.

In 1966, Roger Corman, a producer at American International, hired Rathbone for the film Queen of Blood (Planet of Blood). Rathbone played Dr. Farraday, who, in 1990, sends a team of astronauts to Mars to rescue a crashed spacecraft. The astronauts return with a survivor—a green woman named Velana. The astronauts quickly learn that Velana is a vampire who kills her captors, and that she also has hemophilia.

Velana bleeds to death from a scratch, leaving behind her eggs. I admit, this is one of my all-time favorite science fiction/horror flicks, originally shown at drive-in theaters. NHF chose Basil Rathbone as a celebrity spokesperson for only a short time. We don’t know whether he had a family connection with hemophilia. Although he performed in two movies that include hemophilia,

Rathbone was probably more concerned with earning a salary than promoting a cause. Yet we can always be grateful for any celebrity spokesperson.

Who would you choose to speak for our community today?

For more info, read Rathbone’s autobiography, In and Out of Character (1962); and Michael B. Druxman’s biography/filmography, Basil Rathbone: His Life and His Films (1975).

For more articles like this check out Richard’s Review, published in every issue of PEN!

Uncategorized

When to Suspect an Inhibitor

The Joy of Giving

Bayer Teams Up with WFH to Provide Aid

Basil Rathbone Hemophilia Celebrity Spokesperson

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