Ray Greenidge and Erica Worrell of the Barbados (in red) with Laurie Kelley and Salome Mekhuzla (WFH)
No man is an island,
Entire of itself,
Every man is a piece of the continent, A part of the main. —English poet John Donne
I stepped off the plane in Kingston, Jamaica on Thursday evening and the sultry, warm air enveloped me like a thick blanket. This tropical island has great music, savory food, friendly people, world-class resorts, a raging drug problem… and hemophilia. I was here this past weekend to speak at the first-ever conference for the English-speaking countries of the Caribbean. The Jamaica Haemophilia Committee hosted this landmark World Federation of Hemophilia conference.
Luisa Durante, WFH
I’ve been to Jamaica twice before to help the patients organize, and numerous times over the past 20 years to a few other English-speaking countries here, including Barbados, Bahamas, and St. Vincent and the Grenadines. All were represented here, at long last.
The emerging theme of the conference became isolation, and how
to break it. The poetry of John Donne expresses this so beautifully: so many people with hemophilia feel isolated, as if they are on an island, cut off from the world. Indeed, these people really are on islands! Our challenge is to unite patients and families with one another in their own countries, and then link and connect country with other countries, to learn from one another and to support each other. To make a hemophilia continent, and to reduce isolation.
Topics of the two day conference included: clinical management of hemophilia, outreach to community members, genetics, hemophilia in the Caribbean, WFH programs, comprehensive care, the role of nursing, physiotherapy, and patient involvement.
Dr. Jackie Bird, St. Lucia
Who attended? Erica Worrell from Barbados, mother of a child with hemophilia, who just started a new society there in April and already held a successful walk to raise awareness; Brian and Ritchie Bardalez of Belize, young men who I have known since they were children, as I visited
Belize twice back in the early 2000s (they are now taking a leadership role);
Issa from Trinidad and Tobago, a patient and fiery orator, all my colleagues
and friends from Jamaica (Denton, Alex, Kirt, Berverly, Kerry-Ann, Milton,
Tamaicka, Sharon, Gricell, and the medical staff of UWI), Laurence Bakhsh, a brave young man from Guyana who we have helped for years with factor donations—first time meeting him!
The challenges are vast: Erica shared how there is no national registry of patients in Barbados, and how difficult it is to get factor. While the country buys some—a miracle in itself—you must get a prescription (wait time long), then go to the pharmacy (wait time even longer). You must pay for all ancillaries, right down to butterfly needles, which often are not even available! And tourniquets? Forget it. (We promised to ship her some ASAP.) Ritchie pointed out that Belize has closer ties with the Caribbean than with Central America, where he is located. There are only 16 known patients in Belize, and the terrain is the opposite of flat Barbados; it is mountainous with poor roads. A plane—“puddle jumper”—is the safest way to travel and distribute factor. 56% of the patients do home therapy, which spares them from expensive travel to clinics. There’s no hematologist—anywhere.
Bardalez brothers (Belize) with Valentino (Suriname), Dr. Eric (Belize) and Laurence Bahkesh (Guyana)
There’s also no hematologist in St. Lucia, reported Dr. Jackie Bird, a 58-year-old dynamo who seems to have single-handedly taken on care for all people with hemophilia on this volcanic island. Poverty is 28%, and there are only 6 known patients—all with factor IX deficiency! Factor is not available and never has been.
That made me sit up. Could we be the first to ship factor IX concentrate to St. Lucia? Jackie’s knees buckled, as she made a prayer sign with her hands, smiled and shouted, “Yes, thank you!” The room exploded in applause. This is the magic of these meetings: putting people together, those with and without, sharing honestly, and finding solutions. Yeah for us!
Patient Ray Greenidge, vp, and president Erica Worrell, Barbados Haemophilia Association and Charity
Laurence, a 33-year-old from Guyana, just founded a hemophilia society in 2015 and is struggling. After all, he has limited mobility, limited funding (he cannot work), and there are only 5 known people with hemophilia, including his brother and cousin. Guyana’s population of 755,000 means there are about 75 people with hemophilia. He has his work cut out for him. But with his natural charm and humility, he quickly became a favorite of all, and with his new network of colleagues, is bound to make improvements soon. “Strive to stay alive” is the organization’s tagline.
Laurie Kelley with Laurence Bakhsh (Guyana)
The Bahamas was represented by Florence Roker, mother of 21-year-old Chavez, who I met years ago. She has now stepped forward
to grab the reins of the floundering national organization. She broke into tears describing how Chavez has suffered: “You physically feel their pain,” she sobbed. Now, she said, regaining her composure, there are other children who suffer and who need our help. You have to find the silver lining behind the clouds. “This is a passion for me,” she concluded. “I’m motivated to get the organization to where it should be.”
Agent of change: Florence Roker of the Bahamas
An empowering and passionate speech was given by Issa, chair of the Society for Inherited and Severe Blood Disorders Trinidad and Tobago, an association for those with hemophilia, sickle cell and thalassemia. This is a brilliant strategy, to link up the three blood disorders to gain strength and have a stronger voice. And oh boy, does Issa have a strong voice! Motivating us and making us laugh, he stirred the audience emotionally with obvious leadership skills. T&T has been a WFH national member organization for 25 years, making it the oldest organization for hemophilia in the Caribbean. Issa declared that in the 1980s, West Indies cricket dominated the world. “Dominated!” he shouted. “Dominated! It happened when we united as one! We conquered the world!’ The audience laughed and applauded. Comparing this to diseases and disorders, T&T united as well. But unlike the West Indies cricket team, which had its glory days in the 1980s, “we are still a force to be reckoned with!” Issa declared.
But the most quoted person these two days was Jamaican hemophilia patient Tevon Brown, who stressed how isolated he felt growing up, as if he were the only person with hemophilia. He suffered terribly with pain, which felt like an “electric lead to my heart.” Strong role models and good teachers helped him. And he stressed now that we must unite and bring those who are isolated together, including nations. “We must have stick-to-it-iveness!”
he announced.
I must thank the World Federation of Hemophilia for organizing this conference, funding it, and inviting me, which allowed me to invite so many of the people I’ve met over the past 20 years. Kudos to Luisa Durante, regional manager of the WFH for Latin America, who is as hard working as she is fun as she is beloved by the people she serves. She and her team (Salome and Felipe) provided a magical two days. The WFH does great work, and we are
proud to support them and volunteer when possible. It takes stick-to-it-iveness to make long term changes in hemophilia care, and this is how it happens. Unity, hard work, passion.
In this week’s blog, we hear a personal story from a young
man with hemophilia A, about how his self-perception changes throughout his
life, and how finding the right treatment made such a difference in the person he is
today.
Indications
ELOCTATE is an injectable medicine that is used to help control and prevent bleeding in people with Hemophilia A (congenital Factor VIII deficiency). Your healthcare provider may give you ELOCTATE when you have surgery.
Selected Important Safety Information Do not use ELOCTATE if you have had an allergic reaction to it in the past.
It’s really difficult to find the words to describe living with hemophilia—not only because emotions and words are two completely different animals, but also because meeting new people under the premise of “Hey, I’m Laith, and I have hemophilia!” feels foreign to me.
I was diagnosed with hemophilia A after bleeding for 10 days after birth. For a time, when I was growing up, hemophilia was the coolest thing in the world—it was fascinating. I loved tagging along with my parents to the conferences and meeting other people like me. My dad drilled some strange love for science into my head early on, so the biology of it all was super attractive in my eyes. No time was wasted: I talked to my doctor and I started to self-infuse at the age of 10.
But right around the time I entered 5th grade, things really started to change. My hemophilia treatment center sent letters to my school administrators, who naturally put every teacher in the school on high alert. Something that I had thought was so cool about me suddenly became taboo. When I got to middle school, I ostracized myself. I faded into the background with my hemophilia and preferred invisibility.
The role hemophilia has played in my life became two things for me: steady and secretive.
In high school, I decided to take this opportunity to choose who I wanted to be and became “Laith without hemophilia.” My approach to dealing with my bleeding disorder in high school gave me more in common with a stealthy secret agent than I care to admit. Self-induced solitude was my friend, and I had every intention to master it as an art. “Laith without hemophilia” didn’t need to obey his prophylaxis infusion schedule. It didn’t matter how swollen my arm was, I was going to wait until I was either at home or hidden in some part of the school to infuse.
Abrupt abandonment of friendships, unexplained absences, outrageously convoluted excuses, and unreturned phone calls proudly became my hallmarks.
But, I had awesome physicians my entire life. My hematologist was persistent, justifiably so, in encouraging me to stay on schedule with my infusions. Then I heard about ELOCTATE. It may be due to my lifetime interest in the science of hemophilia, but the prolonged half-life of ELOCTATE piqued my childhood curiosity. My doctor, clever guy that he is, picked up on this. We talked and decided on ELOCTATE as a treatment option because he thought the every-four-days infusion schedule might work for me. A MyELOCTATE™ Coordinator worked with me to make sure my transition to ELOCTATE went smoothly and supplied me with medication while my insurance worked to approve it. After working with my doctor, I’ve settled into the routine of infusing every four days*. I have been able to stay on track. I schedule my infusions ahead of time, and, for the most part, I’ve kept up with them.
No two people are the same, right? Now at 24, what I’ve discovered about people in general is that to secure a place and purpose in life, one must take ownership. Being that hemophilia has been my Achilles heel in this regard, I’m treating it as a priority. I figure only then can I discover who I really am. I’m not “Laith without hemophilia.” I’m not even “Laith with hemophilia.” Hemophilia is one part of the many parts that make me whole.
This is my story; peeling back my layers one at a time and showing others that it is possible to discover who they really are.
Talk to your healthcare provider about whether ELOCTATE may be right for you.
*The recommended starting regimen is 50 IU/kg of ELOCTATE administered every 4 days. Adjust the regimen based on patient response with dosing in the range of 25-65 IU/kg at 3-5 day intervals.
For children <6 years of age, the recommended starting regimen is 50 IU/kg of ELOCTATE administered twice weekly. Adjust the regimen based on patient response with dosing in the range of 25-65 IU/kg at 3-5 day intervals. More frequent or higher doses up to 80 IU/kg may be required.
Important Safety Information
Do not use ELOCTATE if you have had an allergic reaction to it in the past.
Tell your healthcare provider if you have or have had any medical problems, take any medicines, including prescription and non-prescription medicines, supplements, or herbal medicines, have any allergies, are breastfeeding, are pregnant or planning to become pregnant, or have been told you have inhibitors (antibodies) to Factor VIII.
Allergic reactions may occur with ELOCTATE. Call your healthcare provider or get emergency treatment right away if you have any of the following symptoms: difficulty breathing, chest tightness, swelling of the face, rash, or hives.
Your body can also make antibodies called, “inhibitors,” against ELOCTATE, which may stop ELOCTATE from working properly.
The most frequently occurring side effects of ELOCTATE are headache, rash, joint pain, muscle pain and general discomfort. These are not all the possible side effects of ELOCTATE. Talk to your healthcare provider right away about any side effect that bothers you or that does not go away, and if bleeding is not controlled after using ELOCTATE.
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.
ELO-US-1092 10/2016
This blog was sponsored by Biogen for educational purposes.
This
week, I’m excited to tell you about a new company made of up familiar faces within
the hemophilia B community. The people from Emergent who brought you IXINITY [coagulation
factor IX (recombinant)] are now continuing their tradition of being a different kind of company at Aptevo
Therapeutics.
Continue reading for more
information about how Aptevo is still committed to putting people with
hemophilia B first.
Introducing
Aptevo Therapeutics
Aptevo
Therapeutics is a new biotech company focused on improving the lives of people
with bleeding disorders and cancer by combining extensive experience with the
drive to advance the science of what’s next.
By
connecting with people across the country to learn about their needs and continuing
to support community outreach programs that enrich peoples’ lives, our primary goal
is to provide high-quality, specialized therapies for people with rare
conditions.
When
it comes to the hemophilia B community, the same people from Emergent who pride
themselves on working for a different kind of company are now part of Aptevo.
So
while you may see a new business card, IXINITY is still the same, and you’ll
still connect with the same people you’ve come to trust.
Visit IXINITY.com to find out more about this
factor IX and the company behind it.
The content of
this post is provided and sponsored by Aptevo Therapeutics.
IXINITY INDICATIONS
AND IMPORTANT SAFETY INFORMATION
What is IXINITY®?
IXINITY [coagulation factor IX (recombinant)] is a medicine used to
replace clotting factor (factor IX) that is missing in adults and children at
least 12 years of age with hemophilia B.
Hemophilia B
is also called congenital factor IX deficiency or Christmas disease. Hemophilia
B is an inherited bleeding disorder that prevents clotting. Your healthcare
provider may give you IXINITY to control and prevent bleeding episodes or when
you have surgery.
IXINITY is
not indicated for induction of immune tolerance in patients with hemophilia B.
IMPORTANT SAFETY INFORMATION for IXINITY®
·
You should not use IXINITY if you are
allergic to hamsters or any ingredients in IXINITY.
·
You should tell your healthcare provider if
you have or have had medical problems, take any medicines, including
prescription and non-prescription medicines, such as over-the-counter
medicines, supplements, or herbal remedies, have any allergies, including
allergies to hamsters, are nursing, are pregnant or planning to become
pregnant, or have been told that you have inhibitors to factor IX.
·
You can experience an allergic reaction to
IXINITY. Contact your healthcare provider or get emergency treatment right away
if you develop a rash or hives, itching, tightness of the throat, chest pain,
or tightness, difficulty breathing, lightheadedness, dizziness, nausea, or
fainting.
·
Your body may form inhibitors to IXINITY. An
inhibitor is part of the body’s defense system. If you develop inhibitors, it
may prevent IXINITY from working properly. Consult with your healthcare
provider to make sure you are carefully monitored with blood tests for
development of inhibitors to IXINITY.
·
If you have risk factors for developing blood
clots, the use of IXINITY may increase the risk of abnormal blood clots.
·
Call your healthcare provider right away
about any side effects that bother you or do not go away, or if your bleeding
does not stop after taking IXINITY.
·
The most common side effect that was reported with IXINITY during
clinical trials was headache.
· These are
not all the side effects possible with IXINITY. You can ask your healthcare
provider for information that is written for healthcare professionals.
You are encouraged to report side effects of prescription drugs to the
Food and Drug Administration. Visit the www.fda.gov/medwatch, or call
1-800-FDA-1088.
Aptevo BioTherapeutics LLC, Berwyn, PA
19312
IXINITY [coagulation factor IX (recombinant)] and any and all Aptevo BioTherapeutics LLC brand, product, service and feature names, logos, and
slogans are trademarks or registered trademarks of Aptevo
BioTherapeutics LLC in the United States and/or
other countries.
It was a chilly and moist evening in
New York City on September 29, perfect for a fall kick-off and a good reason to
warm up with friends, colleagues and a celebrity to honor 15 years of Save One
Life.
Save One Life was founded in 2001, but
my idea to start a nonprofit to help the world’s children with hemophilia who
live in poverty had been kicking around for at least two years. We are now up
to over 1,500 served over 15 years, and we have great plans to expand our help even further.
It was fitting to hold our celebration
in New York; to give credit where it is due, it was a New York mother of a
child with hemophilia, Lisa O’Connor, who wrote to me back in 1999, on a piece
of yellow-lined paper (yes, people did that at one time!) to suggest we start a
Save the Children-like program. I loved the idea, and the letter coincided with
a trip to Pakistan, in which I sat in the home of an extremely poor family:
two rooms, no kitchen, no plumbing, no electricity, two small children with
hemophilia. The father simply wanted extra money each month to send his son to
a good school, where he would be protected, and learn English, to get a good
job. Then he could buy factor. The cost? $20 a month, he told me. The idea
became a plan.
This evening, so many of our sponsors
and colleagues came out to share our 15 years. I saw so many long term
community friends, some going back 20 years!
Cyclist Barry Haarde
Held at the Manhattan Penthouse, high
above the bustling city below, we gathered to have a lovely dinner, distribute
awards and honor our volunteers and supporters, and to welcome two very special
guests.
First, no surprise, we honored yet
again the amazing Barry Haarde, for his fifth cross-country cycling odyssey to
raise funds for Save One Life. The 2016 journey took two months this time, and
saw Barry traverse the US from west coast to east coast, and then from north to
south! I managed to hop off Kilimanjaro in time to fly to Key West, the most
southern point in the US, to welcome Barry on Route 1, mile 0. Barry takes it
all in stride, but his efforts are Herculean. He has raised awareness as well
as over $200,000 for Save One Life over his five trips, and sponsors a child
with hemophilia from India.
Usha Parthasarathy received our
Inspiration Award. Usha is a dear friend and also volunteer and expert advisor
on hemophilia in India since Save One Life’s formation in 2001. Usha helped
Save One Life to organize and launch its sponsorship program in India. In 2009
Usha joined Save One Life as India program coordinator, assisting with growing
and managing our largest country, with 25 program partners and more than 830
current and graduated beneficiaries. She works tirelessly in a completely
volunteer role. Usha lost both her father to an intracranial bleed and her baby
within two years. She left a career as a national newscaster in India to devote
her life to helping those with hemophilia, and became development vice
president for the Hemophilia Federation (India). Her second son, Sudharsan, born
in 1981, is now a post doctoral researcher in the hematology division of
Children’s Hospital of Philadelphia.
Not present, but honored, was board
member, friend and fellow-mountain climber Eric Hill, COO of Diplomat, who
sponsors 31 children, and has organized our mountain climbs to raise awareness
and money for Save One Life. His efforts have raised over $150,000 for save One
Life and Eric recently has made the first donation to our endowment fund.
Miahi of Romania
After dinner, we were entertained by
Mihai, a lovely young man with hemophilia from Romania. He and his mother flew
all that way to attend, as our guests. Handsome, slight of build and tall, he
was introduced by my long-time friend and mentor Adriana Henderson of North
Carolina (Romanian by birth) who has devoted her life to helping the poor and
suffering in Romania with hemophilia. She explained how Mihai excelled at
playing the piano, despite the limited resources of his family, and frequent
bleeds in his hands. Mihai played several beautiful classical pieces, and
ended with Chopin’s Nocturne Op. 9 No. 2, one of my favorites.
Best of all, Mihai’s sponsor, Reid Coleman of North Carolina, attended and was able to meet his beneficiary of many years!
The final presentation of the evening
belonged to Tony Fernández,
former shortstop for the Toronto Bluejays, and for one year, the New York
Yankees. Tony has been a long time friend to the
hemophilia community in the Dominican Republic. His Toronto Bluejays teammate
and best friend, Damaso García, has a son with hemophilia. When Damaso’s wife
Haydée De García Benoit, founder and president of Fundación
Apoyo Al Hemofílico (FAHEM), the DR’s national
hemophilia organization, asked Tony to attend their first camp in 1999, Tony
gladly said yes. The children were thrilled to have a national hero spend the
day playing baseball with them. He has since attended three camps, now called
Yo sí Puedo! (Yes I Can!) and one of the best hemophilia camps in the world!
FAHEM has since become a Save One Life
program partner.
Tony has had a distinguished Major
League Baseball career, starting in September 1983. He signed on to six teams,
setting a nine-year record for shortstops. He was awarded four consecutive Gold
Glove Awards, from 1986 to 1989, and was also named to five All-Star teams. Today
Tony is an ordained minister and founder of the Tony Fernández Foundation,
which works with underprivileged and troubled children by nurturing and developing
them through counseling, education, training, and physical and spiritual
activities.
Tony shared how Damaso Jr. felt
defeated early in life; his father was a star player, but he could not even
play baseball. Tony counseled him and encouraged him to find some other way to
participate other than actually playing. The encouragement took hold. Today
Damaso is a national sportscaster for the Dominican Republic and the entire
nation tunes in to him at 5 pm daily.
Tony reminded the audience that to give is to receive, and that the world needs
our help in these troubled times. He urged everyone to pick up the envelop in
front of them, and to give. His authority, his devotion to children, made an
impact. We raised over $4,500 right after his speech, with 19 more children
pledge for sponsorship.
Tony Fernández
The next day our team met at KYNE
offices, with David Kyne, president and founder, and Save One Life board
member, for a seven hour strategy meeting. The next 15 years will see tremendous
growth of Save One Life, and many more children reached, and lives changed.
“If
we stay the course, the unthinkable might happen, yes, together we can bring
hope to the hopeless, just be faithful to the vision given to you by God and
He will find a way to bring it to pass.” Tony Fernández, major league
ballplayer, minister, founder of The Tony Fernández Foundation
Thanks to Save One Life staff Martha Hopewell and Tricia Sico for organizing the event; and to our sponsors and donors, and all attendees. Please see www.saveonelife.net for more information or to sponsor a child with a bleeding disorder in a developing country.
I’m preparing for a visit to Russia next
week, a country I have not been to since 1999. So I’ve been reading two
excellent books to prepare: Peter the Great and Nicolas and Alexandra, both by
Pulitzer-prize winning author Robert K. Massie.
These books, and last week’s post on
hemophilia myths, got me thinking: Do you know who the most famous carrier of
hemophilia was? Hint: It’s why hemophilia is dubbed “The Royal Disease.”
Queen
Victoria of England, one of England’s longest reigning monarch. Her 63-year reign became known as the Victorian era, and was the longest reign until
September 9, 2015, when Queen Elizabeth II surpassed her. Her era saw British
power at its zenith across the globe; Victoria believed that the British Empire
existed to civilize people in less developed countries and to protect them from
their own rulers and the aggression of neighboring rulers. Others saw the
purely commercial aspect of this world domination of lands and trade routes.
Victoria took the throne at age 18, and later married
her first cousin, Prince Albert of Saxe-Coburg and Gotha, in 1840. Over the
next seventeen years, she and Albert had nine children: Albert
Edward, (b. 1841), Alice (b. 1843), Alfred (b. 1844), Helena (b. 1846), Louise (b. 1848), Arthur (b. 1850), Leopold (b. 1853) and Beatrice (b. 1857).
The last child, Beatrice, was delivered under the care of Dr. John Snow, who
later became the founder of modern public health by discovering the
transmission mechanism of cholera (infected water). Snow used anesthesia on
Victoria, thus giving her pain-free childbirth for the first time! While Snow
did not invent anesthesia, he created a more convenient and safer way to administer
it.
When their nine children married into royal and noble families throughout Europe (for mostly
political reasons), Victoria was called “the grandmother of Europe,”
and indeed was called “Granny” by all her grandchildren and their spouses.
Hemophilia was not known to exist in the
royal family before, but Victoria carried the gene for hemophilia B. Only
Leopold had hemophilia; two daughters, Beatrice and Alice, were carriers. They
later transmitted hemophilia to the Spanish and Russian royal families.
Leopold grew up to be a tall, intelligent,
affectionate yet stubborn prince, whose willfulness often led to injuries and
bleeds, according to author Massie. The Queen was unusually attached to her
son, and worried over him incessantly. Victoria reported in one letter that
Leopold had been at death’s door four or five times. Eventually Victoria tried
to keep him confined to the upper floors of Buckingham Palace for his own
protection, even as a man! But he managed to get away to Paris for two weeks.
Eventually at age 29, he fell in love with a German princess, Helen of Waldeck.
They had a daughter. When Helen was pregnant a second time, Leopold fell,
suffered a brain hemorrhage and died at age 31.
There was no treatment for hemophilia then, of course. Victoria was informed by telegram that her youngest son had died in Cannes. He was “the dearest of my dear
sons,” she lamented.
According to one of her
biographers, Victoria wrote an average of 2,500 words a day during her adult
life, and kept a detailed journal, which eventually encompassed 122 volumes. From this we
learned a bit about hemophilia in those days. The
Queen didn’t know what type of hemophilia her son had or if there even were
types; only much later, through genetic testing, was the family found to have
hemophilia B. While the mechanisms weren’t entirely understood, the royal
family knew that hemophilia could be passed down from generation to generation.
And it was; when it hit the Russian royal family, it would change the world.
For more information, read Robert K. Massie’s excellent books, or view “Nicolas and Alexandra,” a major motion picture and excellent portrayal of the last years of the Russian monarchy. Available on Amazon.com. Robert K. Massie also has an adult son, Robert K. Massie, Jr., who was later cured of his hemophilia through a liver transplant. His life is exquisitely portrayed in the book Journey.
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It was a chilly and moist evening in
It was fitting to hold our celebration
