Management of Hemophilia with No or Limited Factor

 Greetings from Down Under!
I’m attending the World Federation of Hemophilia Congress in Melbourne, Australia. I’ve been attending these since 1996, when it seemed I knew everyone, and now, with 4,000 attending from 128
countries, it’s at once hard to see familiar faces, and yet, that’s a positive side effect of the success of the WFH’s outreach to so many patients, physicians and partner organizations globally.
Aung from Myanmar: The face of
hemophilia in the Third World
The opening plenary yesterday, led by
WFH president Alain Weill, welcomed everyone, and provided some surprises.
My concern is always access to product for those in developing countries. Indeed, in the opening ceremony Sunday night, Weill announced something to the effect that this was a great time in hemophilia, a great era. Treatment for all! That’s the slogan. Well, yes and no. Not if you’re a child in the heart of Africa.
But there was great news at the plenary Monday morning. Weill said that since 1996 WFH has distributed 248 million IUs of product in 86 countries, helping about 90,000 people with bleeding disorders. (I’m proud to note that Project SHARE, a program run by two people, me and Zoraida, has distributed since 2002 about 60 million IU to about 55 countries) Weill announced some amazing product donation pledges: Biogen Idec and Sobi have agreed to donate 500 million IUs of factor over the next five years to WFH’s Humanitarian Aid Program. Grifols committed to 60 million IUs over a three-year period. And the Italian Centro Nazionale Sangue (CNS) announced donations of up to 30 million IUs of factor VIII per year over a five-year period.
As the Soci CEO said, we want to stop thinking of donating product at the end of the day, and make it the first thing we think about in the morning. In other words, this will allow WFH to plan strategically how to use product donations to improve care, rather than leaving it to act in crisis mode (which is often how Project SHARE operates, and it’s difficult to operate like that). So the donations will be charity used with a strategic purpose to enhance development. Awesome.
Alain Weill and Dr. Gabriele from CNS Italy 
I attended an interesting meeting called the Management of Hemophilia with No or Limited CFCs, with Carlos Gaitan Fitch of Mexico as facilitator.
Four speakers presented how they manage hemophilia with little or no factor.
Ricarte Felipe, new president of HAPLOS of the Philippines, age 50, reviewed his life growing up without factor. He spoke from a wheelchair that day, and from which he was confined for 3-7 years of his young life. He was diagnosed at age 8 with hemophilia A. At age 12 lost his left eyesight in an accident. Yet he managed to get a college diploma despite the challenges. Only in 1986 was he started to be infused with plasma and cryo. In 1988
he received his first infusion of factor due to a tooth extraction. What a survivor!
He shared that factor in the Philippines is $100 for a 300 IU vial. And while in 2010-2013, he was treated mostly with CFC (clotting factor concentrate), factor is not available much in the Philippines. The government does not buy it. And it is so expensive, most families cannot afford it.
So how do Filipinos manage? Support from HAPLOS consists of psychosocial workshops, camps, workshops, and “Mass healing,” where attendees at church pray for the patients.
What was sadly missing from this touching account was this: what is HAPLOS doing to secure factor, to change the destiny of the children being born right now with hemophilia in the Philippines? It’s difficult to listen to the suffering, especially from a leader, and not hear about what is that organization going to do to change this.
Dr. Gilian Wharfe, University of West Indies, Jamaica, also presented. I’ve known Dr. Wharfe for many years and have visited Jamaica twice
to meet with her and the patients. She clearly laid out the issues facing Jamaica: high unemployment, stagnant economy, inflation, and factor costing $50 per unit, beyond what parents can pay (we know; they contact us regularly), makes it impossible for the government to consider putting factor in the federal budget. The only access to factor is from donations.

Laurie Kelley with long time friends Massod Fareed Malik and Deon York, each presidents of their country’s hemophilia organizaiton (Pakistan, New Zealand)
With approximately 200 patients, and only one HTC at UHWI (which is not a public hospital; the patients must pay), one national blood
transfusion center and  several  collection centers, Jamaica struggles to provide care to hemophilia patients. The stopgap measures for care include: prevention of bleeds, early treatment of bleeds (How can you do that with limited access to factor, distances to travel, long waits for cryo or FFP to be thawed, etc?) and educating patients about how to avoid bleeds and recognize bleeds early.
It was a rather bleak assessment, and a classic example of life in the developing world with hemophilia. You have to admire the physicians like Dr.
Wharfe who must practice under these conditions.
Dr. Daniel Hart, hematologist at Barts and The London School of Medicine, spoke about his experiences twinning with Bangladesh and The Royal London Hospital Haemophilia Centre. It was an excellent summary about how twinning can help slowly transform a country. Twinning means the medical center in a developed country pairs with one in a developing to share best practices and hopefully, to help resource the less developed center.

Aussies Steve Russell and Fred Wensing help Aung of Myanmar to attend the Congress and buy a wheelchair to take home
Dr. Hart described how Bangladesh uses “.001 IU per capita,” compared to 5.8 IU per capita in Australia.  Numbers like these give hard, cold facts to global factor disparity. He covered a lot of ground in his talk, but what stuck out were two things:
1. Diagnostic accuracy is fundamental, as well as access to expertise, advocacy (really important—hitting up the government, emergency
centers, pushing the national hemophilia standards everywhere), and patient and physician education.
2. Perspective: Hart showed an old trailer in an old photo, and said this was how the treatment center in London, now a world class center,
started. In just an old trailer. His photos progressed to a high-end sports car!

So conditions now do not have to persist over time. Treatment can improve. But… and the Philippines should heed this… you cannot get anywhere if
you don’t fix up the trailer. Advocacy is a must—pushing the national agenda, having a strategy, and first knowing what you want.
And everyone wants factor.

This Not-So-Royal Disease

by Andrea Trinidad-Echavez

This week, I am posting a wonderful article by colleague and friend Andrea, from the Philippines. I was privileged to travel with her for one week. We both met Jeffrey and his mother Rita.

Every year, Rita Rodriguez, 54, would religiously light a candle in the early mornings of All Saints Day in remembrance of her four sons– Noel, Ricky, Materno and Antonio — who died one after the other in a span of two decades.

Cursed, was how people from her hometown of Tanjay in Negros Oriental, thought of Manang Rita. Little did they know that a rare hereditary bleeding disorder called hemophilia was the culprit behind their deaths.

“I would have wanted to go home to Negros to personally visit their graves but life is hard,” said the 54-year-old mother of eight, who has been struggling to keep another son Jeffrey, from falling into the same fate as his older brothers. In between Jeffrey’s confinements, Manang Rita would make ends meet by offering services as masseuse or cleaning houses and doing laundries.

Jeffrey, 25, a psychology student in Adamson University, was diagnosed with hemophilia when he was barely three, following a supposedly minor injury on the head that caused profuse bleeding. By then, two of Jeffrey’s older siblings had already succumbed to strange internal bleeding episodes.

Manang Rita remembered her older brother, Luis Torres, would also profusely bleed from the smallest injuries. But they did not know that hemophilia runs in their family.

Manang Rita’s oldest child Noel died in the early ’60s from an unexplained ailment. He was only three years old. Noel bumped himself while playing. Shortly after, he developed a tumor-like lesion that wouldn’t go away. He died a few days later.

Another son, Ricky, died in the ’70s at the age of 5. He also died of strange bleeding after getting minor injuries from playing.

In both deaths, Manang Rita hardly understood what happened to her boys. She could only grieve over the loss and hoped well for her other children.

But her reprieve from grief ended when in 1989, Materno, then 25, died after days of suffering from severe headache.

“We gave him pain relievers and put ice on his head. But when it refused to go away, we decided to bring him to the hospital,” Manang Rita said in mixed Cebuano and Tagalog. By then, local doctors already knew the name of the strange disorder that plagued the boys in Manang Rita’s family.

“I was told the boys have hemophilia. It was only then that we became aware of the name of the bleeding disorder in our family,” Manang Rita shared, her voice quivering in between.

Doctors gave Materno a blood transfusion to replenish the blood lost in the internal bleeding. He was also given a dose of the expensive Factor 8 concentrates derived from human plasma that would instantaneously stop the bleeding.

But by that time, it was too late to avert Materno’s condition. He died in the hospital.

In 1997, another son, Antonio, complained of severe stomach pains. He urinated blood, causing her mother to be concerned. Unknown to Manang Rita, Antonio, then 26, fell off from a tricycle. Though he did not sustain any open wound, Antonio’s injury caused internal bleeding which lead to his death in less than one week.

“People thought she was cursed because of the deaths of her four sons,” said Rizalina Magalso, a cousin of Manang Rita and herself a mother of 13-year-old Dan Louie, also a hemophiliac.

In 1999, Manang Rita’s youngest son, Jeffrey sustained injuries from a fall and again suffered excessive bleeding. By then, with four sons dying of bleeding, Manang Rita was already on the verge of despair.

“I told the doctors I could no longer bear it if something bad happened to Jeffrey,” said Manang Rita.

The doctors sympathized with her and advised her to bring Jeffrey immediately to Manila and seek help at the Philippine General Hospital.

She sold all her family’s little landholdings if only to save her youngest son and admitted him at the PGH. After several bags of blood transfusion and over a month of stay at the hospital, Jeffrey became well enough.

But the deaths of her four sons led Manang Rita to decide to stay in Manila for good, where Jeffrey has better chances of surviving.

Called the “royal disease,” hemophilia is a hereditary bleeding disorder that affects the males of the family. Mothers are usually the carriers and they pass on the defective genes to their male offspring.

Several members of noble families in Europe were affected by it, thus the monicker “royal disease”. Written accounts identified Queen Victoria as the first royal carrier of the disorder, with her eighth child Leopold, born in 1853 as the first recorded royalty with the disease. Leopold died at the age of 31 from an intra-cerebral hemorrhage after a fall.

Two of Queen Victoria’s own daughters, Alice and Beatrice, were also carriers of hemophilia. The condition was transmitted through them to several royal families in Europe, including Spain and Russia. The most famous affected individual was the son of Tsar Nicholas II of Russia, Tsarevich Alexis, who was born in 1904. There were speculations that the illness led to severe strain within the royal family, and enabled Rasputin to gain influence over the family, ultimately leading to the downfall of the once powerful dynasty.

The WFH estimates that .01 percent of the population or about 1 in 13,000 are affected by hemophilia.

In the Philippines, the Hemophilia Association of the Philippines for Love & Service (HAPLOS) said about 6,000 to 8,000 could be affected by the disorder. However, only a little over 1,000 have been registered. Most of the sufferers come from the same families.

Thanks to modern technology, hemophiliacs like Jeffrey may soon lead normal lives with the availability of factor concentrates that can instantaneously stop bleeding. The medicines, however, are very expensive and are still unaffordable for people like Manang Rita, who barely manages to survive with her meager income as a masseuse.

“Even though life is hard here, we will endure if only for the sake of Jeffrey. I am thankful to God that despite everything, He has helped us survive this ordeal,” said Manang Rita.

Great Book I Just Read
Daughter of the Desert, by Georgina Howard

If I tried to describe Gertrude Bell to you, you might think I was making it up. But this is a true story about one of the world’s most amazing women. Born into a privileged English family in 1868, Gertrude Bell seemed to have been destined to become a daughter of the desert, with her sharp mind and energetic physique, she excelled at whatever she attempted. Her resume would include archaeologist, spy, Arabist, linguist, author, poet, photographer, mountaineer and nation builder. And despite her intrepid nomadic roaming, her warm and intimate letters to home revealed a sensitive, loving woman. She eventually would become perhaps the most famous woman of her time, exploring the deserts of Arabia on her own, learning Arabic, and meeting privately with the Bedouin. She learned more about Arab life and culture than any other westerner. Her skills and knowledge were only surpassed by her passion: she eventually grew to love the desert and the Arab people. The timing was perfect for England. During World War I, when the Ottoman Turks pushed into Arabia, the British needed someone to help bring the Arabs in as allies. Like her friend Lawrence of Arabia, Bell became the British government’s diplomat. She eventually helped win the Arabs to the British side, helped to build the nation of Iraq and was recognized by the Arabs for her dedication and achievements.

Anyone wanting an informed opinion about Iraq currently should read this book. It’s a sobering reality check when you read about the sacrifices made by the British 100 years ago and you think you are actually reading about what has happened in the last six years. It highlights the long history and complex nature of the Arab culture, beautiful and mysterious, and the obstacles in uniting a diverse group of people. Inspiring, informative. A whopping four stars. I can’t wait to read it again.

The Philippines: Dedication, Love and Service

The Philippines is a large country in the South Pacific, made up of more than 7,000 islands. After enduring 400 years of Spanish rule, and then used as a strategic base by the US during World War II, it has thrown off the yoke of colonialism and struggles as an independent developing country. I am here in Manila until Thursday; so far I’ve seen almost nothing of Manila, due to our heavy schedule, but I have seen devastating amounts of what hemophilia can do when there is no treatment.

The government does not buy factor, despite years of dedicated negotiations by the World Federation of Hemophilia. There are drug distributors here who are licensed to sell some products (NovoSeven, Koate DVI among them) but only the wealthy few with hemophilia can afford them. Even poor patients must pay for basic things like syringes, needles and even band-aids. Life is very hard for the majority of hemophilia patients, and almost every one I meet has some major deformity, trauma or has had a family member die from untreated bleeding.

On Monday we met first thing with a group of reporters representing various national newspapers and magazines. At the invitation of Andrea Trinidad-Echavez, a mother with von Willebrand disease, and media expert, these reporters listened to my presentation of hemophilia, and its symptoms, treatment and effects, followed by extremely emotional testimonials from the patients who attended. One was Angelo Cuevas, 27, who suffered a huge psuedotumor on his hip. He related his story of trying to find treatment, of not being able to afford an operation due to the high cost of factor and the large amount he needed. When he recounted what the pain was like, you could have heard a pin drop. Angelo stammered, bit his lip, and tried to go on. He was flooded with emotions remembering the pain: “It was as if I was going mad. I screamed with pain and my neighbors were scared,” he said in his soft voice. I am not sure many of us know what level of pain that is. I looked around the room as Angelo spoke and saw amputations, twisted hands, feet that cannot be flexed flat, missing eyes, bandages, crutches; so much pain was represented here.

The reporters stayed for 90 minutes and were genuinely moved by what they heard: how could a human not be?

Meeting with Father Don

We spent the afternoon then meeting with members of HAPLOS, which stands for Hemophilia Association of the Philippines for Love and Service. Haplos also means “human touch” roughly translated from the local language. This is headed by Rey Sarmenta, the father of a grown man with hemophilia. Rey worked in commercial business for over 30 years, and is educated, well connected and deeply compassionate. He could have retired many years ago to spend each day resting in comfort, but instead has dedicated himself to helping others like his son. HAPLOS is in excellent standing with the WFH, and struggles to help so many while operating with so little. Like in most developing countries, it’s almost impossible to get the government to pay attention to the needs of those with bleeding disorders when there are economic, political, climate and epidemic issues to confront.

We relaxed that evening at the Columban missionary compound we are visiting, guests of Father Don Kill. Father Don has been our main contact here for about five years. Also compassionate and dedicated, he operates a home for teens in Mindanao, in the south. He met a teen with hemophilia one day, took him in, and has been a champion of people with hemophilia ever since. He has turned to us for factor time and again, and his expert ability to organize and follow through has enabled us to get so much factor to the Philippines that this country represents 23% of all our factor donations, making the Philippines our largest recipient of aid.

On Tuesday, October 14, we met first thing in the morning with the patients again, without HAPLOS, to hear their opinions and record their needs. Filipinos are often characterized by their gentleness, and like many Asian people, avoid public confrontation and frown upon outspokenness. How different that is from the America culture, where speaking your mind in public is not only a privilege, but is considered a right (and good entertainment). Allowing patients to speak openly helps them assess their own feelings and needs.

See photos of the whole trip here.

Not surprisingly, the focus was on factor: how to get more, how to share what little is available. Despite the large amounts we have sent, I could see we were only touching the tip of an iceberg! That was momentarily disturbing, because if the Philippines is our largest recipient and we are only serving the needs of a few, but I can’t afford to think like that. What must do whatever we can do.

One mother told how her son had an intracranial bleed and almost died, but was saved with a donation of factor. A man named Elmer told us how he made history: he was the first Filipino patient to have a successful, no complications amputation, with the factor from Project SHARE. (Imagine thinking of how lucky you are to have a successful amputation!) He was very grateful.

Most touching was Jeffrey, a young man with an expression too old for his years. He was accompanied by his mother, who specializes in alternative therapies like massage. Jeffrey is one of five brothers, all with hemophilia. Four have died. Four. With ancient pain in her eyes, the mother told us how each had died: one from an intracranial bleed, one form a motor accident, one from an operation… Jeffrey looked away, his face becoming blank as she spoke. Clearly, Jeffrey was all she had left, and she was desperate to ensure he would live. He is a university student with impeccable English, but they are not wealthy. Without money, Jeffrey could not pay his tuition bill, and his grades would not be released. Without grades, he cannot enroll in next semester’s session. They were not asking for help but only highlighting the difficulties of life here.

How much does his education cost for one semester? $200. I told him we could cover that and we’d find a sponsor for next year. (If anyone reading would like to sponsor Jeffrey, we have enrolled him in Save One Life; just email me and let me know) Jeffrey is studying to be a psychologist and wants to dedicate his career to helping the patients with hemophilia in the Philippines. With all that he has suffered personally, he has turned his pain into a mission. It’s inspiring to see how resilient the human spirit is!

After this meeting we stepped out into the steamy, tropic air and drove through Manila to attend a luncheon meeting by HAPLOS at a Christian center. Lunch was a simple KFC style box lunch and the presentation highlighted HAPLOS’ accomplishments through the years. There is a strong focus on compassion and community. Most of all, HAPLOS stresses psychological support, more than any other country I have visited. “Without factor, sometimes it’s all we can focus on,” one board member said. The board is composed of mostly parents and patients, all volunteers.

A gathering of patients took place after the presentation, and these were all different patients than the ones we had met with that morning. The need, the pain, the suffering…I have never felt so helpless in all my life. While person after person stood and thanked Project SHARE for the factor that helped with a surgery, with a dark time, with a head bleed, with a life saved, I realized that so much more work needs to be done to find a way to close the gap between the haves and have-nots. One young, delicate mother, Olive, stood and told us her 12 year old, factor IX deficient son had been hospitalized for a week with a head bleed, and she broke down crying as she asked for help. A man with an obvious eye trauma stood and said he could not afford to have surgery for his eye; Father Don stepped in and informed him of how to apply for factor through Project SHARE. Another weeping young mother came forward holding her three year old son, whose elbow was swollen and hot—an active bleed. While her son was not crying, indeed looked bored, she was shaking and crying uncontrollably. Was she suffering from guilt? Later on we spoke to her and learned she was recently divorced, and has two children with hemophilia, no money, and under great emotional duress. This is where HAPLOS can excel in helping.

The meeting was capped by lunches for everyone, and the great news that we would be enrolling everyone in Save One Life, which would help ease some of their burdens by providing each person with a US sponsor who will give $20 a month (we will need those sponsors son after I return, so please consider helping). Everyone’s eyes lit up at the news, because so many people in attendance earn so little, and deal with pain that we rarely have to face. Can you imagine having your child with hemophilia actively bleeding, in pain, and not being able to do a single thing to stop it or to help?

Some of the patients had traveled three hours to attend this meeting — three hours by bus, in pain, spending precious pesos. We pledged to reimburse everyone for travel expenses, also much appreciated. As we filtered out of the center, I saw how the entire group shuffled out, walking unevenly from such crippled limbs.

Around 4 pm we left to visit a patient in his home. This is always the favorite part of my trips, we get to enter the world of the patients and directly see their needs — the better to help meet them. We passed the impressive business center of town and then drove by shanties teetering on the banks of muddy rivers. Yancy is an 18-year-old who lives on a dark and greasy looking alley. Crowded, noisy, hot, Yancy and his mom share their two-room dwelling with four other siblings and their father. Hard as it is to fathom that, this was a step up for them! They had recently moved there from a squatter’s place, under power lines. Yancy is a second year university student (college starts at age 16 here) and determined to graduate, despite poverty, overcrowding, a bed on a concrete floor, and untreated hemophilia. He is also available for sponsorship now. Any takers? We spent that evening at a gathering at Rey’s home, sampling delicious local cuisine, relaxing with the doctors and patients, and of course, celebrating with karaoke. I managed to avoid having to sing somehow, and we were entertained by Father Don, who likes to sing Elvis, and Mayette, a board member, who has a gorgeous voice. I could see that HAPLOS really is about love and service, and deep faith. The Philippines is 95% Roman Catholic and faith enters into every aspect of life for the common person. Perhaps this faith has sustained them this far on a journey of immense suffering and yet strong community. Mayette ended our wonderful evening by singing the adopted theme song of HAPLOS, “If We Hold On Together.”

The best news was for last: as we were leaving I learned from Dra. Flerida one of the main hematologists, that factor for Olive’s son, the one with a head bleed) had just arrived that afternoon from our office. Her son was going to be fine.

See photos of the whole trip here.

Off to the Philippines

It’s Monday in the Philippines and I have landed. I’m visiting for 12 days, to a country that is the single largest recipient of our humanitarian donations of factor. Although we have had contact with the Philippines for years, and have provided aid, I have never visited. This trip will give me an in depth tour of the situation that Filipinos with hemophilia live in. Most are poor, many are crippled. There is not enough factor for everyone, reducing the Filipinos to beggars sometimes. Life can be very hard for them. Several of the teens and young men we have tried to help have died this year.

We provide sponsorship for about 20 children and young men with hemophilia through Save One Life, our sponsorship program. I am hoping to meet most of them, and perhaps enroll some more.

See all the photos of the trip here.

Our guide through this eye-opening trip is Father Don Kill, a Columban priest who is also a shrewd organizer and compassionate humanitarian. We met years ago when Father Don discovered a teen living on the streets who couldn’t walk. In taking the boy to his mission for teens, he discovered the youth had hemophilia. Father Don has since been on a new mission: to find as many boys as possible undiagnosed with hemophilia, and get them the care they need. We are happy to support his efforts when possible.

This trip will take me into clinics in four cities; hemophilia treatment centers; to meetings with the press; meetings with the patients and the patient group, HAPLOS; and best of all, in the very homes of the poor, so we can document their lives and hopefully find help for them when I return to the US. Please check in again in a few days when I hope to have more of our journey posted!

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