2017

La-Va Land

A brief respite from all things hemophilia as I share my adventure to the Nyiragonga Crater in the Democratic Republic of the Congo.
 

After the hemophilia workshop in Rwanda, I decided to have a brief adventure. On Saturday, June 17, I packed
up my hiking gear, and waited in the lobby of the Mille Collines (of “Hotel Rwanda” fame). My driver Faustin arrived,
and we loaded up the jeep and took off. He was nice: clean cut, friendly, like
most Africans seem to be. We drove through Kigali, where there was a bit of Saturday
morning traffic on the narrow, two-lane road.

The drive was a good four hours to the Rwanda border. I looked out the window at the pink stucco houses perched on the
rolling hills of Kigali to my right; to my left, more tropical pink and
turquoise blue painted homes on top of red dirt. Despite the dirt, everything
is clean. No paper, plastic bags, trash. While poor, the Rwandans have pride.
And strict laws that prohibit not only littering, but even bringing plastic
bags into Rwanda. I’ve never seen such a clean developing country before like Rwanda.
The rolling hillsides gave way to lush forests, and tilled fields on graduated steps, all
pattered in different crops. Faustin pointed out, There’s sorghum, there’s
maize, and there’s tea. All these created a patch-work green quilt of crops
spreading out before us. At the top of the quilted fields, the lone trees that
have not been chopped down crown the tops of each hill. Land of a Thousand
Hills.
The roads
are excellent and winding, going up and up, higher and higher, and I thought,
Barry Haarde just has to do Wheels for the World in Rwanda! And sure enough, we
pass impressive, ripped Rwandans cycling up these killer mountains, wearing
professional gear. Jaw-dropping power!
They, like
us, pass by hundreds of people walking. Everyone in Rwanda walks. Children
struggle with big yellow containers holding water. Old women balance primary
color buckets on their heads, filled with potatoes to sell. Women, men and
children sway under 20-foot tree trunks, sometimes as many as five on one
person, to bring home to chop into firewood. Most people don’t have cars, or
running water. So water and fuel for the fire must be sought or bought and
carried home. Mile after mile, men push bicycles up these hilly roads, which
are laden with several enormous sacks of potatoes. We drive by one bicycle
which only had tires visible; the entire bike and rider, pushing it, was engulfed
in a mountain of kindling. No one hitches for a ride. Everyone is working. By
noon we see impeccably dressed people in large groups walking along: Seventh
Day Adventists returning from church. How handsome they all look, in contrast
to the dusty roads. Faustin pulls the car over and we get quizzical stares, but
he introduces me to his father, in one of the groups. Expressions change to
greetings of joy.
At last we reach Gisenyi, a town crouched on the shores of Lake
Kivu, and “Paradis Malahide,” my mid-range hotel. It’s fine with me, quite
nice. It has a Caribbean feel to it. We check in, and two smiling Masaai
promptly approach and ask if I want to buy sandals.
My room is up on the second floor, all wood, with a beautiful view of Lake Kivu. I
instantly want to just curl up with my book on Rwanda and relax. First lunch:
so we order and sit by the lake. We
eat lunch, which is an almost inedible sandwich for me. I don’t eat it all, as
the chicken slivers in it are dark meat, greatly overcooked. But Faustin
unabashedly takes whatever I have left and eats it, telling me that due to the
shortages of food in the country in the past, nothing goes to waste. He eats
everything in front of him.
Laurie Kelley and Faustin

I ascend the
steps to my room, sort out my things, grab my iPad and read an excellent book
about President Paul Kagame, once leader of the rebellion to overthrow the
regime responsible for the genocide, and later president of Rwanda. A
fascinating man and leader. I order African tea, which is so delicious and
spicy, and watch as a sakabaka (black
kite) swoops down to the beach then back up again to a tree top. Then there are
two, doing a ritual dance, up and down, crisscrossing in the cloudless sky.

I read until dinner, and then order pasta,
which maybe is the food least likely to be ruined by cooking (and I would know as I ruin everything I cook). It’s fine, but
too much, and I eat only half the dish and half a glass of wine. I return to my
room, roll down the mosquito netting and read till I fall asleep, serenaded by
the whirring of the crickets and occasional bird calls.
In the morning, Sunday, I pack up and am
ready by 7 am, as planned, with no breakfast. Just a take-out order of bread
and bananas. We head for
the Rwandan border. Rwanda customs is in a huge building, very out of place in this
remote area. It’s cavernous, empty and air conditioned. Gleaming floors and an
efficient process for passing to the Democratic Republic of the Congo (DRC). We
wait in line, get a stamp, and done!

We then walk across the border, showing our
passport and stamps to the agents seated in the searing sun. As soon as we walk
across the border, the quality of life plummets. It’s an incredible contrast to
the pretty streets of Rwanda. Both are poor countries; both have dusty roads
and homes of rock with corrugated metal roofs. The main difference is that
Rwanda is kept clean: no trash anywhere. In the DRC, people toss wrappers and
trash at their feet. In Rwanda in the
morning, you will see women with their home made brooms of corn husks or branches,
sweeping out the streets, of any stray paper or even dried leaves… even far
away from Kigali on the main road to the DRC, these city workers are sweeping
up fallen leaves from the trees that landed on the road side! Not in the DRC.
It is an impoverished country, psychically and mentally.

 

 
Our new driver picks me up, and Faustin waves
good bye as he returns to Rwanda on foot. I climb into the new jeep and away we
go to Mt. Nyiragonga, a large active volcano. My guide, Tresor, can’t wait to
tell me about the DRC. We are now in Goma, a town made famous during the 1994
genocide, into which a huge number of Hutu refugees flowed after the Tutsi massacre
stopped. They crushed this town, and created one of the worst humanitarian
crises of our times. It was only then, when cholera swept through the camp,
killing thousands and orphaning thousands, that the West finally sat up and
noted that Rwanda had a few problems. That’s a story for another time but the
history of Rwanda and its genocide is absolutely fascinating; I’m reading my
fourth book about it.
We stop at a statue in the middle of Goma, of
a man pushing a chukudu, a two-wheel
wooden “motorcycle,” and Tresor explains that this statue, erected in 2009, is
the symbol of the DRC, and how hard its people work. That’s true: everyone is
working. There are no beggars in such a place, as tourism is limited. I find
you mostly have beggars when there is an influx of tourists.
Goma is grey. Everything, from streets to
buildings and even the color of the people, is chalky, dusty grey. Partly it is
from living in lava-land. The last eruption of Nyiragonga was in January 17, 2002,
when the lava lake collapsed, and lava sped at 60 mph (the fastest ever
recorded!) and reached Goma, coating everything in molten rock, which hardened
to a shell. About 147 people died, and 120,000 were left homeless. An ongoing
threat, besides the rumblings of this active volcano, is carbon dioxide
emissions from the ground, called mazuku,
which kills, as recently as 2016.
Now, everywhere you look you see piles of
lava rock, home built of lava rock, and the color charcoal grey.
Laurie Kelley and Tresor
Soon, the
grey gives way to red dust, as we drive along the main road to the national
park. Within 20 minutes we arrive. A group of men shuffle their feet in place
along the roadside, perhaps waiting to be chosen as porters for the mzungu (white person) who will ascend the volcano
today. The driveway to the registration cabin is so steep we have to get out
and walk, so the jeep can make it up. Welcome to La-Va Land!
It takes
about 30 minutes to sign in, and wait for the rest of our group. We have only
a few people in our group, and indeed, only a few can go at a time. First, it’s
dangerous and a strenuous hike. Second, at the rim of the volcano, where we
will camp overnight, there are only a few sheds (A-frames made of metal). Our
group? George, an FBI agent working in Nigeria, and a dead ringer for Tom
Brady. Orlando, a very athletic flight attendant with Edelweiss Air in Switzerland.
And Franziska and Bacadi, a 30-year-old German woman and her Zanzibarian
boyfriend. This was a great group to hike with and with a few porters (mine is
Josef, who will carry my duffel bag), Tresor and two armed rangers, we head
out!


This was
overall a very strenuous hike, more
than I imagined. Despite being in pretty good shape, I found it hard. In less
than a year, I’ve summited Kilimanjaro (a 6-day hike, 19,341 ft), and then Everest
base camp (9 day, 17,500 ft) but this 11,380 footer was a killer! Partly it’s
the 45° angle that strains the quads and calves; partly it’s the loose lava
rocks. At times you ware walking on wet ground; at others, on loose lava scree;
then you will walk on rocks that twist ever which way and are as sharp as
razors. And there’s the altitude. We were all huffing and puffing our way up,
quads screaming at times. And we need to ascend quickly. Up straight to the
top; sleep overnight, then descend first thing in the morning.
Franziska
and I fell in to talking: she and Bacadi work at a hotel in Zanzibar (one of my
favorite places to visit!). She speaks several languages, but he! He speaks
Swahili, English, German, Italian, French and is learning Spanish! And he was
such a nice guy. I admired how kind he was to his girlfriend. Franziska
struggled right from the start. She was nervous as her fitness level was
self-admittedly not high. I tried to reassure by saying it’s not a competition;
we are in no rush. We’ll all wait. And also what I have in fitness is negated
by age! After all, I’m almost twice as old as her.
Still, it
was a slow hike, with Franziska asking childlike at each rest stop, How much
longer till the next stop?
The higher
we went up, the quieter the group became, as we focused on not twisting our
ankles, and breathing deeply. The air was moist, the vegetation thick and rich.
Tresor stopped us on one rocky section to look at something amidst the rock
piles. I saw nothing. It’s a bird! He said. I didn’t see it. Finally, its form
emerged out of the rocks, like a 3-D photo in which you have to blur your eyes
to see the picture. It was a nightjar, sitting quietly, blinking in the sunlight. I
thought it injured, but when Foramen, our guard, touched it with his gun tip,
the bird arched its wings to ward us off, and there underneath it, warm and
safe, was a single white egg. After a quick snap of the camera, we trudged on.
Eventually
the moist forest gave way to an alpine climate, with low scrub brush and trees
similar to those I’ve seen on Kilimanjaro. The air grew very cool and
we donned jackets. The hillside became even more rocky, in fact, all rocks now.
Sometimes you could find where the spilled lava river solidified, and you felt
like you were walking on a smooth river of stone. Then it changed to sharp,
black, porous lava. Our feet, stepping and kicking them, caused a tinkling
sound, like crystal. Despite its menacing look, the lava sounded like the
finest Waterford.
Finally we
reached our goal, the summit of Nyiragonga, 11,384 feet! Clouds moved in,
sometimes obscuring our view. All of us were anxious to see the famed lava
lake. We ditched out backpacks, threw on extra layers and climbed up a few more
feet to perch on the rim. And I mean perch. It’s a sheer drop down into the
volcano, at the bottom of which is a red, thick, ever-shifting lake of lava, complete
with red waves, red flames shooting upward, bubbling molten rock. It’s like a
prehistoric vision of what hell might be like. If I were an uneducated person,
living centuries ago, this would awe and frighten (it still does) and I would
believe that this is Hell, literally. It looks like the doorway to the Underworld,
where people would burn in its fire for eternity.
But, it’s
just a volcano. And a magnificent one.
We stood for
the longest time, mesmerized, silent except for some oohs and ahhs. Clouds came
and went, but finally they parted to give us a good view of the lake. To hold
my cameras still, I crouched down on the lava, which was mighty uncomfortable;
lava is razor-sharp and hurts. After about 30 minutes of trying to think of
poetry or words that could do this vision justice, I groped my way to my metal
tent. Inside was just a bare mattress, onto which I threw my wonderfully warm
sleeping bag and my gear. I brought a headlamp, essential, and all the usual accessories.
I added layers and layers as it was cold, about 40°. Not freezing, but still I
could see my breath. Dinner was called and we fumbled our way on the pitched
slope in the dark to the mess tent, which had a charcoal fire on the ground. The air was nearly unbreathable inside. Tresor asked me if we used charcoal back home.
Laurie Kelley at volcano rim
It was cold
and dirty, unlike my other adventure trips. The food wasn’t particularly good, especially
inedible was the chicken. Franziska and Bacadi joined us; they signed up for
this on a whim and were fairly unprepared! No gloves and no food. We had them
eat ours, gladly, as I ate almost nothing. Then back to bed where I read a bit
before falling into a deep sleep.
I was awake
at 4 am, happy and content to be on this dirty volcano, and didn’t even need a
second look at the lava lake (which was obscured by clouds I later learned). I
wanted to get back to Kigali for my 7:30 pm flight, a long ways away. So I was
ready—even had mascara and eye liner on!—and after a quick bite we started
down.
My quads
ached and my knees buckled from the 7-hour hike yesterday, and minimal food and
nutrients. I actually had to be careful using my left knee so I didn’t wrench
it. Going downhill on loose lava at a 45° angle is tricky. I slipped a few
times, fearful of landing in the mud (the overnight moisture softened all the earth and
made the rocks slippery) so Foramen held his hand out to me often to help me.
It was a long, quiet three-hour hike back to base. But as we descended towards
the end, I heard a whoop and a rush, and Franziska flew past me and grabbed Foramen!
Get me off this mountain! she happily squealed! I teased her that she was
fooling us about her fitness level. She actually felt good while I was trashed!
Back at base
we exchanged email addresses, waved good-bye, and got into our jeeps to head
for home. I tried to hurry Tresor, but he assured me that we had plenty of
time. And he was right. About 30 minutes later, I walked across
the border into Rwanda again with Tresor, who left me in the hands of Chris, my
drive back to Kigali.
I was tired,
and after a 30-minute drive I climbed into the back seat to try to sleep before
the long trip home. After an hour I gave up and climbed back into the front
seat. Chris and I chatted. I told him I had used Gorilla Trek Africa three
years ago to find gorillas in Rwanda and I loved my guides, who were so good, I
decided to use the outfitter again. He asked who they were: Scodius (who is
friends with me on FB) and a young guy whose name I don’t recall… Chris was
silent then said quietly, That was me.
And he laughed and laughed and I felt all barriers falling. Now I recall!
He and Scodius were so nice then, and I even invited them to
dine with me in the dining room, instead of having them go back to the lesser quality guides’
hotel. We sang Christian songs all the way back to Kigali, I recall, and Chris
gave me a CD with all the songs on it. So this was really nice to see him again and connect.
He shared
how he was born in the jungle, and called himself “Son of Gorillas.” His mother
couldn’t get to the hospital or even midwife; she had been out collecting
firewood, and so she gave birth right in the jungle! To cut the umbilical cord,
one of the women she was with said to bite it. No one would bite it! You do it,
you do it, they all shouted at each other. Then finally they sharpened the edge
of a fan palm, which is tough and sharp anyway, and they cut it. Twelve kids
later, they all kid Chris about being the “son of a gorilla.” Chris became
sober when I asked about his mother now: killed in the genocide, along with a
sister. There’s just no escaping the genocide impact on this country.
We stopped
at a convenience store and bought muffins, which I promptly dropped in the dirt
on the roadside, but ate anyway, after dusting it off. I am getting way too chancy in my habits overseas!
Back to
Kigali, to the Mille Collines, which kindly lent me a room for one hour to
shower and repack, and then Chris brought me to the small international airport. We had to
stop at the airport entrance, unload all the luggage and a Belgium malnoir sniffed
through everything. (Rwanda has tight security.)
And that was
that. I tipped Chris, gave him a hug, settled at a coffee shop in the brilliant sunshine,
and waited for my flight. Eventually it would be a 7:40 pm flight to Nairobi
with a 2-hour layover, a 7.5 hour flight to Amsterdam, with a three hour
layover, and a 7.5 hour flight to Boston.
A 29-hour transit but coming from Africa, decades in distance. And I cannot wait to return. Africa is magical, with stunning natural beauty and friendly people, and a lifetime of work in hemophilia to do.
See the full gallery of photos for the DR Congo here.

New Publication Answers Questions About the SIPPET Study

Inhibitors are the most troubling complication of hemophilia A treatment today. In this week’s blog, I share with you a new publication concerning the SIPPET study, which sheds light on factor VIII products and inhibitors.
New Publication Answers Questions About the SIPPET Study


An article written by noted hematologist Dr. Flora Peyvandi and colleagues was recently published in the medical journal Haemophilia. This article directly addresses and answers several questions that were raised about the 2016 publication of the Survey of Inhibitors in Plasma-Product Exposed Toddlers, or SIPPET study.1

With this new article, SIPPET: methodology, analysis and generalizability, the authors respond to the 17 most common questions associated with the design, methodology, and results of the SIPPET study, including1:
  • Is the inhibitor risk higher in SIPPET than in previous reports?
  • Could differences in treatments between countries have affected the results?
  • Could the results have been affected by the way the study was randomized?
  • Do the SIPPET results also apply to other recombinant factor VIII (rFVIII) products
    beyond the 1st and 2nd generation products used in the study?
  • Is there a difference in inhibitor risk between the different brands within the plasmaderived and recombinant groups?
Original SIPPET Study
The SIPPET study, conducted by Dr. Peyvandi and colleagues, was the first randomized trial to compare the incidence of inhibitors in plasma-derived factor VIII (pdFVIII/VWF) products and rFVIII products in previously untreated patients (PUPs).2

Results from this landmark study showed that there was an 87% higher rate of inhibitor development in patients who received rFVIII compared with patients who received pdFVIII containing von Willebrand factor (VWF).2

Based on the results of the SIPPET study, the National Hemophilia Foundation’s Medical and Scientific Advisory Council (MASAC) now recommends that pdFVIII/VWF be considered as one of several treatment options in PUPs.3
Visit www.inhibitorinfo.com to Learn More About Inhibitors
Inhibitors are the most serious and challenging hemophilia A treatment complications. All patients with hemophilia A are at risk for developing inhibitors, regardless of age and disease severity.
Inhibitorinfo.com is a comprehensive website that provides important information and resources about inhibitors and the risk of inhibitors. There is a discussion guide patients can download and use to talk with their hematologists about inhibitors. Visitors can also read about the results of the SIPPET study and watch leading hematologists talk about its implications.
When visitors sign up for updates at inhibitorinfo.com, they will receive access to the full SIPPET study, as well as updates about hemophilia, inhibitors, and the latest clinical data.
References: 1. Peyvandi F, Mannucci PM, Palla R, Rosendaal FR. SIPPET: methodology, analysis
and generalizability [published online ahead of print March 17, 2017]. Haemophilia. doi:
10.1111/hae.13203. 2. Peyvandi F, Mannucci PM, Garagiola I, et al. A randomized trial of factor VIII
and neutralizing antibodies in hemophilia A. N Engl J Med. 2016;374(21):2054-2064. 3. National
Hemophilia Foundation. MASAC Update on SIPPET. National Hemophilia Foundation website.
https://www.hemophilia.org/Newsroom/NHF-Community-News/MASAC-Update-on-SIPPET.
Published March 9, 2016. Accessed April 27, 2017.
This is a paid public announcement from Grifols and does not constitute an endorsement of
products or services. When you click on the links in this blog entry, you will be directed to
the Grifols website. LA Kelley Communications always advises you to be a savvy consumer
when contacting any company; do not reveal identifying information against your will.
BN/A8/0517/0275

“My bones are older than me”

Prince, age 17
“My bones
are older than me,” lamented Prince, the handsome 17-year-old Rwandan with
hemophilia, who I had met previously three years ago when I first came to this
small, lush country. This night, Wednesday, June 14, I welcomed a few families
with hemophilia, who are the founders of the new Rwanda Federation of
Hemophilia. While all competent professionals, they still need some help in
getting their organization jumpstarted. Imagine living in a country that has no
factor, no hemophilia care, and is poor. Of the estimated 800 people with hemophilia here,
no more than 50 are identified.
But this is all about to change.
We gathered socially in the cool evening on the terrace at the famous Hôtel des Mille
Collines, dubbed “Hotel
Rwanda,” a safe haven during the brutal 1994 genocide, where the manager Paul Rusesabagina saved 1,268 Hutu and Tutsi refugees from the Interahamwe militia. (If you haven’t
seen the movie Hotel Rwanda, I urge you to rent it.) The Mille Collines is
a lovely hotel, and while I write this I hear a spry African ensemble playing traditional
music downstairs with chanting and upbeats that make you want to dance!
Shady Sedhom, NNHF, listens to the patients
Prince is rail
thin, and soft spoken, like all Rwandans. I’m glad he arrived first so I could
get caught up with him personally. When I saw him in 2014, I had arrived for
the first time in Rwanda to assist the new Federation. Prince was a stocky
14-year-old then. Now he was lean and taller, with chiseled features. When I
asked him how he was doing, he replied with a phrase that showed his desperate
plight, and poetic aptitude: “My bones are older than me. I have the bones of a
60-year-old, my doctor told me.” His right knee had given him a lot of trouble
three years ago; now the left one was. When was the last time he went to the clinic,
which was only 1 kilometer from his house? Not in years. Why? “Every time I go
they have no factor.”
I’ve been
trying to keep Rwanda supplied with factor; indeed, we are the only ones who
give them factor. And that’s because they are not yet registered with the World
Federation of Hemophilia. Once they register, they will be eligible for much,
much more factor, perhaps regularly. Getting them registered, both with their
own government and then the WFH was my goal this trip.
With me was
Mr. Shady Sedhom, a registered pharmacist and now program manager with the NovoNordisk Haemophilia Foundation, an incredible organization based in
Switzerland, that provides program expertise, management and funding for
hemophilia organizations globally. This was the second time I would work with
them, but the first time in person. We would give a half day workshop on
Thursday, June 15.
Benis’s knee
But this
evening we were here to meet the board members, and hear their stories. I don’t
want to just give a lecture on how to run an organization: I want to know them,
as people, as families, as families with hemophilia, as blood brothers and
sisters in this amazing global family we have.
Little Benis
I met Vivine,
whose son Ness, now age 8, hemophilia B, had a headache as a toddler, and it
continued on. We listened in somber silence, as she continued. She took him to
the doctor, who tested him for malaria, but this was not it. They gave him a painkiller
and sent him home. The headache continued for two more days, getting worse. She
took him to the ER on a Friday night at the public hospital, but they said they
could not give him a CT scan because it was too late. Come back Monday! In a
culture used to respecting authority and not questioning the medics, Vivine’s
strong maternal instinct won out. She went to the King Faisal Hospital (a
private hospital), which would be prohibitively expensive. She went anyway that
night, and they diagnosed him with a head bleed! He got factor and this saved
his life. Her story highlighted the need for education among the country’s
doctors.
A fun evening with many shared stories
We met
James, age 31, also hemophilia B, who was just diagnosed last year! James is a quiet man, lacking a few front teeth. Indeed, he had persistent dental
problems, with constant bleeding. When the doctors here could not figure out
what was causing this, he finally sent his blood to France to be tested, at
cost of $400! This is a ridiculous amount of money in a country where the average
annual household income is about $700. Especially since it could have been
diagnosed in neighboring Kenya.
Sylvestre
has been my email pal for the past few months as we prepared for this visit. He
serves as Secretary of the RFH. Sylvestre is well known to us in the office
back home as he has requested factor for his son Virgil. Little Virgil,
squirming before me with all the energy of a four-year-old, wears glasses for
his still misaligned eyes. He was blind for 18 months after a coma, due to head
bleed as a two year old, but factor from Project SHARE saved his eyesight. Slowly,
Sylvestre told us, he is getting his eyesight back.
Sylvestre
reminded me of how I got involved with Rwanda in the first place—I had actually
forgotten as we have accumulated so many stories working with so many countries.
A nurse named Tracy Kelly was volunteering in Rwanda about five years ago, and
met Sylvestre as he sought help for little Virgil. She contacted her hospital
back home, which eventually found us. We shipped factor over right away. And
when the crisis passed, I asked Sylvestre to consider founding a national
organization for those with hemophilia. Like many we have met, he agreed. And
here we were.
We finished our
juice drinks and tea and cakes, and then they dispersed into the soft night,
hopeful for the next day’s outcomes.
Vivine adds a needs list
The next day
was our workshop. Shady has a prepared slide deck, exercises and came equipped
with markers, post-its, posters. The attendees arrived early, prepared to work!
Besides the RFH we also had several doctors, which was a high point. Doctors in
developing countries have little free time. Most work at two hospitals and/or
have a private practice. They seem on call 24/7. To have them here was an
absolute honor. The day consisted first of a needs assessment, brainstorming
what Rwanda needs to have good hemophilia care. Each attendee wrote out ideas
on a post-it note then attached it to a poster, under one of four areas of
need. The post-its read: A comprehensive center, training, education of health
care workers and families, diagnosing suspected cases, outreach to find more
patients, public awareness to help find patients, and of course… more factor.
Laurie Kelley with patient at CHUK 
But the
greatest need was to register the RFH with the government. Until it becomes an
official, registered nonprofit, nothing much would happen. The WFH needs it
registered and accountable. Project SHARE will keep sending factor of course,
but we can only do so much. Shady said NHF is ready with funding for a project
to help meet these needs… after they get registered.

 
And an
interesting phenomenon: learning how to challenge each other’s ideas. When
someone offered an idea, such as the most important need was to get more
factor, Shady and I challenged that. Spending your time securing factor is
urgent, especially when your child has a bleed. But allowing the registration
issue to languish means you will only get dribs and drabs of donated factor. Focusing
on registering now will open so many doors later. Short term pain for long term
gain. The RFH was learning now to priorities needs.

After this
we did goal setting, based on those needs. And then prioritizing those goals.
This took over an hour. Later, Shady asked the group to plan a birthday party,
as an exercise in planning a hemophilia event later on (like World Hemophilia
Day next April). It was a fun exercise to see who remembered what action item
was needed. And ironic: that very day, June 15, was Shady’s birthday! Unknown
to him I ordered a cake. And right after the birthday party exercise, we took a
break, and out came a cake with candles! It was fun to all join in and sing him
happy birthday. We thanked him for his dedication for spending his birthday working on Rwanda’s hemophilia future. Then we learned that we had another cause to celebrate: James’s wife had just given birth a few hours ago! But he stayed to complete the workshop with us. Such dedication!
After break
came Stakeholder Awareness, an exercise I’ve never done before so this was
educational for me. Even just learning who to identify who is influential and
how much they were influential—Prince offered the media, which was
brilliant—and others offered families, the public, the ministry of health and
more.
By the end
of the day we had the components of a strategic plan, with action items. It
will take many more meetings to hammer out the details but it was a powerful
five hours. Afterward, we dined outside in the night air and had a buffet
dinner together. Shady had to dash off to catch a flight but the rest of us
relaxed and shared our thoughts on this truly historic day.

We planted
the seeds of growth, and now, it’s up to the Rwandans to take next steps on the
road to better hemophilia treatment care.




And they
deserve it and can do it. Rwanda spends more on healthcare per capita than most
African countries. The country is peaceful, functions well and has infrastructure.
It’s a small country, about the size of Massachusetts, my home state. Best of
all, it has interested and dedicated doctors. All ingredients of success.
There will
be challenges. I read a Rwandan proverb that says: If you are building a
house and a nail breaks, do you stop building, or do you change the nail?
We don’t want them to ever stop building.

A reason to celebrate!
My vision?
To see Rwanda join the WFH, and be present next year at the WFH Congress in
Scotland, where they will meet the world community, and their fellow Africans,
to learn, to share and to get the resources that so many others get. When they
join everyone else, they will then be able to determine their treatment and
destiny, and a whole generation of Rwandans will grow up free of the pain and
disabilities they suffer now.

To see photos of the trip, go here. 

“My bones are older than me”

Prince, age 17
“My bones are older than me,” lamented Prince, the handsome 17-year-old Rwandan with hemophilia, who I had met previously three years ago when I first came to this small, lush country. This night, Wednesday, June 14, I welcomed a few families with hemophilia, who are the founders of the new Rwanda Federation of Hemophilia. While all competent professionals, they still need some help in getting their organization jumpstarted. Imagine living in a country that has no factor, no hemophilia care, and is poor. Of the estimated 800 people with hemophilia here, no more than 50 are identified.
 
But this is all about to change.
 
We gathered socially in the cool evening on the terrace at the famous Hôtel des Mille Collines, dubbed “Hotel Rwanda,” a safe haven during the brutal 1994 genocide, where the manager Paul Rusesabagina saved 1,268 Hutu and Tutsi refugees from the Interahamwe militia. (If you haven’t seen the movie Hotel Rwanda, I urge you to rent it.) The Mille Collines is a lovely hotel, and while I write this I hear a spry African ensemble playing traditional music downstairs with chanting and upbeats that make you want to dance!
Shady Sedhom, NNHF, listens to the patients
Prince is rail thin, and soft spoken, like all Rwandans. I’m glad he arrived first so I could get caught up with him personally. When I saw him in 2014, I had arrived for the first time in Rwanda to assist the new Federation. Prince was a stocky 14-year-old then. Now he was lean and taller, with chiseled features. When I asked him how he was doing, he replied with a phrase that showed his desperate plight, and poetic aptitude: “My bones are older than me. I have the bones of a 60-year-old, my doctor told me.” His right knee had given him a lot of trouble three years ago; now the left one was. When was the last time he went to the clinic, which was only 1 kilometer from his house? Not in years. Why? “Every time I go they have no factor.”
I’ve been trying to keep Rwanda supplied with factor; indeed, we are the only ones who give them factor. And that’s because they are not yet registered with the World Federation of Hemophilia. Once they register, they will be eligible for much, much more factor, perhaps regularly. Getting them registered, both with their own government and then the WFH was my goal this trip.
With me was Mr. Shady Sedhom, a registered pharmacist and now program manager with the NovoNordisk Haemophilia Foundation, an incredible organization based in Switzerland, that provides program expertise, management and funding for hemophilia organizations globally. This was the second time I would work with them, but the first time in person. We would give a half day workshop on Thursday, June 15.
Benis’s knee
But this evening we were here to meet the board members, and hear their stories. I don’t want to just give a lecture on how to run an organization: I want to know them, as people, as families, as families with hemophilia, as blood brothers and sisters in this amazing global family we have.
Little Benis
I met Vivine, whose son Ness, now age 8, hemophilia B, had a headache as a toddler, and it continued on. We listened in somber silence, as she continued. She took him to the doctor, who tested him for malaria, but this was not it. They gave him a painkiller and sent him home. The headache continued for two more days, getting worse. She took him to the ER on a Friday night at the public hospital, but they said they could not give him a CT scan because it was too late. Come back Monday! In a culture used to respecting authority and not questioning the medics, Vivine’s strong maternal instinct won out. She went to the King Faisal Hospital (a private hospital), which would be prohibitively expensive. She went anyway that night, and they diagnosed him with a head bleed! He got factor and this saved his life. Her story highlighted the need for education among the country’s doctors.
A fun evening with many shared stories
We met James, age 31, also hemophilia B, who was just diagnosed last year! James is a quiet man, lacking a few front teeth. Indeed, he had persistent dental problems, with constant bleeding. When the doctors here could not figure out what was causing this, he finally sent his blood to France to be tested, at cost of $400! This is a ridiculous amount of money in a country where the average annual household income is about $700. Especially since it could have been diagnosed in neighboring Kenya.
Sylvestre has been my email pal for the past few months as we prepared for this visit. He serves as Secretary of the RFH. Sylvestre is well known to us in the office back home as he has requested factor for his son Virgil. Little Virgil, squirming before me with all the energy of a four-year-old, wears glasses for his still misaligned eyes. He was blind for 18 months after a coma, due to head bleed as a two year old, but factor from Project SHARE saved his eyesight. Slowly, Sylvestre told us, he is getting his eyesight back.
Sylvestre reminded me of how I got involved with Rwanda in the first place—I had actually forgotten as we have accumulated so many stories working with so many countries. A nurse named Tracy Kelly was volunteering in Rwanda about five years ago, and met Sylvestre as he sought help for little Virgil. She contacted her hospital back home, which eventually found us. We shipped factor over right away. And when the crisis passed, I asked Sylvestre to consider founding a national organization for those with hemophilia. Like many we have met, he agreed. And here we were.
We finished our juice drinks and tea and cakes, and then they dispersed into the soft night, hopeful for the next day’s outcomes.
Vivine adds a needs list
The next day was our workshop. Shady has a prepared slide deck, exercises and came equipped with markers, post-its, posters. The attendees arrived early, prepared to work! Besides the RFH we also had several doctors, which was a high point. Doctors in developing countries have little free time. Most work at two hospitals and/or have a private practice. They seem on call 24/7. To have them here was an absolute honor. The day consisted first of a needs assessment, brainstorming what Rwanda needs to have good hemophilia care. Each attendee wrote out ideas on a post-it note then attached it to a poster, under one of four areas of need. The post-its read: A comprehensive center, training, education of health care workers and families, diagnosing suspected cases, outreach to find more patients, public awareness to help find patients, and of course… more factor.
Laurie Kelley with patient at CHUK
But the greatest need was to register the RFH with the government. Until it becomes an official, registered nonprofit, nothing much would happen. The WFH needs it registered and accountable. Project SHARE will keep sending factor of course, but we can only do so much. Shady said NHF is ready with funding for a project to help meet these needs… after they get registered.

 
And an interesting phenomenon: learning how to challenge each other’s ideas. When someone offered an idea, such as the most important need was to get more factor, Shady and I challenged that. Spending your time securing factor is urgent, especially when your child has a bleed. But allowing the registration issue to languish means you will only get dribs and drabs of donated factor. Focusing on registering now will open so many doors later. Short term pain for long term gain. The RFH was learning now to priorities needs.
After this we did goal setting, based on those needs. And then prioritizing those goals. This took over an hour.
Later, Shady asked the group to plan a birthday party, as an exercise in planning a hemophilia event later on (like World Hemophilia Day next April). It was a fun exercise to see who remembered what action item was needed. And ironic: that very day, June 15, was Shady’s birthday! Unknown to him I ordered a cake. And right after the birthday party exercise, we took a break, and out came a cake with candles! It was fun to all join in and sing him happy birthday. We thanked him for his dedication for spending his birthday working on Rwanda’s hemophilia future. Then we learned that we had another cause to celebrate: James’s wife had just given birth a few hours ago! But he stayed to complete the workshop with us. Such dedication!
After break came Stakeholder Awareness, an exercise I’ve never done before so this was educational for me. Even just learning who to identify who is influential and how much they were influential—Prince offered the media, which was brilliant—and others offered families, the public, the ministry of health and more.
By the end of the day we had the components of a strategic plan, with action items. It will take many more meetings to hammer out the details but it was a powerful five hours. Afterward, we dined outside in the night air and had a buffet dinner together. Shady had to dash off to catch a flight but the rest of us relaxed and shared our thoughts on this truly historic day.

We planted the seeds of growth, and now, it’s up to the Rwandans to take next steps on the road to better hemophilia treatment care.

And they deserve it and can do it. Rwanda spends more on healthcare per capita than most African countries. The country is peaceful, functions well and has infrastructure. It’s a small country, about the size of Massachusetts, my home state. Best of all, it has interested and dedicated doctors. All ingredients of success.
There will be challenges. I read a Rwandan proverb that says: If you are building a house and a nail breaks, do you stop building, or do you change the nail? We don’t want them to ever stop building.


A reason to celebrate!
My vision? To see Rwanda join the WFH, and be present next year at the WFH Congress in Scotland, where they will meet the world community, and their fellow Africans, to learn, to share and to get the resources that so many others get. When they join everyone else, they will then be able to determine their treatment and destiny, and a whole generation of Rwandans will grow up free of the pain and disabilities they suffer now.

To see photos of the trip, go here. 


Personalizing Prophylaxis

Written by Leonard A. Valentino, MD

Originally published in YOU, August 2015
Steve is a lifelong Chicago White Sox fan and rarely misses a home game. For his birthday last year, a friend gave him a team ball cap to replace the one he’d worn for years. The new black and white cap looked cool and had a great brim for blocking the sun. But it was a non-adjustable, “one size fits all” model, and after just a couple of innings, Steve had to take it off because it was tight and uncomfortable. The cap was too large for his daughter but fit his son perfectly, and he became its new owner.
Standard prophylaxis is a lot like that baseball cap. For years, we’ve relied on a one size fits all approach to treatment. For many, these standard regimens, intended to keep FVIII or FIX trough levels above 1% of normal, prevent or reduce bleeding. Other patients may need higher trough levels to prevent bleeds, while some may be overtreated with a standard approach. So, how can we improve the “fit” of prophylaxis and reduce the risk of bleeding or overtreatment for everyone? Make the regimen adjustable by customizing treatment to the individual. In other words, personalizing prophylaxis.
Step One: Assessing Bleeding Risk
To personalize a prophylaxis regimen, your physician must first assess your risk for bleeding.1 This risk is partly determined by your bleeding phenotype—how often you bleed when not on prophylaxis. While many people with severe hemophilia have frequent, spontaneous joint bleeds, some rarely experience bleeding episodes. Your bleeding phenotype helps determine the intensity of prophylaxis. If you have a severe phenotype, you may require a higher dose of factor concentrate and/or more frequent infusions to prevent bleeding.
The types of activities a person participates in may be another risk for bleeding. Although an active lifestyle is essential for maintaining overall health, vigorous physical activity may somewhat increase the risk for bleeding in persons with hemophilia. Personalizing prophylaxis by raising FVIII or FIX levels during certain physical activities may help prevent or reduce injury-related (traumatic) bleeds.
Age at the start of prophylaxis and the presence of a target joint also contribute to bleeding risk. Hemophilia treaters continue to debate the ideal age for beginning prophylaxis. Yet they all agree that it is not a good idea to delay prophylaxis until someone experiences several bleeds— particularly, multiple bleeds into the same joint (a target joint). Once this situation develops, personalized prophylaxis may be needed to reduce a pattern of frequent bleeding.
Finally, FVIII or FIX pharmacokinetics (PK) has a major impact on bleeding risk. PK is a scientific approach to study and describe what the body does to a drug—in this case, clotting factor concentrate. Because this topic is so important, let’s consider it separately.
Your Pharmacokinetic Profile
One of the best tools we have for personalizing prophylaxis is a person’s unique PK profile. This profile, derived from blood samples taken before and after factor infusion, provides information about how your body uses factor concentrate. The main determinants of PK are how often you infuse and how long FVIII or FIX remains active afterwards.1
Following an infusion, factor level increases, with the increase determined by the amount of factor administered and the patient’s response to the infused dose. This information is used to calculate the incremental recovery. For example, a FVIII injection of 50 IU/kg body weight would be expected to increase the FVIII level to 100% of normal values, indicating an incremental recovery of 2 IU/dL per IU/kg infused. This level quickly begins to fall, however, and after about 12 hours for FVIII and 24 hours for FIX, the level drops to 50%.This decline is called factor half-life, meaning that your body has used up half of the infused factor.
Figure 1. Factor VIII Half-Life Levels

Figure 2. Factor IX Half-Life Levels

Although incremental recovery is fairly consistent among persons with hemophilia, factor half-life varies widely. In fact, a clinical study of prophylaxis found that some people have a half-life that is more than 2 times longer than that of someone with the shortest half-life. Personalizing prophylaxis for someone with a short factor half-life may involve infusing more often. Persons who are physically active may also benefit from personalizing prophylactic infusions. This strategy has worked well for several of my former patients.

On the other hand, persons with longer than average half-lives and those who are inactive because of severe joint disease or a sedentary lifestyle may be able to infuse less frequently. Ed* is a retired office worker with hemophilia A who has arthritis in both knees and uses a walker for mobility. He started prophylaxis at age 57 to reduce frequent knee bleeds. Although this regimen was effective, thrice-weekly infusions were a struggle for him, and he often missed doses. We measured his personal half-life and found he was at the high end of the normal range. We leveraged this long half-life and modified his FVIII prophylaxis regimen to every third day infusions. Since personalizing prophylaxis a year ago, Ed’s annual bleed rate has decreased from 24 to 30 to just 2.
Optimizing Success
An essential component of any personalized prophylaxis regimen is good adherence.1
Prophylaxis is unquestionably time-consuming and often inconvenient. However, bleeding resulting from missed doses is even more burdensome and can have negative consequences.
Here are a few suggestions for optimizing your success:
  • Become an educated healthcare consumer by learning about your bleeding disorder and current treatment regimen. Consulting your local hemophilia chapter may be an important first step.
  • Record all infusions in an electronic or paper bleed log and share this information with your healthcare providers.
    Discuss your personal goals and aspirations with your healthcare team, making sure to emphasize how you define a successful treatment regimen.
  • If you have questions about any aspect of the treatment plan, contact your hemophilia treatment center (HTC) and speak with a nurse or doctor.
  • If you experience breakthrough bleeding, immediately contact your HTC, as this may indicate a need to modify the prophylaxis regimen.
  • When traveling, make certain to bring extra factor concentrate/infusion supplies in case of flight delays, etc.
Summing up, one size fits all doesn’t necessarily work for baseball caps or prophylaxis. Personalizing the prophylaxis regimen by taking into consideration your individual risk for bleeding and PK profile is key to ensuring a “good fit” that reduces bleeding while avoiding overtreatment. Once you and your physician have personalized your treatment plan, always infuse on schedule and immediately report any breakthrough bleeding. A little fine-tuning of the regimen to account for changes in your lifestyle may be all that is needed to prevent bleeds.
Dr. Valentino is a professor of pediatrics at Rush University in Chicago, Illinois, and the former director of the Rush Hemophilia and Thrombophilia Center. He is now the global head of Hemophilia & Blood Disorders, Global Medical Affairs at Shire.

Interested in more content like this? 
Click Here to subscribe to our Parent Empowerment Newsletter. 
Free to families with hemophilia
* Names and some details have been changed to protect patient confidentiality.
1. Valentino, L. A. “Considerations in Individualizing Prophylaxis in Patients with Haemophilia A.” Haemaphilia 20(5) (2014): 607–15.
HemaBlog Archives
Categories