Hemophilia Notes

Music is figuring prominently in the movies lately: A Star is Born, the third remake with Lady GaGa and Bradley Cooper, is a musically and emotionally rich film about how success changes people. Bohemian Rhapsody… need I say anything at all? A biopic about the irreplaceable and eternal Freddie Mercury, who has frequently been rated as having one of the greatest voices in rock history and who created one of the often-rated top 10 rock songs of all time. He’s my second favorite singer (Jim Morrison of the Doors being first).

Music has long been an art form to express emotions and thoughts that are not easily given to words. Music is so tied to emotions that many of us can hear a song, and remember a place or event that happened when we first heard it. It taps a different part of our brain than reading; it can express feelings that we cannot put into words, unless a verse accompanied by music.

For example, “Love of My Life” was Freddie Mercury’s favorite song, according to one biography, because despite his fame, he often felt alone and mistrusted many people around him.

The bleeding disorder community also has its own music, designed to give a voice to feelings that are not easily expressed. Sometimes music with lyrics, sometimes just music. Blood Vibrations is an on-going, grassroots collaboration with various talented artists in the community, to provide a forum for creativity, expression and learning. With songs like “I Am Alone,” “Everybody Counts,” “Patience is a Virtue,” “Alone,” you can see there are some deep feelings to explore and share. Pain and suffering are often doorways to these deep feelings and dark thoughts, and music is one way to relieve and process these. The artists may even be people you know, like Jenny Massey, Kevin Finkle and Jozef Zwier, though many are using pseudonyms, like Cell Division and Bleeder. Listen to their songs, and understand bleeding disorders more. I write books on hemophilia, but there is great value in hearing music about it.

I love music; I am picking up my piano again, after a 5-year hiatus, inspired by these artists. I play classical piano but am also attending a Metallica concert tomorrow in Raleigh, North Carolina. Music has many voices, all valid! If you haven’t listened to Metallica, I would recommend them. When I heard “For Whom the Bell Tolls,” I could have sworn I heard Bach fugues in it. Sure enough, reading their bio, I learned that the late bassist Cliff Burton studied Bach. He died at age 24 in 1986 in a bus accident, but the band survived and remains a top draw in heavy metal.

Read about music too. I love bios about musicians, to learn what inspired them, how they crafted their songs, what paths their lives took to get them where they are.

My favorite rock bios: Life, by Keith Richards (Rolling Stones)— the best. Intelligently written, brilliant and funny. Weaves music theory into explaining why the Stones were so successful with their music, and who influenced them. Born to Run by Bruce Springsteen—it was a long road with many hardships and lessons to become someone who I think is the hardest working person in rock. The Paths We Choose by Sully Erna (Godsmack)—expelled from preschool for fighting, Sully only hit fame when he trusted his gut instinct and struck out on his own, and oddly, didn’t think he could sing. Mercury and Me by Jim Hutton—the movie took a lot of liberties with Freddie’s life; this one tries to balance out factor and fiction from the viewpoint of Freddie’s partner at the end of his life. No One Here Gets Out Alive: the Biography of Jim Morrison by Danny

Sugarman and Jerry Hopkins—This one doesn’t have a happy ending, but it documents the rise and fall of the 1960s great rock band, their influence and the sad outcome of one of the greatest front men, and poets, in rock.

For fun? Slash by Slash and Dancing with Myself by Billy Idol. Hey, why not?

Explore music. Which is your favorite genre? Band? Singer? Writer? Why? What one song moves you like no other? Music unites like almost no other art form. I will see that for myself when I disappear into a crowd of 50,000 tomorrow, all singing with, cheering at and feeling the power that is Metallica.

 

Explore Blood Vibrations too. You too can submit a song; it doesn’t even have to be about a bleeding disorder. Send to bloodvibrations@yahoo.com.

I’ll be waiting to hear it!

  Long Live Rock!

 

 

 

The Last Hemophilia Princess

The English royals have always interested Americans, even though we gave them the boot back in 1776. “The Crown” is a popular Netflix show, and the princesses, from Diana to Kate and now Meghan, seem to fascinate us. The hemophilia community is even more fascinated, as the most famous carrier in the world must be Queen Victoria, who up until recently was the longest reigning monarch in English history. Only one of her nine children, Leopold, had hemophilia, and she passed along the gene to two daughters, who later had children with the hemophilia gene, including the famous Alexei Romanov, son of Nicholas II, the last tsar of Russia. The Romanovs and Alexei’s story makes for astounding history, and indeed, may have changed the course of world history (order Alexis: The Prince Who Had Hemophilia to learn more).

But who was the last princess? Our researcher nonpareil, Richard Atwood, shares a review about a book on Beatrice, based on the book by Matthew Dennison, The Last Princess: The Devoted Life of Queen Victoria’s Youngest Daughter (2007). 

Over 18 years of marriage, Queen Victoria had four sons and four daughters, aged four to 17. She delivered her last child, Beatrice Mary Victoria Feodore, on April 14, 1857. For the second time, Dr. John Snow administered chloroform as an anesthesia for the birth. Beatrice was a bright and precocious child. While she began lacking playmates as her older siblings were marrying or leaving the household, Beatrice had her dogs, cats, birds, and ponies as company. Her father, the Prince Consort, died in 1861 when she was only four. Her mother would grieve as a widow for the next 40 years. By default, Beatrice became her mother’s comforter, companion, and caretaker.

Beatrice attended her first grown-up meal in 1863, and regularly sat at the Queen’s dinners beginning at 15. Her older brother, Leopold, was a part-time invalid by 13, often confined to bed, due to his hemophilia. Beatrice herself developed acute rheumatism and lameness by her early twenties. She visited European spas to be treated with massage and healing water, plus, later in life, took trips to the warmer Mediterranean. She also had neuritis in her hand, right arm, knee, and foot. Her sister, Princess Alice, who was in poor health due to rheumatism, neuralgia, tiredness, and headache, died of diphtheria in 1878 when only 35. John Brown, the Queen’s companion, died in 1883 after 19 years of service. Leopold died in 1884.

Queen Victoria ended any potential romance between Beatrice and Louis of Battenberg, but allowed the marriage of Beatrice to Henry of Battenberg, called Liko, in 1885, provided that Henry give up his independence, military career, German nationality, plus live with Beatrice and the Queen. Beatrice replaced her sisters, and Liko replaced Leopold, to serve Queen Victoria. Beatrice was the shyest of the 9 siblings, and also the most devoted of the daughters. She had a retiring and unassertive manner, was uncomplaining, and had a habit of avoiding eye contact. Beatrice was radiantly happy with Liko, and, following a miscarriage in 1885, had three sons and a daughter: Alexander, Victoria Eugenie (“Ena”), Leopold, and Maurice. Leopold (1889-1922) had hemophilia. The birth of Ena, a hemophilia carrier, was assisted by forceps and occurred at Balmoral, the first royal birth in Scotland in 300 years.

In 1896, Liko volunteered for the Ashanti Expedition in West Africa and died of malaria. Queen Victoria died in 1901. Beatrice, as literary executor, would edit and rewrite the 111 volumes of Queen Victoria’s journal over the next 30 years. Ena, thought not fluent in Spanish, married Alfonso XIII of Spain in 1906, and converted to Catholicism. Her firstborn and male heir, Alfonso, had hemophilia. Three of Beatrice’ssons served in the military during the Great War. Maurice died in 1914 at Ypres. Leopold died in 1922 following an emergency operation complicated by his hemophilia. Beatrice suffered from rheumatism and bronchitis, unable to walk and breathe. She died an invalid and a widow of 48 years in 1944, the last princess, at 87 years.

Hemophilia is no longer in the English royal family, which is why you don’t hear about it anymore as the “royal disease.” The heirs of Beatrice and Alice who carried or had hemophilia are all gone.   

 

Hemophilia: The B Sci-Fi Movie

I love sci-fi as a movie genre, and am a big fan of the 1950s classics (War of the Worlds, The Amazing Colossal Man, The Day the Earth Stood Still), and even the silly B-movies (Plan 9 From Outer Space), right up to 2001: A Space OdysseyAlien (all time greatest) and hey, even Prometheus. I never knew hemophilia was ever mentioned in sci-fi but it has been!

Our esteemed writer Richard Atwood is a sleuth at finding obscure references to hemophilia. And he found one in an old sci-fi film–with some prominent cast members.

The film is the 1966 Planet of Blood (a.k.a. Queen of Blood) and is about a space-vampire who is a queen on her planet. But her spaceship is in trouble; so Dr. Farrady (starring Basil Rathbone) at the International Institute of Space Technology sends two rescue teams of American astronauts (played by Dennis Hopper, Judi Meredith, and John Saxon) travel from the Moon to Mars in 1990 to search for unidentified aliens in distress.

The astronauts locate only one mute, green-skinned, female alien (Florence Marly) in a red-bodysuit, with platinum blood hair, picked up from the Martian satellite Phobos. The alien must live on human plasma, which leads to the deaths of two astronauts who were hypnotized first, while the crew return to Earth. She has a secret plot to overrun the Earth by laying as many eggs as possible on the rescue ship that recovers her from her crashed interstellar spaceship.

Disdaining human food, however, the alien queen needs human blood to survive. She hypnotizes male crew members, one by one, and sucks them dry. Ordered not to kill her, the surviving astronauts fear for their lives from the killer in their midst. Spoiler alert!! They accidentally end her life by cutting her skin, and she bleeds to death. Her blood is green.

The crew believe that she is what humans would have become if we had evolved on another planet–hemophiliacs.  This female vampire is labeled a hemophiliac, by the crew, and some sort of royalty. She’s dead, but like a queen bee, she leaves behind her red eggs, with “consequences left to the imagination,” Richard writes.  

Interesting take on interstellar aliens, vampires, and hemophilia!  It’s now a cult movie, and you can order it on Amazon.   

Why You’re Afraid to Switch Products

Many consider the new year to be a great time to make life changes. With new factor products in the marketplace we thought this article on the pros and cons of switching products would be helpful to our readers. Enjoy! 

by Cazandra Campos-MacDonald
originally published in PEN, November 2018 

 A few myths about hemophilia linger in the community: “Only men can have hemophilia.” “A person with hemophilia will bleed faster than someone without hemophilia.” “People with hemophilia can’t play sports.” “You will outgrow hemophilia.” Belief in these myths can spread fear, and can lead to harmful biases or behaviors: for example, prohibiting a child from playing sports, or not believing women who claim to have bleeding issues.

One fear that is still felt by many people with hemophilia, particularly those who’ve had an inhibitor, is the development or recurrence of an inhibitor as a result of switching factor products. Though there is some evidence to support this,1 we also know that the risk of developing an inhibitor as a result of switching products is very small—too small to accurately measure. Yet many parents give this risk more weight than it deserves. Fear clouds their judgment when weighing the benefits of finding a better product and treatment regimen with the risk of contracting an inhibitor.

The fear of switching products became ingrained in my mind when my oldest son, Julian, was diagnosed in 1996 with a low-titer inhibitor at age one. I learned to infuse Julian with a first-generation factor VIII product (from which he developed the inhibitor), and after two and a half years of daily infusions, his inhibitor tolerized. My husband and I kept Julian on his treatment regimen while also gathering information about new factor products entering the marketplace. First-generation recombinant factor products reconstituted into 10 cc volumes, while some second-generation products reconstituted into only 2.5 cc. Yet our fear of triggering an inhibitor by switching products outweighed the promise of quicker infusions.

My second son, Caeleb, developed a high-titer inhibitor at age 11 months. He eventually began immune tolerance therapy (ITT) on a recombinant factor product, and after a couple of years of daily infusions, we moved him to a plasma-derived factor, hoping to lower his inhibitor level. Given the many complications Caeleb endured over the years, I feared that switching to a different factor product would cause his titer to spike. And daily infusions meant accessing his port every morning and pushing 30 cc of product from four vials of factor concentrate. This was not conducive to self-infusing, although he stopped experiencing episodic bleeds. Not having bleeds regularly was a great trade-off.

Whenever Caeleb visited the hemophilia treatment center (HTC) to have labs drawn, I brought along an extra-large plastic bag of his factor and supplies. Recently, Caeleb’s hematologist, Dr. Shirley Abraham, suggested, “I think it’s time to switch Caeleb to Hemlibra.”2 I was stunned. Another switch? I honestly couldn’t understand why Caeleb needed to switch. Even though Hemlibra® is not a factor product, I realized that the fear of switching was alive and well in my mind. Caeleb still had an inhibitor, and not knowing how a new treatment would affect him was nerve-wracking. Dr. Abraham pointed to the extra-large bag and said, “That is why it’s time to switch.” Over the years, I had grown accustomed to carrying all those supplies for an infusion, and to accessing his port daily. With Hemlibra, a month of product and supplies fit into a small container, and Caeleb receives one subcutaneous injection weekly into his thigh—no more infusions into his port. In that moment, speaking to Dr. Abraham, I understood. Caeleb’s quality of life could be even better if I trusted medical advice and didn’t give in to old fears.

Products are chosen—and kept—for many reasons. For example, in families where more than one person has hemophilia, the choice may be based on another family member’s experience. And if a treatment regimen or product is working well, change may not be needed. But what if you can do better? A common response to changing products came from one mother on social media. “Same manufacturer for 18 years because [the product] works. No reason to change.” Claudia Mackaron, a retired HTC nurse coordinator in Albuquerque, New Mexico, says, “Even though a new product could be more beneficial, the old saying ‘if it ain’t broke, don’t fix it’ creeps in. It’s very frustrating as a clinician to fight with patients in switching, knowing [a new product] could help them.” Fear of the unknown may stop a patient from changing products even if the data shows that a specific product could be a better fit. “Psychologically, the fear of the unknown, and a potentially harmful and difficult unknown, can shape bias tremendously,” says Dr. Mike Wang, associate professor of pediatrics at University of Colorado’s School of Medicine. Inhibitors are frightening, and it’s possible someone could develop an inhibitor after switching products. The fear lives on, even with no clinical evidence that the inhibitor resulted from a new product.

Why do some people embrace change without fear? Debbie Porter has an adult son with hemophilia and inhibitors who always “thought the idea of staying with the same product forever was counterproductive to advancing new and better treatments.” Debbie wanted more for her son Matt, who suffered for years from inhibitor complications. Matt infused recombinant products, plasma-derived products, and bypassing agents over the years, so switching wasn’t a fear. The day Hemlibra became available, Debbie immediately requested it for Matt. He has been bleed-free for seven months now, and his veins get the rest they desperately need. And of course, because Hemlibra isn’t factor, it can’t cause an inhibitor to factor VIII. Yet some people may be reluctant to switch, because they experience few complications with their inhibitors and their current treatment works. But for people like Matt and Caeleb, the severity of complications pushes them to anxiously wait for new products that promise better results.

 We are learning more about why inhibitors form, and who is most likely to develop one. Physicians can identify patients who are more susceptible to inhibitor development based on genetics, environmental factors, race, and family history. But people seemingly not at high risk may still develop an inhibitor, and this has continued to feed fears about treatments and products. One HTC provider admitted that for years, he and his colleagues encouraged patients without inhibitors not to switch factor brands because they might get an inhibitor. While there is a risk of inhibitor development when switching products, “current evidence does not suggest that switching products significantly influences inhibitor development,” declared a finding in the Eleventh Zürich Haemophilia Forum.3 Yet fears persist, even in the face of scientific evidence.

Letting go of old beliefs isn’t easy. Once a myth has taken root in your belief system, it takes a lot of effort to remove it. We remain captive to treatments, protocols, and products that may not be the most effective. Staying where we are now—based on assumptions not rooted in the facts—prevents us from embracing the many possibilities that exist today.

Have the courage to overcome fear and seek out what’s in the best interest of your loved ones. Gather the facts, and communicate your treatment needs and concerns to your physician. Trying a different regimen or product may change your life.

 

  1. F. R. Rosendaal, et al., “A Sudden Increase in Factor VIII Inhibitor Development in Multitransfused
Hemophilia A Patients in the Netherlands: Dutch Hemophilia Study Group,” Blood 81, no. 8 (1993): 2180–86, available at www.bloodjournal.org. Thierry Calvez, et al., “Recombinant Factor VIII Products and Inhibitor Development in Previously Untreated Boys with Severe Hemophilia A,” Blood 124, no. 23 (2014): 3398-3408, available at www.bloodjournal.org.
  1. For more on Hemlibra, see Paul Clement, “ACE910: The First Disruptor,” PEN, February 2018, 4.
  2. Elena Santagostino, et. al., “Switching Treatments in Haemophilia: Is There a Risk of Inhibitor Development?” European Journal of Haematology 94 (2014): 284–89.
 
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