The History of Hemophilia and Its Evolution Toward Preventive Care

Episode 3


About 40 years ago a mother brought her 6-month-old child into the hospital. He was covered in bruises. Dr. Victor Blanchette, a pediatric hematologist at the Hospital for Sick Children in Toronto, Canada, met the patient that day and, following a severe hemophilia diagnosis, has treated the patient ever since. Dr. Blanchette recounts how during the patient’s childhood in the 80s, the approach to treating hemophilia was reactive, not proactive. This meant that normal childhood activities could lead to devastating bleeds. During this episode, we walk through the history of hemophilia and how its evolution toward preventive care has had an incredible impact on this patient and so many others.

 Victor S Blanchette, SCM, FRCP(C), FRCP

Dr. Victor Blanchette is Professor of Pediatrics, University of Toronto, Canada. He is a Staff Pediatric Hematologist in the Division of Hematology/Oncology and a Senior Associate Scientist (Emeritus) in the Research Institute at the Hospital for Sick Children, Toronto. He is the McCaig Magee Family Medical Director of the SickKids-Caribbean Cancer and Blood Disorders Initiative in the Centre for Global Child Health at the Hospital for Sick Children.

After completing his medical training at the University of Cambridge and St Bartholomew’s Hospital in the United Kingdom, Dr. Blanchette pursued subspecialty training in pediatrics at Johns Hopkins Hospital in Baltimore, USA, followed by fellowship training in pediatric hematology/oncology at McMaster University Medical Centre in Hamilton, Canada.

Dr. Blanchette’s research interests are in the area of the congenital and acquired bleeding disorders of children. He is Co-Director of the Pediatric Comprehensive Care Hemophilia Program at the Hospital for Sick Children, and Chair of the International Prophylaxis Study Group (IPSG). Dr. Blanchette is recipient of the Canadian Pediatric Society 2009 Alan Ross Award, the Canadian Blood Services 2010 Lifetime Achievement Award, the American Society of Pediatric Hematology/Oncology 2012 Distinguished Career Award, the 2018 University of the West Indies (UWI) Vice-Chancellor’s Award, the 2019 Hemostasis and Thrombosis Research Society (HTRS) Lifetime Achievement Award and the 2019 Canadian Hematology Society (CHS) Lifetime Achievement Award.  In 2018 Dr. Blanchette was awarded the Order of Barbados (Silver Crown of Merit), the country of his birth, for his contributions to the field of medicine. Dr. Blanchette is an elected Fellow of the Royal College of Physicians and Surgeons of the United Kingdom.



The History of Hemophilia and Its Evolution Toward Preventive Care

Dr. Raj Bhardwaj: This season of DDx is sponsored by Sanofi. The case study you’re about to hear is a real patient story. 

About 40 years ago a mom brought her 6-month-old baby into a hospital in Toronto, Canada. He was covered in bruises.

Dr. Victor Blanchette: In fact, when I first saw him, it was clear that he was bruising abnormally in places we don’t see at about age 6 months. This was extremely concerning to her because she wondered that something serious might be going on.

Dr. Bhardwaj: This is DDx, a podcast from Figure 1 about how doctors think.

I’m Dr. Raj Bhardwaj.

This season, we’re taking you inside the minds of doctors who specialize in the most common type of hemophilia, Hemophilia A — an inherited bleeding disorder that prevents the blood from clotting properly.

Today’s case is from Dr. Victor Blanchette, a pediatric hematologist at the Hospital for Sick Children in Toronto, Canada.

Dr. Blanchette has been compensated by Sanofi for his participation in this episode.


Dr. Blanchette: This story I’m now going to paint for you is really a journey from the dark days to the good days.

Dr. Bhardwaj: The baby you heard about a moment ago was Dr. Blanchette’s patient. He was diagnosed with Severe hemophilia A in the mid 1980s.

Back in the 80s, the approach to treating hemophilia was different than now. Dr. Blanchette saw his young patient through plenty of dark days. Throughout his childhood there were many setbacks.

Dr. Blanchette: This little fellow had a first spontaneous bleed into his left knee around about 18 months, which is very typical.

Dr. Bhardwaj: This continued throughout the boy’s childhood. These bleeds can be caused by everyday activities. Like bumping a knee. Or twisting an ankle. 

Dr. Blanchette: His first 10 years of life were very difficult because of these recurrent bleeds.

Many visits to the hospital, many, many times where he had pain and was in a wheelchair. And in those early school days, he could not keep up with his peers. He told me that he wasn’t allowed to play gym. And that really bothered him. And you could imagine if he was your son, how tough that would be.

Dr. Bhardwaj: Dr. Blanchette monitored his patient. Whenever he had a significant bleed, the boy was treated with an infusion of a protein called factor VIII (8) that helps blood to clot.

But these treatments could only be given after the fact. They weren’t preventative. So the patient’s bleeds continued — and they were painful. And to complicate matters, the patient had developed an inhibitor to the treatments he’d been getting. Dr. Blanchette remembers one especially dark day.

Dr. Blanchette: Around the age of 8 years, he had a devastating bleed when he was tobogganing. In Canada, in the winter, little kids do that on hills. And this was a trauma-related bleed. He fell off the toboggan. And that whiplash injury caused bleeding in his spinal cord. If we were not successful in controlling the bleed, he would have been paralyzed for life. A devastating situation, and his mother was aware of that.

Dr. Bhardwaj: The inhibitor meant that the usual treatments were no longer working. Dr. Blanchette needed to find another way to deal with this life-threatening injury. So he decided to try an experimental treatment — but it wasn’t available in North America. He didn’t have much time.

Dr. Blanchette: I should emphasize the critical nature of this bleed, because if not treated in a way to stop bleeding within 48 hours of the injury, then there would be severe consequences. I recall being on the phone several times a day in that first 48 hours as we sourced that product to treat this little boy who was in hospital.

Dr. Bhardwaj: The child’s bleeding stopped, and his spine wasn’t damaged. 

Dr. Blanchette: Occasionally, boys with severe hemophilia have life-threatening bleeds into the brain and you may have only hours, very little time, to make the diagnosis and start effective treatment.

Dr. Bhardwaj: After the tobogganing accident, Dr. Blanchette’s patient started on a “desensitizing protocol” to try to convince his body to accept the factor VIII treatments again. It worked. Doctors could treat the boy’s hemorrhages after they happened.

But what hemophilia patients really needed was a way to prevent the bleeding from happening in the first place.

After years of research, a breakthrough.

Dr. Blanchette: We moved him then from being treated just when there was a bleed. And for several days until recovery to something we call prophylaxis. That is the regular administration of the factor VIII at a frequency that is generally two to three times a week.

Dr. Bhardwaj: At first, IV infusions of factor VIII were done in the hospital but then the patient’s mother learned how to give the intravenous treatments at home. When he was 12, the boy learned how to do them himself.   

Dr. Blanchette: He began to self-administer these infusions very effectively through veins, and I recall when I look back that he learned some of that technique at a hemophilia camp, and there are nurses and doctors who are at that camp and that was a pivotal thing in his life.

Dr. Bhardwaj: Despite starting on preventive treatment when he was about 9 years old, the patient had significant damage to his ankles and knee.  

Dr. Blanchette: So unfortunately, while all of these things were happening with him as a very young boy and then as a teenager, he was also developing damage in both ankles. Some of that bleed may have been very silent. Some may have been related to trauma. 

Dr. Bhardwaj: Through the years, synovitis — which is inflammation of the joint lining — was doing cumulative damage.

Dr. Blanchette: The blood that enters the joint leads to inflammation, destruction of cartilage and bone. We needed to give him an experimental treatment with a radioactive isotope, injected into his knee to really quiet that joint down.

Dr. Bhardwaj: That experimental treatment helped. But by the time the patient was 20, there was enough joint damage from synovitis that he needed surgery on one of his ankles. A few years later he needed surgery on the other ankle.

Dr. Blanchette: We call that synovectomy to remove, to clean out the inflamed synovium. That was helpful. He also recalls that he had two injections in his knee, his target left knee, with a type of gel, a lubricating gel, and that helped as well.

Dr. Bhardwaj: This patient is in his 40s now. He has severe arthritis in his left knee and both ankles. His hemophilia is well-controlled.

Apart from dealing with arthritis, his quality of life has improved a great deal. 

Dr. Blanchette: He did go to university and he graduated with an engineering degree. Quite remarkable considering all that he had gone through.

Dr. Bhardwaj: The use of preventative treatments means that the damaging joint bleeds have become much less frequent.

Dr. Blanchette: He’s actually with a partner. He’s just had a son, who is now 2 months old. He’s incredibly proud. He has a steady job. And his activity is constrained. He uses cycling as a very important activity because he can do that without creating soreness from the arthritis.

Dr. Bhardwaj: You can hear the admiration Dr. Blanchette has for this man who he has known for decades.

Dr. Blanchette: I was very proud of him and very close to him and he to me because of his fighting spirit.

Dr. Bhardwaj: When he thinks of this case, Dr. Blanchette is reminded of the dramatic evolution in hemophilia treatments since the 1980s when he first met this patient.

Dr. Blanchette: We are all human, so a physician or a healthcare worker, they want to help people. They want to cure people. And I recall those multiple visits with him through his first 20 years and you know, hospitalizations, painful bleeding episodes.

And when you have to treat patients like that, your heart hurts. You want to do better. And what has been remarkable for me as a physician with a focus in the hemophilia area, is to see the remarkable advances that have occurred moving from almost no therapy through to these recent years, just in the last few years — the spectacular advances in science, bringing us these options of treatment.


Dr. Bhardwaj: If a 6-month-old baby with severe hemophilia came into Dr. Blanchette’s care today, the plan could look very different, depending on the case

Dr. Blanchette: So if I were engaged with a similar young baby now, in the era in which we are living where there is so much access to information  from multiple sources, I would feel it important to tell them the facts about the history of hemophilia, not sugarcoating anything, but reassuring them that with the therapies we have and the multidisciplinary approach, they can be assured that the outcome is really likely to be a very good outcome if they have access to these treatments.

Dr. Bhardwaj: Thanks for speaking with us Dr. Blanchette.

This is DDx, a podcast by Figure 1.

Figure 1 is an app that lets doctors share clinical images and knowledge about difficult to diagnose cases.

I’m Dr. Raj Bhardwaj Bhardwaj, host and story editor of DDx.

Head over to, where you can find full show notes, photos and speaker bios.

This season of DDx is sponsored by Sanofi.

Sanofi is a global healthcare company that develops and delivers medicines and vaccines for millions of people around the world.  Thanks for listening!