Factor VIII and Haemophilia Treatment - A brief history

Blood, the essential life-giving fluid that flows through our veins, has a sophisticated network of proteins and cells working in concert to keep us healthy. Some health conditions can mean that our blood doesn't contain certain things it should. One such condition is Haemophilia, a bleeding disorder caused by the lack of a critical clotting protein, Factor VIII. This article traces the journey of Factor VIII from its origins to the present day, focusing on the risks associated with Factor VIII concentrates used in the 1970s and 1980s.

The Role of Factor VIII in the Body

Factor VIII is a crucial protein in the blood coagulation process, a complex series of events that lead to the formation of a blood clot to stop bleeding. In individuals with haemophilia A, the most common form of haemophilia, the body does not produce enough Factor VIII, which can result in spontaneous or prolonged bleeding. 

Factor VIII Concentrates: A Double-Edged Sword

In the 1970s and 1980s, commercial manufacturers sold Factor VIII concentrates, made by mixing together many thousands of plasma donations. These concentrates were one way in which a haemophiliacs missing Factor VIII could be replaced through simple infusion into the bloodstream. Infusion of Factor VIII, like Cryoprecipitate (Cryo), would replace the missing Factor VIII and help to prevent or stop bleeding. Concentrates were sold as a revolutionary treatment because they were more convenient to use than Cryo, but in reality, they were a foreseeably lethal step backwards.

Concentrates were made by pooling and processing plasma from tens of thousands of people. This method of production created a high-risk path for viral transmission, amplifying infection risk exponentially.

Such unsafe products were used in the UK for over a decade and the results were devastating. Every haemophiliac treated with Factor VIII concentrates in the UK was exposed to Hepatitis C and around 1,250 were also infected with HIV. The treatment that was sold as being more convenient was in fact a death sentence.

Some countries, such as Finland, avoided the infected blood scandal by refusing dangerous Factor VIII concentrates. Instead, they kept Cryo as their primary treatment owing to its greater safety. Finland specifically refused concentrates due to their high risk of Hepatitis transmission, which manufacturers and the medical community worldwide appreciated from the very beginning. Although Cryo was less convenient, because it had to be kept frozen and took more time to prepare and administer, it was thousands of times safer and as effective.

By the end of 1985, the UK only used heat-treated Factor VIII. Heat treatment could kill Hepatitis and HIV present in Factor VIII. But, in the UK heat-treated Factor VIII came too late and the damage of more than a decade of using unsafe products had been done. Factor VIII should have been heat-treated before it was ever made available for patient use.

The successful production of safe Albumin, a blood protein essential for maintaining the body's fluid balance, offers a compelling case. Since the 1940s, Albumin has been subjected to heat treatment, made possible through a chemical stabiliser. This vital step in ensuring safety raises a powerful question: why was Factor VIII not accorded the same treatment before its release for patient use?

The heat treatment of Albumin, a process that involves heating the product to a specific temperature for a particular duration, serves as a gold standard in ensuring the safety of this blood product. However, this wouldn't have been possible without a chemical stabiliser. This chemical compound plays a crucial role in preserving Albumin's structure and function during the heating process, effectively inactivating potential viruses, including hepatitis, without compromising the product's integrity.

Factor VIII concentrate made its entry into medical treatment in the 1970s. Yet, until the mid-1980s, concentrates derived from the plasma of thousands of donors, were not subjected to a similar heat-treatment process. As a result, the high risk of transmission of blood-borne viruses like hepatitis and HIV became a reality.

The key issue here was the need for a suitable chemical stabiliser for Factor VIII concentrate that would allow it to be heat-treated without losing its efficacy. While finding such a stabiliser was undoubtedly challenging, it was a necessary step before Factor VIII should have been made available to patients.

Documentary evidence shows that it was only well into the 1980s that manufacturers began to make more serious efforts to heat-treat Factor VIII.

Owing to the Albumin precedent, heat treatment should have been a prerequisite for releasing Factor VIII concentrates.

Haemophilia Treatment Today: A New Era

Today, we live in a different era for haemophilia treatment, defined by increased safety. Innovations in the late 20th and early 21st centuries have transformed haemophilia care.

The advent of recombinant technology in the 1990s allowed for the production of Factor VIII in a lab, removing the need for human plasma and, with it, the plasma-based risks of viral transmission. These recombinant Factor VIII concentrates have become the standard treatment for haemophilia A.

Moreover, gene therapy, a field that has seen significant advancements in recent years, offers the hope of a long-term or even permanent solution for haemophilia. By introducing a healthy copy of the faulty gene into a patient's cells, this therapy aims to enable the body to produce Factor VIII naturally.

Despite these advances, the shadow of the past still looms large over the haemophilia community. The Factor VIII concentrate crisis of the 70s and 80s is a stark reminder of the need for rigorous safety standards in medical treatments. Never again should scientific endeavour, research or profits trump patient safety.