von Willebrand disease
On April 17, the global bleeding disorders community celebrates World Hemophilia Day, which aims to educate the public about the many challenges faced by people with these rare disorders. The theme this year is “Access for All: Prevention of bleeds as the global standard of care.”
Rawda, a mother of four from North Lebanon, has three boys with von Willebrand disease (VWD) and has first-hand experience of the difficulties patients with VWD face in receiving a correct diagnosis and accessing prophylactic treatment.
When Rawda’s oldest son was nine months old, he fell and bled heavily from his nose and mouth. The bleeding would not stop. “We took him to the Islamic Hospital in Tripoli, where tests showed he had a bleeding disorder, but I didn't know what kind it was,” she recalls.
There is a history of bleeding disorders in Rawda’s family. Her father suffered with heavy nosebleeds throughout his life but was never properly diagnosed. “Later, we discovered that von Willebrand disease is present in the family, and it has been passed down through generations. Now it has reappeared in the new generation.” Like their brother, Rawda’s other two sons were subsequently also diagnosed with von Willebrand Disease, as were Rawda and her husband.
Despite her family’s history of bleeding disorders, Rawda and her husband did not know anything about von Willebrand disease. “I knew it was a bleeding disorder, but we didn't know what bleeding disorder it was.”
Before having access to preventative treatment, at least two of her boys would bleed every day. The amount of blood loss was devasting “I really felt a knot in my stomach from the smell of blood. I couldn't smell it or see its colour anymore,” recalls Rawda.
Rawda faced many challenges. Suffering constant nose bleeds, her boys were paralyzed in bed for days. She was so afraid of the disease that she took care of them at home, isolating them from the community, where they did not attend school or have any friends. “They grew up far from other children and do not have social relationships with others,” she admits. “I was so scared for them that I spoiled them all the time, and they depended on me for everything.”
After several years using on-demand treatment, Rawda took her sons to her sons to Beirut, where she met Dr Claudia Khayat, a paediatric haematologist at Hotel Dieu de France Hospital, St Joseph's University, and Professor of Paediatrics at the Saint Joseph University in Beirut, Lebanon. Dr Khayat suggested switching to prophylactic treatment with Octapharma’s VWF/FVIII concentrate, after which the children took the medication twice a week to prevent bleeding.
Today, Rawda is grateful for the switch to prophylactic treatment. “For me, there is a significant difference between my life before and after preventive treatment,” she says. “Before my children took the preventive treatment, their bleeding was frequent, but since they have started using it to prevent bleeding, they have only had 1 or 2 bleeds in a whole year.”
Rawda knows that they will live with VWD for the rest of their lives, but she is grateful that prophylactic treatment has reduced their bleeding rate by more than 80%.
“The prophylactic treatment has improved my children's quality of life,” she said. “After the prophylactic treatment, they started playing, and their memory is much better than before.”
Octapharma is committed to providing new healthcare solutions to advance human life and to providing patients like Rawda and her family with access to the best possible treatments. On June 26, Octapharma will present new clinical data from the recently completed WIL-31 study into the use of von Willebrand factor replacement therapy for prophylaxis in VWD at the annual meeting of the International Society on Thrombosis and Haemostasis in Montreal.
Octapharma has a long-term commitment to improving the treatment of bleeding disorders, as well as to raising global awareness of them. As a company, we are proud to support World Hemophilia Day and to play an active part in the haemophilia community.
von Willebrand disease