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Takeda Continues to Advance the Diagnosis and Treatment of Bleeding Disorders at ASH 2022

By Jorge Caicedo, M.D., Senior Medical Director Hematology, U.S. Medical

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More than 47,000 people are living with bleeding disorders in the U.S. today. For more than 70 years, Takeda has been dedicated to addressing unmet needs in the bleeding disorders community.1,2 We embrace a patient-first, personalized approach and continue to explore ways to improve the standard of care for people with hemophilia and von Willebrand disease (VWD), including harnessing real-world data to help healthcare professionals make more informed decisions about their patients’ care.

In December 2022, Takeda joins the hematology community at the 64th American Society of Hematology (ASH) Annual Meeting in New Orleans to share new research focused on increasing the speed of diagnosis and improving the overall care of individuals with bleeding disorders.

Improving VWD diagnosis through machine learning

For women with VWD, it takes an average of 16 years between the onset of their bleeding symptoms and accurate diagnosis.2 This extensive delay in diagnosis often leads to complications, frustration with not feeling heard, significant impact on quality of life and ultimately a delay in appropriate treatment. Our new research presented at ASH explored the potential of machine learning, using the largest database of people in the U.S. with confirmed VWD, to help identify specific characteristics that can help signal a potential VWD diagnosis to healthcare providers.3

“There are a significant number of people living with symptomatic, undiagnosed VWD,” said Robert F. Sidonio Jr., M.D., MSc., Associate Professor of Pediatrics, Emory University School of Medicine. “Increasing awareness of VWD symptoms, especially in women, and exploring new technologies, such as this machine learning model, are critical to improving earlier diagnosis of VWD and reducing the physical, mental and economic burden faced by individuals who are still searching for an answer.”

Time to diagnosis continues to be one of the most significant hurdles for people with rare conditions, and harnessing new technologies, such as machine learning, is a key part of our commitment to addressing this issue.

Examining the impact of recurrent gastrointestinal bleeding in VWD

People with VWD may suffer with recurrent bleeding in the gastrointestinal (GI) tract. These episodes are difficult to identify and frequently require hospitalization, which can cause financial burden and a poor quality of life. Those who experience this complication, often undergo a variety of costly and invasive procedures including colonoscopies and endoscopies.4

“Studies like these are important to drive awareness of the impact these complications have on people with bleeding disorders,” said Nathan T. Connell, M.D., M.P.H., Associate Professor of Medicine, Harvard Medical School. “Understanding the real-world experience of those who suffer from these conditions, will help clinicians develop personalized treatment plans for their patients, including evaluating potential prophylactic solutions.”

Identifying the risk factors for intracranial hemorrhages

Intracranial hemorrhage (ICH) can be a devastating and life-threatening complication of hemophilia A, so understanding the risk factors of these potential events is critical. We undertook the first study in over a decade to examine the risk factors associated with ICH by analyzing data from the ATHNdataset, the largest database of people with hemophilia A in the U.S.

Hemophilia A treatment has evolved significantly since the last analysis was completed in 2008. Our study revealed that exposure to factor VIII and prophylactic treatment both play a role in reducing the risk of ICH in people with hemophilia A.5

Harnessing the power of research to change the status quo

Takeda is committed to reducing the burdens faced by the bleeding disorders community. Harnessing real-world data to reduce time to diagnosis, advance treatment and mitigate serious complications is just one way we are continuing to drive medical innovation and changing the status quo.

1 CDC. Data and Statistics on Hemophilia. https://www.cdc.gov/ncbddd/hemophilia/data.html

2 CDC. Data and Statistics on von Willebrand Disease. https://www.cdc.gov/ncbddd/vwd/data.html

3 Sidonio RF, et al. Development of a Machine Learning Algorithm for Early Diagnosis of Persons With von Willebrand Disease Using Real-World Data. 64th American Society of Hematology (ASH) Annual Meeting, 12/2022.

4 Connell NT, et al. Real-World Analysis of Healthcare Resource Utilization and Costs among Patients Diagnosed with Von Willebrand Disease and Angiodysplasia. 64th American Society of Hematology (ASH) Annual Meeting, 12/2022.

5 Hu B, et al. Risk of Intracranial Hemorrhage in US Patients with Hemophilia A: Real-world Retrospective Cohort Study Using the ATHNdataset. 64th American Society of Hematology (ASH) Annual Meeting, 12/2022.