BLUE BELL, PA — Slipstream has co-authored a landmark study on Hereditary Hemorrhagic Telangiectasia (HHT), offering the most comprehensive analysis to date of the economic and clinical burden of this rare genetic bleeding disorder. The study, recently published in the American Journal of Hematology, represents the largest examination of HHT patients ever conducted, analyzing data from more than 24,000 individuals across the U.S.
Drawing on Komodo Health’s Healthcare Map®—a database encompassing records from over 330 million patients—the research found that direct annual medical costs for HHT exceed $450 million nationally. The majority of these expenses are driven by treatments addressing severe bleeding complications, particularly anemia.
“We now know that nearly 60% of patients diagnosed with HHT have bleeding so severe that it causes anemia, and a significant portion of those patients receive frequent IV iron and/or red cell transfusions,” said Tracy J. Mayne, PhD, Senior Vice President of Regulatory and Life Science Research at Slipstream. “The burden for both patients and the healthcare system is enormous, with significant implications for advocacy and drug development.”
The study, conducted in collaboration with Massachusetts General Hospital, Cure HHT, and Diagonal Therapeutics, offers striking cost comparisons. On average, annual per-patient costs exceeded $19,000 for all HHT patients—about 20% higher than those for sickle cell disease. Costs rose to $27,000 for patients with anemia and reached approximately $40,000 for those requiring hematologic support such as iron infusions or blood transfusions.
Other findings revealed that while patients with anemia comprise nearly 60% of the HHT population, they account for around 80% of the disorder’s direct medical expenses. Additionally, HHT patients experience liver transplantation rates 40 times higher than the general U.S. population, largely due to complications from liver arteriovenous malformations.
Slipstream CEO Brandon McKay emphasized the study’s broader significance, noting, “Studies like this one validate how the right data, paired with the right expertise, can redefine what is possible in healthcare. This collaboration is exactly the kind of work we strive to do, partnering with innovators to unlock the power of real-world data and digital platforms.”
HHT currently lacks disease-modifying treatments, highlighting a critical unmet need in the rare disease community. The data from this study may help inform future drug development and policy decisions aimed at improving patient outcomes.
The research highlights the potential of real-world evidence to illuminate hidden burdens in underserved patient populations, paving the way for more targeted and effective interventions.
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