This Landmark HHT Study Could Shift National Attention

We’re excited to share some truly important news for the HHT community.

A major new study done in collaboration between Cure HHT, Massachusetts General Hospital, and Diagonal Therapeutics — just published in the American Journal of Hematology — delivers something we’ve never had before: hard data that proves HHT is a serious, high-impact disease that’s been overlooked for far too long.

The data outlined in this study will be transformational in our efforts to bring more funding and attention to HHT research, treatment, and care.  This publication gives us the incredibly powerful facts and figures we need to change the conversation around HHT.

Educate yourself by reading more below.

Key Findings:

The study, “Characterizing the Healthcare Utilization and Costs of Hereditary Hemorrhagic Telangiectasia,” analyzed real-world healthcare claims data from thousands of HHT patients across the U.S. The numbers speak volumes:

• The average HHT patient incurs nearly $19,000 per year in healthcare costs
• For patients with anemia (57% of our community), that rises to $27,000 per year
• Those needing iron infusions or blood transfusions face over $40,000 annually
• Liver transplants among HHT patients occur at 40 times the rate of the general population

Total U.S. costs exceed $450 million each year!

 

Why This Study Matters:

This is more than just numbers — it’s proof of what we’ve always known: HHT is not rare in its impact — only in its recognition.

This new data shows that the cost burden of HHT exceeds or matches that of several better-known and more prevalent diseases. HHT costs are:

• 20% higher than sickle cell disease
• Comparable to muscular dystrophy
• Comparable to cystic fibrosis

These conditions have long attracted research funding, biotech investment, and federal attention. It’s time HHT does too.

 

What This Means For the Future:

This study gives Cure HHT — and all of us — the tools to:

• Push for increased federal research funding
• Draw the attention of biotech and pharmaceutical companies
• Make a stronger case with lawmakers, regulators, and insurers
• Build momentum for clinical trials and, eventually, approved therapies

Despite its burden, HHT still has no FDA-approved treatments. These findings make that reality impossible to ignore — and impossible to accept any longer. We’re working every day to be the catalyst that changes this.

 

Cure HHT’s Role:

This research wouldn’t have happened without Cure HHT’s leadership and your support. We proudly partnered on this effort from concept to publication — working side-by-side with Dr. Hanny Al-Samkari and Diagonal Therapeutics — to ensure the challenges HHT families face were front and center.

 

“This study provides the first detailed analysis of the healthcare resource demands and costs associated with HHT, highlighting the substantial economic impact this disease places on patients and the health system. The findings underscore the urgent need for treatments that can effectively address the underlying cause of HHT and reduce the complications related to chronic bleeding and anemia, which are major cost drivers requiring hematologic support, hospital admissions, and emergency care.”

— Hanny Al-Samkari, MD

Peggy S. Blitz Endowed Chair in Hematology/Oncology at the Massachusetts General Hospital, Associate Professor of Medicine at Harvard Medical School, and lead author of the study

 

You can read the full press release from Diagonal Therapeutics here.

Slipstream, a technology partner serving the Biopharmaceutical industry who helped co-author this groundbreaking study, also issued a press release today on the findings. You can read their release here.