Speaker: Julien Haroche, MD, PhD
Time: 9:35 AM – 9:55 AM
In the world of Erdheim-Chester Disease (ECD), few names are more widely respected than Dr. Julien Haroche. For more than two decades, Dr. Haroche has led groundbreaking research in histiocytosis, particularly ECD. At this year’s Patient and Family Gathering in Barcelona, his session titled “ECD Treatment Options: Current and on the Horizon” promises to be a powerful overview of how far treatment has come—and what lies ahead.
A Global Leader in ECD Treatment Innovation
Dr. Haroche is based at Hôpital Pitié-Salpêtrière in Paris, France, where he and his team have treated more than 250 patients with targeted therapies—making their program one of the most experienced ECD centers in the world. His clinical research has played a significant role in defining treatment protocols that are now used globally, particularly those involving targeted therapy for patients with BRAF or other MAPK pathway mutations.
In 2012, his team was the first to use these therapies in adults with histiocytosis, a development that changed the trajectory for many patients. Before that, options were limited and outcomes unpredictable. Now, while ECD remains complex, treatment is more tailored, precise, and often more effective.
What You’ll Learn in This Session
This talk will serve as a clear, non-technical guide for patients and families seeking to understand how treatment decisions are made. Dr. Haroche will address several critical questions, including:
- What treatments are available today, and how do doctors determine which one is appropriate?
- What role do genetic mutations—especially BRAFV600E—play in determining treatment?
- How are targeted therapies different from traditional approaches, and what can patients expect in terms of administration, duration, and monitoring?
- What research and clinical trials are underway that might change the landscape in the coming years?
He’ll also cover considerations around treatment intensity, including when it’s possible to take a “watchful waiting” approach and when more aggressive intervention is necessary.
Why This Session Matters
Understanding treatment options can be one of the most empowering aspects of living with ECD. For many patients, the road to diagnosis is long and filled with uncertainty. Gaining clarity around what can be done—and why—often brings a sense of hope and control.
Dr. Haroche has an unmatched ability to translate complex science into language that patients can understand. He treats not just diseases, but people—an approach that aligns closely with the values of the ECDGA and the spirit of this Gathering.
If you or your loved one is considering treatment, currently undergoing therapy, or seeking insight into what the future may hold, this is a must-attend session.
The ECDGA encourages all attendees to bring questions and engage. While Dr. Haroche and his colleagues cannot offer personal medical advice in this forum, the information shared may help you have more informed conversations with your care team at home.