September brought plenty of opportunities to learn and connect across the RRP community. In this issue, we share helpful new resources, research updates, and conversations about patient experiences and clinical care. |
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| Sirolimus Clinical Trial Expands to Oregon |
Based on encouraging early observations among the first participants, the Low-Dose Sirolimus Pilot Study has expanded: - Oregon Health & Science University (OHSU) in Portland has begun enrolling.
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A limited number of additional spots have opened at MD Anderson Cancer Center in Houston.
The study is testing whether a low daily dose of a drug called sirolimus, also known as rapamycin, is safe and well tolerated by adults with RUNX1-FPD who have not developed a blood cancer.
The research behind the trial comes from Anupriya Agarwal, PhD, a recipient of the RRP-American Cancer Society LEAP Grant, whose lab is at OHSU. Interested in joining? Contact Amanda Eggen with any questions. |
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Learning about RUNX1-FPD often means learning a whole new vocabulary. Our new glossary offers plain-language definitions to help patients and families understand terms they may hear from a care team, see in medical reports, or encounter while reading about research.
This is a living resource that will continue to grow based on community feedback. If you don't see a term you think should be included, please email us and let us know. |
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| 2027 Virtual RUNX1 Scientific Conference & Patient Meeting |
Registration is open for both the 11th Annual Virtual RUNX1 Scientific Conference (May 4–5, 2027) and the 2027 Virtual RUNX1 Patient Meeting (May 14, 2027).
The Scientific Conference brings together researchers and clinicians from around the world to share the latest discoveries in RUNX1-FPD research.
The Patient Meeting brings together individuals and families for updates on research and clinical trials, as well as opportunities to connect with others in the community.
Agendas and speaker details will be shared closer to the events. |
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| New RUNX1 Pulse Blog:
A Lifetime of Bleeding: What I Wish I Had Known |
Christina, an individual with RUNX1-FPD, shares what she wishes she had known sooner about navigating bleeding issues as a woman. From her first period through diagnosis, family planning, pregnancy, perimenopause, and hysterectomy, her story shows how heavy menstrual bleeding can shape every stage of life. Adapted from a recent medical education webinar, the blog also includes clinical perspectives from Drs. Hilary Whitworth and Robert Silver.
Together, these perspectives offer important insights for patients, families, and clinicians about helping girls understand what normal menstrual bleeding looks like, continuing to seek answers when initial testing is inconclusive, and understanding what an inherited diagnosis could mean across generations. |
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Bleeding gums and tooth enamel issues may seem like ordinary dental concerns, but in RUNX1-FPD, they may be linked to the condition through more than one route. A new insight from the RUNX1 Patient Data Hub explores what our community reported and why the pattern may be relevant to RUNX1 biology. |
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| 2026 RUNX1 Research Update Webinar |
Thank you to RRP Executive Director Katrin Ericson, PhD, and RRP Scientific Advisory Board Chair Nancy Speck, PhD, for guiding attendees through the latest RUNX1 research updates.
The webinar covered progress in clinical trials, emerging areas of research, and what these developments could mean for our community.
If you missed it, the recording is now available. |
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| Family Planning & Fertility Considerations for RUNX1-FPD |
For individuals with RUNX1-FPD, questions about family planning and fertility are deeply personal and often complex.
In this 2020 webinar, a genetic counselor and a fertility specialist walk through reproductive options for families with inherited variants that increase blood cancer risk, including IVF with embryo testing (now called PGT-M), as well as fertility preservation considerations for those facing a blood cancer diagnosis and treatment. While some details have evolved since 2020, the foundational concepts remain accurate, and the recording is a helpful starting point for conversations with your clinical team. The discussion is also relevant to other predisposition syndromes. |
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2027 RRP-ALSF Early Career Investigator Grant |
The 2027 RRP-ALSF Early Career Investigator Grant offers up to $180,000 over three years to support early-career scientists pursuing basic or translational research focused on preventing the progression from premalignancy to blood cancer in individuals with RUNX1-FPD.
Applications are due Thursday, December 17, 2026, by 8:00 p.m. ET. Click below for the application guidelines and the application link. |
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Medical Education Webinar: "A Lifetime of Bleeding: Inherited Platelet Disorders in Women" |
RRP and the Foundation for Women & Girls with Blood Disorders recently co-hosted a webinar exploring heavy menstrual bleeding as an early clinical clue to an inherited platelet disorder. The personal and clinical perspectives shared offered valuable insight into how inherited platelet disorders can shape care across the female lifespan. Thank you to Christina for sharing her deeply personal journey as a woman living with RUNX1-FPD across multiple life stages, and to Dr. Hilary Whitworth (Children's Hospital of Philadelphia) and Dr. Robert Silver (University of Utah) for providing clinical context throughout. |
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