PREEMPTIVE STEM CELL TRANSPLANT
REFLECTION TOOL
Thinking about a stem cell transplantA procedure that replaces a person's blood-forming stem cells with healthy donor stem cells, most often used to treat or prevent certain blood cancers and blood disorders. before blood cancer develops can feel overwhelming, especially when there may not be one clearly right answer. This tool is designed to help people with RUNX1-FPD and their families understand preemptive stem cell transplant and the questions they may want to consider. If you have already been diagnosed with blood cancer, talk with your care team about the treatment options that best apply to you.
This isn't intended to tell you what to choose. It can help you feel more prepared to discuss your questions and preferences with your care team. The RUNX1 Research Program (RRP) does not recommend for or against preemptive stem cell transplant.
This topic may not feel relevant to you right now, especially if you are newly diagnosed, and that is okay. Everyone's journey with RUNX1-FPD looks different. Take in what feels useful and relevant to you, and return to this page if and when the time feels right.
A donor stem cell transplant is currently the only potentially curative treatment for blood cancer that develops in some individuals with RUNX1-FPD. In certain circumstances, a stem cell transplant may also be considered preemptively, before blood cancer develops, depending on a person's medical situation, priorities, and feelings about the potential benefits and risks.
Continued monitoring is another option. Some individuals may be eligible to join clinical trials testing other kinds of interventions. These approaches are not currently considered curative, but they may be worth discussing with your care team.
Even if continued monitoring feels right for you now, it can still be helpful to ask your care team what might lead you to revisit the conversation and whether speaking with a transplant specialist would make sense. Having these conversations does not commit you to a particular path. It can simply help you feel more prepared if your situation changes.
This reflection tool is based on a published paper developed by RUNX1-FPD clinicians and members of the RRP community. The framework was created to support informed and productive conversations among patients, their families, and their healthcare teams about whether and when to consider a preemptive stem cell transplant.
Your medical details are an important part of decisions about your care, and so are your personal values and preferences. By values, we mean the priorities and perspectives that matter to you, including how you feel about uncertainty, risk, and the possible benefits and complications of transplant.
You'll consider questions such as:
- How do you feel about continued monitoring while living with some uncertainty about future blood cancer risk?
- How do you feel about undergoing a major medical procedure before blood cancer develops, with the aim of preventing it?
- How do the potential serious complications of transplant affect your interest in exploring it?
There are no right or wrong answers. When you finish, you can email yourself a summary of your responses to help you organize your thoughts for discussion with your care team.
Compare continued active monitoring and preemptive stem cell transplant by exploring key questions you may want to discuss with your care team. Select any question to see both paths side by side.
The graphic below shows how clinical considerations for each approach are weighed together with personal and practical considerations. The importance of each consideration will differ from person to person. It is a framework for discussion, not a recommendation or scoring tool.
To actively monitor blood counts, bone marrow, and genetic changes over time, and revisit treatment options if the person's medical situation or preferences change.
To replace bone marrow stem cells carrying the RUNX1 variant with healthy donor stem cells, with the goal of preventing blood cancers from developing.
Avoids the immediate risks and side effects of transplant while allowing the care team time to monitor for meaningful changes. It also avoids transplant-related effects on fertility for now and may provide more time for family planning.
Has the potential to prevent blood cancer associated with RUNX1-FPD. It may also alleviate bleeding symptoms and other health effects associated with RUNX1-FPD and reduce the psychological burden of living with an elevated cancer risk, particularly for individuals with a strong family history of blood cancer.
The underlying genetic condition remains within the blood system. RUNX1-FPD is associated with a 35-50% lifetime risk of developing a blood cancer. Blood cancers can cause serious complications. Active surveillance does not reduce that risk; it monitors for it. A person with RUNX1-FPD may never develop blood cancer.
A transplant is a major medical procedure that can cause serious complications. Some effects may be long-term, recovery can take months to years, and outcomes cannot be guaranteed.
Monitoring usually continues throughout life. Your care team may recommend regular blood counts and bone marrow testing to watch for early signs of disease progression.
After transplant, close medical monitoring is required, including frequent visits, medications to prevent complications, and long-term follow-up. Recovery varies and may take months to years. Some effects may be long-term.
When blood counts and other monitoring findings remain stable, when no suitable donor is available, or when a person prefers continued monitoring after considering the potential risks and benefits to their unique health situation.
When a person has a suitable donor, a strong family history of blood cancer, signs of disease progression, significant bleeding-related or other effects on quality of life, or other clinical factors the care team identifies as high-risk.
If no donor is currently available, active monitoring is typically the path forward while a donor search continues, if desired. Donor availability can change over time.
A suitable donor is required for transplant. Because RUNX1-FPD is hereditary, potential related donors must be tested to make sure they do not also have RUNX1-FPD. Matched unrelated or other donor types may also be considered, and outcomes with these donors have improved significantly. Finding the right donor can take time and is not guaranteed.
Monitoring does not reduce the blood cancer risk associated with RUNX1-FPD. It allows the care team to watch for changes over time, but clinicians cannot yet predict with certainty who will or will not develop cancer, or when.
Transplant may prevent blood cancer, but carries real risks and long-term effects. There is currently no consensus on optimal timing or patient selection for preemptive transplant for RUNX1-FPD, and long-term follow-up data are still limited.
May be chosen if disease risk appears stable, if there is limited to no family history of blood cancer, if the caregiving, logistical, or financial demands of transplant would be difficult to manage at this time, or if the person prefers to avoid transplant risks unless clearly necessary.
May be considered when there are concerning changes in blood counts, bone marrow, or clonal or genetic findings; a significant family history of blood cancer; substantial bleeding-related effects on quality of life; or a suitable donor.
Some individuals prefer to hold off on a major medical procedure with serious risks and possible long-term effects. Factors such as fertility goals, family circumstances, quality-of-life considerations, and comfort with uncertainty can all influence this preference.
Some people prefer to act early to reduce their future risk of developing blood cancer. For those with a strong family history of blood cancer, taking action before cancer may develop could provide a greater sense of control or peace of mind.
Regardless of where you are in your thinking, this topic doesn't need to be settled in one conversation. You can revisit it as your preferences, medical situation, or circumstances change.
If you complete the Reflection Quiz, you can email yourself a summary of your responses and print a copy to bring to your care team to help guide a conversation about your questions, preferences, and what, if any, next steps may make sense for you.
We plan to add more resources to this reflection tool over time, including videos. Have questions about the tool, or want to share feedback? Contact Dr. Amanda Eggen, Director of Patient Engagement & Clinical Programs.
Questions about your individual care or whether transplant may be appropriate for you should be discussed with your care team.
For people with RUNX1-FPD, the decision to pursue a preemptive stem cell transplant is complex and deeply personal.
A new framework helps patients and their care teams navigate it together.