Dear MPN Community:
Re: Additional MF Focused QoL study focused around Stem Cell Transplant
We would like to invite you to participate in the steering committee for a new study in which we seek funding from the federal Patient-Centered Outcomes Research Institute (PCORI). This effort is separate (i.e. different PCORI funding mechanism and different trial), but complementary to the efforts of Ruben Mesa and his team focus focusing on alleviating MPN Fatigue in a randomized clinical trial. This parallel effort includes representatives from the National Marrow Donor Program, and the Center for International Blood and Marrow transplant registry, and MPN investigators focused on stem cell transplant.
Our goal in this study is to comprehensively understand the quality of life in patients who have advanced myelofibrosis to help inform patients and caregivers regarding different therapeutic options they have. As many patients consider different therapeutic options for advanced myelofibrosis, including clinical trials, standard supportive care, or allogeneic stem cell transplant, there is very little to guide them about the relative impact these options will have on their quality of life. Although, as providers, we can give estimates of survival, we cannot advise how that survival will look. In a disease such as myelofibrosis, where symptoms play a major role in the disease, what the patient experiences if often unknown.
We would like to understand this experience. We plan on doing this through several efforts. First, to guide our approach, we would like to survey patients regarding their feelings on transplant (link below). This will explore what factors are important to them when considering a transplant, and why they have or have not considered this therapeutic option. Using this information, we then will design a clinical trial that will evaluate the trajectory of symptoms and quality of life in patients with myelofibrosis. This will help identify which patients are likely to benefit from transplant, and who may be likely to benefit from standard supportive care or clinical trials. We may also identify different areas of concern that could be addressed by interventions or policy change.
The input from the MPN community at large will be critical in designing a subsequent trial to obtain the most relevant information to help patients. We look forward to your insight and suggestions as they will be of tremendous help in designing the most informative trial we can.
Thank you in advance for your time and consideration.
Here is the link to the live survey: https://redcap2.mayo.edu/redcap/surveys/?s=mjAdrIxJtX
Jeanne Palmer, MD
Director of MPN Transplant – Mayo Clinic in Arizona
Ruben A. Mesa, MD Director, UT Health San Antonio Cancer Center