SEATTLE — Aug. 27, 2026 — A newly funded national clinical trial initiative will gather critical evidence to determine the best treatment strategies for older adults with acute myeloid leukemia (AML), an aggressive, fast-moving blood cancer that leaves a substantial proportion of older patients facing uncertainty about whether to proceed to stem cell transplantation or continue non-transplant therapy.
“For decades, patients, families and physicians have had to make these life-changing decisions without sufficiently strong comparative evidence,” said principal investigator Mohamed Sorror, MD, MSc, a professor in Fred Hutch Cancer Center’s Clinical Research Division and an associate professor of medicine at University of Washington School of Medicine.
“Transplantation may offer the best chance of long-term disease control for some patients, but it also carries substantial short-term risks and long-term burdens. For other patients, modern non-transplant therapies may provide a better balance of survival, quality of life and treatment burden,” Sorror said.
The initiative is supported by $15 million from the Patient-Centered Outcomes Research Institute (PCORI), which announced the funding August 25.
The landmark clinical trial program to be led by Sorror and colleagues across an 18-center U.S. consortium will compare stem cell transplantation with non-transplant therapy after patients achieve remission. The treatment strategies will be compared for overall survival and patient-centered outcomes, including quality of life and function.
For some older adults with AML, disease biology and overall health make the preferred treatment strategy relatively clear. But Sorror estimates that roughly one-third to one-half of older adults with AML do not have a clear treatment strategy.
The new trials will focus on patients in remission for whom both upfront transplantation and non-transplant therapy are medically reasonable options, but no randomized trials have directly answered this question in these three clinically defined groups of older adults with AML.
Patients, caregivers, clinicians and other stakeholders helped shape the study design and the outcomes that will be measured, including survival, quality of life, physical and cognitive function, frailty, disability, treatment toxicity and hospitalization.
“As a clinician, I can tell an AML patient that a transplant is potentially helpful, but then the burden is on the patient to decide whether they want to leave home, disrupt their life and go to a transplant center for treatment,” said Sorror, a physician whose clinical research focuses on helping older patients with blood cancers achieve longer survival while preserving quality of life and function.
“If I can tell a patient there is strong evidence that transplant offers a substantial survival benefit, that burden may be easier to justify. But right now, the lack of randomized trials to help make that decision creates uncertainty and frustration,” he added.
The award builds on more than a decade of multicenter AML research led by Sorror. Over the next few years, Sorror and collaborators will enroll about 800 patients at 18 medical centers across the United States, including at Fred Hutch. The new funding will support randomized controlled studies of three clinically distinct groups:
- Patients with relatively favorable combined disease and health risk, for whom it is uncertain whether any additional survival benefit from proceeding immediately to transplant outweighs its toxicity and quality-of-life burden.
- Patients in first remission with persistent measurable-residual-disease (MRD) and intermediate health vulnerability, for whom both immediate transplantation and continued non-transplant or MRD-directed treatment may represent reasonable strategies.
- Patients with adverse or high-risk AML who also have increased health vulnerability, including frailty, substantial comorbidity or related health limitations, for whom transplantation may improve disease control but may also carry substantial early morbidity and non-relapse mortality.
The project will also examine whether treatment effects differ between patients aged 60-69 and those aged 70 and older.
"My goal for this research project is to provide the evidence clinicians need to make informed treatment decisions for older patients with AML," said Sorror. "When a patient walks into a clinic anywhere in the country, doctors should have a clearer understanding of whether the potential benefits of stem cell transplantation outweigh the risks."
The funding award to Fred Hutch has been approved pending completion of PCORI’s business and programmatic review and issuance of a formal award contract.
PCORI is a nonprofit organization with a mission to fund patient-centered CER designed to provide patients and those who care for them with evidence to make better-informed healthcare decisions.
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Media Contact:
Molly McElroy
mwmcelro@fredhutch.org
Fred Hutch Cancer Center
Fred Hutch Cancer Center is globally recognized for trailblazing discoveries that have helped save more than a million lives worldwide. Based in Seattle, Fred Hutch is the only NCI-designated cancer center in Washington state and is home to Nobel Prize–winning scientists and cancer care experts who have made major breakthroughs in preventing and eliminating cancer and infectious disease — including bone marrow transplantation, immunotherapy and the HPV vaccine. Fred Hutch’s role as a hub for national research initiatives and clinical trial networks helps scale discoveries for greater impact. An independent cancer center, Fred Hutch also serves as UW Medicine’s cancer program. Learn more at FredHutch.org.