A new five-year, $3.5 million grant from the National Cancer Institute will kick off a large clinical trial aimed at improving the quality of life and survival of medically vulnerable patients after they receive a transplant of donated blood-forming stem cells. Grantee Dr. Mohamed Sorror of Fred Hutchinson Cancer Research Center will lead the new trial.
“These patients do the worst after transplant. How are we going to change that? From there came the idea of a clinical trial,” Sorror said.
The new trial is unusual in its focus on the needs of older, frailer patients and those with multiple health problems besides cancer, who are usually excluded from cancer clinical trials. It should yield results that could be applied right away to improve patient care, Sorror explained. “I think it carries a lot of promise for patients who come to us with many issues. So that’s why we’re so excited about it,” he said.
Sorror’s research focuses on improving care for these “marginalized” blood cancer patients, especially those who might benefit from a transplant of blood-forming stem cells (such as a bone marrow transplant) from a donor. Transplantation can cure these cancers, but it is a difficult treatment that comes with many health risks. An older patient in poor health who receives a transplant could die early of complications or have gravely diminished quality of life in their remaining years. At the same time, an elderly patient who is otherwise in good health could do very well from a transplant.
While seniors make up the majority of blood-cancer patients, developing optimal treatment regimens for them, and for those with medical complications, is not possible if they are excluded from novel research studies.
“My trial here is totally the opposite. We’re excluding young patients; we’re excluding healthy patients,” Sorror said. “We’re actually focusing for the first time on the patient that has been excluded for too long. We want to open the door for them, to get them better care.”
The new Phase 2/3 trial will enroll 600 participants who are scheduled for a donor (or allogeneic) transplant, and only those who are age 65 and older, have multiple other health conditions and/or have a slow gait — a measure of frailty. In its initial stage, the trial will test several interventions before and after transplant to see which improves quality of life three months after transplant: 1) supportive, or palliative, care focused on relieving physical or emotional symptoms; 2) specialized care for nontransplant-related health conditions (such as heart disease); 3) a combination of both; or 4) standard of care.
Then, the best approach that emerges from the first stage will be compared head-to-head with standard of care to provide definitive evidence of improvement in quality of life. The researchers will also determine whether either intervention can improve patient survival, the use of medical and financial resources, and the patients’ long-term quality of life and function (like avoiding disability or nursing-home care).
The trial will be carried out at multiple institutions around the country. Sorror anticipates being able to begin recruiting participants by early next year.
From this trial, “we can provide enough evidence to say that other institutions might need to follow this [the regimen that emerges from this trial] because it provides more promising results, and get insurance approval for that additional care,” Sorror said.
Sorror acknowledges the “bridge” funding he received earlier this year from the American Society of Hematology to sustain his research on this and related projects during the months he awaited the activation of his new NCI grant.
Susan Keown is an associate editor at Fred Hutchinson Cancer Research Center. She has written about health and research topics for a variety of research institutions, including the National Institutes of Health and the Centers for Disease Control and Prevention. Reach her at firstname.lastname@example.org or on Twitter @sejkeown.