New methods make transplant safer, enhance survival for patients with leukemia and other blood cancers
When Shernan Holtan, MD, began her career as a physician-scientist, patients receiving bone marrow or stem cell transplants for blood cancers faced significant risks. A serious complication called graft-versus-host disease (GVHD) — where the donor's immune cells attack the patient's tissues — was life-threatening for around 10% of transplant recipients. Today, as Chief of Blood and Marrow Transplantation at Roswell Park Comprehensive Cancer Center, Dr. Holtan has ushered in transplant procedures that have nearly eliminated the risk of severe GVHD.
Patients diagnosed with blood or bone marrow cancers usually begin treatment with chemotherapy. However, if chemotherapy alone is unlikely to be curative, your care team may recommend a stem cell transplant. "If we're talking about a transplant, it's because chemotherapy alone might not be enough to cure the cancer," Dr. Holtan explains. Your team carefully reviews test results and the unique molecular characteristics of your cancer to determine the most effective treatment plan.
In the past, stem cell donors underwent a painful procedure in which cells were extracted directly from their bone marrow, typically from the hip. Today, the process is far more comfortable. Donors sit in a reclining chair, and stem cells are collected from their blood—no surgery required.
The hospital stay required for transplant recipients has also undergone significant improvement. Historically, patients stayed in the hospital for four to six weeks. Now, thanks to new treatment protocols, most patients are able to go home in under two weeks. These advancements not only shorten hospital stays but also improve patients’ quality of life, lower the risk of GVHD, and enhance overall survival rates.
Building a new immune system
Previously, patients receiving transplants faced GVHD because the donor’s immune cells recognized the patient's body as foreign and attacked it. To prevent this, patients had to take anti-rejection medications for years, and severe GVHD was often fatal. "When I started about 20 years ago, patients with severe GVHD survived about six weeks on average after diagnosis," says Dr. Holtan. "Understandably, people were concerned, but we're now in a new era. Transplants today are dramatically safer."
Modern transplant strategies now allow nearly all patients, even those without a fully matched donor, to receive a transplant safely. "Our goal is curative therapy by giving the patient a completely new immune system," Dr. Holtan says. "With our current donor selection strategies, we identify suitable donors for approximately 99% of our patients." Preparation for transplant involves chemotherapy and sometimes radiation, mainly to suppress the patient’s immune system and ensure the body accepts the new cells.
Why choose Roswell Park for your stem cell transplant?
A stem cell or bone marrow transplant is an aggressive and complex procedure that requires the expertise of oncologists who specialize in delivering transplant treatments and preventing and managing side effects.
"Think of it like planting a garden," Dr. Holtan explains. "Gardeners don’t just scatter seeds onto hard soil and expect them to thrive. They prepare the soil first. Similarly, we prepare your body so that the new cells have the best environment in which to grow. It typically takes about 16 days for these new cells to begin flourishing.
As the new immune system begins to establish itself, some immune cells become overly active, causing GVHD. The new approach involves administering chemotherapy after the transplant to target these rapidly dividing, problematic cells. Cancer and harmful immune cells tend to grow quickly, making them easy targets for this treatment, which helps protect the healthy cells you need.
With this innovative method, the risk of developing any GVHD is now below 15%, and the chance of experiencing severe, life-threatening GVHD is less than 1%.
What we do to optimize stem cell transplant success
After the transplant, most patients are discharged from the hospital within two weeks. "We encourage our patients to recover at home because recovery is generally quicker and more comfortable outside of a hospital setting," Dr. Holtan says. We provide daily outpatient visits for supportive treatments, such as antibiotics, fluids, or blood products, allowing patients to spend most of their recovery time in the comfort of their own homes.
This patient-centered approach has become standard practice at Roswell Park, significantly improving patient outcomes. In fact, among patients who had transplants at Roswell Park in 2024, the six-month survival rate was 100%. Dr. Holtan anticipates that the one-year survival rate for her allogeneic transplant program will exceed 90%, making it one of the highest rates of survival for any transplant program worldwide.
