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Personalized Therapeutics Improve Outcomes for Leukemia

Amy Vittetoe and family
Amelia (Amy) Vittetoe, pictured here with her family, was diagnosed with acute myeloid leukemia (AML) in May 2007. After aggressive chemotherapy and a stem cell transplant, she is now in remission.

Researchers at the University of Chicago Medicine Comprehensive Cancer Center are working tirelessly to identify genetic pathways involved in hematologic malignancies, such as leukemia, because by understanding and characterizing these pathways, we are able to develop novel, molecularly targeted treatments.

Patients from the Chicago area and beyond know that when they come to the University of Chicago for treatment, they will receive the unique expertise and leading-edge treatment strategies that have made our hematologic malignancies program a program of distinction for pediatrics, as well as for adult patient care.

Novel Treatment Approach Helps Teen Swimmer Dive Back in Pool

Kris Egebrecht knew something was wrong when Brandon, her teen who loves to swim, could not get out of bed.

"I slept for 15 hours a day for 3 days straight," Brandon said.

Kris took him to an urgent care clinic near their home in Northwest Indiana and was convinced a blood test would reveal a diagnosis of mononucleosis, a viral illness common in teens. Instead, Brandon tested positive for acute myeloid leukemia (AML).

While Kris grappled with what she called her "worst nightmare," Brandon's primary care physician set the wheels in motion for Brandon's treatment. With an aggressive disease like AML, time is of the essence.

Brandon's pediatric oncologist referred him to Comer Children's Hospital at the University of Chicago, where Brandon was diagnosed with a genetic subtype of AML that was first described at the University of Chicago in 1983 by Michelle M. Le Beau, PhD, and colleagues.

Brandon was enrolled in an innovative clinical trial affiliated with St. Jude Children's Research Hospital in Memphis, Tenn., that utilized an aggressive treatment plan. The drug clofarabine, which is effective in patients with relapsed AML, was administered as the first cycle of induction therapy, followed by four cycles of standard chemotherapy.

"The goal of this study is to achieve better remission rates by using this more intense drug as part of the initial therapy for newly diagnosed patients," explained Jennifer McNeer, MD, associate professor of pediatrics.

Overcoming Setbacks

Brandon's first treatment was the most challenging. He developed tumor lysis syndrome, which occurs when chemotherapy very quickly breaks down leukemic cells, explained Dr. McNeer. It caused Brandon's kidneys and lungs to fail. Dr. McNeer said Brandon's care team worked together to support him until his kidneys resumed their normal function.

Kris said she was impressed with how Brandon's care team explained every step of the treatment to the family. "They answered questions before I even knew I had questions."

Brandon's treatment ended earlier than initially planned because studies have demonstrated that four cycles of chemotherapy are as effective as five, according to Dr. McNeer. "Survival rates in AML are certainly better than they were years ago, but we are always working to improve them," she said. "It's great we were able to offer Brandon a unique treatment protocol."

Brandon was diagnosed with leukemia in April 2011. By August, his treatment was completed and he began his junior year of high school.

"I've grown a lot from this experience," Brandon said. "It has definitely changed my perspective."

Stem Cell Donation Saves Life of Adult AML Patient

Another success story in AML revolves around a woman whose life was forever altered by a stranger.

In May 2007, Amelia (Amy) Vittetoe was feeling tired and thought her thyroid medication needed adjusting. But after extensive blood and bone marrow studies confirmed a diagnosis of AML, she was immediately admitted to a local hospital near her home in Crystal Lake, Ill.

An attending physician suggested that Vittetoe transfer to a hospital that was better equipped to treat her disease. She considered several Chicago area hospitals, but chose the University of Chicago, a decision that she said saved her life.

"That was the farthest hospital that we could have gone to, but I know for sure that it was the best," she said.

Vittetoe's multidisciplinary care team, including Wendy Stock, MD, professor of medicine, used molecular diagnostics to reveal that Vittetoe had the same cytogenetic abnormality as Brandon Egebrecht. This particular subtype of leukemia is usually associated with a relatively good prognosis with standard chemotherapy.

Vittetoe's treatment kept her in and out of the hospital and required frequent visits to the outpatient clinic for blood transfusions.

She also participated in a national clinical trial and received additional chemotherapy. "It was, for sure, the most difficult time of my life," Vittetoe said. "The hardest part was being away from my kids."

A Setback Forces a New Approach

Just two weeks after completing her treatments, Vittetoe's leukemia relapsed and a stem cell transplant was recommended.

"Even with specific subsets of leukemia, there's a lot of heterogeneity," Dr. Stock said. "We still have a lot to learn about why some cases like Amy's relapse and require a transplant whereas others are cured with standard chemotherapy."

None of Vittetoe's relatives was a match, but she found a perfect match through the national bone marrow registry.

After undergoing more chemotherapy to control her disease, Vittetoe was admitted for her stem cell transplant where she received intensive chemotherapy followed by the intravenous infusion of stem cells from an unrelated donor -- a 41-year-old man from Germany.

Three years later, Vittetoe is still in remission and has new priorities. She now works as a school nurse and spends as much time as possible with her children. She has also become active in the national marrow donor program.

"I can't donate stem cells or bone marrow to anyone anymore because of my condition, but I can help someone else find a match," she said.

Dr. Stock said cancer researchers are currently looking closer at these leukemias to see whether certain new, targeted therapies, in addition to chemotherapy, will improve their outcome.

"We take each case individually and tailor the therapy as best we can using the most sophisticated diagnostic and therapeutic strategies available," Dr. Stock said. "If specific chemotherapeutic options don't work, then immune therapies like transplants can work, and Amy is a perfect example of that."