Treatment Is Waiting
For patients with chronic blood cancers, the early intervention mantra of cancer does not always apply.
By Sue Rochman
Photo © iStock/ OGphoto / royaltystockphoto / EzumeImages
A few days after a routine physical in July 2009, Geoff Grubbs got a phone call from his nurse practitioner. His white blood cell count was higher than normal, and she wanted him to come back in for a second blood test. “She thought maybe it was a mistake,” recalls Grubbs. He did, too. At age 58, he was “happy as a clam,” he says, exercising regularly and spending a lot of time outdoors with his wife and children. He had the second blood test, expecting everything to be fine. Instead, his nurse practitioner called to say he should see an oncologist—soon.
Several anxiety-filled weeks later, Grubbs learned he had chronic lymphocytic leukemia (CLL), a type of blood cancer that begins in the bone marrow when certain white blood cells, called lymphocytes,
begin multiplying too rapidly. Not feeling at all ill, Grubbs was stunned by the diagnosis. But even “harder to get my mind around,” says the Washington, D.C., resident, was his oncologist’s recommendation: waiting to treat the cancer until it advanced. “You are afraid you are going to die,” he says, “and then the doctor tells you the best thing to do is nothing at this time.
Not treating a potentially deadly cancer seems counter-intuitive. But for some patients with slow-growing (indolent) blood cancers, an approach known as “watchful waiting”—or as some patients call it, “watchful worrying”—is the standard of care. (See “Who Can Watch and Wait?”
.) For some, the watchful waiting period may last a decade or more. For others, it may be two years or less before symptoms appear and drug therapies are needed to put the cancer into remission—when watching and waiting for symptoms begins again.
It’s not always easy for patients—or their loved ones—to trust a doctor who says watch and wait. But for certain blood cancers, the research shows starting treatment before symptoms develop doesn’t improve survival. Instead, it exposes patients to unnecessary treatment-related side effects.
Uma Borate, a medical oncologist and hematologist at Oregon Health & Science University in Portland, says when she treats patients diagnosed with a slow-growing blood cancer, one of her first goals is to ease their anxiety. “I try to help them understand the nature of the disease,” she says. “I explain that this is not something that happened overnight and that it is not likely to get out of control soon.”
Watchful waiting isn’t actually the same as doing nothing. Patients like Grubbs who are put on watch-and-wait protocols are observed closely, typically coming in for blood tests every three to six months. By testing patients regularly, says Loretta Nastoupil, a medical oncologist and hematologist at the University of Texas M. D. Anderson Cancer Center in Houston, “we learn about the tempo of their disease.” It is not until the pace at which the cells are dividing picks up, she says, that “the risk of disease outweighs the risk of therapy.”
Symptoms also relay the news that treatment is near. Grubbs had been having blood tests every six months for five years when, in late 2014, he found himself extremely exhausted, losing weight quickly and with a white blood cell count doubling rapidly. “It was quite clear I was no longer watch and wait,” he says.
Grubbs enrolled in a study at the Georgetown Lombardi Comprehensive Cancer Center in Washington, D.C., comparing a standard CLL treatment—the chemotherapy drug Treanda (bendamustine) and the targeted therapy Rituxan (rituximab)—with the standard treatment accompanied by Zydelig (idelalisib), a new type of targeted therapy that had been approved recently for previously treated CLL. He was randomly assigned to the group that received the standard treatment and Zydelig. Partway into the trial, Grubbs developed severe side effects and was taken off Zydelig, but he remained on the standard treatment. (Ultimately, trials testing Zydelig as a first-line treatment for CLL were halted because of the side effects it caused.) Even so, Grubbs says he doesn’t regret his decision to take part in the clinical trial. The treatment put his cancer in remission, and he says he intends to look for another trial if and when he needs treatment again. “Without new data, the science can’t advance,” he says.
After two years of watchful waiting, John D’Orazio, who was diagnosed with CLL in 2005 at age 35, had a similar experience. “When you start getting lymph nodes the size of tennis balls popping out of you, you know you need treatment,” says the Medford, Massachusetts, resident and father of four. D’Orazio entered a study at the Dana-Farber Cancer Institute in Boston investigating whether Revlimid (lenalidomide), a drug in trials for many different types of blood cancer, was effective in treating CLL. His cancer didn’t respond to the drug, and he switched to a standard-of-care regimen known as FCR—Fludara (fludarabine), Cytoxan (cyclophosphamide) and Rituxan—that put him into remission. (Revlimid is no longer being studied in CLL. It has been approved to treat multiple myeloma, myelodysplastic syndromes and mantle cell lymphoma.)