Through their participation in a national study, patients being treated for a rare form of leukemia by doctors at North Shore University Hospital (NSUH) and Long Island Jewish (LIJ) Medical Center have helped confirm that an arsenic compound, coupled with standard chemotherapy treatments, significantly increases long-term survival.
Patients all had newly-diagnosed acute promyelocytic leukemia, or APL, and participated in a multi-year national phase III clinical study. Twenty eight patients from NSUH and LIJ enrolled in the clinical trials, the largest number of participants from any facility in the New York metropolitan area - and the second largest nationwide. The study involved 582 patients nationally over six years -- from June 1999 through March 2005. It was sponsored by the National Institutes of Health's National Cancer Institute. The study findings were recently released prior to formal publication so that all APL patients could benefit sooner from the therapy, according to the NCI.
Jonathan Kolitz, MD, director of the leukemia service at NSUH's Don Monti Division of Medical Oncology at the Monter Cancer Center in Lake Success, said the trial shows that APL is essentially curable in many patients, a term he doesn't use lightly. "I believe arsenic trioxide will become part of the new standard protocol for treatment of APL in many patients," he said.
Vicki Murphy, a 59-year-old resident of Oyster Bay, was diagnosed with APL in 2000. She was the first patient to enroll in the study at NSUH. After going through several rounds of chemotherapy to put her leukemia into remission, she said she went back to the hospital daily for nearly three months to receive the additional arsenic trioxide therapy. "I was scared but I also knew this trial was my chance for a cure," she explained. "It's always scary when you're facing the unknown."
In the randomized trials, patients with APL were given either several rounds of standard chemotherapy drugs combined with the retinoid ATRA, a vitamin A derivative plus a post-remission regimen of additional chemotherapy and ATRA followed by a year of maintenance chemotherapy using oral agents, including ATRA, or the identical regimen plus two 25-day courses of arsenic trioxide prior to the post-remission therapy. Patients receiving the arsenic trioxide had significantly better likelihood of remaining disease-free and surviving than those receiving standard chemotherapy alone. Patients in remission also underwent an additional year of ATRA, with or without oral chemotherapy, to ensure they remained cancer-free.
"These patients endured additional months of therapy, with 25 days of two-hour infusions of arsenic given over five weeks," explained Dr. Kolitz. "Then, after a short break, they had to do it all over again." Study patients also underwent extensive follow-up, including weekly blood tests and regular bone marrow tests. They will be monitored for life.
Ms. Murphy said study participants got through the ordeal thanks to the support of each other and staff at NSUH and LIJ. "I wouldn't be here, I wouldn't have been able to endure the treatments if it wasn't for their support and encouragement," said Ms. Murphy, a grandmother, journalist, musician and activist. "The entire staff was incredibly supportive and provided lots of encouragement, especially during the times I wasn't sure I could get through this," she said.
In acute leukemia, the bone marrow doesn't mature properly, and eventually crowds out normal cells. APL, a subtype of acute myeloid leukemia (AML), affects about 1,500 patients each year, about 10 percent of AML cases. It primarily strikes middle-aged adults. AML is an acquired, not genetic, condition, which helps to explain why previously healthy people can develop it. While scientists are still unsure of what causes AML, they believe long-term exposure to certain chemicals, such as benzene, may be a risk factor.
Ms. Murphy has been in remission for nearly seven years and says she owes more than she can ever fully express to Dr. Kolitz and his healthcare team. "I took it one day at a time, and although there were good days and bad days, the support I got meant I never completely lost hope," she said. She is now looking forward to many long and productive years with her family, friends and colleagues.