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24/7 support may improve outcomes in acute promyelocytic leukemia
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Around-the-clock availability of experts in acute promyelocytic leukemia — a hotline, of sorts, for clinicians who treat this rare disease — may help patients survive the critical first month of treatment, according to study results.
The mortality rate for acute promyelocytic leukemia (APL) in the first month can be as high as 30%, largely due to a unique set of complications that may be difficult to recognize for clinicians who rarely treat the disease, Vamsi Kota, MD, assistant professor in the department of hematology and medical oncology at Winship Cancer Institute of Emory University, and colleagues wrote.
Standard treatment includes all-trans retinoic acid and arsenic trioxide combined with anthracyclines. However, the complications that occur are not always well understood because community hospitals may treat just one patient with APL every few years, Kota said.
Investigators devised a strategy that they believe could significantly reduce the early mortality rate and increase the likelihood of long-term management or cure for these patients.
The strategy incorporates use of standardized guidelines and a simplified checklist for clinicians, as well as consultative support and sharing of expertise. Use of the strategy contributed to a significant reduction in early fatalities at the participating center.
HemOnc Today spoke with Kota about the study, the support services such an intervention would provide, and the potential impact this strategy could have on early outcomes of patients with APL.
Question: What prompted this study?
Answer: This idea started in failure. Anand P. Jillella, MD, the study chair for this project, and I noticed — on review of charts between 2005 and 2009 — that a high number of deaths occurred among patients with APL during the first month. We had to overcome this burden, as APL is supposed to be a curable disease, probably the most curable of all cancers. We created a two-page algorithm, a sort of checklist, of what needed to happen during early treatment. In addition, we felt that patients would benefit from dedicated oversight from a core group of physicians with interest in APL. We took this concept to multiple funding agencies and, because it is not a treatment trial, we had difficulty obtaining funding. However, eventually the Leukemia & Lymphoma Society funded us for 5 years to improve the outcomes in our region. We enrolled 120 patients, mostly from Georgia and South Carolina. We have shown that, with this concept, we can improve the outcomes and decrease the death rate in the first month from as high as 30% to 8.5%. The study has expanded and is to NCI Community Oncology Research Program sites across the country.
Q: How did you conduct this study?
A: This trial is unique in that we do not have any new treatments being introduced. We are simply ensuring that the available and excellent treatment options are helping patients, even outside of a clinical trial. A core group of seven physicians will be available to take calls from physicians for any patients newly diagnosed with APL at the participating NCORP sites. The treating physician can call one of these experts and discuss the best way to treat the patient. We want to have an ongoing conversation in the first few days, when the risks to the patient are high due to treatment complications.
Q: What support services will be provided?
A: The participating expert has to call as soon as the diagnosis is made so we can participate in the discussion about the patient from the beginning. The goal is to avoid complications, and we feel that two physicians discussing the patient’s condition at the start offers the best chance to improve outcomes. The patient must sign a consent form that describes the nature of the trial and the conversations that will ensue. This also allows us to collect the data from the patient’s therapy.
Q: How labor-intensive is this intervention?
A: APL is a rare disease and, although the process of following a patient over 30 days without actually seeing the patient might not be how we are used to seeing a trial run, it is a unique effort on the part of the experts.
Q: How might this support improve patient outcomes?
A: APL has very high cure rate if we can get over the complications of the disease and its therapy in the first month. The relapse risk is small. Mortality in the first month has been as high as 30% in some studies. We have shown in our region that this can be brought down to less than 10% in a real-world, unselected patient population. We hope we can replicate the results in this large nationwide study.
Q: Could this study serve as a basis for similar programs in other malignancies?
A: We certainly think this will be a starting point for other diseases. In one way, our approach is telemedicine, which has already been used in multiple diseases but has been slow to start in cancer. We hope to extend this concept to other diseases. – by Rob Volansky
Reference:
Jillella AP and Kota VK. Blood Rev. 2017;doi:10.1016/j.blre.2017.09.001.
For more information:
Vamsi Kota, MD, can be reached at Winship Cancer Institute, 1365-C Clifton Road NE, Atlanta, GA 30322; email: vamsi.kota@emory.edu.
Disclosure: Kota reports research funding from Leukemia & Lymphoma Society and NCI.