A panel of experts discuss the most important goals of therapy for patients with chronic lymphocytic leukemia, mantle cell lymphoma, and small lymphocytic lymphoma and the largest unmet needs in leukemia/lymphoma.
Ryan Haumschild, PharmD, MS, MBA: I’m curious about what the goals of therapy are as we treat patients with medication therapies. Dr Coombs, I’ll turn to you. When you’re treating these patients, what are the most important goals you’re thinking about and talking through with your patients?
Callie Coombs, MD: The first thing I’ll say is that it depends on the patient. I don’t believe in a 1-size-fits-all treatment plan because goals are different depending on the individual. Let me go for 2 extremes. As a doctor who works at a referral center, I see some very young patients who need decades of life to see their kids grow up, thrive, and support their families. Those patients may want the most aggressive therapy possible, but we have to weigh what the actual benefit is. CLL [chronic lymphocytic leukemia] is not generally considered curable. There’s an exception to that rule. Young, very fit patients with high-risk markers who have been failed by multiple therapies can consider allogeneic transplant, and that can be curative. That’s 1 extreme, and that may be 1 patient’s goal.
On the other extreme, I saw someone last week who is 97 years old. She did have symptoms from her disease and did warrant treatment. However, this woman wants to enjoy whatever time she has left, and none of us knows how much time that is. The goal is to relieve whatever symptoms she has without causing any suffering. Globally, in cancer, something I learned when I was a trainee is that our goals should be to live longer, live better, or feel better. I totally understand how tough it can be to hear “watch and wait” and feel like you’re doing nothing. One of the ways I explain to patients with CLL the reason why we’re not treating aggressively early on is that it’s never been shown to make individuals live longer, and patients can feel worse only when they start with no symptoms at all. It’s always important to No. 1, find out the patient’s goals because they’re all different; and No. 2, minimize suffering while prolonging their quality of life, especially if they have symptoms from the disease.
Ryan Haumschild, PharmD, MS, MBA: Excellent. I want to turn to Dr Beveridge. What do you think are the largest unmet needs in leukemia and lymphoma? We’ve heard a lot about some of the treatments, extending life out. But what are some of the biggest unmet needs?
Roy Beveridge, MD: What we’ve heard today is that we have an abundance of new treatments compared with when you and I did our fellowship, when we used chlorambucil and a few other things like that. We have many more treatments, but none of this is curative. This is a big problem. We’re moving toward a cure for many other diseases. But if I’m going to put my hat on and represent the payer community, including Medicare, there’s a fair amount of confusion in terms of what the pathways say and what standard therapy is for a typical patient. We all know that a 37-year-old patient with this is going to have different needs, expectations, and desires. But that’s rare, relatively speaking. There’s a standard patient, and what insurance companies are looking for is that median +/-1 standard deviation.
How do you treat that group? The biggest problems for payers are understanding what the pathway should be and what comparisons of drug X and drug Y should be because there are a lot of drug treatments for which there’s no comparison. That’s what patients worry about. Then they go to key opinion leaders. There’s a diversity of opinion there, which makes it very difficult if you’re trying to actuarially understand what your costs are going to be in the following year. That’s what payers spend a lot of time thinking about.
Ryan Haumschild, PharmD, MS, MBA: I appreciate the payers’ perspective because they’re the ones helping to design the benefit and thinking about the future needs in terms of providing better value to our patients and to the employer groups, but ultimately steward health care dollars. I’d like to turn it over to Dr Koffman. What are some of the largest unmet needs in the leukemia and lymphoma space?
Brian Koffman, MD: I totally agree. The biggest unmet need for patients is curative therapies. Every therapy in CLL, with the exception of the few individuals with allotransplants and a few young, healthy patients with the best possible markers—FCR [fludarabine, cyclophosphamide, rituximab]. Some of those patients are 17, 18 years out and look like they’re cured. But that’s a tiny minority of patients, and it’s a very small group that that therapy should be considered on. Every therapy is palliative. If you’re young enough—and it doesn’t have to be that young; you can be in your 50s or 60s—you’re going to run out of therapies. Then what do you do? We’re looking at what therapies are potentially curative. But there are some other significant unmet needs, and something the pandemic brought to the front is how we reconstitute our immune system. Dr Coombs will tell you that a lot of patients that she has aren’t dying of CLL. They’re dying of the second malignancy. They’re dying of an overwhelming infection. These are big problems that patients with CLL have. These are often end-of-life events for patients with CLL because it’s a cancer of the immune system. How we reconstitute the immune system is another thing that we have to look at.
What do we do for patients who failed the approved therapies? What do we have outside clinical trials? What can we do? About 5% to 10% of the time, patients with CLL transform into Richter syndrome, and the prognosis is dismal for that. We did hear some encouraging data at ASH [American Society of Hematology Annual Meeting], but they were small numbers. It’s exciting, but these are early data. A lot of studies have shown that survival with Richter syndrome is measured in months, not years. That’s a tremendous unmet need for that small minority, but it’s 5% to 10%.
Transcript edited for clarity.