Tuesday, January 13, 2015

Rituxan Maintenance -- Still Not Settled

The medical website Healio published an interesting piece a couple of days ago called "Robust Data Needed to Settle Maintenance Therapy Debate in Hematologic Malignancies." It's a little broader than a discussion about just Rituxan Maintenance in Follicular Lymphoma, looking at other maintenance treatments, and other blood-related issues, but what it has to say applies easily to R-Maintenance for FL.

(For those who don't know, maintenance therapy is the process of giving an additional treatment after a treatment has achieved a response, in an attempt to "maintain" that response. One common maintenance therapy for Follicular Lymphoma is Rituxan Maintenance -- after receiving R-CHOP and getting a response, the patient then gets Rituxan every six months for two years, with the idea that the Rituxan will clean up any left over FL cells that had been hiding.)

In a sense, it reviews (and doesn't necessarily add any new data to) the debate over whether R-Maintenance is worth it. Instead, the author, Dr. John Sweetenham, calls for some new research on the effectiveness of maintenance, and on how that effectiveness is measured.

It's a debate that's been going on for a while -- is maintenance therapy worth it? The studies that have attempted to answer that question (and Dr. Sweetenham refers to several of them) have not been able to give us a definite answer.

One problem is the way it has been measured. For the most part, researchers looking into maintenance have been measuring Progression Free Survival (PFS), or the amount of time it takes before the patient needs another treatment. Most studies show that R-Maintenance shows a PFS benefit -- that is, maintenance helps the patient go longer before another treatment is needed than would have been the case if there was no maintenance. However, most studies also show that R-Maintenance has no real effect on Overall Survival (OS). Dr. Sweetenham points out how difficult it is to decide between the two measurements. On the one hand, PFS isn't really the best measure, because most of us will probably need multiple treatments over time anyway. On the other hand, OS isn't really much better, and for the same reason -- if we need several different treatments over time, it's hard to say which one contributed most to OS. 

Dr. Sweetenham also points out that, as we shift our view of treating FL from looking for a cure to considering it a chronic disease, it is possible that we will be taking daily treatments -- maybe in the form of oral pills. If that's the case, the cost could be overwhelming to patients and to health care systems. (Imagine 40,000 Follicular Lymphoma patients each taking a pill every day that costs $100 each. The cost would likely go down, but maybe not as much as we'd like.)

Dr. Sweetenham's suggestion is that from here, studies on the effectiveness of maintenance also consider factors such as cost and cost effectiveness. In other words, while studies typically measure PFS and/or OS to answer the "is it worth it?" question, they should also consider whether a treatment justifies its potentially high price. If we need to justify it later on, having that kind of cost-effectiveness data will be very helpful.

All in all, this was a pretty thought-provoking article. (And for the record, Dr. Sweetenham would seem to be in the pro-maintenance camp, since he does prescribe it for his patients.)

I think what I like most about it is that it makes the assumption that Follicular Lymphoma is being seen as a chronic disease, that it is hard to measure its effectiveness because it is essentially chronic, and that the kinds of treatments that we will be focusing on in the future will treat it as such. It brings up some hard questions, but they are built on a foundation of hope.


William May said...

Since fNHL is now being considered a treatable illness, any increase in PFS is meaningful because the number of available treatments is finite.

Anonymous said...

I had R-maintenance for 2 years...I have MALT Lymphoma that was progressing and caused b- symptoms. I know my oncologist was trying to give me the best chance of a longer remission, but I am now suffering from side effects that even the docs at Mayo have not seen. Specifically, my b-cells have not recovered at almost 4 years out from my last dose...I have also been diagnosed with cytokine release syndrome which is causing b-type symptoms...another side effect of Rituxan that usually occurs during treatment. Rituxan is a relatively new drug in the grand scheme of things...how many other people are going to develope long term side effects. In hindsight, maintenance may not have been the best route to go in my case. The initial 8 weekly doses I recieved may have been sufficient.

Lymphomaniac said...

I'm sorry to hear about your troubles. I know there are lots of experts who are very positive about R-M, but lots of experts who have questions about it that, unfortunately, you seem to be answering. I hope things can turn around for you soon. Thanks for sharing your experience.