This release about a small trial of immunotherapy applied to a dozen leukemia patients who are at unusually high risk of relapse leaps to premature interpretations and inflates the meaning of the results far beyond the limited guidance for future research that this sort of study can offer.
It inappropriately elevates the importance of relapse rates, implying that they predict vital outcomes, such as survival or quality life improvements, which this trial did not measure. Even as it puffs up the potential benefits, the release neglects to note either the short-term harms reported by researchers in their scientific conference abstract or the potential life-threatening harms that immunotherapy may expose patients to. The release doesn’t provide context for how these trial results relate to existing or other experimental treatments, and it makes makes only an oblique reference to the personal financial stakes of a lead researcher. It does not make any reference to cost, even though recently-approved immunotherapy treatments suggest the price tag could be in the hundreds of thousands of dollars.
On the positive side, the release does a good job of explaining why adjuvant treatment to enhance the immune system after bone marrow transplant may be necessary in certain leukemia patients.
Relapse rates may reveal important clues to how long patients may live or how they will feel, but then again they may not. Any surmised connection between this sort of intermediate endpoint and the things that really mean something to patients must be tested. Until survival or quality of life trials are performed, news releases should not imply that an intermediate endpoint result, such as relapse rate, predicts better or longer life for patients.
Although this research in still in early stages, the release could have noted that immunotherapy can be very expensive. Some courses of treatment cost $100,000, while others need to be given continually at a cost that can exceed $10,000/month.
The release reports that all 12 participants in the trial (those who received the T-cell therapy after a transplant) remained in remission during a median follow-up period of more than two years, while more than a quarter of transplant-only patients relapsed within 10 months. Although that seems like clear quantification of benefits, there are many examples of cancer treatments that have produced higher remission rates, but ultimately failed to help patients live longer or better. To be clear, we have no problem with the release reporting on the surrogate outcome, which may be the only measure available to estimate the treatment’s effects in these patients. However, we believe that the release should have cautioned that the remission rates do not necessarily predict survival or even quality of life…and that these factors, the ones that really matter to patients, will be measured only in future experiments.
Not only does the release omit side effects or other potential harms from this type of treatment, it emphasizes the harms of conventional treatment, thus presenting an unbalanced comparison. The conference abstract summarizing the research noted that adverse events included short-term flu-like symptoms that are common to certain types of immunotherapy, as well as temporary reductions in white blood cell counts. In addition to failing to mention these reported harms, the release did not alert readers to potential life-threatening risks of immunotherapy which sometimes do not become apparent until the therapies have been given to many people over many years.
Of note: a similar experimental immunotherapy trial by the same company (with acute lymphoblastic leukemia rather than AML patients) was halted after some patients died, according to this conference recap. The growing awareness of potential hazards of immunotherapy was recently highlighted in this post on HealthNewsReview.org.
The release should have mentioned that the absence of life-threatening complications in this small trial does not prove the treatment is safe.
The release reports that the trial involved only 12 patients and that the comparison group was made up of “similar” patients. But it does not explain that this comparison is not as clear-cut as a trial that randomizes patients to different treatments. Also, there is no mention of the amount or type of study that remains to be done.
The release notes that the trial involved only acute myeloid leukemia (AML) patients considered at high risk of relapse. It gives readers a sense that these patients are not typical of patients with AML. The release would have been better if it had provided some sense of the proportion of AML patients who fit the description of those in this trial.
The release reports that funding came from the National Institutes of Health and from Juno Therapeutics. It says that one of the researchers is “a scientific co-founder” of Juno Therapeutics. So we will give the release a passing grade on this criterion. However, readers have to have sharp eyes to intuit that this disclosure means that the researcher owns part of the company and so stands to profit from any treatments that result from this work.
Without any information on the extent of the type of leukemia relapse risk that is the target of this research or any evidence that a reduction in relapse rates ultimately gives patients longer or better quality life, there is no way to make an informed comparison with existing treatments. Nevertheless, the release makes unsupported assertions that this treatment appears to be superior to conventional treatment. The release also leaves out any mention of similar experimental therapies.
The release reports that this is the first test of this particular treatment, so it is clear that it is just at the beginning of the research process.
The release does not discuss similar experiments, including one that was outlined in the conference abstract, so readers cannot get a sense of how this experiment fits into a broad view of efforts to improve treatment of AML.
The release doesn’t rely on sensationalism to describe the findings. However, researchers are quoted as saying the experimental treatment “might actually be helping patients who have a high risk of relapsing to not relapse down the line.” Then again, the reverse may be true and it might not be.
While leukemia relapse is unwelcome, it cannot be known from this trial whether lower relapse rates mean longer or better lives for patients. Highlighting this sort of intermediate (or surrogate) endpoint distracts attention from the things that really matter to patients: the length and quality of their lives.