This transcript has been edited for clarity.
Hello. I'm Dr Maurie Markman, from City of Hope. I wanted to briefly discuss an interesting paper that recently appeared in the Journal of Clinical Oncology, not so much because the results are earth-shattering — in fact, they're quite negative — but rather to highlight and applaud what the authors have done.
They've done what I consider to be a critical, simple, and pragmatic trial t o answer a question that has nothing to do with new drugs, the development of novel agents, patents, or Nobel prizes, but everything to do with that patient being seen in front of the doctor today, certainly oncologists. We need to do many more of the trials I'm going to talk about now. I think they're important to emphasize.
What was the question? Well, here's the trial: “Methylphenidate Versus Placebo for Treating Fatigue In Patients With Advanced Cancer: Randomized, Double-Blind, Multicenter, Placebo-Controlled Trial.” As I noted, it was published in JCO earlier this year.
Fatigue is one of the worst, if not possibly the worst, feeling for patients with advanced cancer, and certainly for many patients receiving anticancer therapy.We don't have good therapies for it. Think about that. We've been working on this for 50-70 years and we don't have an answer to the question of fatigue, certainly not in the advanced cancer setting, considering quality of life and impact on what people would like to do in the time they have.
It was a negative trial, as I said.What do we have for fatigue? There's very limited guidance available. The National Comprehensive Cancer Network guidelines say you can consider methylphenidate. Does it work? Does it do anything? Do we give it to patients and does it have side effects? Does it help at all? Where are the data? The data are not very good or we don't have any. This study answered the question.
There were162 patients randomized, 77 on placebo 82 on methylphenidate, and some had to be excluded. It was a 6-week trial. The trial actually ended after 10 weeks of therapy. They evaluated quality-of-life scores with the well-documented Functional Assessment of Chronic Illness Therapy ‒ Fatigue, FACIT-F.
It was very simple,pragmatic, and straightforward. There was no benefit — none — of this therapy. There were side effects but no benefit. There is no point in using the therapy, having any guidelines, or saying that patients might try it. It doesn't work. It was a very pragmatic and critically important question answered in this trial.
We need to do many, many more of these trials to answer questions.Of course, who's going to do them? Who's going to pay for them? No drug company wants to be involved in this so there’s no sponsor. If there’s no cure for cancer, can you get the National Cancer Institute to pay for it? I would hope so. How about insurance companies? We have a mechanism for doing this. Obviously, I'm asking important questions.
I want to emphasize the quality and the importance of these types of studies and of this study, and the need for more pragmatic trials answering clinically relevant questions. Oncologists are seeing patients every day. We need more of these.
Thank you for your attention.
COMMENTARY
Disrupting the Status Quo: We Need More Pragmatic Trials
Maurie Markman, MD
DISCLOSURES
| December 23, 2024This transcript has been edited for clarity.
Hello. I'm Dr Maurie Markman, from City of Hope. I wanted to briefly discuss an interesting paper that recently appeared in the Journal of Clinical Oncology, not so much because the results are earth-shattering — in fact, they're quite negative — but rather to highlight and applaud what the authors have done.
They've done what I consider to be a critical, simple, and pragmatic trial t o answer a question that has nothing to do with new drugs, the development of novel agents, patents, or Nobel prizes, but everything to do with that patient being seen in front of the doctor today, certainly oncologists. We need to do many more of the trials I'm going to talk about now. I think they're important to emphasize.
What was the question? Well, here's the trial: “Methylphenidate Versus Placebo for Treating Fatigue In Patients With Advanced Cancer: Randomized, Double-Blind, Multicenter, Placebo-Controlled Trial.” As I noted, it was published in JCO earlier this year.
Fatigue is one of the worst, if not possibly the worst, feeling for patients with advanced cancer, and certainly for many patients receiving anticancer therapy.We don't have good therapies for it. Think about that. We've been working on this for 50-70 years and we don't have an answer to the question of fatigue, certainly not in the advanced cancer setting, considering quality of life and impact on what people would like to do in the time they have.
It was a negative trial, as I said.What do we have for fatigue? There's very limited guidance available. The National Comprehensive Cancer Network guidelines say you can consider methylphenidate. Does it work? Does it do anything? Do we give it to patients and does it have side effects? Does it help at all? Where are the data? The data are not very good or we don't have any. This study answered the question.
There were162 patients randomized, 77 on placebo 82 on methylphenidate, and some had to be excluded. It was a 6-week trial. The trial actually ended after 10 weeks of therapy. They evaluated quality-of-life scores with the well-documented Functional Assessment of Chronic Illness Therapy ‒ Fatigue, FACIT-F.
It was very simple,pragmatic, and straightforward. There was no benefit — none — of this therapy. There were side effects but no benefit. There is no point in using the therapy, having any guidelines, or saying that patients might try it. It doesn't work. It was a very pragmatic and critically important question answered in this trial.
We need to do many, many more of these trials to answer questions.Of course, who's going to do them? Who's going to pay for them? No drug company wants to be involved in this so there’s no sponsor. If there’s no cure for cancer, can you get the National Cancer Institute to pay for it? I would hope so. How about insurance companies? We have a mechanism for doing this. Obviously, I'm asking important questions.
I want to emphasize the quality and the importance of these types of studies and of this study, and the need for more pragmatic trials answering clinically relevant questions. Oncologists are seeing patients every day. We need more of these.
Thank you for your attention.
Any views expressed above are the author's own and do not necessarily reflect the views of WebMD or Medscape.
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