acute myeloid leukemia (AML)
Decitabine, a drug usually seen as a milder form of chemotherapy, may be an improved treatment for patients with acute myeloid leukemia (AML) who also have mutations in the TP53 gene, according to this news release from Washington University in St. Louis. People with AML who have this gene mutation usually also have a more difficult-to-treat disease. In this study, researchers identified patients with TP53 mutations and then ran a drug trial to see if there was a correlation between the mutations and remission with decitabine. Remission with decitabine lasted about a year, while other remissions for this group following traditional chemotherapy usually average 4 to 6 months. The researchers found a similar response to the drug in patients with myelodysplastic syndrome, often a precurser to AML.
The release makes it abundantly clear that this is a preliminary trial result while providing a good description of benefits and an adequate overview of the trial. It would have been stronger with a brief discussion of costs for both decitabine and traditional treatment for AML. And since one of the benefits of decitabine is its lower toxicity, a brief discussion on reduced side effects would have been helpful to patients.
About 20,000 Americans will be diagnosed with AML this year and about 10,400 will die of the disease, according to the National Cancer Institute’s surveillance data. Patients with TP53 mutations are considered to have poor prognosis using conventional chemotherapy, but according to this small study, use of decitabine appeared to induce remission in all TP53 gene mutation patients.
Currently decitabine is used to treat elderly AML patients because it is an easier drug to tolerate than other chemotherapies. If a milder chemotherapeutic drug proves in a larger trial to increase the length of remission for a subgroup of AML patients, that’s welcome news. The news release, however, offers numerous cautions about the findings and it underscores that the drug treatment is not a cure.
There is no discussion of cost in this story. Decitabine is an FDA-approved drug and costs are readily available, but nothing is said of the costs or the relative costs of other treatments.
The release describes the benefit of the preliminary trial in sufficient depth and with appropriate cautions. It relates that each of the 21 patients with TP53 mutations had a remission, compared to 46 percent of the entire trial group of 116 that achieved some remission following treatment. According to the release, standard treatment typically induces remission in about 20 to 30 percent of patients with TP53 mutations. While the numbers look good from this trial, the researchers note that larger studies need to be done.
The release doesn’t directly address harms from either decitabine or standard chemotherapy for AML. What are the common harms from chemotherapy and which of these side effects are reduced in decitabine? Because the focus of the release is on the trial results of a drug described as less toxic, we would have liked some context on what reduced toxicity means.
The release points out that the trial was fairly small but that the objective of identifying TP53 mutation holders and assessing their response to decitabine appears to be successful. It describes how patients were selected and how their genetic mutations were identified. The release also reinforces the limitations of the study in various ways. Among them: the trial was quite small with just 21 of the 116 patients treated with decitabine having the TP53 mutation, and the researchers do not know why this subgroup of patients — who typically have a poorer prognosis — responded better than the whole to the drug.
There is no disease mongering. The release provides appropriate context on the disease.
The funding sources for this trial are clearly stated. However, the conflicts of interest disclosed in the published paper are not included in the news release. The disclosure states:
“Dr. Uy reports receiving fees for serving on an advisory board and clinical-trial support from Novartis; Dr. Jacoby, receiving consulting fees and fees for end-point adjudication from Quintiles and clinical-trial support from Sunesis Pharmaceuticals; and Dr. Graubert, receiving consulting fees from Agios.”
Each of the companies mentioned market cancer drugs so its appropriate to include the researchers’ financial ties with them in the release.
While the alternative drugs used in chemotherapy are not mentioned by name, they are referred to as the standard therapy. Because it is already known that TP53 patients have a worse prognosis than other patients with the conventional drugs, it appears that therapy with this milder drug shows better outcomes.
It is clear that decitabine is an existing FDA-approved drug. However, its off-label use in this trial does not mean that it is now available for other AML patients. The researchers note repeatedly that larger trials are required.
The release claims that patients with a specific gene mutation may live longer if treated with a less intensive chemotherapy drug. A researcher is quoted saying: “What’s really unique here is that all the patients in the study with TP53 mutations had a response to decitabine and achieved an initial remission.”
While the drug is not novel, its usefulness in treating this population of patients with a typically poor prognosis is a novel application.
There is no unjustifiable language used and it is made clear that decitabine is not a cure for these patients.
Systematic, Criteria-Driven
The tale of two “negative” studies. Or, how spin is applied to make research findings look more impressive. A must read. https://johnmandrola.substack.com/p/the-tale-of-two-negative-studies
The @nytimes chose to interview one of the editors of the @AnnalsofIM (publisher of this paper) who "gushed with extraordinary glee: It’s huge! There are very few things that reduce your mortality by 30%."
Turns out coffee is not one of those few things despite all that glee. https://twitter.com/garyschwitzer/status/1533202075928215558
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