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www.jco.org on January 22, 2013
■■■
Inclusion of Patients With Advanced
Cancer in Phase I Trials: Is This a Tool
for Improving Optimism and
Emotional Well-Being?
TO THE EDITOR: Fu et al
1
recently published an article concern-
ing the advance care planning for patients with cancer who are
referred to early clinical trials. Via an investigator-designed survey,
the authors assessed the attitudes of patients who were included in
phase I clinical trials at the point of end-of-life planning. This study
revealed that despite the severity of illness and lack of proven
benefit from aggressive care in a phase I trial, a minority of these
patients (49%) wanted to address advance care planning. Many of
them avoided discussing this topic, with strong feelings in opposi-
tion to considering advance care planning. Confronting the subject
of planning for death was extremely traumatic for them because
they remained hopeful that they could beat their cancer. Compared
with similar patients with cancer, those who choose to be enrolled
onto a phase I trial tend to be more optimistic than might be
expected.
2,3
However, this result contrasts with the results of the
recent study of Fang et al,
4
who reported that patients with cancer
have an increased risk of suicide, particularly after a cancer diag-
nosis, especially in those with a poor prognosis. On the other hand,
two recent studies
5,6
hypothesized that depression was associated
with worse survival in patients with metastatic cancer and that
improvement in depression mediated a survival benefit.
Therefore, how can we explain that, despite a short life expec-
tancy, patients who choose to pursue treatment in phase I clinical trials
remain optimistic? Critically, the authors hypothesized that this high
degree of optimism could be generated by denial mechanisms in
response to diagnosis and misconceptions about the therapeutic ob-
jectives of phase I trials. In this way, the study reported by Fu et al
1
is of
major interest and suggests the need for a fine balance between phase
I trial patients’ optimism and the qualified chance of success of the
protocol therapy.
We previously reported the evaluation of sexuality, health-
related quality of life (HRQoL), and depression in 63 patients with
advanced cancer who were treated in phase I trials in the Gustave
Roussy Institute Drug Development Unit.
7
To our knowledge, we
reported the first prospective study evaluating sexuality, HRQoL,
and depression in patients with advanced cancer who were treated
in early clinical trials of predominantly targeted anticancer drugs.
All of the patients enrolled onto phase I trials had advanced or
metastatic cancer that was refractory to classic lines of chemother-
apy. Interestingly, the prevalence of major depression was only 2%,
respectively, at baseline and after 1 month of treatment. This result
contrasts with the reported prevalence of major depression in the
general healthy population of 5%
7
and in the cancer population of
10% to 25%.
8
In principle, we agree with the authors,
1
but we suggest that
the disparity between patients’ optimism and the poor expectancy
for success of the phase I trials could be explained in more detail.
Arguably, patients who are eligible for entry in a phase I protocol
have several specific characteristics, including good performance
status, despite the failure of conventional chemotherapy and their
progressing disease. Given that the key aim of phase I trials is to
investigate toxicity and determine a recommended phase II dose,
patients enrolled onto phase I trials are usually those for whom
there is no standard therapy. Therefore, potential candidates for
phase I trials are enrolled at a specific point along their disease
trajectory when they may feel hopeless and experience fear of
death. Moreover, participants have high expectations regarding
the success of experimental therapy, with the hope for disease
regression, and may discount potential toxicity. Available data on
motivations and expectations of patients who are enrolled onto
phase I clinical trials show that hope of a personal clinical benefit is
the main reason patients agree to be included.
3
However, a central
issue to be addressed is that most of the patients who are treated in
a phase I trial for advanced cancer feel less depressed, with HRQoL
scores that are comparable with those of the general healthy pop-
ulation. Additional studies are needed to investigate the psycholog-
ical impact of inclusion in early-phase clinical trials and the
changes in depression symptoms from baseline to trial exit.
Mathieu Rouanne
Institut Gustave Roussy, Universite ´ Paris XI, Villejuif; Ho ˆ pital Foch, Suresnes;
Universite ´ Versailles, Saint-Quentin-en-Yvelines, France
Elodie Jacquelet and Antoine Hollebecque
Institut Gustave Roussy, Universite ´ Paris XI, Villejuif, France
Thierry Lebret
Ho ˆ pital Foch, Suresnes; Universite ´ Versailles, Saint-Quentin-en-Yvelines,
France
Jean-Charles Soria and Christophe Massard
Institut Gustave Roussy, Universite ´ Paris XI, Villejuif, France
AUTHORS’ DISCLOSURES OF POTENTIAL CONFLICTS OF INTEREST
The author(s) indicated no potential conflicts of interest.
REFERENCES
1. Fu S, Barber FD, Naing A, et al: Advance care planning in patients with
cancer referred to a phase I clinical trials program: The MD Anderson Cancer
Center experience. J Clin Oncol 30:2891-2896, 2012
2. Daugherty C, Ratain MJ, Grochowski E, et al: Perceptions of cancer
patients and their physicians involved in phase I trials. J Clin Oncol 13:1062-
1072, 1995
3. Agrawal M, Grady C, Fairclough DL, et al: Patients’ decision-making process
regarding participation in phase I oncology research. J Clin Oncol 24:4479-4484,
2006
Correspondence
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