Article

This study aimed to gain a better understanding of the psychological impact
of participating in a clinical trial for patients with Pompe disease (Acid
Maltase Deficiency). Attitudes and expectations of adult patients with
neuromuscular diseases regarding medical trials are as yet unreported. In order
to learn about the psychological consequences of participating in a clinical
trial, we conducted a prospective assessment of patients with late-onset Pompe
Disease, a rare genetic condition, for which no treatment had been available
before. This psychological study was carried out as an ancillary study to the
randomized double-blind placebo-controlled trial described elsewhere (van der
Ploeg et al., 2010). SUBJECTS AND METHODS: We assessed patients (n=8) at
inclusion, and at 12 and 18 months for six psychological dimensions: depression
(Beck Depression Inventory, BDI), hopelessness (Beck Hopelessness Scale, BHS),
anxiety (STAI A-B), quality of life (Whoqol-26), social adjustment
(S.A.S-self-report) and locus of control (IPC Levenson). We produced a
self-administered questionnaire in order to assess the attitudes, motivations and
expectations of patients during the trial. RESULTS: At 12 months, mean social
adjustment (SAS-SR, P=0.02) had improved, and at 18 months mean depression score
had improved as well (BDI, P=0.03). The quality of life of patients (Whoqol-26)
remained unchanged. Throughout the study, patients were more likely to have an
internal locus of control than an external one (IPC Levenson). The
self-administered questionnaire showed that patients' expectations were
disproportionate compared to the medical information they had received starting
the trial. For all patients, the first motivation for being enrolled in a
clinical trial was "to help research", for half of them the motivation was to
"improve their health". Whether patients believed to be part of one group or
another (placebo or treatment) depended on their subjective perception of
improvement during the trial. CONCLUSION: Given the small sample size, the
conclusions of this study are preliminary. However, findings do suggest that
there is a positive psychological impact of participating in a treatment trial.
Moreover, the patients' reactions upon unblinding have led us to recommend that
patients be asked whether they would like their group assignation disclosed to
them or not.
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Langue : ANGLAIS