title: Attitudes and expectations of patients with neuromuscular diseases about their participation in a clinical trial.
abstract: OBJECTIVE
  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).
  
  
  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.
  
  
  CONCLUSIONS
  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.
authors: Institut de myologie, groupe hospitalier Pitié-Salpêtrière, Assistance publique-Hôpitaux de Paris, 47, boulevard de l'Hôpital, 75013 Paris, France; Laboratoire de psychologie clinique et psychopathologie (EA 4056), université Paris Descartes, Sorbonne Paris Cité, institut de Psychologie, 71, avenue Édouard-Vaillant, 92100 Boulogne-Billancourt, France. Electronic address: marcela.gargiulo@parisdescartes.fr.
authors: M
authors: M
authors: Gargiulo
authors: A
authors: A
authors: Herson
authors: C C
authors: CC
authors: Michon
authors: J Y
authors: JY
authors: Hogrel
authors: V
authors: V
authors: Doppler
authors: K
authors: K
authors: Laloui
authors: S
authors: S
authors: Herson
authors: C
authors: C
authors: Payan
authors: B
authors: B
authors: Eymard
authors: P
authors: P
authors: Laforêt
mesh_terms: D000328:Adult; D000368:Aged; D001290:Attitude; D002986:Clinical Trials as Topic / Q000523:psychology* / Q000706:statistics & numerical data; D056947:Enzyme Replacement Therapy / Q000523:psychology; D005260:Female; D005500:Follow-Up Studies; D006009:Glycogen Storage Disease Type II / Q000188:drug therapy / Q000523:psychology; D006801:Humans; D008297:Male; D008875:Middle Aged; D009468:Neuromuscular Diseases / Q000453:epidemiology / Q000523:psychology* / Q000628:therapy; D010358:Patient Participation / Q000523:psychology* / Q000706:statistics & numerical data; D010465:Perception; D011795:Surveys and Questionnaires
keywords: Essais cliniques; Maladies neuromusculaires; Medical trial; Neuromuscular disease; Psychological; Psychologie