Perceptions of symptoms and expectations of advanced therapy for Parkinson’s disease: preliminary report of a Patient-Reported Outcome tool for Advanced Parkinson’s disease (PRO-APD)

BACKGROUND: What do patients expect from a treatment? A patient-centred approach to treatment is becoming necessary given the choices for invasive treatments for Parkinson’s disease. Patient’s perceptions of severity and expectations from complex therapies have not been studied. We describe the rationale and concept of developing a Patient-Reported Outcome (PRO) tool to assess perceptions of symptom severity and expectations of therapy. We report preliminary findings from use of the tool, association with clinical factors, and illustrate the potential use in individual patients awaiting therapy. METHODS: Patient symptoms were grouped into four domains, with 8 motor, 7 non-motor, 7 psychological and 4 social questions. For each question, symptom severity was rated on a Likert scale scoring from 0 (no problem) to 7 (perceived as a severe problem). Similarly, the expectation for each symptom to change after therapy was rated on a Likert scale: score −3 (expected to be very much worse) to + 3 (expected to be very much improved). RESULTS: 22 consecutive patients, routinely planned to receive one of Deep Brain Stimulation/Intrajejunal Levodopa Infusion/Apomorphine Infusion therapies, were recruited: 13 male, mean (+/−sd) age: 65.6 (+/−9.5) years, mean (+/−sd) disease duration: 14.3 (+/−5.7) years. Subjective severity scores are reported as mean (+/−sd) / maximum possible score: (i) motor 23.5 (+/−7.5) / 56, (ii) non-motor 15.5 (+/−5.6) / 49, (iii) cognitive - psychological 12.4 (+/−5.8) / 49, (iv) social 9.3 (+/−4.1) / 28. Expectation of change (improvement) scores are reported as mean (+/−sd) / maximum possible score of: (i) motor 14.0 (+/−5.6) / 24, (ii) non-motor 8.5 (+/−4.1) / 21, (iii) cognitive - psychological 7.4 (+/−4.4)/ 21, and (iv) social 5.5 (+/−2.8) / 12. For each domain, Spearman correlation coefficient showed significant associations between severity and expectation within-domain. CONCLUSION: This tool (PRO-APD) provides a description of perceived problem severity and expectation of treatments encompassing a holistic patient-driven view of care. PD patients about to receive complex therapy have moderately high perception of symptom load in multiple domains, and expect substantial improvements in multiple domains. These preliminary findings may be useful in documenting multi-domain symptoms, as well as counseling patients to help them reach realistic expectations and reduce potential dissatisfaction following therapy.