Patient-Reported Treatment Patterns of Charcot-Marie-Tooth Disease Type 1A in Europe and the US Measured Using a Digital APP
Author(s)
Thomas FP1, Sivera R2, Gray A3, Moore A4, Rego M5, Genovese F6, Boutalbi Y7, Monteiro K7, Paoli X7, Hall K8, Llewellyn S8, Ouyang C8, Larkin M8
1Hackensack Meridian School of Medicine and Hackensack University Medical Center, Hackensack, NJ, USA, 2Hospital Francesc de Borja, Gandia, Spain, 3Charcot-Marie-Tooth Association, Glenolden, PA, USA, 4Hereditary Neuropathy Foundation, New York, NY, USA, 5Federación Española de Enfermedades Neuromusculares, Barcelona, Spain, 6ACMT-Rete per la malattia di Charcot-Marie-Tooth OdV, Bologna, Italy, 7Pharnext, Issy-les-Moulineaux, France, 8Vitaccess Ltd, Oxford, UK
OBJECTIVES Patients with Charcot-Marie-Tooth disease type 1A (CMT1A) often require a variety of treatment modalities, such as rehabilitation therapies, medications, orthotics and surgery. The purpose of this interim analysis was to examine patient-reported treatment patterns for CMT1A in Europe and the US. METHODS Adults from Germany, Italy, Spain, UK and USA with CMT1A were recruited to a two-year observational study exploring the real-world impact of the disease. Data were collected via CMT&Me, a digital app developed for this study, through which participants answered questions on demographics and treatment patterns. RESULTS Participants reporting severe and moderate symptom severity, relative to mild, were significantly more likely to report visiting rehabilitation therapists, using analgesics and ankle/leg braces. In contrast, only severe symptom-severity participants indicated higher antidepressants use, relative to mild (n=270, p<0.05). Compared to the rest of Europe, UK and US participants significantly more often reported visiting occupational therapists and using ankle/leg braces. The proportion of UK participants reporting insole use was significantly higher vs the US, whereas antidepressant use was significantly higher in the US vs Europe (n=270, p<0.05). Male participants were significantly less likely to report the use of physical therapies or medications. Similarly, increasing age was associated with reporting using no physical therapies, and higher body mass index (BMI) with the use of no medication (n=270, p<0.05). No associations were found in the types of surgical procedures received by participants. CONCLUSIONS Patient-reported data confirmed CMT1A is associated with use of physical therapies, medications and orthotics. Greater reported use was seen in participants with higher symptom severity, whereas associations in the type of treatment used varied between regions. Differences in treatment patterns among patients were also seen across participant age, sex and BMI.
Conference/Value in Health Info
2020-11, ISPOR Europe 2020, Milan, Italy
Value in Health, Volume 23, Issue S2 (December 2020)
Code
PRO134
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare and Orphan Diseases
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