PRO45 PATIENT-REPORTED REAL-WORLD TREATMENT PATTERNS FOR CHARCOT-MARIE-TOOTH DISEASE IN THE UK AND US ASSESSED USING A DIGITAL 'BRING YOUR OWN DEVICE PLATFORM'

Abstract

The objective of this analysis was to examine patient-reported treatment patterns for Charcot-Marie-Tooth disease (CMT) in UK and US real-world practice. Adults with CMT were recruited to a two-year international observational study exploring the real-world impact of the disease. Data were collected via CMT&Me, a ‘bring your own device’ app specifically developed for this study, through which participants were asked questions about demographic, CMT management-related and quality-of-life variables. This interim analysis examined CMT treatment patterns, including use of surgical and physical therapies, medicines, and rehabilitative aids/orthoses in UK and US participants. The majority of study participants reported having received some form of physical therapy for their CMT, the most common being physio/physical therapy and occupational therapy, each received by over half of participants. Analgesics/painkillers and antidepressants were the most frequently used medicines. Antianxiety medications, cannabidiol oil and neuroleptics were also used by substantial proportions of participants. Use of pain medication for CMT was often long-term, with many participants reporting several decades of analgesic use. Most participants used some form of orthosis or walking aid, insoles and ankle/leg braces being the most common, with walking sticks and other walking aids also frequently reported. Additionally, around half of participants had received a surgical procedure for their CMT. Common procedures included osteotomy, arthrodesis, and plantar fascia-, achilles-, or tendon release. On average, US participants received a slightly greater number of different types of physical therapy, medication, and surgical procedure than UK participants did, who used a greater number of aids/orthoses. The management of CMT in the UK and US is multifaceted, involving the use of physical and surgical therapies, as well as multiple medications, orthoses and aids. This ongoing registry will provide further real-world insights into the treatment CMT to enable gaps in care to be identified and addressed.