The CMT&Me real-world digital lifestyle study self-reports data from patients with all types of CMT and is collected on a regular basis in both US and Europe. The objective of the study is to better understand the impact of the disease on patients’ daily lives and help them manage their condition and treatment, as well as raise awareness and assess the value of potential new treatments. This study is managed by the company Vitaccess in collaboration with HNF and other patient advocacy groups and key opinion leaders in the field, with the support of Pharnext.
The recent findings presented at the annual Peripheral Nerve Society Meeting represent the impact CMT has on quality of life and the importance of addressing the needs of patients.
Depression in Patients with Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Participants were asked questions on demographic and employment variables. This interim analysis examined 937 participants from France, Germany, Italy, Spain, UK, and the US. Of the participants who responded to this question, 38% of the participants reported having been diagnosed with depression in addition to CMT1A; higher than in the general population. Of these, 54% and 35% reported moderate or severe CMT1A symptom severity, respectively. And 43% of participants diagnosed with depression reported that they used, or had previously used antidepressants. Reported diagnosis of depression varied considerably by country. The highest rates were among participants in the US and UK (48% and 40% respectively), while lowest rates were among participants in France and Italy (29% and 18% respectively). Of participants who responded to the EuroQol 5 Dimensions 5 Levels (EQ-5D-5L) instrument, 62% reported concerns with anxiety/depression.
Over a third of participants reported a diagnosis of depression in addition to CMT1A. This is not surprising for a disease with this symptom burden; however, depression itself as a comorbid condition represents a significant disease burden and can affect treatment and outcomes for CMT1A.
***This warrants further analysis and exploration.
Work Impacts in Patients with Charcot-Marie-Tooth Disease Type 1A (CMT1A)
Participants were asked questions on demographic and employment variables. This interim analysis examined 937 participants from France, Germany, Italy, Spain, the UK and the US.
Of participants who responded to this question, 54% reported working for pay; this was similar across countries with the lowest being France. Twenty percent reported not working due to disability; this was highest in the US (27%) and lowest in Italy (10%).
Of those working for pay, 74% reported their work-life was affected by CMT1A. The highest rates were in Spain (96%), while the lowest were in Italy (65%). Frequently reported ways that CMT1A affected work-life were the type of job (54%) who specified ways in which work-life was affected, number of sick days (30%), and working part-time (30%). Participants reported missing a mean of 1.4 workdays in the past two weeks due to CMT1A, equivalent to approximately 36 days per year.
Of those unemployed (7%), 71% reported that CMT1A was a contributing factor. The highest rates were in Italy (91%), while the lowest were in Germany (0%).
CMT1A has a substantial impact on patients’ ability to work, which is comparable across European countries and the US. Patients are absent from work approximately 36 days per year due to CMT1A.
***Further research is needed to explore indirect costs associated with these losses and to better manage the impact on patients’ work lives.
Patient-Reported Symptom Burden of Charcot-Marie-Tooth Disease Type 1A (CMT1A)
This analysis aimed to examine patient-reported symptom burden for Charcot-Marie-Tooth disease type 1A (CMT1A) in European and US real-world practice. CMT1A patients were recruited and asked questions via patient-reported outcome measures. This interim analysis examined 937 participants from France, Germany, Italy, Spain, the UK, and the US.
The CMT1A symptoms ranked with the highest importance by participants were weakness in hands and fingers (most important, 32%), difficulty walking (15.5%), weakness in the feet (13.0%), fatigue (8.7%), weakness in the legs (5.7%), and problems with balance (sixth most important, 4.2%). The majority of participants reported the severity of their symptoms to be moderate (58.5%) or severe (24.4%), with almost half of participants (48%) experiencing a worsening of CMT1A symptom severity from initial diagnosis.
Anxiety and depression were each reported by over a third of participants (39.3%, and 37.9%, respectively), higher than the prevalence in the general global population.
There was a high recorded use of rehabilitative interventions (86.5% reported using physical therapy), medications (72.5% reported using painkillers), and orthotics/walking aids (48.6% reported using off-the-shelf insoles). There was a similar reported use of these across all countries.
Patients with CMT1A experience a high level of symptom burden, aligned with clinical observations and literature describing a wide variety of symptoms. Patients first suffer from using limbs, fatigue, pain symptoms, and depression, causing impairment to quality of life.
***It is apparent that there remains a high unmet need in CMT1A caused by the burden on patients.
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