Treatment of Charcot-Marie-Tooth (CMT) disease represents a substantial economic burden, which increases with age, disease severity, comorbidities, obesity and perceived impairments, according to a German study.
The study, “Cost of illness in Charcot-Marie-Tooth neuropathy: Results from Germany,” was published in the journal Neurology.
The team intended to assess the cost of CMT treatment in German patients, identified via a national patient registry. A total of 480 patients completed a questionnaire in 2015 regarding health status, consumption of healthcare resources, other CMT-related expenses, and perceived impairments. Of these, 397 had genetically confirmed diagnosis and were included in the analysis.
Among study participants (age range 3–95, median age 47 years), the most frequent CMT subtype was CMT type 1A (62%). Assessment of the CMT Neuropathy Score in 274 patients revealed that most patients self-reported impairments in daily or work activities. One-third lived in a household where at least one more person had CMT, most often children of the patient (54.9%) or parents (49.6%).
Also, 71.5% of the patients reported CMT-associated comorbidities, such as scoliosis (11.3%), foot or toe deformities (6.5%), restless legs syndrome and hip dysplasia (4.3% each) or hearing loss (4.0%).
As for medical and non-medical resources associated with CMT, medical aids (84.6%), outpatient visits (83.6%) and physiotherapy (67.6%) were frequently used. The participants had a median of six physician visits per year.
Mobility aids were used by 81.5%, while those supporting daily living or care were used by 44.5% and 7.7%, respectively. Overall, the patients used four different types of aids. Of those reporting further use, physiotherapy was the most frequent type of aid, used by 91.7% of participants, followed by foot care (33.0%) and occupational therapy (30.8%). In contrast, inpatient treatment (11.2%) and psychological assistance (11.1%), were among the least used.
Almost half of the patients (47.0%) received care by relatives or friends, most frequently partners and spouses (39.9%). Also, 35.9% needed changes in their workplace, house, or car. Other reported resources included fitness classes (28.6%) and legal advice (7.4%).
In total, this led to a calculated annual per-person cost of €17,427 euros ($22,362 US), which is lower than those reported in Germany for Duchenne muscular dystrophy (DMD, €78,913, or $88,936) and spinal muscular atrophy (SMA, €70,566, or $79,528), the team wrote.
Two-thirds of this was direct costs — €11,735, or $15,059 — mainly related to physiotherapy, foot care, and occupational therapy. Direct non-medical costs represented 40.5% of the total.
In turn, indirect costs due to missing days at work and other factors were estimated at €5.931, or $ 7,610.
Patients unable to work due to CMT, those married or living in a partnership, with CMT-associated or other comorbidities, reporting impairments in daily or school/work activities and needing permanent attendance, had higher total costs.
As for CMT subtypes, patients with CMT2 (mean €17,664, $19,907) and CMT4 (€24,235, $27,313) had the highest costs. Also, those aged 46-60, patients overweight or obese, and patients with a medium educational level had higher costs compared to younger patients, those with normal weight, and patients with high educational level, respectively.
The data further revealed that costs were higher with greater disease severity, but tended to be lower with each year after symptoms onset. According to the researchers, this may be due to better coping strategies along disease duration.
Taken together, the annual total cost of CMT in Germany was estimated at €572.8 million ($644.8 million), which, due to its higher prevalence, is higher than that of DMD and SMA in the country, the scientists added.
Of note, 15 patients (age 13-71, diverse CMT subtypes) reported no costs. All were slightly or moderately affected by the disease.
In conclusion, “CMT is associated with a substantial economic burden,” the scientists wrote. They added that besides serving “as important input to decision-making in health policy,” the findings “indicate the importance of the patient-reported perception of disease severity related to the consumption of resources.”
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