Myasthenia gravis affects about 60,000 individuals in the United States.
Understanding the complex needs of patients with rare diseases is often limited, and a small survey of physicians who treat patients with myasthenia gravis (MG) sought to provide real-world knowledge of this disorder.
MG, which affects about 60,000 individuals in the United States, is created by the production of pathogenic immunoglobulin G (IgG) autoantibodies against the neuromuscular junction (NMJ), where nerve cells connect with the muscles they control. These IgG autoantibodies block transmission to muscle fibers, including the acetylcholine receptor; up to 88% of patients have acetylcholine receptor (AChR) autoantibody-positive MG.
AChR autoantibodies trigger the classical complement cascade and damage the NMJ, resulting in impaired muscle contraction ability.
Looking to collect real-world evidence, researchers used the Adelphi Real World MG Disease Specific Programme to query US physicians between March and July 2020. The data included demographics, comorbidities, symptoms, disease history, treatments, and health care resource utilization.
Seventy-eight providers responded to the survey, with information from 456 patients, who had a mean age of 54.5; most of the patients (53.1%) were male. Most (75%) had 1 or more comorbidities. More than one-third listed hypertension as a comorbidity, followed by dyslipidemia and anxiety.
Among patients who had 1 or more comorbidities (n = 334), 88.3% took medication for their conditions. There was a mean total of 2.9 prescribed and non-prescribed medications taken per person for all conditions. Statins were the most commonly prescribed medications for comorbidities, followed by antidepressants and angiotensin II receptor blockers.
Mean time from symptom onset to diagnosis was 9.0 months, and eye symptoms were prevalent, reported in 71.7% of patients.
“Delays in diagnosis can be due to the fluctuating nature of MG symptoms, making MG difficult to diagnose on clinical examination,” the researchers reported. “Diagnoses may also be missed in primary and non-neurology specialist care as patients can have normal serology and electrophysiology results, particularly in ocular MG, suggesting there is a need for education in primary care and of non-neurology specialists, and more reliable methods of detection.”
General fatigue affected 47.1% of patients and over half of those reported the severity as moderate or severe (59.5%, n = 128).
The most frequently prescribed first-line treatment were acetylcholinesterase inhibitors and/or steroids in patients with moderate-to-severe symptoms.
The most prescribed acute treatments were high-dose steroids and intravenous immunoglobulin; symptom exacerbations or myasthenic crises were the most common reasons for acute treatment.
On average, 2.5 healthcare professionals were involved in patient management and 5.0 consultations were made per patient over the last 12 months.
The mean number of symptoms per patient was the same (5) after treatment as it was at diagnosis, suggesting that many patients were still impacted by disease, despite treatment.
Over one-third of the patients in this cohort required acute treatment at some point, predominantly for the treatment of exacerbations or myasthenic crisis.
“Our findings showed that current maintenance treatment does not fully control the disease, as patients continued to experience a high burden of disease and healthcare resource utilization despite treatment, highlighting the need for improved treatment options in MG,” the authors concluded.
Mahic M, Bozorg AM, DeCourcy JJ, et al. Physician-reported perspectives on myasthenia gravis in the United States: A real-world survey. Neurol Ther. 2022 Dec;11(4):1535-1551. doi: 10.1007/s40120-022-00383-3. Epub 2022 Jul 20.