Thymectomy may improve MG outcomes while lowering care costs

A thymectomy, the surgical removal of the thymus gland, was projected to improve health outcomes and lower lifetime care costs when added to prednisolone-based care for eligible adults with myasthenia gravis (MG) in the U.K.

Simulations in the study, which focused on eligible adults with generalized MG and no thymus tumor, found thymectomy was projected to save £13,014, or about $17,549, per patient in the base-case analysis. When researchers assumed thymectomy’s benefits lasted a patient’s lifetime, projected savings rose to £49,629, or about $66,874, per patient.

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Model points to long-term savings with thymectomy

“This analysis provided clear proof that the [U.K.’s National Health Service] is getting its money’s worth with thymectomy,” Gil I. Wolfe, MD, a professor at the University of Buffalo and one of the study’s authors, said in a university news story. “The surgery is a one-time cost and, in the long run, much less expensive than the very costly medications that have now become available for myasthenia gravis.”

According to Wolfe, those treatments can cost between $300,000 and $500,000 per year. “Even in the U.S., where surgery costs more, it does not approach a $300,000 price tag,” he said, adding that this is also the case in other countries.

The study, “Economic Evaluation of Thymectomy for the Treatment of Nonthymomatous Myasthenia Gravis,” was published in JAMA Network Open.

In most cases of MG, self-targeting antibodies attack proteins that are key for nerve-muscle communication. Such attacks lead to MG’s hallmark symptoms of muscle weakness and fatigue.

MG treatment focuses on controlling symptoms, maintaining remission, and preventing myasthenic crises, a life-threatening MG complication marked by severe muscle weakness that can lead to respiratory failure, hospitalization, and breathing support.

Thymectomy may reduce need for higher steroid doses

U.K. guidelines recommend starting with pyridostigmine (sold as Mestinon, with generics available), a medication that helps improve nerve-muscle signaling. Even so, most patients also need immunosuppressive therapies, such as corticosteroids, which carry risks with long-term use.

The thymus gland, part of the immune system, is thought to play a central role in the abnormal immune response that drives MG. Thymectomy is required when MG patients have a thymus tumor and may be recommended for certain patients without a tumor, including eligible people with generalized MG.

“If patients do not undergo thymectomy, corticosteroids like prednisolone have to be used in higher doses,” Wolfe said. “As a result, all of the complications of that class of agents — weight gain, sleep disturbance, [high blood sugar], bone loss, skin changes, mood changes — are more likely.”

Despite evidence supporting the clinical benefit of thymectomy, the researchers noted that no studies had assessed its cost-effectiveness in a U.K. context, including within the National Health Service, a publicly funded healthcare system that provides residents access to care at no or low cost.

Because thymectomy entails considerable upfront costs, it remains unclear whether its long-term benefits justify those costs. To find out, Wolfe and colleagues, most of whom were in the U.K., built a mathematical model that simulated what happens to MG patients over a lifetime under two treatment strategies: thymectomy plus prednisolone-based care or prednisolone-based care alone.

The simulated patient population was based on the 126 adults with generalized MG without a thymus tumor who participated in the Phase 3 MGTX clinical trial (NCT00294658), which tested thymectomy plus prednisone versus prednisone alone.

Costs included the surgical procedure, medications, routine monitoring, management of crises and exacerbations, and end-of-life care. Health outcomes were measured in quality-adjusted life years (QALYs), a unit that combines both the length and quality of life, with one QALY equaling one year in perfect health.

Base-case model projected better outcomes and lower costs

Assuming thymectomy’s benefits last for 10 years before waning, patients who received thymectomy plus prednisolone-based care were projected to accumulate more QALYs over their lifetime compared with those on prednisolone-based care alone (14.63 vs. 14.11 QALYs), a gain of 0.52 QALYs.

At the same time, the lifetime cost of care for the thymectomy group was projected to be £181,716 (about $245,090), compared with £194,730 (about $262,663) for the medication-only group, indicating that thymectomy was associated with a cost saving of £13,014 (about $17,549) per patient.

When the model assumed that the benefits of thymectomy lasted the patient’s entire lifetime rather than 10 years, the advantages were even larger. In that scenario, the thymectomy group gained 1.25 additional QALYs, and the cost savings grew to £49,629 (about $66,874) per patient.

Although researchers tested a range of other scenarios, including lower or higher procedure costs or halving the costs of managing crises and exacerbations, thymectomy generally remained dominant, meaning it was projected to be both more effective and less costly. At the U.K.’s standard cost-effectiveness threshold of £25,000 (about $33,729) per QALY gained, more than 99% of simulations judged thymectomy cost-effective.

A final sensitivity analysis found that the most influential factors on the results were the cost of the thymectomy procedure, the cost of managing a myasthenic crisis, the risk of dying during a crisis, and the cost of managing an MG exacerbation. Even when these factors were set to their highest and lowest plausible values, thymectomy remained cost-effective.

“This trial provided a prime example of testing whether a surgical therapy is more effective than medical management alone,” Wolfe said. “Surgery tends to be expensive, but in certain settings, it not only provides improved quality of life but may also lower long-term health care costs.”