Thymectomy A Rational Choice For Some Patients With Myasthenia Gravis Interview with:

Photograph: Douglas Levere

Dr. Gil Wolfe

Gil I. Wolfe, MD, FAAN
Irvin and Rosemary Smith Professor and Chair
Dept. of Neurology/Jacobs Neurological Institute
Univ. at Buffalo Jacobs School of Medicine and Biomedical Sciences/SUNY
Buffalo General Medical Center
Buffalo, NY 14203-1126 What is the background for this study? What are the main findings?

Response: Thymectomy has been used in myasthenia gravis (MG), in particular those patients who do not have a tumor of the thymus gland, known as a thymoma, for over 75 years without randomized data to support its use. A practice parameter in 2000 on behalf of the American Academy of Neurology stated that the benefits of thymectomy in this population of non-thymomatou smyasthenia gravis patients remained uncertain, classified thymectomy as a treatment option in this group, and called for rigorous, randomized studies.

What we found is that compared to an identical prednisone protocol alone, that extended transsternal thymectomy confers significant benefits to non-thymomatous MG patients over a period of three years after the procedure. The benefits include better disease status, reduced prednisone requirements, fewer hospitalizations to manage  myasthenia gravis worsenings, and a lower symptom profile related to side effects from medications used to control the disease state. What should readers take away from your report?

Response: That thymectomy is a rational approach to treating patients with generalized, non-thymomatous  myasthenia gravis. It provides benefits to patients beyond those of conventional medications alone, such as corticosteroids and other immunsuppressive agents.

What recommendations do you have for future research as a result of this study?

Response: We are still “mining” the large amounts of data collected from patients in the study. One of the first things we plan to investigate now is whether the microscopic appearance of the thymus glands that were removed from patients predicts whether they will have a good response to thymectomy or not. We are also looking at patient blood samples to help develop useful biomarkers for the disease. Is there anything else you would like to add?

Response: This was a challenging study that involved not just neurologists but also thoracic surgeons and pathologists, along with many supportive research staff. It took a long time to complete, some 15 years from start to finish, but in the end it was gratifying to see results that essentially all fell in line, demonstrating benefits for a procedure that we have wondered about for decades.

The trial leadership is grateful to all of our colleagues who contributed their time and energy to answer these questions. I would also like to mention that two members of the trial leadership are no longer with us — Drs. John Newsom-Davis and Fred Jaretzki. That we were able to initiate and complete the study is a tribute to them. And we must thank the patients who put their trust in us — we asked a lot from them as they had to surrender their own decision on whether to undergo a major surgery or not in order to be randomized. Finally the study could not have been completed without the support of the NIH/National Institute of Neurological Disorders and Stroke, the Muscular Dystrophy Association and the Myasthenia Gravis Foundation of America. Thank you for your contribution to the community.


Randomized Trial of Thymectomy in Myasthenia Gravis

Gil I. Wolfe, M.D., Henry J. Kaminski, M.D., Inmaculada B. Aban, Ph.D., Greg Minisman, M.A., Hui-Chien Kuo, M.S., Alexander Marx, M.D., Philipp Ströbel, M.D., Claudio Mazia, M.D., Joel Oger, M.D., J. Gabriel Cea, M.D., Jeannine M. Heckmann, M.B., Ch.B., Ph.D., Amelia Evoli, M.D., Wilfred Nix, M.D., Emma Ciafaloni, M.D., Giovanni Antonini, M.D., Rawiphan Witoonpanich, M.D., John O. King, M.D., Said R. Beydoun, M.D., Colin H. Chalk, M.D., Alexandru C. Barboi, M.D., Anthony A. Amato, M.D., Aziz I. Shaibani, M.D., Bashar Katirji, M.D., Bryan R.F. Lecky, M.D., Camilla Buckley, M.D., Angela Vincent, M.B., B.S., Elza Dias-Tosta, M.D., Ph.D., Hiroaki Yoshikawa, M.D., Ph.D., Márcia Waddington-Cruz, M.D., Ph.D., Michael T. Pulley, M.D., Ph.D., Michael H. Rivner, M.D., Anna Kostera-Pruszczyk, M.D., Robert M. Pascuzzi, M.D., Carlayne E. Jackson, M.D., Guillermo S. Garcia Ramos, M.D., Jan J.G.M. Verschuuren, M.D., Janice M. Massey, M.D., John T. Kissel, M.D., Lineu C. Werneck, M.D., Ph.D., Michael Benatar, M.D., Ph.D., Richard J. Barohn, M.D., Rup Tandan, M.D., Tahseen Mozaffar, M.D., Robin Conwit, M.D., Joanne Odenkirchen, M.P.H., Joshua R. Sonett, M.D., Alfred Jaretzki, III, M.D., John Newsom-Davis, M.D., and Gary R. Cutter, Ph.D., for the MGTX Study Group*

N Engl J Med 2016; 375:511-522
August 11, 2016DOI: 10.1056/NEJMoa1602489

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Last Updated on August 12, 2016 by Marie Benz MD FAAD