Aim: Myasthenia gravis (MG) is a rare and heterogeneous disease for which there is no generally accepted standard of care. Thus, it is critical that MG experts develop consensus guidelines based on their practice and ...Aim: Myasthenia gravis (MG) is a rare and heterogeneous disease for which there is no generally accepted standard of care. Thus, it is critical that MG experts develop consensus guidelines based on their practice and disease management to assist clinicians and provide advice for insurance companies, health organizations and institutional review boards. Methods: An international treatment guidance was developed based on national guidelines established in the US, Denmark, Norway, Germany, Japan, Netherlands, United Kingdom and Europe. The RAND/UCLA appropriateness method (RAM) was applied to reach consensus among 15 worldly renowned experts and experienced clinicians. Results: This paper introduced the RAM procedure with its principles and applications and conducted a brief review of the resulting 2016 international consensus guidance for MG in comparison to clinical experience and management of Chinese MG patients. Conclusion: The 2016 international consensus guidance is a major contribution to the treatment and management of MG, providing an up-to-date expert consensus to assist clinicians around the world, especially those with limited experience and/or practice in countries/regions that have limited resources to develop local treatment guidelines. It is also an important contribution showing how RAM can help to develop consensus guidance for treatment of rare diseases based on scientific findings and expert experience.展开更多
文摘Aim: Myasthenia gravis (MG) is a rare and heterogeneous disease for which there is no generally accepted standard of care. Thus, it is critical that MG experts develop consensus guidelines based on their practice and disease management to assist clinicians and provide advice for insurance companies, health organizations and institutional review boards. Methods: An international treatment guidance was developed based on national guidelines established in the US, Denmark, Norway, Germany, Japan, Netherlands, United Kingdom and Europe. The RAND/UCLA appropriateness method (RAM) was applied to reach consensus among 15 worldly renowned experts and experienced clinicians. Results: This paper introduced the RAM procedure with its principles and applications and conducted a brief review of the resulting 2016 international consensus guidance for MG in comparison to clinical experience and management of Chinese MG patients. Conclusion: The 2016 international consensus guidance is a major contribution to the treatment and management of MG, providing an up-to-date expert consensus to assist clinicians around the world, especially those with limited experience and/or practice in countries/regions that have limited resources to develop local treatment guidelines. It is also an important contribution showing how RAM can help to develop consensus guidance for treatment of rare diseases based on scientific findings and expert experience.
