Abstract Details

Title Refractory Myasthenia Gravis Treated with Autologous Hematopoietic Stem Cell Transplantation

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) S34 - Updates on Myasthenia Gravis (2:24 PM-2:36 PM)

Poster/Presentation
Number 008

Background Patients with refractory MG have few treatment options. HSCT has been used to treat immune diseases, however, its use in the treatment of MG is not broadly considered.

Objective Our objective is to report on the efficacy and safety of autologous hematopoietic stem cell transplantation (HSCT) in refractory myasthenia gravis.

Design/Methods Twenty-one patients who underwent HSCT for MG between were retrospectively reviewed. All patients had severe MG refractory to multiple therapies. Stem cells were mobilized with cyclophosphamide and granulocyte colony stimulating factor. The grafts were depleted of immune cells by selecting CD34+ cells. HSCT conditioning consisted of high dose cytoreductive therapy and anti-thymocyte globulin. The primary efficacy outcome was achieving Myasthenia Gravis Foundation of America Post-intervention Status (MGFA-PIS) of clinically stable remission or minimal manifestations without treatment and remaining as such until most recent follow-up. Primary safety outcome was death unrelated to MG.

Results The median (range) time from MG diagnosis to HSCT was 4.0 years (1.9-21.8 years). The primary outcome was reached in 16 of 18 evaluable patients (88.9%) at a median of 1.7 years and maintained with a median follow-up of 6.7 years (range 1.0-21.9 years). Two remaining evaluable patients achieved the PIS of minimal manifestations but required ongoing anti-MG treatment. Three patients were not evaluable for the primary outcome: one due to confounding illness and two due to death within 100 days post-HSCT. The primary safety outcome was reached by 4 patients (19%) as two additional patients died without active MG at 8.6 and 9.4 years post-HSCT.

Conclusions After HSCT for refractory MG, most patients achieved sustained disease remission. However, HSCT-related mortality in medically complex MG patients may be high. Prospective studies investigating the efficacy and safety of HSCT in the treatment of refractory MG are warranted.

Authors/Disclosures
Benjamin Beland, MD PRESENTER	Dr. Beland has nothing to disclose.
Jan Storek, MD, PhD	Dr. Storek has nothing to disclose.
Liam Quartermain	An immediate family member of Mr. Quartermain has received personal compensation for serving as an employee of Wish Collaboration.
Christopher Hahn, MD (Alberta Health Services)	Dr. Hahn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Akcea. Dr. Hahn has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Anylim.
C E. Pringle, MD	Dr. Pringle has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for argenx.
Pierre R. Bourque, MD (Ottawa Hospital, Civic Campus)	Dr. Bourque has nothing to disclose.
Michael Kennah, MD	Dr. Kennah has received personal compensation in the range of $500-$4,999 for serving as a Case Review consultant with Ontario Health/Cancer Care Ontario.
Natasha Kekre, MD	Dr. Kekre has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Gilead.
Chris Bredeson, MD	Dr. Bredeson has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bms. Dr. Bredeson has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Allogene. Dr. Bredeson has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Niaid. Dr. Bredeson has received personal compensation in the range of $100,000-$499,999 for serving as a Clinical lead complex malignant hematology with Cancer care Ontario .
Kareem Jamani, MD	Dr. Jamani has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Vertex. Dr. Jamani has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Jazz.
Christopher White, MD (South Health CampusDepartment of Neurology)	Dr. White has nothing to disclose.
Harold Atkins, MD	The institution of Dr. Atkins has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Mallinckrodt Biotech.

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