Abstract Details

Title Lenalidomide in Anti-MAG Neuropathy: Phase 1b Study

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) Neuromuscular and Clinical Neurophysiology (EMG) Posters (7:00 AM-5:00 PM)

Poster/Presentation
Number 060

Objective

This study aims to determine the maximum tolerated dose (MTD) and examine the safety profile and efficacy of Lenalidomide in patients with anti-myelin associated glycoprotein (MAG) neuropathy.

Background Anti-MAG neuropathy is a rare autoimmune demyelinating disorder of the peripheral nerves that presents with weakness, gait imbalance, and sensory loss, and is generally refractory to all available treatments used in acquired demyelinating neuropathy. Case reports suggest potential efficacy of Lenalidomide, an immunomodulatory drug used in plasma cell dyscrasias.

Design/Methods

We prospectively enrolled patients ≥ 18yo with Anti-MAG titer positivity and distal acquired demyelinating sensorimotor (DADS). Patients received Lenalidomide 10, 15, or 25mg (days 1-21 of 28-day cycle) and dexamethasone 20mg (days 1,8,15 and 22) following a Bayesian optimal interval design. The primary outcome was MTD of Lenalidomide. Secondary outcomes of treatment efficacy included measures of strength (MRCSS, grip strength), disability (INCAT, I-RODS, SARA), and quality of life (EQ-5D-5L). All patients received aspirin or anticoagulation for DVT prophylaxis.

Results 7 subjects (6 men, mean age 67y), out of our target cohort of 20, have already enrolled in the year-1 dose-escalation phase. All patients have tolerated the 25 mg Lenalidomide dose, with no dose-limiting toxicities (DLTs), although one patient developed pulmonary embolus (expected). 4 subjects have completed 9-12 months of enrollment, with 3 patients showing positive trends across both I-RODS (baseline range 32-46, post-treatment range 39-47) and Jamar grip strength (baseline range across both hands 68-98, post-treatment range 75-100). We plan to share additional preliminary findings by the time of the meeting.

Conclusions Initial findings suggest Lenalidomide is well tolerated and safe in anti-MAG patients, with most patients appearing to tolerate the 25mg dose with no significant DLTs. Preliminary efficacy trends are encouraging, and warrant continued and larger study exploration.

Authors/Disclosures
Amro Stino, MD (Michigan Medicine - University of Michigan) PRESENTER	Dr. Stino has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for CSL Behring. Dr. Stino has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Argenx. Dr. Stino has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Dr. Stino has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. Dr. Stino has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Annexon. The institution of Dr. Stino has received research support from GBS-CIDP Foundation. The institution of Dr. Stino has received research support from Bristol Myers Squibb.
Bakri Elsheikh, MD, F�鶹��ýӳ�� (The Ohio State University Wexner Medical Center)	Dr. Elsheikh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biogen . Dr. Elsheikh has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argnex . The institution of Dr. Elsheikh has received research support from Biogen. The institution of Dr. Elsheikh has received research support from Cure SMA.
	No disclosure on file
	No disclosure on file
	No disclosure on file

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