Abstract Details

Title Cladribine to treat Relapsing and Progressive Multiple Sclerosis - experience in >200 patients

Topic Multiple Sclerosis

Presentation(s) S26 - MS and CNS Inflammatory Disease: Clinical Considerations II (2:17 PM-2:28 PM)

Poster/Presentation
Number 008

Background

Oral cladribine (Mavenclad) has been licensed by the EMA in 2017 as a disease-modifying treatment (DMT) for people with highly active relapsing MS (RMS) and is currently under FDA consideration. We have used a subcutaneously injected preparation of cladribine (Litak®?) to treat pwMS since 2014.

Objective

To report safety and efficacy data using cladribine as an immunotherapy for people with MS (pwMS).

Design/Methods Litak®? was offered as an off-label treatment option to pwMS who either (i) did not met NHS-England (NHSE) criteria for licensed DMT or (ii) met NHSE DMT criteria, but chose Litak®? instead. Assessment of disease activity was based on MRI and/or cerebro-spinal fluid neurofilament light chain level. Litak®? 10mg was administered on three days (four in pwMS >90kg) during week 1. Based on individual week 4 lymphocyte count, pwMS were given another 0-30mg Litak®? (personalised dosing, PD) in week 5. A second course of treatment was given after 48 weeks. Follow-up included adverse events (AEs), annual EDSS and MRI, and regular blood counts. The proportion of pwMS with no evidence of disease activity (NEDA) and no evidence of progression and active disease (NEPAD) was calculated.

Results

A total of 202 pwMS (99 RMS, 103 progressive MS) underwent at least one treatment cycle. Age at baseline was 45 (17-72) years, disease duration 11 (1- 48) years. AEs included 1 death (influenza; EDSS 8.5), 2 cases of erythema multiforme, 1 myocardial infarct, mild infections, pain, injection site reactions. Lymphopenia grade 3 was detected in 3/202, and grade 4 in none. At two years 69% of pwRMS were NEDA, and at two years NEPAD in pwPMS was 31%.

Conclusions

Litak®? PD was well tolerated and prevented significant lymphopenia in 98% of pwMS. NEDA and NEPAD data resemble those reported with clinical trials of highly effective DMTs including oral cladribine and ocrelizumab.

Authors/Disclosures
Stefania De Trane PRESENTER	Stefania De Trane has nothing to disclose.
	No disclosure on file
	No disclosure on file
Kimberley Allen-Philbey, MSc (The Blizard Institute (Neuroscience and Trauma))	Miss Allen-Philbey has nothing to disclose.
Ozlem Yildiz, PhD (Blizard Institute)	Dr. Yildiz has nothing to disclose.
Tom Campion	No disclosure on file
	No disclosure on file
Benjamin Turner, MD, MBBS (Barts and The Royal London Hospitals)	No disclosure on file
	No disclosure on file
	No disclosure on file
Sharmilee Gnanapavan, MB (Institute of Neurology)	Dr. Gnanapavan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Merck. The institution of Dr. Gnanapavan has received research support from NIHR.
	No disclosure on file
Joela T. Mathews, MRPSGB (Barts Health NHS Trust)	Miss Mathews has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Novartis. Miss Mathews has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Roche. Miss Mathews has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Celegene.
Gavin Giovannoni, MD (QMUL)	Dr. Giovannoni has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Dr. Giovannoni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sanofi. Dr. Giovannoni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Merck KGaA. Dr. Giovannoni has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Novartis. Dr. Giovannoni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Roche-Genentech. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Janssen. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Moderna. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GSK. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Aurinia Pharmaceuticals. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sandoz. Dr. Giovannoni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Astoria Biologica. Dr. Giovannoni has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Viracta. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zenas. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Novartis. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Biogen. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Sanofi. Dr. Giovannoni has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Medscape. Dr. Giovannoni has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Oxford Health Policy Forum. Dr. Giovannoni has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Peervoice. Dr. Giovannoni has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Bristows .
David Baker	No disclosure on file
Klaus Schmierer, MD, PhD (The Blizard Institute, Centre for Neuroscience, Surgery & Trauma)	Dr. Schmierer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for AXDEV. Dr. Schmierer has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for MedScape. Dr. Schmierer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for NeuroDiem. Dr. Schmierer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurology Academy. Dr. Schmierer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck KGaA. Dr. Schmierer has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Merck KGaA. Dr. Schmierer has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for F. Hoffmann-La Roche AG. Dr. Schmierer has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Novartis. Dr. Schmierer has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Teva.

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