The goal of this observational study is to study the long-term effects of autologous hematopoietic stem cell transplantation (aHSCT) in people with highly active relapsing-remitting multiple sclerosis. The study will evaluate the following items: 1. Disease activity 2. Safety and tolerability of aHSCT 3. Changes in the immune system Participants will be subjected to frequent visits for five years after treatment with aHSCT. During these visits, clinical testing, evaluation by questionnaires, MRI scans and blood sampling will be performed.
Study Type
OBSERVATIONAL
Enrollment
24
Amsterdam UMC
Amsterdam, Netherlands
RECRUITINGSt. Antonius Hospital
Nieuwegein, Netherlands
RECRUITINGTreatment efficacy
The proportion of patients with ne evidence of disease activity-3 (NEDA-3) as defined by: no clinical relapse, no disability progression, no radiological disease activity.
Time frame: 2 years
Annual relapse rate
Time frame: 2 years
Time to first relapse
Time frame: 2 years
Confirmed disability progression (CDP)
Measured by the Expanded Disability Status Scale (EDSS) CDP-EDSS is defined as an increase of one point in the EDSS score from baseline to month 24.
Time frame: 2 years
Confirmed disability improvement (CDI)
Measured by the Expanded Disability Status Scale (EDSS) CDI-EDSS is defined as a decrease of one point in the EDSS score from baseline to month 24, with an absence of relapse at the point of assessment.
Time frame: 2 years
Progression independent of relapse activity (PIRA)
Defined as an episode of CDP without relapse during the 90 days before EDSS increase and during the 6-month period between the EDSS increase and the confirmation of disability progression.
Time frame: 2 years
Changes on the multiple sclerosis functional composite (MSFC)
MSFC consists of the timed 25-foot walk test, 9-hole peg test and standard digit modalities test (SDMT)
Time frame: 2 years
Brain/spinal cord MRI
Presence and number of contrast-enhancing lesions at baseline and the development of new or enlarging lesions between baseline and follow-up will be assessed.
Time frame: 2 years
Optical coherence tomography (OCT)
Developments of optic neuritis and longitudinal changes of the retinal nerve fiber layer will be assessed.
Time frame: 2 years
Side-effects and toxicity
Frequency and type of (serious) adverse events such as infections, secondary auto-immunity, fertility problems, clinical relevant changes on physical examination and use of concomitant medications will be assessed
Time frame: 2 years
Multiple Sclerosis Impact Scale-29 (MSIS-29)
Patient reported outcome measures about the impact of MS on daily life
Time frame: 2 years
EuroQoL 5D (EQ-5D-5L)
Patient reported outcome measures about quality of life
Time frame: 2 years
Modified Fatigue Impact Scale (MFIS-5)
Patient reported outcome measures about fatigue
Time frame: 2 years
Hospital Anxiety and Depression Scale (HADS)
Patient reported outcome measures about anxiety and depression
Time frame: 2 years
iMTA Medical Cost Questionnaire (iMCQ)
Patient reported outcome measures about medical consumption
Time frame: 2 years
iMTA Productivity Cost Questionnaire (iPCQ)
Patient reported outcome measures about productivity
Time frame: 2 years
Treatment Satisfaction Questionnaire for Medication (TSQM)
Patient reported outcome measures about medication
Time frame: 2 years
Biomarkers
Serum NfL and GFAP over time
Time frame: 2 years
Proteomics
Olink discover panel
Time frame: 2 years
Immune phenotyping
Characterization of innate and adaptive immune subsets with mass spectrometry imaging
Time frame: 2 years
Genetic analysis
Immune gene profiling for gene signatures predictive for response to aHSCT
Time frame: 2 years
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