Abstract Details

Despite over 20 disease-modifying therapies (DMTs), MS remains a significant cause of morbidity and mortality. Poor treatment response to DMTs for some patients led to exploration of autologous hematopoietic stem cell transplantation as treatment for MS. However, not all patients met inclusion criteria for aHSCT clinical trials, prompting some patients to seek aHSCT outside of clinical trials at centers in the United States and elsewhere. Whether clinical outcomes of these patients differs from those meeting trial inclusion criteria is currently unknown and will be evaluated with this study.

To characterize the University of Utah patient population with multiple sclerosis (MS) who received autologous hematopoietic stem cell transplantation (aHSCT) outside of clinical trials and their clinical response

We performed a retrospective review of patients within the University of Utah Healthcare system meeting the following criteria: (1) ICD-10 code G35 (multiple sclerosis), (2) aHSCT performed outside of a clinical trial for treatment of MS, and (3) have been seen at the University of Utah between 1/1/2014 and 12/1/2020

Thirteen patients met inclusion criteria. Fifty-seven percent were female, and average age at time of aHSCT was 41 years. The majority (64%) had relapsing-remitting MS while 36% of patients had secondary progressive MS. Sixty-four percent of patients had no relapses in the preceding 12 months and 43% had no new disease activity on MRI. During a median follow up duration of 30 months, 9 patients (64%) had neither relapse or gradual progression. Seventy-nine percent of patients had no new disease activity on MRI post-aHSCT. Two patients were restarted on DMT due to disease progression. There was no significant difference in EDSS or Hauser ambulation index pre and post-aHSCT.

Despite only one patient meeting inclusion criteria for contemporary aHSCT clinical trials, the majority of our patients had no clinical or radiographic disease progression post-aHSCT.