Treatment with autologous hematopoietic stem cell transplant (aHSCT) led to a safe and rapid lessening of disability and no clinical relapses in patients with aggressive multiple sclerosis (MS), according to a new study.

The research, “The use of autologous hematopoietic stem cell transplantation as a first line disease modifying therapy in patients with ‘aggressive’ multiple sclerosis,” was presented at the recent 34th congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) , which concluded in Berlin, Germany last week.

The data was presented by Joyutpal Das from the department of neurology, Sheffield Teaching Hospitals NHS Foundation Trust, United Kingdom.

aHSCT is a procedure that involves collecting stem cells from a patient’s bone marrow, peripheral blood or umbilical cord blood, followed by partial or total impairment of the immune system with a conditioning regimen (called immunoablation), and then infusing the stem cells back into the patient to reconstitute and reactivate the immune system.

This method has been very effective in patients with highly active relapsing MS, who had failed to respond to standard disease modifying therapies (DMTs). The European Society for Blood and Marrow Transplantation guidelines recommend its use as a first-line therapy in patients with aggressive MS.

Aiming to address the procedure’s safety and effectiveness as a first-line strategy among these patients, the study focused on 20 with aggressive MS who received aHSCT at five centers — seven patients in Sheffield, U.K., seven in Uppsala, Sweden, four in Ottawa, Canada, one in Florence, Italy, and one in Chicago, Illinois.

No patient received any standard DMT for more than three months before aHSCT. Patients were treated with either BEAM (carmustine, etoposide, cytarabine, melphalan) chemotherapy plus antithymocyte globulin (ATG) to lessen transplant rejection, cyclophosphamide with ATG, or a triple combination of cyclophosphamide, ATG and busulphan were used as conditioning regimens.

Patients had a mean age at diagnosis of 28 years (range 19-52 years), and a median pre-aHSCT Expanded Disability Status Scale (EDSS) score of 6.5, which means requiring two walking aids to walk about 20 meters (almost 22 yards) without resting. (The higher the EDSS score, the greater the patient’s disability.) Median follow-up duration was 29.5 months.

Patients showed a statistically significant median EDSS score improvement (reduction) of 2.5 between pre-aHSCT and the last follow-up visit. The median EDSS score at the last visit was 2.0, which indicates a minimal disability level. Das also noted that “no patients had post-transplant disability progression.”

No patients experienced a clinical relapse following aHSCT.

New brain/spinal cord lesions were found in three patients during the first follow-up using magnetic resonance imaging (MRI) scan, but no further lesions were found in subsequent scans.

Treatment with aHSCT led to routine toxicities, but did not cause death.

To further support the successful approach with aHSCT, Das reported that among the patients treated “one female naturally conceived and gave birth to a healthy baby,” and “one male patient fathered a healthy baby.”

Overall, the team concluded: “aHSCT was safe and highly effective in inducing rapid and sustained remission in this cohort and was associated with a significant improvement of patients’ level of disability,” the researchers wrote. “This demonstrates the potential role of aHSCT as first line therapy in ‘aggressive’ MS,” they added.

Of note, these results build on data presented in April at the 2018 Annual Meeting of the American Academy of Neurology.