AAN 2023: Low rates of disease activity after stem cell transplant

There was no evidence of multiple sclerosis (MS) disease activity for at least two years in patients who underwent an autologous hematopoietic stem cell transplant (aHSCT) at a center in Mexico.

And by the most recent follow-up — a median of about seven years after the procedure — more than two-thirds still saw no disease activity, according to data presented in a poster titled, “Autologous hematopoietic stem cell transplantation in multiple sclerosis: A 20-year single center’s experience in Mexico.”at the American Academy of Neurology (AAN) 2023 Annual Meeting this week in Boston and virtually.

aHSCT, or stem cell therapy, is a one-time procedure to reset a person’s immune system by collecting blood stem cells (which give rise to immune cells) from a patient’s bone marrow then administering intensive chemotherapy and/or radiation to destroy existing immune cells. The stem cells are then transplanted back into the patient, with the aim of repopulating healthy immune cells that are less prone to the type of inflammatory attack that drives MS.

The full aHSCT protocol usually requires several weeks in the hospital. It’s not specifically authorized to treat MS in the U.S., but has been recognized as a feasible option for select groups of patients by the National MS Society.

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NEDA3 after two, seven years follow-up

In the poster, scientists shared outcomes for 22 MS patients who underwent aHSCT at a center in Mexico City between 2000 and 2020 and had at least one year of follow-up.

About two-thirds were men. Half had secondary progressive MS (SPMS), six had relapsing-remitting MS (RRMS), and five had primary progressive MS (PPMS). Most had tried more than one MS therapy before aHSCT.

To assess outcomes, the researchers reviewed a score called NEDA3 (no evidence of disease activity 3). In order to meet NEDA3 criteria, there must be no new relapses, no sustained worsening of disability, and no new disease activity on MRI scans.

In the two years after aHSCT, all 22 patients achieved NEDA3, results showed. Sixteen (72.1%) were still in NEDA3 after a total median follow-up of more than seven years.

The most common reason for not remaining in NEDA3 was new relapse activity, which occurred in five (22.7%) patients. New MRI activity was reported in two patients and only two had any notable disability progression throughout the study.

The patients who didn’t achieve NEDA3 after aHSCT generally had more active disease before the procedure, the researchers noted.

Seven of the 22 patients had significant safety issues related to the aHSCT procedure itself, including severe infections and fever, though none died due to complications related to it.

Over long-term follow-up, two patients developed epilepsy (seizures), one developed a type of blood cancer called non-Hodgkin’s lymphoma, and another had premature ovarian failure, where the ovaries stop making eggs and hormones earlier than would be expected for menopause. The poster didn’t specify if there was any apparent connection between the procedure and these long-term complications.

“This retrospective single-center observational study shows that AHSCT using a low-intensity regimen is relatively safe when done in highly experienced centers, with good long term outcomes in RRMS and SPMS patients. Given the lack of treatment option for SPMS, AHSCT may be a viable option for mitigating disease progression when other options are not available,” the scientists said.

Note: The Multiple Sclerosis News Today team is providing coverage of the American Academy of Neurology (AAN) 2023 Annual Meeting April 22-27. Go here to see the latest stories from the conference.