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Hematopoietic Stem Cell Transplantation Shown to Benefit MS Prognoses

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A national single-center observational retrospective study reveals the long-term benefits of hematopoietic stem cell transplantation in the treatment of multiple sclerosis (MS).

Hematopoietic stem cell transplantation (HSCT) has demonstrated great promise in the long-term treatment of multiple sclerosis (MS) and disease-free survival, according to a recent study published in Multiple Sclerosis Journal.

Stem Cell Transplant Model | image credit: freshidea - stock.adobe.com

Stem Cell Transplant Model | image credit: freshidea - stock.adobe.com

In Norway, patients with aggressive forms of MS that are resistant to disease-modifying therapies (DMTs) have the option to engage with HSCT. As the authors of the present study mention, HSCT has demonstrated great efficacy in the treatment of relapsing-remitting MS (RRMS). To expand on the literature available in this subject, they conducted a study in Norway to assess the long-term patient outcomes for those who underwent HSCT.

From January 2015 through January 2018, 29 patients with RRMS who were treated with HSCT were retrospectively analyzed. Patients were followed alongside clinical controls at 3, 6, and 12 months after HSCT at Haukeland University Hospital. There was also a 5-year follow-up. The progression or improvement of their disease was defined as a 1-point increase or decrease of the Expanded Disability Status Scale (EDSS; otherwise defined as a 0.5 difference if their baseline EDSS was at or above 5.5). Furthermore, no evidence of disease activity (NEDA-3) was marked by a composite score comprising a lack of clinical relapse, sustained disability progression, and the absence of new MRI disease activity.

Before their HSCT, 7% of patients (n = 2) were exposed to 1 DMT, 48% (n = 14) had received 2 DMTs, 24% (n = 7) had exposure to 3 DMTs, and 21% (n = 6) had over 3. At baseline, those with active disease exhibited significantly higher average EDSS scores compared with patients without active disease (3.7 vs 2.4; P = .02).

After an average follow-up of 70 months, 69% of patients (n =20) reached NEDA-3 status. By the end of follow-up, clinical outcomes showed patients were relapse free, MRI event free, and progression free at rates of 82.8% (n = 24), 79.3% (n = 23), and 89.7% (n = 26), respectively.

An average 1.5-point decrease in EDSS was observed in 38% (n = 11) of patients, and a stable EDSS in 52% (n = 15). Ten percent of patients exhibited disease progression (n = 3), 21% had new MRI T2-lesions (n = 6), and 17% experienced clinical relapses (n = 5).

The researchers also considered changes in working status among the patients who underwent HSCT. Baseline data showed that 1 patient had a full-time working status, and 5 patients were receiving full-time disability benefits. At the 2-year mark post HSCT, 10 patients were working full time; this number increased to 15 patients at the 5-year mark.

Of the later adverse events recorded, autoimmune thyroid diseases were diagnosed in 17% of patients (n = 5): 3 cases of Graves’ disease and 2 of hypothyroidism. Among 21 of the female patients, 62% (n = 13) experienced chronic symptoms of ovarian failure.

In their concluding thoughts, the authors highlighted that HSCT is a 1-time treatment that can benefit patients who are more vulnerable to infections or long-term immunosuppression. Furthermore, the long-term disease activity and work participation benefits observed in patients treated with HSCT starts to form an argument for the use of HSCT over modern DMTs.

Reference

Kvistad CE, Lehmann AK, Kvistad SAS, et al. Autologous hematopoietic stem cell transplantation for multiple sclerosis: Long-term follow-up data from Norway. Mult Scler. Published online February 12, 2024. doi:10.1177/13524585241231665

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