HSCT for MS Success Rate: What the Published Evidence Actually Shows

There is no single HSCT success rate for multiple sclerosis. The published figures range from 46 per cent to 81 per cent at five years, and the reason for that spread is not the procedure. It is who was treated.

In relapsing remitting MS treated while the disease is still active, the largest routine care series reports 73 per cent of patients free of any disease activity at five years. In cohorts made up mostly of progressive MS with high disability, the same measure falls to roughly half that. The question “what is the success rate” is answered by the patient, not by the treatment.

Every figure below is attributed to a named peer reviewed study, with the population it came from stated alongside it.

What “Success” Means in MS Research

MS studies rarely use the word success. The measure they use is NEDA, which stands for no evidence of disease activity. A patient counts as NEDA only when all three of the following are true across the follow up period.

• No clinical relapses.
• No new or enlarging lesions on MRI.
• No confirmed worsening of disability on the EDSS scale.

NEDA is a demanding standard. A patient who feels well, walks better and has had no relapse can still fall out of NEDA on a single new MRI spot. That is why NEDA figures are always lower than the proportion of patients who report feeling better, and it is why the three components are usually published separately as well. In the UK cohort, 88.6 per cent were relapse free at five years while combined NEDA sat at 46.2 per cent, because the MRI and disability components pull the combined number down.

Readers comparing HSCT against a drug should check which measure each figure uses. Comparing a drug’s relapse rate reduction against an HSCT NEDA rate compares two different things.

Why the Success Rate Differs So Much Between Patients

The single largest factor in the published data is MS type. Relapsing remitting MS responds better than progressive MS, and the gap widens over time.

Italian multicentre cohort, 210 patients. Boffa, Neurology, 2021.

Three further factors were measured in the largest long term registry analysis of 281 patients, published in JAMA Neurology in 2017. Progressive MS carried more than double the risk of disability progression compared with relapsing MS. Each additional year of age raised that risk. Having already failed more than two disease modifying drugs raised it again.

Read together, those three findings point the same way. The published outcomes are strongest in patients treated while the disease is still inflammatory and before disability has accumulated. That is the practical answer to the success rate question, and it is why an eligibility assessment is worth doing early rather than after another year of decline.

Progressive MS is not excluded. A 2023 analysis in Neurology of patients with active secondary progressive MS found sustained disability improvement at three years in 34.7 per cent of those treated with HSCT, against 4.6 per cent of a matched group on other therapies. The word that matters in that sentence is active. Assessment is individual, and the HSCT specialists at HSCT Hospital India review each case on its own scans and history.

What the Randomised Trials Show

Most HSCT evidence comes from cohorts and registries. Two randomised controlled trials have reported, and several more are running.

MIST, published in JAMA in 2019, randomised 110 patients with relapsing remitting MS to non myeloablative HSCT or to continued disease modifying therapy. Disease progression occurred in 3 of 55 transplanted patients against 34 of 55 on drug therapy. Mean EDSS score improved from 3.38 to 2.36 at one year in the transplant group. There were no deaths. Median follow up was two years.

ASTIMS, published in Neurology in 2015, was a smaller phase two trial comparing HSCT against mitoxantrone. It found 79 per cent fewer new MRI lesions after transplant, and no measurable difference in disability progression between the two groups over its follow up.

Larger comparisons against modern high efficacy drugs are still in progress. BEAT-MS in the United States, RAM-MS in Norway and StarMS in the United Kingdom are all running and none has published a primary results paper. Anyone quoting a figure from those trials today is quoting something that does not yet exist.

In the meantime, matched comparisons from the MSBase registry, published in JAMA Neurology in 2023, found HSCT produced lower relapse rates than fingolimod and greater disability improvement than natalizumab, with results broadly similar to ocrelizumab over three years.

Safety: What the Same Studies Report

HSCT is a hospital procedure involving chemotherapy, and the published series report treatment related mortality alongside the outcome figures. The number has fallen sharply as regimens moved from myeloablative to non myeloablative protocols and as patient selection improved.

The pattern across those rows is consistent. The two series using non myeloablative conditioning in relapsing remitting patients report no treatment related deaths at all, and where deaths did occur in the UK series they were confined to patients already at EDSS 6.5. Careful selection is what moves this number, which is the reason the assessment stage matters as much as the transplant itself.

HSCT Hospital India uses a non myeloablative protocol, and treatment is given in a deluxe private room with triple level HEPA air filtration for the period when the immune system is rebuilding.

Four Claims to Treat With Care

• A single success rate figure. Published five year NEDA runs from 46 per cent to 81 per cent depending on who was in the cohort. Any source quoting one number without naming the population has left out the part that determines it.
• Relapsing figures presented as progressive figures. The ten year gap between the two is 14 percentage points in the Italian cohort. They are not interchangeable.
• Zero risk. Treatment related mortality across the published literature runs from 0 to 2.8 per cent depending on era, regimen and baseline disability.
• Results from trials that have not reported. BEAT-MS, RAM-MS and StarMS have no published primary results.

What Patients Say Themselves

Fourteen patients treated at HSCT Hospital India have filmed what changed for them, and their videos and full transcripts are published on this site. Gudrun R from the United Kingdom arrived pushing a rollator and was walking with a stick five weeks later. Evert from Canada came for myasthenia gravis with a drooping eyelid, weak hands and difficulty breathing, and reports his hand strength, calf strength and breathing all responded.

Individual accounts are not a success rate, and the studies above are the place to look for that. What the accounts do show is the shape of a real recovery week by week, which no percentage can convey.

Read Next

• HSCT for multiple sclerosis: the full treatment guide
• Patient results and video testimonials
• What the HSCT treatment package includes
• HSCT cost compared by country
• EDSS score calculator

References

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2. Muraro PA et al. Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurology, 2017. PMID 28241268
3. Burt RK et al. Effect of nonmyeloablative HSCT vs continued disease-modifying therapy on disease progression in relapsing-remitting MS (MIST). JAMA, 2019. PMID 30644983
4. Mancardi GL et al. Autologous hematopoietic stem cell transplantation in multiple sclerosis: a phase II trial (ASTIMS). Neurology, 2015. PMID 25672923
5. Silfverberg T et al. Haematopoietic stem cell transplantation for relapsing-remitting multiple sclerosis in Sweden. JNNP, 2024. PMID 37748927
6. Boffa G et al. Long-term clinical outcomes of hematopoietic stem cell transplantation in multiple sclerosis. Neurology, 2021. PMID 33472915
7. Boffa G et al. Hematopoietic stem cell transplantation in people with active secondary progressive multiple sclerosis. Neurology, 2023. PMID 36543569
8. Kalincik T et al. Comparative effectiveness of AHSCT vs fingolimod, natalizumab and ocrelizumab in highly active relapsing-remitting MS. JAMA Neurology, 2023. PMID 37437240
9. Muraro PA et al. Real-world effectiveness of AHSCT for multiple sclerosis in the UK. JNNP, 2026. PMID 40912910
10. Kazmi M et al. AHSCT for multiple sclerosis in the UK: a 20-year retrospective analysis. British Journal of Haematology, 2025. PMID 40500866
11. Brittain G et al. StarMS trial protocol. BMJ Open, 2024. PMID 38316583