AHSCT as an MS cure

The AHSCT holds the potential to being a “cure” for MS

Some patients who underwent transplant (i.e., AHSCT) before 2010 have achieved 15 consecutive years of NEDA-3 (No Evidence of Disease Activity), without having used any other DMT (Disease Modifying Treatment) post-transplant. These patients meet the essential criteria of the working definition of being “cured” of MS.

For more details about working definition of cure read “Working definition of cure MS” section.

🔹Back to index.

Rationale of AHSCT

AHSCT has the potential to eliminate the immunological mechanism causing CNS (Central Nervous System) lesions and renew the immune system, making it less aggressive toward the patient’s own CNS.

Other therapies like Alemtuzumab (Lemtrada) and Cladribine (Mavenclad) are also classified as IRTs (Immune Reconstitution Therapies), however the strongest scientific data regarding the possibility of achieving an MS “cure” comes from studies on AHSCT, and this website is focused on this procedure.

The figure above provides a simplified overview of the factors underlying the pathogenesis of MS. Many evidences suggest that to develop MS, it is necessary, but not sufficient, to have both a genetic predisposition and to have contracted an infection, in most cases asymptomatic, of EBV (Epstein-Barr Virus).

To these two conditions, environmental factors must be added, such as childhood obesity, vitamin D deficiency, smoking, environmental pollution, alterations in intestinal flora, and perhaps others yet unknown, and whose specific role is not yet defined.

The combination of these conditions leads to modifications of the immune system which is responsible for defending our body against pathogens such as viruses and bacteria and consists of certain types of white blood cells (lymphocytes, monocytes), lymph nodes, spleen, and thymus.

The immune system in people who develop MS, in addition to performing the normal defense functions of the body, also becomes aggressive against the CNS; this activation occurs in different phases. Firstly, in the minority of patients, the immune system produces autoantibodies against the nervous system; subsequently, lymphatic cells are formed that are auto-aggressive, meaning they can attack the nervous system, particularly the myelin.

Auto-aggressive lymphatic cells multiply (clones) under particular conditions, enter the CNS, and cause inflammation, which can lead to symptoms (new lesions evident on MRI sometimes with neurological symptoms, i.e., clinical attacks or relapses).

How do therapies against MS work?

Therapies capable of modifying the course of MS (DMTs, Disease Modifying Therapies) all act on the immune system, with different mechanisms. DMTs can be divided into therapies administered chronically because their interruption can lead to the resumption of the disease, and IRTs that are administered only in short cycles in the first two years because they can have a longer lasting effect on the immune system.
 

The IRTs are cladribine (Mavenclad) (Giovannoni et al., 2022) and alemtuzumab (Lemtrada) (von Essen et al., 2023); all other DMTs are administered chronically.

AHSCT is an IRT procedure, performed only once. Some authors consider anti-CD20 therapies as IRTs, such as Lünemann et al., 2020. For a comparison between AHSCT vs. DMTs, see table here.

🔹Back to index.

What is AHSCT?

AHSCT is a lengthy procedure performed once if a patient is deemed suitable for it. After signing informed consent, a series of tests are conducted to mitigate risks. If necessary, fertility preservation procedures are performed (applicable to both genders).

Subsequently, stem cells are collected through a process called mobilization, where they are stimulated to move from the bone marrow to the bloodstream. These cells are then collected via a procedure called leukapheresis and subsequently preserved and frozen.

Once a sufficient number of stem cells have been collected, the next phase, called conditioning, proceeds. This involves the elimination of autoreactive lymphocytes clones, including those aggressive to the nervous system.

Currently, several conditioning regimens are available, classified by the EBMT guidelines based on their “myeloablative potential” as high-, intermediate-, or low-intensity regimens. Find insights on conditioning regimens here.

After the conditioning phase, the patient’s own stem cells are infused intravenously. The stem cells migrate to the bone marrow where they regenerate new blood and immune system cells with lower CNS reactivity.

Conditioning phase requires hospitalization which can last approximately 1.5-3 weeks in a hematology ward.

Below is an extended version of a video describing the different phases of a transplant.

📌 For a shorter version of the video, click here.

🟢 For more information, see insights below

🟢 For detailed information, see “procedures“.

Some history about AHSCT

This therapy was developed by Dr. E. Donnall Thomas in 1957. Thomas, a pioneer in the field of bone marrow transplants, conducted this groundbreaking procedure using a patient’s hematopoietic stem cells to treat a hematologic disease. Thanks to his discovery, in 1990 he was awarded the Nobel Prize along with Joseph Murray. AHSCT is currently used to treat hematological tumors.

In 1997, Fassas et al. were pioneers in this approach in MS; employing AHSCTs for patients with progressive MS.

Throughout the history of AHSCT in MS, the number of pwMS undergoing AHSCT has increased, patient selection criteria has improved, various conditioning procedures have been developed, and, importantly, the associated risks, including mortality, have decreased over time.

Learn more about the benefits here and the risks here. For a comparison between AHSCT vs. DMTs, please click here.

🔹Back to index.

Patient’s Right to Be Informed

Patient’s Right

to Be Informed

pwMS (People with MS) have the essential right to be fully informed about all treatment options, including their risks, benefits, and outcomes. This knowledge empowers them to make informed decisions about their health. Among these options, it is vital that patients are also informed about AHSCT.

The following is Dr. Bertolotto’s personal opinion, based on his experience:

  1. Neurologist MUST explain and illustrate at the pwMS all the treatment options, including AHSCT.
  2. The evaluation and the weight of the risk is personal, everyone weights risk and benefits in a different way. Also the neurologist weight risk and benefit of the pwMS they have in front, but from his personal point of view; his personal evaluation can not replace that of the patient.
  3. Neurologist do not have the right to take a decision instead of the patient, in particular for treatment with risk of death; neurologists can not choose “a priori” the patient who will be offered AHSCT or DMTs. If a patient ask me “doctor, what would you do if you were in my shoes?” I explain my choice, but I add, “if you ask another neurologist it is very likely that you will receive a different answer, as the evaluation of risks and benefits is very personal”.
  4. Legal problems and responsibility: before AHSCT a detailed flow-chart must be followed, with meetings among HCPs and patient (and relatives), written informed consent, answers to all the questions. This procedure takes time, a lot of time, but it is time-saving considering that the management of post-AHSCT is, in the great majority of cases, very easy for many years.
  5. To present AHSCT to pwMS is not only a respectful approach, but also prevents the future accusation of wrong information to pwMS.
  6. We need an up-date of the 2013 working definition of “cure of MS” (Banwell et al., 2013).
  7. Progression of disability: the pwMS must be informed, from the time of communication of the diagnosis, that he/she has a RISK of progression.

Articles Worth Reading: here an insightful article by Prof. Giovannoni (AHSCT vs. Alemtuzumab).

🔹Back to index.

AHSCT Eligible Patient

The profile of the “eligible candidate” for AHSCT

The title of this website, curems.net, can allow the hope of curing all the persons with MS, but I want to clearly state that transplant cannot repair damaged old CNS lesions, and it is riskier in older persons with disability and is more dangerous in patients with more disability. It is not indicated for progressive forms of MS without evidence of active inflammation (i.e. relapses or new MRI lesions).

The following sentence reflects what the majority of experts think about the selection of patients for AHSCT: “For advanced disease stages with a long duration, older age and greater impairment, data argue against a benefit that would justify the risks of transplantation” (Position paper of German neurologists and experts in stem cells transplantation; Bayas et al., 2023).

Medicine is constantly evolving. The criteria for eligibility for transplant are continuously evolving as well. Previously, it was only performed for cases that had failed all available DMTs; then, it was limited to RRMS (relapse-remitting MS) cases with at least two failed DMTs.

Currently some clinical trials enroll pwMS that fail only one DMT, and case reports show very good results as a first line treatment for very aggressive forms of MS (see Das et al., 2021).

Figure from Muraro et al. “Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder — recommendations from ECTRIMS and the EBMT“. Nature Rev Neural (2025).

The numbers—age, disease duration, and Expanded Disability Status Scale (EDSS) score—are provided as illustrative examples to convey the principles and should not be considered strict cut-off values. Consequently, the profile on the far left (in green) represents the optimal candidate for AHSCT.

Clinical radiological characteristics: Which pwMS should consider AHSCT as a possible treatment?

Before undergoing AHSCT, several points need to be taken into consideration. We present an evaluation of various parameters used by a group of German experts.

2023 German criteria: Position paper of German neurologists and experts in stem cells transplantation (Bayas et al., 2023)

The German experts have considered several aspects, including various clinical features, indicated in the table below, and have developed two criteria: core criteria and extended criteria.

According to the authors, the term “Extended criteria” in this context refers to additional factors or conditions beyond the core criteria that are considered when evaluating a patient’s suitability for transplant. While the core criteria provide a basic framework for eligibility, extended criteria may include additional indicators or considerations that can further inform the decision-making process.

These extended criteria may vary depending on specific patient characteristics, disease progression, the neurologists’ experience or other relevant factors.

Essentially, with the term “extended criteria”, the authors broaden the scope of evaluation beyond the essential requirements outlined in the core criteria. The following table outlines this data.

Several parameters must be taken into consideration, in particular

  1. The clinical characteristic of the pwMS: the disease course of MS, age, EDSS, duration of illness, clinical and MRI activity in the last years, speed of clinical progression, therapy failure. (Bayas et al., 2023).
  2. The risk of adverse effects: TRM, the risk to fertility, infections, appearance of new auto-immune diseases, the long-term risks of cancer. Read here more about risks.

Table from Bayas et al. “Autologous haematopoietic stem cell transplantation for multiple sclerosis: a position paper and registry outline“. Therapeutic Advances in Neurological Disorders (2023)

“Active RRMS despite highly effective immunotherapy is the key indication for AHSCT. In younger patients with active SPMS and a conversion from RRMS no longer than within the preceding 3 years without severe impairment (EDSS ⩽ 6.5), the use of AHSCT can be considered.” (Bayas et al., 2023)

“A lower regenerative capacity of the nervous system, ageing of the immune and nervous systems, comorbidities, and the risk of other comorbidities argue against AHSCT in pwMS beyond 50 years. In older age, a short disease duration and high inflammatory activity are a pre-requisite to consider AHSCT.” (Bayas et al., 2023)

“A free walking distance of at least 100 m with assistance (EDSS 6.0) is generally considered the upper limit of mobility impairment for AHSCT treatment, based on the idea that the degeneration in the nervous system should not be too advanced.

In individual cases, AHSCT can be considered beyond EDSS 6.0 if the patient is very young, has a short, highly aggressive course and has a high level of inflammatory activity.

In the same way, a patient who is currently only mildly affected can be treated if the inflammatory activity (many relapses, high MRI activity) and neurological impairment are high during relapses. The dynamics of progression must also be taken into account. Thus, according to the EBMT criteria based on Menon et al., 2013 an EDSS 6.0 after a maximum of 5 years of disease progression or an EDSS 6.0 before age 40 are also accepted as criteria.” (Bayas et al., 2023)

“In general, based on the available studies, MS should not be present for longer than 10 years (first certain manifestation, not time of diagnosis), which also conforms to the idea that degeneration plays an increasing role after a longer disease course. Here, too, differentiation must be made for individual cases. If MS starts in childhood or adolescence, AHSCT can also be considered after 15 years with MS.

In older patients with such a long disease period, AHSCT treatment should only be considered in cases with very high inflammatory activity”. (Bayas et al., 2023)

“In principle, relapse activity should be present in the period before transplantation. Given the heterogeneity of relapses and the uncertainty in diagnosing relapses, ideally, only relapses with EDSS-relevant changes should be evaluated.

However, in highly active disease courses, differentiation between relapse and progression can be difficult. In case of an uncertain relapse classification, other criteria become more important for determining the indication: MRI activity, age and disease duration.

In the criteria according to Menon et al., 2013 an SPMS conversion since less than 3 years is mentioned. Here, however, a distinction from PPMS with superimposed relapses remains blurred.” (Bayas et al., 2023)

“Basically, MRI activity, that is, new contrast enhancement(s) or new/size-progressive T2 lesions in the last year, is a pre-requisite for AHSCT, although there is no international consensus on the necessary number of lesions.

However, highly effective therapy can potentially lead to suppression of MRI activity in the presence of clear clinical progression. Whether this progression is neurodegenerative or an expression of diffuse inflammation not showing up on MRI is unclear.

There is no doubt that contrast enhancement and T2 lesions represent only parts of the MRI inflammatory activity in MS. Therefore, affected persons without MRI activity are also suitable in individual cases, provided they are young, have a short disease duration and show considerable progression dynamics.” (Bayas et al., 2023)

“In principle, treatment failure with one highly potent medication (ocrelizumab, ofatumumab, rituximab, natalizumab, alemtuzumab or a similar active substance) is required before selecting AHSCT, also in light of negotiating cost coverage with health insurance companies” (Germany, Bayas et al., 2023)

In 174 Swedish patients, it was observed that if rituximab was the last DMT before AHSCT, this was associated with a lower risk of inflammatory activity after transplant (Noui et al., 2025).

“In cases of aggressive disease and marked disease progression under an initial medication, AHSCT should already be considered in order not to miss the most favourable time of opportunity for this treatment. In individuals with clearly aggressive disease, AHSCT might be justified even as a first-line treatment on a case-by-case basis.” (Bayas et al., 2023)

Table from Mancardi et al. “Intense immunosuppression followed by autologous haematopoietic stem cell transplantation as a therapeutic strategy in aggressive forms of multiple sclerosis.” Multiple Sclerosis Journal (2018)

🔹Back to index.

Types of MS and AHSCT

The course of MS is classically subdivided in 3 forms: RRMS, SPMS and PPMS.

However the course of MS is so highly variable and influenced by treatments that the scientific literature identifies others subsets of MS: malignant MS, Aggressive MS, Highly active MS, non-responder to DMT, non-responders to high efficacy (HE)-DMT or low efficacy (LE)-DMTs, transitional MS, SPMS with relapses, SPMS without relapses, MS with PIRA (Progression Independent of Relapse Activity), benign MS.

 

The great majority of these subsets do not have a single accepted definition and some of them are overlapping. For example, it is hard to distinguish among “Malignant” “Aggressive” and “Highly Active” MS. To reduce the confusion, each scientific paper specifies its particular definition.

An attempt to define the aggressive forms of MS is presented by Boffa et al., 2025: see figure below.

Differences between ‘highly active MS’, characterized by severe CNS inflammation responsible for heightened disease severity in the short term, and ‘aggressive MS”, encompasses both inflammatory and neurodegenerative components. Clinical, radiological and biological markers predicting the risk of highly active and aggressive MS. Figure by Boffa et al., 2025

Currently, the classification into distinct forms is being questioned because many experts believe that MS is a continuum: the inflammatory component and the degenerative component coexist from the onset of the disease, and the clinical manifestations are the result of various genetic factors, immunological factors and neurologic reserve. (Vollmer T. et al., 2021, Giovannoni et al., 2022, Kuhlmann et al., 2023).

Different Courses of MS

In this section, with respect to subtypes of MS, we summarize some of the guidelines and recommendations of scientific societies as well as position papers of experts. Each document subdivides MS in subsets that can be different or partially overlapping in comparison with other ones.

We report the original subdivision of each paper in order to avoid misinterpretation of the original point of view of the authors.
A consensus on AHSCT in MS by ECTRIMS, EBMT, and lead representatives of ACTRIMS has been published in Nature Reviews Neurology on January 2025:

🟢 The consensus statement endorses AHSCT for RRMS after failure of high-efficacy DMTs and before the onset of irreversible disability.

🟢 AHSCT is recommended for highly aggressive MS with poor prognostic factors within a clinical trial or study.

🟢 AHSCT may have a role in early progressive disease with clear clinical and/or radiological signs of inflammation.

🔴 AHSCT is not recommended for late-stage MS, typically progressive forms.

Read the consensus statement here (Muraro et al., 2025).

🟢 An important consensus endorses AHSCT for selected indications: “In this Consensus Statement, ECTRIMS and the EBMT, as well as lead representatives of ACTRIMS, endorse AHSCT for selected indications. In relapsing–remitting MS, AHSCT should be offered to appropriate candidates, normally after failure of high-efficacy DMT but within the window of opportunity before the development of irreversible disability.” (Muraro et al., 2025)

  • France – French experts and collaborators from MATHEC (Maladies Auto-immunes et Thérapie Cellulaire) list the characteristics for undergoing HSCT in MS patients: failure at least of one highly effective treatment for at the minimum of six months. (Farge et al., 2024)
  • UK –  The
    ABN
    (Rashid et al., 2024) has published updated guidance on the use of DMTs in MS, supporting the appropriate use of AHSCT for treatment-resistant active inflammatory MS, in line with EBMT guidelines.
  • United Arab Emirates – AHSCT is offered as second line for aggressive MS. (Jacob et al., 2024).
  • Germany –  This subset of
    pwMS
    is the critical for a patient to be considered for AHSCT and it has all the “core criteria” for AHSCT according the German recommendations (Bayas et al., 2023).
  • Italy – Guidelines from the Italian Society of Neurology
    SIN
    and the
    ISS
    indicate AHSCT if no response to HE-DMT. For additional information click here and see at page 101.

Source: ISS-Società Italiana di Neurologia SIN (2022)

Australia & New Zealand: AHSCT is used selectively for younger adults (<65) with severe, treatment-resistant RRMS and lower disability (EDSS ≤6.5) who have failed at least two high-efficacy DMTs due to ongoing disease activity. (Shipley et al., 2025)

  • According to the Alberta Bone and Marrow Transplant Clinic (2021), “eligibility for AHSCT includes poorly controlled
    RRMS
    or apparent pseudo-progression in highly select group of patients”. According these guidelines, “pseudo-progression” is referred to as a very active inflammatory disease. The last revision of the MS section dates back to February 6th, 2024.

🟢 A consensus on AHSCT in MS, developed by ECTRIMS, EBMT, and lead representatives of ACTRIMS (Muraro et al., 2025) recommends AHSCT for patients with highly aggressive MS and poor prognostic factors within the context of a clinical trial or study. 

🟢 Recommendation of German experts (Bayas et al., 2023): “In individuals with clearly aggressive disease, AHSCT might be justified even as a first-line treatment on a case-by-case basis.”

The indications for AHSCT as a first-line treatment are rapidly evolving. Other references on this topic:

  • A review on AHSCT as a first line-treatment by Mariottini et al., 2023 (“Should autologous hematopoietic stem cell transplantation be offered as a first-line disease modifying therapy to patients with multiple sclerosis?“). Click here for their review.
  • For a retrospective study by Das et al., 2021 (“Autologous haematopoietic stem cell transplantation as a first-line disease-modifying therapy in patients with “aggressive” multiple sclerosis”), on 20 patients with “aggressive” MS who received AHSCT as a first-line treatment. Click here.

The Star-MS trial has enrolled patients aged between 16 and 55 with highly active RRMS. This trial has enrolled 90 patients in the study from 19 sites across the UK. Click here for more details.

  • “Extended criteria” according to German recommendations (Bayas et al., 2023). See here the table for more information about the extended criteria.
  • The following clinical trials are recruiting or have recruited patients with RRMS non responder to
    LE-DMTs
    . For more details: BEAT-MS, NET-MS, RAM-MS and STAR-MS.

📌 Click here for a table comparing RCTs. Click here for a detailed description of clinical trials in the “Prospective” section. 

🟢 The EBMT guidelines consider AHSCT as a “clinical option” (Sharrack et al., 2020) with a grade II level of evidence. For additional details about the levels of evidence, click here.

🟢 The ASBMT (Cohen et al., 2019) considers AHSCT as the “standard of care” for SPMS with high disease activity, specifically for patients who are at significant risk for progression. The ASBMT Task Force states that “The ASBMT Task Force recommends revising the recommended indication for AHCT in MS to “standard of care, clinical evidence available”, for patients with relapsing forms of MS (RRMS or progressive MS with superimposed activity) who have prognostic factors that indicate a high risk of future disability, including ongoing clinical relapse or MRI lesion activity despite treatment with available DMTs, especially if disease activity continues despite treatment with high-efficacy DMTs and/or worsening disability.”

Presented below are additional significant perspectives on this topic from various MS societies worldwide:

🟢 According to German experts (Bayas et al., 2023), a patient is suitable for AHSCT, if affected by SPMS with progression for ≤ 2 years (core criteria) or if affected by SPMS with progression ≤ 5 years (extended criteria).

In the “DMT guidelines for MS in the United Arab Emirates” (Jacob et al., 2024), AHSCT is offered as second line for aggressive MS: “Treatment naïve SPMS patients are typically begun on Siponimod or anti CD20 agents (though any drug used in RRMS can be used as per FDA). Breakthrough disease activity in the form of new MRI lesions and relapses warrants escalation to anti CD20 agents, cladribine, natalizumab, alemtuzumab, AHSCT or mitoxantrone”.

Other references on this topic:

  • A study of pwMS with active secondary progressive by Boffa et al., 2022 (Hematopoietic Stem Cell Transplantation in People With Active Secondary Progressive Multiple Sclerosis). Click here.

🟢 “Generally not recommended”: according to EBMT guidelines (Sharrack et al., 2020).

🟢 Recommendation of German experts (Bayas et al., 2023): “Most immunotherapies do not work convincingly in PPMS; hence AHSCT should be considered with great caution.”.

They have suggested to evaluate AHSCT in selected cases of PPMS, especially if patients show inflammatory activity on MRI or relapses. “Without MRI activity, PPMS could only be considered in cases with an aggressive course, that is, EDSS 6.0 after 5 years or EDSS 6.0 before age 40 and with enhanced consideration of the benefit versus risk balance”.

🟢 According to the review by the EBMT Autoimmune Diseases (ADWP) and Paediatric Diseases (PDWP) Working Parties (2022), “Autologous and allogeneic HSCT have been used successfully to induce disease control and often apparent cure of severe treatment-refractory autoimmune diseases (ADs) in children.” (Achini-Gutzwiller et al., 2022)

In this review, the authors state that HSCT, particularly AHSCT, is considered a therapeutic option for pediatric MS cases  that are refractory to conventional treatments such as interferon beta (IFN β), glatiramer acetate (GA), natalizumab, or rituximab. However, a careful assessment of the risks and benefits in a multidisciplinary setting is essential. (Achini-Gutzwiller et al., 2022)

🟢 A detailed description of pediatric MS has been developed in Chapter 15 of the Hematopoietic Stem Cell Transplantation for Neurologic Diseases, Handbook of Clinical Neurology by Elsevier (2024). 

Other references on this topic:

  • Read here an important study on 7 pediatric patients with AIDs treated with AHSCT, with a median follow-up of 17 years. “All patients are still alive and 4/7 are in clinical remission on medication, 3/7 in clinical remission off medication” (Satirer et al., 2024).
  • A study on 21 young patients (aged 9-18) treated with AHSCT (Burman et al., 2017) reported encouraging outcomes, with 100% overall survival and progression-free survival (PFS) at three years after AHSCT.

🔹Back to index.

Other Neurological AIDs

The following is a list of autoimmune diseases of both central and peripheral nervous system, on which publications have been made since 2024 to date. It is beyond the scope of this website to detail other diseases but MS.

 

 

Other Neurological Autoimmune Diseases Paper Title, Authors and Journal 
Anti-Caspr1 antibody nodopathy 

“Successful autologous hematopoietic stem cell transplantation in a refractory anti-Caspr1 antibody nodopathy”. Afanasiev et al., 2023. Journal of the Peripheral Nervous System
Autoimmune retinopathy

“Hematopoietic stem cell transplantation as rescue therapy for refractory autoimmune retinopathy: a case report“. Wong et al., 2025. Frontiers in Immunology

Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)

“Hematopoietic stem cell transplantation (HSCT) for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP): Is it CIDP?“. Burt et al., 2024. Hematopoietic Stem Cell Transplantation for Neurologic Diseases (Chap. 13). Handbook of Clinical Neurology. Elsevier.
MOGAD

“An aggressive form of MOGAD treated with AHSCT: A case report”. Sbragia et al., 2023. Multiple Sclerosis Journal
Myasthenia Gravis

“An updated review on the utility of hematopoietic stem cell transplant in the treatment of refractory myasthenia gravis“. Li et al., 2025. RRNF Neuromuscular Journal

“HSCT for stiff person syndrome and myasthenia gravis“. Boccia et al., 2024. Hematopoietic Stem Cell Transplantation for Neurologic Diseases (Chap. 14). Handbook of Clinical Neurology. Elsevier.
Neuromyelitis Optica Spectrum Disorder

“Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder — recommendations from ECTRIMS and the EBMT“. Muraro et al., 2025. Nature Rew Neurol

“Hematopoietic stem cell transplantation for neuromyelitis optica spectrum disorder. Can immune tolerance be reestablished?“. Burt et al., 2024. Hematopoietic Stem Cell Transplantation for Neurologic Diseases (Chap. 12). Handbook of Clinical Neurology. Elsevier.

“Late Relapse After Prolonged Remission Post-autologous Hematopoietic Stem Cell Transplantation in Two Patients with AQP4-IgG+ Neuromyelitis Optica Spectrum Disorder”. Vorasoot et al., 2025. JAMA Netw Open
Paraneoplastic Cerebellar Degeneration

 “Autologous Hematopoietic Stem Cell Transplantation for Paraneoplastic Cerebellar Degeneration“. Guerra et al., 2025. Neurol Neuroimmunol Neuroinflamm. 

Sporadic late-onset nemaline myopathy (SLONM) 

 “Sporadic late-onset nemaline myopathy with monoclonal gammapathy of unknown significance treated with melphalan and autologous hematopoietic stem cell transplantation“. Kierdaszuk et al., 2025. Neuromuscular Disorders. 
Stiff Person Spectrum Disorders

“HSCT for stiff person syndrome and myasthenia gravis“. Boccia et al., 2024. Hematopoietic Stem Cell Transplantation for Neurologic Diseases (Chap. 14). Handbook of Clinical Neurology. Elsevier.

“Autologous hematopoietic stem cell transplantation in a patient with multi-refractory stiff person syndrome“. Alsuliman et al., 2024. Bone Marrow Trans

“Successful Autologous Hematopoietic Stem Cell Transplant in Glycine Receptor Antibody-Positive Stiff Person Syndrome”. Celli et al., 2024. Neurol Neuroimmunol Neuroinflamm.

“Clinical Outcome of Autologous Hematopoietic Stem Cell Transplantation in Stiff Person Syndrome: A Literature Review”. Supantini et al., 2026. JMM
Systemic Autoimmune Diseases with Neurologic Involvement (*) “HSCT for systemic autoimmune diseases with neurologic involvement“. Alexander et al., 2024. Hematopoietic Stem Cell Transplantation for Neurologic Diseases (Chap. 16). Handbook of Clinical Neurology Elsevier.

Anti-Caspr1, anti-contactin-associated protein 1. MOGAD, myelin oligodendrocyte glycoprotein antibody-associated disease. AQP4-IgG+, aquaporin-4 (AQP4) antibodies.

(*) Systemic lupus erythematosus, Antiphospholipid Syndrome, Sjogren’s Syndrome, Sarcoidosis, Behçet’s disease, Granulomatosis with polyangiitis, IgG4-related disease, Giant cell arteritis.

The table lists various neurological autoimmune diseases where AHSCT has been performed, offering an overview of treated cases.

For further insights on transplant in other neurological autoimmune disorders:

  • Download the EBMT Handbook for free here, authored by over 200 experts and renowned authorities in the fields of HSCT and Cellular Therapies (2024).

🔹Back to index.