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Clinical Outcome of Autologous Hematopoietic Stem Cell Transplantation in Stiff Person Syndrome: A Literature Review
Abstract
Stiff person syndrome (SPS) is a rare immune-mediated neurological disorder characterized by progressive muscle stiffness and painful spasms that significantly impair functional ability. Although symptomatic and immunomodulatory therapies are commonly used, some patients remain refractory to standard treatments. Autologous hematopoietic stem cell transplantation (aHSCT) has been proposed as a potential therapeutic option for severe or treatment-resistant cases. This literature review aims to evaluate the clinical outcomes and safety of aHSCT in patients with SPS. A narrative literature review was conducted using published studies reporting the use of aHSCT in SPS, including clinical trials, cohort, case series, and case reports. Relevant articles were identified through PubMed and Google Scholar, limited to studies published within the last ten years. A total of 134 articles were initially identified, followed by a selection process, and five eligible articles were included in this review. The reviewed studies demonstrated that aHSCT may lead to clinical improvement in selected patients with refractory SPS. Autologous HSCT reduced muscle stiffness and improved functional status. Treatment responses varied, and some non-responders were reported; however, overall clinical outcomes were favorable with acceptable safety profiles. In conclusion, aHSCT may be considered a potential therapeutic option for severe or treatment-resistant SPS, although further prospective studies are needed to confirm its efficacy and safety.
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Alexandra Muranova, A., & Shanina Affiliations, E. (n.d.). Stiff Person Syndrome Continuing Education Activity. Retrieved https://www.ncbi.nlm.nih.gov/books/NBK573078/?report=printable
Alsuliman, T., Psimaras, D., Stocker, N., Sestili, S., Banet, A., Van de Wyngaert, Z., Bonnin, A., Badoglio, M., Puyade, M., Farge, D., Mohty, M., & Marjanovic, Z. (2025). Autologous hematopoietic stem cell transplantation in a patient with multi-refractory stiff person syndrome. Bone Marrow Transplantation, 60(1), 86–88. https://doi.org/10.1038/s41409-024-02440-x
Boccia, V. D., Boffa, G., & Inglese, M. (2024). HSCT for stiff person syndrome and myasthenia gravis (pp. 239–247). https://doi.org/10.1016/B978-0-323-90242-7.00020-1
Bose, S., & Jacob, S. (2025). Stiff-person syndrome. Practical Neurology, 25(1), 6–17. https://doi.org/10.1136/pn-2023-003974
Burman, J., Tolf, A., Hägglund, H., & Askmark, H. (2018). Autologous haematopoietic stem cell transplantation for neurological diseases. Journal of Neurology, Neurosurgery & Psychiatry, 89(2), 147–155. https://doi.org/10.1136/jnnp-2017-316271
Burt, R. K., Balabanov, R., Han, X., Quigley, K., Arnautovic, I., Helenowski, I., Rose, J., & Siddique, T. (2021). Autologous Hematopoietic Stem Cell Transplantation for Stiff-Person Spectrum Disorder. Neurology, 96(6).
https://doi.org/10.1212/WNL.0000000000011338
Celli, S. I., Nash, R., Money, K. M., Garza, M., Borko, T. L., Mizenko, C., McMenamin, C., Von Geldern, G., Georges, G., & Piquet, A. L. (2024). Successful Autologous Hematopoietic Stem Cell Transplant in Glycine Receptor Antibody-Positive Stiff Person Syndrome. Neurology Neuroimmunology & Neuroinflammation, 11(2). https://doi.org/10.1212/NXI.0000000000200197
Dalakas, M. C. (2021). Limited Benefits Halt Enrollment in Hematopoietic Stem Cell Transplantation Trial for Stiff-Person Syndrome. Neurology, 96(6), 239–240. https://doi.org/10.1212/WNL.0000000000011349
Dalakas, M. C. (2022). Stiff-person Syndrome and GAD Antibody-spectrum Disorders: GABAergic Neuronal Excitability, Immunopathogenesis and Update on Antibody Therapies. Neurotherapeutics, 19(3), 832–847. https://doi.org/10.1007/s13311-
-01188-w
Dalakas, M. C. (2024). Stiff-person syndrome and related disorders — diagnosis, mechanisms and therapies. Nature Reviews Neurology, 20(10), 587–601. https://doi.org/10.1038/s41582-024-01012-3
Kass-Iliyya, L., Snowden, J. A., Thorpe, A., Jessop, H., Chantry, A. D., Sarrigiannis, P. G., Hadjivassiliou, M., & Sharrack, B. (2021). Autologous haematopoietic stem cell transplantation for refractory stiff-person syndrome: the UK experience. Journal of Neurology, 268(1), 265–275. https://doi.org/10.1007/s00415-020-10054-8
Lenglet, T., Honnorat, J., & Attarian, S. (2025). Systematic Review of Immune and Symptomatic Treatments for Stiff‐Person Syndrome. European Journal of Neurology, 32(11). https://doi.org/10.1111/ene.70435
Mariottini, A., Bulgarini, G., Cornacchini, S., Damato, V., Saccardi, R., & Massacesi, L. (2023). Hematopoietic Stem Cell Transplantation for the Treatment of Autoimmune Neurological Diseases: An Update. Bioengineering, 10(2), 176.
https://doi.org/10.3390/bioengineering10020176
Perera, T., Tchajkov, I., & Storek, J. (2024). Antibody-Negative Stiff Person Syndrome Non-Responder After Hematopoietic Cell Transplant. Canadian Journal of Neurological Sciences / Journal Canadien Des Sciences Neurologiques, 51(5), 712–713. https://doi.org/10.1017/cjn.2023.296
Rajmohan, R., Baveja, S., Nguyen, D., Shah, E., Sy, M., Attaripour, S., & Swope, D. (2023). Case report: Approaches to treatment-refractory and super-refractory glutamic acid decarboxylase antibody-spectrum disorders. Frontiers in Immunology, 14. https://doi.org/10.3389/fimmu.2023.1297340
Ritter, J., Marco, T., Angeletti, M., Barnett, D., Horak, H., & Husnain, M. (2025). Autologous hematopoietic stem cell transplantation in severe, refractory stiff person syndrome: a case series. Bone Marrow Transplantation, 60(10), 1387–1389. https://doi.org/10.1038/s41409-025-02686-z
Sanders, S., Bredeson, C., Pringle, C. E., Martin, L., Allan, D., Bence-Bruckler, I., Hamelin, L., Hopkins, H. S., Sabloff, M., Sheppard, D., Tay, J., Huebsch, L., & Atkins, H. L. (2014). Autologous Stem Cell Transplantation for Stiff Person Syndrome. JAMA Neurology, 71(10), 1296. https://doi.org/10.1001/jamaneurol.2014.1297
Sharrack, B., Saccardi, R., Alexander, T., Badoglio, M., Burman, J., Farge, D., Greco, R., Jessop, H., Kazmi, M., Kirgizov, K., Labopin, M., Mancardi, G., Martin, R., Moore, J., Muraro, P. A., Rovira, M., Sormani, M. P., Snowden, J. A., Snowden, J., … Zaccara, E. (2020). Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and recommendations from the EBMT Autoimmune Diseases Working Party (ADWP) and the Joint Accreditation Committee of EBMT and ISCT (JACIE). Bone Marrow Transplantation, 55(2), 283–306. https://doi.org/10.1038/s41409-019-0684-0
Vlad, B., Wang, Y., Newsome, S. D., & Balint, B. (2023). Stiff Person Spectrum Disorders—An Update and Outlook on Clinical, Pathophysiological and Treatment Perspectives. Biomedicines, 11(9), 2500. https://doi.org/10.3390/biomedicines11092500
Yahyaoui, A. E., Sayhi, S., Guediche, N. E., Saied, Z., Arfaoui, B., Ajili, F., Bedoui, I., Sassi, S. Ben, & Abdelhafidh, N. Ben. (2024). A rare presentation of stiff-person syndrome with a nonspecific focal myositis: A case report. Romanian Journal of Rheumatology, 33(3), 181–186. https://doi.org/10.37897/RJR.2024.3.8
Yasiry, Z., Burt, R. K., & Sharrack, B. (2021). Autologous Hematopoietic Stem Cell Transplantation in Stiff Person Spectrum Disorder. In Hematopoietic Stem Cell Transplantation and Cellular Therapies for Autoimmune Diseases (pp. 580–588). CRC Press. https://doi.org/10.1201/9781315151366-61
DOI: https://doi.org/10.33024/jmm.v10i1.25044
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