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Autologous hematopoietic stem cell transplantation in severe, refractory stiff person syndrome: a case series

Bone Marrow Transplantation volume 60, pages 1387–1389 (2025)Cite this article

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To the Editor:

Stiff Person Syndrome (SPS) is a rare autoimmune neurological disorder characterized by progressive muscle stiffness, painful spasms, and impaired mobility, often triggered by stimuli like noise or stress [1]. With a prevalence of 1–2 per million and a female predominance, SPS is primarily driven by autoantibodies against glutamic acid decarboxylase-65 (GAD-65), disrupting γ-aminobutyric acid neurotransmission [2, 3]. Classic SPS, comprising 70–80% of cases, features truncal and proximal limb spasticity due to continuous muscle co-contraction. Neuroradiologic studies generally do not reveal pathognomonic abnormalities. Electromyography (EMG) can support the diagnosis with continuous motor unit activity with abnormal co-contraction of opposing muscle groups; however, it is often normal or unremarkable [4, 5]. Standard treatments, including benzodiazepines (e.g., diazepam), baclofen, and immunomodulatory therapies (e.g., intravenous immunoglobulin [IVIG], rituximab), often provide only transient relief in refractory cases. According to the European Society for Blood and Marrow Transplant (EBMT) guidelines, autologous hematopoietic stem cell transplantation (auto-HSCT) can be considered a clinical option for severe, treatment-resistant SPS [6,7,8]. We report two cases of refractory SPS treated with auto-HSCT at the University of Arizona Cancer Center, highlighting clinical outcomes and complications.

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Authors and Affiliations

  1. University of Arizona College of Medicine, Tucson, AZ, USA

    Jesse Ritter, Tom Marco, Monica Angeletti, Daniel Barnett & Muhammad Husnain

  2. University of Arizona Cancer Center, Tucson, AZ, USA

    Daniel Barnett & Muhammad Husnain

  3. University of Arizona Department of Neurology, Tucson, AZ, USA

    Holli Horak

Authors
  1. Jesse Ritter
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  2. Tom Marco
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  3. Monica Angeletti
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  6. Muhammad Husnain
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Corresponding author

Correspondence to Jesse Ritter.

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Competing interests

The authors declare no competing interests.

Ethics approval and consent to participate

This case series does not meet the definition of human subjects research as defined by 45 CFR 46.102 and was therefore not subject to IRB oversight. All methods were performed in accordance with relevant institutional guidelines and regulations.

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Written informed consent was obtained from all participants for publication of de-identified clinical data presented in this report. No identifiable images are included in the manuscript. JR conceived and designed the study, collected data, analyzed and interpreted results, wrote the manuscript, and finalized the edits. TM collected data and contributed to writing the manuscript. MA contributed to writing the manuscript. DB analyzed and interpreted data and contributed to final manuscript editing and approval. HH reviewed and approved the final manuscript. MH conceived and designed the study, analyzed and interpreted data, supervised the project, and approved the final manuscript.

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Ritter, J., Marco, T., Angeletti, M. et al. Autologous hematopoietic stem cell transplantation in severe, refractory stiff person syndrome: a case series. Bone Marrow Transplant 60, 1387–1389 (2025). https://doi.org/10.1038/s41409-025-02686-z

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