Autologous Hematopoietic Stem Cell Transplantation: A Promising Therapeutic Avenue for Refractory Stiff Person Syndrome
Jourdan Carroll1, Paul Elsbernd1
1Neurology, Brooke Army Medical Center
Objective:
To describe a case of refractory stiff person syndrome (SPS) and co-morbid autoimmune vitiligo successfully treated with autologous hematopoietic stem cell transplant (aHSCT).
Background:

A single patient with a 10 year history of refractory SPS and advanced vitiligo (>90% total body surface area) was treated via non-myeloablative aHSCT at a military medical training facility. The patient required frequent increases of centrally acting medications to control his symptoms of progressive muscle cramping, spasticity, and stiffness. Several disease modifying therapies for both conditions were trialed with little to no long-term improvement including prednisone, IVIG, plasma exchange, rituximab, and tofacitinib. The patient also experienced worsening side effects of his symptomatic medications due to escalating doses which impacted his ability to work and participate in daily tasks, ultimately leading to consideration of aHSCT. Response to the transplant was assessed via the Stiff Person Syndrome Validated Assessment Scale at follow up appointments as well as ability to decrease dosages of the patient’s symptomatic treatment without increase in SPS symptoms. Post-aHSCT, the patient’s serum GAD65 antibody (Ab) titers were re-tested at regular intervals and compared to pre-transplant levels.

Design/Methods:
NA
Results:
Patient successfully completed aHSCT only complicated by grade 1 mucositis. Following aHSCT, the patient’s symptoms both subjectively and objectively improved. There was an 80% reduction in Heightened Sensitivity Scale score, a 15% improvement in Quality of Life Index score, and no worsening in Distribution of Stiffness score. His daily benzodiazepine dose was reduced by nearly 50% without increase in symptoms. The patient’s GAD65 Ab titer level decreased following the transplant from 1,600 U/mL to 333 U/mL. Finally, there was drastic clinical improvement of the patient’s secondary autoimmune condition, vitiligo (>90% to estimated 40% post-aHSCT).
Conclusions:

aHSCT represents a safe and effective potential treatment for refractory SPS with long-term improvements in symptoms and medication requirements.

10.1212/WNL.0000000000213220
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