Lewis Kass-Iliyya1,2, John A Snowden3,4, Alice Thorpe3, Helen Jessop3, Andrew D Chantry3,4, Ptolemaios G Sarrigiannis5, Marios Hadjivassiliou5,6, Basil Sharrack5,6. 1. Academic Department of Neurosciences, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. lewis.kass-iliyya@nhs.net. 2. The Academic Unit of Medical Education, University of Sheffield, Sheffield, UK. lewis.kass-iliyya@nhs.net. 3. Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. 4. Department of Oncology & Metabolism, University of Sheffield, Sheffield, UK. 5. Academic Department of Neurosciences, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK. 6. University of Sheffield, Sheffield, UK.
Abstract
Stiff Person Syndrome (SPS) is a rare immune-mediated disabling neurological disorder characterised by muscle spasms and high GAD antibodies. There are only a few case reports of autologous haematopoietic stem cell transplantation (auto-HSCT) as a treatment for SPS. OBJECTIVE: To describe the UK experience of treating refractory SPS with auto-HSCT. METHODS: Between 2015 and 2019, 10 patients with SPS were referred to our institution for consideration of auto-HSCT. Eight patients were deemed suitable for autograft and four were treated. Of the treated patients, three had classical SPS and one had the progressive encephalomyelitis with rigidity and myoclonus variant. All patients were significantly disabled and had failed conventional immunosuppressive therapy. Patients were mobilised with Cyclophosphamide (Cy) 2 g/m2 + G-CSF and conditioned with Cy 200 mg/kg + ATG followed by auto-HSCT. RESULTS: Despite their significantly reduced performance status, all patients tolerated the procedure with no unexpected toxicities. Following autograft, all patients improved symptomatically and stopped all forms of immunosuppressive therapies. Two patients were able to ambulate independently from being wheelchair dependent. One patient's walking distance improved from 300 meters to 5 miles and one patient's ambulation improved from being confined to a wheelchair to be able to walk with a frame. Two patients became seronegative for anti-GAD antibodies and normalised their neurophysiological abnormalities. CONCLUSIONS: Auto-HSCT is an intensive but well tolerated and effective treatment option for patients with SPS refractory to conventional immunotherapy. Further work is warranted to optimise patient selection and establish the efficacy, long-term safety, and cost-effectiveness of this treatment.
Stiff Person Syndrome (SPS) is a rare immune-mediated disabling neurological disorder characterised by muscle spasms and high GAD antibodies. There are only a few case reports of autologous haematopoietic stem cell transplantation (auto-HSCT) as a treatment for SPS. OBJECTIVE: To describe the UK experience of treating refractory SPS with auto-HSCT. METHODS: Between 2015 and 2019, 10 patients with SPS were referred to our institution for consideration of auto-HSCT. Eight patients were deemed suitable for autograft and four were treated. Of the treated patients, three had classical SPS and one had the progressive encephalomyelitis with rigidity and myoclonus variant. All patients were significantly disabled and had failed conventional immunosuppressive therapy. Patients were mobilised with Cyclophosphamide (Cy) 2 g/m2 + G-CSF and conditioned with Cy 200 mg/kg + ATG followed by auto-HSCT. RESULTS: Despite their significantly reduced performance status, all patients tolerated the procedure with no unexpected toxicities. Following autograft, all patients improved symptomatically and stopped all forms of immunosuppressive therapies. Two patients were able to ambulate independently from being wheelchair dependent. One patient's walking distance improved from 300 meters to 5 miles and one patient's ambulation improved from being confined to a wheelchair to be able to walk with a frame. Two patients became seronegative for anti-GAD antibodies and normalised their neurophysiological abnormalities. CONCLUSIONS: Auto-HSCT is an intensive but well tolerated and effective treatment option for patients with SPS refractory to conventional immunotherapy. Further work is warranted to optimise patient selection and establish the efficacy, long-term safety, and cost-effectiveness of this treatment.
Entities:
Keywords:
Stem cell transplantation; Stiff person syndrome
Authors: John A Snowden; Isabel Sánchez-Ortega; Selim Corbacioglu; Grzegorz W Basak; Christian Chabannon; Rafael de la Camara; Harry Dolstra; Rafael F Duarte; Bertram Glass; Raffaella Greco; Arjan C Lankester; Mohamad Mohty; Bénédicte Neven; Régis Peffault de Latour; Paolo Pedrazzoli; Zinaida Peric; Ibrahim Yakoub-Agha; Anna Sureda; Nicolaus Kröger Journal: Bone Marrow Transplant Date: 2022-05-19 Impact factor: 5.174
Authors: Marios Hadjivassiliou; Panagiotis Zis; David S Sanders; Nigel Hoggard; Ptolemaios G Sarrigiannis Journal: Nutrients Date: 2021-04-20 Impact factor: 5.717