Autologous haematopoietic stem cell transplantation for refractory stiff-person syndrome: the UK experience

go back to reference Meinck HM, Thompson PD (2002) Stiff man syndrome and related conditions. Mov Disord 17:853–866CrossRef Meinck HM, Thompson PD (2002) Stiff man syndrome and related conditions. Mov Disord 17:853–866CrossRef

go back to reference Rakocevic G, Alexopoulos H, Dalakas MC (2019) Quantitative clinical and autoimmune assessments in stiff person syndrome: evidence for a progressive disorder. BMC Neurol 19:1CrossRef Rakocevic G, Alexopoulos H, Dalakas MC (2019) Quantitative clinical and autoimmune assessments in stiff person syndrome: evidence for a progressive disorder. BMC Neurol 19:1CrossRef

go back to reference Dinkel K, Meinck HM, Jury KM, Karges W, Richter W (1998) Inhibition of gamma-aminobutyric acid synthesis by glutamic acid decarboxylase autoantibodies in stiff-man syndrome. Ann Neurol 44:194–201CrossRef Dinkel K, Meinck HM, Jury KM, Karges W, Richter W (1998) Inhibition of gamma-aminobutyric acid synthesis by glutamic acid decarboxylase autoantibodies in stiff-man syndrome. Ann Neurol 44:194–201CrossRef

go back to reference Baizabal-Carvallo JF, Jankovic J (2015) Stiff-person syndrome: insights into a complex autoimmune disorder. J Neurol Neurosurg Psychiatry 86:840–848CrossRef Baizabal-Carvallo JF, Jankovic J (2015) Stiff-person syndrome: insights into a complex autoimmune disorder. J Neurol Neurosurg Psychiatry 86:840–848CrossRef

go back to reference Carvajal-Gonzalez A, Leite MI, Waters P, Woodhall M, Coutinho E, Balint B, Lang B, Pettingill P, Carr A, Sheerin UM, Press R, Lunn MP, Lim M, Maddison P, Meinck HM, Vandenberghe W, Vincent A (2014) Glycine receptor antibodies in PERM and related syndromes: characteristics, clinical features and outcomes. Brain 137:2178–2192CrossRef Carvajal-Gonzalez A, Leite MI, Waters P, Woodhall M, Coutinho E, Balint B, Lang B, Pettingill P, Carr A, Sheerin UM, Press R, Lunn MP, Lim M, Maddison P, Meinck HM, Vandenberghe W, Vincent A (2014) Glycine receptor antibodies in PERM and related syndromes: characteristics, clinical features and outcomes. Brain 137:2178–2192CrossRef

go back to reference Hinson SR, Lopez-Chiriboga AS, Bower JH, Matsumoto JY, Hassan A, Basal E, Lennon VA, Pittock SJ, McKeon A (2018) Glycine receptor modulating antibody predicting treatable stiff-person spectrum disorders. Neurol Neuroimmunol Neuroinflamm 5:e438CrossRef Hinson SR, Lopez-Chiriboga AS, Bower JH, Matsumoto JY, Hassan A, Basal E, Lennon VA, Pittock SJ, McKeon A (2018) Glycine receptor modulating antibody predicting treatable stiff-person spectrum disorders. Neurol Neuroimmunol Neuroinflamm 5:e438CrossRef

go back to reference McKeon A, Robinson MT, McEvoy KM, Matsumoto JY, Lennon VA, Ahlskog JE, Pittock SJ (2012) Stiff-man syndrome and variants: clinical course, treatments, and outcomes. Arch Neurol 69:230–238CrossRef McKeon A, Robinson MT, McEvoy KM, Matsumoto JY, Lennon VA, Ahlskog JE, Pittock SJ (2012) Stiff-man syndrome and variants: clinical course, treatments, and outcomes. Arch Neurol 69:230–238CrossRef

go back to reference Sanders S, Bredeson C, Pringle CE, Martin L, Allan D, Bence-Bruckler I, Hamelin L, Hopkins HS, Sabloff M, Sheppard D, Tay J, Huebsch L, Atkins HL (2014) Autologous stem cell transplantation for stiff person syndrome: two cases from the Ottawa blood and marrow transplant program. JAMA Neurol 71:1296–1299CrossRef Sanders S, Bredeson C, Pringle CE, Martin L, Allan D, Bence-Bruckler I, Hamelin L, Hopkins HS, Sabloff M, Sheppard D, Tay J, Huebsch L, Atkins HL (2014) Autologous stem cell transplantation for stiff person syndrome: two cases from the Ottawa blood and marrow transplant program. JAMA Neurol 71:1296–1299CrossRef

go back to reference Hadjivassiliou M, Aeschlimann D, Grunewald RA, Sanders DS, Sharrack B, Woodroofe N (2011) GAD antibody-associated neurological illness and its relationship to gluten sensitivity. Acta Neurol Scand 123:175–180CrossRef Hadjivassiliou M, Aeschlimann D, Grunewald RA, Sanders DS, Sharrack B, Woodroofe N (2011) GAD antibody-associated neurological illness and its relationship to gluten sensitivity. Acta Neurol Scand 123:175–180CrossRef

go back to reference 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 PA, Rovira M, Sormani MP, Snowden JA, Snowden J, Saccardi R, McGrath E, Bambi F, Sanchez-Guijo F, Worel N, Snowden J, Alexander T, Badolglio M, Abinun M, Arnold R, Brierley C, Burman J, Castilla-Llorente C, Cooper N, Daikeler T, del Papa N, Farge D, Finke J, Greco R, Hagglund H, Henes J, Hiepe F, Jessop H, Kiely D, Labopin M, Kazmi M, Kirgizov K, Mancardi G, Marjanovic Z, Martin R, Martin T, Ma D, Moore J, Miller P, Muraro P, Oliveira M-C, Polushin A, Onida F, Simoes B, Puyade M, Resnick I, Rovira M, Saccardi R, Saif M, Sakellari I, Sharrack B, Snarski E, Scherer HU, Sossa C, de Vries-Bouwstra J, Wulffraat N, Zaccara E, for the European Society for B, Marrow Transplantation Autoimmune Diseases Working P, the Joint Accreditation Committee of the International Society for Cellular T, Ebmt (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 55:283–306CrossRef 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 PA, Rovira M, Sormani MP, Snowden JA, Snowden J, Saccardi R, McGrath E, Bambi F, Sanchez-Guijo F, Worel N, Snowden J, Alexander T, Badolglio M, Abinun M, Arnold R, Brierley C, Burman J, Castilla-Llorente C, Cooper N, Daikeler T, del Papa N, Farge D, Finke J, Greco R, Hagglund H, Henes J, Hiepe F, Jessop H, Kiely D, Labopin M, Kazmi M, Kirgizov K, Mancardi G, Marjanovic Z, Martin R, Martin T, Ma D, Moore J, Miller P, Muraro P, Oliveira M-C, Polushin A, Onida F, Simoes B, Puyade M, Resnick I, Rovira M, Saccardi R, Saif M, Sakellari I, Sharrack B, Snarski E, Scherer HU, Sossa C, de Vries-Bouwstra J, Wulffraat N, Zaccara E, for the European Society for B, Marrow Transplantation Autoimmune Diseases Working P, the Joint Accreditation Committee of the International Society for Cellular T, Ebmt (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 55:283–306CrossRef

go back to reference ClinicalTrials.gov NCT02282514 (2019, December 13) Stem cell transplantation for stiff person syndrome. https://clinicaltrials.gov/ct2/show/NCT02282514 ClinicalTrials.gov NCT02282514 (2019, December 13) Stem cell transplantation for stiff person syndrome. https://​clinicaltrials.​gov/​ct2/​show/​NCT02282514

go back to reference Levy LM, Dalakas MC, Floeter MK (1999) The stiff-person syndrome: an autoimmune disorder affecting neurotransmission of gamma-aminobutyric acid. Ann Intern Med 131:522–530CrossRef Levy LM, Dalakas MC, Floeter MK (1999) The stiff-person syndrome: an autoimmune disorder affecting neurotransmission of gamma-aminobutyric acid. Ann Intern Med 131:522–530CrossRef

go back to reference Rakocevic G, Raju R, Dalakas MC (2004) Anti-glutamic acid decarboxylase antibodies in the serum and cerebrospinal fluid of patients with stiff-person syndrome: correlation with clinical severity. Arch Neurol 61:902–904CrossRef Rakocevic G, Raju R, Dalakas MC (2004) Anti-glutamic acid decarboxylase antibodies in the serum and cerebrospinal fluid of patients with stiff-person syndrome: correlation with clinical severity. Arch Neurol 61:902–904CrossRef

go back to reference Barker RA, Revesz T, Thom M, Marsden CD, Brown P (1998) Review of 23 patients affected by the stiff man syndrome: clinical subdivision into stiff trunk (man) syndrome, stiff limb syndrome, and progressive encephalomyelitis with rigidity. J Neurol Neurosurg Psychiatry 65:633–640CrossRef Barker RA, Revesz T, Thom M, Marsden CD, Brown P (1998) Review of 23 patients affected by the stiff man syndrome: clinical subdivision into stiff trunk (man) syndrome, stiff limb syndrome, and progressive encephalomyelitis with rigidity. J Neurol Neurosurg Psychiatry 65:633–640CrossRef

go back to reference Lynch JW (2009) Native glycine receptor subtypes and their physiological roles. Neuropharmacology 56:303–309CrossRef Lynch JW (2009) Native glycine receptor subtypes and their physiological roles. Neuropharmacology 56:303–309CrossRef

go back to reference Crisp SJ, Balint B, Vincent A (2017) Redefining progressive encephalomyelitis with rigidity and myoclonus after the discovery of antibodies to glycine receptors. Curr Opin Neurol 30:310–316CrossRef Crisp SJ, Balint B, Vincent A (2017) Redefining progressive encephalomyelitis with rigidity and myoclonus after the discovery of antibodies to glycine receptors. Curr Opin Neurol 30:310–316CrossRef

go back to reference Alexopoulos H, Akrivou S, Dalakas MC (2013) Glycine receptor antibodies in stiff-person syndrome and other GAD-positive CNS disorders. Neurology 81:1962–1964CrossRef Alexopoulos H, Akrivou S, Dalakas MC (2013) Glycine receptor antibodies in stiff-person syndrome and other GAD-positive CNS disorders. Neurology 81:1962–1964CrossRef

go back to reference Skorstad G, Hestvik AL, Vartdal F, Holmoy T (2009) Cerebrospinal fluid T cell responses against glutamic acid decarboxylase 65 in patients with stiff person syndrome. J Autoimmun 32:24–32CrossRef Skorstad G, Hestvik AL, Vartdal F, Holmoy T (2009) Cerebrospinal fluid T cell responses against glutamic acid decarboxylase 65 in patients with stiff person syndrome. J Autoimmun 32:24–32CrossRef

go back to reference Burman J, Tolf A, Hagglund H, Askmark H (2018) Autologous haematopoietic stem cell transplantation for neurological diseases. J Neurol Neurosurg Psychiatry 89:147–155CrossRef Burman J, Tolf A, Hagglund H, Askmark H (2018) Autologous haematopoietic stem cell transplantation for neurological diseases. J Neurol Neurosurg Psychiatry 89:147–155CrossRef