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Successful Autologous Hematopoietic Stem Cell Transplant in Glycine Receptor Antibody-Positive Stiff Person Syndrome: A Case Report
Celli, Sofia I; Nash, Richard; Money, Kelli M; Garza, Madeline; Borko, Tyler L; Mizenko, Christopher; McMenamin, Constance; Von Geldern, Gloria; Georges, George; Piquet, Amanda L.
Afiliação
  • Celli SI; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Nash R; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Money KM; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Garza M; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Borko TL; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Mizenko C; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • McMenamin C; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Von Geldern G; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Georges G; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
  • Piquet AL; From the Department of Neurology (S.I.C., K.M.M., M.G., T.L.B., C. Mizenko, C. McMenamin, A.L.P.), University of Colorado, Aurora Colorado Blood Cancer Institute (R.N.), Presbyterian St. Luke's Medical Center, Denver, CO; Department of Neurology (G.V.G.); Fred Hutchinson Cancer Center (G.G.), Univer
Neurol Neuroimmunol Neuroinflamm ; 11(2): e200197, 2024 03.
Article em En | MEDLINE | ID: mdl-38170953
ABSTRACT
BACKGROUND AND

OBJECTIVES:

To describe a case of glycine receptor (GlyR) antibody-positive stiff person syndrome (SPS) treated with autologous hematopoietic stem cell transplant (aHSCT).

METHODS:

This was a multicenter collaboration for the treatment of a single patient who underwent aHSCT as part of a clinical trial (NCT00716066). To objectively assess the response to transplantation, several clinical outcome measures were evaluated pretransplant and up to 18 months post-transplant, including modified Rankin Score (mRS), stiffness index, Hauser Ambulation Score (HAS), hypersensitivity index, timed 25-foot walk, and Montreal Cognitive Assessment.

RESULTS:

After transplant, the patient achieved sustained clinical improvement evidenced across various clinical scales, including mRS, stiffness index, HAS, and 25-foot walk time.

DISCUSSION:

aHSCT represents a promising treatment option for SPS, including for GlyR-positive patients. In addition, this case represents the need to validate and standardize best clinical outcome measures for patients with SPS. CLASSIFICATION OF EVIDENCE Class IV; this is a single observational study without controls.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Rigidez Muscular Espasmódica / Transplante de Células-Tronco Hematopoéticas Tipo de estudo: Clinical_trials / Observational_studies Limite: Humans Idioma: En Ano de publicação: 2024 Tipo de documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Rigidez Muscular Espasmódica / Transplante de Células-Tronco Hematopoéticas Tipo de estudo: Clinical_trials / Observational_studies Limite: Humans Idioma: En Ano de publicação: 2024 Tipo de documento: Article