Successful Autologous Hematopoietic Stem Cell Transplant in Glycine Receptor Antibody-Positive Stiff Person Syndrome: A Case Report
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.
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