An Open-Label, Prospective Study Evaluating the Clinical and Immunological Effects of Higher Dose Granulocyte Colony-Stimulating Factor in ALS.
Journal
Journal of clinical neuromuscular disease
ISSN: 1537-1611
Titre abrégé: J Clin Neuromuscul Dis
Pays: United States
ID NLM: 100887391
Informations de publication
Date de publication:
Mar 2020
Mar 2020
Historique:
entrez:
20
2
2020
pubmed:
20
2
2020
medline:
15
12
2020
Statut:
ppublish
Résumé
We evaluated the safety and tolerability of higher-dose granulocyte colony-stimulating factor (G-CSF) in patients with amyotrophic lateral sclerosis. In addition, rates of disease progression and serum G-CSF levels and other immunological and hematological markers were measured. Three patients with advanced amyotrophic lateral sclerosis were treated with G-CSF subcutaneously at 5 μg/kg twice daily for 5 consecutive days monthly for 4-12 months. Patients were monitored for adverse effects, and disease progression was assessed with ALSFRS-R and other measures. Patients tolerated higher-dose G-CSF well with no serious adverse events. Adverse effects were mild to moderate with musculoskeletal pain and malaise being most often reported. No significant change in the rate of disease progression was noted for ALSFRS-R or other measures. Bone marrow progenitor cells were rapidly mobilized for a duration of approximately 9 days with transient and variable effect on cytokines. Higher-dose G-CSF was well tolerated in this cohort with no apparent effect on disease progression up to 1 year.
Identifiants
pubmed: 32073458
doi: 10.1097/CND.0000000000000275
pii: 00131402-202003000-00001
doi:
Substances chimiques
Granulocyte Colony-Stimulating Factor
143011-72-7
Types de publication
Clinical Trial
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
127-134Références
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