An Open-Label, Prospective Study Evaluating the Clinical and Immunological Effects of Higher Dose Granulocyte Colony–Stimulating Factor in ALS
| Autor: | Chuanhai Cao, Yazan Suradi, Tuan Vu, Xiaoyang Lin, Kristyn Pocock, Samuel Dang, Brittany Harvey, Niraja Suresh, Kyle Sutherland, Clifton L. Gooch |
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| Rok vydání: | 2020 |
| Předmět: |
Male
medicine.medical_specialty Pilot Projects Gastroenterology Malaise Internal medicine Granulocyte Colony-Stimulating Factor Humans Medicine Prospective Studies Amyotrophic lateral sclerosis Prospective cohort study Adverse effect business.industry Amyotrophic Lateral Sclerosis Mesenchymal Stem Cells General Medicine Middle Aged medicine.disease Granulocyte colony-stimulating factor Clinical trial Treatment Outcome Neurology Tolerability Cohort Disease Progression Female Neurology (clinical) medicine.symptom business |
| Zdroj: | Journal of Clinical Neuromuscular Disease. 21:127-134 |
| ISSN: | 1522-0443 |
| Popis: | Objective 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. Methods 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. Results 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. Conclusions Higher-dose G-CSF was well tolerated in this cohort with no apparent effect on disease progression up to 1 year. |
| Databáze: | OpenAIRE |
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