ABSTRACT
Granulocyte-colony stimulating factor (G-CSF) is used to mobilize CD34+ haematopoietic stem cells from the bone marrow to the peripheral blood. We proposed to use cell subsets induced by G-CSF to slow down disease progression in patients with amyotrophic lateral sclerosis (ALS). Patients with definite or probable ALS were assigned in a double-blind manner to receive G-CSF or placebo every three months for a year. The primary outcome measure was the functional decline, measured by the revised ALS Functional Rating Scale, Revised (ALSFRS-R) score. Secondary outcome measures included vital capacity, manual muscle strength, compound muscle action potential amplitudes, neurophysiological index, and McGill single item quality of life score (QoL). Thirty-nine patients were enrolled. Seventeen patients who received G-CSF and 18 who received placebo were evaluated. G-CSF was effective in mobilizing CD34+ to blood. The outcome measures used showed no statistically significant benefit, although there was a trend of slowing disease progression following two G-CSF treatments, as shown by lower slopes of ALSFRS-R and QoL in the first six treatment months. The treatment had no major side-effects. G-CSF administration in ALS patients caused successful mobilization of autologous bone marrow cells, but was not effective in slowing down disease deterioration.
Subject(s)
Amyotrophic Lateral Sclerosis/drug therapy , Granulocyte Colony-Stimulating Factor/administration & dosage , Hematopoietic Stem Cell Mobilization/methods , Action Potentials/drug effects , Adult , Aged , Amyotrophic Lateral Sclerosis/physiopathology , Disease Progression , Female , Follow-Up Studies , Granulocyte Colony-Stimulating Factor/adverse effects , Humans , Male , Middle Aged , Muscle Strength/drug effects , Patient Dropouts , Pilot Projects , Placebos , Recombinant Proteins , Treatment Failure , Vital Capacity/drug effectsABSTRACT
BACKGROUND: Statins are increasingly recognized as causing muscle damage and, more rarely, peripheral neuropathy. A preliminary report that there are more cases of amyotrophic lateral sclerosis (ALS) among people treated with statins caused considerable concern. We considered the possibility that statins could affect survival in patients already diagnosed as having amyotropic lateral sclerosis who were taking statins for dyslipidemia. METHODS: We reviewed the clinical charts of 459 patients with ALS followed-up in our clinic between 1997 and 2007. Retrieved data included age, all administered medications, form of ALS at onset and survival. We compared the survival rates of patients taking any statins with that of patients not taking statins, while adjusting for other factors which influence disease progression, such as age, gender and ALS form at onset. RESULTS: 72 patients were on statins for dyslipidemia at disease onset. The doses ranged from 10-60 mg daily and varied throughout the disease course. As expected, the patients on statins were older than the non-treated ones (65.7+/-9 versus 57.5+/-13 years, respectively). After correcting for age, gender and disease form, there was no significant difference in survival between the groups. CONCLUSION: Our findings indicate that statins treatment for dyslipidemia in patients with ALS does not carry any survival risks.
