ABSTRACT
BACKGROUND AND PURPOSE: The aim of this study was to determine the efficacy and tolerability of granulocyte colony-stimulating factor (G-CSF) in subjects with amyotrophic lateral sclerosis (ALS). METHODS: Forty subjects with ALS were randomly assigned to two groups, which received either subcutaneous G-CSF (5 microg/kg/q12h) or placebo for 5 days. The subjects were then followed up for 3 months using the ALS Functional Rating Scale-Revised (ALSFRS-R), manual muscle testing, ALS Assessment Questionnaire-40, and nerve conduction studies. CD34+/CD133+ cell count and monocyte chemoattractant protein-1 (MCP-1) levels were evaluated at baseline. RESULTS: The rate of disease progression did not differ significantly between the two groups. The reduction in ALSFRS-R scores was greater in female subjects in the G-CSF group than in their counterparts in the placebo group. There was a trend toward a positive correlation between baseline CSF MCP-1 levels and the change in ALSFRS-R scores in both groups (Spearman's rho=0.370, p=0.070). CONCLUSIONS: With the protocol implemented in this study, G-CSF is not a promising option for the treatment of ALS. Furthermore, it may accelerate disease progression in females.
Subject(s)
Female , Humans , Amyotrophic Lateral Sclerosis , Cell Count , Chemokine CCL2 , Disease Progression , Granulocyte Colony-Stimulating Factor , Neural ConductionABSTRACT
BACKGROUND: The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is a quantitative measure of ALS-related physical deficit with established reliability and validity in prospective clinical research. This study aimed to test the reliability and validity of the Korean version of ALSFRS-R (K-ALSFRS-R). METHODS: The subjects of the study were 51 patients with ALS diagnosed on the basis of El Escorial criteria. The patients were evaluated by one rater using K-ALSFRS-R, Norris scale, and Appel ALS rating scale on the same day. Subsequently, K-ALSFRS-R was measured by two other raters using a videotape design. Test-retest data were obtained within the time interval of 7 days. RESULTS: Internal consistency and test-retest reliability were high. Inter-rater comparisons showed significant reliability with Kappa or Kendall's tau-b value. Moreover, K-ALSFRS-R scores correlated significantly with clinical status as measured by Norris and Appel ALS rating scale. CONCLUSIONS: Our results showed that K-ALSFRS-R would be a reliable and useful instrument for the evaluation of functional status in patients with ALS.
Subject(s)
Humans , Amyotrophic Lateral Sclerosis , Pilot Projects , Prospective Studies , Reproducibility of Results , Videotape RecordingABSTRACT
Objective To evaluate proton MR spectroscopy (1H-MRS) for detection of the motor cortex and adjacent brain in amyotrophic lateral sclerosis (ALS) patients with apparent upper motor neuron involvement after olfactory ensheathing cells(OECs) transplantation. Methods From December 2004 to February 2005, 7 patients with clinically definite ALS who could safely undergo MRS were admitted into the perspective study. The neurological status, ALS functional rating scale (ALSFRS), EMG, and 1H-MRS taken before and 2 weeks after operations were carefully analyzed. The NAA/Cr and Cho/Cr ratios were measured in the cerebral peduncle, genu and posterior limb of the internal capsule, corona radiata and precentral gyrus. Results The ALSFRS in 2 cases improved obviously whose ALSFRS increased from 30 to 33 and from 29 to 34 respectively. And 5 cases remained stable 2 weeks after OECs transplantation. Statistical analyses for all seven cases showed both the NAA/Cr and Cho/Cr ratios decreased, but in the two cases with ALSFRS improvement the NAA/Cr increased in the certain anatomic position which confirmed the neurological and EMG findings. Conclusion The proton MR spectroscopy is a suitable noninvasive measure for ALS evaluation. The preliminary study suggests that two of the seven ALS cases improved apparently short-term after OECs transplantation. More patients are required for the clinical study and longer follow-up duration is needed for future research.