C37 AGGRESSIVE APPROACHES TO SYMPTOM MANAGEMENT IN PATIENTS WITH MOTOR NEURON DISEASE
ROSENFELD J
Carolinas ALS Center, Charlotte, North Carolina, United States
E-mail address for correspondence: [email protected]
Keywords: therapies, symptom control, multidisciplinary care
The diversity and severity of symptoms arising from motor neuron disease (MND) is vast. Despite the large variety of symptoms encountered in patients with MND, there is a paucity of evidence-based recommendations for treatment.
An aggressive and proactive treatment for symptomatic control in MND frequently implicates empiric or anecdotal treatment trials of medications, therapies or devices. These may have an obvious benefit to the patient and be commonly utilized in selected clinics despite a lack of formal data from randomized, controlled trials. The disparity between the large number of available treatment options for control of disabling symptoms and the limited number of approved therapies has resulted in great variability between specialized treatment centers and the care received in more general neurology practice settings.
This presentation will highlight a variety of successful therapies which, although not formally approved for patients with MND, are commonly utilized or discussed in the context of aggressive, proactive multidisciplinary care. In addition, data from trials regarding symptom control in other neurodegenerative disease states will be reviewed.
Management of spasticity, sialorrhea, dyspnea, secretion management, cramping, muscle spasm and fatigue will be discussed. In addition, emerging technology for augmentative communication will be included. Available data and empiric observations for the use of intrathecal baclofen, botulinum toxin, modafinil, noninvasive ventilation during PEG placement, diaphragm pacing, in-exsufflation, standing frame and motorized pedlar will be included.
This overview presentation will emphasize many of the most problematic symptoms and the multidisciplinary approaches toward their effective resolution.
C38 RANDOMIZED PLACEBO-CONTROLLED CROSSOVER TRIAL WITH THC (DELTA 9-TETRAHYDROCANNABINOL) FOR THE TREATMENT OF CRAMPS IN AMYOTROPHIC LATERAL SCLEROSIS (ALS)
WEBER M, HARTMANN S, GOLDMAN BEA
Neuromuscular Dieases Unit, Kantonsspital St.Gallen, St.Gallen, Switzerland
E-mail address for correspondence: [email protected]
Keywords: Randomized trial, Delta 9-tetrahydrocannabinol, cramps
Background: Many patients with ALS experience cramps. Severity varies from mild, without affecting daily activities and sleep, to disabling, where almost any voluntary muscle activity induces long standing, severely painful cramping. So far, there has not been any proven benefit of medications used for the treatment of cramps in ALS.
Objective: To determine the tolerability, safety and efficacy of THC in the treatment of cramps in ALS. It is hypothesized that the severity of cramps will lessen significantly in patients treated with THC compared to treatment with placebo.
Methods: Standard 2x2 crossover study design was used. Patients who had a cramp severity score of 5 or more on the visual analogue scale (VAS) became eligible. After a 14-day run-in period patients were treated for 2 weeks with either THC 5 mg bid or matching placebo in a double-blinded manner. After a 14-day washout period, a new 2-week treatment period followed. The primary variable of effectiveness was severity of cramps measured daily by a VAS. Secondary outcome measures were number of cramp days and nights and severity of fasciculations. Possible side effects were also monitored.
Results: A total of 27 patients were enrolled. Two patients experienced spontaneous lessening of cramps during the run-in period and withdrew from the study, one patient died during the study. Tolerability of the chosen dosage (THC 5 mg bid) was excellent. Only one patient experienced side effects (dizziness). No patient showed a significant change of monitored haematology and blood chemistry values. Three quarters of the patients were able to identify a treatment period with lessening of cramps. Currently statistical analysis is under way. A repeated measure ANOVA is applied with treatment and carry-over as within factors. The severity of cramps at each day will enter as a covariate in the model.
Discussion: For the treatment of cramps in ALS a dosage of THC 5 mg bid is well tolerated and safe. The fact that three quarters of the patients identified a treatment period with lessening of cramps suggests that THC is also effective. This was not caused by central side effects (e.g. euphoria). Statistical results and efficacy data will be included in the presentation
C39 EVALUATION OF A “FAST TRACK” PROCESS FOR THE EVALUATION AND INVESTIGATION OF PEOPLE WITH SUSPECTED ALS
MITCHELL D, CALLAGHER P, ADDISON-JONES R, BENNETT W, GARDHAM J
Preston MND Care & Research Centre, Preston, Lancashire, United Kingdom
E-mail address for correspondence: [email protected]
Keywords: Fast track, diagnosis, investigation
Background: There is a perception in UK neurological practice that the evaluation of people with suspected ALS can sometimes take a relatively long period of time and that current structures are inconsistent with the expedited processes that often pertain for the investigation of people suspected of having other critical illnesses such as cancer. We have been able to implement a “fast track” (FT) process for the investigation of people suspected of having ALS
Aim: To compare timelines in the process of the evaluation of people suspected of having ALS going through the FT process with those traversing more traditional pathways
Methods: Times from referral to first neurological consultation and from referral to diagnosis, along with other details relating to management pathways were determined in 144 patients with ALS. Comparisons relating to the patient journey were made between those patients going through the FT process and those following non-fast track (NFT) pathways
Results: Median time from referral to diagnosis was 51 days for the FT patients and 104.5 days for the NFT patients. The theoretical target for this timeline for the FT patients was 8 weeks. 60% of the FT patients achieved this target compared with 30% of the NFT patients (OR 0.29, 95%CI 0.08–0.99). At 9 weeks the figures were 76% & 30% (OR 0.14, 95%CI 0.04–0.51), 12 weeks 88% & 30% (OR 0.06, 95%CI 0.01–0.29) and at 18 weeks 88% & 60% (OR 0.21, 95%CI 0.05–0.96) for FT and NFT patients respectively.
Discussion: Twice the proportion of patients in the FT process achieved diagnosis within the theoretical target of the FT clinic as those seen in the general clinics. This difference increases sharply in favour of the FT process as time following referral increases until 18 weeks when only 1.47 times the proportion of FT patients to NFT patients achieved diagnosis. The FT process therefore offers the chance of a much-accelerated patient pathway to diagnosis up to 18 weeks after referral. An important barrier to initiating the FT process is the identification of ALS as a potential diagnosis. It is possible that however efficient the diagnostic process from a theoretical perspective, a small proportion of service users will always have a relatively prolonged diagnostic journey. This can arise through atypical presentation, confusing co-morbidities etc. The UK NHS seeks to achieve diagnosis and treatment within 18 weeks of referral for all NHS patients and this data shows that in most cases this is being achieved. This work is currently being used in discussions with the UK Department of Health in the development of a generally applicable 18-week pathway for people suspected of having ALS