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Evidence summaries

Treatment for Familial Amyotrophic Lateral Sclerosis

The treatment response in patients with familial and sporadic forms of ALS might possibly not be different, but the evidence is insufficient. Level of evidence: "D"

A Cochrane review [Abstract] 1 included 5 RCTs with a total of 895 patients with amyotrophic lateral sclerosis (ALS), 52 patients had familial ALS (FALS). The active agent in two trials was creatine monohydrate, in other trials celecoxib, topiramate and arimoclomol. In 3 trials the primary outcomes were the rate of decline of strength of arm muscles, death, tracheostomy and persistent assisted ventilation-free survival and in the arimoclomol trial tolerability and safety. When combining the data from all 5 studies, the difference was not statistically significant for the treatment response of patients with FALS as compared with ALS (HR 0.90, 95% Cl -0.13 to 0.84). The pooled estimate of HR suggested a more beneficial response with respect to survival among patients with FALS, but the result was not statistically significant. Estimates of the rate of decline on the arm muscle strength also suggested a slightly better response to treatment among those with FALS, but the result was not statistically significant either.

Comment: The quality of evidence is downgraded by reporting bias (only few of the reported trials could be included in the review), inconsistency (differences in interventions and outcomes) and imprecise results (few patients and wide confidence intervals).

References

  • Benatar M, Kurent J, Moore DH. Treatment for familial amyotrophic lateral sclerosis/motor neuron disease. Cochrane Database Syst Rev 2009;(1):CD006153. [PubMed]

Primary/Secondary Keywords