Dr. Dale and his research colleagues compared the modified Rankin Scale (mRS) outcomes of 16 patients with opsoclonus myoclonus ataxia syndrome who received rituximab within one year of first-line treatment with those of 16 patients who received rituximab after one year of first-line treatment. Among patients who started rituximab early, 93.3% had an mRS of 0–2 (ie, better outcomes), compared with 62.5% of patients who started rituximab later. This result “really makes sense. As a general principle of medicine, earlier treatment is better,” said Dr. Dale.
The comparison was part of a larger retrospective study of 144 children who were treated with rituximab for neurologic indications. The researchers concluded that the data support an off-label use of rituximab, but the significant risk of infectious complications suggests that the therapy should be restricted to disorders with significant morbidity and mortality.
In another recent study, Mitchell et al compared the outcomes of a historic cohort of patients with opsoclonus myoclonus ataxia syndrome with those of a new cohort of patients that the researchers treated more aggressively and with newer therapies, including rituximab. The newer patients had significantly higher adaptive behavior, cognitive scores, and motor outcome scores, compared with the historic cohort. Considered together, the studies provide uncontrolled evidence that early, aggressive treatment of severe disease improves outcomes, Dr. Dale concluded.
—Jake Remaly