Letter to the Editor concerning 'cost-effectiveness of velmanase alfa vs. bone marrow transplantation or no causal therapy in patients with mild to moderate alpha-mannosidosis'.

The letter to the editor critiques a cost-effectiveness analysis of velmanase alfa (VA) for alpha-mannosidosis treatment, pointing out limitations in the modeling approach used. While the study suggests that VA is not cost-effective compared to other treatments, it fails to consider key symptoms and relies on data from studies that did not specifically address VA efficacy. In contrast, the UK's NICE recommended VA for non-neurological symptoms of alpha-mannosidosis in children under 18, based on a Markov model incorporating long-term efficacy data. The letter emphasizes the need for caution in interpreting the results of the cost-effectiveness analysis and highlights the importance of ongoing research to refine understanding of treatment options for alpha-mannosidosis. [Extracted from the article]