CPC-086 Miglustat Off-Label in a Paediatric Formulation For a Rare Metabolic Disease: Early Infantile GM1 Gangliosidosis

Abstract

A196Eur J Hosp Pharm 2013;20(Suppl 1):A1-A238Results A total of 52 patients (84% women) were prescribed aprepitant during the study period. The average age was 49 years (age range: 19-69 years). The following data were collected: diagnosis and stage of disease, chemotherapy scheme, anti-emesis change cycle number, combination with radiotherapy and alcohol intake. 65% of patients had breast cancer followed by non-small lung cancer (5%). 27% and 25% of cancers were in stages IA and IIA respectively. The most common chemotherapy scheme (55%) for which the change of antiemetic therapy was seen, was FEC 500-100-500. 26% of patients started ondansetron 4 or 8 mg before aprepitant was prescribed. The rest (74%) received aprepitant directly after failing the first line antiemetic therapy. In 40% of patients the antiemetic regimen was changed to the study drug in cycle 2 and in 25% in cycle 3, demonstrating that aprepitant is not used as first-line antiemetic. Only 5 patients received radiotherapy combined with chemotherapy and only in 4 was alcohol intake recorded. Conclusions In our hospital aprepitant is mainly used in chemotherapy regimens that include anthracyclines in combination with cyclophosphamide. It is prescribed after first line antiemetic regimen failure; meeting the indications established by Drug and Therapeutics Committee. However, it would be advisable to cheque the antiemetic guidelines periodically for compliance with reference guides such as NCCN, ASCO, MASCC etc.No conflict of interest.

Substrate reduction therapy with Miglustat in pediatric patients with GM1 type 2 gangliosidosis delays neurological involvement: A multicenter experience

“…Indeed, GM1 gangliosidosis mouse models demonstrated Miglustat effectiveness in reducing brain GM1 ganglioside content (Kasperzyk et al., 2004; Treiber, Morand, & Clozel, 2007). Data on Miglustat efficacy in patients with GM1 gangliosidosis are scarce but promising, as reported in a few pediatric and adult patients affected by type 1 and 2 forms (Deodato et al., 2017; Garzone et al., 2013; Tifft, Adams, & Morgan, 2007).…”