Comorbidities and pharmacotherapies in patients with Gaucher disease type 1: The potential for drug-drug interactions.

摘要: Abstract Purpose Clinical care for patients with rare diseases may be complicated by comorbidities. Administration of medications to treat comorbidities elicit potentially harmful drug–drug interactions (DDIs). Genetic background also influence DDI occurrence. We investigated the range comorbid conditions in Gaucher disease type I (GD1), pharmacotherapies prescribed and potential enzyme replacement substrate reduction therapies additional medications, specifically cytochrome P450 (CYP) metabolizing medications. Methods A literature review examined reported GD1. Analysis two national databases real-world prescription practices GD1 (Germany, N = 87; US, N = 374). Prescribed drugs were assessed known isoenzymes from hepatic CYP family. Results The symptomatology broad agreement clinical picture. German received 86 different whereas US 329 An average 3.2 (Germany) 7 (US) per patient prescribed. Moderate/strong inhibitors 20% 57% Germany, respectively. Conclusion This study describes extensive number GD1, importance determining isoenzyme interaction reduce risk.