Purpose: Clinical care for patients with rare diseases may be complicated by comorbidities. Administration of medications to treat comorbidities may elicit potentially harmful drug-drug interactions (DDIs). Genetic background may also influence DDI occurrence. We investigated the range of comorbid conditions in patients with Gaucher disease type I (GD1), the pharmacotherapies prescribed and the potential for DDI with enzyme replacement and substrate reduction therapies and additional medications, specifically cytochrome P450 (CYP) metabolizing medications. Methods: A literature review examined comorbid conditions and pharmacotherapies reported in GD1. Analysis of two national databases reported real-world prescription practices in patients with GD1 (Germany, N = 87; US, N = 374). Prescribed drugs were assessed for known interactions with isoenzymes from the hepatic CYP enzyme family. Results: The literature reported GD1 symptomatology and comorbid conditions in broad agreement with the known clinical picture. German patients received 86 different medications whereas US patients received 329 different medications. An average of 3.2 medications (Germany) and 7 medications (US) per patient were prescribed. Moderate/strong inhibitors of CYP isoenzymes were prescribed to 20% and 57% of patients in the US and Germany, respectively. Conclusion: This study describes the extensive number of comorbid conditions and drugs prescribed to patients with GD1, and the importance of determining CYP isoenzyme interaction to reduce DDI risk.
Bibliographical noteFunding Information:
Writing and editorial assistance in the preparation of this manuscript were provided by Katie Bickford and Andrew Smith PhD of Fishawack Communications GmbH, Basel, Switzerland. This support was funded by Actelion Pharmaceuticals Ltd. Prescription data were obtained from IMS Health and paid for by Actelion Pharmaceuticals Ltd.
- Clinical decisions
- Drug-drug interactions
- Gaucher disease type 1
- Treatment decisions