Enzyme replacement therapy "drug holiday": Results from an unexpected shortage of an orphan drug supply in Australia

Blood Cells, Molecules, and Diseases

Volumn 46, Issue 1, 2011, Pages 107-110

  Goldblatt, Jack ^a,b   Fletcher, Janice M ^c   McGill, Jim ^d   Szer, Jeffrey ^e   Wilson, Meredith ^f  

^a UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^b KING EDWARD MEMORIAL HOSPITAL   (Australia)

^c WOMEN S AND CHILDREN S HOSPITAL   (Australia)

^d ROYAL CHILDREN'S HOSPITAL   (Australia)

^e ROYAL MELBOURNE HOSPITAL   (Australia)

^f UNIVERSITY OF SYDNEY   (Australia)

Author keywords

Drug holiday; Enzyme replacement therapy; Gaucher disease

Indexed keywords

CHITOTRIOSIDASE; IMIGLUCERASE; NONSTEROID ANTIINFLAMMATORY AGENT;

ADULT; ADVERSE OUTCOME; AGED; ARTICLE; AUSTRALIA; CLINICAL ARTICLE; DISEASE SEVERITY; DRUG CONTAMINATION; ENZYME REPLACEMENT; FATIGUE; FEMALE; GAUCHER DISEASE; HEMOGLOBIN BLOOD LEVEL; HUMAN; LIMB PAIN; MAINTENANCE THERAPY; MALE; PATIENT MONITORING; PRIORITY JOURNAL; THROMBOCYTE COUNT;

ADOLESCENT; ADULT; AGED; AUSTRALIA; ENZYME REPLACEMENT THERAPY; FEMALE; GAUCHER DISEASE; GLUCOSYLCERAMIDASE; HUMANS; MALE; MIDDLE AGED; ORPHAN DRUG PRODUCTION; PHARMACEUTICAL PREPARATIONS; TREATMENT OUTCOME; YOUNG ADULT;

EID: 78650816915     PISSN: 10799796     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.bcmd.2010.05.002     Document Type: Article

References (8)

1. Goldblatt J., Szer J., Fletcher J.M., McGill J., Rowell J.A., Wilson M. Enzyme replacement therapy for Gaucher disease in Australia. Intern. Med. J. 2005, 35:156-161.
2. Hollak C.E., Vom Dahl S., Aerts J.M., et al. Force majeure: therapeutic measures in response to restricted supply of imiglucerase (Cerezyme) for patients with Gaucher disease. Blood Cells Mol. Dis. 2010, 44:41-47.
3. Charrow J., Andersson H.C., Kaplan P., et al. The Gaucher registry: demographics and disease characteristics of 1698 patients with GD1. Arch. Intern. Med. 2000, 9:2835-2843.
4. Boomsma J.M., van Dussen L., Wiersma M., et al. Spontaneous regression of disease manifestations can occur in type 1 Gaucher disease; results of a retrospective cohort study. Blood Cells Mol. Dis. 2010, 44:181-187.
5. Weinreb N.J., Cappellini M.D., Cox T.M., et al. A validated disease severity scoring system for adults with type 1 Gaucher disease. Genet. Med. 2010, 12:44-51.
6. Andersson H.C., Charrow J., Kaplan P., et al. Individualization of long-term enzyme replacement therapy for Gaucher disease. Genet. Med. 2005, 7:105-110.
7. Kishnani P.S., DiRocco M., Kaplan P., et al. A randomized trial comparing the efficacy and safety of imiglucerase (Cerezyme) infusions every 4weeks versus every 2weeks in the maintenance therapy of adult patients with Gaucher disease type 1. Mol. Genet. Metab. 2009, 96:164-170.
8. Elstein D., Abrahamov A., Hadas-Halpern I., Zimran A. Withdrawal of enzyme replacement therapy in Gaucher's disease. Br. J. Haematol. 2000, 110:488-492.