New England Journal of Medicine Article Reports Inferior Outcome in Using Alternative Treatments to Counter Mechlorethamine Shortage


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ASCO Immediate Past President Michael P. Link, MD, recently coauthored a perspectives piece in The New England Journal of Medicine on the impact of drug shortages on children with cancer.

The paper, “The Impact of Drug Shortages on Children with Cancer — The Example of Mechlorethamine,” describes the impact of using alternative treatments to counter shortages of the drug mechlorethamine (Mustargen), also known as nitrogen mustard. Mechlorethamine was one of the first anticancer agents created and has been used in a 12-week chemotherapy regimen for treating children and adults with Hodgkin lymphoma that includes vinblastine, doxorubicin, vincristine, bleomycin, etoposide, and prednisone. When mechlorethamine went into shortage in 2009, it was substituted with cyclophosphamide in the regimen.

Greater Event-free Survival with Mechlorethamine

A retrospective analysis through the Pediatric Hodgkin Lymphoma Consortium compared the probability of event-free survival among 181 patients who were treated with the original regimen including mechlorethamine with 40 patients treated with the modified regimen including cyclophosphamide. The study found that 88% of patients treated with the established regimen with mechlorethamine were expected to be event-free survivors at 2 years, compared to only 75% of those treated with the cyclophosphamide-containing regimen. The article noted:

Almost 80% of children and adolescents with cancer can be cured with current therapy. Most of the curative treatment regimens are based on chemotherapeutic agents that have been available for decades, but some of these have recently been in short supply. These shortages are likely to have devastating effects on patients with cancer and must be prevented. For many of these agents, no adequate substitute drugs are available. Our results suggest that even promising substitute regimens should be examined carefully before adoption; what might appear to be a suitable alternative regimen may result in an inferior outcome—an intolerable situation for young people with curable diseases.

ASCO is currently in the process of surveying its members to gauge how shortages are impacting their practice and will continue to monitor the situation. Please continue to visit ascoaction.asco.org for the latest news on drug shortages, research and cancer policy. ■

© 2013. American Society of Clinical Oncology. All rights reserved.



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