There has been a shortage of certain medications for about three years now. Particularly hard hit have been generic injectable drugs. The shortage of one medication has affected kids with cancer.
Young Hodgkin's lymphoma patients relapsed more during the time when mechlorethamine (Mustargen) was unavailable. No patient died because of the shortage, which has now been resolved.
However, the substituted treatment is very intensive, and possible long-term complications can include infertility.
St. Jude Children's Research Hospital led a national clinical trial involving 181 youngsters with Hodgkin's lymphoma. Various institutions were collaborating under the umbrella of The Pediatric Hodgkin Lymphoma Consortium. The Stanford University School of Medicine and Lucille Packard Children's Hospital at Stanford; Dana-Farber Cancer Institute and Boston Children's Hospital; Massachusetts General Hospital; and Maine Medical Center participated in the Consortium.
The trial was testing the effectiveness of a 7-drug cocktail, which included mechlorethamine. The study goal was to examine how to reduce the risk of second cancers, infertility and other problems associated with earlier treatment methods.
Hodgkin's lymphoma is a cancer that affects the lymph system, which helps the body fight off infection. It makes up about 4 percent of childhood cancers and is the most common cancer in youngsters between the ages of 15 and 19. About 90 percent of patients live many years after diagnosis.
Mechlorethamine has been used to treat a number of cancers since the 1960s. The shortage of this medication began after the trial was started in 2002. Doctors substituted another commonly used anti-cancer medicine – cyclophosphamide, which is sold under various names including Cytoxin.
Trial members ranged in age from 3 to 21, with half being under the age of 14. The number of youngsters who were disease-free for two years dropped from 88 percent when mechlorethamine was available to 75 percent when cyclophosphamide had to be used in its place in 40 patients.
"This puts a face on the problem of drug shortages and shows that the problem is real, not theoretical,” said Michael Link, MD, the senior author of a new report and professor of pediatrics in hematology-oncology at Stanford.
“This is about a curative therapy that we were unable to administer because the drug we needed was not available."
He concluded, “Despite heroic efforts by the drug shortage office of the Food and Drug Administration to solve the shortages of a number of medically necessary drugs, it is clear that patients are still suffering from the unavailability of life-saving drugs. A more systematic solution to the problem is needed."
Patients who relapsed received intensive chemotherapy and a stem cell transplant using their own stem cells. Researchers say it’s too early to know about the long-term survival rates among these patients compared to those who did not relapse.
Lundbeck Inc., the manufacturer of mechlorethamine blamed the shortage on a change in the facility where it was made. In mid November 2012, the company reported that supplies have stabilized.
A perspective on the study was published December 27 in the New England Journal of Medicine.