Adding thalidomide to standard treatment improves the survival for patients suffering from a blood cancer by 18 months, according to research published in The Lancet today. In the study 447 newly diagnosed patients with multiple myeloma, aged 65 to 75 who were ineligible for intensive bone marrow transplantation regimens, were randomly assigned to receive one of the following: melphalan and prednisone (MP), which is standard therapy for multiple myeloma in the EU; standard therapy plus thalidomide (MP-T); or reduced intensity stem cell transplantation using melphalan 100mg/m2 (MEL100). The primary endpoint was overall survival. Overall survival rates were 51.6 months for MP-T therapy, compared to 33.2 months for MP and 38.3 months for the third group.
Commenting on the study, Professor Gareth Morgan, Professor of Haematology at The Royal Marsden Hospital, London, said: "The results of this study will have an enormous impact on how multiple myeloma is treated in the future. It redefines the standard treatment for patients with multiple myeloma, and makes the combination of melphalan-predisone-thalidomide the gold standard." For older patients, who are ineligible for intensive bone marrow transplantation regimens, melphalan-prednisone has been the standard of care since the 1960s for multiple myeloma.
The disease is more common in men, and usually occurs in people over age 65. Eric Low, Chief Executive of Myeloma UK said: "Thalidomide is undoubtedly an important treatment for patients. It is given orally, has flexible administration and has manageable side-effects. However, patients should not have to receive unlicensed treatments. Thalidomide should be licensed to ensure safety, prevent unregulated supply and impose better regulation, and also help address inconsistent approaches to prescribing and improve patient access to thalidomide in the UK."
Based on these data, Pharmion is seeking authorisation for the use of thalidomide in combination with melphalan and prednisone for the treatment of patients with untreated multiple myeloma aged 65 years or older or in patients ineligible for high dose chemotherapy. Thalidomide must be prescribed and dispensed through the Pharmion Risk Management Programme. This is a carefully regulated programme aimed at preventing pregnancy in patients receiving thalidomide as part of their therapy. There are between 14,000 and 20,000 people with multiple myeloma in Britain. Every year, about 3,700 people are diagnosed with the disease, and about 2,400 will die because of it. It is the second-most common blood cancer after non-Hodgkins lymphoma.
The study was carried out by the French researchers, headed by Professor Thierry Facon from Intergroupe Francophone du Myélome. The researchers say in The Lancet: "After 40 years of unsuccessful attempts to find a more effective treatment than the standard MP, the MP-T combination opens an era of progress for elderly patients with this disease."