Innovative Drug Approved for Long-term Treatment of Chronic Myelogenous Leukemia
2018-01-23 |

Innovative Drug Approved for Long-term Treatment of Chronic Myelogenous Leukemia

Chronic myelogenous leukemia (CML) is a type of cancer that affects white blood cells and bone marrow. It is a form of leukemia caused by a genetic disorder disease of the hematopoietic system, with an increased and unregulated growth of white blood cells, predominantly myeloid cells in the bone marrow. After being produced, these cells are accumulated in the blood, where they can further multiply and crowd out healthy blood cells and platelets.


CML accounts for nearly 10% of all leukemias. It is the second most common form of chronic leukemia. Patients with CML have non-specific signs, which depending on the stage of disease may range from fever, malaise, loss of weight to an enlarged spleen, infections, swollen glands, and bone pain. There are various treatment options for CML. The optional therapy depends largely on the stage of the disease.


Treatment for CML with the tyrosine kinase inhibitor Imatinib is also effective over the course of eleven years and has few side effects. To this conclusion came a team of authors headed by Prof. Andreas Hochhaus from the University Hospital Jena in a new evaluation of a study that has been published in the New England Journal of Medicine. The so-called IRIS study was started in 2000 to compare the efficacy of a new drug, the tyrosine kinase inhibitor Imatinib, with the previous standard therapy, the immune stimulator Interferon alpha. The new active ingredient Imatinib selectively inhibits the activity of the altered protein, which triggers the continued division in the leukemia cells. The therapeutic response and tolerability of Imatinib were so good that the majority of patients in the interferon group eventually switched to this therapy and Imatinib became the standard drug for the treatment of CML.


The follow-up of study participants now provides valuable data for long-term therapy. Dr. Andreas Hochhaus, the Head of Internal Medicine the Department of the University Hospital Jena says: "The drug is also effective in long-term use so that patients live for ten years and longer without major CML symptoms. The IRIS study has also shown that no critical side effects accumulate or increase during this time".


Today, 83 percent of CML patients also live ten years after diagnosis. This is close to the survival rates of the normal population. The effectiveness of CML therapy is reflected in the decline in the proportion of remaining genetically modified blood cells. For their determination, a quantitative molecular genetic analysis method has been developed for the IRIS study, which is now being used by default for follow-up in CML. If the proportion of white blood cells with the characteristic mutation falls below a threshold, the disease is considered to be completely suppressed.


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