According to a story from PR Newswire, presentations at the 60th annual American Society of Hematology Meeting and Exposition are highlighting the results of four large scale clinical trials that demonstrate meaningful progress in the treatment of blood disorders and blood cancer. These trials are poised to cause major changes in the treatment of these rare diseases.
Diffuse Large B-Cell Lymphoma
One study focuses on the use of chemotherapy as a treatment for diffuse large B-cell lymphoma. This is a form of blood cancer that affects white blood cells and has a tendency to progress rapidly. This trial monitored patients for a median of five years; some patients were monitored in the study for 11 years. Currently, it is common for patients with diffuse large B-cell lymphoma to receive six cycles of chemotherapy in combination with rituximab, a monoclonal antibody. However, the study found that four cycles of chemo was just as effective as six. Considering that chemotherapy can have severe and unpleasant side effects, the news that cycles can be reduced without sacrificing effectiveness is welcome news for patients. A reduced number of cycles means that quality of life will improve and patients will be able to return to their normal routines after treatment more quickly.
Chronic Lymphocytic Leukemia
In another trial, the targeted therapy ibrutinib was shown to be more effective in treating chronic lymphocytic leukemia than the current treatment standard, at least in older patients. Chronic lymphocytic leukemia is another cancer of the white blood cells which tends to appear in old age. Although ibrutinib was approved as a treatment for this disease in 2016, it is common for older patients to receive a combination of chemotherapy and rituximab instead. However, the researchers found that ibrutinib was able to reduce disease progression more effectively in this group of patients. Progression free survival with ibrutinib and combined chemo and rituximab at two years was 87 percent and 74 percent respectively.
Luspatercept in Myelodysplastic Syndromes and Beta Thalassemia
There were also two separate studies of the investigational drug luspatercept as a treatment for the rare blood disorder beta thalassemia and myelodysplastic syndromes. Beta thalassemia is characterized by reduced production of hemoglobin, which is vital to the transportation of oxygen on red blood cells. In the study, 70 percent of patients with the disorder were able to reduce blood transfusions by a third on average over a 12 week period. When the drug was tested against myelodysplastic syndromes, a cancer affecting the bone marrow, 53 percent of patients were able to reduce the frequency of their blood transfusions.
These findings indicate substantial advancements in the treatment of these rare diseases.