According to a story from Specialty Pharmacy Times, data from a Phase II clinical trial has recently been released which suggests that the drug tagraxofsup is a useful treatment in a rare blood cancer called blastic plasmacytoid dendritic cell neoplasm (BPDCN). This rare neoplasm is commonly regarded as aggressive. Tagraxofsup is the only therapy approved by the US Food and Drug Administration (FDA) for its treatment. The drug was first approved in December of 2018.
About Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
Blastic plasmacytoid dendritic cell neoplasm (BPDCN) is a rare type of blood cancer. This an aggressive cancer with features similar to leukemia and cutaneous lymphoma. It can occur in children but it most often appears in older adults. The disease is associated with myelodysplastic syndromes and can also evolve into acute myeloid leukemia. The cause of BPDCN is unknown. Symptoms of this blood cancer include skin lesions on the head and torso, enlarged lymph nodes and/or other organs, anemia, leukopenia, and thrombocytopenia. BPDCN often initially responds well to treatment with chemo regimens used for other blood cancers, but remission is brief and the disease relapses with greater resistance. Stem cell transplant combined with chemo can be curative in some cases. The five year survival rate for BPDCN is very low. More effective treatment options are direly needed. To learn more about BPDCN, click here.
About The Study
The clinical trial included a total of 47 BPDCN patients. Some of them had not received any treatment and other had already relapsed. A total of 32 patients were receiving tagraxofsup in the study as a first-line treatment; 15 patients had already received other therapies. Among the previously untreated group, the overall response rate reached 90 percent. The patients that had been previously treated saw an overall response of 67 percent. Median survival rate was 8.5 months for this group. In the previously untreated group, survival rates were 59 percent and 18 months and 52 percent at 24 months.
Using tagraxofsup can lead to serious adverse effects including capillary leak syndrome, which appeared in 19 percent of patients. These results indicate that this therapy has the potential to prolong survival compared to other approaches.
Check out the original study here.