Rare blood cancers, the Italian experience with target drugs

A rare disease, difficult to recognize, for which specific therapy was not available until last year. Today, things have changed: in recent months, Italy was the second country in Europe to make the new therapy available, after Germany, after AIFA approved the reimbursement of tagraxofusp as monotherapy for the first-line treatment of affected patients from this neoplasm. The Italian experience of IRST in Meldola was presented in Paris during the Congress of the European Bone Marrow Transplantation.

The illness

Blast plasmacytoid dendritic cell neoplasm (BPDCN) is a rare and aggressive blood cancer with a severe prognosis characterized by overexpression of the CD123 antigen, affecting 75% of males. “It is a pathology that is difficult to diagnose early precisely because it presents a complex symptomatology to interpret. It is a systemic pathology, which often presents itself at the onset with multiple and infiltrating dark-coloured skin lesions and in its course it can affect the bone marrow, the central nervous system and the lymph nodes”, explained Claudio Cerchione, researcher at the IRCCS “Dino Amadori” of Meldola and president of SOHO Italy (Society of Hematologic Oncology Italy), who in his speech in Paris underlined the importance of the multidisciplinary team and early diagnosis which can only take place in reference centres, such as Meldola.

Before the new therapy

Until the approval of tagraxofusp, there were no authorized drugs and in clinical practice intensive chemotherapy regimens normally used for the treatment of leukemia and lymphoma were used. The tumor is characterized by an intrinsic resistance to standard chemotherapy therefore the responses were often transient and the results obtained did not translate into advantages from the point of view of survival. In addition, these were therapies associated with serious toxicities, particularly heavy especially in elderly patients.

The new drug

Tagraxofusp has been available since last month, a drug that acts selectively and can lead to complete remission, significantly improving tolerability but at the same time increasing the number of patients who can be referred for allogeneic bone marrow transplantation. “In Meldola we follow many patients affected by this neoplasm (the team is composed, in addition to Cerchione, by Giovanni Marconi, Maria Benedetta Giannini, Marianna Norata, Irene Zacheo, by the Head of the Hematology Division Gerardo Mmisuraca and by the Scientific Director Giovanni Martinelli , Editor’s note), and it is important to explain to patients and caregivers the usefulness of reference centres, where it is possible to arrive at a diagnosis as early and precise as possible, which allows for the best treatment to be started in a short time”, underlines Cerchione. “These rare haematological pathologies, in fact, must see the involvement of several figures, the haematologist in the first place, the anatomo-pathologist, the dermatologist, the molecular biologist, in order to be able to offer the patient the best management”.