Chronic Myeloid Leukemia

According to updated data from the phase II ELIANA study, CD19-targeted CAR T-cell therapy tisagenlecleucel as treatment of pediatric and young adult patients with relapsed or refractory acute lymphoblastic leukemia sustained rates of relapse-free survival and overall survival at 24 and 18 months. 

CLL is a malignant disease characterized by progressive accumulation of immature, immunophenotypically distinct lymphocytes in the blood, bone marrow, and lymphatic tissues. Although the clinical course of CLL can be unpredictable, this disease is typically slow-progressing and occurs most often in patients ≥65 years of age. As such, treatment, if indicated, is typically conservative. Current CLL treatments are not considered to be curative; however, long-term remission can be achieved in some patients using allogenic stem cell transplantation.

Chronic lymphocytic leukemia is characterized by the clonal proliferation and accumulation of small, mature-appearing CD5-positive B lymphocytes in the blood, bone marrow, and secondary lymphoid tissues. A CLL diagnosis is established by the presence of more than 5x109/L peripheral lymphocytes co-expressing CD5, CD19, and CD23, and weakly expressing CD20, CD79b, and surface immunoglobulin. Small lymphocytic lymphoma represents a clinical variant of CLL and is similarly managed.

Dasatinib has been granted approval by the European Commission as a treatment for children and adolescent patients aged 1 to 18 years with Philadelphia chromosome–positive chronic myeloid leukemia in chronic phase, according to Bristol-Myers Squibb, the manufacturer of the tyrosine kinase inhibitor.

According to results presented at the 2018 ASCO Annual Meeting, moxetumomab pasudotox, a first-in-class recombinant immunotoxin targeting CD22, demonstrated deep and durable responses in a substantial proportion of pretreated patients with relapsed/refractory hairy cell leukemia.

The FDA approved several indications throughout the month of April 2018. A number of drugs were granted priority review and Fast Track designation. The FDA also halted all clinical trials using tazemetostat as treatment, and new initiatives were introduced to help ease the development of genetic and genomic-based tests. Check out our list of all FDA happenings from April 2018.

Based on data from 2 clinical trials, nilotinib (Tasigna) has been approved by the FDA for the first- and second-line treatment of pediatric patients aged 1 year and older with Philadelphia chromosome–positive chronic myeloid leukemia in the chronic phase.