SAN FRANCISCO—Nilotinib in combination with chemotherapy is highly effective and well tolerated as the first-line treatment of elderly patients with newly diagnosed Philadelphia chromosome (Ph)-positive and/or BCR-ALB1-positive acute lymphocytic leukemia (ALL), a European Working Group study (Abstract 798) presented at the 56th American Society of Hematology (ASH) Annual Meeting and Exposition has shown.

As of October 2014, researchers had enrolled 56 patients with a median age of 65 years with Ph+ and/or BCR-ABL1-positive ALL. Patients first received dexamethasone on days -7 to -3 with or without cyclophosphamide IV on days -3 to -1 in a pre-phase.

Then, all patients received nilotinib 400 mg twice daily beginning during induction and continuing thereafter, in addition to vincristine and dexamethasone on 2 days, weekly for 4 weeks. The consolidation phase consisted of nilotinib twice daily, methotrexate on day 1, and asparaginase on day 2 for cycles 1, 3, and 5, and cytarabine on days 1, 3, and 5 for cycles 2, 4, and 6.

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Patients then received nilotinib twice daily, 6-mercaptopurine daily, and methotrexate once weekly for 4 weeks every other month, and dexamethasone and vincristine in 2-month intervals for up to 2 years.

 Of those enrolled, 47 were evaluable for efficacy by the time of this analysis. Patients were followed for a median of 8.5 months. The complete hematologic response rate following treatment was 87% with a median time to complete response of 41 days. In addition, 4% of patients experienced a partial response or failure, 6% discontinued treatment before assessment, and 2% died.

“Looking at those patients who had achieved a complete response, 83% are still in ongoing remission,” said Oliver G. Ottmann, MD, of Goethe University in Frankfurt, Germany, while presenting at the meeting. At 30 months, the overall survival rates were 72.7% for patients who did not undergo stem cell transplantation and 67.1% for all patients (P=0.8).

“There was no significant difference between these groups, and I would like to note the short follow-up in these patients,” Dr. Ottmann noted. “The overall survival estimate, though preliminary, is very encouraging.”

In regard to safety, “there were quite a number of adverse effects,” Dr. Ottmann said. Cytopenias, infections, and febrile neutropenia were among the most common.


  1. Ottmann, Oliver G., MD, et al. “798 Nilotinib (Tasigna®) and Chemotherapy for First-Line Treatment in Elderly Patients with De Novo Philadelphia Chromosome/BCR-ABL1 Positive Acute Lymphoblastic Leukemia (ALL): A Trial of the European Working Group for Adult ALL (EWALL-PH-02).” ASH 2014. Oral Presentation. December 9, 2014.