The pharmaceutical arsenal for the treatment of patients with leukemias or myelodysplastic syndromes (MDS) currently in the pipeline continues to grow, with some drugs showing particularly promising results. Updates on many studies for drugs that are currently in development were presented at ASH 2013.
New Orleans, LA—The role of anti-CD20 antibodies in unfit patients with chronic lymphocytic leukemia (CLL) was discussed in several presentations at ASH 2013.
New Orleans, LA—A planned interim analysis of the first phase 3 clinical trial shows that idelalisib, a first-in-class selective oral kinase inhibitor, when combined with rituximab (Rituxan), is superior to rituximab alone in progression-free survival (PFS) in patients with heavily pretreated chronic lymphocytic leukemia (CLL).
New Orleans, LA—Three large randomized phase 3 trials demonstrate the superiority of nilotinib (Tasigna) over imatinib (Gleevec) in achieving molecular response (MR) and complete cytogenetic response (CCyR) across various populations of patients with Philadelphia chromosome–positive (Ph+) chronic myeloid leukemia (CML), including patients who have a suboptimal response to frontline imatinib.

A new selective inhibitor of the FLT3 gene, quizartinib, produced complete remission in more than 33% of patients with an aggressive form of acute myeloid leukemia (AML). The treatment allowed these patients to bridge to potential­ly curative allogeneic stem-cell transplant, said principal investigator, Mark Levis, MD, PhD, Associate Professor of Oncology and Medicine, Johns Hopkins Kimmel Can­­­cer Center, Baltimore, MD, at ASH 2012.

Atlanta, GA—The new oral tyrosine kinase inhibitor (TKI) ponatinib (Iclusig) has significant activity and is well tolerated in patients with highly pretreated chronic myeloid leukemia (CML) or with Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL) who otherwise lack therapeutic options. Twelve-month follow-up data from a pivotal phase 2 trial of ponatinib in this population were reported by Jorge E. Cortes, MD, Professor of Medicine and Deputy Chair, Department of Leukemia, at M.D. Anderson Cancer Center, Houston, at the 2012 ASH meeting.

Atlanta, GA—Targeting Bruton’s tyrosine kinase (BTK) in patients with chronic lymphocytic leukemia (CLL) resulted in high response rates, without severe toxicity, according to the results of 2 studies that were presented at the 2012 ASH meeting.

Data from one trial showed a 70% response rate in 116 patients treated with ibrutinib and a 22-month survival of 96% in previously untreated patients and 76% in the subgroup with relapsed/refractory CLL.

An early major molecular re­sponse (MMR) identifies pa­tients with chronic myeloid leukemia (CML) who are more likely to achieve undetectable BCR-ABL1 transcripts while receiving imatinib therapy, and represent candidates for entry into studies of discontinuing therapy, said Susan Branford, PhD, Department of Genetics and Molecular Pathology, University of Adelaide School of Medicine, Australia.

The combination of the nucleoside analog cytarabine (Cytosar-U) and the anthracycline daunorubicin (Daunoxome), the so-called “7 + 3” schedule, has been the standard of care for the treatment of acute myeloid leukemia (AML) for several years. Recent data suggest that better induction options exist, especially for younger patients, said Alan K. Burnett, MD, Head of Hematology, Department of Medical Genetics, Hematology and Pathology, Cardiff University, Heath Park, United Kingdom. He challenged the current 7 + 3 paradigm during the Ham-Wasserman Lecture delivered at ASH 2012.

The initial choice of therapy for patients with chronic myeloid leukemia (CML) is probably not important, because differences in efficacy are not dramatic. What matters is that response is assessed early, and that side effects are managed swiftly, said David Marin, MD, Hammersmith Hospital, Imperial College London, United Kingdom.

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