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New therapies and gene target advance the treatment and understanding of hard-to-treat leukemias
Date:12/6/2010

cent (43 patients) remaining on therapy and 36 percent (24 patients) discontinuing therapy. Of 32 evaluable patients with CML in chronic phase, 30 patients (94 percent) had a complete hematologic response (meaning blood cell counts are in the normal range), and 20 patients (63 percent) had a major cytogenetic response (meaning that no cells containing the Philadelphia chromosome were detected in the bone marrow). Of those achieving a major cytogenetic response, 12 patients had a complete cytogenetic response and eight patients had a partial cytogenetic response. Eighteen of these patients remained on ponatinib for a mean duration of 326 days without progression (range 142 to 599 days) with 13 of these patients having a confirmed response at a second assessment.

Of 11 evaluable patients with CML in chronic phase with a T315I mutation, 11 patients (100 percent) achieved a complete hematologic response and nine patients (82 percent) had a major cytogenetic response (eight of these patients had a complete cytogenetic response). Of 16 evaluable patients with either CML in accelerated or blast phase or with Ph+ ALL, five patients (31 percent) had a major hematologic response, three patients (19 percent) had a major cytogenetic response, and one patient (6 percent) had a minor cytogenetic response. Of nine evaluable patients with CML in accelerated or blast phase or patients with Ph+ ALL with a T315I mutation, three patients (33 percent) had a major hematologic response, and two patients (20 percent) had a major cytogenetic response.

Responses also were seen in heavily refractory patients with no mutations as well as in patients with other mutations who were resistant to currently approved tyrosine kinase inhibitors. For example, there was one complete cytogenetic response and one partial cytogenetic response in two patients with an F317L mutation who had previously failed therapy with imatinib, dasatinib, and nilotinib. Another patient with an F359C muta
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Contact: Lindsey Love
llove@hematology.org
540-538-9326
American Society of Hematology
Source:Eurekalert

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