Chronic Myeloid Leukemia: A Handbook for Hematologists and Oncologists PDF

Preface

The past three decades have witnessed chronic myeloid leukemia (CML), a clonal disease that results from an acquired genetic change in a single pluripotential hemopoietic stem cell, to have been served remarkably well by advancements in cellular and molecular biology. The speed at which these findings were translated to one of the great cancer medicine success stories of the past three decades is remarkable. Although CML is a rare disease, the lessons learned have led to a major paradigm shift in cancer medicine in general. For CML patients, the introduction of the tyrosine kinase inhibitor, imatinib mesylate, in 1998 was an important therapeutic milestone with most patients achieving a complete cytogenetic response and prolongation of survival compared with the previous therapies, other than stem cell transplantation. With the more recent regulatory approval in USA and many other countries of the second generation of tyrosine kinase inhibitors, dasatinib and nilotinib for first-line therapy of CML in chronic phase, and other candidate drugs in clinical trials, the pace at which the treatment algorithm for patients with CML is changing is unprecedented. There is however some uncertainty with regard to how best to assess efficacy of these and other potential next-generation drugs, such as ponatinib, in particular with regards to the most appropriate surrogate markers for overall survival. Currently the first-line studies of nilotinib use molecular markers and dasatinib use cytogenetic responses. There are also important differences in the precise definitions of common endpoints, such as event-free survival, progression-free survival and others.

In this inaugural edition of the CML handbook, I aim to provide important preclinical and clinical aspects of CML, for hematologists, oncologists and other health professionals interested in the disease. The opinions expressed are mine and I apologize for any errors or omissions.