Few examples in clinical medicine demonstrate such a fascinating interaction as chronic myeloid leukemia (CML) and hematopoietic stem cell transplantation (HSCT). CML did provide first proof of principle that an incurable disease was amenable to successful treatment, that immune mediated mechanism could control a malignancy and that the risk of HSCT could be quantified by a few key factors. HSCT was nearly abandoned at the introduction of targeted therapy with imatinib. Today, it has a clear position and exemplifies the modern risk adapted approach.
Since early 2002, the Glivec International Patient Assistance Program (GIPAP) has provided a bridge to Glivec access for CML patients from 80 countries in Latin America, Africa, Asia Pacific and Eastern Europe. Developed and sponsored by Novartis and launched just a few months after the drug received FDA approval, the program has provided unprecedented access to state of the art treatment in so-called developing countries almost simultaneously with the Western World. As a result, for the past eight years more than 1,000 hematologists and 30,000 CML patients in developing countries have been able to benefit from access to Glivec treatment.
How close are we to achieving the ultimate goal of global standardization
for BCR-ABL measurement by RQ-PCR in CML?
Over recent years various studies have demonstrated that serial analysis of BCR-ABL mRNA levels by real-time quantitative PCR (RQ-PCR) accurately reflects the level of leukemic inhibition induced by therapy and provides an effective monitoring strategy for patients with CML.