Imatinib is the most effective and practical treatment for CML .

There is only one rational potential cure for CML, one of the several TKI's like imatinib (1st generation). If the

If a complete molecular ablation of the bcr/abl product is achieved, half of these patients will remain disease free.

Hi Antonio,

The Tyrosine Kinase Inhibitors (TKI) are effective in the clinical approach, patients achieve major hematological, cytogenetic and molecular responses with great clinical responses.

Despite the clinical improvement, Hematopoietic Stem Cell Transplantation is the unique curative approach for CML patients. Recently, some protocols are investigating the opportunity to discontinue TKI in patients in deep molecular response (>4.5 log), we are looking forward to these results.

Best regards,

Juan.

Perhaps now on a desk top instead of a phone which restricted the amount I could write, I'll have a chance to answer fully. While allogenic transplant is a curative approach for CML, this has become rare. The reason being that these diseases are almost always sensitive to TKI drugs and if no bcr/abl mutation develops that may be resistant to the available drugs (and some are specific for specific mutations) the large majority of patients can be put into a deep molecular remission. After sometime (a few years) in remission, many have been taken off TKI's and about 50% have been seen to remain in remission, i.e., cure. For those who show a recurrence of bcr/abl, the TKI is re-started with no apparent progression of the recurrent disease.

For the few cases where there is progressive disease on TKI, allogenic transplant remains an option but at considerable morbidity and mortality.

Thank you Abdul, Jason and Juan for your answers. Best of luck in your field of research :)

Allogeneic transplant is no longer the treatment of choice in eligible patients (young patients w/o comorbidities) over about the last 20 years, since the introduction of bcr-abl TKIs; imatinib, dasatinib, nilotinib, ponatinib, etc. As stated earlier by the other colleagues, these drugs induce molecular responses, i.e. 3.0-4.5 log reduction of bcr-abl transcripts in an appreciable % of patients. In the case that resistance develops, in particular the T315I mutation that is TKI resistant, then allogeneic HCT is indicated...!!!

Treatment Development of Chronic Myeloid Leukemia

Article Treatment Development of Chronic Myeloid Leukemia

TARGET THERAPY AND MONITORING OF CHRONIC MYELOID

LEUKEMIA

Treatment of chronic myeloid leukemia (CML) with target therapy

approved 5 different tyrosine kinase inhibitors (TKIs; imatinib,

nilotinib, dasatinib, bosutinib and ponatinib) according to the disease

risk, disease stage, comorbidities and BCR-ABL genotype. Imatinib

was the most common drug of choice for treatment of newly diagnosed

CML patients in the last 15 years. In the last 3 years nilotinib, dasatinib

and bosutinib, three newer drugs with higher potency against BCR-ABL and against imatinib resistant BCR-ABL mutations.

Ponatinib is the newest TKI indicated for chronic phase, accelerated phase, or blast ph ase chronic myeloid leukemia (CML) who are resistant or intolerant to dasatinib, nilotinib or bosutinib. Allogenic stem cell transplantation

is a curative treatment for patients with CML, but the excellent curative remission with TKI therapy have challenged the role of

allogeneic stem cell transplantation as a first line therapy.

(8) (PDF) TARGET THERAPY AND MONITORING OF CHRONIC MYELOID LEUKEMIA. Available from: https://www.researchgate.net/publication/281405796_TARGET_THERAPY_AND_MONITORING_OF_CHRONIC_MYELOID_LEUKEMIA [accessed Nov 21 2019].