Tasigna is a kinase inhibitor indicated for the treatment of:...Adult and pediatric patients greater than or equal to 1 year of age with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase...Pediatric patients greater than or equal to 1 year of age with Ph+ CML-CP and CML-AP resistant or intolerant to prior tyrosine-kinase inhibitor (TKI) therapy.

Tasigna is indicated for the treatment of:...adult and paediatric patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukaemia (CML) in the chronic phase…

CONTRADICTING EVIDENCE:...pts diagnosed with Ph+ CML-CP who were initiated on a 2G TKI (dasatinib, nilotinib, or bosutinib) as 2L therapy...In 2L TKI therapy, 41% of pts received nilotinib, 19% dasatinib, and 40% bosutinib...Although 2G TKIs have higher efficacy and lower progression rates in 1L vs 1G TKI, a substantial proportion of pts still do not achieve the important treatment response milestones; almost 50% did not achieve MMR within 12 months of 2L TKI therapy.

Patients consistently in MR4.5 during consolidation had higher TFR rates (50.6%) than patients with ≥ 1 assessment without MR4.5 during consolidation (35.0%). In a landmark analysis, 96-week TFR rates for patients with MR4.5, MR4 (BCR-ABL1IS ≤ 0.01%) but not MR4.5, and MMR but not MR4 at TFR week 12 were 82.6, 23.1, and 0%, respectively. There were no reports of disease progression or death due to CML; overall adverse event frequency decreased following TFR. Within the follow-up period, TFR did not adversely affect disease outcomes.

...Cumulative rate of BCR-ABL1 fusion transcripts ≤ 0.0032%, measured by real-time quantitative polymerase chain reaction and standardized to the international scale`Cumulative rate of molecular response 4.5 by 24 months...

The safety profile of nilotinib was consistent with those of earlier reports. No on-treatment deaths occurred. These long-term (up to ∼5 years) data support the efficacy and safety of nilotinib in pediatric patients with Ph+ CML-CP.

We conducted a retrospective study including patients treated for CML between 2003 and 2021….BCR-ABL1 transcript was detected at diagnosis by Reverse Transcriptase-Polymerase Chain Reaction....Second line therapy with second generation TKIs (Dasatinib or Nilotinib) was required in 35% of cases due to the treatment failure in 32% and treatment related toxicity in 3% of cases. DMR was reported in 78% of cases....Our study showed that TKIs first and second generation are highly effective in the treatment of CML, achieving complete hematological response and high rates of molecular response and leading to good long-term outcomes for patients.

BCR-ABL-positive CML-CP patients with ≥3 years of frontline NIL...At 12 months, 37/51 patients remained in MR4.0 (BCR-ABL1IS ≤0.01%) (72.6%; 95% CI:58.3%–84.1%); 14 patients (27.5%) lost MR4.0, and 31/51 patients remained in MR4.5 (60.8%; 95% CI:46.1%–74.2%); 20 patients (39.2%) lost MR4.5. No patient experienced disease progression...

Data from 63 patients treated with TKIs were collected (first-line imatinib [n = 27], nilotinib [n = 27], dasatinib [n = 8], or ponatinib [n = 1]). A higher percentage of patients in the nilotinib and dasatinib groups versus the imatinib group reached a deep molecular response (BCR-ABL1IS ≤ 0.01%) at 12 months (32% and 25%, respectively, vs. 23%) and 18 months (42% and 38%, respectively, vs 27%).

At three centers, from May 25, 2004, to May 4, 2005, we enrolled 119 patients whose disease was resistant to imatinib... As of June 15, 2005, 66 patients remained in the study, including all 17 patients with chronic-phase CML….Among 17 patients with the chronic phase of disease, the median duration of therapy was 4.9 months...and all of the patients have continued therapy. At the present time, 11 of 12 patients with active disease have had a complete hematologic remission. There were cytogenetic responses in 9 of 17 patients who could be evaluated, including 6 responses that were complete... All trough levels exceeded the 50 percent inhibitory concentration of cellular phosphorylation of BCR-ABL (20 to 57 nM, depending on cell type) and 32 of 33 BCR-ABL kinase mutants (19 to 709 nM)…nilotinib has a relatively favorable safety profile, and preliminary results obtained with a relatively short follow-up period indicate that the drug is active in CML.

...nilotinib demonstrated efficacy and a manageable safety profile in pediatric patients with Ph+ CML-CP.

A 58-year-old female...was diagnosed as CML based on blood film, bone marrow aspiration, and biopsy results and was confirmed by the detection of Philadelphia chromosome P210 in 46% of cells….She was shifted to nilotinib with the absence of the previous toxicities and QPCR reduction....PET- CT was negative after four cycles, and QPCR remained undetectable. Therefore, the patient is continuing nilotinib with regular monthly follow-up for lymph node ultrasonography.

A 40-year-old male patient was diagnosed with BCR-ABL1 positive chronic stage CML 2 years ago and achieved complete molecular response on nilotinib...