MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. A patient was counted as having MMR at 6 cycles if the patient met the MMR criteria at the Cycle 6 Visit.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. A patient was counted as having MMR by 12 cycles if the patient met the MMR criteria at least once at any time between first study drug intake and Cycle 12 visit included.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as CCyR at 12 cycles if the patient met the CCyR criteria at the Cycle 12 Visit.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Major molecular response (MMR) was defined as BCR-ABL/ABL % ≤ 0.1% by IS as measured by RQ-PCR, confirmed by duplicate analysis of the same sample.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Major molecular response (MMR) was defined as BCR-ABL/ABL % ≤ 0.1% by IS as measured by RQ-PCR, confirmed by duplicate analysis of the same sample.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. BCR-ABL ratio by percentage: > 0.0032 to ≤ 0.01% is equal to a log reduction category of >= 4 to <4.5 -log reduction (MR4); BCR-ABL ratio by percentage: <=0.0032% is equal to a log reduction category of >= 4.5-log reduction (MMR4.5)

Full Analysis Set (FAS): All patients who received at least one dose of study medication

MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR (Real time quantitative polymerase chain reaction). BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. BCR-ABL ratio by percentage: > 0.0032 to ≤ 0.01% is equal to a log reduction category of >= 4 to <4.5 -log reduction (MR4); BCR-ABL ratio by percentage: <=0.0032% is equal to a log reduction category of >= 4.5-log reduction (MMR4.5)

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Time from first study drug intake to first MMR amongst imatinib or dasatinib resistant or intolerant patients with CML-CP computed only for patients who achieved MMR.

Full Analysis Set (FAS): All patients who received at least one dose of study medication.

Time to MMR is the time from first study drug intake to first major molecular response computed only for participants who achieved MMR.

Full Analysis Set (FAS): All patients who received at least one dose of study medication.

Duration of MMR is defined as the time between the date of the first MMR and the date of confirmed loss of MMR (i.e. the earliest of confirmed loss of MMR, CML-related death or progression to AP or BC). Participants without loss of MMR were censored at the last molecular assessment date.

Full Analysis Set (FAS): All patients who received at least one dose of study medication.

Duration of MMR is defined as the time between the date of the first MMR and the date of confirmed loss of MMR (i.e. the earliest of confirmed loss of MMR, CML-related death or progression to AP or BC). Participants without loss of MMR were censored at the last molecular assessment date.

Full Analysis Set (FAS): All patients who received at least one dose of study medication.

• Complete cytogenetic response (CCyR) - 0% Ph+ metaphases
• Partial cytogenetic response (PCyR) - >0 to 35% Ph+ metaphases
• Minor cytogenetic response (mCyR) - >35 to 65% Ph+ metaphases
• Minimal - >65 to 95% Ph+ metaphases
• None - >95 to 100% Ph+ metaphases
• Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.

Full Analysis Set (FAS): All patients who received at least one dose of study medication.

Complete cytogenetic response (CCyR) - 0% Ph+ metaphases No response - >95 to 100% Ph+ metaphases

Full Analysis Set (FAS): All patients who received at least one dose of study medication.

Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as having CCyR by 6 cycles (respectively 12 cycles) if the patient met the CCyR criteria at least once at any time between first study drug intake and cycle 6 (cycle 12 respectively) visit included.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as having CCyR by 6 cycles (respectively 12 cycles) if the patient met the CCyR criteria at least once at any time between first study drug intake and cycle 6 (cycle 12 respectively) visit included.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Cytogenetic response is assessed as the percentage of Philadelphia positive (Ph+) metaphases in the bone marrow. Complete Cytogenetic Response (CCyR) is defined as 0% of Ph+ metaphases. A patient was counted as having CCyR by 6 cycles (respectively 12 cycles) if the patient met the CCyR criteria at least once at any time between first study drug intake and cycle 6 (cycle 12 respectively) visit included.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Major cytogenetic response (MCyR) - 0 to 35% Ph+ metaphases. A major response combines both complete and partial responses.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Complete Hematological Response (CHR) was defined as

• WBC count <10×109/L
• platelet count <450×109/L
• basophils <5%
• no blasts and promyelocytes in peripheral blood
• myelocytes+metamyelocytes <5% in peripheral blood
• no evidence of extramedullary disease, including spleen and liver
• Assessment confirmation after at least 4 weeks for newly diagnosed Ph+ CML-CP

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Complete Hematological Response (CHR) was defined as

• WBC count <10×109/L
• platelet count <450×109/L
• basophils <5%
• no blasts and promyelocytes in peripheral blood
• myelocytes+metamyelocytes <5% in peripheral blood
• no evidence of extramedullary disease, including spleen and liver
• Assessment confirmation after at least 4 weeks for newly diagnosed Ph+ CML-CP

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Complete Hematological Response (CHR) was defined as

• WBC count <10×109/L
• platelet count <450×109/L
• basophils <5%
• no blasts and promyelocytes in peripheral blood
• myelocytes+metamyelocytes <5% in peripheral blood
• no evidence of extramedullary disease, including spleen and liver
• Assessment confirmation after at least 4 weeks for newly diagnosed Ph+ CML-CP

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Time to disease progression is the time from the date of first study drug intake to the date of event defined as the first progression to AP or BC (from CP) or to BC (from AP) or the date of CML-related death occurring on treatment, whichever was earlier.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Event Free Survival is defined as the time from the date of first study drug intake to the first occurrence of any of the following loss of CHR, loss of MCyR ( PCyR + CCyR), progression to AP/BC (from CP) or to BC (from AP), or death from any cause. (Including events only during treatment)

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Event Free Survival is defined as the time from the date of first study drug intake to the first occurrence of any of the following loss of CHR, loss of MCyR ( PCyR + CCyR), progression to AP/BC (from CP) or to BC (from AP), or death from any cause. (Including events only during treatment)

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Overall survival is defined as the time from the date of first study drug intake to the date of death due to any cause. If a patient is not known to have died, survival will be censored at the date of their last assessment for patients on study and date of last contact for patients in follow-up.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

Overall survival is defined as the time from the date of first study drug intake to the date of death due to any cause. If a patient is not known to have died, survival will be censored at the date of their last assessment for patients on study and date of last contact for patients in follow-up.

Full Analysis Set (FAS): All patients who received at least one dose of study medication

BCR-ABL is the fusion gene from breakpoint cluster region and Abelson genes. BCR-ABL transcript levels were summarized by cohort and time point. MMR is defined as ≤ 0.1% BCR-ABL/control gene (ABL) % by international scale, or equivalent to ≥ 3 log reduction of BCR-ABL transcript from standardized baseline, measured by RQ-PCR.

Full Analysis Set (FAS): All patients who received at least one dose of study medication.

PK was analyzed only when all patients has completed 12 cycles on treatment or discontinued the study treatment early.

Pharmacokinetics Analysis Set (PAS): All patients with at least one evaluable PK blood sample. Of the 32 patients included in the PAS, only 30 had evaluable Ctrough.

PK was analyzed only when all patients has completed 12 cycles of treatment or discontinued the study treatment early.

Pharmacokinetics Analysis Set (PAS): All patients with at least one evaluable PK blood sample.

To assess long term effect on growth, development and maturation of nilotinib treatment in pediatric patients with Ph+ CML in participants with both a baseline and post-baseline value.

Safety Set (SAF): All patients who received at least one dose of study medication in participants with both a baseline and post-baseline value.

Acceptability of the study drug was evaluated from a questionnaire completed by patients, with the help from parents or caregivers at visits.

The Questionnaire to capture patient assessment of palatability (very good to very bad) and acceptability of taking the medication (very easy to very hard to administration).

Safety Set (SAF): All patients who received at least one dose of study medication.

Emerging signs of resistance to nilotinib

Full Analysis Set (FAS): All patients who received at least one dose of study medication

The summary of bone age and Dual-energy X-ray absorptiometry (DEXA) by cohort. Alteration of bone biochemical markers of hand and wrist X-Ray evaluation was observed in bone age standard deviation scores (SDS) and for bone mineral density for DEXA before and after treatment with nilotinib.

Safety Set (SAF): All patients who received at least one dose of study medication.

On treatment deaths were collected from FPFT up to 30 days after study drug discontinuation, for a maximum duration of 64.5 months (treatment duration ranged from 0.7 to 63.5 months).

Deaths post treatment survival follow up were collected after the on- treatment period, up to approx. 7 years.

Clinical Database Population: All treated participants.