cancersCancer

Chronic myeloid leukaemia (CML)

Chronic myeloid leukaemia is a blood cancer driven by a single fused gene, BCR-ABL. Imatinib in 2001 turned it from a disease with a median survival of about five years into one most people live with, and many can now stop treatment altogether.

CML is defined by the Philadelphia chromosome t(9;22) and its product, the constitutively active BCR-ABL1 tyrosine kinase. It is the paradigm of oncogene addiction: tyrosine kinase inhibitors (TKIs) restore near-normal life expectancy in chronic phase, and treatment response is tracked by quantitative BCR-ABL1 PCR on the International Scale (IS), with milestones at 3, 6 and 12 months (ELN 2020).

First-line options are imatinib, the second-generation TKIs dasatinib, nilotinib and bosutinib, and since 2024 asciminib (ASC4FIRST), the first allosteric STAMP inhibitor. Second-generation drugs achieve deeper responses faster but have not improved overall survival over imatinib; choice is driven by comorbidity (cardiovascular risk with nilotinib and ponatinib, pleural effusions with dasatinib) and by the goal of treatment-free remission (TFR). Resistance is largely through ABL1 kinase-domain mutations; T315I is covered by ponatinib and asciminib. Allogeneic transplant is reserved for blast phase or multi-TKI failure.

The frontier is TFR (about half of patients with sustained deep molecular response can stop, EURO-SKI), safer T315I coverage, olverembatinib in Asia, and the small residue of accelerated/blast-phase disease, where outcomes remain poor.

State of the art today

Asciminib is the first allosteric BCR-ABL1 inhibitor and, since ASC4FIRST (2024), a first-line option with better tolerability than second-generation TKIs.
Roughly half of eligible patients maintain remission off therapy; predictors are duration of deep response and prior interferon exposure.
Blast-phase CML remains the unsolved problem, and TKI cost is the barrier in low- and middle-income countries despite generic imatinib.

Show survival figures (1)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

Life expectancy in chronic phase on TKI is close to the general population; the goal has moved from survival to deep molecular response and treatment-free remission.

Who it affects

About 1-2 cases per 100,000 per year; because patients now live near-normal lifespans, prevalence keeps rising (SEER).

Group

haematologic

Where the cases are

Cases by country · GLOBOCAN 2022

All countries →

No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.

Standard of care

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Chronic phase, first line

Imatinib 400 mg, or a second-generation TKI (dasatinib, nilotinib, bosutinib), or asciminib (ASC4FIRST, approved 2024). Choice by comorbidity and treatment goal; monitor BCR-ABL1 IS at 3, 6, 12 months against ELN milestones.

NCCN · Category 1 (imatinib, dasatinib, nilotinib,…

ImatinibDasatinibNilotinibBosutinibAsciminib

Resistance or intolerance

Switch TKI guided by mutation analysis; ponatinib or asciminib for T315I; allogeneic HSCT if two or more TKIs fail or in advanced phase.

ELN 2020

PonatinibAsciminibAllogeneic stem cell transplantationBosutinib

Sustained deep molecular response

Treatment-free remission attempt after ≥3-5 years of TKI and ≥2 years of MR4 or better, with monthly PCR for the first six months (EURO-SKI); restart on loss of MMR.

ELN 2020; EURO-SKI (Lancet Oncol 2018)

Minimal / molecular residual disease (MRD)

Blast phase

TKI plus acute-leukaemia-type induction (ponatinib or dasatinib with chemotherapy), then allogeneic HSCT.

PonatinibDasatinibAllogeneic stem cell transplantation

Subtypes & biomarkers

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Subtypes

Chronic phase (~95% at diagnosis)
Accelerated phase
Blast phase (myeloid or lymphoid)
Atypical CML / BCR-ABL1-negative (separate MDS/MPN entity)

Biomarkers clinicians test

BCR-ABL1 by RT-qPCR on the International Scale (MMR = ≤0.1%, MR4.5 = ≤0.0032%)
ABL1 kinase-domain mutations (T315I, F317L, E255K/V, Y253H)
ELTS score at diagnosis
Additional cytogenetic abnormalities
Cardiovascular risk profile (drug selection)

Target prevalence in this cancer

History

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1960Philadelphia chromosome
Nowell and Hungerford describe a minute chromosome in CML: the first consistent chromosomal abnormality in a human cancer.
1973t(9;22) translocation
Janet Rowley shows the Philadelphia chromosome is a reciprocal translocation.
1990BCR-ABL causes CML in mice
Daley, Van Etten and Baltimore demonstrate the fusion kinase is sufficient to cause a CML-like disease.
1998Imatinib enters the clinic
Druker's phase 1 trial of STI571: complete haematologic responses in nearly every chronic-phase patient.
Imatinib
2001Imatinib approved
FDA approval in under three months; IRIS (2003) confirms superiority over interferon-cytarabine.
Imatinib
2006Second-generation TKIs
Dasatinib (2006) and nilotinib (2007) approved for resistance; later first line (DASISION, ENESTnd).
DasatinibNilotinib
2012Ponatinib covers T315I; bosutinib approved
PonatinibBosutinib
2018Treatment-free remission validated
EURO-SKI: about half of patients stopping TKI stay in remission.
Minimal / molecular residual disease (MRD)
2021Asciminib: allosteric STAMP inhibitor approved
ASCEMBL vs bosutinib in third line.
Asciminib
2024Asciminib first line
ASC4FIRST: higher MMR at 48 weeks than investigator-selected TKI; FDA accelerated approval October 2024.
Asciminib

Pipeline

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ApprovedSmall-molecule allosteric ABL1 inhibitor (STAMP)

Asciminib · Scemblix

Allogeneic stem cell transplantation

Open problems

Blast-phase CML: median survival still under a year.
Predicting who can stop TKI safely; second TFR attempts.
Cardiovascular toxicity of nilotinib and ponatinib.
Access to any TKI and to PCR monitoring in LMICs.

Trials

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Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov

Chronic myeloid leukaemia (CML)

condition: Chronic myeloid leukemia

Open on ClinicalTrials.gov →

within

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Expert centres

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Where the expertise is

Centres linked to this cancer in OnCo

GIMEMARome, IT
via Imatinib, Minimal / molecular residual disease (MRD)
OHSU Knight Cancer InstitutePortland, OR, USNCI comprehensive
via this cancer, Imatinib
Seoul St. Mary's HospitalSeoul, KR
via Imatinib, Nilotinib
American Society of HematologyWashington, DC, US
via Minimal / molecular residual disease (MRD)
Australasian Gastro-Intestinal Trials GroupSydney, AU
via Minimal / molecular residual disease (MRD)
Cancer Center Clínica Universidad de Navarra / CIMAPamplona, ES
via Minimal / molecular residual disease (MRD)
Central Drugs Standard Control OrganizationNew Delhi, IN
via Imatinib
European Hematology AssociationThe Hague, NL
via Minimal / molecular residual disease (MRD)
Fox Chase Cancer CenterPhiladelphia, PA, USNCI comprehensive
via this cancer
Fred & Pamela Buffett Cancer CenterOmaha, NE, USNCI clinical
via Allogeneic stem cell transplantation
Hospital Universitario 12 de OctubreMadrid, ES
via Minimal / molecular residual disease (MRD)
HOVONRotterdam, NL
via Minimal / molecular residual disease (MRD)
INCLIVA Biomedical Research Institute / Hospital Clínico Universitario de ValenciaValencia, ES
via Minimal / molecular residual disease (MRD)
Indiana University Melvin and Bren Simon Comprehensive Cancer CenterIndianapolis, IN, USNCI comprehensive
via Allogeneic stem cell transplantation
IUCT Oncopole – Institut Universitaire du Cancer de ToulouseToulouse, FR
via Minimal / molecular residual disease (MRD)
Juravinski Cancer Centre / Escarpment Cancer Research InstituteHamilton, ON, CA
via Allogeneic stem cell transplantation
Masonic Cancer Center, University of MinnesotaMinneapolis, MN, USNCI comprehensive
via Allogeneic stem cell transplantation
National Cancer Center Hospital EastKashiwa, Chiba, JP
via Minimal / molecular residual disease (MRD)
Sydney Children's Hospitals Network / Children's Cancer InstituteSydney, AU
via Minimal / molecular residual disease (MRD)
Sylvester Comprehensive Cancer Center, University of MiamiMiami, FL, USNCI clinical
via Minimal / molecular residual disease (MRD)
University of Chicago Medicine Comprehensive Cancer CenterChicago, IL, USNCI comprehensive
via this cancer
University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer CenterBaltimore, MD, USNCI comprehensive
via Allogeneic stem cell transplantation

Seeking a second opinion: ask your oncologist for a referral to a high-volume centre; most accept records and pathology by mail or telehealth. In the US, use the NCI's Find a Cancer Center tool or the nonprofit Cancer Commons, which navigates options for advanced cancers at no cost.

Questions to ask

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Bring to your appointment

Questions to ask your oncologist about Chronic myeloid leukaemia

Generated from this cancer's standard of care, biomarkers, and pipeline · 16 questions

Newly diagnosed

What is my exact diagnosis, stage, and grade, and which tests established them?
Why: Everything else follows from an accurate stage and subtype.
Which biomarkers have been tested on my tumour (for example BCR-ABL1 by RT-qPCR on the International Scale, ABL1 kinase-domain mutations, ELTS score at diagnosis, Additional cytogenetic abnormalities, Cardiovascular risk profile), and what were the results?
Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
Which subtype is my cancer, and does that change the recommended treatment?
Why: Recognised subtypes for this cancer include Chronic phase, Accelerated phase, Blast phase.
Is germline (inherited) genetic testing recommended for me or my family?
Why: Inherited variants can change treatment and matter for relatives.

Chronic phase, first line

For my situation (chronic phase, first line), which of the standard options do you recommend and why?
Why: Guideline options include: Imatinib 400 mg, or a second-generation TKI (dasatinib, nilotinib, bosutinib), or asciminib (ASC4FIRST, approved 2024). Choice by comorbidity and treatment goal; monitor BCR-ABL1 IS at 3, 6, 12 months against ELN milestones.
Am I a candidate for Imatinib, Dasatinib, Nilotinib or related drugs, and what side effects should I expect?
Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Resistance or intolerance

For my situation (resistance or intolerance), which of the standard options do you recommend and why?
Why: Guideline options include: Switch TKI guided by mutation analysis; ponatinib or asciminib for T315I; allogeneic HSCT if two or more TKIs fail or in advanced phase.
Am I a candidate for Ponatinib, Asciminib, Bosutinib, and what side effects should I expect?
Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Sustained deep molecular response

For my situation (sustained deep molecular response), which of the standard options do you recommend and why?
Why: Guideline options include: Treatment-free remission attempt after ≥3-5 years of TKI and ≥2 years of MR4 or better, with monthly PCR for the first six months (EURO-SKI); restart on loss of MMR.

Blast phase

For my situation (blast phase), which of the standard options do you recommend and why?
Why: Guideline options include: TKI plus acute-leukaemia-type induction (ponatinib or dasatinib with chemotherapy), then allogeneic HSCT.
Am I a candidate for Ponatinib, Dasatinib, and what side effects should I expect?
Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Any stage

Are there clinical trials I could join, for example of Asciminib, Allogeneic stem cell transplantation?
Why: Trials are how the next standard of care is set; asking early keeps options open.
Would a second opinion at a high-volume centre change anything, and can you help arrange it?
Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
Why: Supportive care improves quality of life and helps patients complete treatment.
I read that “Blast-phase CML: median survival still under a year”. How does that affect my plan?
Why: Open problems are where trials and second opinions matter most.
I read that “Predicting who can stop TKI safely; second TFR attempts”. How does that affect my plan?
Why: Open problems are where trials and second opinions matter most.

Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.

Everything relevant

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Direct links plus the targets, companies, and technologies of this cancer's products.

Key papers

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rctNew England Journal of Medicine 2003changed practice

IRIS: imatinib versus interferon plus cytarabine as first treatment for chronic myeloid leukaemia

IRIS made imatinib the first-line standard for CML worldwide and established the tyrosine kinase inhibitor as a chronic, life-long oral therapy. For most patients CML became a manageable condition with near-normal life expectancy. Later generations of TKIs (dasatinib, nilotinib, asciminib) produce faster, deeper responses but have not shown a survival advantage over imatinib.

translationalNew England Journal of Medicine 2001changed practice

First imatinib trial: a pill that switched off the enzyme driving chronic myeloid leukaemia

Druker's 2001 imatinib paper turned the idea of hitting a cancer's specific molecular engine into a working medicine. For people with CML it began the shift from a fatal disease treated with interferon or transplant to one managed with a daily tablet. It also set expectations, later tempered, that every cancer might have its own imatinib.