Can Chronic Myeloid Leukemia (CML) progress to Acute Myeloid Leukemia (AML)?

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Can CML Progress to AML?

Yes, CML can progress to blast phase that resembles acute leukemia, including acute myeloid leukemia (AML), though this represents disease transformation rather than development of a separate AML. 1

Understanding CML Disease Progression

CML naturally progresses through three distinct phases if left untreated or inadequately treated:

  • Chronic phase (CP-CML)Accelerated phase (AP-CML)Blast phase (BP-CML) 1
  • Without effective treatment, untreated CP-CML will progress to AP-CML or BP-CML in 3 to 5 years on average 1
  • With modern tyrosine kinase inhibitor (TKI) therapy, the annual progression rate has dramatically decreased to 1-1.5% from the historical >20% 2

Blast Phase: The "AML-Like" Transformation

Blast phase CML is defined as ≥20% blasts (WHO criteria) or ≥30% blasts (International Bone Marrow Transplant Registry criteria) in blood or bone marrow 1, 2:

  • 70-80% of blast phase cases are myeloid phenotype, resembling AML 1
  • Approximately 30% are lymphoid phenotype 3
  • Mixed phenotypes can occur 1

The key distinction is that blast phase CML remains BCR-ABL1 positive disease (Philadelphia chromosome positive), whereas de novo AML is BCR-ABL1 negative 1, 2. This is a critical difference because:

  • BP-CML represents transformation of the original CML clone 1
  • The genetic instability of BCR-ABL1-positive cells drives this progression through accumulation of additional mutations 1, 4
  • Gene expression profiling shows the bulk of genetic changes occur during the transition from CP to AP, with AP and BP showing similar expression patterns 1

Rare Exception: True AML Development

In extremely rare cases, true BCR-ABL1-negative AML can develop in CML patients during TKI therapy 5:

  • This represents a separate clonal event, not CML transformation 5
  • Can occur even in patients achieving cytogenetic response to imatinib 5
  • Does not require development of secondary cytogenetic abnormalities 5

Clinical Implications and Prognosis

Survival in blast phase is generally less than 1 year without successful intervention 6, 7:

  • Blast phase CML responds poorly to standard AML chemotherapy compared to de novo AML 2
  • Allogeneic stem cell transplantation is considered the only curative therapy for BP-CML 6, 3
  • Early indicators of progression include additional chromosomal aberrations, somatic mutations, and clinical deterioration 6

Monitoring to Prevent Progression

Regular monitoring with quantitative PCR for BCR-ABL1 transcripts every 3 months is essential 6, 2:

  • Early molecular response milestones: BCR-ABL1 ≤10% at 3 months and ≤1% at 12 months 2
  • Bone marrow examination should be performed if progression is suspected to determine CML phase 6
  • BCR-ABL1 kinase domain mutation analysis guides selection of subsequent TKI therapy if resistance develops 6

Common pitfall: Confusing blast phase CML (which is BCR-ABL1 positive) with de novo AML (which is BCR-ABL1 negative). Always confirm Philadelphia chromosome/BCR-ABL1 status, as this fundamentally changes treatment approach and prognosis 1, 2.

References

Guideline

Guideline Directed Topic Overview

Dr.Oracle Medical Advisory Board & Editors, 2025

Guideline

CML and AML: Key Differences in Disease Characteristics and Treatment Approaches

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Guideline

Leukopoiesis Regulation and Pathological Alterations

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Guideline

Management of Chronic Myeloid Leukemia Progression on Tyrosine Kinase Inhibitors

Praxis Medical Insights: Practical Summaries of Clinical Guidelines, 2025

Research

Targeted chronic myeloid leukemia therapy: seeking a cure.

Journal of managed care pharmacy : JMCP, 2007

Professional Medical Disclaimer

This information is intended for healthcare professionals. Any medical decision-making should rely on clinical judgment and independently verified information. The content provided herein does not replace professional discretion and should be considered supplementary to established clinical guidelines. Healthcare providers should verify all information against primary literature and current practice standards before application in patient care. Dr.Oracle assumes no liability for clinical decisions based on this content.

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