Prolonged indolent clinical course in a high-risk chronic phase-CML patient without cytogenetic or molecular response: a case challenging established response paradigms
7 affiliations
- 1Division of Hematology, AOU Policlinico “Rodolico - S. Marco”, University of Catania, Catania, Italy
- 2Department of Clinical and Experimental Medicine, University of Catania, Catania, 95123 Italy
- 3Center of Experimental Oncology and Hematology, A.O.U. Policlinico “G. Rodolico-S. Marco”, Catania, 95123 Italy
- 4Provincial Health Agency of Ragusa, Ragusa, RG Italy
- 5Department of General Surgery and Medical-Surgical Specialties, University of Catania, Catania, 95123 Italy
- 6Hematology Section, AOU Policlinico “G. Martino”, University of Messina, via Consolare Valeria 1, Messina, 98124 Italy
- 7Department of Human Pathology in Adulthood and Childhood “G. Barresi”, University of Messina, Messina, 98124 Italy
Abstract
Chronic-phase chronic myeloid leukemia (CP-CML) is currently treated with tyrosine kinase inhibitors (TKIs), and the early achievement of cytogenetic and molecular milestones is strongly associated with improved clinical outcomes. Here, we describe the case of a 55-year-old woman diagnosed with CP-CML in 2002 who received sequential TKI therapy for more than 20 years. Despite continuous treatment, she never achieved a complete cytogenetic response (CCyR) or a major molecular response (MMR), and the BCR::ABL1IS transcript levels remained above 10% for most of the disease course. At diagnosis, she presented with high-risk features, including p190 BCR::ABL1 transcript expression, and exhibited multiple mutations, but not driver ones, over time. Nevertheless, along with several TKIs treatments, she maintained a stable chronic-phase disease with sustained hematologic control and preserved a good quality of life throughout long-term follow-up. This case highlights the biological heterogeneity of CML, the timing and trajectory of BCR::ABL1-driven disease, the importance of patient-centered outcomes and the role of TKI therapy in maintaining durable hematologic remission and long-term disease control, even in the absence of optimal molecular milestones.
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BibTeX
@article{longo2026prolonged,
title = {{Prolonged indolent clinical course in a high-risk chronic phase-CML patient without cytogenetic or molecular response: a case challenging established response paradigms}},
author = {Ermelinda Longo and Uros Markovic and Stefania Stella and Agostino Antolino and Michele Massimino and Manlio Fazio and Francesco Di Raimondo and Alessandro Allegra and Fabio Stagno},
journal = {Annals of Hematology},
year = {2026},
month = aug,
doi = {10.1007/s00277-026-07233-4},
url = {https://doi.org/10.1007/s00277-026-07233-4},
note = {CC BY-NC-ND 4.0},
}
RIS
TY - JOUR
TI - Prolonged indolent clinical course in a high-risk chronic phase-CML patient without cytogenetic or molecular response: a case challenging established response paradigms
AU - Ermelinda Longo
AU - Uros Markovic
AU - Stefania Stella
AU - Agostino Antolino
AU - Michele Massimino
AU - Manlio Fazio
AU - Francesco Di Raimondo
AU - Alessandro Allegra
AU - Fabio Stagno
PY - 2026
DA - 2026/08/10
JO - Annals of Hematology
DO - 10.1007/s00277-026-07233-4
UR - https://doi.org/10.1007/s00277-026-07233-4
AB - Chronic-phase chronic myeloid leukemia (CP-CML) is currently treated with tyrosine kinase inhibitors (TKIs), and the early achievement of cytogenetic and molecular milestones is strongly associated with improved clinical outcomes. Here, we describe the case of a 55-year-old woman diagnosed with CP-CML in 2002 who received sequential TKI therapy for more than 20 years. Despite continuous treatment, she never achieved a complete cytogenetic response (CCyR) or a major molecular response (MMR), and the BCR::ABL1IS transcript levels remained above 10% for most of the disease course. At diagnosis, she presented with high-risk features, including p190 BCR::ABL1 transcript expression, and exhibited multiple mutations, but not driver ones, over time. Nevertheless, along with several TKIs treatments, she maintained a stable chronic-phase disease with sustained hematologic control and preserved a good quality of life throughout long-term follow-up. This case highlights the biological heterogeneity of CML, the timing and trajectory of BCR::ABL1-driven disease, the importance of patient-centered outcomes and the role of TKI therapy in maintaining durable hematologic remission and long-term disease control, even in the absence of optimal molecular milestones.
SN - 0939-5555
SN - 1432-0584
ER -