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Open research questions in Chronic Myeloid Leukemia Treatments

119 gap statements mined from Chronic Myeloid Leukemia Treatments papers in our 4.5M-paper local library, which holds 619 papers on the topic — drawn mostly from each paper's own stated research gap, future-work, challenge and limitation notes, and its abstract. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one.

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What the literature leaves open

  • One of the challenges is the potential for molecular relapse after tyrosine kinase inhibitor discontinuation. Another challenge is the need for lifelong treatment with tyrosine kinase inhibitors. The study also faced challenges in terms of patient recruitment and retention.

    Randomized phase 3 trial of Ropeginterferon alfa-2b versus surveillance after tyrosine kinase inhibitor discontinuation in chronic myeloid leukemia (ENDURE/CML-IX) · 2026 · DOI

  • The co-occurrence of BCR::ABL1 rearrangement with concomitant Ph-negative MPN is rarely reported. There is a lack of understanding of the clinical outcomes and treatment responses in patients with Ph-negative MPN and CML. Prior work considered BCR::ABL1 rearrangement and MPN driver mutations to be mutually exclusive.

    Myeloproliferative neoplasms with concomitant chronic myeloid leukemia are associated with TKI resistance and poor outcomes · 2026 · DOI

  • The complexity of ribosome biology and the need to develop novel methods to analyze ribosome collisions. The challenge of targeting ribosome collisions as a therapeutic strategy for CML. The need to further understand the mechanisms by which ribosome collisions activate the ribotoxic stress response.

    BCR::ABL1 tyrosine kinase inhibitors induce ribosome collisions to activate ZAK-dependent ribotoxic stress and apoptosis in chronic myeloid leukemia · 2026 · DOI

  • There is a need to optimize asciminib treatment for patients with CML-CP who have not responded optimally to prior TKI therapy. The study aims to address the gap in treatment options for these patients. The study evaluates the efficacy and safety of asciminib in this patient population.

    ASC4OPT: asciminib treatment optimization study in patients with chronic myeloid leukemia in chronic phase previously treated with two or more tyrosine kinase inhibitors · 2026 · DOI

  • The clinical and molecular characteristics of BRAF-mutated AML are not well understood. The prognostic and therapeutic implications of BRAF mutations in AML are not clear. Prior studies have limited BRAF gene coverage, which may lead to underestimation of the actual frequency of BRAF mutations in AML.

    Prognostic and therapeutic implications of BRAF mutations in acute myeloid leukemia · 2026 · DOI

  • The gap in current research is the lack of accurate methods for quantifying blood basophils. The paper identifies the need for improved quantitation of basophils using AI-assisted digital processing and machine learning methods.

    Chronic myeloid leukaemia: have basophils gone the way of the Dodo bird? · 2026 · DOI

  • The lack of clinical variables in administrative databases. The need for an integrative infrastructure to combine administrative and clinical data.

    From data to evidence: why administrative databases alone cannot measure real-world effectiveness in rare diseases – the case of chronic myeloid leukaemia · 2026 · DOI

  • Ensuring precise inter-laboratory quantitation of BCR::ABL1 RNA is a significant challenge. Inter-laboratory variability in BCR::ABL1 quantitation can impact chronic myeloid leukemia disease management. The study identifies the need for standardized reporting on the International Scale to improve inter-laboratory reproducibility.

    Improved inter-lab precision after CML international scale adoption: a 15-year history of BCR::ABL1 proficiency testing · 2026 · DOI

  • The impact of tyrosine kinase inhibitors on the health status of long-term chronic myeloid leukemia survivors is unknown. There is a lack of studies comparing the health status of TKI-treated CML patients to non-CML controls and CML patients treated with blood or marrow transplantation.

    Health status impairments in tyrosine kinase inhibitor-treated chronic myeloid leukemia patients versus blood or marrow transplant survivors and non-cancer controls · 2026 · DOI

  • The mechanisms by which RUNX1 aberrations contribute to the development of blast-phase chronic myeloid leukaemia and therapeutic resistance are not well understood. The role of post-transcriptional dysregulation in the development of blast-phase CML is not well characterised.

    RUNX1 aberrations in blast-phase chronic myeloid leukaemia induce the RNA-binding protein SPATS2L, which promotes growth, survival and stress granule assembly · 2026 · DOI

  • The study had a limited sample size, with only 39 patients attempting treatment-free remission. The study had to contend with the complexity of tyrosine kinase inhibitor management strategies and their impact on treatment-free remission outcomes.

    Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia · 2025 · DOI

  • Dasatinib-associated pleural effusion is not limited to the early treatment phase and may develop at any time during therapy, resulting in an increased cumulative incidence with long-term follow-up [ 15 ].

    Long-term outcomes of low-dose dasatinib in older patients with chronic myeloid leukemia in chronic phase: an extended follow-up of the DAVLEC phase 2 trial · 2026 · DOI

  • Although imatinib's nephrotoxic potential is established, the renal safety profiles of second-generation TKIs-dasatinib, nilotinib, and flumatinib-remain incompletely characterized.

    Renal dysfunction in patients with CML on long-term first-line TKI therapy: a single-center analysis · 2026 · DOI

  • Thus, all patients in this study were candidates for allo-HCT, but it was not performed when a suitable donor was unavailable or when the patient’s performance status was insufficient.

    Poor prognostic implication of CDKN2 deletion in adult patients with Philadelphia chromosome-positive ALL · 2025 · DOI

  • Knowledge on survival among patients with CML after the introduction of a later generation of TKIs has been based on information from clinical trials, whereas data on survival in a real-world setting remain sparse [ 12 ].

    Temporal changes in survival among adult patients with chronic myeloid leukemia in the period 1995–2022—a Danish nationwide population-based register study · 2025 · DOI

  • Concerns about the integrity of the figures were identified. The authors' explanations were not deemed sufficient to resolve the concerns.

    RETRACTED: Li et al. Blockade of Y177 and Nuclear Translocation of Bcr-Abl Inhibits Proliferation and Promotes Apoptosis in Chronic Myeloid Leukemia Cells. Int. J. Mol. Sci. 2017, 18, 537 · 2026 · DOI

  • Nonetheless, adverse events still occur, survival remains suboptimal and the long-term health impact of the disease and its treatment is poorly understood.

    Adverse Health Events in Chronic Myeloid Leukaemia Patients Treated With Tyrosine Kinase Inhibitors 2009–2019: A Real‐World Study From the UK 's Haematological Malignancy Research Network · 2026 · DOI

  • These findings could aid clinicians in the early detection of these rarely reported but fatal hematologic AEs, thereby reducing the risk of severe toxicities in blinatumomab recipients.

    Disseminated intravascular coagulation is an underestimated but fatal adverse event associated with blinatumomab therapy: A pharmacovigilance analysis of FAERS · 2024 · DOI

  • The study had a limited sample size of 203 patients, - The study had a relatively short follow-up period of 36 months, - The study only included patients with stable deep molecular remission, - The study did not include a placebo…

    Randomized phase 3 trial of Ropeginterferon alfa-2b versus surveillance after tyrosine kinase inhibitor discontinuation in chronic myeloid leukemia (ENDURE/CML-IX) · 2026 · DOI

  • The data on fluctuating remission has to be considered as exploratory and needs further validation, - The study did not have data on the validation sample for fluctuating remission, - The EURO-SKI Biomarker Study included only 240 of the 728 EURO-SKI…

    Comprehensive molecular characterization of treatment-free remission and molecular relapse in chronic myeloid leukemia patients: the EURO-SKI Biomarker Study · 2026 · DOI

  • Investigating the role of ZAK in other types of cancer, - Examining the potential of targeting ZAK-dependent RSR as a therapeutic…

    BCR::ABL1 tyrosine kinase inhibitors induce ribosome collisions to activate ZAK-dependent ribotoxic stress and apoptosis in chronic myeloid leukemia · 2026 · DOI

  • No future research directions are explicitly suggested, - Further studies could investigate the impact of IS adoption on patient outcomes, - Research on other variables that could affect inter-laboratory precision…

    Improved inter-lab precision after CML international scale adoption: a 15-year history of BCR::ABL1 proficiency testing · 2026 · DOI

  • Investigating the interaction between stress response and dysregulated RiBi/translation, - Examining the role of RUNX1 as a regulator of 5'TOP motifs, - Studying the effects of SPATS2L on therapeutic resistance in RUNX1 mutated…

    RUNX1 aberrations in blast-phase chronic myeloid leukaemia induce the RNA-binding protein SPATS2L, which promotes growth, survival and stress granule assembly · 2026 · DOI

  • Further studies are needed to elucidate TKI management strategies that will increase the rates of sustained TFR without compromising patient safety, - Research on the long-term outcomes of TFR…

    Real-World Evidence of Treatment-Free Remission Strategies and Outcomes in Chronic Myeloid Leukemia · 2025 · DOI

  • Although limited by a small number of patients, our data suggest concomitant genetic alterations did not substantially contribute to the poor survival outcomes observed in patients with CDKN2 del…

    Poor prognostic implication of CDKN2 deletion in adult patients with Philadelphia chromosome-positive ALL · 2025 · DOI

Most-cited papers in Chronic Myeloid Leukemia Treatments

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Open questions raised by papers listed on this page include: (1) There is a lack of understanding of the clinical outcomes and treatment responses in patients with Ph-negative MPN and CML (“Myeloproliferative neoplasms with concomitant chronic myeloid leukemia are…”, 2026); (2) There is a need to optimize asciminib treatment for patients with CML-CP who have not responded optimally to prior TKI therapy (“ASC4OPT: asciminib treatment optimization study in patients with chronic…”, 2026); (3) The clinical and molecular characteristics of BRAF-mutated AML are not well understood (“Prognostic and therapeutic implications of BRAF mutations in acute myeloid…”, 2026); (4) The gap in current research is the lack of accurate methods for quantifying blood basophils (“Chronic myeloid leukaemia: have basophils gone the way of the Dodo bird?”, 2026); (5) The impact of tyrosine kinase inhibitors on the health status of long-term chronic myeloid leukemia survivors is unknown (“Health status impairments in tyrosine kinase inhibitor-treated chronic myeloid…”, 2026). 119 gap statements have been mined from Chronic Myeloid Leukemia Treatments papers in our 4.5M-paper local library, which holds 619 papers on the topic; the gaps come from whichever of those papers state one. They are mostly the research gaps the authors state and the papers' abstracts, plus future-work, limitations and challenges passages. The ones listed below are a selection still marked open; each names the study that raised it, with a DOI link where the paper has one, so you can read the original claim in context.

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