Bone Marrow CD34+/lin− Cells of Patients with Chronic-Phase Chronic Myeloid Leukemia (CP-CML) After 12 Months of Nilotinib Treatment Exhibit a Different Gene Expression Signature Compared to the Diagnosis and the Corresponding Cells from Healthy Subjects

Simple Summary: Nilotinib and other tyrosine kinase inhibitors (TKIs) target the BCR-ABL1 oncoprotein in Chronic Myeloid Leukemia (CML), reducing the growth of leukemic stem cells (LSCs) and promoting their death. However, some LSCs persist in the bone marrow (BM), which may lead to relapse and dise...

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Publicado en:Cancers Vol. 17; no. 6; pp. 1022 - 1036
Autores principales: Trojani, Alessandra, Pungolino, Ester, Di Camillo, Barbara, Bossi, Luca Emanuele, Palumbo, Cassandra, D'adda, Mariella, Perego, Alessandra, Turrini, Mauro, Elena, Chiara, Borin, Lorenza Maria, Iurlo, Alessandra, Malato, Simona, Spina, Francesco, Latargia, Maria Luisa, Spedini, Pierangelo, Artale, Salvatore, Anghilieri, Michela, Carraro, Maria Cristina, Bucelli, Cristina, Beghini, Alessandro
Formato: research tables/charts Journal Article
Publicado: MDPI Mar2025
Acceso en línea:Ver este registro en EBSCOhost
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      dt: Mar2025
      vid: 17
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      pid: 97109
      pub: MDPI
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        184104579
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        10.3390/cancers17061022
        184104579
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        atl: Bone Marrow CD34+/lin− Cells of Patients with Chronic-Phase Chronic Myeloid Leukemia (CP-CML) After 12 Months of Nilotinib Treatment Exhibit a Different Gene Expression Signature Compared to the Diagnosis and the Corresponding Cells from Healthy Subjects
      aug:
        au:
          Trojani, Alessandra
          Pungolino, Ester
          Di Camillo, Barbara
          Bossi, Luca Emanuele
          Palumbo, Cassandra
          D'adda, Mariella
          Perego, Alessandra
          Turrini, Mauro
          Elena, Chiara
          Borin, Lorenza Maria
          Iurlo, Alessandra
          Malato, Simona
          Spina, Francesco
          Latargia, Maria Luisa
          Spedini, Pierangelo
          Artale, Salvatore
          Anghilieri, Michela
          Carraro, Maria Cristina
          Bucelli, Cristina
          Beghini, Alessandro
        affil: Department of Hematology and Oncology, ASST Grande Ospedale Metropolitano Niguarda, 20162 Milan, Italy
      sug:
        subj:
          Gene Expression Profiling
          Bone Marrow Physiopathology
          Antigens, Surface Analysis
          Leukemia, Myeloid, Chronic Drug Therapy
          Nilotinib Therapeutic Use
          Human
          Leukemia, Myeloid, Chronic Diagnosis
          Signal Transduction Physiology
          Genes Evaluation
          Stem Cells Drug Effects
          Stem Cells Physiopathology
          Protein Kinases Drug Effects
          Recurrence Risk Factors
          Transplant Donors
      ab: Simple Summary: Nilotinib and other tyrosine kinase inhibitors (TKIs) target the BCR-ABL1 oncoprotein in Chronic Myeloid Leukemia (CML), reducing the growth of leukemic stem cells (LSCs) and promoting their death. However, some LSCs persist in the bone marrow (BM), which may lead to relapse and disease progression. We analyzed the gene expression profiling of BM CD34+/lin− cells from 79 Chronic-Phase CML patients at diagnosis and after 12 months of nilotinib treatment, comparing them to the healthy cells from 10 donors (CTRLs). Our study identified key genes involved in eight crucial biological pathways: CML, cell cycle, JAK-STAT, PI3K-Akt, MAPK, Ras, NF-kB, and ABC transporters. We observed a progressive down-regulation of the expression levels of several genes across these pathways from diagnosis to 12 months of nilotinib treatment and to CTRLs. This could suggest that nilotinib effects create selective pressure, potentially supporting the survival and self-renewal of LSCs. Background: Chronic-Phase Chronic Myeloid Leukemia (C-PCML) is defined by the presence of the BCR-ABL1 fusion gene, which encodes a tyrosine kinase protein that drives the uncontrolled proliferation and survival of leukemic stem cells (LSCs). Nilotinib, a tyrosine kinase inhibitor, targets the activity of BCR-ABL1 by reducing aberrant signaling pathways, which drive the regeneration of LSCs. Despite nilotinib's action, a population of resilient LSCs persist in the bone marrow (BM) and can indeed drive relapse and progression in CML patients. Methods: Our study investigated the gene expression profiling (GEP) of BM CD34+/lin− cells from 79 CP-CML patients at diagnosis, compared to the BM CD34+/lin− cells from the same patients after 12 months of nilotinib treatment and to the normal counterpart cells from 10 donors (CTRLs). Results: GEP analyses identified 3012 significantly differentially expressed genes across these comparisons. Among these, we focused on certain key genes associated with eight crucial KEGG pathways: CML, cell cycle, JAK-STAT, PI3K-Akt, MAPK, Ras, NF-kB, and ABC transporters. Within these pathways, we observed the up-regulation of several genes at diagnosis compared to both 12 months of nilotinib treatment and the CTRLs. Conclusions: We observed that certain transcriptome features present at diagnosis persisted after 12 months of nilotinib treatment, compared to CTRLs. This suggests that nilotinib may exert selective pressure, potentially supporting the survival and self-renewal of LSCs. Future insights into these pathways could help identify therapeutic targets to improve outcomes in CML.
      pubtype: Academic Journal
      doctype:
        research
        tables/charts
        Journal Article
      ougenre: Article
    language: English
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