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ID

45784

Description

Principal Investigator: Catherine Wu, MD, Dana Farber Cancer Institute, Boston, MA, USA MeSH: Leukemia, Lymphocytic, Chronic, B-Cell https://www.ncbi.nlm.nih.gov/projects/gap/cgi-bin/study.cgi?study_id=phs001091 We analyzed clonal evolution in serial samples from five CLL patients who became resistant to the Bruton's tyrosine kinase (BTK) inhibitor ibrutinib, using whole-exome and deep targeted sequencing. We observe a BTK-C481S mutation in one of five patients, and multiple PLCG2 mutations in a second patient. The other patients had an expansion of clones harboring del(8p) carrying additional driver mutations (EP300, MLL2, EIF2A), with one patient developing trans-differentiation into CD19-negative histiocytic sarcoma. We calculated the growth kinetics of ibrutinib-resistant subclones and estimated the size of the resistant clones at treatment initiation, which we validated by droplet-microfluidic technology. Haplo-insufficiency of TRAIL-R, a consequence of del(8p), led to TRAIL insensitivity which may contribute to development of ibrutinib resistance. These findings demonstrate that ibrutinib therapy has the potential to lead to clonal selection and expansion of rare cell populations already present at the time of treatment initiation. They also provide insight into the heterogeneity of genetic changes associated with ibrutinib resistance, previously attributed solely to mutations in BTK and related pathway molecules.

Link

dbGaP-study=phs001091

Keywords

  1. 6/21/23 6/21/23 - Chiara Middel
Copyright Holder

Catherine Wu, MD, Dana Farber Cancer Institute, Boston, MA, USA

Uploaded on

June 21, 2023

DOI

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License

Creative Commons BY 4.0

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    dbGaP phs001091 Clonal Evolution in Patients with Chronic Lymphocytic Leukemia

    This subject phenotype table contains subject IDs, gender, age, primary disease, mutational status of the IgHV genes, and chromosomal alterations as identified by FISH.

    pht006065
    Description

    pht006065

    Alias
    UMLS CUI [1,1]
    C3846158
    Unique Participant Identifier
    Description

    SUBJID

    Data type

    string

    Alias
    UMLS CUI [1,1]
    C2348585
    Participant's gender as Male or Female
    Description

    Gender

    Data type

    string

    Alias
    UMLS CUI [1,1]
    C0079399
    Age of patient
    Description

    Age

    Data type

    string

    Measurement units
    • years
    Alias
    UMLS CUI [1,1]
    C0001779
    years
    Primary disease state of patient
    Description

    Primary Disease

    Data type

    string

    Alias
    UMLS CUI [1,1]
    C0205225
    UMLS CUI [1,2]
    C0012634
    UMLS CUI [1,3]
    C0449438
    Mutational status of the Immunoglobulin Heavy Chain Variable Region Genes (IgHV)
    Description

    IgHV

    Data type

    text

    Alias
    UMLS CUI [1,1]
    C0026882
    UMLS CUI [1,2]
    C0449438
    UMLS CUI [1,3]
    C4321381
    UMLS CUI [1,4]
    C0017337
    Chromosomal alterations as identified by Fluorescence in situ Hybridization (FISH)
    Description

    FISH

    Data type

    string

    Alias
    UMLS CUI [1,1]
    C0008625
    UMLS CUI [1,2]
    C0205396
    UMLS CUI [1,3]
    C0162789

    Similar models

    This subject phenotype table contains subject IDs, gender, age, primary disease, mutational status of the IgHV genes, and chromosomal alterations as identified by FISH.

    Name
    Type
    Description | Question | Decode (Coded Value)
    Data type
    Alias
    Item Group
    pht006065
    C3846158 (UMLS CUI [1,1])
    SUBJID
    Item
    Unique Participant Identifier
    string
    C2348585 (UMLS CUI [1,1])
    Item
    Participant's gender as Male or Female
    string
    C0079399 (UMLS CUI [1,1])
    Code List
    Participant's gender as Male or Female
    CL Item
    Female (Female)
    C0086287 (UMLS CUI [1,1])
    CL Item
    Male (Male)
    C0086582 (UMLS CUI [1,1])
    Age
    Item
    Age of patient
    string
    C0001779 (UMLS CUI [1,1])
    Item
    Primary disease state of patient
    string
    C0205225 (UMLS CUI [1,1])
    C0012634 (UMLS CUI [1,2])
    C0449438 (UMLS CUI [1,3])
    Code List
    Primary disease state of patient
    CL Item
    Chronic Lymphocytic Leukemia (Chronic Lymphocytic Leukemia)
    Item
    Mutational status of the Immunoglobulin Heavy Chain Variable Region Genes (IgHV)
    text
    C0026882 (UMLS CUI [1,1])
    C0449438 (UMLS CUI [1,2])
    C4321381 (UMLS CUI [1,3])
    C0017337 (UMLS CUI [1,4])
    Code List
    Mutational status of the Immunoglobulin Heavy Chain Variable Region Genes (IgHV)
    CL Item
    Unknown (1)
    CL Item
    Unmutated (2)
    Item
    Chromosomal alterations as identified by Fluorescence in situ Hybridization (FISH)
    string
    C0008625 (UMLS CUI [1,1])
    C0205396 (UMLS CUI [1,2])
    C0162789 (UMLS CUI [1,3])
    Code List
    Chromosomal alterations as identified by Fluorescence in situ Hybridization (FISH)
    CL Item
    del 11q (del 11q)
    CL Item
    del 13q, del 11q (del 13q, del 11q)
    CL Item
    del 17p, del 13q (del 17p, del 13q)
    CL Item
    del 17p, del 13q, del 11q (del 17p, del 13q, del 11q)
    CL Item
    del 17p, del 13q, trisomy 12 (del 17p, del 13q, trisomy 12)

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