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Rogers G, Hoyle M, Thompson Coon J, et al. Dasatinib and Nilotinib for Imatinib-Resistant or -Intolerant Chronic Myeloid Leukaemia: A Systematic Review and Economic Evaluation. Southampton (UK): NIHR Journals Library; 2012 Apr. (Health Technology Assessment, No. 16.22.)

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Dasatinib and Nilotinib for Imatinib-Resistant or -Intolerant Chronic Myeloid Leukaemia: A Systematic Review and Economic Evaluation.

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Abelson oncogene (ABL)

An oncogene is a cancer-causing gene. The ABL gene is located on part of chromosome 9 that translocates to chromosome 22 in chronic myeloid leukaemia (CML).

Allogeneic transplant

A bone marrow (BM) or stem cell transplant (SCT) using marrow from another person.


An excess number of basophils, a rare type of white blood cell (WBC) found in the peripheral blood (PB).

Blast cells

Immature cells found in and produced by the BM. Not normally found in the PB.


An oncogene fusion protein consisting of breakpoint cluster region (BCR) and ABL.

Bone marrow (BM)

The soft substance that fills bone cavities. It is composed of mature and immature blood cells and fat. Red and WBCs and platelets are formed in the BM.

Breakpoint cluster region (BCR)

The region of on a chromosome where breaks cluster. In the case of CML, it is the narrow part of chromosome 22 where the translocation to chromosome 9 occurs which includes the ABL (BCR–ABL). The BCR–ABL protein product results in the excessive proliferation of a tyrosine kinase.

Bone marrow transplant

A procedure in which a patient's BM is replaced by healthy BM. The BM to be replaced may be deliberately destroyed by high doses of chemotherapy and/or radiation therapy. The replacement marrow may come from another person or it may have been previously harvested from the patient's own marrow.


The treatment of a disease by chemicals to destroy cancer cells. Chemotherapy can affect the whole body.

Cytogenetic response (CyR)

A response to treatment at the level of chromosomal abnormalities. In the case of CML, assessed by counting the number of Philadelphia-positive (Ph+) cells in metaphase (usually 20 metaphases are analysed). A complete response generally means no Ph+ cells, a partial response leaves up to 35% Ph+ cells evident and with a minor response from 35% to 95% Ph+ cells are still evident.


A reduction in the number of cells circulating in the blood.


An adapter protein that becomes tyrosine phosphorylated by BCR–ABL.

European Quality of Life-5 Dimensions (EQ-5D)

A European quality of life questionnaire containing five physical and psychological dimensions.

Extramedullary disease

Disease occurring outside the BM.

Haematological response (HR)

A HR refers to the normalisation of blood cell counts. CML causes overproliferation of WBCs, which treatments aims to lower and categories of response indicate the extent to which this occurs. Typically, the HR is classified as complete if the WBC count is < 10 × 109/l, platelet count is < 450 × 109/l, there are no immature cells in the PB with normal differential count, and there is a disappearance of symptoms and signs.


Hydroxycarbamide (Hydrea®, Bristol-Myers Squibb) is a drug used in the treatment of CML which inhibits deoxyribonucleic acid (DNA) synthesis.

Incremental cost-effectiveness ratio (ICER)

Demonstrates the total additional cost per quality-adjusted life-year (QALY) gained of one alternative over another. There is no particular point at which an alternative is said to be ‘cost-effective’ as this will be a policy decision. The larger the ICER, the less likely the treatment is to be cost-effective.

Interferon-α (IFN)

Interferon is a protein derived from human cells. It has a role in fighting viral infections by preventing virus multiplication in cells. IFN-α is made by leucocytes. It is often used as a first-line therapy in CML.

Landmark analysis

A form of survival analysis that includes only patients who have survived for a specified period of time.


WBCs which are responsible for fighting infections.


A process of removing excess WBCs from the PB.


A reduced number of WBCs in the blood – it may affect a single cell type or all WBCs.


The second phase of mitosis (cell division). Cells in this phase of division are used for cytogenetic analysis in CML to identify the proportion of Ph+ chromosomes.


A division of cells which consists of four phases: prophase, metaphase, anaphase and telophase.


Committed progenitor cells produced by, and found in, the BM which develop into mature leucocytes.


A decrease in neutrophils (WBCs) circulating in the blood.

Peripheral blood (PB)

In this report PB refers to blood in the circulatory system.


Committed progenitor cells produced by and found in the BM which develop into myelocytes.


A non-receptor protein tyrosine kinase.

Stem cells

Very early progenitor cells which divide and mature to become all the types of cells which make up the blood and the immune system.


Platelets (fragments of BM cells) found in the blood which help to form clots and control bleeding.


A reduced number of thrombocytes (platelets) in the blood.


The quality of being poisonous. The National Cancer Institute grades toxicity levels of treatments as: 1 = mild, 2 = moderate, 3 = severe and 4 = life-threatening.

Tyrosine kinase

An enzymatic protein which adds phosphate residues to other proteins in the cell. In CML, the abnormal tyrosine kinase, BCR–ABL, phosphorylates proteins which cause cellular proliferation.

Weibull curve

A mathematical function which is often used in modelling to describe survival times and in which the chance of survival varies with time.

© 2012, Crown Copyright.

Included under terms of UK Non-commercial Government License.

Bookshelf ID: NBK98821


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