Chronic myeloid leukemia (CML) is a clonal malignant disease characterized by excessive proliferation of myeloid lineage, followed by a progressive loss of cell differentiation, with evolution including progression from chronic phase to accelerated or blastic phase. The disease is associated with a specific cytogenetic abnormality, the Philadelphia chromosome, which results from a reciprocal translocation between the long arms of chromosomes 9 and 22 [t(9;22)(q34;q11)], leading to the generation of the new leukemia-specific gene BCR-ABL, resulting in the production of an oncoprotein with tyrosine kinase activity, responsible for the pathophysiology of disease. Effective treatments with tyrosine kinase inhibitors (TKI) are available for CML patients and differences in adverse events (AE) and incidences occurring during therapy are reported in the literature depending on the type of TKI.


Improve the standard of resources used and reduce later treatment costs of treatment-related haematological and non-hematological adverse events of CML patients, from the perspective of the Brazilian National Health System. Cost of treatment per event will be presented as a result.


A Delphi methodology was applied in Brazil to study the management of adverse events related to CML treatment by collecting data on resource used., considering the resource use and its cost associated.  Based on the literature, a questionnaire was developed on the available resources for the treatment of select adverse events. Ten experts completed the questionnaires and the results were compiled and validated. The data collected were related to percentage of patients affected by each AE and percentage of each resource used to manage the AE. A consensus meeting was held after questionnaire completion. The unit cost of resources was based on the Bank Price and Health Management System Procedures Table of SUS (SIGTAP). Cost of treatment of the AEs consisted of the cost of the treatment options and monitoring costs (e.g., outpatient visits, laboratory tests and procedures, hospitalizations) in the Brazilian Public Health System.


Among non-hematologic adverse events grade 3-4, gastrointestinal bleeding and central nervous system (CNS) hemorrhage presented the highest costs of treatment per event (R$ 1.022,90 and R$ 1.173,34, respectively).  Hospitalization is the resource with more impact; mainly in CNS hemorrhage, with an average length of hospitalization reported by the experts of 1 week and also being demanded by 100% of patients affected by this AE. Management of cardiac events is R$973,35 and Pericardial effusion is R$3.896,55. Cytopenias are the most commonly occurring events in patients with CML receiving BCR–ABL inhibitors. Experts suggest the use of growth factors, such as granulocyte colony-stimulating factor, interleukin-11 and erythropoietin in patients with CML receiving TKI therapy. The Panel experts agreed the cost associated with the management the hematologic adverse events are: Anemia: R$14,93, Thrombocytopenia: R$14,47 and neutropenia: R$211,54.


All adverse events related to treatment of CML, with differences in the occurrence and intensity should be valued and recorded as contributing to the estimated total cost of treating the disease. Adverse event management in CML patients has important clinical and economic impact.

I Banco de Preço em Saúde. Disponível em:  - Accessed in 08/04/2013

II Sistema de Gerenciamento da Tabela de Procedimentos, Medicamentos e OPM do SUS. DATASUS – SIGTAP.  Available in  -  Accessed in: 08/04/2013


Alves: Novartis: Employment. Lemos: Novartis: Employment. Boquimpani: Novartis: Consultancy.

Author notes


Asterisk with author names denotes non-ASH members.

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