TARGET: a survey of real‐world management of chronic myeloid leukaemia across 33 countries

Abstract

Summary Despite the availability of guidelines for the management of chronic myeloid leukaemia (CML), various issues may prevent their successful implementation. The TARGET survey examined real‐world management of CML patients compared with international recommendations. This online survey was completed in 2017. Results were discussed by a Steering Committee (SC) of eight international haematologists, challenges were identified and practical solutions developed. Of the 1008 haematologists invited (33 countries), 614 completed the survey. Gaps regarding treatment efficacy and molecular monitoring were identified. Half of the physicians did not perform three‐monthly testing of during the initial 12 months of treatment, citing cost as the major barrier, although they know it should be done. Treatment‐free remission was not considered a primary treatment goal or as a priority factor influencing treatment decisions. European Leukemia Net guidelines interpretation was generally acceptable, but awareness regarding management of persistent adverse events was poor. Practical solutions proposed by the SC were mostly focused on enhancing physician education and awareness, or encouraging hospitals to work with the government, in order to improve the quality of BCR‐ABL testing. Gaps in current CML management were identified compared with international recommendations, which the proposed practical solutions would help to address.

Nilotinib-Induced Diabetes Mellitus in a Young Female with Chronic Myeloid Leukemia

“…Most patients present in chronic phase CML, which is typically manifest as leukocytosis with immature myeloid cells, with or without anemia, thrombocytopenia, constitutional symptoms, splenomegaly, and/or bleeding. Chronic phase CML can progress from a relatively indolent disorder to the more aggressive disorders, accelerated phase (AP) or blast crisis (BC) [1].…”

“…Available treatment options for patients with CML include allogeneic hematopoietic cell transplantation (HCT), tyrosine kinase inhibitors (TKIs), and palliative therapy with cytotoxic agents [1]. Oral TKIs target the constitutively active tyrosine kinase implicated in the pathogenesis of CML.…”

“…It mainly affects adults and is rarely seen in children. The average age at diagnosis is around 64 years [1]. CML typically runs a biphasic or triphasic course (chronic phase, accelerated phase, and blastic phase), with a diagnosis usually made in the chronic phase.…”

Extreme Levels of Platelet Count in Essential Thrombocythemia: Management and Outcome, Report of Two Cases

“…Although approximately half of the patients with ET are asymptomatic at the time of diagnosis, most patients develop thrombotic, vasomotor, or hemorrhagic features at some point during the course of their disease; venous or arterial thromboembolic incidents, including stroke, myocardial infarction, venous thromboembolism, and first-trimester pregnancy loss (either spontaneously or during an otherwise hypercoagulable state). The incident appears to be increased when the white blood cell count is elevated [1][2][3][4]. Mostly patients with essential thrombocytosis possess mutations in one of three genes: first, MPL mutations have been associated with around 3-5% of essential thrombocytosis cases.…”

Chronic Myeloid Leukemia Preceded by Tuberculosis

“…In the absence of treatment, CML follows a triphasic or biphasic clinical course as it progresses from a chronic phase to an accelerated phase and on to a terminal blast crisis. CML accounts for approximately 15-20% of all leukemia cases in adults [2]. It has an annual incidence rate of 1-2 cases per 100,000, with a slight male predominance [3].…”