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Budget Impact Analysis of Discontinuing Tyrosin Kinase Inhibitors in Patients With Chronic Myeloid Leukemia Achieving a Complete Molecular Response by Using Probabilistic Markov Approach

NCT03540654 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 355

Last updated 2020-04-28

No results posted yet for this study

Summary

Chronic myeloid leukemia (CML) is a model of targeted therapy for human malignancies. Over the past decade, a broad array of drugs designed to selectively inhibit protein tyrosine kinases (PTK) \[i.e., tyrosine kinase inhibitors, (TKI)\] have emerged as novel therapies for cancer patients. Hence, CML is an hematopoietic stem cell disorder in which a t(9;22) (q34;q11) reciprocal chromosomal translocation gives rise to Philadelphia chromosome (Ph) and generates the BCR-ABL1 fusion gene encoding a constitutively activated PTK. TKIs, such as imatinib by blocking BCR-ABL1 kinase activity, selectively eradicate CML cells and induce durable responses and prolong survival. CML patients treated with TKI are monitored by quantitative RT-PCR to detect leukemic BCR-ABL1 transcript performed from peripheral blood samples (1).

Since TKI treated CML patients have a near-normal life expectancy two important issues must be considered in the future:

1. the quality of life and ethical aspects of a lifetime treatment,
2. the budget impact for healthcare providers of treating patients during lifetime.

One of the best ways to consider these two points is to ask the question about stopping TKI in good responder patients. We first reported a pilot study where imatinib was withdrawn in 12 CML patients treated and maintained in complete molecular remission (CMR), defined by undetectable residual disease (with sensitivity of 4.5 log) on quantitative RT-PCR, for at least two years. Then, we demonstrated in a multicenter study entitled STIM trial that imatinib could be safely discontinued in patients with CMR for at least 2 years (2). All molecular relapsing patients were sensitive when imatinib was re-challenged (3). Around 40% of these patients remain in a prolonged treatment-free remission (TFR) after treatment cessation (4). Taking into account the cost of imatinib and the number of months without treatment in STIM trial, the savings in France were estimated to be 9 million €. However, since only 40 % of patients are in treatment free remission, a study, assessing the real budget impact of stopping TKI in the eligible population seems necessary as no published study has ever addressed this question in France.

Our aim is to assess the budget impact of discontinuing TKI treatment in patients with CML in deep molecular response since at least two years, compared with treatment during lifetime, from the French healthcare system point of view. This budget impact will be expressed as a "net benefit" and will be based on the difference between total costs incurred by this strategy and total costs avoided also.

One of the originality of our study is to raise the issue of treatment cessation in the context of a chronic disease from an economic point of view.

The other originality of this study is to use a decision model to perform this French budget impact analysis of TKI discontinuation, without setting up another trial. Besides the literature review and meta-analysis; the proposed probabilistic Markov model will use direct costs (including treatment costs and all health care related costs as well as costs related to relapse) extracted mainly from the French Health Insurance Databases.

Conditions

Interventions

OTHER

Budget impact

Budget impact, using a probabilistic markov model, of discontinuing TKI treatment in patients with CML in deep molecular response since at least two years, compared with current practice (treatment during entire life), from the healthcare system point of view

Sponsors & Collaborators

  • Bordeaux PharmacoEpi

    collaborator OTHER

  • University Hospital, Bordeaux

    lead OTHER

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2016-12-07
Primary Completion
2018-10-31
Completion
2019-04-30

Countries

  • France

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT03540654 on ClinicalTrials.gov