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 | Completed

• 1 other identifier: CHUBX 2015/28
• Study Type: observational
• Target: 355
• Locations: 1 country
• Sites: 1

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

Conditions

Hematologic Diseases; Chronic Myeloid Leukemia

Outcome Measures

Primary Outcomes (1)

• Budget impact of discontinuing TKI in patients with CML achieving a complete molecular response

  Budget impact, 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), between 2008 and 2015 from the healthcare system point of view, by using a probabilistic Markov model

  Patients treated since at least 3 years and achieving deep molecular response compared with current practice (treatment during entire life), between 2008 and 2015

Study Arms (1)

Patients with CML discontinuing TKI treatment

Other: Budget impact

Interventions

Budget impact OTHER

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

Patients with CML discontinuing TKI treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

A cohort of CML patients treated with TKI will be identified in the French nationwide claims and hospital database (SNIIRAM)

You may qualify if:

• History: 3-year period;
• Patients aged 18 years or over;
• Patients with LTD registration or hospitalization for CML (primary, associated and linked ICD-10 diagnosis code, i.e. C92.1 or C921) during the study period;

You may not qualify if:

• Patients who proceeded to allogeneic or autogenic hematopoietic stem-cell transplant (hospitalization ICD-10 code diagnosis Z94.80) in the 3 year-period prior to or in the month following the last TKI reimbursement identified before TKI discontinuation;
• Patients with HIV/AIDS (hospitalization ICD-10 code diagnosis B24) or chronic Hepatitis C or B (hospitalization ICD-10 code diagnosis B18) in the 3 year-period prior to or in the month following the last TKI reimbursement identified before TKI discontinuation;
• Recent (i.e. in the previous year) or ongoing pregnancy at TKI discontinuation date identified by an algorithm based on codes of hospitalization diagnoses and medical procedures.
• Causes of TKI discontinuation will be further investigated in the cohort in order to conduct analyses only patients with a TKI discontinuation due to a complete molecular response.

Sponsors & Collaborators

• University Hospital, Bordeaux: lead
• Bordeaux PharmacoEpi: collaborator

Study Sites (1)

CHU Bordeaux

Bordeaux, France

Location

MeSH Terms

Conditions

Hematologic Diseases; Leukemia, Myelogenous, Chronic, BCR-ABL Positive

Condition Hierarchy (Ancestors)

Hemic and Lymphatic Diseases; Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Myeloproliferative Disorders; Bone Marrow Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 9, 2018
• First Posted: May 30, 2018
• Study Start: December 7, 2016
• Primary Completion: October 1, 2018
• Study Completion: April 1, 2019
• Last Updated: April 28, 2020

Record last verified: 2020-04

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)