Medication adherence, molecular monitoring, and clinical outcomes in patients with chronic myelogenous leukemia in a large HMO

Haque, Reina; Shi, Jiaxiao; Chung, Joanie; Xu, Xiaoqing; Avila, Chantal; Campbell, Christopher; Ahmed, Syed; Chen, Lei; Schottinger, Joanne E.

doi:10.1016/j.japh.2017.01.004

Cited by 23 publications

(49 citation statements)

References 17 publications

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“…In one study, comparing 3 to 4 annual qPCR tests with no annual qPCR tests was found to yield a significant reduction in disease progression . However, in another study, 1 to 2 annual qPCR tests did not appear to reduce disease progression significantly . The results of the current study provide greater granularity to these findings in that even one tPCR may lead to better outcomes for patients.…”

Section: Discussionmentioning

confidence: 49%

“…8 However, in another study, 1 to 2 annual qPCR tests did not appear to reduce disease progression significantly. 14 The results of the current study provide greater granularity to these findings in that even one tPCR may lead to better outcomes for patients. It is interesting to note that the results of the current study demonstrate that patients who have timely monitoring also tend to have more frequent monitoring.…”

Section: Discussionmentioning

confidence: 73%

“…Cancer February 15, 2019 To the best of our knowledge, 2 studies to date have examined whether frequent molecular monitoring is associated with better outcomes in patients with CML. 8,14 In one study, comparing 3 to 4 annual qPCR tests with no annual qPCR tests was found to yield a significant reduction in disease progression. 8 However, in another study, 1 to 2 annual qPCR tests did not appear to reduce disease progression significantly.…”

Section: Discussionmentioning

confidence: 99%

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Molecular monitoring of therapeutic milestones and clinical outcomes in patients with chronic myeloid leukemia

Klil-Drori

Yin

Azoulay

et al. 2018

Cancer

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Background In the current study, the authors determined whether adhering to molecular monitoring guidelines in patients with chronic myeloid leukemia (CML) is associated with major molecular response (MMR) and assessed barriers to adherent monitoring. Methods Newly treated patients with CML from the Quebec province‐wide CML registry from 2005 to 2016 were included. Timely polymerase chain reaction (tPCR) was defined as the molecular assessment of BCR‐ABL1 at the 3‐month, 12‐month, and 18‐month time points from the initiation of tyrosine kinase inhibitor (TKI) therapy. The cohort was analyzed as a nested case‐control study. Cases with a first‐ever MMR (BCR‐ABL1 ≤0.1%, assessed at any time during follow‐up) were matched to up to 5 controls by duration of TKI therapy, volume of patients with CML at the treatment center, year of cohort entry, and age. Odds ratios (ORs) for the performance of tPCR and MMR were adjusted for sex, comorbidities, type of TKI, and other important covariates. Results The cohort included 496 patients. Of 392 MMR events, 67.9% occurred before 18 months. The performance of tPCR was associated with a doubling of the MMR rate (OR, 2.23; 95% confidence interval [95% CI], 1.56‐3.21) and was similar with 1 to 3 tPCRs performed (P = .67). Furthermore, tPCRs at 3 months (OR, 2.77; 95% CI, 1.81‐4.23) and 12 months (OR, 3.00; 95% CI, 1.64‐5.49) were associated with achieving early MMR, whereas tPCRs at 18 months were not (OR, 1.23; 95% CI, 0.80‐1.89). Low‐volume centers were found to have lower adherence to tPCR (OR, 0.60; 95% CI, 0.40‐0.89). Conclusions Timely molecular assessment at 3 months and 12 months appears to benefit patients with CML. Adherence to timely monitoring should be encouraged, especially in low‐volume treatment centers.

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Section: Discussionmentioning

confidence: 49%

Section: Discussionmentioning

confidence: 73%

Section: Discussionmentioning

confidence: 99%

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Molecular monitoring of therapeutic milestones and clinical outcomes in patients with chronic myeloid leukemia

Klil-Drori

Yin

Azoulay

et al. 2018

Cancer

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“…Another potential approach for an OCM to reduce the total cost of care of CML patients might be to improve the frequency of molecular monitoring, since several studies have reported routine molecular monitoring is associated with improved patient outcomes, which could lead to a reduction in healthcare costs [27][28][29][30][31][32][33] . Two separate studies have also reported cost reductions associated with earlier and more frequent monitoring of CML patients to proactively manage TKI treatment responses 31,34 .…”

Section: Discussionmentioning

confidence: 99%

Economic modeling to evaluate the impact of chronic myeloid leukemia therapy management on the oncology care model in the US

Jabbour

Mendiola

Lingohr-Smith³

et al. 2019

Journal of Medical Economics

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Objective: To develop an economic model to evaluate changes in healthcare costs driven by restricting usage of branded tyrosine kinase inhibitors (TKIs) through substitution with generic imatinib among chronic myeloid leukemia (CML) patients in a typical Oncology Care Model (OCM) practice, and examine the impact on Performance-Based Payment (PBP) eligibility. Methods: An Excel-based economic model of an OCM practice with 1,000 cancer patients during a 6-month episode of care was developed. Cancer types and proportions of patients treated in the practice were estimated from an OCM report. All-cause healthcare costs were obtained from published literature. It was assumed that if a practice restricts usage of branded TKIs for newly-diagnosed CML patients, 80% of the market share of branded imatinib and 50% of the market shares of 2 nd-gen TKIs would shift to generic imatinib. Among established TKI-treated patients, it was assumed that 80% of the market share of branded imatinib and no patients treated with 2 nd-gen TKIs would shift to the generic. Results: Four CML patients were estimated for a 1,000-cancer patient OCM practice with a total baseline healthcare cost of $51,345,812 during a 6-month episode. If the practice restricts usage of branded TKIs, the shift from 2 nd-gen TKIs to generic imatinib would reduce costs by $12,970, while shifting from branded to generic imatinib lowers costs by $25,250 during a 6-month episode. Minimum reductions of $3,013,832 in a one-sided risk model and $2,372,010 in a two-sided risk model are required for PBP eligibility; the shift from 2 nd-gen TKIs to generic imatinib would account for 0.4% and 0.5% of the savings required for a PBP, respectively. Conclusions: This analysis indicates that the potential cost reduction associated with restricting branded TKI usage among CML patients in an OCM setting will represent only a small proportion of the cost reduction needed for PBP eligibility.

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“…Tolerability Detailed data on adverse events occurring early during treatment 22 Treatment discontinuation 22,25,26 Long-term treatment side effects and complications 39 CML research studies Response Molecular, cytogenetic, and hematologic response rates at discrete time points 22,25,26 Kinetics of reduction in BCR-ABL1 transcript levels 40 Assessment of whether a patient meets designated treatment response landmarks (eg, target response levels at 3, 6, and 12 months from the start of treatment) 40 Rates of CML progression at early time points 40 Development of evidence supporting new treatment practices, such as criteria for attempting treatment-free remission 34,41e47 Long-term treatment effects 27 Long-term rates of CML progression 32 Rates of treatment failure in clinical practice 14,30 Analysis of treatment switching patterns, including rates of treatment switch due to treatment failure 14,30 Analysis of treatment-free remission practices and outcomes in clinical practice 48 Outcomes in patients not typically included in RCTs 29,30 Response rates in clinical practice 31 Real-world disease management practices 31…”

Section: Qol 38mentioning

confidence: 99%

The Case for Real-world Evidence in the Future of Clinical Research on Chronic Myeloid Leukemia

Webster

Smith

2019

Clinical Therapeutics

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Purpose: In light of recently published guidelines from the US Food and Drug Administration (FDA) on the communication of real-world data (RWD) and real-world evidence (RWE) to support regulatory decision making, it is important to understand how such data are developed, the limitations of these data, and how to best use RWD to improve patient care. Historically, the use of RWE has been approached with skepticism because of its often-retrospective nature compared with data from conventional randomized controlled trials (RCTs). This review discusses the role and function of RWE and RWD in clinical research. We summarize the types of RWE used in clinical research, outline the challenges and limitations involved with these data, and suggest how these types of analyses can supplement results from clinical trials to foster a more complete understanding of a drug or disease area of interest. In particular, we focus on the role of RWE in investigating chronic myeloid leukemia (CML) and tyrosine kinase inhibitor therapy for CML. Methods: We reviewed FDA guidance on the use of RWE and conducted a PubMed literature search to evaluate published data from real-world studies in CML. Findings: RWE includes analysis of RWD gathered from nonconventional sources, including patient registries, observational studies, and social media, among others. Importantly, although real-world studies do not adhere to the same degree of controlled conditions and predefined patientmanagement strategies as do conventional clinical trials, analyses resulting from these studies can be held to a high degree of validation and standardization, making them as meaningful as those from RCTs. In CML, RWE has informed early treatment milestones and has provided a window into patient perspectives regarding treatment. These types of analyses have already informed and can continue to inform disease management. These improvements in disease management, in turn, will help clinicians to better forecast treatment challenges and allow for the optimization of future treatment paradigms. Implications: Real-world studies are different from conventional RCTs and therefore provide insight into distinct aspects of treatment and patient outcomes. Together with results from clinical trials, RWE can help to illustrate a more complete picture of the tolerability, effectiveness, and impact of a drug. The recently published guidelines indicate that the FDA expects a growing role for RWE. (Clin Ther.

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Medication adherence, molecular monitoring, and clinical outcomes in patients with chronic myelogenous leukemia in a large HMO

Abstract: Our results suggest that close clinical monitoring, which includes PCR monitoring in patients with high and low TKI drug adherence, is associated with a lower risk of progression or mortality.

Cited by 23 publications

References 17 publications

Molecular monitoring of therapeutic milestones and clinical outcomes in patients with chronic myeloid leukemia

Molecular monitoring of therapeutic milestones and clinical outcomes in patients with chronic myeloid leukemia

Economic modeling to evaluate the impact of chronic myeloid leukemia therapy management on the oncology care model in the US

The Case for Real-world Evidence in the Future of Clinical Research on Chronic Myeloid Leukemia

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