Patients’ priorities in selecting chronic lymphocytic leukemia treatments

Mansfield, Carol; Masaquel, Anthony; Sutphin, Jessie; Weiss, Elisa S.; Gutierrez, Meghan; Wilson, Jennifer G.; Boeri, Marco; Li, Jia; Reyes, Carolina

doi:10.1182/bloodadvances.2017007294

Cited by 34 publications

(41 citation statements)

References 22 publications

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“…Patients in the early stages typically do not receive treatment, and upon disease progression to intermediate and advanced stages, various therapeutic options including chemotherapy, immunotherapy, targeted drug therapy, as well as bone marrow transplantation are applied. Despite major advances in therapeutic approaches, CLL still remains an incurable disease due to the short-term benefit of present modalities and high relapse rates [2-5]. One possible explanation for inefficient therapy in CLL is the inability of patients to mount potent, effective, and durable immune responses against cancerous cells [6].…”

Section: Introductionmentioning

confidence: 99%

Dysregulated Expression of Tim-3 and NKp30 Receptors on NK Cells of Patients with Chronic Lymphocytic Leukemia

et al. 2019

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Background: In this study, the expression pattern of NKp30 and T cell immunoglobulin and mucin domain-containing molecule-3 (Tim-3), as candidates for activating and inhibitory receptors of NK cells, were evaluated in patients with chronic lymphocytic leukemia (CLL). Patients and Methods: 24 CLL patients and 19 healthy controls were enrolled. Fresh peripheral blood was collected from all subjects and stained with fluorochrome-conjugated antibodies. The frequency of CD56+/CD3–/NKp30+ and CD56+/CD3–/Tim-3+ cells was determined by multicolor flow cytometry. Results: Our results revealed that Tim-3 is significantly upregulated on natural killer (NK) cells of CLL patients in comparison to healthy controls. NK cells of CLL patients showed lower expression of NKp30-activating receptor compared to controls. Tim-3 expression pattern on NK cells of CLL patients was correlated with poor prognostic factors including low hemoglobin level, high absolute lymphocyte count, and high serum C-reactive protein level. Conclusion: Dysregulated expression of Tim-3 and NKp30 receptors confirms the exhaustion state of NK cells in CLL. Our data introduce Tim-3 as a promising biomarker and potential target for immunotherapy of CLL.

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Section: Introductionmentioning

confidence: 99%

Dysregulated Expression of Tim-3 and NKp30 Receptors on NK Cells of Patients with Chronic Lymphocytic Leukemia

et al. 2019

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“…However, patients recruited to participate in preference research by these organizations can often only self-report their diagnosis or symptoms. 19 , 20 In addition, patients recruited through patient organizations may include fewer racial and ethnic minorities and be of higher socioeconomic status than the overall population of patients.…”

Section: Discussionmentioning

confidence: 99%

Parkinson’s Patients’ Tolerance for Risk and Willingness to Wait for Potential Benefits of Novel Neurostimulation Devices: A Patient-Centered Threshold Technique Study

Hauber

Mange

Zhou

et al. 2021

MDM Policy & Practice

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Background. Parkinson’s disease (PD) is neurodegenerative, causing motor, cognitive, psychological, somatic, and autonomic symptoms. Understanding PD patients’ preferences for novel neurostimulation devices may help ensure that devices are delivered in a timely manner with the appropriate level of evidence. Our objective was to elicit preferences and willingness-to-wait for novel neurostimulation devices among PD patients to inform a model of optimal trial design. Methods. We developed and administered a survey to PD patients to quantify the maximum levels of risks that patients would accept to achieve potential benefits of a neurostimulation device. Threshold technique was used to quantify patients’ risk thresholds for new or worsening depression or anxiety, brain bleed, or death in exchange for improvements in “on-time,” motor symptoms, pain, cognition, and pill burden. The survey elicited patients’ willingness to wait to receive treatment benefit. Patients were recruited through Fox Insight, an online PD observational study. Results. A total of 2740 patients were included and a majority were White (94.6%) and had a 4-year college degree (69.8%). Risk thresholds increased as benefits increased. Threshold for depression or anxiety was substantially higher than threshold for brain bleed or death. Patient age, ambulation, and prior neurostimulation experience influenced risk tolerance. Patients were willing to wait an average of 4 to 13 years for devices that provide different levels of benefit. Conclusions. PD patients are willing to accept substantial risks to improve symptoms. Preferences are heterogeneous and depend on treatment benefit and patient characteristics. The results of this study may be useful in informing review of device applications and other regulatory decisions and will be input into a model of optimal trial design for neurostimulation devices.

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“…The empirical application is based on a study by Mansfield et al (2017). The survey instrument included screening questions and informed consent, followed by disease experience and treatment experience questions.…”

Section: Methodsmentioning

confidence: 99%

“…Example discrete choice experiment question (a) and the two follow‐up questions with the cost attribute (b and c) (Mansfield et al, 2017) [Colour figure can be viewed at wileyonlinelibrary.com]…”

Section: Methodsmentioning

confidence: 99%

Assessing the impact of excluded attributes on choice in a discrete choice experiment using a follow‐up question

Mansfield

Sutphin

Boeri³

2020

Health Economics

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Health researchers design discrete choice experiments (DCEs) to elicit preferences over attributes that define treatments. A DCE can accommodate a limited number of attributes selected by researchers based on numerous factors (e.g., respondent comprehension, cognitive burden, and sample size). For situations where researchers want information about the possible impact of an attribute excluded from the DCE, we propose a method to use a question after the DCE. This follow‐up question includes the attributes in the DCE with fixed levels and an additional attribute originally excluded from the DCE. The DCE data can be used to predict the probability that respondents would select one treatment profile over another without the additional attribute. Comparing the prediction to the percentage of the sample who selected each profile when it includes the additional attribute provides information on the potential impact of the additional attribute. We provide an example using data from a DCE on treatments for chronic lymphocytic leukemia. Cost was excluded from the DCE, but the survey included a follow‐up question with two fixed treatment profiles, similar to two treatments currently on the market, and a cost for each. Preferences were sensitive to modest changes in cost, highlighting the importance of gathering this information.

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Patients’ priorities in selecting chronic lymphocytic leukemia treatments

Abstract: Key Points CLL patients value higher PFS but would accept significant reductions in PFS to avoid serious adverse events. Adding even modest out-of-pocket costs changed treatment choices for hypothetical treatments, suggesting patients are sensitive to cost.

Cited by 34 publications

References 22 publications

Dysregulated Expression of Tim-3 and NKp30 Receptors on NK Cells of Patients with Chronic Lymphocytic Leukemia

Dysregulated Expression of Tim-3 and NKp30 Receptors on NK Cells of Patients with Chronic Lymphocytic Leukemia

Parkinson’s Patients’ Tolerance for Risk and Willingness to Wait for Potential Benefits of Novel Neurostimulation Devices: A Patient-Centered Threshold Technique Study

Assessing the impact of excluded attributes on choice in a discrete choice experiment using a follow‐up question

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