Trial Outcomes & Findings for The EMPATHY Pilot Study (NCT NCT04384848)

Last Updated: 2024-03-04

Results Overview

Adherence to Refills and Medications Scale (ARMS). The ARMS-7 consists of seven items evaluating adherence to taking medications and refilling prescriptions. Items are rated on a four-point Likert scale ranging between 7 and 28, with higher scores indicating lower medication adherence.

Recruitment status

COMPLETED

Study phase

Target enrollment

94 participants

Primary outcome timeframe

6 months

Results posted on

2024-03-04

Participant Flow

Participant milestones

Participant milestones
Measure	Chronic Myeloid Leukemia (CML) Patients CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the Patient Reported Outcome (PRO-CTCAE) Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Overall Study STARTED	94
Overall Study COMPLETED	90
Overall Study NOT COMPLETED	4

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

The EMPATHY Pilot Study

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Chronic Myeloid Leukemia (CML) Patients n=94 Participants CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Age, Continuous	56.92 years STANDARD_DEVIATION 14.35 • n=93 Participants
Sex: Female, Male Female	42 Participants n=93 Participants
Sex: Female, Male Male	52 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	1 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	1 Participants n=93 Participants
Race (NIH/OMB) White	81 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	11 Participants n=93 Participants
Region of Enrollment United States	16 participants n=93 Participants
Region of Enrollment Italy	78 participants n=93 Participants

PRIMARY outcome

Timeframe: 6 months

Population: 94 patients were consented. 93 patients provided baseline information. 83 patients provided adherence outcome data

Outcome measures

Outcome measures
Measure	Chronic Myeloid Leukemia (CML) Patients n=83 Participants CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Medication Adherence	7.81 score on a scale Standard Deviation 1.31

PRIMARY outcome

Timeframe: 6 months

Population: although 94 patients signed consent, one withdrew prior to baseline and so the number of participants analyzed is 93

Prescription refill data extracted from hospital pharmacy records. Prescription refill data were evaluated over 6 months. Missed refills, for reasons other than mortality or physician change of medication, were counted as non-adherence. Adherence was defined as the number of days medicine not available between each refill over the number of days between first prescription and last refill during the study period.

Outcome measures

Outcome measures
Measure	Chronic Myeloid Leukemia (CML) Patients n=93 Participants CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Number of Participants Adherent To Their Medication	86 Participants

SECONDARY outcome

Timeframe: 6 months

Population: 94 patients were consented. 93 patients provided baseline information. 84 patients provided adherence outcome data

Functional Assessment of Cancer Therapy-General. The possible score range of this measure is 0-128. Higher scores denote better quality of life

Outcome measures

Outcome measures
Measure	Chronic Myeloid Leukemia (CML) Patients n=84 Participants CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Health-Related Quality of Life	81.4 score on a scale Interval 78.4 to 84.3

SECONDARY outcome

Timeframe: 6 months

Population: 94 patients were consented. 93 patients provided baseline information. 84 patients provided adherence outcome data

Functional Assessment of Chronic Illness Therapy-Fatigue. the possible score range of this measure is 0-52. Higher scores reflect less fatigue

Outcome measures

Outcome measures
Measure	Chronic Myeloid Leukemia (CML) Patients n=84 Participants CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Fatigue	41.9 score on a scale Interval 40.0 to 43.8

SECONDARY outcome

Timeframe: 6 months

Population: 94 patients were consented. 93 patients provided baseline information. 81 patients provided adherence outcome data

Cytogenetic response (CyR) is defined based on the percentage of Ph pos metaphases, as evaluated by chromosome banding analysis of at least 20 marrow cell metaphases (cytogenetics). Complete CyR is when the percentage of Ph pos metaphases is 0; Partial CyR is when the percentage of Ph pos metaphases ranges from 1 to 35, Minor CyR if the percentage of Ph pos metaphases ranges from 36 to 65, minimal CyR if the percentage of Ph pos metaphases ranges from 66 to 95, No CyR if the percentage of Ph pos metaphases is higher than 95.

Outcome measures

Outcome measures
Measure	Chronic Myeloid Leukemia (CML) Patients n=81 Participants CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Cytogenetic Response	61 Participants

Adverse Events

Chronic Myeloid Leukemia (CML) Patients

Serious events: 13 serious events

Other events: 0 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	Chronic Myeloid Leukemia (CML) Patients n=94 participants at risk CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy EMPATHY Pilot: The goal of this pilot is to develop and pilot a tailored monitoring intervention targeting symptomatic, Patient-reported adverse events (AEs) in chronic myeloid leukemia (CML) Patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy. The tailored monitoring intervention will draw primarily from the PRO-CTCAE Item Library, with additional items drawn from the FACIT and EORTC Item libraries as necessary. After identifying the full set of AEs to be monitored, we will load the Patient assessment and report program into tablets for electronic administration in the busy clinic setting. We will then pilot a six-month intervention aimed to monitor and manage emerging AEs, coupled with assessment of intervention feasibility, Patient acceptability and satisfaction, provider acceptability and clinical management utility, adherence to TKI therapy, and HRQoL. We expect that adherence to therapy, clinical response, and HRQoL will be enhanced by such an intervention.
Gastrointestinal disorders abdominal pain	1.1% 1/94 • 12 months
Immune system disorders Neutropenia	5.3% 5/94 • 12 months
Blood and lymphatic system disorders thrombocytopenia	4.3% 4/94 • 12 months
Immune system disorders neutropenia	6.4% 6/94 • 12 months
Cardiac disorders myocardial infarction	1.1% 1/94 • 12 months

Other adverse events

Adverse event data not reported

Additional Information

David Cella

Northwestern University

Phone: 312-503-1725

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place