The EMPATHY Pilot Study

February 28, 2024 updated by: David Cella, Northwestern University

Evaluating Patient-Reported Outcomes Monitoring in Routine Care of Patients With Chronic Myeloid Leukemia for Increasing Adherence and Clinical Response to THerapY: The EMPATHY Pilot Study

The proposed study, may significantly contribute to improve healthcare delivery in patients with Chronic Myeloid Leukemia (CML) treated with modern tyrosine kinase inhibitors (TKIs) in two ways. First, it may provide novel empirical data on the positive effects of systematically monitoring of patient-reported adverse events (AEs) in routine practice for improving symptom management and adherence to therapy. Second, it will inform the development of a large international randomized controlled trial (RCT) to test whether systematic collection of patient-reported AEs, could improve clinical response to TKI therapy.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Detailed Description

The evolution in the understanding of the biology of Chronic Myeloid Leukemia (CML), that eventually translated into highly effective molecular targeted therapies, is unparalleled in cancer medicine. This knowledge led the development of a number of orally active molecule tyrosine kinase inhibitors (TKIs) that have dramatically improved clinical outcomes. In less than two decades, the 10-year survival probability increased from 20 to 53% with previous (IFN)-therapies to about 90% in the TKI era. Life expectancy of these patients now approaches that of the general population. While oral TKIs are now the standard of care for CML, it should be considered that therapy is lifelong and patients are requested to take medication on a daily basis. Importantly, there is convincing evidence that full adherence to therapy is a critical factor to obtain and maintain an optimal response to therapy. However, non-adherence is a major challenge in CML, since side effects induced by these drugs negatively impact on patient's quality of life (QoL), seriously undermine full adherence with treatment schedule and thereby often lead to sub-optimal clinical responses to drugs. From previous Preliminary Data we extrapolated the following evidences: Systematic monitoring of Patient-Reported Outcomes (PRO) in routine care has several advantages, such as improved symptom control, enhanced patient-physician communication, as well as patient satisfaction and wellbeing. Furthermore, it was found that Adverse Events (AEs) are the most frequent cause for non-adherence to CML therapy and that even experienced physicians tend to underestimate burden of TKIs of their patients. This mismatch might have major clinical implications in disease management, as physicians might not be able to early identify those patients who might be at heightened risk of poor adherence behavior. Given these findings, we hypothesized that systematic electronic monitoring of Patient-Reported AEs in CML routine practice may improve adherence to therapy, quality of life, and clinical response to therapy. This hypothesis will be addressed in the experiments of the following Specific Aims: 1) To develop an online platform for systematic monitoring of patient-reported AE assessment that is tailored to the unique demands of TKI therapy for CML. 2) To assess patient and physician acceptability and satisfaction with use of this platform in CML routine practice and evaluate its value in improving symptom management, quality of life, adherence to therapy as well as preliminary efficacy. We anticipate this study will provide unprecedented information on the value of systematically collecting patient-reported AEs information in CML routine care. If positive, our results will inform the development of large international randomized controlled trial (RCT) to investigate whether this experimental approach can improve depth and rates of clinical responses to TKI therapies in CML patients.

Dr. Fabio Efficace is also a Principal Investigator on this study. He is also a Professor in the Department of Medical Social Sciences at Northwestern University, Feinberg School of Medicine.

Study Type

Interventional

Enrollment (Actual)

94

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Italy
      • Bologna, Italy
        • Policlinico Sant'Orsola Malpighi - UOC Ematologia - Azienda Ospedaliero-Universitaria di Bologna
      • Cagliari, Italy
        • Ospedale "A. Businco" - Struttura Complessa di Ematologia e CTMO - Azienda Ospedaliera G. Brotzu
      • Milano, Italy
        • Milano Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - UOC Oncoematologia - Padiglione Marcora
      • Napoli, Italy
        • Azienda Ospedaliera Universitaria - Università degli Studi di Napoli "Federico II" - UOC Ematologia
      • Novara, Italy
        • Azienda Ospedaliera Universitaria Maggiore della Carità di Novara - SCDU Ematologia
      • Reggio Emilia, Italy
        • AUSL Reggio Emilia - Arcispedale S. Maria Nuova, IRCSS - SC Ematologia
      • Roma, Italy
        • Azienda Ospedaliera Universitaria Policlinico Umberto I - Università degli Studi "Sapienza" - UOC Ematologia
      • Roma, Italy
        • Ospedale Sant'Eugenio
      • Rome, Italy, 00161
        • The GIMEMA Foundation (Italian Group for Adult Hematologic Diseases)
      • Torino, Italy
        • Ospedale Mauriziano Umberto I - Torino - Scdu Ematologia
      • Torino, Italy
        • Azienda Ospedaliero-Universitario Città della Salute e della Scienza di Torino - Ospedale S. Giovanni Battista Molinette - SC Ematologia
      • Udine, Italy
        • Asui Di Udine - Presidio Ospedaliero "Santa Maria Della Misericordia" - Clinica Ematologica
      • Verona, Italy
        • Azienda Ospedaliera Universitario Integrata di Verona, Policlinico G.B. Rossi - UOC Ematologia
      • Vicenza, Italy
        • USL 6 - Ospedale San Bortolo - Vicenza
  • United States
    • Georgia
      • Augusta, Georgia, United States, 30912
        • Augusta University, Hematology and Oncology
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Northwestern University, Robert H. Lurie Comprehensive Cancer Center Comprehensive Cancer Center (RHLCCC)

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Diagnosis of Philadelphia chromosome positive and/or BCR-ABL positive CML confirmed by cytogenetic and/or molecular analysis;
  2. Newly diagnosed chronic phase (CP)-CML Patients planned to receive one of the following TKI approved as first line treatment: imatinib, dasatinib, nilotinib or bosutinib;
  3. Adult Patients (≥18 years) at the time of study entry; Children under the age of 18 will be excluded from the study. The exclusion of children is justified by the following circumstances: a) The condition is relatively rare in children, as compared to adults; b) Issues of study preclude direct applicability of hypotheses and/or interventions to both adults and children.
  4. Written informed consent.
  5. Written informed consent from Patient's physician as a participant.
  6. Newly diagnosed Chronic Phase (CP)-CML Patients who are within 4 weeks of first line TKI therapy (anyone of the TKI approved in the USA and Europe, that is: imatinib, dasatinib, nilotinib or bosutinib).
  7. Ability to read/converse in English (Northwestern University and Augusta University Sites). Ability to read/converse in Italian (GIMEMA Centers).

Patient exclusion criteria will include:

  1. Major cognitive deficits or psychiatric problems hampering a self-reported evaluation;
  2. Having received any CML treatment - other than TKI - for more than 3 months prior to receiving current TKI therapy.

This project will focus on CML, which affect both men and women. Therefore, there are no exclusion/inclusion criteria based on sex/gender. In addition, there are no exclusion/inclusion criteria based on race and ethnicity.

Physician inclusion criteria will include:

1)Provider of clinical care for Patient who meets inclusion criteria for the study

Please note that Physician consent is requisite for the Patient to be enrolled as a participant in the study.

Exclusion Criteria:

  1. Major cognitive deficits or psychiatric problems hampering a self-reported evaluation
  2. Having received any CML treatment prior to therapy with imatinib, dasatinib, bosutinib or nilotinib for more than three months

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Supportive Care
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Chronic myeloid leukemia (CML) patients
CML patients undergoing first-line tyrosine kinase inhibitor (TKI) therapy
Other: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Medication Adherence
Time Frame: 6 months
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.
6 months
Number of Participants Adherent To Their Medication
Time Frame: 6 months
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.
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Health-Related Quality of Life
Time Frame: 6 months
Functional Assessment of Cancer Therapy-General. The possible score range of this measure is 0-128. Higher scores denote better quality of life
6 months
Fatigue
Time Frame: 6 months
Functional Assessment of Chronic Illness Therapy-Fatigue. the possible score range of this measure is 0-52. Higher scores reflect less fatigue
6 months
Cytogenetic Response
Time Frame: 6 months
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.
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Northwestern University

Collaborators

National Cancer Institute (NCI)
Augusta University
Fondazione GIMEMA - Franco Mandelli Onlus

Investigators

  • Principal Investigator: David Cella, PhD, Northwestern University
  • Principal Investigator: Fabio Efficace, PhD, Fondazione GIMEMA - Franco Mandelli Onlus

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2019

Primary Completion (Actual)

June 10, 2022

Study Completion (Actual)

July 31, 2022

Study Registration Dates

First Submitted

April 17, 2020

First Submitted That Met QC Criteria

May 7, 2020

First Posted (Actual)

May 12, 2020

Study Record Updates

Last Update Posted (Estimated)

March 4, 2024

Last Update Submitted That Met QC Criteria

February 28, 2024

Last Verified

February 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 1R21CA230367-01A1 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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