Clinical trial NCT04384848: The EMPATHY Pilot Study
Official title: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Leukemia, Chronic Myeloid
Cancer
Keywords: Cancer
Leukemia, Chronic Myeloid
Health-Related Quality Of Life
Patient Reported Outcome
Lead sponsor: Northwestern University
Sponsor class: OTHER
Collaborators: name: National Cancer Institute (NCI)
name: Fondazione GIMEMA
name: Augusta University
First posted: 2020-05-12
Enrollment: 94
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: SUPPORTIVE_CARE

Brief summary: 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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Medication Adherence
timeFrame: 6 months
measure: Number of Participants Adherent To Their Medication
timeFrame: 6 months
Secondary outcomes:
measure: Health-Related Quality of Life
timeFrame: 6 months
measure: Fatigue
timeFrame: 6 months
measure: Cytogenetic Response
timeFrame: 6 months
Other outcomes:
Locations:
city: Augusta
country: United States
facility: Augusta University, Hematology and Oncology
state: Georgia
city: Chicago
country: United States
facility: Northwestern University, Robert H. Lurie Comprehensive Cancer Center Comprehensive Cancer Center (RHLCCC)
state: Illinois
city: Bologna
country: Italy
facility: Policlinico Sant'Orsola Malpighi - UOC Ematologia - Azienda Ospedaliero-Universitaria di Bologna
city: Cagliari
country: Italy
facility: Ospedale "A. Businco" - Struttura Complessa di Ematologia e CTMO - Azienda Ospedaliera G. Brotzu
city: Milano
country: Italy
facility: Milano Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico - UOC Oncoematologia - Padiglione Marcora
city: Napoli
country: Italy
facility: Azienda Ospedaliera Universitaria - Università degli Studi di Napoli "Federico II" - UOC Ematologia
city: Novara
country: Italy
facility: Azienda Ospedaliera Universitaria Maggiore della Carità di Novara - SCDU Ematologia
city: Reggio Emilia
country: Italy
facility: AUSL Reggio Emilia - Arcispedale S. Maria Nuova, IRCSS - SC Ematologia
city: Roma
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico Umberto I - Università degli Studi "Sapienza" - UOC Ematologia
city: Roma
country: Italy
facility: Ospedale Sant'Eugenio
city: Rome
country: Italy
facility: The GIMEMA Foundation (Italian Group for Adult Hematologic Diseases)
city: Torino
country: Italy
facility: Azienda Ospedaliero-Universitario Città della Salute e della Scienza di Torino - Ospedale S. Giovanni Battista Molinette - SC Ematologia
city: Torino
country: Italy
facility: Ospedale Mauriziano Umberto I - Torino - SCDU Ematologia
city: Udine
country: Italy
facility: ASUI di Udine - Presidio Ospedaliero "Santa Maria della Misericordia" - Clinica Ematologica
city: Verona
country: Italy
facility: Azienda Ospedaliera Universitario Integrata di Verona, Policlinico G.B. Rossi - UOC Ematologia
city: Vicenza
country: Italy
facility: USL 6 - Ospedale San Bortolo - Vicenza
Study officials:
affiliation: Northwestern University
name: David Cella, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Fondazione GIMEMA
name: Fabio Efficace, PhD
role: PRINCIPAL_INVESTIGATOR
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