A Disease Registry of Patients With Mantle Cell Lymphoma

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to create a patient registry in order to assess treatment patterns, physician reported clinical outcomes and patient-reported health-related quality of life among patients diagnosed with Mantle Cell Lymphoma (MCL) who newly initiated a novel therapy in the past 6 months and whose treatment is ongoing at the time of enrollment.

Detailed Description
-----------------
Newer targeted therapies (monotherapy or in combination with other agents) have been recently approved in the United States for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least 1 prior therapy. The approval of these newer therapies will have an impact on the treatment patterns, toxicity patterns, and outcomes in the MCL population. A prospective, observational study will help to better understand the evolving real-world treatment outcomes (including treatment patterns, reasons for discontinuation/dose reduction, treatment interruption or treatment switches), physician-reported clinical outcomes, and patient-reported symptoms and health-related quality of life (HRQoL) among patients diagnosed with Mantle Cell Lymphoma (MCL) who newly initiated a novel therapy in the past 6 months and whose treatment is ongoing at the time of enrollment.

Official Title
-----------------
Treatment Patterns, Outcomes, and Patient-Reported Health-Related Quality of Life: A Prospective Disease Registry of Patients With Mantle Cell Lymphoma Treated With Novel Agents

Conditions
-----------------
Mantle Cell Lymphoma

Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Patient diagnosed with Mantle Cell Lymphoma (MCL) Informed consent for participation Age ≥ 18 years old, as of the first observed diagnosis of MCL Patients for whom a clinical decision has been made to initiate novel therapy in the last 6 months, limited to the following novel agent categories: Bcl-2 inhibitors BTK inhibitors Immunomodulatory agents Phosphoinositide 3-kinase inhibitors The novel agent must have been granted approval in at least one haematological cancer. Treatment must be ongoing at the time of enrollment. Exclusion Criteria: • Patient is participating in a clinical study that prohibits participation in non-interventional studies, or where treatment is blinded, at the time of consent.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years
Maximum Age: 130 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Single cohort (registry) of MCL patients<br>Patients diagnosed with MCL who have initiated a novel therapy meeting inclusion/exclusion criteria in the past 6 months and treatment is ongoing at the time of enrollment. | |

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The frequency and proportion of patients exposed to each novel agent therapy | MCL novel agent treatment types are part of the primary study objective relating to treatment patterns and will be descriptive only and use aggregated patient data. | 24 to 60 months | 
| The frequency and proportion of patients exposed to novel agent by therapy regimen | MCL treatment regimens are part of the primary study objective relating to treatment patterns and will be descriptive only and use aggregated patient data. | 24 to 60 months | 
| The frequency and proportion of patients exposed to novel agent by line of therapy | MCL treatment line is part of the primary study objective relating to treatment patterns and will be descriptive only and use aggregated patient data. | 24 to 60 months | 
| The frequency and proportion of patients exposed to novel agent by therapy class | MCL treatment class is part of the primary study objective relating to treatment patterns and will be descriptive only and use aggregated patient data. | 24 to 60 months. | 
| The rate of patients who change novel agent therapy dose (in months) | Summarizing MCL novel agent treatment dose changes are part of the primary study objective relating to treatment patterns and will be descriptive only. Time-to-dose change will be assessed. The rate of dose modification (in months) will be estimated using aggregated patient data. Summary statistics (mean, SD, median, IQR, minimum, and maximum) will be used to describe time on a particular dose, which will be measured from the start of treatment until the date of dose change or death. | 24 to 60 months. | 
| The rate of patients who interrupt novel agent therapy (in months) | Summarizing MCL treatment interruptions are part of the primary study objective relating to treatment patterns and will be descriptive only. The rate of treatment interruption (in months) will be estimated using aggregated patient data. Summary statistics (mean, SD, median, IQR, minimum, and maximum) will be used to describe time on therapy, which will be measured from the start of treatment until the date of interruption or death. | 24 to 60 months | 
| The rate of patients who discontinue novel agent therapy (in months) | Summarizing MCL treatment discontinuations are part of the primary study objective relating to treatment patterns and will be descriptive only. The rate of treatment discontinuation (in months) will be estimated using aggregated patient data. Reasons for discontinuations will be collected and summarized categorically. Summary statistics (mean, SD, median, IQR, minimum, and maximum) will be used to describe time on therapy, which will be measured from the start of treatment until the date of discontinuation or death. | 24 to 60 months | 
| The duration of MCL treatment | Summarizing MCL treatment duration is part of the primary study objective relating to treatment patterns and will be descriptive only. The duration and number of cycles of each targeted treatment (mean, SD, median, IQR, minimum, and maximum) will be summarized. Summary statistics (mean, SD, median, IQR, minimum, and maximum) will be used to describe time on therapy (in months), which will be measured from the start of treatment until the date of discontinuation, interruption, switch or death. | 24 to 60 months | 
| Estimate the overall response rate (ORR) among patients diagnosed with MCL and initiating treatment with novel therapies | ORR will be measured as the frequency and proportion of patients with a complete or partial response based on physician assessment during the observation period. | 24 to 60 months. | 
| Estimate the complete response rate (CR) among patients diagnosed with MCL and initiating treatment with novel therapies. | The CR will be calculated as the frequency and proportion of patients with a complete response based on physician assessment during the observation period. | 24 to 60 months. | 
| Estimate progression-free survival (PFS) among patients diagnosed with MCL and initiating treatment with novel therapies. | All survival outcome measures will be descriptive only. PFS will be calculated as the time from the start of novel MCL treatment until progression or death. Summary statistics (mean, median, SD, IQR, minimum and maximum) will be used to describe PFS. Kaplan-Meier curves will be used to graphically show PFS. | 24 to 60 months | 
| Estimate event-free survival (EFS) among patients diagnosed with MCL and initiating treatment with novel therapies. | All survival outcome measures will be descriptive only. EFS will be calculated as the time from the start of novel MCL treatment to disease progression, death, or discontinuation of treatment for any reason (eg, toxicity, patient preference, or initiation of a new treatment without documented progression). | 24 to 60 months | 
| Estimate overall survival (OS) among patients diagnosed with MCL and initiating treatment with novel therapies. | All survival outcome measures will be descriptive only. OS will be captured using summary statistics (mean, SD, median, IQR, minimum and maximum), which will be used to describe time from diagnosis to death, time from enrollment to death, time from index novel MCL treatment to death and time from second treatment (if applicable) to death. Kaplan-Meier curves will be used to graphically show patient survival. | 24 to 60 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The frequency of adverse events (AEs) in patients with MCL, where the term AE is used to include both serious and non-serious AEs. | The frequency of patients with AEs will be tabulated from the start of index novel MCL treatment. AEs (both in terms of the MedDRA Preferred Terms (PTs) and Common Terminology Criteria for Adverse Events [CTCAE] grade) will be listed individually by patient and described by System Organ Class (SOC) and PT. | 24 to 60 months | 
| The proportion of adverse events (AEs) in patients with MCL, where the term AE is used to include both serious and non-serious AEs. | The proportion of patients with AEs will be calculated from the start of index novel MCL treatment. AEs (both in terms of the MedDRA Preferred Terms (PTs) and Common Terminology Criteria for Adverse Events [CTCAE] grade) will be listed individually by patient and described by System Organ Class (SOC) and PT. | 24 to 60 months | 
| Estimate the frequency of serious adverse events (SAEs) in patients with MCL | The proportion and frequency patients with SAEs will be tabulated from the start of index novel MCL treatment. | 24 to 60 months | 
| Estimate the frequency of reported serious adverse safety events and adverse events leading to treatment changes associated with novel agents in patients with MCL | The proportion and frequency of all SAEs and any AEs leading to treatment changes will be tabulated overall, and will also include the frequency and proportion of: (1) drug discontinuations, (2) dose interruptions or (3) dose changes. | 24 to 60 months | 
| Estimate the frequency and proportion of patients experiencing a clinical event of interest (related to MCL or MCL treatment) | The clinical events of interest that have an economic impact will be tabulated with the frequency and proportion of patients experiencing the event. Medical interventions used for managing clinical events of interest including but not limited to: diagnostic tests, procedures and medications. | 24 to 60 months | 
| Estimate the frequency and proportion of patients experiencing healthcare resource utilization (HCRU), such as inpatient or emergency department visits | Each type of physician visit will be tabulated at the aggregate level using the mean, SD, median, IQR, minimum and maximum. The total number of visits across all facilities will also be calculated. | 24 to 60 months | 
| Tabulate HRQoL responses for the validated EORTC QLC-C30 Patient Reported Outcome (PRO) in patients with MCL | Descriptive statistics for each validated PRO will include continuous measures (mean, median, SD, IQR, minimum, and maximum) or achievement of a particular threshold (frequency and proportion), if applicable. This study will collect patient perceptions of HRQoL based on PRO measures in patients with MCL by collecting the European Organization for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30) on a quarterly basis. The EORTC QLQ-C30 has a recall/observation period during the past week. The questionnaire uses a 4-point Likert scale ranging from 1: Not at all to 4: Very much, and scores by dimension range from 0 to 100. | 24 to 60 months | 
| Tabulate HRQoL responses for the three selected, validated PRO-CTCAE PRO measures in patients with MCL | Descriptive statistics for each validated PRO will include continuous measures (mean, median, SD, IQR, minimum, and maximum) or achievement of a particular threshold (frequency and proportion), if applicable. This study will collect patient perceptions of HRQoL by collecting the PRO version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) (3 questions regarding muscle pain, joint pain, heart palpitations) on a quarterly basis. The PRO-CTCAE is intended for individuals 18 years or older and has a recall of the past 7 days. Responses to each question are scored from 0 to 4 (in order of increasing frequency and severity). | 24 to 60 months | 
| Tabulate HRQoL responses for the validated PRO EuroQoL 5-Dimension 5-Level (EQ-5D-5L) in patients with MCL | Descriptive statistics for each validated PRO will include continuous measures (mean, median, SD, IQR, minimum, and maximum) or achievement of a particular threshold (frequency and proportion), if applicable. This study will collect patient perceptions of HRQoL based on PRO measures in patients with MCL by collecting the EQ-5D-5L on a quarterly basis. This questionnaire is comprised of 5 dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression) and 1 visual analog scale used to assess a patient's self-rated health. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. A unique health state is defined by combining 1 level from each of the 5 dimensions. Each health state is referred to in terms of a 5-digit code. | 24 to 60 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Mantle Cell Lymphoma, Registry, MCL novel therapy, MCL treatment patterns, MCL reported outcomes