A Study to Assess Disease Burden, in Terms of Health-related Quality of Life and Direct Healthcare Costs, in Patients With Newly Diagnosed Multiple Myeloma Who Are Not Candidates for Autologous Stem Cell Transplant (ASCT) in Spain

Study Overview
=================
Brief Summary
-----------------
Those patients with newly diagnosed Multiple Myeloma (MM) who are not candidates for Autologous Stem Cell Transplant (ASCT) and who meet the screening criteria described in this protocol can participate. The participating patients must sign an informed consent, which the investigator who will collect the study variables in an electronic case report form (eCRF) will provide to them. The investigators must recruit at least 450 patients in a 24-month period.

Detailed Description
-----------------
Following the baseline enrolment visit, the following data corresponding to the patient's visits scheduled according to routine clinical practice will be collected in accordance with the following model: Recruitment period: data collection at the baseline visit, every 4 months +/- 15 days in the first year (month 4, 8 and 12) and every 6 months +/- 1 month in the second year (month 18 and 24). Follow-up period: (study will end at 4 years following enrolment of the first patient) From month 24 until up to 4 years from enrolment of the first patient, follow-ups will be performed with semi-annual data collections coinciding with the patient's routine clinical practice visits. In case of progression, suspension or unexpected termination of treatment (for example due to toxicity), or death, a data collection coinciding with the visit in which any of the cases presented occurs will be performed. If a new line of treatment is started, the follow-up model will be started with the same frequency of follow-ups described in point 1 above (month 4, 8, 12, 18 and 24 and semi-annually according to routine clinical practice until up to 4 years from enrolment of the first patient in the study). Observation period: Following this 4-year period, an additional 5-year follow-up will be performed only to evaluate the onset of second primary malignant neoplasms. This follow-up during the 5-year observational period will be performed semi-annually (± 1 month) according to routine clinical practice at each site.

Official Title
-----------------
Observational Study to Assess Disease Burden, in Terms of Health-related Quality of Life and Direct Healthcare Costs, in Patients With Newly Diagnosed Multiple Myeloma Who Are Not Candidates for Autologous Stem Cell Transplant (ASCT) in Spain

Conditions
-----------------
Multiple Myeloma

Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Patient ≥ 18 years of age. Patient with newly diagnosed multiple myeloma (MM) according to IMWG criteria who are not candidates for ASCT and who start treatment for the study disease. Patient who is capable of understanding and filling in the study questionnaires. Patient who has granted his or her informed consent in writing. Exclusion Criteria: Patient who is participating in the active phase of treatment of any clinical trial during the study period.

Ages Eligible for Study
-----------------
Minimum Age: 18 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 |
| --- | --- |
| Patient with MM who are not candidates for ASCT<br>Patients with newly diagnosed MM who are not candidates for ASCT and who are going to start drug treatment for the study disease and patient who is capable of understanding and filling in the study questionnaires At least 450 patients will be enrolled in a 2-year period. Patients must meet all of the inclusion criteria and none of the exclusion criteria and must have previously granted their informed consent in writing. | |

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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Health-related quality of life (HRQoL) | This questionnaire consists of 30 items grouped into 8 dimensions: number of drugs, gait velocity, mood, activities of daily living, health status, nutrition, mental state and comorbidity/habits. It is an instrument that was developed and validated to assess the health status of elderly patients diagnosed with different hematological diseases, including MM. This scale may be a useful instrument in clinical practice as a support tool to predict tolerance to treatment in advanced age. The time required to complete the questionnaire is approximately 10-12 minutes. It will be provided only at the baseline visit for the patient to fill in before starting treatment. The patient must fill it in again each time he or she starts a new line. The questionnaire will be provided by the physician during the visit. | 4 years | 
| EuroQoL 5-Dimension, 5-Level Questionnaire (EQ-5D-5L) | This is a generic HRQoL questionnaire that contains a descriptive system and a visual analogue scale (VAS) for self-assessment of health status. The EQ-5D-5L's descriptive system contains a description of health state in five dimensions (mobility, self-care, usual activities, pain/discomfort and anxiety/depression). Each dimension has 5 possible responses. The respondent must mark the level of problems, within each dimension, that best describes his or her health state today. It will be provided at each visit for the patient to fill in before starting treatment: at the baseline visit, approximately every 4 months in the first year (the visits must coincide with the patient's routine clinical practice visit) and after the first year every 6 months to progression. In case of progression and start of a second line of treatment, the questionnaire will start to be filled in with the same frequency initially described (baseline, every 4 months, etc.). | 4 years | 
| Quality of Life Questionnaire (QLQ-C30) | This is a cancer-specific questionnaire that consists of 30 items structured into 5 functional scales (physical functioning, role functioning, emotional functioning, cognitive functioning and social functioning), 3 symptom scales (fatigue, nausea and vomiting, pain), a global health status scale and, finally, 6 independent items (dyspnoea, insomnia, appetite loss, constipation, diarrhea and financial difficulties). Values from 1 to 4 are assigned depending on the patient's responses to the item. Only items 29 and 30 are evaluated with scoring from 1 to 7. Scores are standardised to obtain a score from 0 to 100, which determines the level of impact of the cancer on the patient for each scale. High scores on the overall health and functional status scales indicate a better QoL, while high scores on the symptoms scale would indicate a decrease in QoL since they indicate the presence of cancer-related symptoms. | 4 years | 
| Quality of Life Questionnaire - Multiple Myeloma Module 20 (QLQ-MY20) | This is a specific questionnaire used in combination with the QLQ-C30 generic questionnaire to evaluate quality of life in patients with MM. It consists of 20 items that address four specific domains - disease symptoms, side effects of treatment, future perspective and body image - and analyses various aspects of each dimension. Three of the four domains of the QLQ-MY20 are multi-item scales: Disease symptoms , side effects of treatment and future perspective (includes worry about death and health in the future and thinking about illness). The body image scale is a single-item scale that addresses physical attractiveness. The scores obtained are transformed linearly to a scale from 0 to 100. A high score in the disease symptoms and side effects domains represents a high level of symptoms or problems, while a high score in the future perspective and body image domains represents better outcomes. | 4 years | 
| Health care cost for Multiple Myeloma (MM) patients | MM-related direct healthcare resources and costs will be evaluated in each line MM treatment will be evaluated. The questionnaire will be provided by the physician during the visit. The resources used since the previous study visit will be collected at each visit. | 4 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Time to progression (TTP) | Time that elapses from the start of a treatment up to disease progression. | 4 years | 
| Progression-free survival (PFS) | Time that elapses from the start of a treatment to disease progression or death, whichever occurs first. | 4 years | 
| Overall survival (OS) | Time that elapses from the diagnosis of the disease up to the death of the patient for any reason. | 9 years | 
| Overall response rate | Response measured through the IMWG criteria: Stringent complete response; complete response (CR); very good partial response; partial response (PR); stable disease (SD); progression (P) | 4 years | 
| Response duration | For each line of treatment for multiple myeloma (MM) during the study period | 4 years | 
| Treatment-free interval [TFI] | Time between relapse and start of a new treatment (second/third lines) | 4 years | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Multiple Myeloma, autologous stem cell transplant (ASCT), QoLMMBuS, healthcare costs