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
- Conditions
- Multiple Myeloma
- Registration Number
- NCT02946333
- Lead Sponsor
- Celgene
- 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:
1. 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).
2. 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).
3. 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 355
- 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.
- Patient who is participating in the active phase of treatment of any clinical trial during the study period.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Quality of Life Questionnaire - Multiple Myeloma Module 20 (QLQ-MY20) 4 years 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.
Quality of Life Questionnaire (QLQ-C30) 4 years 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.
Health care cost for Multiple Myeloma (MM) patients 4 years 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.
Health-related quality of life (HRQoL) 4 years 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.
EuroQoL 5-Dimension, 5-Level Questionnaire (EQ-5D-5L) 4 years 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.).
- Secondary Outcome Measures
Name Time Method Time to progression (TTP) 4 years Time that elapses from the start of a treatment up to disease progression.
Progression-free survival (PFS) 4 years Time that elapses from the start of a treatment to disease progression or death, whichever occurs first.
Treatment-free interval [TFI] 4 years Time between relapse and start of a new treatment (second/third lines)
Response duration 4 years For each line of treatment for multiple myeloma (MM) during the study period
Overall response rate 4 years Response measured through the IMWG criteria: Stringent complete response; complete response (CR); very good partial response; partial response (PR); stable disease (SD); progression (P)
Overall survival (OS) 9 years Time that elapses from the diagnosis of the disease up to the death of the patient for any reason.
Trial Locations
- Locations (64)
Hospital Puerto Real
🇪🇸Puerto Real, Andalusia, Spain
Hospital ICO Durán y Reynals
🇪🇸Hospitalet de Llobregat, Barcelona, Spain
Hospital de la Princesa
🇪🇸Madrid, Comunidad De Madrid, Spain
Hospital Arnau Vilanova de Lleida
🇪🇸Lleida, Cataluña, Spain
Hospital General Universitario Gregorio Marañon
🇪🇸Madrid, Comunidad De Madrid, Spain
Hospital Son Espases
🇪🇸Palma de Mallorca, Islas Baleares, Spain
Hospital de Txagorritxu
🇪🇸Vitoria-Gasteiz, País Vasco, Spain
Hospital de Antequera
🇪🇸Antequera, Andalusia, Spain
Hospital Punta de Europa
🇪🇸Algeciras, Andalusia, Spain
Hospital Huercal Olvera
🇪🇸Huercal Olvera, Andalusia, Spain
Hospital Reina Sofía
🇪🇸Córdoba, Andalusia, Spain
Hospital Jerez de la Frontera
🇪🇸Jerez de la Frontera, Andalusia, Spain
Hospital de Motril
🇪🇸Motril, Andalusia, Spain
Hospital General Jaén
🇪🇸Jaén, Andalusia, Spain
Clinico Univ. Virgen de la Victoria
🇪🇸Málaga, Andalusia, Spain
Hospital de Costa del Sol
🇪🇸Málaga, Andalusia, Spain
Hospital Valle de los Pedroches Pozoblanco
🇪🇸Pozoblanco, Andalusia, Spain
Hospital de Macarena
🇪🇸Sevilla, Andalusia, Spain
Hospital Miguel Servet
🇪🇸Zaragoza, Spain
ICO Duran i Reynals
🇪🇸Hospitalet de Llobregat, Barcelona, Spain
Hospital Universitario de Burgos
🇪🇸Burgos, Castilla Y León, Spain
Hospital Clínico Universitario de Salamanca
🇪🇸Salamanca, Castilla Y León, Spain
Hospital Vall Hebrón
🇪🇸Barcelona, Cataluña, Spain
Hospital del Mar
🇪🇸Barcelona, Cataluña, Spain
Hospital Santa Creu i Sant Pau
🇪🇸Barcelona, Cataluña, Spain
Hospital General de Granollers
🇪🇸Granollers, Cataluña, Spain
Hospital Universitario de Getafe
🇪🇸Getafe, Comunidad De Madrid, Spain
Parc Taulí
🇪🇸Sabadell, Cataluña, Spain
Fundació Althaia
🇪🇸Manresa, Cataluña, Spain
Mutua Terrassa
🇪🇸Terrassa, Cataluña, Spain
Hospital Universitario Fundación Jiménez Díaz
🇪🇸Madrid, Comunidad De Madrid, Spain
Hospital Clínico San Carlos
🇪🇸Madrid, Comunidad De Madrid, Spain
Hospital 12 de Octubre
🇪🇸Madrid, Comunidad De Madrid, Spain
Hospital Doctor Peset
🇪🇸Valencia, Comunidad Valenciana, Spain
Hospital Arnau Vilanova Valencia
🇪🇸Valencia, Comunidad Valenciana, Spain
Hospital General Castellón
🇪🇸Castellón de la Plana, Comunidad Valenciana, Spain
Hospital La Fe Valencia
🇪🇸Valencia, Comunidad Valenciana, Spain
Complejo Hospitalario Llerena-Zafra
🇪🇸Llerena, Extremadura, Spain
Complexo Hospitalario Universitario de A Coruña
🇪🇸A Coruña, Galicia, Spain
Hospital de Mérida
🇪🇸Mérida, Extremadura, Spain
Hospital Lucus Augustí
🇪🇸Lugo, Galicia, Spain
Complexo Hospitalario Universitario de Ourense
🇪🇸Ourense, Galicia, Spain
Complexo Hospitalario Universitario de Vigo
🇪🇸Vigo, Galicia, Spain
Complexo Hospitalario de Pontevedra
🇪🇸Pontevedra, Galicia, Spain
Hospital Dr. Negrín
🇪🇸Las Palmas de Gran Canaria, Islas Canarias, Spain
Hospital Universitario Nuestra Señora de Candelaria
🇪🇸Santa Cruz de Tenerife, Islas Canarias, Spain
Hospital Quirón
🇪🇸Pozuelo de Alarcón, Madrid, Spain
Hospital Infanta Sofía
🇪🇸San Sebastián de los Reyes, Madrid, Spain
Complejo Hospitalario Navarra
🇪🇸Pamplona, Navarra, Spain
Hospital de Basurto
🇪🇸Bilbao, País Vasco, Spain
Hospital de Galadakao
🇪🇸Usansolo, País Vasco, Spain
Hospital de La Ribera
🇪🇸Alzira, Valencia, Spain
Hospital La Paz
🇪🇸Madrid, Spain
Hospital Infanta Leonor
🇪🇸Madrid, Spain
Hospital Ramón y Cajal
🇪🇸Madrid, Spain
Complejo Hospitalario de Navarra
🇪🇸Navarra, Spain
Hospital Universitario La Fe
🇪🇸Valencia, Spain
Complejo Hospitalario de Ávila
🇪🇸Ávila, Spain
Hospital La Ribera
🇪🇸Valencia, Comunidad Valenciana, Spain
Hospital Clínico de Valladolid
🇪🇸Valladolid, Castilla Y León, Spain
Hospital Carlos Haya
🇪🇸Málaga, Andalusia, Spain
Hospital Universitario Canarias
🇪🇸Santa Cruz de Tenerife, Islas Canaria, Spain
Hospital Universitario Dr. Negrín
🇪🇸Las Palmas de Gran Canaria, Canarias, Spain
Hospital Son Llàtzer
🇪🇸Palma de Mallorca, Islas Baleares, Spain