Clinical trial NCT02946333: 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
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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Multiple Myeloma
Keywords: Multiple Myeloma
autologous stem cell transplant (ASCT)
QoLMMBuS
healthcare costs
Lead sponsor: Celgene
Sponsor class: INDUSTRY
First posted: 2016-10-27
Enrollment: 355
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: At least 450 patients with newly diagnosed multiple myeloma (MM) according to IMWG criteria who are not candidates for ASCT and who start drug treatment for the study disease will be enrolled.

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.

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.

Primary outcomes:
measure: Health-related quality of life (HRQoL)
timeFrame: 4 years
measure: EuroQoL 5-Dimension, 5-Level Questionnaire (EQ-5D-5L)
timeFrame: 4 years
measure: Quality of Life Questionnaire (QLQ-C30)
timeFrame: 4 years
measure: Quality of Life Questionnaire - Multiple Myeloma Module 20 (QLQ-MY20)
timeFrame: 4 years
measure: Health care cost for Multiple Myeloma (MM) patients
timeFrame: 4 years
Secondary outcomes:
measure: Time to progression (TTP)
timeFrame: 4 years
measure: Progression-free survival (PFS)
timeFrame: 4 years
measure: Overall survival (OS)
timeFrame: 9 years
measure: Overall response rate
timeFrame: 4 years
measure: Response duration
timeFrame: 4 years
measure: Treatment-free interval [TFI]
timeFrame: 4 years
Other outcomes:
Locations:
city: Algeciras
country: Spain
facility: Hospital Punta de Europa
state: Andalusia
city: Antequera
country: Spain
facility: Hospital de Antequera
state: Andalusia
city: Córdoba
country: Spain
facility: Hospital Reina Sofía
state: Andalusia
city: Huercal Olvera
country: Spain
facility: Hospital Huercal Olvera
state: Andalusia
city: Jaén
country: Spain
facility: Hospital General Jaén
state: Andalusia
city: Jerez de la Frontera
country: Spain
facility: Hospital Jerez de la Frontera
state: Andalusia
city: Motril
country: Spain
facility: Hospital de Motril
state: Andalusia
city: Málaga
country: Spain
facility: Clinico Univ. Virgen de la Victoria
state: Andalusia
city: Málaga
country: Spain
facility: Hospital Carlos Haya
state: Andalusia
city: Málaga
country: Spain
facility: Hospital de Costa del Sol
state: Andalusia
city: Pozoblanco
country: Spain
facility: Hospital Valle de los Pedroches Pozoblanco
state: Andalusia
city: Puerto Real
country: Spain
facility: Hospital Puerto Real
state: Andalusia
city: Sevilla
country: Spain
facility: Hospital de Macarena
state: Andalusia
city: Zaragoza
country: Spain
facility: Hospital Miguel Servet
state: Aragón
city: Hospitalet de Llobregat
country: Spain
facility: Hospital ICO Durán y Reynals
state: Barcelona
city: Hospitalet de Llobregat
country: Spain
facility: ICO Duran i Reynals
state: Barcelona
city: Las Palmas de Gran Canaria
country: Spain
facility: Hospital Universitario Dr. Negrín
state: Canarias
city: Burgos
country: Spain
facility: Hospital Universitario de Burgos
state: Castilla Y León
city: Salamanca
country: Spain
facility: Hospital Clínico Universitario de Salamanca
state: Castilla Y León
city: Valladolid
country: Spain
facility: Hospital Clínico de Valladolid
state: Castilla Y León
city: Barcelona
country: Spain
facility: Hospital del Mar
state: Cataluña
city: Barcelona
country: Spain
facility: Hospital Santa Creu i Sant Pau
state: Cataluña
city: Barcelona
country: Spain
facility: Hospital Vall Hebrón
state: Cataluña
city: Granollers
country: Spain
facility: Hospital General de Granollers
state: Cataluña
city: Lleida
country: Spain
facility: Hospital Arnau Vilanova de Lleida
state: Cataluña
city: Manresa
country: Spain
facility: Fundació Althaia
state: Cataluña
city: Sabadell
country: Spain
facility: Parc Taulí
state: Cataluña
city: Terrassa
country: Spain
facility: Mutua Terrassa
state: Cataluña
city: Getafe
country: Spain
facility: Hospital Universitario de Getafe
state: Comunidad De Madrid
city: Madrid
country: Spain
facility: Hospital de la Princesa
state: Comunidad De Madrid
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Marañon
state: Comunidad De Madrid
city: Madrid
country: Spain
facility: Hospital Clínico San Carlos
state: Comunidad De Madrid
city: Madrid
country: Spain
facility: Hospital Universitario Fundación Jiménez Díaz
state: Comunidad De Madrid
city: Madrid
country: Spain
facility: Hospital 12 de Octubre
state: Comunidad De Madrid
city: Castellón de la Plana
country: Spain
facility: Hospital General Castellón
state: Comunidad Valenciana
city: Valencia
country: Spain
facility: Hospital Arnau Vilanova Valencia
state: Comunidad Valenciana
city: Valencia
country: Spain
facility: Hospital Doctor Peset
state: Comunidad Valenciana
city: Valencia
country: Spain
facility: Hospital La Fe Valencia
state: Comunidad Valenciana
city: Valencia
country: Spain
facility: Hospital La Ribera
state: Comunidad Valenciana
city: Llerena
country: Spain
facility: Complejo Hospitalario Llerena-Zafra
state: Extremadura
city: Mérida
country: Spain
facility: Hospital de Mérida
state: Extremadura
city: A Coruña
country: Spain
facility: Complexo Hospitalario Universitario de A Coruña
state: Galicia
city: Lugo
country: Spain
facility: Hospital Lucus Augustí
state: Galicia
city: Ourense
country: Spain
facility: Complexo Hospitalario Universitario de Ourense
state: Galicia
city: Pontevedra
country: Spain
facility: Complexo Hospitalario de Pontevedra
state: Galicia
city: Vigo
country: Spain
facility: Complexo Hospitalario Universitario de Vigo
state: Galicia
city: Palma de Mallorca
country: Spain
facility: Hospital Son Espases
state: Islas Baleares
city: Palma de Mallorca
country: Spain
facility: Hospital Son Llàtzer
state: Islas Baleares
city: Las Palmas de Gran Canaria
country: Spain
facility: Hospital Dr. Negrín
state: Islas Canarias
city: Santa Cruz de Tenerife
country: Spain
facility: Hospital Universitario Nuestra Señora de Candelaria
state: Islas Canarias
city: Santa Cruz de Tenerife
country: Spain
facility: Hospital Universitario Canarias
state: Islas Canaria
city: Pozuelo de Alarcón
country: Spain
facility: Hospital Quirón
state: Madrid
city: San Sebastián de los Reyes
country: Spain
facility: Hospital Infanta Sofía
state: Madrid
city: Pamplona
country: Spain
facility: Complejo Hospitalario Navarra
state: Navarra
city: Bilbao
country: Spain
facility: Hospital de Basurto
state: País Vasco
city: Usansolo
country: Spain
facility: Hospital de Galadakao
state: País Vasco
city: Vitoria-Gasteiz
country: Spain
facility: Hospital de Txagorritxu
state: País Vasco
city: Alzira
country: Spain
facility: Hospital de La Ribera
state: Valencia
city: Madrid
country: Spain
facility: Hospital Infanta Leonor
city: Madrid
country: Spain
facility: Hospital Ramón y Cajal
city: Madrid
country: Spain
facility: Hospital La Paz
city: Navarra
country: Spain
facility: Complejo Hospitalario de Navarra
city: Valencia
country: Spain
facility: Hospital Universitario La Fe
city: Zaragoza
country: Spain
facility: Hospital Miguel Servet
city: Ávila
country: Spain
facility: Complejo Hospitalario de Ávila
Study officials:
affiliation: Celgene
name: Giselle Lostaunau, Senior Medical Advisor MM
role: STUDY_DIRECTOR
References:
citation: Engelhardt M, Ihorst G, Singh M, Rieth A, Saba G, Pellan M, Lebioda A. Real-World Evaluation of Health-Related Quality of Life in Patients With Multiple Myeloma From Germany. Clin Lymphoma Myeloma Leuk. 2021 Feb;21(2):e160-e175. doi: 10.1016/j.clml.2020.10.002. Epub 2020 Oct 24.