Clinical trial NCT04888039: Follow-up of Patients With Multiple Myeloma in the West-Occitanie Region "Living With a Myeloma in West-Occitanie"
Official title: Therapeutic and Support Oncologic Medical Care Evaluation in Patients With Multiple Myeloma in West-Occitanie Region. Factors Influencing Medical Care and Predictive and Prognostic Impact.
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Multiple Myeloma
Keywords: Multiple Myeloma
Real life
Health care pathways
Quality of life
Lead sponsor: University Hospital, Toulouse
Sponsor class: OTHER
Collaborators: name: Institut National de la Santé Et de la Recherche Médicale, France
name: Takeda
name: Sanofi
First posted: 2021-05-17
Enrollment: 2000
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Adult patients with a diagnosis of symptomatic multiple myeloma and living in the west-Occitanie region.

Brief summary: Actually very few real life data are available for patients with multiple myeloma (MM), whereas they're playing a more and more important role in health care decisions. Treatments choice for medical care of patient with MM depends of their age, their general status, their eligibility to high dose treatment (autograft), and also based on cytogenetic risk (standard/high risk). Therapeutic strategies are multiple and based on drugs associations including proteasome inhibitors, immuno-modulators and monoclonal antibodies.

Therapeutic medical care objective is to improve quality and response duration through more effective induction schemas, systematic consolidation for patients who have undergone high dose therapy and/or maintenance treatment, ensuring patients safety and well-being in the health care pathway.

Quality of life evaluation has to take in consideration disease outcome and secondary effects impact from treatments prescribed for MM.

With clinical trials, new therapeutic strategies are proposed with innovative drugs but participants are selected and do not represent all patients with MM. Therefore, there is a large gap between clinical trials and real life data.

That's why the CHU Toulouse intends to set up a prospective cohort to evaluate the health care pathway of patients with MM in West-Occitanie region and studies impact of treatments prescribed on the disease and on the patients' quality of life.

With this research, standard of care practices for patients with MM will be followed, prognostic scores and clinical trials results will be validated in real life, impact of outpatient support procedure will be assessed (AMA procedure) and sociodemographic/quality of life data will be available for research teams.

Detailed description: Primary objective :

Describe health care pathways of patients with MM living in West Occitanie according to socio-demographic patients' caracteristics, their comorbidities and their initial disease severity. These pathways will be described until the patients' death if the death occurs before the end of their follow-up in this study.

Secondary objectives :

* The best response at each line of therapy
* The progression free survival and overall survival
* Quality of life of patients with MM along their health care pathway
* Second primary malignancy and neuropathy grade 3 or more occurrence during patients'care
* Socio-demographic, clinic and biology factors identification to predict response to treatments, progression free survival, overall survival and quality of life.

Study size calculation :

With the hypothesis of 80% of patients informed about the study will agree to participate and will accept to have their health care data collected, and with 500 to 550 patients' medical files presented each year for MM care to West Occitanie multidisciplinary committee meeting (approximately 400 different patients), a 5-years recruitment period will lead to 1600 patients enrollment.

This size will be able to generate enough precisions for descriptive analyses. Indeed, as example, with a percentage of 50%, conservative situation to estimate percentages, expected precision should be more or less 2.5% according to Clopper-Pearson exact method.

Precision of more or less 5% should be also obtained for sub-groups of 400 persons.

Eligibility criteria: Inclusion Criteria:

* Patient living in the West-Occitanie region
* Patient with a diagnosis of symptomatic multiple myeloma (Rajkumar et al, Lancet Oncology 2014)

Exclusion Criteria:

* Patient opposed to this research
* Patient under legal protection

Primary outcomes:
measure: Treatment lines and procedures followed by the patients
timeFrame: 5 to 10 years
measure: Transplants
timeFrame: 5 to 10 years
measure: Treatments discontinuation
timeFrame: 5 to 10 years
measure: Therapeutic medical care description
timeFrame: 5 to 10 years
measure: Unconventional alternative medicine
timeFrame: 5 to 10 years
measure: Outpatient support structure
timeFrame: 5 to 10 years
Secondary outcomes:
measure: Best response (BR)
timeFrame: 5 to 10 years
measure: Progression-free Survival (PFS)
timeFrame: 5 to 10 years
measure: PFS after the second therapeutic line
timeFrame: 5 to 10 years
measure: Overall Survival (OS);
timeFrame: 5 to 10 years
measure: Quality of life (QOL) during the treatment course
timeFrame: 5 to 10 years
measure: Second primary cancers (SPC) and grade 3 and higher neuropathies
timeFrame: 5 to 10 years
Other outcomes:
Locations:
city: Toulouse
country: France
facility: IUCT-Oncopole - Toulouse University Hospital
state: West-Occitanie
Study officials:
affiliation: IUCT-Oncopole - Toulouse University Hospital
name: Aurore PERROT, MD
role: PRINCIPAL_INVESTIGATOR
References: