An Observational Study of Presentation, Treatment Patterns, and Outcomes in Multiple Myeloma Participants

Browse trials for

• Multiple Myeloma 3,632
• Neoplasms, Plasma Cell 2,306

Keywords

• Drug Therapy

Multiple Myeloma

3,632 studies on the registry are indexed under Multiple Myeloma; 745 are open to participants now.

This study's enrollment of 4,253 is above the median of 140 across 468 observational studies indexed under Multiple Myeloma.

Lead sponsor

Takeda is the lead sponsor of 1,002 studies on the registry; 92 are open to participants now.

Of its 173 completed or terminated interventional studies of FDA-regulated products, 149 (86%) have results posted.

Study population

Groups and cohorts

• Newly Diagnosed Multiple Myeloma

  Participants newly diagnosed with MM within 3 months from initiation of treatment were enrolled for 3 years, and followed for at least 2 years, until death, or the end of the study, whichever comes first (up to approximately 5 years).

  Other: No Intervention

• Relapsed/Refractory Multiple Myeloma

  Participants diagnosed with RRMM who previously received 1 to 3 prior lines of therapy were enrolled for 3 years, and followed for at least 2 years, until death, or the end of the study, whichever comes first (up to approximately 5 years).

  Other: No Intervention

Interventions

• OtherNo Intervention

  As this was an observational study, no intervention was administered.

Primary outcomes

1. Number of Participants With Co-morbidities

   Charlson Comorbidity Index (CCI) was used to represent number of participants with co-morbidities. CCI is a method of categorizing comorbidities of participants. Each comorbidity category has an associated weight (from 1 to 6), based on the adjusted risk of mortality or resource use, and the sum of all the weights results in a single comorbidity score for a participant. A score of 0 = no comorbidities found, 1 = not ill, 2 = mildly ill, 3 = moderately ill, 4 = severely ill, and ≥5 = moribund. The higher the score, the more likely the predicted outcome resulted in mortality or higher resource use.

   Time frame: Baseline up to 5 years

2. Number of Participants Diagnosed With Newly Diagnosed Multiple Myeloma (NDMM) and Relapsed/Refractory Multiple Myeloma (R/RMM)

   Participants diagnosed with NDMM and R/RMM were determined at the start of the study.

   Time frame: At Baseline

3. Number of Participants Diagnosed With Symptoms of ND MM and R/R MM During the Study

   Time frame: Baseline up to 5 years

4. Sites of Disease Diagnosed With ND MM and R/R MM

   Time frame: Baseline up to 5 years

5. Number of Participants With ECOG (Eastern Cooperative Oncology Group) Performance Status

   ECOG-PS measured on-therapy (time between first dose and last dose date with a 30-day lag) assessed participant's performance status on 6 point scale: 0=Fully active/able to carry on all pre-disease activities without restriction; 1=restricted in physically strenuous activity, ambulatory/able to carry out light or sedentary work; 2=ambulatory (\>50% of waking hours), capable of all self care, unable to carry out any work activities; 3=capable of only limited self care, confined to bed/chair \>50% of waking hours; 4=completely disabled, cannot carry on any self care, totally confined to bed/chair; 5=dead. The line of Therapy was determined at study entry.

   Time frame: At Baseline

6. Number of Participants With Myeloma Frailty Index

   Frailty is defined as the combination of unintentional weight loss, exhaustion, low physical activity, slow walking speed, and muscular weakness. The Myeloma Frailty Index is a composite index that was calculated using the points system, which produces a range of values from 0 to 5. Participants with score 0= fit, score 1= intermediate, and score ≥2= frail. Higher score indicates likeliness that the predicted outcome will result in frailty. The line of Therapy was determined at study entry.

   Time frame: At Baseline

7. Number of Participants Evaluated for Minimal Residual Disease (MRD)

   Time frame: Baseline up to 5 years

8. Number of Participants Evaluated for Gene Expression Profiling (GEP)

   Time frame: Baseline up to 5 years

9. Number of Participants Evaluated for Cytogenetics Using Fluorescence in Situ Hybridization (FISH)

   FISH methodology was reported with Yes/No results for the following tests: deletion (17p)/p53 \[Del(17p)/p53\], translocation (4,14) \[t(4,14)\], and translocation (14,16) \[t(14,16)\].

   Time frame: At Baseline

10. Number of Participants Evaluated for International Staging System (ISS)/ Revised (R)-ISS Stage

    ISS disease stages were defined as I:low risk, β2-Microglobulin\<3.5mg/L, albumin≥3.5g/dL, II:not stage I or III, III:high risk,β2-Microglobulin≥5.5mg/L). R-ISS is based on ISS, chromosomal abnormalities (CA), and lactate dehydrogenase (LDH). R-ISS disease stages were defined as I: ISS Stage I and standard risk CA by FISH and normal LDH (i.e. \<=300 U/L), II: Neither R-ISS Stage I nor Stage III, III: ISS Stage III and either high risk CA by FISH or high LDH (i.e. \>300 U/L).

    Time frame: At Baseline

11. Duration of Treatment for Participants With and Without Stem Cell Transplant

    Data was analyzed for participants with and without stem cell transplant for all enrolled population, included all participants who signed the inform consent form, out of which 990 participants were excluded during the final analysis due to concerns around robustness of data.

    Time frame: Baseline up to 5 years

12. Overall Survival (OS)

    Overall Survival was defined as the number of months from the index regimen start date within each line of therapy, starting with the line during study entry, until the date of death. The Kaplan Meier estimates was used for the analysis.

    Time frame: Baseline up to 5 years

13. Disease Progression Status on Each Regimen

    Disease progression status was assessed by physician interpretation of IMWG Response criteria.

    Time frame: Baseline up to 5 years

14. Response to Each Regimen

    Time frame: Baseline up to 5 years

15. Time to Next Therapy

    The line of Therapy was determined at study entry. The Kaplan Meier estimates was used for the analysis.

    Time frame: Baseline up to 5 years

16. Number of Participants With Stem Cell Transplant

    Time frame: Baseline up to 5 years

17. Number of Participants With Global Health Status Scale/Quality of Life (QoL) Among MM Participants

    The Global Health Status scale/QoL scale included 2 questions measured with a 7-point numeric rating scale (very poor to excellent). Raw scores are converted into scale scores ranging from 0 to 100. A higher score represents better HRQoL.

    Time frame: Baseline up to 5 years

Secondary outcomes

1. Number of Participants Receiving Different Treatment Combinations

   Time frame: Baseline up to 5 years

2. Number of Treatment Sequencing

   Drug classes were based on the earliest regimen in each corresponding Line of Therapy. The data for this outcome measure was analyzed as per line of therapy.

   Time frame: Baseline up to 5 years

3. Number of Participants in the Treatment Rechallenge

   Time frame: Baseline up to 5 years

4. Number of Clinical Outcomes for Different Strategies

   Time frame: Baseline up to 5 years

5. Number of Clinical Outcomes Between Continuous Treatment and Intermittent Treatment Strategy

   Time frame: Baseline up to 5 years

6. Triggers of Treatment Initiation at Relapse Including Biochemical Progression or Symptomatic Progression

   Time frame: Baseline up to 5 years

7. Reasons for Treatment Modifications

   Time frame: Baseline up to 5 years

8. Healthcare Resource Utilization (HRU) Among MM Participants

   Time frame: Baseline up to 5 years

9. Associations Between Presentation and Disease Characteristics

   Time frame: Baseline up to 5 years

10. Associations Between Choice Of Therapy and Clinical Outcomes

    Time frame: Baseline up to 5 years

11. Number of Participants With Atleast One Treatment-emergent Adverse Events Leading to Treatment Discontinuation

    An Adverse Event (AE) is defined as any untoward medical occurrence in a clinical investigation participant administered a drug; it does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (e.g., a clinically significant abnormal laboratory finding), symptom, or disease temporally associated with the use of a drug, whether or not it is considered related to the drug. A treatment-emergent adverse event (TEAE) is defined as an adverse event with an onset that occurs after receiving study drug. Treatment discontinuation includes temporary and permanent discontinuation, drug modification, and second primary malignancies.

    Time frame: Baseline up to 5 years

Baseline characteristics

Publications

• Hajek R, Minarik J, Straub J, Pour L, Jungova A, Berdeja JG, Boccadoro M, Brozova L, Spencer A, van Rhee F, Vela-Ojeda J, Thompson MA, Abonour R, Chari A, Cook G, Costello CL, Davies FE, Hungria VT, Lee HC, Leleu X, Puig N, Rifkin RM, Terpos E, Usmani SZ, Weisel KC, Zonder JA, Barinova M, Kuhn M, Silar J, Capkova L, Galvez K, Lu J, Elliott J, Stull DM, Ren K, Maisnar V. Ixazomib-lenalidomide-dexamethasone in routine clinical practice: effectiveness in relapsed/refractory multiple myeloma. Future Oncol. 2021 Jul;17(19):2499-2512. doi: 10.2217/fon-2020-1225. Epub 2021 Mar 26. PubMed 33769076 ↗
• Costello C, Davies FE, Cook G, Vela-Ojeda J, Omel J, Rifkin RM, Berdeja J, Puig N, Usmani SZ, Weisel K, Zonder JA, Terpos E, Spencer A, Leleu X, Boccadoro M, Thompson MA, Romanus D, Stull DM, Hungria V. INSIGHT MM: a large, global, prospective, non-interventional, real-world study of patients with multiple myeloma. Future Oncol. 2019 May;15(13):1411-1428. doi: 10.2217/fon-2019-0013. Epub 2019 Feb 28. PubMed 30816809 ↗

Study documents

• Study protocol · Apr 11, 2018
• Statistical analysis plan · Sep 28, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Supporting information: Study protocol, Sap, Icf, Csr