Real-World Treatment Patterns and Clinical Outcomes in Patients With Multiple Myeloma Previously Treated With Lenalidomide and an Anti-CD38 Monoclonal Antibody.

Background: This analysis explored real-world characteristics, treatment patterns and clinical outcomes in patients with relapsed or refractory multiple myeloma (RRMM) previously treated with lenalidomide and an anti-CD38 monoclonal antibody (mAb) and requiring subsequent treatment.

Materials And Methods: The PREAMBLE and Connect MM prospective registries of patients with multiple myeloma (MM), and the US nationwide Flatiron Health electronic health record-derived de-identified database were analysed. MM-specific treatment patterns (prior/index therapies) and outcomes (progression-free survival [PFS]/overall survival [OS]) were assessed.

The Effect of Age and Other Patient Characteristics on Outcomes Among Nontransplanted Patients Who Were Treated With First-Line Lenalidomide, Bortezomib, and Dexamethasone: Results From the Connect MM Registry.

Background: Lenalidomide (R), bortezomib (V), and dexamethasone (d) is a standard-of-care regimen in newly diagnosed multiple myeloma (NDMM); however, characteristics and outcomes for nontransplanted patients receiving frontline RVd are not well understood.

Patients: The Connect MM Registry is a large, US, multicenter, prospective observational cohort study of NDMM patients.

Methods: This analysis investigated characteristics and outcomes of patients who received RVd alone or followed by Rd or R (RVd ± Rd/R) who did not undergo frontline autologous stem cell transplantation.

Pomalidomide, Bortezomib, and Dexamethasone in Lenalidomide-Pretreated Multiple Myeloma: A Subanalysis of OPTIMISMM by Frailty and Bortezomib Dose Adjustment.

Introduction: A proportion of patients with multiple myeloma (MM) are older and/or have comorbidities, requiring dose adjustments. Data from OPTIMISMM (NCT01734928) supported the use of pomalidomide, bortezomib, and dexamethasone (PVd) for treating relapsed/refractory MM. This subanalysis of OPTIMISMM assessed outcome by frailty and/or bortezomib dose adjustment.

A Meta-Analysis of the Efficacy of Pomalidomide-Based Regimens for the Treatment of Relapsed/Refractory Multiple Myeloma After Lenalidomide Exposure.

Introduction: The objective was to assess the benefit of pomalidomide-based combination regimens in patients with relapsed/refractory multiple myeloma (RRMM) previously treated with lenalidomide. A pooled estimate was obtained for efficacy outcomes including overall response rate (ORR), complete response (CR) rate, and progression-free survival (PFS) based on multiple trials conducted in this patient population.

Patients And Methods: A literature search was conducted on March 22, 2022 for relevant trials published between January 1, 2016 and the search date.

Building a Healthcare Alliance for Resourceful Medicine Offensive Against Neoplasms in Hematology Added Value Framework for Hematologic Malignancies: A Comparative Analysis of Existing Tools.

Objectives: The Innovative Medicines Initiative-funded, multistakeholders project Healthcare Alliance for Resourceful Medicine Offensive Against Neoplasms in Hematology (HARMONY) created a task force involving patient organizations, medical associations, pharmaceutical companies, and health technology assessment/regulator agencies' representatives to evaluate the suitability of previously established value frameworks (VFs) for assessing the clinical and societal impact of new interventions for hematologic malignancies (HMs).

Methods: Since the HARMONY stakeholders identified the inclusion of patients' points of view on evaluating VFs as a priority, surveys were conducted with the patient organizations active in HMs and part of the HARMONY network, together with key opinion leaders, pharmaceutical companies, and regulators, to establish which outcomes were important for each HM. Next, to evaluate VFs against the sources of information taken into account (randomized clinical trials, registries, real-world data), structured questionnaires were created and filled by HARMONY health professionals to specify preferred data sources per malignancy.

Indirect treatment comparison of idecabtagene vicleucel versus conventional care in triple-class exposed multiple myeloma.

To compare the efficacy of idecabtagene vicleucel (ide-cel, bb2121) versus conventional care (CC) in triple-class exposed relapsed and refractory multiple myeloma (RRMM) patients. A matching-adjusted indirect comparison was conducted using individual patient-level data from the pivotal, phase II, single-arm KarMMa trial (NCT03361748) and aggregate-level data from MAMMOTH, the largest independent observational study of CC in heavily pretreated RRMM patients. Ide-cel improved overall response rate (odds ratio: 5.

Treatment Patterns and Outcomes in Triple-Class Exposed Patients With Relapsed and Refractory Multiple Myeloma: Findings From the Multinational ITEMISE Study.

Purpose: Patients with relapsed and refractory multiple myeloma (RRMM) have a poor prognosis and limited treatment options after exposure to an immunomodulatory drug, proteasome inhibitor (PI), and anti-CD38 antibody (triple-class exposure [TCE]). However, current understanding about the management of these patients and associated health care resource use (HCRU) is limited outside the United States. The objective of the International Treatment pattern and resource use Evaluation for Multiple myeloma In a Study of triple-class Exposed patients (ITEMISE) study was to use a physician-developed survey fielded to hematologists across Europe and Canada to assess the treatment, management, HCRU, and end-of-life care for patients with RRMM after TCE.

Real-world analysis of patient characteristics, treatment outcomes, and healthcare resource utilization across Europe in patients with newly diagnosed multiple myeloma ineligible for stem cell transplantation who received lenalidomide- or bortezomib-based regimens.

We aimed to compare real-world outcomes, resource use, and costs for patients with newly diagnosed multiple myeloma (NDMM) treated with continuous first-line (1 L) lenalidomide or fixed bortezomib in Europe. We performed a multicenter, retrospective, observational chart review of transplant-ineligible NDMM patients across 7 countries. Of 453 eligible patients, 220 received 1 L lenalidomide-based regimens; 105 (47.

Matching adjusted indirect comparisons of efficacy outcomes for idecabtagene vicleucel (ide-cel, bb2121) versus selinexor + dexamethasone and belantamab mafodotin in relapsed and refractory multiple myeloma.

Idecabtagene vicleucel (ide-cel, bb2121), a chimeric antigen receptor (CAR) T cell therapy, has been investigated in patients with relapsed and refractory multiple myeloma (RRMM) who have received an immunomodulatory drug, proteasome inhibitor, and anti-CD38 antibody in the single-arm phase 2 KarMMa clinical trial. Two therapies with distinct mechanisms of action - selinexor plus dexamethasone (Sd) and belantamab mafodotin (BM) - are currently approved in the United States for heavily pretreated patients, including those who are triple-class refractory. To compare ide-cel versus Sd and ide-cel versus BM, matching-adjusted indirect comparisons were performed.

Lenalidomide as maintenance treatment for patients with multiple myeloma after autologous stem cell transplantation: A pharmaco-economic assessment.

Objective: Autologous stem cell transplantation (ASCT) has improved progression-free survival (PFS) and overall survival in eligible patients with newly diagnosed multiple myeloma (NDMM); however, relapse occurs. Maintenance therapy with lenalidomide (Len-Mt) extends survival and delays relapse and the subsequent initiation of costly second-line regimens. Here, we report the cost-effectiveness of Len-Mt following ASCT from a Dutch healthcare service perspective.

Health-related quality-of-life results from the phase 3 OPTIMISMM study: pomalidomide, bortezomib, and low-dose dexamethasone versus bortezomib and low-dose dexamethasone in relapsed or refractory multiple myeloma.

In the randomized phase-3 OPTIMISMM study, the addition of pomalidomide to bortezomib and low-dose dexamethasone (PVd) resulted in significant improvement in progression-free survival (PFS) in lenalidomide-pretreated patients with relapsed or refractory multiple myeloma (RRMM), including lenalidomide refractory patients. Here, we report health-related quality of life (HRQoL) results from this trial. Patients received PVd or Vd in 21-day cycles until disease progression or discontinuation.

Health-related quality of life results from the IFM 2009 trial: treatment with lenalidomide, bortezomib, and dexamethasone in transplant-eligible patients with newly diagnosed multiple myeloma.

The Intergroupe Francophone du Myelome 2009 trial (NCT01191060) assessed health-related quality of life (HRQoL) in patients with newly diagnosed multiple myeloma (NDMM) receiving lenalidomide/bortezomib/dexamethasone (RVd) induction therapy followed by consolidation therapy with either autologous stem cell transplantation (ASCT) plus RVd (RVd-ASCT) or RVd-alone; both groups then received lenalidomide maintenance therapy for 1 year. Global HRQoL, physical functioning, and role functioning scores significantly improved for both cohorts from baseline to the end of consolidation and were sustained during maintenance and follow-up, with clinically meaningful changes (RVd-alone:  = .0002; RVd-ASCT:  < .

Relative efficacy of treatment options in transplant-ineligible newly diagnosed multiple myeloma: results from a systematic literature review and network meta-analysis.

Established treatments for transplant-ineligible (TNE) patients with newly diagnosed multiple myeloma (NDMM) include melphalan and prednisone (MP) combined with either bortezomib (VMP) or thalidomide (MPT), or lenalidomide plus low-dose dexamethasone (Rd). New treatments for TNE NDMM include Rd plus bortezomib (RVd) and daratumumab plus VMP (VMP + D), daratumumab plus lenalidomide and dexamethasone (D + Rd). Relative efficacy of these treatments was compared using a network meta-analysis.

Productivity losses in patients with newly diagnosed multiple myeloma following stem cell transplantation and the impact of maintenance therapy.

Objective: This study examined productivity losses in European patients with newly diagnosed multiple myeloma (NDMM) undergoing autologous stem cell transplantation (ASCT), to better understand and model the impact of NDMM and lenalidomide maintenance therapy on productivity from a patient and societal perspective.

Methods: A cross-sectional online patient survey was conducted across the UK, Germany, France, Spain and Italy. A partitioned survival model was used to estimate productivity loss and the impact of maintenance therapy, using human capital (HC) and friction cost approaches.

The Role of Real-World Evidence in UK Reimbursement: Case Study of Lenalidomide in Myelodysplastic Syndrome Deletion 5q.

Background: Uncertainty within cost-effectiveness analysis, often driven by lack of mature data from large clinical trials, plays a key role in decisions made by the National Institute for Health and Care Excellence (NICE), particularly for early access medicines and orphan drugs.

Objectives: In this case study, we used real-world evidence to address the uncertainty in the cost-effectiveness case for lenalidomide in transfusion-dependent low- and intermediate-1-risk myelodysplastic syndrome (MDS) deletion 5q [del(5q)], affecting a small but unique subpopulation with an orphan disease.

Methods: As part of a submission to NICE, we developed a cost-effectiveness model for lenalidomide, resulting in eventual recommendation.

Health related quality of life for multiple myeloma patients according to treatment strategy after autologous stem cell transplant: a cross-sectional study using EORTC, EQ-5D and MY-20 scales.

Maintenance (MT) may be prescribed after autologous stem cell transplant (ASCT) but there are often concerns about the impact on quality of life (QoL). QoL was compared between baseline patients (30-100 days post-ASCT and had not commenced MT); MT patients (>100 days post-ASCT and receiving MT), and no MT (>100 days post-ASCT and not receiving MT). Patients completed the EuroQoL five dimension (EQ-5D), the European Organization for Research and Treatment of Cancer QoL Questionnaire Core 30 (EORTC QLQ-C30), and the QoL Questionnaire Myeloma 20 module (QLQ-MY20).

Chart review across EU5 in MM post-ASCT patients.

Aim: To understand the current treatment patterns, clinical outcomes and healthcare resource utilization-associated costs for multiple myeloma patients, post autologous stem cell transplant (ASCT) across Europe.

Patients & Methods: Medical records were used to abstract data for 337 multiple myeloma patients who had received ASCT.

Results: Following ASCT, 7% received maintenance therapy prior to progression.

The Cost Impact of Lenalidomide for Newly Diagnosed Multiple Myeloma in the EU5.

Introduction: Lenalidomide is an active agent that was approved for use in the EU in 2015 as a first-line therapy for previously untreated, non-transplant eligible multiple myeloma patients. Our objective was to assess the cost impact of lenalidomide when selected as a first-line treatment for transplant-ineligible patients in France, Germany, Italy, Spain, and the United Kingdom (EU5).

Methods: We developed a cost-impact model of the total costs associated with newly diagnosed multiple myeloma over 5 years in the EU5 based on treatment duration and time to progression (TTP) (taken from trial data).

Association between Transfusion Status and Overall Survival in Patients with Myelodysplastic Syndromes: A Systematic Literature Review and Meta-Analysis.

Introduction: Multiple studies show that transfusion independence (TI) in myelodysplastic syndrome (MDS) has a positive impact on overall survival (OS). To assess this, a systematic review and meta-analysis of the association between TI and OS in patients with MDS was conducted (PROSPERO ID: CRD42014007264).

Methods: Comprehensive searches of 5 key bibliographic databases were conducted and supplemented with additional search techniques.

Overall survival of relapsed and refractory multiple myeloma patients after adjusting for crossover in the MM-003 trial for pomalidomide plus low-dose dexamethasone.

In the phase III MM-003 trial, pomalidomide plus low-dose dexamethasone (POM+LoDEX) improved overall survival (OS) versus high-dose dexamethasone (HiDEX) in 455 patients with relapsed and refractory multiple myeloma (RRMM) after treatment with bortezomib and lenalidomide. Here, a two-stage Weibull method was used to adjust for the crossover of patients in the HiDEX arm to pomalidomide-based therapy. The adjusted difference in median OS between patients in the POM+LoDEX and HiDEX arms was 7·0 months (12·7 vs.

Lenalidomide for multiple myeloma: cost-effectiveness in patients with one prior therapy in England and Wales.

Purpose: To determine the cost effectiveness of lenalidomide plus dexamethasone (LEN/DEX) versus DEX alone in managing multiple myeloma (MM) patients who have failed one prior therapy.

Materials And Methods: An individual simulation model was designed to capture the costs and outcomes of LEN/DEX versus DEX therapy in relapsed refractory MM patients. MM009/010 efficacy data were adjusted for treatment cross-over and extrapolated to patient lifetime.

Economic impact of services for first-episode psychosis: a decision model approach.

Aim: To assess the impact of early intervention (EI) services on service costs for people with first-episode psychosis.

Methods: A decision model was constructed to map the care pathways following input from EI services and from standard care. A Markov process was used to run the model over 18 2-month cycles.