A roadmap towards improving outcomes in multiple myeloma.

Multiple myeloma (MM) is a chronic hematologic malignancy that remains incurable, because most patients eventually relapse or become refractory to current treatments. MM is a major health problem, with a globally increasing incidence. While, increase in the choice of MM treatment, including new immunotherapies (bispecific monoclonal antibodies and chimeric antigen receptor (CAR)-T cell therapy), may allow to further improve MM patients' outcomes, some non-therapy-related key issues may represent a pre-requisite towards improving MM outcomes in the next few years.

Potential future direction of measurable residual disease evaluation in multiple myeloma.

Multiple myeloma remains an incurable disease plagued by high relapse rates. Deeper and more sustainable responses, however, have been consistently shown to improve outcomes and could eventually pave the way to achieving a cure. Our understanding of disease response has surpassed complete response (CR), because the current definitions are suboptimal, and the treatment goal should aim even beyond stringent CR, toward molecular and flow CR, that is, measurable residual disease (MRD) negativity.

Current Advances in Multiple Myeloma: A Post International Myeloma Society (IMS 2022) Round Table Debate by the International Academy for Clinical Hematology (IACH).

This round table discussion organized by the International Academy for Clinical Hematology (IACH) was dedicated to the 19th annual meeting of the International Myeloma Society (IMS), which was held in Los Angeles between the 25th and 27th August 2022. After some key meetings of the discipline of the field of clinical hematology, the IACH organizes regular round table discussion in order to summarize the flow of information and get the opinion of a panel of experts and the key take-home messages. As part of this discussion, the panellists debated 6 key topics: disease monitoring, management of high-risk multiple myeloma (MM), induction for newly-diagnosed MM, management of relapsed MM, immune reconstitution, and vaccination and cellular therapy in MM.

Beyond medicines' barriers: exploring the true cost of multiple myeloma.

Aim: The goal of this research was to quantify and qualify all the costs associated with multiple myeloma (MM) from a healthcare and societal perspective and to highlight certain costs that are often underestimated.

Materials And Methods: The study used a mixed methods approach that consisted of three phases: a systemic literature review (SLR), a virtual roundtable discussion based on the results of the SLR, and an online survey.

Results: In total, 4321 records were identified by literature and snowball searches.

Systematic literature review of health economic models developed for multiple myeloma to support future analyses.

Aims: The goal of this study was to review the economic evaluations of health technologies in multiple myeloma (MM) and provide guidance and recommendations for future health economic analyses.

Materials And Methods: A systemic literature review (SLR) was conducted on original economic assessment studies and structured review papers focusing on the studies in MM. The search was limited to English language papers published from 1 January 2000 onwards.

Patient and Caregiver Experience Decision Factors in Treatment Decision Making: Results of a Systematic Literature Review of Multiple Myeloma Decision Aids.

Objectives: Decision-aids (DAs) may facilitate shared decision-making for patients and caregivers, by providing evidence-based information to assist healthcare professionals, patients, and caregivers in making choices about aspects of care, and/or highlighting decision factors to discuss with the potential of altering the treatment decision. These decision factors may not be well integrated in DAs.

Methods: A systematic literature review was conducted in the field of multiple myeloma (MM) on peer-reviewed publications, extended with a gray literature search.

Induction therapy prior to autologous stem cell transplantation (ASCT) in newly diagnosed multiple myeloma: an update.

The current standard of care model for newly diagnosed fit multiple myeloma (NDMM) patients is the sequential treatment of induction, high dose melphalan, autologous stem cell transplantation (ASCT), and maintenance. Adequate induction is required to achieve good disease control and induce deep response rates while minimizing toxicity as a bridge to transplant. Doublet induction regimens have greatly fallen out of favor, with current international guidelines favoring triplet or quadruplet induction regimens built around the backbone of the proteasome inhibitor bortezomib and dexamethasone (Vd).

Requirements for operational cure in multiple myeloma.

Multiple myeloma is usually considered an incurable disease. However, with the therapeutic improvement observed in the past few years, achievement of an operational cure is increasingly becoming a realistic goal. The advent of novel agents, with or without high-dose chemotherapy or autologous transplantation, revealed a correlation between depth of response to treatment and outcome.

Treatment of relapsed and refractory multiple myeloma: recommendations from the International Myeloma Working Group.

This Policy Review presents the International Myeloma Working Group's clinical practice recommendations for the treatment of relapsed and refractory multiple myeloma. Based on the results of phase 2 and phase 3 trials, these recommendations are proposed for the treatment of patients with relapsed and refractory disease who have received one previous line of therapy, and for patients with relapsed and refractory multiple myeloma who have received two or more previous lines of therapy. These recommendations integrate the issue of drug access in both low-income and middle-income countries and in high-income countries to help guide real-world practice and thus improve patient outcomes.

Relapsed refractory multiple myeloma: a comprehensive overview.

Most patients with relapsed/refractory multiple myeloma (RRMM) have been treated with drug combinations including a proteasome inhibitor (PI) and/or an immunomodulatory drug (IMiD). The goal of therapy for such patients is therefore to achieve disease control with acceptable toxicity and patient-defined decent quality of life. Physicians face a difficult task not only deciding who to treat, but also when to treat and how to treat, utilizing knowledge of previously administered therapies, patient comorbidities, potential adverse events, and patient wishes to make such a critical decision.

Analysis of Availability and Access of Anti-myeloma Drugs and Impact on the Management of Multiple Myeloma in Latin American Countries.

Introduction: Latin American countries (LATAMC) represent a large fraction of patients treated for multiple myeloma (MM) worldwide. In order to understand the difficulty of access to anti-myeloma therapy in LATAMC, we designed this study that explores areas involved in the availability of drugs, such as health care systems, approval times, coverage of new agents, old drugs, use of generics, and the first-line treatments.

Material And Methods: We collected data from 16 countries in 2015.

Minimal residual disease negativity using deep sequencing is a major prognostic factor in multiple myeloma.

The introduction of novel agents has led to major improvements in clinical outcomes for patients with multiple myeloma. To shorten evaluation times for new treatments, health agencies are currently examining minimal residual disease (MRD) as a surrogate end point in clinical trials. We assessed the prognostic value of MRD, measured during maintenance therapy by next-generation sequencing (NGS).

How I treat first relapse of myeloma.

The standard treatment of relapsed multiple myeloma has been either lenalidomide-dexamethasone (RD) or bortezomib-dexamethasone (VD) but it is changing rapidly for 2 reasons. First, lenalidomide and bortezomib are currently used in frontline treatment and many patients become resistant to these agents early in the course of their disease. Second, 6 second-line new agents have been recently developed and offer new possibilities (pomalidomide, carfilzomib and ixazomib, panobinostat, elotuzumab, and daratumumab).

Lenalidomide, Bortezomib, and Dexamethasone with Transplantation for Myeloma.

Background: High-dose chemotherapy plus autologous stem-cell transplantation has been the standard treatment for newly diagnosed multiple myeloma in adults up to 65 years of age. However, promising data on the use of combination therapy with lenalidomide, bortezomib, and dexamethasone (RVD) in this population have raised questions about the role and timing of transplantation.

Methods: We randomly assigned 700 patients with multiple myeloma to receive induction therapy with three cycles of RVD and then consolidation therapy with either five additional cycles of RVD (350 patients) or high-dose melphalan plus stem-cell transplantation followed by two additional cycles of RVD (350 patients).

Addition of Androgens Improves Survival in Elderly Patients With Acute Myeloid Leukemia: A GOELAMS Study.

Purpose Elderly patients with acute myeloid leukemia (AML) have a poor prognosis, and innovative maintenance therapy could improve their outcomes. Androgens, used in the treatment of aplastic anemia, have been reported to block proliferation of and initiate differentiation in AML cells. We report the results of a multicenter, phase III, randomized open-label trial exploring the benefit of adding androgens to maintenance therapy in patients 60 years of age or older.

Multiple myeloma treatment at relapse after autologous stem cell transplantation: A practical analysis.

Over the past decade, significant advances have been made in the field of multiple myeloma. Introduction of the so-called novel agents, proteasome inhibitors (PI) and immunomodulatory drugs (IMiD), and improved supportive care have resulted in significantly better outcome. Standard first line treatment in fit patients include PI and IMiD based induction, high dose melphalan with autologous hematopoietic stem cell transplantation (ASCT) and consolidation/maintenance.

Late Complications and Quality of Life after Reduced-Intensity Conditioning Allogeneic Stem Cell Transplantation.

Late complications (LC) and quality of life (QOL) were analyzed in 110 adult patients who underwent reduced-intensity conditioning (RIC) allogeneic stem cell transplantation (allo-SCT) and were alive for more than 2 years after allo-SCT. Overall survival of these patients was 93% (95% confidence interval [CI], 88% to 99%) and 81% (95% CI, 71% to 94%) at 5 and 10 years, respectively. The primary cause of death was a recurrence of primary malignancy.