The New England Journal of Medicine May 30, 2019

Daratumumab plus Lenalidomide and Dexamethasone for Untreated Myeloma

Thierry Facon, Shaji K. Kumar, Torben Plesner, et al.

Bottom Line

The frontline addition of the CD38-directed monoclonal antibody daratumumab to lenalidomide and dexamethasone significantly improved progression-free survival and depth of response in transplant-ineligible patients with newly diagnosed multiple myeloma.

Key Findings

1. At a median follow-up of 28.0 months, daratumumab plus lenalidomide and dexamethasone (D-Rd) reduced the risk of disease progression or death by 44% compared to lenalidomide and dexamethasone alone (Rd) (HR 0.56; 95% CI, 0.43 to 0.73; P<0.001) [2.1.2].
2. Median progression-free survival (PFS) was not reached in the D-Rd arm, whereas it was 31.9 months in the Rd control arm.
3. The estimated 30-month progression-free survival rate was 71% in the D-Rd group compared to 56% in the Rd group.
4. D-Rd induced significantly deeper clinical responses, with 47.6% of patients achieving a complete response or better compared to 24.9% with Rd (P<0.001).
5. Minimal residual disease (MRD) negativity (at a threshold of 10^-5) was achieved by 24.2% of patients in the D-Rd arm versus 7.3% in the Rd arm (P<0.001).
6. The most common grade 3 or 4 adverse events in the D-Rd group were neutropenia (50.0% vs. 35.3% in the Rd group) and pneumonia (13.7% vs. 7.9%).

Study Design

Design
Phase 3 RCT
Open-Label
Sample
737
Patients
Duration
28.0 mo
Median
Setting
14 countries
Population Patients aged 18 years or older with newly diagnosed multiple myeloma who were ineligible for high-dose chemotherapy with autologous stem-cell transplantation due to age (≥65 years) or comorbidities.
Intervention Daratumumab (16 mg/kg IV weekly for cycles 1-2, every 2 weeks for cycles 3-6, and every 4 weeks thereafter) plus Lenalidomide (25 mg PO on days 1-21) and Dexamethasone (40 mg PO weekly) administered in 28-day cycles until disease progression.
Comparator Lenalidomide (25 mg PO on days 1-21) and Dexamethasone (40 mg PO weekly) in 28-day cycles until disease progression.
Outcome Progression-free survival (PFS)

Study Limitations

The open-label design of the trial could potentially introduce bias in investigator-assessed secondary outcomes and the reporting of adverse events.
At the time of the primary 2019 publication, overall survival (OS) data were immature and had not yet demonstrated a statistically significant difference.
The addition of continuous daratumumab led to higher incidences of severe infections, notably grade 3 or 4 pneumonia, requiring vigilant monitoring in an elderly, transplant-ineligible population.
Continuous indefinite triplet therapy featuring a monoclonal antibody introduces significant and sustained financial toxicity and logistical infusion burden compared to an oral-dominant regimen.

Clinical Significance

The MAIA trial established D-Rd as a foundational, standard-of-care frontline regimen for transplant-ineligible newly diagnosed multiple myeloma. By achieving unprecedented complete response and MRD-negativity rates in an older demographic, it proved that the integration of monoclonal antibodies into upfront continuous therapy provides massive survival and disease-control benefits without prohibitive toxicity.

Historical Context

Prior to the MAIA trial, continuous lenalidomide and dexamethasone (Rd) was established by the FIRST trial as a primary standard of care for older patients with newly diagnosed multiple myeloma who were ineligible for autologous stem-cell transplantation. Daratumumab, a first-in-class CD38-directed monoclonal antibody, had already transformed the relapsed/refractory treatment paradigm (POLLUX and CASTOR trials). MAIA successfully combined daratumumab with the preferred Rd backbone in the frontline setting, fundamentally shifting the treatment paradigm for older adults away from doublet regimens toward highly effective, targeted triplet therapies.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

What is the mechanism of action of daratumumab, and why is CD38 a highly targeted antigen in the pathophysiology of multiple myeloma?

Key Response

Daratumumab is an IgG1 kappa monoclonal antibody that binds to CD38, a transmembrane glycoprotein that is highly and uniformly expressed on the surface of multiple myeloma cells. Once bound, it induces rapid tumor cell death through multiple immune-mediated mechanisms, including complement-dependent cytotoxicity (CDC), antibody-dependent cell-mediated cytotoxicity (ADCC), antibody-dependent cellular phagocytosis (ADCP), and direct apoptosis, making it a highly specific targeted therapy.

Resident
Resident

Based on the MAIA trial results, how does the upfront use of Dara-Rd change the standard of care for a 75-year-old newly diagnosed, transplant-ineligible multiple myeloma patient compared to historical Rd or VRd regimens?

Key Response

Dara-Rd demonstrated unprecedented progression-free survival and deep responses in older, frail patients, establishing it as a preferred frontline regimen. It is often favored over VRd (bortezomib, lenalidomide, dexamethasone) in this population to avoid proteasome inhibitor-induced peripheral neuropathy while achieving highly durable efficacy, shifting the paradigm to monoclonal antibody-based triplet therapy even for non-transplant candidates.

Fellow
Fellow

How should we interpret the high rates of MRD (minimal residual disease) negativity achieved with Dara-Rd in a transplant-ineligible population, and does achieving MRD negativity justify early de-escalation of continuous therapy in these older patients?

Key Response

While the MAIA trial showed that Dara-Rd leads to significantly higher MRD negativity (which strongly correlates with improved PFS), the study was not designed to test therapy discontinuation based on MRD status. Fellows must balance the proven benefit of continuous therapy against cumulative toxicity in frail patients, acknowledging that MRD-guided de-escalation in this specific cohort remains an active area of investigation rather than current standard practice.

Attending
Attending

With the profound efficacy of Dara-Rd moving to the frontline setting, how does this alter our sequencing strategy for first relapse, particularly considering these patients will now be refractory to both an anti-CD38 antibody and an IMiD?

Key Response

Moving the most effective agents upfront yields the best initial survival curves but creates a challenging 'double-refractory' state at first relapse. Attendings must proactively plan for subsequent therapies, shifting agents like proteasome inhibitors (e.g., carfilzomib), next-generation IMiDs (e.g., pomalidomide), bispecific antibodies, or CAR-T cell therapy earlier in the disease course, fundamentally changing the relapsed/refractory treatment algorithm.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The MAIA trial utilized a continuous treatment approach until disease progression or unacceptable toxicity. How might the inclusion of a 'treat-to-target' fixed-duration arm have impacted the trial's power to detect a PFS difference, and what statistical methodologies are required to handle the competing risk of non-myeloma mortality in this elderly population?

Key Response

Continuous therapy in an elderly cohort introduces significant non-disease-specific mortality and cumulative toxicity dropouts, complicating standard Kaplan-Meier estimates. A fixed-duration arm would require non-inferiority margins or dynamic treatment effect modeling. Researchers must utilize Fine-Gray subdistribution hazard models to properly isolate myeloma-specific progression from age-related competing mortality risks.

Journal Editor
Journal Editor

A critical reviewer might note the absence of a triplet therapy control arm, such as VRd. How does comparing a novel triplet (Dara-Rd) to a doublet (Rd) rather than a contemporary standard-of-care triplet affect the editorial evaluation of the trial's magnitude of benefit and clinical applicability?

Key Response

At the time of trial design, Rd was a valid standard; however, VRd subsequently became a standard for transplant-ineligible patients. The lack of a VRd control means the trial proves Dara-Rd is superior to Rd but cannot definitively establish superiority over other triplets. Editors must highlight this limitation to ensure readers do not misinterpret the effect size as an absolute victory over all existing upfront regimens.

Guideline Committee
Guideline Committee

Given the unprecedented progression-free survival benefit seen in the MAIA trial, should NCCN and ESMO guidelines universally mandate Dara-Rd as the sole Category 1 preferred regimen for transplant-ineligible newly diagnosed multiple myeloma, or should VRd remain a co-preferred option?

Key Response

NCCN guidelines currently list Dara-Rd as a Category 1 preferred regimen for transplant-ineligible NDMM based on MAIA data. However, the committee must weigh factors like patient frailty, financial toxicity, access to daratumumab, and the presence of high-risk cytogenetics (where proteasome inhibitors in VRd might still offer unique advantages) to justify keeping VRd as a co-preferred option, ensuring individualized patient care.

Clinical Landscape

Noteworthy Related Trials

2014

FIRST Trial

n = 1,623 · NEJM

Tested

Continuous Lenalidomide and Dexamethasone (Rd)

Population

Transplant-ineligible newly diagnosed multiple myeloma

Comparator

Melphalan, prednisone, and thalidomide (MPT)

Endpoint

Progression-free survival (PFS)

Key result: Continuous Rd significantly improved PFS (25.5 vs 21.2 months) and overall survival compared to MPT.
2016

POLLUX Trial

n = 569 · NEJM

Tested

Daratumumab plus lenalidomide and dexamethasone (D-Rd)

Population

Relapsed or refractory multiple myeloma

Comparator

Lenalidomide and dexamethasone (Rd)

Endpoint

Progression-free survival (PFS)

Key result: D-Rd significantly prolonged PFS and induced higher rates of overall response compared to Rd alone in patients with relapsed or refractory disease.
2018

ALCYONE Trial

n = 706 · NEJM

Tested

Daratumumab plus bortezomib, melphalan, and prednisone (D-VMP)

Population

Transplant-ineligible newly diagnosed multiple myeloma

Comparator

Bortezomib, melphalan, and prednisone (VMP)

Endpoint

Progression-free survival (PFS)

Key result: The addition of daratumumab to VMP reduced the risk of disease progression or death by 50 percent compared to VMP alone.

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