The New England Journal of Medicine February 08, 2018

Daratumumab plus Bortezomib, Melphalan, and Prednisone for Untreated Myeloma

María-Victoria Mateos, Meletios A. Dimopoulos, Michele Cavo, Kenshi Suzuki, Andrzej Jakubowiak, Stefan Knop, et al.

Bottom Line

In transplant-ineligible patients with newly diagnosed multiple myeloma, the addition of the anti-CD38 monoclonal antibody daratumumab to bortezomib, melphalan, and prednisone significantly improved progression-free survival and depth of response.

Key Findings

1. At a median follow-up of 16.5 months, the 18-month progression-free survival rate was 71.6% in the daratumumab group versus 50.2% in the control group (Hazard Ratio for disease progression or death, 0.50; 95% CI, 0.38 to 0.65; P<0.001) [5.2.1].
2. The overall response rate (ORR) was significantly higher in the daratumumab group compared to the control group (90.9% vs. 73.9%; P<0.001).
3. The rate of complete response or better (including stringent complete response) was 42.6% in the daratumumab arm versus 24.4% in the control arm (P<0.001).
4. Minimal residual disease (MRD) negativity (at a threshold of 10^-5) was achieved by 22.3% of patients in the daratumumab group compared with only 6.2% of patients in the control group (P<0.001).
5. Grade 3 or 4 hematologic adverse events included neutropenia (39.9% in the daratumumab arm vs. 38.7% in the control arm), thrombocytopenia (34.4% vs. 37.6%), and anemia (15.9% vs. 19.8%).
6. Daratumumab-associated infusion-related reactions occurred in 27.7% of patients in the interventional group.

Study Design

Design
RCT
Open-Label
Sample
706
Patients
Duration
16.5 mo
Median
Setting
Multicenter, international
Population Patients with newly diagnosed multiple myeloma who were ineligible for high-dose chemotherapy with autologous stem-cell transplantation due to age (≥65 years) or substantial comorbidities.
Intervention Nine 6-week cycles of bortezomib, melphalan, and prednisone (VMP) plus daratumumab (16 mg/kg intravenously), followed by daratumumab maintenance every 4 weeks until disease progression or unacceptable toxicity.
Comparator Nine 6-week cycles of bortezomib, melphalan, and prednisone (VMP) alone, with no maintenance therapy after completion of cycle 9.
Outcome Progression-free survival (PFS)

Study Limitations

The open-label design of the trial could potentially introduce bias in the investigator assessment of outcomes and reporting of toxicity.
The control arm did not receive any maintenance therapy after the 9 induction cycles, while the interventional arm received continuous daratumumab maintenance until progression, confounding the assessment of whether the primary benefit comes from the initial 4-drug induction or the prolonged maintenance phase.
The backbone regimen of bortezomib, melphalan, and prednisone (VMP) is widely used in Europe and other regions but is not standard in the United States, where lenalidomide-based regimens (like Rd or VRd) are generally favored for transplant-ineligible patients.
At the time of this primary analysis, overall survival data were immature, though later follow-up confirmed an overall survival benefit.

Clinical Significance

The ALCYONE trial established daratumumab in combination with VMP as a highly efficacious, standard-of-care frontline regimen for older or transplant-ineligible patients with newly diagnosed multiple myeloma. It proved that adding a monoclonal antibody to a standard triplet backbone significantly deepens responses (including MRD negativity rates) and prolongs progression-free survival without compromising deliverability, signaling a paradigm shift toward quadruplet upfront therapies.

Historical Context

Historically, the standard of care for newly diagnosed multiple myeloma patients ineligible for autologous stem-cell transplantation consisted of doublet or triplet regimens such as VMP (bortezomib, melphalan, prednisone) or Rd (lenalidomide, dexamethasone). Daratumumab, a first-in-class human IgG1k monoclonal antibody targeting CD38, had already revolutionized the treatment of relapsed or refractory multiple myeloma as both monotherapy and in combinations (CASTOR and POLLUX trials). The ALCYONE trial was the first major phase 3 study to demonstrate its profound efficacy when moved into the first-line setting for transplant-ineligible patients.

Guided Discussion

High-yield insights from every perspective

Med Student
Medical Student

Daratumumab is a monoclonal antibody targeting CD38, which was added to the VMP regimen in this trial. What are the primary mechanisms by which daratumumab induces myeloma cell death, and why is CD38 an ideal target in multiple myeloma?

Key Response

CD38 is a transmembrane glycoprotein highly and uniformly expressed on myeloma cells, but expressed at relatively low levels on normal lymphoid and myeloid cells. Daratumumab causes cell death via multiple mechanisms: complement-dependent cytotoxicity (CDC), antibody-dependent cell-mediated cytotoxicity (ADCC), antibody-dependent cellular phagocytosis (ADCP), direct apoptosis upon cross-linking, and immunomodulatory effects such as the depletion of CD38+ regulatory T-cells and myeloid-derived suppressor cells (MDSCs).

Resident
Resident

In adding daratumumab to the VMP regimen for transplant-ineligible patients, what specific adverse events must a clinician be prepared to manage, particularly during the initial phases of therapy?

Key Response

Daratumumab is known for infusion-related reactions (IRRs), occurring in nearly half of patients, predominantly during the first infusion. Clinicians must administer premedication (corticosteroids, antipyretics, antihistamines) and use slow initial infusion rates. Additionally, the ALCYONE trial showed a higher rate of grade 3/4 infections (especially pneumonia) in the D-VMP arm, necessitating vigilance, potential antiviral prophylaxis (e.g., for herpes zoster), and consideration of IVIG for severe hypogammaglobulinemia.

Fellow
Fellow

The ALCYONE trial demonstrated the superiority of D-VMP over VMP; however, VMP is largely an older, predominantly European regimen. How does the choice of backbone (VMP vs. Rd as seen in the MAIA trial) influence your frontline management of transplant-ineligible multiple myeloma in modern practice?

Key Response

While ALCYONE proved the efficacy of adding daratumumab, the MAIA trial (Daratumumab plus lenalidomide and dexamethasone) has largely become the standard of care in the US. This is due to the continuous nature of lenalidomide/dexamethasone therapy compared to the fixed-duration, alkylator-heavy VMP regimen, avoiding melphalan-associated toxicity and leveraging the superior progression-free survival seen with D-Rd, pushing D-VMP to a secondary option in regions where lenalidomide is readily accessible.

Attending
Attending

The ALCYONE trial highlighted a dramatic increase in MRD negativity (at a threshold of 10^-5) when daratumumab was added to VMP. How should the achievement of MRD negativity in a highly frail, transplant-ineligible population guide our discussions regarding continuous versus fixed-duration therapy and potential de-escalation?

Key Response

While MRD negativity is strongly prognostic of PFS and OS, it is not yet standard practice to use MRD to stop therapy in frontline myeloma outside of clinical trials. The clinical challenge is determining whether pushing for deep responses (MRD-) justifies the added toxicity and financial burden of continuous daratumumab in older, frailer patients, and whether achieving sustained MRD negativity might eventually serve as a biomarker to safely de-escalate or pause therapy.

Scholarly Review

Critical appraisal through the lens of expert reviewers and guideline development

PhD
PhD

The trial utilized progression-free survival (PFS) as the primary endpoint. From a methodological standpoint, what are the limitations of using PFS and overall survival (OS) comparisons in a trial with a continuous therapy arm (daratumumab maintenance) versus a fixed-duration control arm (VMP alone)?

Key Response

Comparing a continuous intervention (D-VMP followed by daratumumab maintenance until progression) against a fixed-duration control (VMP for 9 cycles then observation) inherently biases longer-term endpoints like PFS in favor of the continuous arm. It prevents researchers from distinguishing whether the benefit is derived from the synergistic D-VMP induction or simply the effect of single-agent daratumumab maintenance delaying progression.

Journal Editor
Journal Editor

As a peer reviewer evaluating the internal validity of the ALCYONE trial, how does the asymmetry of the post-induction treatment phase between the two arms threaten the study's ability to isolate the specific benefit of quadruplet induction?

Key Response

A rigorous reviewer would flag that the experimental arm received D-VMP followed by continuous daratumumab, while the control arm stopped VMP after 9 cycles with no maintenance or placebo. Because the control arm lacked a maintenance phase, it is impossible to determine if the massive PFS benefit is driven by deep responses from the upfront quadruplet combination or merely the continuous suppression of disease by ongoing daratumumab monotherapy.

Guideline Committee
Guideline Committee

Based on the ALCYONE and MAIA trial results, how should NCCN and ESMO guidelines classify CD38-directed therapies for transplant-ineligible newly diagnosed multiple myeloma, and what level of evidence supports displacing traditional doublets (e.g., Rd or Vd) from Category 1 preferred status?

Key Response

NCCN and ESMO updated their guidelines to include D-VMP and D-Rd as Category 1, preferred options for transplant-ineligible NDMM based on unprecedented PFS and OS benefits. The standard of care has definitively shifted from doublets to triplets (D-Rd) or quadruplets (D-VMP) for patients who can tolerate them, supported by high-quality randomized phase 3 data, reserving older doublets only for highly frail populations who cannot tolerate multi-agent regimens.

Clinical Landscape

Noteworthy Related Trials

2008

VISTA Trial

n = 682 · NEJM

Tested

Bortezomib, Melphalan, and Prednisone (VMP)

Population

Newly diagnosed, transplant-ineligible multiple myeloma patients

Comparator

Melphalan and Prednisone (MP)

Endpoint

Time to progression

Key result: VMP significantly improved time to progression (24.0 vs 16.6 months) and overall survival compared to MP.
2016

CASTOR Trial

n = 498 · NEJM

Tested

Daratumumab, Bortezomib, and Dexamethasone (D-Vd)

Population

Patients with relapsed or refractory multiple myeloma

Comparator

Bortezomib and Dexamethasone (Vd)

Endpoint

Progression-free survival

Key result: Daratumumab combined with Vd significantly prolonged progression-free survival and resulted in higher rates of overall response.
2019

MAIA Trial

n = 737 · NEJM

Tested

Daratumumab, Lenalidomide, and Dexamethasone (D-Rd)

Population

Newly diagnosed, transplant-ineligible multiple myeloma patients

Comparator

Lenalidomide and Dexamethasone (Rd)

Endpoint

Progression-free survival

Key result: Adding daratumumab to Rd significantly reduced the risk of disease progression or death by 44% compared to Rd alone.

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