The prognostic value of MRD in high-risk AML*

Panel discussion with Professors Stephane de Botton, Michael Heuser & Roberto Lemoli

This promotional feature is sponsored by Jazz Pharmaceuticals who have had an influence on the content

Vyxeos® Liposomal (44 mg/100 mg powder for concentrate for solution for infusion daunorubicin/cytarabine) (CPX-351) is indicated for the treatment of adults with newly diagnosed, therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC). *High-risk AML is defined as t-AML or AML-MRC
Click here for the prescribing information for Vyxeos® Liposomal | Refer to the SmPC for full safety information

Patients with high-risk AML – AML with myelodysplasia-related changes (AML-MRC) or treatment-related AML (t-AML), represent a subset of patients with generally poorer outcomes due to adverse cytogenetics and advanced age.1 For these patients, the main curative pathway remains intensive chemotherapy followed by allogeneic hematopoietic stem cell transplantation (alloHSCT).2

Only a few patients with high-risk disease achieve deep and durable remission with conventional chemotherapy1 and attempts to improve outcomes for patients after induction therapy by the addition of other agents or intensification of post-remission therapy have failed.3 However, in patients with high-risk disease, CPX-351 (a liposomal formulation of daunorubicin and cytarabine),4 has demonstrated higher remission rates, improved overall survival compared with conventional cytarabine plus daunorubicin chemotherapy (7 + 3 regimen) (9.56 months [6.60–11.86] vs 5.95 months [4.99–7.75]: HR: 0.69, 95% CI: 0.52–0.90, p=0.005 [2-sided]), and maintained long term (18% 5-year overall survival [95% CI 12–25%] vs. 8% [4–13%] for the 7+3 group).5 In the phase 3 study, the overall frequency and severity of adverse events was comparable for CPX-351 and 7+3.5

In part 1, the panel discuss the use of CPX-351 in their clinical practice, and the importance of molecular profiling in these patients.

Part 1: Induction therapy for high-risk AML

“CPX-351 is approved…in Europe for the treatment of patients with myelodysplasia-related changes or therapy-related AML…and [has] showed to be superior to 3+7 conventional chemotherapy.” – Prof. Roberto Lemoli

There is increasing evidence that suggests achieving MRD pre-transplantation is an important prognostic biomarker in AML. Achieving a deep and durable remission, as evidenced by MRD negativity, has only recently become a more realistic goal with the advent of therapies like CPX-351.1
In patients receiving CPX-351, MRD-negativity rates of 36–64% have been reported across retrospective studies in adults with newly diagnosed disease.1

Part 2: MRD in practice

In this video, the panel discuss the use and prognostic value of MRD in AML – including in difficult-to-treat patient subgroups.

“The real-world data from Germany on CPX-351, that’s the patients who are MRD-negative, have an excellent transplantation outcome…none of these patients who made it to transplantation had a relapse or died in this follow-up.” – Prof. Michael Heuser

CPX-351 proved to be “effective in inducing MRD-negative CR. In our Italian trial, which has been published in the end of 2020.
Prof. Roberto Lemoli

Part 3: Case study discussion

In this final part of the discussion, the panel discuss the case of a male patient treated with CPX-351, who received an HSCT.

“In some cases, a consolidation [course with CPX-351] results in a deeper [response] with MRD-negative CR.” –  Prof. Roberto Lemoli

“After two consolidation courses [with CPX-351], MRD negativity was achieved. So, this is a very interesting case.”
Prof. Stephane de Botton

Click here for the prescribing information for Vyxeos® Liposomal

Adverse events should be reported. Healthcare professionals are asked to report any suspected adverse events via their national reporting system. Adverse events should also be reported to Jazz Pharmaceuticals by email to 


1. Cluzeau T, et al. Measurable residual disease in high-risk acute myeloid leukemia. Cancers (Basel) 2022;1:14:1278.

2. Bewersdorf JP et al. Are we witnessing the start of a therapeutic revolution in acute myeloid leukemia? Leuk Lymphoma 2019;60:1354–69.

3. Rautenberg C, et al. Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia. Blood Cancer Journal 2021;11:164

4. Vyxeos Liposomal. European Summary of Product Characteristics January 2021.

5. Lancet JE et al. CPX-351 versus 7+3 cytarabine and daunorubicin chemotherapy in older adults with newly diagnosed high-risk or secondary acute myeloid leukaemia: 5-year results of a randomised, open-label, multicentre, phase 3 trial. Lancet Haematol. 2021 Jul;8(7):e481-e491.

Relevant Disclosures

Stephane de Botton:
• Honoraria: BMS, AbbVie, Servier, Jazz Pharmaceuticals, Astellas;
• Consulting/Advisory role: Servier, BMS, GSK, Syndax
• Speakers Bureau: Servier, BMS, Jazz Pharmaceuticals, Astellas, AbbVie
• Research funding: Forma, Auron
• Travel/Accommodation/Expenses: AbbVie, Servier

Roberto Lemoli:
• Honoraria: BMS, Sanofi, StemLine, Jazz Pharmaceuticals, Pfizer

Michael Heuser:
• Research funding: Abbvie, Astellas, BergenBio,BMS, Bayer Pharma AG, Daiichi Sankyo, Jazz Pharmaceuticals, Karyopharm, Novartis, Pfizer, PinotBio, Roche
• Honoraria: Abbvie, Jazz Pharmaceuticals, Janssen, Novartis
• Consulting: Abbvie, BMS, Daiichi Sankyo, Jazz Pharmaceuticals, Novartis, Pfizer, PinotBio, Roche, Tolremo

INT-VYX-2200108 | June 2022

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