Research Papers:

Improved outcome in acute myeloid leukemia patients enrolled in clinical trials: A national population-based cohort study of Danish intensive chemotherapy patients

Lene Sofie Granfeldt Østgård _, Mette Nørgaard, Henrik Sengeløv, Bruno C. Medeiros, Lars Kjeldsen, Ulrik Malthe Overgaard, Marianne Tang Severinsen, Claus Werenberg Marcher, Morten Krogh Jensen and Jan Maxwell Nørgaard

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Oncotarget. 2016; 7:72044-72056. https://doi.org/10.18632/oncotarget.12495

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Lene Sofie Granfeldt Østgård1,2, Mette Nørgaard2, Henrik Sengeløv3, Bruno C. Medeiros4, Lars Kjeldsen3, Ulrik Malthe Overgaard5, Marianne Tang Severinsen6,7, Claus Werenberg Marcher8, Morten Krogh Jensen9, Jan Maxwell Nørgaard1

1Department of Hematology, Aarhus University Hospital, Aarhus, Denmark

2Department of Clinical Epidemiology, Aarhus University Hospital, Aarhus, Denmark

3Department of Hematology, The University Hospital Rigshospitalet, Copenhagen, Denmark

4Stanford University School of Medicine, Stanford, CA, United States

5Department of Hematology, Copenhagen University Hospital, Herlev, Denmark

6Department of Hematology, Aalborg University Hospital, Aalborg, Denmark

7Department of Clinical Medicine, Aalborg University, Aalborg, Denmark

8Department of Hematology, Odense University Hospital, Odense, Denmark

9Department of Hematology, Copenhagen University Hospital, Roskilde, Denmark

Correspondence to:

Lene Sofie Granfeldt Østgård, email: [email protected]

Keywords: acute myeloid leukemia, prognosis, chemotherapy, population-based, trials

Received: June 21, 2016     Accepted: September 28, 2016     Published: October 06, 2016


Clinical trials are critical to improve AML treatment. It remains, however, unclear if clinical trial participation per se affects prognosis and to what extent the patients selected for trials differ from those of patients receiving intensive therapy off-trial.

We conducted a population-based cohort study of newly diagnosed Danish AML patients treated with intensive chemotherapy between 2000–2013. We estimated accrual rates and compared characteristics, complete remission (CR) rates, and relative risks (RRs) of death at 90-day, 1-year, and 3-years in clinical trial patients to patients treated off-trial.

Of 867 patients, 58.3% (n = 504) were included in a clinical trial. Accrual rates were similar across age groups (p = 0.55). Patients with poor performance status, comorbidity, therapy-related and secondary AML were less likely to be enrolled in trials. CR rates were 80.2% in trial-patients versus 68.6% in patients treated off- trial. Also, trial-patients had superior survival at 1-year; 72%, vs. 54% (adjusted RR of death 1.28(CI = 1.06–1.54)), and at 3 years; 45% vs. 29% (adjusted RR 1.14(CI = 1.03–1.26)) compared to patients treated off-trial.

Despite high accrual rates, patients enrolled in clinical trials had a favorable prognostic profile and a better survival than patients treated off-trial. In conclusion, all trial results should be extrapolated with caution and population-based studies of “real world patients” have a prominent role in examining the prognosis of AML.

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