In a German phase III trial reported in JAMA Oncology, Bornhäuser et al found that allogeneic hematopoietic cell transplantation (HCT) was not associated with improved overall survival vs standard consolidation chemotherapy in patients aged ≤ 60 years with cytogenetically defined intermediate-risk acute myeloid leukemia (AML) in first complete remission.
Study Details
The open-label multicenter trial included 143 patients in first complete remission/complete remission with incomplete blood cell count recovery after conventional induction therapy and availability of a human leukocyte antigen–matched sibling or unrelated donor. Patients were randomly assigned between February 2011 and July 2018 to allogeneic HCT (n = 76) or high-dose cytarabine for consolidation, with salvage HCT in case of relapse (n = 67). The primary outcome measure was overall survival.
Key Findings
Median follow-up among surviving patients was 50 months (interquartile range [IQR] = 48–66 months). Overall survival at 2 years was 74% (95% confidence interval [CI] = 62%–83%) in the allogeneic HCT group vs 84% (95% CI = 73%–92%) in the consolidation chemotherapy group (P = .22).
Disease-free survival at 2 years was 69% (95% CI = 57%–80%) in the allogeneic HCT group vs 40% (95% CI =28%–53%) in the consolidation chemotherapy group (P = .001). The cumulative incidence of relapse at 2 years was 20% (95% CI = 13%–31%) vs 58% (95% CI = 47%–71%, P < .001). Nonrelapse mortality at 2 years was 9% (95% CI= 5%–19%) vs 2% (95% CI= 0%–11%, P = .005). All 41 patients with relapse after consolidation chemotherapy underwent salvage allogeneic HCT.
Serious adverse events of any grade were reported in 74% of both groups. Median hospital stay was 42.5 days (IQR = 31.0–62.0 days) in the allogeneic HCT group vs 106.0 days (IQR = 72.0–143.0 days) in the consolidation chemotherapy group (P < .001). No significant differences in health-related quality of life measures were observed between groups on the Medical Outcomes Study 36-Item Short-Form Health Survey.
The investigators concluded, “Primary allogeneic HCT during first complete remission was not associated with superior overall survival compared with consolidation chemotherapy in patients 60 years or younger with intermediate-risk AML during the first complete remission and an available donor.”
Martin Bornhäuser, MD, of University Hospital Carl Gustav Carus, Technical University Dresden, and Matthias Stelljes, MD, of University Hospital Münster, are the corresponding authors for the JAMA Oncology article.
Disclosure: The study was funded by the Deutsche Forschungsgemeinschaft. For full disclosures of the study authors, visit jamanetwork.com.
