Academic community partnership in acute promyelocytic leukemia and early mortality: The ECOG-ACRIN EA9131 trial

Authors

Anand P. Jillella, Division of Hematology and Oncology, Georgia Cancer Center, Augusta University, Augusta, Georgia.
Sandra J. Lee, Dana-Farber Cancer Institute, Boston, Massachusetts.
Jessica K. Altman, Robert H. Lurie Comprehensive Cancer Center, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
Selina M. Luger, Abramson Cancer Center, University of Pennsylvania School of Medicine, Philadelphia.
Martin S. Tallman, Robert H. Lurie Comprehensive Cancer Center, Northwestern University Feinberg School of Medicine, Chicago, Illinois.
James M. Foran, Mayo Clinic Comprehensive Cancer Center, Jacksonville, Florida.
Danielle Bradshaw, Division of Hematology and Oncology, Georgia Cancer Center, Augusta University, Augusta, Georgia.
Lisa Y. Law, Roseville Medical Center, Roseville, California.
Locke J. Bryan, Division of Hematology and Oncology, Georgia Cancer Center, Augusta University, Augusta, Georgia.
Abdallah Abou Zahr, Sanford Roger Maris Cancer Center, Fargo, North Dakota.
Kebede H. Begna, Mayo Clinic Comprehensive Cancer Center, Jacksonville, Florida.
Alexander E. Perl, Abramson Cancer Center, University of Pennsylvania School of Medicine, Philadelphia.
Joseph J. Vadakara, Geisinger Medical Center, Danville, Pennsylvania.
Rubina Qamar, Advocate Health - MidwestFollow
Raymond C. Bergan, Fred and Pamela Buffett Cancer Center, University of Nebraska, Omaha.
Michael J. Fisch, Department of General Oncology, The University of Texas MD Anderson Cancer Center, Houston.
Ruth C. Carlos, Department of Radiology, University of Michigan, Ann Arbor.
Lynne I. Wagner, Department of Health Policy and Management, University of North Carolina at Chapel Hill.
Vamsi K. Kota, Division of Hematology and Oncology, Georgia Cancer Center, Augusta University, Augusta, Georgia.
Mark R. Litzow, Mayo Clinic Comprehensive Cancer Center, Jacksonville, Florida.

Recommended Citation

Jillella AP, Lee SJ, Altman JK, et al. Academic Community Partnership in Acute Promyelocytic Leukemia and Early Mortality: The ECOG-ACRIN EA9131 Trial. JAMA Oncol. Published online February 27, 2025. doi:10.1001/jamaoncol.2024.7033

Abstract

Importance:Acute promyelocytic leukemia (APL) is an acute illness that presents with cytopenia, infections, and disseminated intravascular coagulation. Achieving remission has been shown to make a major difference in patient outcomes; however, early death rates in the first month have been as high as 30% due to acute presentation, comorbidities, the rarity of APL, and clinician inexperience.

Objective:To develop treatment strategies that would decrease estimated 1-month mortality from 30% to below 15%.

Design, setting, and participants:In this nonrandomized clinical trial, a treatment algorithm that focused on supportive care was used to prevent early death in patients with APL treated at academic and community health centers between August 2017 and July 2021. Because of the rarity of the disease, expert support was available 24/7 from 7 designated APL experts at 6 participating academic lead centers, and included an additional 293 community centers. When a patient presented with APL, an expert was contacted and a consensus treatment plan was developed using the algorithm and expert suggestions. There were no exclusion criteria and all patients with a confirmed diagnosis of APL regardless of age or comorbid conditions were enrolled. Expert support was provided throughout induction. Initial data analysis was conducted May 2023.

Main outcomes and measures:One-month mortality; additional objectives were to compare outcomes in academic and community centers and assess 1-year and overall survival.

Results:A total of 201 patients were enrolled from 43 centers; 62 at lead centers and 139 from 37 community centers. The median age was 53 years (range, 18-91 years), with 72 patients (35.8%) who were aged 60 years or older; 105 patients (52.2%) were male. Fifty-two patients (26.4%) were diagnosed with high-risk APL. The 1-month mortality rate was 6 deaths of 201 patients (3.0%; 95% CI, 1.1%-6.4%) after adjusting for 1 interim analysis. In a secondary analysis using the Kaplan-Meier method, the 1-month overall survival (OS) rate was 97.0% (95% CI, 93.5%-98.6%) and the 1-year OS rate was 94.5% (95% CI, 90.3%-96.9%).

Conclusions and relevance:In this nonrandomized clinical trial, use of an algorithm combined with expert support resulted in a dramatic decrease in early death in academic and community centers. Population-wide survival improved in this highly curable disease, which suggests that implementing an accessible support system with APL experts for comanagement is the most logical next step.

Type

Article

PubMed ID

40014329