Abstract
Acute graft-versus-host disease (aGVHD) following allogeneic hematopoietic cell transplant (HCT) is the major reason for nonrelapse mortality (NRM), and thus is a major determinant of long-term survival. Clinical trials of new aGVHD treatments are needed to identify approaches that will ultimately improve upon HCT survival. At present, it is not clear how quickly response to GVHD treatment needs to be established to reliably categorize patients at high risk for death or to promptly identify those who might benefit from alternate treatment. Therefore, we analyzed time to response from onset of aGVHD treatment in 180 patients who were enrolled on a national, randomized, phase II aGVHD treatment clinical trial whose initial treatment of GVHD consisted of high-dose steroids plus a second immunosuppressive agent. The aim of this analysis was to determine whether time to aGVHD treatment response predicts patient outcomes, especially survival. We used response at 14, 28, and 56 days from initiation of aGVHD treatment to categorize patients for NRM and survival. Multivariate analyses and specificity/sensitivity analyses identified that day 28 response (complete or partial response) best categorized patients by NRM and survival at 9 months from start of aGVHD treatment. If verified as a reliable predictor of late outcomes following other aGVHD treatment approaches, day 28 response should serve as a standard early endpoint for future trials of aGVHD therapy.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 1693-1699 |
| Number of pages | 7 |
| Journal | Biology of Blood and Marrow Transplantation |
| Volume | 16 |
| Issue number | 12 |
| DOIs | |
| State | Published - Dec 2010 |
Bibliographical note
Funding Information:Financial disclosure: This work was supported in part by the National Heart, Lung, and Blood Institute and the National Cancer Institute . The authors gratefully acknowledge the participating centers and coinvestigators for the BMT CTN 0302 study: MD Anderson Cancer Research Center (Amin M. Alousi, MD), Johns Hopkins University (Javier Bolaños-Meade, MD, and Georgia Vogelsang, MD), University of Minnesota (Daniel Weisdorf, MD), Dana Farber Brigham Women's Partners (Vincent Ho, MD, Robert Soiffer, MD, and Joseph Antin, MD), University of Michigan (John Levine, MD, MS, and James L. Ferrara, MD), Oregon Health Sciences University (Eneida Nemecek, MD), University of Florida College of Medicine (John Wingard, MD), University of Pennsylvania Hospital Center (Steven Goldstein, MD, and Edward Stadtmauer, MD), University of Nebraska Medical Center (Marcel Devetten, MD), Washington University of St. Louis (John DiPersio, MD, and Peter Westervelt, MD), Stanford Hospital and Clinics (Laura Johnston, MD), University of California at San Diego (Edward Ball, MD), Duke University Medical Center (Nelson Chao, MD, and Joanne Kurtzberg, MD), University Hospitals of Cleveland (CWRU) (Hillard Lazarus, MD), Memorial Sloan-Kettering Cancer Center (Nancy Kernan, MD, and Miguel-Angel Perales, MD), Texas Transplant Institute (Carlos Bachier, MD, Michael Grimley, MD, and Paul Shaughnessy, MD), City of Hope National Medical Center (Pablo Parker, MD), Fred Hutchinson Cancer Research Center (Richard Nash, MD), and Hackensack University Medical Center (Joel Brochstein, MD).
Keywords
- Allogeneic
- BMT
- GVHD
- Prognosis
- Response
- Survival
