title: A refined risk score for acute graft-versus-host disease that predicts response to initial therapy, survival, and transplant-related mortality.
abstract: To develop a novel acute graft-versus-host disease (GVHD) risk score, we examined the GVHD clinical stage and grade of 1723 patients at the onset of treatment with systemic steroids. Using clinical grouping, descriptive statistics and recursive partitioning, we identified poorly responsive, high-risk (HR) acute GVHD by the number of involved organs and severity of GVHD at onset. The overall response (complete response/partial response) rate 28 days after initiation of steroid therapy for acute GVHD was lower in the 269 patients with HR-GVHD than in the 1454 patients with standard risk (SR)-GVHD (44% [95% confidence interval (CI) 38% to 50%] versus 68% [95% CI, 66% to 70%], P < .001). Patients with HR-GVHD were less likely to respond at day 28 (odds ratio [OR], .3; 95% CI, .2 to .4; P < .001) and had higher risks of mortality (relative risk, 2.1; 95% CI, 1.7 to 2.6; P < .001) and transplant-related mortality (relative risk, 2.5; 95% CI, 2.0% to 3.2%, P < .001) than patients with SR-GVHD. This refined definition of acute GVHD risk is a better predictor of response, survival, and transplant-related mortality than other published acute GVHD risk scores. Patients with HR-GVHD are candidates for studies investigating new treatment approaches. Likewise, patients with SR-GVHD are candidates for studies investigating less toxic therapy.
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota. Electronic address: macmi002@umn.edu.
authors.forename: Margaret L
authors.initials: ML
authors.lastname: MacMillan
authors.affiliation: Hôpital Saint Louis, Paris, France.
authors.forename: Marie
authors.initials: M
authors.lastname: Robin
authors.affiliation: Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan.
authors.forename: Andrew C
authors.initials: AC
authors.lastname: Harris
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota; Department of Biostatistics and Informatics Core, Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota.
authors.forename: Todd E
authors.initials: TE
authors.lastname: DeFor
authors.affiliation: Fred Hutchinson Cancer Research Center, University of Washington, Seattle, Washington.
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Martin
authors.affiliation: MD Anderson Cancer Center, Houston, Texas; Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin.
authors.forename: Amin
authors.initials: A
authors.lastname: Alousi
authors.affiliation: Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin; Dana-Farber Cancer Institute, Boston, Massachusetts.
authors.forename: Vincent T
authors.initials: VT
authors.lastname: Ho
authors.affiliation: Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin; Johns Hopkins University, Baltimore, Maryland.
authors.forename: Javier
authors.initials: J
authors.lastname: Bolaños-Meade
authors.affiliation: Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan; Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin.
authors.forename: James L M
authors.initials: JL
authors.lastname: Ferrara
authors.affiliation: Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin; Johns Hopkins University, Baltimore, Maryland.
authors.forename: Richard
authors.initials: R
authors.lastname: Jones
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota; Department of Medicine, University of Minnesota, Minneapolis, Minnesota.
authors.forename: Mukta
authors.initials: M
authors.lastname: Arora
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota.
authors.forename: Bruce R
authors.initials: BR
authors.lastname: Blazar
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota; Department of Medicine, University of Minnesota, Minneapolis, Minnesota.
authors.forename: Shernan G
authors.initials: SG
authors.lastname: Holtan
authors.affiliation: Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin; Children's National Medical Center, Washington, District of Columbia.
authors.forename: David
authors.initials: D
authors.lastname: Jacobsohn
authors.affiliation: Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin; Medical College of Wisconsin, Milwaukee, Wisconsin.
authors.forename: Marcelo
authors.initials: M
authors.lastname: Pasquini
authors.affiliation: Hôpital Saint Louis, Paris, France.
authors.forename: Gerard
authors.initials: G
authors.lastname: Socie
authors.affiliation: Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin; Dana-Farber Cancer Institute, Boston, Massachusetts.
authors.forename: Joseph H
authors.initials: JH
authors.lastname: Antin
authors.affiliation: Blood and Marrow Transplant Program, University of Michigan, Ann Arbor, Michigan; Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin.
authors.forename: John E
authors.initials: JE
authors.lastname: Levine
authors.affiliation: Blood and Marrow Transplant Program, University of Minnesota, Minneapolis, Minnesota; Blood and Marrow Transplant Clinical Trials Network GVHD Study Committee, Milwaukee, Wisconsin; Department of Medicine, University of Minnesota, Minneapolis, Minnesota.
authors.forename: Daniel J
authors.initials: DJ
authors.lastname: Weisdorf
mesh_terms: D000208:Acute Disease; D000293:Adolescent; D000328:Adult; D000368:Aged; D064591:Allografts; D002648:Child; D002675:Child, Preschool; D018572:Disease-Free Survival; D005260:Female; D006086:Graft vs Host Disease / Q000209:etiology / Q000401:mortality*; D019337:Hematologic Neoplasms / Q000401:mortality* / Q000628:therapy*; D006801:Humans; D007223:Infant; D008297:Male; D008875:Middle Aged; D012307:Risk Factors; D033581:Stem Cell Transplantation; D015996:Survival Rate
keywords: Allogeneic hematopoietic cell transplantation; Grading systems; Graft-versus-host disease; Risk score; Survival; Transplant-related mortality