title: Quality-adjusted survival analysis shows differences in outcome after immunosuppression or bone marrow transplantation in aplastic anemia.
abstract: Bone marrow transplantation (BMT) and immunosuppression (IS) have improved the prognosis of aplastic anemia; both treatments have specific advantages and drawbacks but similar survival rates. Analysis of additional endpoints may help in treatment decisions. In a single-center study, patients with aplastic anemia treated with IS (n=155) or BMT (n=52) were compared for survival, event-free survival, and quality-adjusted time without symptoms and toxicity (Q-TWiST). Probability of overall and event-free survival at 15 years was similar among both groups (BMT 51+/-15% and 25+/-14%, IS 53+/-10% and 27+/-8%), with more early deaths in the transplant group and more late deaths in the IS group. There were differences in terms of mean duration of seven analyzed health states: time with symptoms from treatment-related toxicity (IS 0.36 years, BMT 0.27), transfusion dependency (IS 0.66 years, BMT 0.1 years), partial remission (IS 3.27 years, BMT 1.42), and secondary clonal disorder (IS 0.68 years, BMT 0.04) was significantly longer for IS compared to BMT (p< or =0.001). Patients treated with BMT spent more time with extensive chronic graft-versus-host disease (GvHD) (IS 0 years, BMT 0.96, p<0.023) and in CR without drugs (IS 1.22 years, BMT 2.43, p=0.056). In conclusion, survival, event-free survival, and Q-TWiST are similar. BMT-treated patients had longer periods free from symptoms, while IS-treated patients needed closer medical care, transfusion support, and medications.
authors.affiliation: Transplantation Unit, University Hospitals of Basel, Petersgraben 4, 4031 Basel, Switzerland.
authors.forename: Romaine
authors.initials: R
authors.lastname: Viollier
authors.forename: Jakob
authors.initials: J
authors.lastname: Passweg
authors.forename: Michael
authors.initials: M
authors.lastname: Gregor
authors.forename: Geneviève
authors.initials: G
authors.lastname: Favre
authors.forename: Thomas
authors.initials: T
authors.lastname: Kühne
authors.forename: Catherine
authors.initials: C
authors.lastname: Nissen
authors.forename: Alois
authors.initials: A
authors.lastname: Gratwohl
authors.forename: André
authors.initials: A
authors.lastname: Tichelli
mesh_terms: D000293:Adolescent; D000328:Adult; D000741:Anemia, Aplastic / Q000401:mortality / Q000628:therapy*; D016026:Bone Marrow Transplantation / Q000401:mortality*; D002423:Cause of Death; D002648:Child; D002675:Child, Preschool; D005260:Female; D006801:Humans; D007165:Immunosuppression Therapy / Q000401:mortality*; D008297:Male; D008875:Middle Aged; D019057:Quality-Adjusted Life Years; D012189:Retrospective Studies; D016019:Survival Analysis; D016896:Treatment Outcome