Quality-adjusted survival analysis shows differences in outcome after immunosuppression or bone marrow transplantation in aplastic anemia

Ann Hematol. 2005 Jan;84(1):47-55. doi: 10.1007/s00277-004-0930-3. Epub 2004 Aug 31.

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.

MeSH terms

  • Adolescent
  • Adult
  • Anemia, Aplastic / mortality
  • Anemia, Aplastic / therapy*
  • Bone Marrow Transplantation / mortality*
  • Cause of Death
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Immunosuppression Therapy / mortality*
  • Male
  • Middle Aged
  • Quality-Adjusted Life Years*
  • Retrospective Studies
  • Survival Analysis
  • Treatment Outcome