title: Long-term followup of health status in patients with severe rheumatoid arthritis after high-dose chemotherapy followed by autologous hematopoietic stem cell transplantation.
abstract: OBJECTIVE
  High-dose chemotherapy (HDC) followed by autologous hematopoietic stem cell transplantation (HSCT) is a new treatment for patients with severe, refractory rheumatoid arthritis (RA). The present study was undertaken to assess the health status of patients with severe RA over a long-term followup period after treatment with HDC + HSCT.
  
  
  METHODS
  Health status and utility scores were assessed in 8 patients before and after treatment with HDC + HSCT. Patients were followed up for 5 years posttransplantation. Health status was assessed by the Health Assessment Questionnaire (HAQ), the RAND-36 version of the Short Form 36 (SF-36) health survey, and the Arthritis Impact Measurement Scales (AIMS). Utility scores were calculated using the EuroQol (EQ-5D) questionnaire and the SF-36-derived utility index (called the SF-6D), from which quality-adjusted life years (QALYs) were derived.
  
  
  RESULTS
  Most measures of health status improved compared with baseline in the first 2 years posttransplantation, notably HAQ and AIMS scores and scores on the functional status, general health, and health change summary scales of the RAND-36 version of the SF-36. Utility scores derived from the EQ-5D questionnaire and the SF-6D also increased significantly after transplantation. This was reflected in the 0.28 QALYs gained compared with baseline. For a putative 50-year-old RA patient with a life expectancy of 20 years, a threshold analysis revealed that HDC + HSCT yielded more QALYs than conventional therapy when treatment-related mortality (TRM) was <2.8%.
  
  
  CONCLUSIONS
  HDC + HSCT temporarily increased the functionality and health status of patients with severe, refractory RA. With a reported TRM of 1.3%, HDC + HSCT can be considered a realistic treatment option for patients with severe RA.
authors.affiliation: Leiden University Medical Center, The Netherlands.
authors.forename: Y K O
authors.initials: YK
authors.lastname: Teng
authors.forename: R J
authors.initials: RJ
authors.lastname: Verburg
authors.forename: J K
authors.initials: JK
authors.lastname: Sont
authors.forename: W B
authors.initials: WB
authors.lastname: van den Hout
authors.forename: F C
authors.initials: FC
authors.lastname: Breedveld
authors.forename: J M
authors.initials: JM
authors.lastname: van Laar
mesh_terms: D000328:Adult; D018501:Antirheumatic Agents / Q000008:administration & dosage* / Q000627:therapeutic use; D001172:Arthritis, Rheumatoid / Q000503:physiopathology* / Q000628:therapy*; D004305:Dose-Response Relationship, Drug; D005260:Female; D006304:Health Status; D018380:Hematopoietic Stem Cell Transplantation; D006801:Humans; D008137:Longitudinal Studies; D008297:Male; D008875:Middle Aged; D019057:Quality-Adjusted Life Years; D012720:Severity of Illness Index; D018434:Sickness Impact Profile
chemical_list: D018501:Antirheumatic Agents