Background
Allogeneic hematopoietic cell transplant (HCT) cures many patients, but often with the risk of late effects and impaired quality of life. The value of quantifying patient-reported outcomes (PRO) is increasingly recognized, but routine collection of PROs is uncommon. We evaluated feasibility of prospective PRO collection by an outcomes registry, at multiple time points, in unselected HCT patients transplanted in centers contributing clinical data to the CIBMTR, and correlated those with clinical and demographic data.
Methods
FACT-BMT, SF-36 and PedsQL measures were administered pre-HCT, day 100, 6 and 12 months. Patients were recruited by the transplant center, but post-transplant PRO collection was managed centrally by CIBMTR.
Results
Of 580 eligible patients, 390 (67%) enrolled. Feasibility was shown by high time-specific retention rates (1-year: 176/238, 74%) and participant satisfaction. Factors associated with higher response rate were age >50 (OR 1.58, 95% CI 1.03-2.41, p=0.0355), Caucasian race (OR 4.61, 95% CI 2.66-7.99, p<0.0001) and being married (OR 2.28, 95% CI 1.42-3.65, p=0.0006) in adults; and higher family income in children (OR 4.99, 95% CI 2.12-11.75, p=0.0002). Importantly, pre-HCT PRO scores independently predicted survival after adjusting for patient-, disease- and transplant-related factors. Adjusted probabilities of 1-year survival by increasing quartiles of pre-HCT FACT-BMT and physical component score of SF-36 scores were 56%, 67%, 75%, 76% and 58%, 72%, 62%, 82%, respectively.
Conclusions
A hybrid model of local consent for centralized PRO collection is feasible, and pre-transplant PROs provide critical prognostic information for HCT outcomes.