Correlates of Patient-Reported Outcomes Following Allogeneic Unrelated Donor Hematopoietic Stem Cell Transplantation

Background: Allogeneic hematopoietic cell transplantation (HCT) is a critical therapy for various hematological disorders. This study investigated the impact of socioeconomic, demographic, and clinical factors on patient-reported outcomes (PROs) using Functional Assessment of Cancer Therapy - Bone Marrow Transplantation (FACT-BMT) and Short Form-36 Health Survey (SF-36) scores following allogeneic HCT.

Methods: We conducted a retrospective analysis using the P-5805 dataset from the publicly available Center for International Blood and Marrow Transplant Research (CIBMTR). The study included 118 patients who underwent allogeneic HCT, with information on PROs, social determinants of health, and clinical and transplant-related characteristics. PROs are crucial for understanding patient quality of life post-transplant. FACT-BMT evaluates the multidimensional well-being of bone marrow transplant recipients, while SF-36 assesses broad health domains, offering insights into physical and mental health. The association between patient characteristics and PROs was determined by unpaired t-tests for categorical variables and univariate linear regression for continuous variables. Mean differences (MD), along with their 95% confidence intervals (CI), were calculated for categorical variables, while the estimates and standard errors were reported for continuous variables. All statistical analyses were performed in R version 4.3.2, and statistical significance was defined as p<0.05.

Results: The median age at HCT was 54.2 years (range: 18.9-73.9); 58.5% were male, and 91.5% were Caucasian. Of 55 patients with household income data, 61.8% had an income of at least $60,000/year. Most patients (74.8%) were married or living with a partner, and 55.6% were employed full-time. Hematologic diagnoses were myeloid (61%), lymphoid (31%), and others (8%). Karnofsky's performance score of 90 and higher was noted in 62% of patients, and 60% of patients had an HCT-comorbidity index (HCT-CI) of less than 3. Myeloablative conditioning was used in 53% of patients. Donor types were matched unrelated (73%), mismatched unrelated (11%), and cord blood (16%). Graft sources were bone marrow (17%), peripheral blood stem cells (67%), and cord blood (16%). GVHD prophylaxis regimens included CD34 selection (71%), Tacrolimus or Cyclosporine-based (13%), and others (16%). The median FACT-BMT score was 103.2 (range: 52.8-141.0) and the median SF-36 score was 90.4 (range: 56.8-118.4). Lower household gross annual income (<$60,000) was associated with lower FACT-BMT (MD -12.19, 95% CI -21.92 to -2.46, p=0.02) and SF-36 scores (MD -8.90, 95% CI -16.92 to -0.88, p=0.03). Non-myeloid/lymphoid diseases were associated with lower FACT-BMT scores (MD -13.14, 95% CI -26.46 to 0.17, p=0.05). Karnofsky scores less than 90 correlated with lower FACT-BMT (MD -7.30, 95% CI -13.32 to -1.27, p=0.02) and SF-36 scores (MD -6.27, 95% CI -11.47 to -1.07, p=0.02). HCT-CI of 3 and higher was associated with lower SF-36 scores (MD -9.34, 95% CI -15.54 to -3.13, p=0.004). Transplant-related factors, including conditioning, donor and graft type, and GVHD prophylaxis, did not significantly impact PROs.

Conclusions: Low household income, poor performance status, and higher comorbidities adversely influence patient-reported outcomes after allogeneic HCT; however, no significant association was observed with transplant-related characteristics. Further studies are warranted to confirm these findings in larger, more diverse cohorts.