Psychosocial Assessment of Candidates for Transplant (PACT) as a tool for psychological and social evaluation of allogeneic hematopoietic cell transplantation recipients.

Psychosocial Assessment of Candidates for Transplant (PACT) is a tool originally developed to address psychosocial risks in solid organ transplant recipients and has the potential for application to hematopoietic cell transplantation (HCT) recipients. In a retrospective cohort study, we reviewed 404 adult allogeneic HCT cases from 2003 to 2014 to identify predictors of adverse psychosocial status as determined by PACT. Final PACT rating was poor/borderline (score 0-1) in 5%, acceptable (score 2) in 22%, good (score 3) in 44%, and excellent (score 4) in 29% recipients. In multivariable regression, higher PACT score was associated with White race (odds ratio [OR] 2.95, P < 0.001), having a related donor (OR 1.61, P = 0.015), and a higher quality of life score (OR 1.22/ 10-point increase in FACT-BMT total score, P < 0.001). PACT score correlated with all quality of life subscales. The final PACT score was associated with non-relapse mortality (HR 0.82/ 1-point increase, p = 0.03) in multivariable analysis that considered patient and disease factors, but not in models that also included transplant-related factors and performance status. PACT score was not associated with overall survival. PACT can be considered as part of a comprehensive psychosocial assessment for identifying patients who may require additional resources around allogeneic HCT.

Assessment of Impact of HLA Type on Outcomes of Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Lymphocytic Leukemia.

Chronic lymphocytic leukemia (CLL) is a common hematologic malignancy with many highly effective therapies. Chemorefractory disease, often characterized by deletion of chromosome 17p, has historically been associated with very poor outcomes, leading to the application of allogeneic hematopoietic stem cell transplantation (allo-HCT) for medically fit patients. Although the use of allo-HCT has declined since the introduction of novel targeted therapy for the treatment of CLL, there remains significant interest in understanding factors that may influence the efficacy of allo-HCT, the only known curative treatment for CLL. The potential benefit of transplantation is most likely due to the presence of alloreactive donor T cells that mediate the graft-versus-leukemia (GVL) effect. The recognition of potentially tumor-specific antigens in the context of class I and II major histocompatibility complex on malignant B lymphocytes by donor T cells may be influenced by subtle differences in the highly polymorphic HLA locus. Given previous reports of specific HLA alleles impacting the incidence of CLL and the clinical outcomes of allo-HCT for CLL, we sought to study the overall survival and progression-free survival of a large cohort of patients with CLL who underwent allo-HCT from fully HLA-matched related and unrelated donors at Center for International Blood and Marrow Transplant Research transplantation centers. We found no statistically significant association of allo-HCT outcomes in CLL based on previously reported HLA combinations. Additional study is needed to further define the immunologic features that portend a more favorable GVL effect after allo-HCT for CLL.

Association of Socioeconomic Status with Outcomes of Autologous Hematopoietic Cell Transplantation for Multiple Myeloma.

Autologous hematopoietic cell transplantation (AHCT) is standard therapy for eligible patients with multiple myeloma. Health care disparities can influence transplantation outcomes. However, the association of socioeconomic status (SES), a major indicator of health care disparities, with outcomes in patients with myeloma after AHCT has not been previously described. We analyzed 346 consecutive AHCT recipients with myeloma who underwent transplantation between 2003 and 2013 in this retrospective cohort study. Zip code of residence at the time of AHCT was obtained to assess annual household income based on 2010 US census data (median, $49,054; range, $16,546 to $127,313). SES groups were divided into < $45,000 (low; n = 120), $45,000 to $60,000 (middle; n = 116), and > $60,000 (high; n = 110). The low-income cohort had smallest portion of Caucasians (69% versus 89% versus 91%); otherwise, patient, disease, and transplantation characteristics were comparable among cohorts or different without significant patterns found. Median follow-up was 49 months. There was no difference among SES groups in overall survival, progression-free survival, nonrelapse mortality, or relapse in univariate and multivariable analysis. Similarly, SES was not associated with survival in a subset analysis of 303 patients who had survived for 1 year after transplantation.

Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation.

Because the outcome of allogeneic hematopoietic cell transplantation (HCT) is predominantly influenced by disease type and status, it is essential to be able to stratify patients undergoing HCT by disease risk. The Disease Risk Index (DRI) was developed for this purpose. In this study, we analyzed 13,131 patients reported to the Center for International Blood and Marrow Transplant Research who underwent HCT between 2008 and 2010. The DRI stratified patients into 4 groups with 2-year overall survival (OS) ranging from 64% to 24% and was the strongest prognostic factor, regardless of age, conditioning intensity, graft source, or donor type. A randomly selected training subgroup of 9849 patients was used to refine the DRI, using a multivariable regression model for OS. This refined DRI had improved prediction ability for the remaining 3282 patients compared with the original DRI or other existing schemes. This validated and refined DRI can be used as a 4- or 3-group index, depending on the size of the cohort under study, for prognostication; to facilitate the interpretation of single-center, multicenter, or registry studies; to adjust center outcome data; and to stratify patients entering clinical trials that enroll patients across disease categories.

A Decision analysis to determine the appropriate treatment for low-risk myelodysplastic syndromes.

BACKGROUND: The myelodysplastic syndromes (MDS) are divided into low-risk and high-risk diseases. Predictive models for response to growth factors (GF) have been developed based on red blood cell transfusion needs and erythropoietin levels. For low-risk MDS the optimal initial therapy (GF vs nongrowth factor [NGF] therapies, including differentiation and immunomodulatory agents) based on response rates to NGF and GF and survival, has not been defined. METHODS: A Markov decision analysis was performed on 799 low-risk MDS patients treated with either GF or NGF to determine the appropriate initial therapy. The treatment strategies analyzed included initial GF or NGF therapies, assuming 3 different states: Patients were either in the good GF predictive group (low transfusion needs and low erythropoietin levels), intermediate, or the poor GF predictive group (high transfusion needs and high erythropoietin levels). RESULTS: In the good GF predictive group, initial therapy with GF improved survival compared with NGF therapies at 3.38 years vs 2.57 years for a typical MDS patient. The advantage of GF to NGF was lost when NGF therapies produced a response in >or=46% of patients. In the intermediate or poor GF predictive groups, NGF maximized survival, provided response rates for NGF were >14% and 4%, respectively, for each predictive group. Quality of life adjustment did not alter the preferred strategy. CONCLUSIONS: Modeling estimates suggest that patients who fall into a good GF predictive group should almost always receive GF initially, whereas those in intermediate and poor predictive groups should almost always be treated with NGF.