Karen K. Ballen

BACKGROUND: Outcomes among older patients with acute lymphoblastic leukemia remain poor. This study sought to determine the efficacy of an intensified, multi‐agent approach derived from a Dana‐Farber consortium trial in younger adults for patients older than 50 years (trial identifier NCT00973752).METHODS: The primary endpoint was overall survival (OS) at 1 year. Patients received induction chemotherapy with vincristine, prednisone, doxorubicin, and pegylated asparaginase. Imatinib was incorporated for Philadelphia chromosome–positive disease. After induction, the first consolidation incorporated clofarabine. Patients in remission could proceed to allogeneic hematopoietic cell transplantation (HCT) after induction and consolidation I. Those not receiving HCT went on to receive central nervous system, consolidation II, and continuation phases of treatment.RESULTS: Thirty patients were enrolled: 19 achieved a complete remission (CR) after induction and 1 achieved CR after consolidation I for a CR rate of 67%. Sixteen patients underwent HCT. The proportion surviving at 1 year was 63%, and this met the primary endpoint. The 2‐year OS rate was 52% (n = 30), and the 2‐year disease‐free survival rate was 52% for patients achieving CR (n = 20). There was no survival advantage among those undergoing HCT. Therapy‐related hyperbilirubinemia prompted adjustments and limitations to asparaginase dosing.CONCLUSIONS: Intensified chemotherapy can result in improved outcomes in comparison with historical data. Additional studies of similarly intensive regimens are warranted in this population. Cancer 2016;122:2379–2388. © 2016 American Cancer Society.

Summary: Umbilical cord blood is a haematopoietic progenitor cell source for patients with acute myeloid leukaemia (AML), other haematological malignancies and metabolic diseases who can be cured by allogeneic haematopoietic cell transplantation, but who do not have a human leucocyte antigen compatible related or unrelated donor. Although the first cord blood transplants were done in children, there are currently more cord blood transplants performed in adults. In this review, we explore the history of umbilical cord blood transplantation, paediatric and adult outcome results, and novel trends to improve engraftment and reduce infection. Umbilical cord blood transplantation cures approximately 30–40% of adults and 60–70% of children with AML. Controversial issues, including the use of double versus single cord blood units for transplantation, optimal cord blood unit selection, infection prophylaxis, conditioning regimens and graft versus host disease prophylaxis, will be reviewed. Finally, comparison to other graft sources, cost, access to care, and the ideal graft source are discussed.

215 Background: Patients undergoinghematopoietic stem cell transplantation (HCT) experience a steep deterioration in quality of life (QOL) and mood during hospitalization for HCT. The impact of this deterioration on patients’ long-term QOL and post-traumatic stress disorder (PTSD) symptoms is unknown. Methods: We conducted a prospective longitudinal study of patients hospitalized for HCT. At baseline (day-6), day+1, day+8, and 6 months post-HCT, we assessed QOL (Functional Assessment of Cancer Therapy-Bone Marrow Transplantation [FACT-BMT]) and mood (Hospital Anxiety and Depression Scale [HADS]). We used the PTSD Checklist to assess for PTSD symptoms at 6 months. We used multivariable linear regression models to identify predictors of QOL and PTSD symptoms at 6 months post-HCT. Results: We enrolled 97% (90/93) of consecutively eligible patients undergoing autologous (n = 30), myeloablative allogeneic (n = 30), or reduced intensity allogeneic (n = 30) HCT. Overall, patients’ QOL at 6 months (mean FACT-BMT: 110, 95%CI [104-116]) recovered to baseline pre-transplant values (mean FACT-BMT: 110, 95% CI [107-115]). At 6 months, 28.4% of participants met provisional diagnostic criteria for PTSD, and 43.3% had clinically significant depression. In multivariable regression analyses adjusting for baseline QOL, mood, other covariates, change in QOL and depression scores during hospitalization for HCT predicted impaired QOL (DQOL β = 1.13, P < 0.0001, D HADS-depression β = 2.51, P = 0.001) and PTSD symptoms (DQOL β = 0.50, P < 0.0001, DHADS-depression β = 1.22, P < 0.0001) at 6 months post-HCT. Conclusions: While patients’ overall QOL at 6 months post-HCT returned to baseline values, a significant proportion met provisional diagnostic criteria for PTSD and depression. The decline in QOL and increase in depressive symptoms during hospitalization for HCT were the most important predictors of long-term QOL impairment and PTSD symptoms. Future studies should evaluate whether interventions to improve QOL and reduce psychological distress during HCT may improve long-term QOL and reduce the risk of PTSD symptoms.

219 Background: In advanced cancers, patients’ prognostic perceptions influence their medical decisions. However, little is known about how patients with hematologic malignancies undergoing HCT and their family caregivers (FC) understand their prognosis. We examined prognostic perception in these patients during hospitalization for HCT and its relationship with QOL and mood. Methods: We conducted a longitudinal study of patients (and FC) hospitalized for HCT. At baseline (6 days pre-HCT), we used a 10-item questionnaire to measure patients’ and FC information preferences, and perception of prognosis. Using 2-items, we also asked oncologists regarding the patients’ prognosis. At day-6, day+1, day+8 of HCT, we assessed QOL (Functional Assessment of Cancer Therapy-Bone Marrow Transplantation FACT-BMT), and mood (Hospital Anxiety and Depression Scale). We examined the relationship between patients’ prognostic perception and their QOL and mood during hospitalization for HCT using multivariable linear mixed models. Results: We enrolled 97% of consecutively eligible patients undergoing autologous (n=30), myeloablative (n=30) or reduced intensity (n=30) allogeneic HCT. Most patients (80/90, 88.9%) and FC (41/47, 87.1%) believed that it is ‘extremely’ or ‘very’ important to know about prognosis. However, the majority of patients (66/85, 77.6%) and FC (33/46, 71.7%) reported an inaccurate and more optimistic perception of the patients’ prognosis compared to the oncologist (P < 0.0001). Patients with accurate prognostic understanding reported lower QOL (P = 0.03) and worse depressive symptoms (P = 0.04) with a steeper increase in depression (P = 0.006) over time compared to patients with an inaccurate optimistic prognostic perception. Conclusions: The majority of patients and FC reported inaccurate and more optimistic perception of prognosis compared with the oncologist. Patients with an accurate perception of their prognosis had lower QOL, higher depression, and a steeper increase in depression during hospitalization for HCT. Interventions are needed to improve prognostic understanding while providing adequate psychosocial support during HCT.