Background: Stem cell transplant is a complex process with risk for transplant related morbidity and mortality. A potential contributor to poor outcomes is lack of comprehension and compliance to complicated medication regimens post discharge. The University of Virginia Health System Stem Cell Transplant (SCT) Program noted cases of non-adherence to immunosuppressive regimens and other key prophylactic antimicrobials post-discharge which significantly threatened the quality of outcomes. The purpose of this initiative was to develop methodology that would serve to improve patient understanding and adherence for discharge medications. Methods: Lean methodology was identified as the guiding framework and an A3 utilized as the problem solving tool. An interdisciplinary team consisting of SCT physicians, nurses, pharmacists, case managers, and nurse practitioners developed a process map incorporating all pre-and post-transplant steps of the discharge medication process. Results: Major opportunities identified included timing, consistency, and frequency of medication discharge teaching during the inpatient stay. The A3 highlighted variation in current practice, and the team recognized an opportunity to develop standard educational materials that would systematically be shared with the patient and family at strategic times throughout their inpatient stay. The collaborative discussions during the A3 served to showcase the strengths brought by each discipline, leading the team to decide the teaching approach would combine efforts between pharmacists and nurses. Bedside delivery of medications was implemented to ensure the patient left the unit with filled prescriptions and could demonstrate the ability to self-administer correct doses. The "teach-back" method was utilized to assess for patient comprehension of material presented. Discussion & Conclusion: Results demonstrate effectiveness of the strategy through qualitative feedback from patients and providers. In addition to improving understanding and adherence, the strategy has also proved to be a valuable quality control tool allowing the team to identify and correct two errors of omission prior to patient discharge. It is anticipated that patient satisfaction scores related to medication discharge teaching will also quantitatively reflect success of the new standard approach. Active engagement by frontline staff served to support development of a strategic multidisciplinary approach that improves outpatient adherence to critical medication regimens by involving the patient and their family at key times throughout the inpatient stay. The strategy could be replicated across a myriad of care spectrums thereby improving quality outcomes in complex care environments.

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Background: HCT is the treatment of choice for many children with non-malignant diseases. The aim of this study is to determine major transplant outcomes among pediatric patients (pts) transplanted in Brazil. Patients and methods: This retrospective study included 300 pts with Bone Marrow Failures syndromes (65%, n = 193), Primary Immunodeficiencies (24%, n = 72), Inborn Errors of Metabolism (7%, n = 22) and Hemoglobinopathies (4%, n = 13) who received first allo-HCT between 2010 and 2014 at 11 Brazilian BMT centers. All pts received unmanipulated bone marrow (BM, 86%) or cord blood (CB, 14%) grafts. HCT donors included match related (MRD, n = 140, 47%), fully matched unrelated (MUD, n = 108, 36%) and mismatched unrelated (MM-URD, n = 52, 17%). Reduced intensity conditioning was given to 167 pts (56%). Most (78%) received GVHD prophylaxis with calcineurin inhibitor plus methotrexate. Grading of aGVHD and cGVHD were per Glucksberg and NIH criteria and were uniform among all centers. Results: Median age was 8 y/o (range, .6 to 17) and 62% (n = 185) were male. With median follow up of 4.5 years, 4yoverall survival (OS) was better for MRD compared to matched URD or mismatched URD transplants (84% versus 71% versus 69%, respectively; P = .01). When compared MM-URD BM (n = 26), matched (n = 11) or mismatched unrelated CB (n = 26) transplant pts had a lower OS (HR = 2.73; P = .04). Twentytwo pts died before D+21 and therefore were not evaluable for engraftment. Primary graft failure (GF) was observed in 21 pts and was significantly higher after mismatched CB transplants (OR = 7.43; P = .03) compared to MM-URD BM transplants. 257 pts engrafted and 22 developed secondary GF at a median of 193 days (range, 39-431). Acute GVHD grade II-IV occurred in 47 pts at a median of 28 days after HCT with a cumulative incidence (CumInc) at 3 months of 16% (95% CI, 11-20). Chronic GVHD was reported in 60 pts at a median of 189 days after HCT with a CumInc at 2 years of 20% (95% CI, 15-24). Acute and chronic GVHD CumInc were not significantly different across all donor types. Seventy-three pts died at a median of 69 days after HCT. Major causes of death included infection (51%) and GVHD (15%). One-year and 100days TRM were 20% (95% CI, 15-24) and 17% (95% CI, 13-22), respectively. Conclusions: Children and adolescents with non-malignant diseases can achieve excellent outcomes after HCT from MRD in Brazil. We observed a high early mortality after HCT from unrelated CB donors in our study. For children lacking MRD, an URD donor is a suitable option, particularly from a MUD. Confirming earlier reports, incidence of chronic GVHD was low in this setting. This is the first pediatric multicenter study in Brazil for nonmalignant diseases and future studies will focus on strategies to reduce early mortality after unrelated CB HCT and will explore the role of HCT from haploidentical donors for children lacking MRD or MUD.

extramedullary leukemia. All of our patients who underwent haplo-HCT received G-CSF-mobilized T-cell-replete grafts followed by post-transplant high-dose cyclophosphamide on days +3 and +4. Data was collected on the following baseline characteristics: patient and donor sex, disease risk, type of hematologic malignancy, disease etiology, intensity of conditioning regimen, use of total body irradiation, CMV reactivation and hematopoietic cell transplantation-comorbidity index (HCT-CI) score. The study endpoints were overall survival (OS), event-free survival (EFS), cumulative incidence of transplant-related mortality (TRM) and rates of acute and chronic GVHD. Multivariate analyses were performed for OS and EFS using the Cox proportional hazard model. Fine-Grey regression model was used for TRM and acute and chronic GVHD. Of the 147 patients, 75 were MUDs, 46 were MRDs and 26 received haplo-HCTs. Patients who received haplo-HCTs had similar EFS (P ¼ 0.64; Fig. 1) and OS (P ¼ 0.61; Fig. 2) compared to patients who received MUD transplants. Patients who received MUD vs. MRD transplants had similar EFS (P ¼ 0.99; Fig. 1) and OS (P ¼ 0.83; Fig. 2). There was no difference in cumulative incidence of transplant-related mortality (P ¼ 0.26) or chronic GVHD (P ¼ 0.2) in the three cohorts. The cumulative incidence of acute GVHD was lower in the MRD cohort compared to the haplo-HCT and MUD cohorts (P ¼ 0.02). Based on our results, haplo-HCT for patients with active disease AML and MDS led to survival outcomes similar to those who received MUD or MRD transplants. These results suggest that for patients with AML or MDS and who have persistent disease at time of transplant, haplo-HCT is a viable option for patients without a matched related or unrelated donor.

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