Early Integration of Palliative Care in Families of Children with Single Ventricle Congenital Heart Defects: A Quality Improvement Project to Enhance Family Support.

Children with single ventricle congenital heart defects (SVCHD) experience a significant risk of early mortality throughout their lifespan, particularly during their first year of life. Due to the intense care needed for these children and families, pediatric palliative care (PPC) team consults should be routine; however, medical staff are often reluctant to broach the idea of PPC to families. The involvement of PPC for many carries with it an association to end-of-life (EOL) care. Setting the standard of PPC involvement from the time of admission for the first palliative surgery led to increased family support, decreased days to consult, improved acceptance and communication. The purpose of this article is to describe a quality improvement project of early integration of PPC with families of children with SVCHD. Lessons learned will be presented, including the resources needed and the barriers encountered in assimilating PPC into the standard of care for all patients with SVCHD. The single ventricle (SV) and PPC teams collaborated to enhance the support given to SV families. Education was initiated with cardiology and PPC providers to understand the goal of consistent PPC consults beginning after birth for patients with SVCHD. Parents were educated during fetal consultation regarding the involvement of the PPC team. The SV team ensured compliance with the PPC initiative by identifying eligible patients and requesting consult orders from the primary providers. PPC consultation increased significantly over the 40 month study period to nearly 100% compliance for children with SVCHD who are undergoing pre-Fontan surgery. In addition, mean days to consult decreased dramatically during the study to a current average of 3 days into the patient's hospitalization; the data likely suggest that more PPC consults were routinely ordered versus urgently placed for unexpected complications. Data indicate that patients are being followed by the PPC team at an earlier age and stage in their SV journey which allows for more opportunity to provide meaningful support to these patients and families. The early involvement of the PPC team for children with SV physiology was operationally feasible and was accepted by families, thus allowing PPC providers to establish a therapeutic relationship early in the disease trajectory with the family. It allowed more continuity throughout the SV journey in a proactive fashion rather than a reactive manner.

Establishing psychosocial palliative care standards for children and adolescents with cancer and their families: An integrative review.

BACKGROUND: Despite standardization in disease assessments and curative interventions for childhood cancer, palliative assessments and psychosocial interventions remain diverse and disparate. AIM: Identify current approaches to palliative care in the pediatric oncology setting to inform development of comprehensive psychosocial palliative care standards for pediatric and adolescent patients with cancer and their families. Analyze barriers to implementation and enabling factors. DESIGN: Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines framed the search strategy and reporting. Data analysis followed integrative review methodology. DATA SOURCES: Four databases were searched in May 2014 with date restrictions from 2000 to 2014: PubMed, Cochrane, PsycINFO, and Scopus. A total of 182 studies were included for synthesis. Types of studies included randomized and non-randomized trials with or without comparison groups, qualitative research, prior reviews, expert opinion, and consensus report. RESULTS: Integration of patient, parent, and clinician perspectives on end-of-life needs as gathered from primary manuscripts (using NVivo coding for first-order constructs) revealed mutual themes across stakeholders: holding to hope, communicating honestly, striving for relief from symptom burden, and caring for one another. Integration of themes from primary author palliative care outcome reports (second-order constructs) revealed the following shared priorities in cancer settings: care access; cost analysis; social support to include primary caregiver support, sibling care, bereavement outreach; symptom assessment and interventions to include both physical and psychological symptoms; communication approaches to include decision-making; and overall care quality. CONCLUSION: The study team coordinated landmark psychosocial palliative care papers into an informed conceptual model (third-order construct) for approaching pediatric palliative care and psychosocial support in oncology settings.

Sorafenib treatment following hematopoietic stem cell transplant in pediatric FLT3/ITD acute myeloid leukemia.

BACKGROUND: FLT3/ITD is associated with poor outcomes in adult and pediatric acute myeloid leukemia (AML). Allogeneic hematopoietic stem cell transplantation (HSCT) can improve cure rates, however relapse is still common. Recent studies demonstrate the activity of FLT3 inhibitors, including sorafenib, in targeting the underlying mutation. PROCEDURE: We conducted a retrospective study of 15 pediatric patients with FLT3/ITD+ AML treated with sorafenib within 18 months after receiving HSCT. Sorafenib was administered either as prophylaxis in patients considered at very high risk for relapse (n = 6) or at the time of disease recurrence (n = 9). RESULTS: Sorafenib was initiated at a median of 100 days post HSCT. Overall, 11/15 (73%) of patients experienced medically significant toxicities. Among patients who experienced toxicity, 6/11 (55%) received treatment at doses above what was later determined to be the maximum tolerated dose of sorafenib for pediatric leukemia. Importantly, sorafenib did not appear to exacerbate graft versus host disease. Our findings suggest that sorafenib may be of particular efficacy in patients with minimal residual disease (MRD); all patients who received sorafenib for MRD immediately prior to transplant or with emergence post-HSCT are alive and remain in complete remission at a median of 48 months post HSCT. CONCLUSIONS: Our case series suggests that sorafenib administration is feasible and tolerable in pediatric FLT3/ITD+ AML patients early post HSCT. Ongoing prospective controlled studies are needed to further define the dosing of sorafenib in the post-HSCT period and to determine the optimal context for this treatment approach.

Palliative Care as a Standard of Care in Pediatric Oncology.

The study team conducted a systematic review of pediatric and adolescent palliative cancer care literature from 1995 to 2015 using four databases to inform development of a palliative care psychosocial standard. A total of 209 papers were reviewed with inclusion of 73 papers for final synthesis. Revealed topics of urgent consideration include the following: symptom assessment and intervention, direct patient report, effective communication, and shared decision-making. Standardization of palliative care assessments and interventions in pediatric oncology has the potential to foster improved quality of care across the cancer trajectory for children and adolescents with cancer and their family members.

Transitions of Care in Patients With Inborn Errors of Immunity.

Inborn errors of immunity (IEI) are a group of inherited conditions caused by damaged monogenic variants that result in impairment and/or dysregulation within the immune system. IEI are typically diagnosed in infancy or early childhood, with clinical presentations that include increased susceptibility to infections, immune dysregulation, autoinflammation, bone marrow failure, and/or malignancy. Historically, transitions of care experienced by patients with IEI have not been well described in the literature. However, with treatment advances extending the long-term survival of patients, this has become a primary area of research. It is crucial to establish guidelines and recommendations specific to the transition of patients with IEI. Transitions may include patients who naturally progress from pediatric to adult care, from inpatient to outpatient settings, or from their established health care team to a new team (ie, moving from one geographic area to another). This narrative review summarizes the current data on transitions of care and describes the health care challenges and patient-related barriers impacting transitions of care. Frameworks with practical guidance on how health care practitioners can better manage care transitions faced by patients with IEI are presented.

Metabolic syndrome and endocrine dysfunctions after HSCT in children.

MS and endocrine dysfunction(s) are common well-recognized complications after HSCT. We retrospectively analyzed our data on 160 patients with a median age at transplant of five yr (0.3-23), who had been followed for a median of seven yr (range 3-18) at Nationwide Children's Hospital after transplant. Dyslipidemia and MS were seen in 13% and 7.5% patients, respectively, and 58% of these patients were <20 yr of age. Twelve patients met the criteria for diagnosis of MS, but four of these did not meet the International Diabetic Federation or WHO criteria. Variation in the diagnostic criteria for MS leading to underdiagnosis is discussed. Female gonadal failure (27%) and hypothyroidism (21%) were the most common endocrine dysfunctions, followed by short stature and GH deficiency (17%) each. TBI and younger age at HSCT were associated with the highest burden of long-term effects, and female sex was more significantly associated with MS-related dysfunction (p < 0.05). Uniform diagnostic criteria for MS and close follow-up after transplant are important for the early diagnosis and management of these late effects, thereby improving the overall quality of life of these patients.

Disseminated Rhizomucor pusillus causing early multiorgan failure during hematopoietic stem cell transplantation for severe aplastic anemia.

Matched sibling donor hematopoietic stem cell transplantation is the standard of care for severe aplastic anemia, with an overall survival of 80% to 90%. Only 60% to 70% of patients respond to treatment with immunosuppressive therapy. The main life threatening complications are infections, graft failure, and graft versus host disease. A 10-year-old patient with severe aplastic anemia underwent matched sibling donor hematopoietic stem cell transplantation, but developed sudden onset of fatal multiorgan failure on day +12. The cause of death was found only after autopsy.

A Retrospective Review to Determine If Children with Sickle Cell Disease Receive Hydroxyurea Monitoring.

INTRODUCTION: Sickle Cell Disease (SCD) guidelines recommend that patients on hydroxyurea receive monitoring at least every 2-3 months, but it is unknown if this occurs in clinical practice. This study aimed to determine if patients with SCD at Nationwide Children's Hospital (NCH) had at least four, in-person monitoring visits during a 12-month period and if frequent monitoring was associated with hydroxyurea adherence and clinical outcomes. METHODS: We performed a retrospective analysis of children on hydroxyurea for at least 12 months during 2010-2015. Patients' demographics, laboratory studies, prescriptions, and number of hydroxyurea and acute visits were recorded from their 12-month period that met eligibility criteria. Patients were considered frequently monitored if they had ≥4 hydroxyurea visits and adherent if they had prescriptions for hydroxyurea for ≥80% of the days in their 12-month period. RESULTS: Seventy-four children met the eligibility criteria and 57 (77%) had frequent monitoring. The most common reason for not obtaining frequent monitoring was missing a scheduled appointment. A greater proportion of frequently monitored patients were adherent to hydroxyurea (66.7% vs. 17.7%, p<0.001) and they had significantly fewer acute visits (median 1 vs. 2 visits, p=0.032) compared to infrequently monitored patients. CONCLUSIONS: Our study shows that most children on hydroxyurea at NCH received frequent monitoring and that it was associated with improved adherence and outcomes. Our results suggest that frequent in-person monitoring could be an opportunity to identify poorly adherent patients. These data inform our next quality improvement initiative that will maximize adherence to these monitoring guidelines.