The codesign of an interdisciplinary team-based intervention regarding initiating palliative care in pediatric oncology.

PURPOSE: Children with advanced cancer are often not referred to palliative or hospice care before they die or are only referred close to the child's death. The goals of the current project were to learn about pediatric oncology team members' perspectives on palliative care, to collaborate with team members to modify and tailor three separate interdisciplinary team-based interventions regarding initiating palliative care, and to assess the feasibility of this collaborative approach. METHODS: We used a modified version of experience-based codesign (EBCD) involving members of the pediatric palliative care team and three interdisciplinary pediatric oncology teams (Bone Marrow Transplant, Neuro-Oncology, and Solid Tumor) to review and tailor materials for three team-based interventions. Eleven pediatric oncology team members participated in four codesign sessions to discuss their experiences with initiating palliative care and to review the proposed intervention including patient case studies, techniques for managing uncertainty and negative emotions, role ambiguity, system-level barriers, and team communication and collaboration. RESULTS: The codesign process showed that the participants were strong supporters of palliative care, members of different teams had preferences for different materials that would be appropriate for their teams, and that while participants reported frustration with timing of palliative care, they had difficulty suggesting how to change current practices. CONCLUSIONS: The current project demonstrated the feasibility of collaborating with pediatric oncology clinicians to develop interventions about introducing palliative care. The procedures and results of this project will be posted online so that other institutions can use them as a model for developing similar interventions appropriate for their needs.

Changing practice: red blood cell typing by molecular methods for patients with sickle cell disease.

BACKGROUND: Extended red blood cell (RBC) antigen matching is recommended to limit alloimmunization in patients with sickle cell disease (SCD). DNA-based testing to predict blood group phenotypes has enhanced availability of antigen-negative donor units and improved typing of transfused patients, but replacement of routine serologic typing for non-ABO antigens with molecular typing for patients has not been reported. STUDY DESIGNS AND METHODS: This study compared the historical RBC antigen phenotypes obtained by hemagglutination methods with genotype predictions in 494 patients with SCD. For discrepant results, repeat serologic testing was performed and/or investigated by gene sequencing for silent or variant alleles. RESULTS: Seventy-one typing discrepancies were identified among 6360 antigen comparisons (1.1%). New specimens for repeat serologic testing were obtained for 66 discrepancies and retyping agreed with the genotype in 64 cases. One repeat Jk(b-) serologic phenotype, predicted Jk(b+) by genotype, was found by direct sequencing of JK to be a silenced allele, and one N typing discrepancy remains under investigation. Fifteen false-negative serologic results were associated with alleles encoding weak antigens or single-dose Fy(b) expression. CONCLUSIONS: DNA-based RBC typing provided improved accuracy and expanded information on RBC antigens compared to hemagglutination methods, leading to its implementation as the primary method for extended RBC typing for patients with SCD at our institution.

Neural tube defects in Latin America and the impact of fortification: a literature review.

OBJECTIVE: Data on the prevalence of birth defects and neural tube defects (NTD) in Latin America are limited. The present review summarizes NTD prevalence and time trends in Latin American countries and compares pre- and post-fortification periods to assess the impact of folic acid fortification in these countries. DESIGN: We carried out a literature review of studies and institutional reports published between 1990 and 2010 that contained information on NTD prevalence in Latin America. RESULTS: NTD prevalence in Latin American countries varied from 0·2 to 9·6 per 1000 live births and was influenced by methods of ascertainment. Time trends from Bogota, Costa Rica, Dominican Republic, Guatemala City, Mexico and Puerto Rico showed average annual declines of 2·5 % to 21·8 %. Pre- and post-fortification comparisons were available for Argentina, Brazil, Chile, Costa Rica, Puerto Rico and Mexico. The aggregate percentage decline in NTD prevalence ranged from 33 % to 59 %. CONCLUSIONS: The present publication is the first to review data on time trends and the impact of folic acid fortification on NTD prevalence in Latin America. Reported NTD prevalence varied markedly by geographic region and in some areas of Latin America was among the lowest in the world, while in other areas it was among the highest. For countries with available information, time trends showed significant declines in NTD prevalence and these declines were greater in countries where folic acid fortification of staples reached the majority of the population at risk, such as Chile and Costa Rica.

Quality of Outpatient Pediatric Palliative Care Telehealth: A Retrospective Chart Review.

CONTEXT: Studies suggest the feasibility and acceptability of telehealth in outpatient pediatric palliative care. However, there is a need for data that describes the implementation and quality of telehealth, relying on objective and validated measures. OBJECTIVE: We sought to compare the provision of pediatric palliative care by delivery method. METHODS: We conducted a retrospective electronic health record review of patients seen by our outpatient palliative care team over a two-year period. Demographic, diagnostic, and health utilization data as well as encounter characteristics were compared between patients seen in person (IP), through telehealth (TH), and both (IP/TH). RESULTS: Three hundred ninety-four patients were evaluated with 889 outpatient pediatric palliative care encounters. Non-English speaking patients were less likely to receive palliative care through TH, as were patients without active patient portals. Median follow-up time was longer for patients seen through TH or IP/TH. Patients with malignancies were seen more frequently IP while children with neurologic diagnoses, technology dependence, and a higher number of complex chronic conditions were seen more frequently via TH. Health outcomes, end of life quality metrics, and encounter-level quality indicators were similar across care delivery methods. Review of systems, pain, and mood management, and advance care planning happened more frequently IP while goals of care discussions and medical decision-making happened more through TH. CONCLUSION: Despite differences in patients seen and palliative interventions provided in person compared to telehealth, health outcomes, and quality indicators were similar across care delivery methods. These data support the continued practice of telehealth in palliative care and highlight the need for equity in its evolution.

Provider Perceptions for Withdrawing Life Sustaining Therapies at a Large Pediatric Hospital.

CONTEXT: More than 74% of pediatric deaths occur in an intensive care unit (ICU), with 40% occurring after withdrawal of life-sustaining therapies (WOLST). No needs assessment has described provider needs or suggestions for improving the WOLST process in pediatrics. OBJECTIVES: This study aims to describe interdisciplinary provider self-reported confidence, needs, and suggestions for improving the WOLST process. METHODS: A convergent parallel mixed-methods design was used. An online survey was distributed to providers involved in WOLSTs in a quaternary children's hospital between January and December 2018. The survey assessed providers' self-reported confidence in their role, in providing guidance to families about the WOLST, experiences with the WOLST process, areas for improvement, and symptom management. Kruskal-Wallis testing was used for quantitative data analysis with P values <0.05 considered significant. Analysis was performed with SPSS v27. Qualitative data were thematically analyzed using Atlas.ti.8 and NVivo. RESULTS: A total of 297 surveys were received (48% survey completion) that consisted of multiple choice, Likert-type, and yes/no questions with options for open-ended responses. Mean provider self-rated confidence was high and varied significantly between disciplines. Qualitative analysis identified four areas for refining communication: 1) between the primary team and family, 2) within the primary team, 3) between the primary team and consulting providers, and 4) logistical challenges. CONCLUSIONS: While participants' self-rated confidence was high, it varied between disciplines. Participants identified opportunities for improved communication and planning before a WOLST. Future work includes development and implementation of a best practice guideline to address gaps and standardize care delivery.

Delphi Method to Develop a Palliative Care Tool for Children and Families in Sub-Saharan Africa.

CONTEXT: In sub-Saharan Africa, there is no standardized approach to pediatric palliative care assessment. Because of this, there is a critical demand for evidence-based assessment tools that identify the specialized needs of children and their families requiring palliative care in developing countries. OBJECTIVES: To develop a standardized approach to pediatric palliative care (PPC) assessment that includes an individualized plan of care for use in sub-Saharan Africa. METHODS: A Delphi method approach used five rounds to explore core elements that define the essential assessment attributes mandatory for providing excellence in PPC. Using the Delphi method, the consensus from 11 PPC experts was obtained during four Delphi rounds regarding the most important questions to include in a PPC assessment tool and plan of care. During the final Delphi round 5, the expert consensus was confirmed in a separate group of 36 childhood cancer/palliative care clinical providers. RESULTS: Five core elements were developed as the foundation for a PPC assessment. A symptom assessment tool was developed that includes 15 symptoms that PPC experts agreed occurred more than 65% of the time in their patients. CONCLUSION: The Delphi method was an effective tool to develop a consensus on a PPC assessment tool to use with children and their families in sub-Saharan Africa. This standardized approach will enable the collection of data to drive outcomes and research.

Palliative Care Services within a Pediatric Hematology-Oncology Program in a Low-Resource Setting.

CONTEXT: Outcomes for children with cancer in sub-Saharan Africa (SAA) are dismal due to delayed diagnosis and limited access to curative therapy. When establishing a pediatric hematology-oncology (PHO) program in low-resource settings, early integration of palliative care services becomes essential. While palliative care is a human right, equitable distribution is lacking. OBJECTIVES: We aim to describe our experience establishing a palliative care program, the services offered, and the distribution of patients served. METHODS: This is a brief description of our PHO palliative care program in Lilongwe, Malawi at a tertiary care center and a three-year retrospective review of activities (2017-2020). Services offered include inpatient, outpatient, home visits, end of life care, and strengthening of referral systems. RESULTS: Over the three-year period, 315 patients were enrolled. 57% (n=179) were male. The median age was seven years (5 months-22 years). Patients served were from 17 of 28 districts within Malawi. Diagnoses of patients included 43% solid tumors (n=135), 22% lymphoma (n=68), 15% leukemia (n=47), and 21% hematologic disease (n=65). 40% of patients have died (n=125), with 53% of deaths occurring at home (n=66), 22% in the hospital (n=28), and 25% at unknown locations (n=31). CONCLUSION: Palliative care is a critical component of PHO programs worldwide. Programs must leverage existing networks to ensure optimal care to children and families. We demonstrate the feasibility of integrating palliative care services within a PHO program in a low-resource setting, which could serve as a model for other countries in SSA.

Quality Indicators in Pediatric Palliative Care: Considerations for Latin America.

Pediatric palliative care is a growing field in which the currently available resources are still insufficient to meet the palliative care needs of children worldwide. Specifically, in Latin America, pediatric palliative care services have emerged unevenly and are still considered underdeveloped when compared to other regions of the world. A crucial step in developing pediatric palliative care (PPC) programs is delineating quality indicators; however, no consensus has been reached on the outcomes or how to measure the impact of PPC. Additionally, Latin America has unique sociocultural characteristics that impact the perception, acceptance, enrollment and implementation of palliative care services. To date, no defined set of quality indicators has been proposed for the region. This article explores the limitations of current available quality indicators and describes the Latin American context and how it affects PPC development. This information can help guide the creation of standards of care and quality indicators that meet local PPC needs while considering the sociocultural landscape of Latin America and its population.

Using Quality Improvement Science to Create a Navigator in the Electronic Health Record for the Consolidation of Patient Information Surrounding Pediatric End-of-Life Care.

BACKGROUND: It is important to document the domains surrounding end-of-life (EOL) care in the electronic health record (EHR). No pediatric navigator exists for these purposes. MEASURES: Medical charts were reviewed for documentation surrounding code status and care at the time of death from January 2017 to June 2019. INTERVENTION: Creation of a navigator in the EHR to consolidate advance care planning documents, code status orders and notes and EOL flowsheets. OUTCOMES: After implementing the navigator, 96% code status changes had supporting documentation, an increase of 35%. The percentage of deaths supported by a psychosocial team (social worker, chaplain and certified child life specialist) increased by 25%. Post-mortem documentation became electronic. Patient level metrics began to be electronically collected. CONCLUSIONS/LESSONS LEARNED: Little has been published regarding use of the EHR to consolidate EOL documentation in pediatrics. Development of a systematic approach to documentation is critical to providing EOL care and standardizing care delivered.

Mutation-specific signaling profiles and kinase inhibitor sensitivities of juvenile myelomonocytic leukemia revealed by induced pluripotent stem cells.

Juvenile myelomonocytic leukemia (JMML) is an uncommon myeloproliferative neoplasm driven by Ras pathway mutations and hyperactive Ras/MAPK signaling. Outcomes for many children with JMML remain dismal with current standard-of-care cytoreductive chemotherapy and hematopoietic stem cell transplantation. We used patient-derived induced pluripotent stem cells (iPSCs) to characterize the signaling profiles and potential therapeutic vulnerabilities of PTPN11-mutant and CBL-mutant JMML. We assessed whether MEK, JAK, and PI3K/mTOR kinase inhibitors (i) could inhibit myeloproliferation and aberrant signaling in iPSC-derived hematopoietic progenitors with PTPN11 E76K or CBL Y371H mutations. We detected constitutive Ras/MAPK and PI3K/mTOR signaling in PTPN11 and CBL iPSC-derived myeloid cells. Activated signaling and growth of PTPN11 iPSCs were preferentially inhibited in vitro by the MEKi PD0325901 and trametinib. Conversely, JAK/STAT signaling was selectively activated in CBL iPSCs and abrogated by the JAKi momelotinib and ruxolitinib. The PI3Kδi idelalisib and mTORi rapamycin inhibited signaling and myeloproliferation in both PTPN11 and CBL iPSCs. These findings demonstrate differential sensitivity of PTPN11 iPSCs to MEKi and of CBL iPSCs to JAKi, but similar sensitivity to PI3Ki and mTORi. Clinical investigation of mutation-specific kinase inhibitor therapies in children with JMML may be warranted.