Community-Based Pediatric Palliative Care: How Services Support Children's and Families' Quality of Life.

The Massachusetts Department of Public Health's Pediatric Palliative Care Network (PPCN) provides Community-Based Pediatric Palliative Care (CBPPC) to children with life-limiting conditions and their families. CBPPC services aim to improve children and families' quality of life (QOL). To identify perceived domains of QOL important for children and families and to understand whether and how CBPPC supports QOL.

AAV gene therapy for Tay-Sachs disease.

Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA). Here, we describe an adeno-associated virus (AAV) gene therapy expanded-access trial in two patients with infantile TSD (IND 18225) with safety as the primary endpoint and no secondary endpoints. Patient TSD-001 was treated at 30 months with an equimolar mix of AAVrh8-HEXA and AAVrh8-HEXB administered intrathecally (i.

Executive Summary of Recommendations and Expert Consensus for Plasma and Platelet Transfusion Practice in Critically Ill Children: From the Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding (TAXI-CAB).

Objectives: Critically ill children frequently receive plasma and platelet transfusions. We sought to determine evidence-based recommendations, and when evidence was insufficient, we developed expert-based consensus statements about decision-making for plasma and platelet transfusions in critically ill pediatric patients.

Design: Systematic review and consensus conference series involving multidisciplinary international experts in hemostasis, and plasma/platelet transfusion in critically ill infants and children (Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding [TAXI-CAB]).

What Laboratory Tests and Physiologic Triggers Should Guide the Decision to Administer a Platelet or Plasma Transfusion in Critically Ill Children and What Product Attributes Are Optimal to Guide Specific Product Selection? From the Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding.

Objectives: To present consensus statements and supporting literature for plasma and platelet product variables and related laboratory testing for transfusions in general critically ill children from the Transfusion and Anemia EXpertise Initiative-Control/Avoidance of Bleeding.

Design: Systematic review and consensus conference of international, multidisciplinary experts in platelet and plasma transfusion management of critically ill children.

Setting: Not applicable.

Pediatric ECMO Candidates at Non-ECMO Centers: Transfer, Cannulate, or Treat Locally?

Pediatric inpatient and intensive care specialists working outside of tertiary medical centers confront difficult clinical scenarios related to how best to care for extremely ill children who may or may not benefit from advanced medical technology, and these clinicians are often faced with limited local availability. Extracorporeal membrane oxygenation (ECMO) is a technology that is only available at a subset of tertiary care centers, and the decision to risk the transfer of a child for the potential benefit of ECMO is challenging. This article is aimed at addressing the main factors and ethical principles related to this decision-making: (1) whether ECMO is the standard of care, (2) clinical decision analysis of the risks and benefits, (3) informed consent and education of the parents and/or guardians, and (4) institutional leadership decision-making.

Consensus Recommendations for RBC Transfusion Practice in Critically Ill Children From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To date, there are no published guidelines to direct RBC transfusion decision-making specifically for critically ill children. We present the recommendations from the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of multidisciplinary, international experts in RBC transfusion management of critically ill children.

Implementation of the Recommendations for RBC Transfusions for Critically Ill Children From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To provide context for the implementation of the Pediatric Critical Care Transfusion and Anemia Expertise Initiative recommendations for RBC transfusions including a review of prior research related to implementation of transfusion guidelines, efforts to facilitate implementation through Transfusion and Anemia Expertise Initiative, and to provide a framework for recommendation implementation.

Design: Review of existing clinical literature and description of a comprehensive approach to implementation based on Implementation Science principles.

Results: The Transfusion and Anemia Expertise Initiative recommendations on RBC transfusions are based on clinical evidence and aim to limit unnecessary and potentially harmful transfusions.

Recommendations on Selection and Processing of RBC Components for Pediatric Patients From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present the recommendations and supporting literature for selection and processing of RBC products in critically ill children developed by the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of international, multidisciplinary experts in RBC transfusion management of critically ill children METHODS:: The panel of 38 experts developed evidence-based, and when evidence was lacking, expert-based clinical recommendations as well as research priorities for RBC transfusions in critically ill children. The RBC processing subgroup included five experts.

Recommendations on the Indications for RBC Transfusion for the Critically Ill Child Receiving Support From Extracorporeal Membrane Oxygenation, Ventricular Assist, and Renal Replacement Therapy Devices From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present recommendations and supporting literature for RBC transfusions in critically ill children supported with extracorporeal membrane oxygenation, ventricular assist devices, or renal replacement therapy.

Design: Consensus conference series of international, multidisciplinary experts in RBC transfusion management of critically ill children.

Methods: The panel of 38 experts developed evidence-based, and when evidence was lacking, expert-based clinical recommendations as well as research priorities for RBC transfusions in critically ill children.

Recommendations on RBC Transfusion Support in Children With Hematologic and Oncologic Diagnoses From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present the recommendations and supporting evidence for RBC transfusions in critically ill children with hematologic and oncologic disease from the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of international, multidisciplinary experts in RBC transfusion management of critically ill children.

Methods: The panel of 38 experts developed evidence-based and, when evidence was lacking, expert-based clinical recommendations and research priorities for RBC transfusions in critically ill children.

Recommendations on RBC Transfusion in Infants and Children With Acquired and Congenital Heart Disease From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present the recommendations and supporting literature for RBC transfusions in critically ill children with acquired and congenital heart disease developed by the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of 38 international, multidisciplinary experts in RBC transfusion management of critically ill children.

Methods: Experts developed evidence-based and, when evidence was lacking, expert-based clinical recommendations and research priorities for RBC transfusions in critically ill children.

Recommendations on RBC Transfusion in Critically Ill Children With Nonlife-Threatening Bleeding or Hemorrhagic Shock From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present the recommendations and supporting literature for RBC transfusions in critically ill children with bleeding developed by the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of international, multidisciplinary experts in RBC transfusion management of critically ill children.

Methods: The panel of 38 experts developed evidence-based and, when evidence was lacking, expert-based clinical recommendations as well as research priorities for RBC transfusions in critically ill children.

Recommendations on RBC Transfusions for Critically Ill Children With Nonhemorrhagic Shock From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present the recommendations and supporting literature for RBC transfusions in critically ill children with nonhemorrhagic shock developed by the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of international, multidisciplinary experts in RBC transfusion management of critically ill children.

Methods: The panel of 38 experts developed evidence-based, and when evidence was lacking, expert-based clinical recommendations as well as research priorities for RBC transfusions in critically ill children.

Recommendations on RBC Transfusions in Critically Ill Children With Acute Respiratory Failure From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present the recommendations and supporting literature for RBC transfusions in critically ill children with bleeding developed by the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of international, multidisciplinary experts in RBC transfusion management of critically ill children.

Methods: The panel of 38 experts developed evidence-based and, when evidence was lacking, expert-based clinical recommendations as well as research priorities for RBC transfusions in critically ill children.

Recommendations on RBC Transfusion in General Critically Ill Children Based on Hemoglobin and/or Physiologic Thresholds From the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Objectives: To present the consensus recommendations and supporting literature for RBC transfusions in general critically ill children from the Pediatric Critical Care Transfusion and Anemia Expertise Initiative.

Design: Consensus conference series of international, multidisciplinary experts in RBC transfusion management of critically ill children.

Methods: The panel of 38 experts developed evidence-based and, when evidence was lacking, expert-based recommendations and research priorities regarding RBC transfusions in critically ill children.

The Pediatric Critical Care Transfusion and Anemia Expertise Initiative Consensus Conference Methodology.

Objectives: This article describes the methodology used for Pediatric Critical Care Transfusion and Anemia Expertise Initiative Consensus Conference.

Design: Consensus conference of international experts in pediatric critical care and transfusion medicine, following standards set by the Institute of Medicine, using the Research and Development/UCLA Appropriateness Method, modeled after the Pediatric Acute Lung Injury Consensus Conference. Topics related to RBC transfusion in children with or at risk for critical illness were divided into nine subgroups with a systematic review of the literature.

Inpatient management of children with recessive dystrophic epidermolysis bullosa: A review.

Recessive dystrophic epidermolysis bullosa is a disorder marked by skin and mucosal blistering after minimal trauma. Even the most routine procedures in the hospital, if done incorrectly, can precipitate extensive skin loss, pain, and scarring. Most providers have little experience working with patients with this degree of skin fragility.

Determinants of red blood cell transfusion in pediatric trauma patients admitted to the intensive care unit.

Background: There are no well-designed prospective studies evaluating transfusion practices in pediatric trauma. We sought to describe red blood cell (RBC) transfusion practices in trauma patients who were admitted to a pediatric intensive care unit (PICU).

Study Design And Methods: This study is a post-hoc analysis of a prospective, 6-month observational study in 30 PICUs.

Early High-Frequency Oscillatory Ventilation in Pediatric Acute Respiratory Failure. A Propensity Score Analysis.

Rationale: The use of high-frequency oscillatory ventilation (HFOV) for acute respiratory failure in children is prevalent despite the lack of efficacy data.

Objectives: To compare the outcomes of patients with acute respiratory failure managed with HFOV within 24-48 hours of endotracheal intubation with those receiving conventional mechanical ventilation (CMV) and/or late HFOV.

Methods: This is a secondary analysis of data from the RESTORE (Randomized Evaluation of Sedation Titration for Respiratory Failure) study, a prospective cluster randomized clinical trial conducted between 2009 and 2013 in 31 U.

The wrap-up: a unique forum to support pediatric residents when faced with the death of a child.

Objective: The project intended to describe the format of the Wrap-up, a unique multidisciplinary guided debriefing following a child's death. Specific feedback from pediatric residents was sought to assess the model.

Methods: The Wrap-ups were timely (within 48 hours of a death), consistent (conducted after each pediatric intensive care unit (PICU) death), multidisciplinary (all care providers were invited), and specifically conducted by someone trained in postdeath facilitation.

Isoflurane for life-threatening bronchospasm: a 15-year single-center experience.

Background: Children with severe bronchospasm requiring mechanical ventilation may become refractory to conventional therapy. In these critically ill patients, isoflurane is an inhaled anesthetic agent available in some centers to treat bronchospasm. We hypothesized that isoflurane is safe and would lead to improved gas exchange in children with life-threatening bronchospasm refractory to conventional therapy.