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ObjectivesCritically 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.DesignSystematic 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]).SettingNot applicable.PatientsChildren admitted to a PICU at risk of bleeding and receipt of plasma and/or platelet transfusions.InterventionsNone.Measurements and main resultsA panel of 29 experts in methodology, transfusion, and implementation science from five countries and nine pediatric subspecialties completed a systematic review and participated in a virtual consensus conference series to develop recommendations. The search included MEDLINE, EMBASE, and Cochrane Library databases, from inception to December 2020, using a combination of subject heading terms and text words for concepts of plasma and platelet transfusion in critically ill children. Four graded recommendations and 49 consensus expert statements were developed using modified Research and Development/UCLA and Grading of Recommendations, Assessment, Development, and Evaluation methodology. We focused on eight subpopulations of critical illness (1, severe trauma, intracranial hemorrhage, or traumatic brain injury; 2, cardiopulmonary bypass surgery; 3, extracorporeal membrane oxygenation; 4, oncologic diagnosis or hematopoietic stem cell transplantation; 5, acute liver failure or liver transplantation; 6, noncardiac surgery; 7, invasive procedures outside the operating room; 8, sepsis and/or disseminated intravascular coagulation) as well as laboratory assays and selection/processing of plasma and platelet components. In total, we came to consensus on four recommendations, five good practice statements, and 44 consensus-based statements. These results were further developed into consensus-based clinical decision trees for plasma and platelet transfusion in critically ill pediatric patients.ConclusionsThe TAXI-CAB program provides expert-based consensus for pediatric intensivists for the administration of plasma and/or platelet transfusions in critically ill pediatric patients. There is a pressing need for primary research to provide more evidence to guide practitioners.

ABBREVIATION. DPT, Demerol/Phenergan/Thorazine cocktail. Patients and parents find lumbar puncture and bone marrow examinations uncomfortable and anxiety-producing. In addition, many radiologic studies and radiotherapy treatments require young patients to be [...]

Juvenile chronic myelogenous leukemia accounts for about two percent of all juvenile leukemia, with 90% of all cases being reported in children under the age of four years. The disease is characterized by abdominal discomfort, hemorrhagic complications, and in many cases a rash and enlarged liver. Blood tests usually reveal disintegration of white blood cells, variable red blood cell precursors and an abnormally low number of platelets. A newborn baby who appeared otherwise healthy showed signs of abdominal distention and small red spots, and the oxygen saturation in his blood began to drop. An elevated white blood cell count and other serum level measurements led to a diagnosis of juvenile chronic myelogenous leukemia. The infant received a bone marrow transplant and subsequent blood transfusion from his sister in an effort to reduce his white blood cell count and associated complications.

Gaucher's disease is a lysosomal storage disorder. Normally, the tiny lysosomes within a cell serve to degrade and recycle various cellular components as well as some materials imported from outside the cell. In Gaucher's disease, the lysosomes are deficient in a particular enzyme called glucocerebrosidase. The consequences of this enzyme deficiency are seen mainly in the lysosomes of macrophages, the large blood cells that digest both bacteria and normal cellular debris. Since the enzyme is lacking, a very special type of fat called a sphingolipid can not be broken down and it accumulates within the lysosome. Ultimately, many organs of the body become affected, and the normal cells of the bone marrow are progressively displaced by macrophages with excess fat they can do nothing with. Most patients develop anemia, enlargement of the internal organs, and skeletal abnormalities. A few patients develop neurologic abnormalities as well. One approach to treating such a disease would be to give the patient large amounts of the enzyme glucocerebrosidase. This has been attempted with variable results. However, the fact that the major defect is in the lysosomes of the macrophages allows for a therapeutic approach not possible in other genetic disorders. If the enzyme could be altered in such a way that the macrophages would eat it, as they do many foreign particles, then the macrophages themselves would place the enzyme exactly where it belongs. This approach was tried in the treatment of 12 patients with Gaucher's disease. The patients were treated with the chemically modified enzyme to enhance the likelihood its accumulation within macrophages. After three to four months of twice-weekly infusions of the enzyme, all 12 patients experienced an improvement in their anemia. The platelet count, an indicator of normal blood clotting, increased in seven patients. During the course of the study, many of the observed symptoms were decreased, including bone pain, easily bruising, and chronic fatigue. The long-term treatment with the enzyme was well tolerated by the patients. (Consumer Summary produced by Reliance Medical Information, Inc.)