Hemolytic Uremic Syndrome Research Articles

A-153 Study of the evaluation of fragment flag and percentage schistocyte count in MINDRAY® BC-6800 PLUS in a public pediatric hospital in Argentina

Abstract Background Schistocytes are fragmented red blood cells (FRCs) that arise from the mechanical destruction of red blood cells (RBCs). They indicate the possible presence of a thrombotic microangiopathy (TMA). TMA includes hemolytic uremic syndrome (HUS) and thrombotic thrombocytopenic purpura(TTP). ICSH defined schistocytes as 4 well stablish shapes always smaller than intact RBCs. The gold standard for the identification of schistocytes is microscopic evaluation. A schistocyte count should be considered clinically meaningful if it represent the main morphological RBCs abnormality in the periferic blood smear (PBS). The results should be expressed as a percentage, after counting at least 1000 RBCs in optimal areas of the PBS. The cut off for TMA was previously stablished in above 1% (adult population) and a higher percentaje in pre term newborns (above 5%). Some blood cell count analyzers have a flag for FRC, and some of them also provide methods for an automated FRC count, through the measurement of forward scatter and the intensity of fluorescence or 2-dimensional optical analysis. The Mindray® BC 6800 PLUS provides a flag for fragments and the research FRC% parameter that quantifies fragmented RBCs. When the flag and the FRC% are combined, can bring value to diagnostic TMA. Objetives Evaluate the sensitivity and specificity of the fragment flag to predict schistocyte on the PBS of pediatric samples. Evaluate the utility of FRC% as TMA diagnostic tool and compare with the gold standard. Methods 725 pediatric samples were analyzed (0-18 years).16 of them was patient samples diagnosed with TMA. All blood cell counts were determined using Mindray®BC-6800 PLUS. Cut off 1% was considered for FRC% in TMA. Inclusion criteria for TMA samples include thrombocytopenia, increased lactate dehydrogenase and decreased hemoglobin concentration. Schistocyte counting was carried out from May-Grunwald-Giemsa-stained PBS according to the recommendations proposed by ICSH. Only ICSH established shapes were kept as valid. Results Regarding the sensitivity of the fragment flag, it was 84.62% with a specificity of 91.13% to detect the presence of schistocytes in PBS samples. In the Bland-Altman analysis, the FRC% shows a difference with a tendency to underestimation with respect to the observation on the PBS, which was less at lower percentages of schistocytes. The sensitivity of the FRC% to TMA was 75% while the specificity was 94.36% and a negative predictive value (NPV) of 99.41%. Conclusions Fragments flag represents a potentially powerful tool for screening of squistocytes in pediatric samples. Although sensibility and NPV for FRC% authomatical counts were acceptable, a few false-negative samples were found. Presence of FRC% below a threshold of 1% could exclude TMA.However PBS microscopic observation is still the gold standard to determine squistocytes presence. For the purpose of complete our study and to get further larger scale prospective data, we started collecting all data for all samples entering the laboratory in order to improve an authomatical method that should have a more rapid turn-around time at lower costs.

The current diagnosis and treatment situation of rare disease in the pediatric intensive care unit

Objective: To analyze the diagnosis and treatment of children with rare diseases in the pediatric intensive care unit (PICU), the distribution of disease types and populations, clinical characteristics, and the use of orphan drugs. Methods: A retrospective case summary was conducted. Data were collected from 105 children aged 29 days to <18 years with a confirmed diagnosis of rare diseases according to the "First Batch of Rare Disease Catalogue in China" who were admitted to the PICU of Beijing Children's Hospital, Capital Medical University from January 2020 to December 2022. Data including general information, auxiliary examinations, and treatment details for each patient were collected from the hospital's electronic medical record system. Patients were divided into age groups: infancy (29 days to<1 year), early childhood (1 to <3 years), preschool age (3 to<7 years), school age (7 to<13 years), and adolescence (13 to<18 years). The chi-square test was used to compare gender distribution differences among various rare diseases. Results: A total of 105 patients with 130 cases meeting the diagnostic criteria were included, accounting for 4.7% (130/2 754) of the total admissions to the PICU. The age at PICU admission was 5.3 (0.8, 9.5) years and there were 81 cases in male. The 3 most common types of diseases were endocrine, nutritional, and metabolic diseases (37 cases); followed by neurological disorders(32 cases); and congenital malformations, deformities, and chromosomal abnormalities(17 cases). The 5 most common rare diseases were methylmalonic acidemia (14 cases), mitochondrial encephalomyopathy (14 cases), atypical hemolytic uremic syndrome (12 cases), autoimmune encephalitis (12 cases), and idiopathic cardiomyopathy (9 cases). The distributions of common rare diseases varied among different age groups. In infants, atypical hemolytic uremic syndrome was most common (6 children). There was no statistically significant difference regarding gender among children with mitochondrial encephalomyopathy (13.6% (11/81) vs. 6.1% (3/49), χ2=1.77, P=0.184). Respiratory failure (36 cases) was the primary reason for rare diseases children to be admitted to the PICU. A total of 95 cases underwent mechanical ventilation, 39 cases received multidisciplinary collaborative diagnosis and treatment, and only 6 children received orphan drug therapy during their stay in the PICU. Conclusions: Rare diseases are not uncommon in PICU. Endocrine, nutritional and metabolic disorders, neurological disorders, congenital malformations, deformities, and chromosomal abnormalities were common. Methylmalonic acidemia, mitochondrial encephalomyopathy, atypical hemolytic uremic syndrome and autoimmune encephalitis have higher cases. Many children with rare diseases in the PICU have complex conditions those are challenging to treat, requiring multidisciplinary collaboration. The utilization rate of orphan drugs among children with rare diseases in PICU needs to be improved.

Recurrence of Atypical Hemolytic Uremic Syndrome (HUS) after Kidney Transplantation in Patients with the Complement C3 p.Arg161Trp Variant

Recurrence of Atypical Hemolytic Uremic Syndrome (HUS) after Kidney Transplantation in Patients with the Complement C3 p.Arg161Trp Variant

Evaluation of Ravulizumab Trough Levels in Pediatric Atypical Hemolytic Uremic Syndrome at Remission

Evaluation of Ravulizumab Trough Levels in Pediatric Atypical Hemolytic Uremic Syndrome at Remission

A Case of Thrombotic Thrombocytopenic PurpuraInitially Suspected as Shiga Toxin-Producing Escherichia coli Hemolytic Uremic Syndrome Due to the Preceding Symptoms of Hemolytic Anemia with Vomiting, Hematochezia, and AKI

A Case of Thrombotic Thrombocytopenic PurpuraInitially Suspected as Shiga Toxin-Producing Escherichia coli Hemolytic Uremic Syndrome Due to the Preceding Symptoms of Hemolytic Anemia with Vomiting, Hematochezia, and AKI

S2988 A Rare Case of Postoperative Atypical Hemolytic Uremic Syndrome Following Partial Colectomy

S2988 A Rare Case of Postoperative Atypical Hemolytic Uremic Syndrome Following Partial Colectomy

S4740 A Case of Small and Large Bowel Ischemic Ulcerations in a Patient With Atypical Hemolytic Uremic Syndrome

S4740 A Case of Small and Large Bowel Ischemic Ulcerations in a Patient With Atypical Hemolytic Uremic Syndrome

Energy Score Analysis in Immune Cells Linked to Atypical Hemolytic Uremic Syndrome Using Single-Cell Sequencing

Energy Score Analysis in Immune Cells Linked to Atypical Hemolytic Uremic Syndrome Using Single-Cell Sequencing

Safety and Effectiveness of Switching to Ravulizumab from Eculizumab in Kidney Transplant Recipients with Atypical Hemolytic Uremic Syndrome: A Global Registry Analysis

Safety and Effectiveness of Switching to Ravulizumab from Eculizumab in Kidney Transplant Recipients with Atypical Hemolytic Uremic Syndrome: A Global Registry Analysis

Atypical Even for Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical Even for Atypical Hemolytic Uremic Syndrome (aHUS)

A Case of Atypical Hemolytic Uremic Syndrome with a Complement Factor I Mutation Triggered by a Femoral Neck Fracture in an Elderly Japanese Patient

A Case of Atypical Hemolytic Uremic Syndrome with a Complement Factor I Mutation Triggered by a Femoral Neck Fracture in an Elderly Japanese Patient

Atypical Hemolytic Uremic Syndrome with Peripheral Gangrene

Atypical Hemolytic Uremic Syndrome with Peripheral Gangrene

Complete Recovery from AKI: A Rare Case of Postpartum Atypical Hemolytic Uremic Syndrome

Complete Recovery from AKI: A Rare Case of Postpartum Atypical Hemolytic Uremic Syndrome

Incidence and Severity of Extra-Kidney Manifestations and Outcomes in Pediatric Hemolytic Uremic Syndrome: A Retrospective Cohort Study

Incidence and Severity of Extra-Kidney Manifestations and Outcomes in Pediatric Hemolytic Uremic Syndrome: A Retrospective Cohort Study

Epidemiological and Clinical Characteristics of Atypical Hemolytic Uremic Syndrome in China

Epidemiological and Clinical Characteristics of Atypical Hemolytic Uremic Syndrome in China

Azithromycin for the Prevention of Hemolytic Uremic Syndrome in Shiga Toxin-Positive Diarrhea: A Proof of Concept

Azithromycin for the Prevention of Hemolytic Uremic Syndrome in Shiga Toxin-Positive Diarrhea: A Proof of Concept

Late Relapse of Atypical Hemolytic Uremic Syndrome 36 Months after Discontinuation of Complement Blockade Therapy

Late Relapse of Atypical Hemolytic Uremic Syndrome 36 Months after Discontinuation of Complement Blockade Therapy

A Unique and Successful Story of Atypical Hemolytic Uremic Syndrome (aHUS)

A Unique and Successful Story of Atypical Hemolytic Uremic Syndrome (aHUS)

Influenza B Triggering Atypical Hemolytic Uremic Syndrome (aHUS) in a Patient with Cobalamin C (CblC) Disease Carrier State and a Complement Factor H (CFH) Mutation

Influenza B Triggering Atypical Hemolytic Uremic Syndrome (aHUS) in a Patient with Cobalamin C (CblC) Disease Carrier State and a Complement Factor H (CFH) Mutation

Real-World Effectiveness of Ravulizumab among C5 Inhibitor-Naive Patients with Atypical Hemolytic Uremic Syndrome (aHUS): A Physician Panel-Based Chart Review Study (aHUS IMPACT)

Real-World Effectiveness of Ravulizumab among C5 Inhibitor-Naive Patients with Atypical Hemolytic Uremic Syndrome (aHUS): A Physician Panel-Based Chart Review Study (aHUS IMPACT)