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Smitha Hosahalli Vasanna, Maria A Pereda, Jignesh Dalal Department of Pediatrics, Division of Pediatric Hematology Oncology, Rainbow Babies and Children’s Hospital, University Hospitals, Cleveland, OH, USACorrespondence: Jignesh DalalDepartment of Pediatrics, Division of Pediatric Hematology Oncology, Rainbow Babies and Children’s Hospital, University Hospitals, 11100 Euclid Ave, Cleveland, OH, 44106, USATel +1 2169831027Fax +1 2168445431Email jignesh.dalal@uhhospitals.orgAbstract: Wiskott–Aldrich syndrome (WAS) is a rare X-linked recessive inborn error of immunity (IEI) first described in 1937. Classic WAS is characterized by the triad of thrombocytopenia with small platelets, recurrent infections due to combined immunodeficiency, and eczema. Hematopoietic stem cell transplantation (HSCT) was the only curative option available for five decades, with excellent outcomes reported for matched sibling donors (MSD) and matched unrelated donors (MUD). More recently, alternative donor transplants such as umbilical cord blood (UCB) and haploidentical transplant have emerged as viable options due to improvements in better graft selection, cell dosing, and effective allograft manipulation measures. Gene therapy is another potential curative option with promising results, yet currently is offered only as part of a clinical trial.Keywords: Wiskott–Aldrich syndrome, X-linked thrombocytopenia, supportive care, hematopoietic stem cell transplantation, gene therapy

Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with relapsed or refractory SAA., (© 2024 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.)

Severe aplastic anemia (SAA) is a rare potentially fatal hematologic disorder. Although overall outcomes with treatment are excellent, there are variations in management approach, including differences in treatment between adult and pediatric patients. Certain aspects of treatment are under active investigation in clinical trials. Because of the rarity of the disease, some pediatric hematologists may have relatively limited experience with the complex management of SAA. The following recommendations reflect an up-to-date evidence-based approach to the treatment of children with newly diagnosed SAA., (© 2024 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.)

Competing Interests: The authors declare they have no conflicts of interest.

Objectives: To determine the association between mean airway pressure and 90-day mortality in patients with acute respiratory failure requiring mechanical ventilation and to compare the predictive ability of mean airway pressure compared with inspiratory plateau pressure and driving pressure., Design: Prospective observational cohort., Setting: Five ICUs in Lima, Peru., Subjects: Adults requiring invasive mechanical ventilation via endotracheal tube for acute respiratory failure., Interventions: None., Measurements and Main Results: Of potentially eligible participants (n = 1,500), 65 (4%) were missing baseline mean airway pressure, while 352 (23.5%) were missing baseline plateau pressure and driving pressure. Ultimately, 1,429 participants were included in the analysis with an average age of 59 ± 19 years, 45% female, and a mean PaO2/FIO2 ratio of 248 ± 147 mm Hg at baseline. Overall, 90-day mortality was 50.4%. Median baseline mean airway pressure was 13 cm H2O (interquartile range, 10-16 cm H2O) in participants who died compared to a median mean airway pressure of 12 cm H2O (interquartile range, 10-14 cm H2O) in participants who survived greater than 90 days (p < 0.001). Mean airway pressure was independently associated with 90-day mortality (odds ratio, 1.38 for difference comparing the 75th to the 25th percentile for mean airway pressure; 95% CI, 1.10-1.74) after adjusting for age, sex, baseline Acute Physiology and Chronic Health Evaluation III, baseline PaO2/FIO2 (modeled with restricted cubic spline), baseline positive end-expiratory pressure, baseline tidal volume, and hospital site. In predicting 90-day mortality, baseline mean airway pressure demonstrated similar discriminative ability (adjusted area under the curve = 0.69) and calibration characteristics as baseline plateau pressure and driving pressure., Conclusions: In a multicenter prospective cohort, baseline mean airway pressure was independently associated with 90-day mortality in mechanically ventilated participants and predicts mortality similarly to plateau pressure and driving pressure. Because mean airway pressure is readily available on all mechanically ventilated patients and all ventilator modes, it is a potentially more useful predictor of mortality in acute respiratory failure.

Background: Hematopoietic stem cell transplant (HSCT) can cure or alleviate a wide range of nonmalignant childhood conditions. However, few studies have examined longitudinal national trends of frequency or short-term complications of HSCT before 2006 when an HSCT became a reportable procedure by US law. By using a US nationally representative database, we conducted nationwide longitudinal analyses on demographics, in-hospital mortality, and short-term complications in nonmalignant HSCT from 2000 to 2012., Procedure: We analyzed 2504 admissions for children < 20 years old who underwent an allogeneic HSCT for a nonmalignant condition by using the Kids' Inpatient Database for the years 2000, 2003, 2006, 2009, and 2012. Changes in in-hospital mortality and other outcomes were assessed over the study period using weighted analyses, which enabled generation of national estimates in each year., Results: The number of admissions for HSCT increased from 334 to 667 from 2000 to 2012, respectively; among them, the use of bone marrow decreased (66.5% to 34.1%, P < 0.001). In-hospital mortality declined (13.4% to 7.1%, P = 0.04), as did bacteremia (28.7% to 10.1%, P < 0.001) and vascular catheter infections (18.8% to 8.7%, P = 0.006), but cytomegalovirus infections increased (4.9% to 15.9%, P < 0.001), as did adenovirus infections (1.8% to 6.9%, P < 0.001) from 2000 to 2012., Conclusion: Population-based analyses demonstrated a substantial expansion of the utilization of HSCT occurred for pediatric nonmalignancies from 2000 to 2012 in the United States, whereas the in-hospital mortality declined by approximately a half. Further research is needed to identify distinct contributing factors., (© 2019 Wiley Periodicals, Inc.)

Massive splenic infarction (MSI) is a rare complication of sickle cell disease, as the spleen generally atrophies within the first few years of life. We report a case of MSI in a 12-year-old boy with homozygous sickle cell anemia (Hb SS) whose chronic transfusion therapy resulted in hypersplenism. The occurrence of a complicated MSI in our patient should perhaps further encourage elective splenectomy in such patients, despite known potential perioperative complications and postsplenectomy risks of infection and thrombosis.

Objectives: Acute chest syndrome is the leading cause of death in children with sickle cell disease and is generally due to respiratory failure. Epidemiologic factors for a need for mechanical ventilation in children with acute chest syndrome require further clarification., Design: Retrospective observational study., Setting: Nationally representative pediatric inpatient records in the United States by using the Kids' Inpatient Database for the years 2003, 2006, 2009, and 2012., Patients: Patients age less than 20 years old with a discharge diagnosis of acute chest syndrome., Measurements and Marin Results: Data were weighted to estimate annual hospitalizations according to hospital characteristics in the United States. Multivariable logistic regression was conducted to ascertain factors associated with use of mechanical ventilation, after adjusting for patient and hospital characteristics. Total hospitalizations for acute chest syndrome were 5,018 in 2003, 6,058 in 2006, 6,072 in 2009, and 6,360 in 2012. Mechanical ventilation use was associated with comorbidities of obesity (odds ratio, 3.35; 95% CI, 1.94-5.78), obstructive sleep apnea (odds ratio, 3.72; 95% CI, 2.23-6.20), and heart disease (odds ratio, 2.19; 95% CI, 1.47-3.27). In addition, nonblack compared with black children (odds ratio, 1.53; 95% CI, 1.02-2.31) and the fall season (p = 0.018) were associated with mechanical ventilation use., Conclusions: Comorbidity of obesity, obstructive sleep apnea, or heart disease could be potentially associated with mechanical ventilation use during an episode of acute chest syndrome. Prospective observational studies would be required to confirm these findings and infer potential interventions for preventing illness severity.

Background and Objective: Pneumonia is the leading cause of death of children. Diagnostic tools include chest radiography, but guidelines do not currently recommend the use of lung ultrasound (LUS) as a diagnostic method. We conducted a meta-analysis to summarize evidence on the diagnostic accuracy of LUS for childhood pneumonia., Methods: We performed a systematic search in PubMed, Embase, the Cochrane Library, Scopus, Global Health, World Health Organization-Libraries, and Latin American and Caribbean Health Sciences Literature of studies comparing LUS diagnostic accuracy against a reference standard. We used a combination of controlled key words for age <18 years, pneumonia, and ultrasound. We identified 1475 studies and selected 15 (1%) for further review. Eight studies (765 children) were retrieved for analysis, of which 6 (75%) were conducted in the general pediatric population and 2 (25%) in neonates. Eligible studies provided information to calculate sensitivity, specificity, and positive and negative likelihood ratios. Heterogeneity was assessed by using Q and I(2) statistics., Results: Five studies (63%) reported using highly skilled sonographers. Overall methodologic quality was high, but heterogeneity was observed across studies. LUS had a sensitivity of 96% (95% confidence interval [CI]: 94%-97%) and specificity of 93% (95% CI: 90%-96%), and positive and negative likelihood ratios were 15.3 (95% CI: 6.6-35.3) and 0.06 (95% CI: 0.03-0.11), respectively. The area under the receiver operating characteristic curve was 0.98. Limitations included the following: most studies included in our analysis had a low number of patients, and the number of eligible studies was also small., Conclusions: Current evidence supports LUS as an imaging alternative for the diagnosis of childhood pneumonia. Recommendations to train pediatricians on LUS for diagnosis of pneumonia may have important implications in different clinical settings., (Copyright © 2015 by the American Academy of Pediatrics.)