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Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the treatment options for myelodysplastic syndromes (MDS). This treatment is indicated as first-line treatment for high-risk MDS according to the IPSS and R-IPSS classifications and improves overall survival and progression-free survival. However, allo-HSCT is not indicated in first intention for low-risk MDS. It can be discussed in case of cytopenias needing transfusions, poor evolution under other treatment, or in case of poor prognosis molecular anomaly. Allo-HSCT is a treatment that can be complicated by early or late toxicities (graft versus host disease, infections, chemotherapy toxicity…). The decision to do an allo-HSCT is based on the benefit/risk ratio between the risk of progression from MDS to myeloid leukemia and the risk of transplant related mortality, which increases with the patient's age and comorbidities. The indication of a cytoreductive treatment before allo-HSCT depends on the blasts count, and on the delay before the allograft. The use of reduced intensity conditioning regimen and alternative donors such as haploidentical donors, expanded the indications for allo-HSCT. Relapse remains one of the main causes of mortality after allo-HSCT. Some genetic mutations and karyotype anomalies increase the risk of post-transplant relapse. Preventive treatments for relapse are currently being studied. Treatments such as azacytidine, donor lymphocytes infusions or targeted therapies can be used, prophylactically or preemptively., (Copyright © 2023 Société Française du Cancer. Published by Elsevier Masson SAS. All rights reserved.)

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the most effective treatment for selected patients with acute myeloid leukemia (AML) and relies on a "graft-versus-leukemia" effect (GVL) where donor T lymphocytes mediate control of malignant cell growth. However, relapse remains the major cause of death after allo-HSCT. In various malignancies, several immunoregulatory mechanisms have been shown to restrain antitumor immunity, including ligand-mediated engagement of inhibitory receptors (IRs) on effector cells, and induction of immunosuppressive cell subsets, such as regulatory T cells (Tregs) or myeloid-derived suppressor cells (MDSCs). Relapse after HSCT remains a major therapeutic challenge, but immunoregulatory mechanisms involved in restraining the GVL effect must be better deciphered in humans. We used mass cytometry to comprehensively characterize circulating leukocytes in 2 cohorts of patients after allo-HSCT. We first longitudinally assessed various immunoregulatory parameters highlighting specific trends, such as opposite dynamics between MDSCs and Tregs. More generally, the immune landscape was stable from months 3 to 6, whereas many variations occurred from months 6 to 12 after HSCT. Comparison with healthy individuals revealed that profound alterations in the immune equilibrium persisted 1 year after HSCT. Importantly, we found that high levels of TIGIT and CD161 expression on CD4 T cells at month 3 after HSCT were distinct features significantly associated with subsequent AML relapse in a second cross-sectional cohort. Altogether, these data provide global insights into the reconstitution of the immunoregulatory landscape after HSCT and highlight non-canonical IRs associated with relapse, which could open the path to new prognostic tools or therapeutic targets to restore subverted anti-AML immunity., (© 2022 by The American Society of Hematology.)

A patent ductus arteriosus, very common in the preterm infant (incidence up to 70% in infants less than 28 weeks gestational age), causes a left-to-right shunt leading to overload of the pulmonary circulation and low systemic blood flow. These hemodynamic anomalies are associated with a higher incidence of respiratory, neurological, and digestive complications of prematurity, as well as a higher mortality rate. Although growing knowledge about the natural history, the pathophysiology, and the mechanisms of closure of the ductus arteriosus has resulted in therapeutic progress, management of the patent ductus arteriosus in the preterm infant is still a subject of intense controversy. We describe here the different treatment modalities, their benefits and side effects, and the therapeutic strategies that have been tested in the last four decades. Based on this evidence, the current trend is a personalized approach to the patent ductus arteriosus, adapted to each preterm infant's individual characteristics and risk factors., (Copyright © 2016 Elsevier Masson SAS. All rights reserved.)

Objectives: Pediatric practice is a difficult task requiring physicians to discriminate potentially serious situations among a variety of benign situations. The goal of this study was to assess the knowledge of students beginning their residency in family medicine on these situations., Methods: One hundred and three students were evaluated on pediatric "traffic lights" using 103 true/false questions. Pediatric and family medicine teachers of the Nantes University Hospital defined "traffic lights" as the fundamentals of pediatrics, misleading situations, and diagnosis and treatment that should not to be missed. Emergency levels were defined by colors, with "red light" corresponding to life-threatening emergencies., Results: Thirty-six percent of the questions (n=103) had a correct response rate below 75%. Thirty-two percent of the questions on emergency situations ("red lights") (n=37) had a response rate below 75%. Fifteen percent of the questions (n=103) had a correct response rate below 50%, half of which were "red light" (e.g., on meningitis, diabetic acidocytosis, or shock). Questions concerning infants (n=24) had significantly fewer correct answers (correct response rate below 50%: 29% versus 10%; P=0.047). All the students answered seven questions correctly. The students' mean score (percentage of good answers) was 76% (±6%). No student had 100% or less than 50% good answers., Conclusions: Some life-threatening situations or situations concerning infants had not been mastered by most of the students. It is therefore essential to optimize the teaching of pediatrics during the second cycle of medical studies., (Copyright © 2014 Elsevier Masson SAS. All rights reserved.)

Objective: To assesses the results of our education program relative to anticoagulation therapy and self-monitoring of INR., Materials and Methods: All children treated with oral anticoagulation therapy and followed using INR self-monitoring in Tours and Nantes, France, were included. A questionnaire on the treatment and its management was sent to the patients and their family. We analyzed the quality of anticoagulation using the proportion of INR within the target range., Results: Thirty-three children were included, with a mean age of 10.9years. Thirty-one questionnaires could be analyzed. Insufficient knowledge on the treatment objective and risks, the INR target range, and the interpretation of the INR was observed. We found 65.8% of INRs within the target range. The proportion of INRs within the target range was better with the INR self-testing than with the laboratory test (69.7% vs. 49.1%, P=0.003). There was no major complication observed during the study period. A moderate correlation (K=0.57) was noted between the INRs measured with the point-of-care monitor and the laboratory test on the same day., Conclusion: However, home-monitoring of oral anticoagulation therapy increases the quality of anticoagulation. Both education and the training program need improvement in order to provide patients and their families with better knowledge on anticoagulation therapy., (Copyright © 2013. Published by Elsevier SAS.)

Coarctation of the aorta is an obstructive aortic lesion that causes secondary hypertension. Diagnosis is sometimes difficult because patients could present no other symptom. An asymptomatic 20-month-old child was referred in the context of isolated high blood pressure. Echocardiography was not effective, but CT scan established the diagnosis of coarctation. This case report underlines the importance of blood pressure measurement in the detection of aortic obstructions in the pediatric population., (Copyright © 2011 Elsevier Masson SAS. All rights reserved.)

To explore international variation in implementation of point-of-care ultrasound in the neonatal intensive care unit (NICU), contributions were invited from neonatologists and paediatric cardiologists in six countries. The contributors show variation in national implementation that ranges from almost total coverage through to a minority of NICUs having point-of-care ultrasound capability. To a varying degree in all systems the main barriers have been concerns from the consultative specialties that traditionally use ultrasound, relating to the risk of misdiagnosis but also involving different clinical needs, liability concerns and lack of outcome-based evidence. All contributors agreed that safe point-of-care ultrasound depends on close collaboration with the consultative specialties and also that there is a need to develop training and accreditation structures for neonatologists using ultrasound., (Crown Copyright © 2010. Published by Elsevier Ltd. All rights reserved.)

Background: Patent ductus arteriosus is a common complication of prematurity that frequently requires surgical or medical treatment. The benefit of prophylactic treatment by indometacin, a cyclo-oxygenase inhibitor, remains uncertain compared with curative treatment. This benefit could be improved with ibuprofen, another cyclo-oxygenase inhibitor with fewer adverse effects than indometacin on renal, mesenteric, and cerebral perfusion. We aimed to compare prophylactic and curative ibuprofen in the treatment of this abnormality in very premature infants., Methods: We did a randomised controlled trial in infants younger than 28 weeks of gestation, who were randomly assigned to receive either three doses of ibuprofen or placebo within 6 h of birth. After day 3, symptomatic patent ductus arteriosus was treated first by open curative ibuprofen, then back-up indometacin, surgery, or both. The primary endpoint was need for surgical ligation. Analysis was per protocol., Findings: The study was stopped prematurely after 135 enrollments because of three cases of severe pulmonary hypertension in the prophylactic group. 65 infants received prophylactic ibuprofen, and 66 received placebo. Prophylaxis reduced the need for surgical ligation from six (9%) to zero (p=0.03), and decreased the rate of severe intraventricular haemorrhage from 15 (23%) to seven (11%) (p=0.10). However, survival was not improved (47 [71%] placebo vs 47 [72%] treatment, p=1.00), because of high frequency of adverse respiratory, renal, and digestive events., Interpretation: In premature infants, prophylactic ibuprofen reduces the need for surgical ligation of patent ductus arteriosus, but does not reduce mortality or morbidity. Therefore, it should not be preferred to early curative ibuprofen.

Unlabelled: Refractory hypoxemia in the newborn requires an accurate diagnostic investigation for an optimal and prompt management., Case Report: After a short delivery, a post-term newborn developed a severe hypoxemia with patchy pulmonary alveolar opacities and systolic right-to-left extrapulmonary shunting through the ductus arteriosus. Echocardiography ruled out a cardiac malformation and showed dilated left atrium with left-to-right shunting by the ovale foramen due to left ventricular dysfunction. Inotropic support associated with mechanical ventilation allowed a rapid improvement., Conclusion: A pathophysiological analysis is needed in case of severe neonatal hypoxemia. Doppler ultrasound is a non invasive and rapid method allowing the adjustment of the therapeutic strategy.

We report three cases of severe hypoxaemia after ibuprofen administration during a randomised controlled trial of prophylactic treatment of patent ductus arteriosus with ibuprofen in premature infants born at less than 28 weeks of gestation. Echocardiography showed severely decreased pulmonary blood flow. Hypoxaemia resolved quickly on inhaled nitric oxide therapy. We suggest that investigators involved in similar trials pay close attention to pulmonary pressure if hypoxaemia occurs after prophylactic administration of ibuprofen.

Unlabelled: The aim of the study was to report the incidence and causes of preoperative deaths in isolated transposition of the great vessels and to describe the clinical findings in these neonates., Patients and Methods: In five French centers of pediatric cardiology, data of all the neonates with isolated transposition of the great vessels who died before arterial switch operations between January 1986 and June 1996 were obtained from reviewing hospital files, echocardiography records and autopsy reports., Results: Among 199 neonates with transposition of the great vessels, 20 (9.9%) died before surgery. The death was related to intracranial haemorrhage in one premature neonate, severe and early hypoxemia in 13 full-term patients (group A) and later sudden collapse in six patients (group B). In group A, the symptoms occurred within 20 minutes after the birth and included cyanosis (n = 12), acute respiratory distress (n = 8), and shock (n = 4). Despite assisted ventilation (n = 13), bicarbonate infusion (n = 12), prostaglandin E1 (n = 7), inotropic drugs (n = 5) and balloon atrioseptostomy (n = 7), death occurred at the median age of five hours. The patent foramen ovale was absent or tiny in ten patients, normal in one patient and not specified in two patients. The ductus arteriosus was patent in ten patients and not specified in three patients. In group B, the neonates were initially in a good hemodynamic condition. Unexplained death occurred between two and five days after the birth: one infant with a large patent foramen ovale did not receive prostaglandin E1, four patients died a few hours after an angiographic study or a balloon atrioseptostomy was performed in a catheterization laboratory, and one child suffered from a cerebral anoxia due to a tightened cord., Conclusion: We conclude that the high preoperative mortality rate in isolated transposition of the great vessels is mainly due to absent or small atrial shunt. These findings suggest that only prenatal diagnosis of transposition of the great vessels with immediate balloon atrioseptostomy could avoid a fatal outcome.

Background: Refractory hypoxemia in the newborn requires a precise diagnostic investigation for optimal and fast management., Case Report: A full term newborn presented with refractory hypoxemia associated with radiologically clear lung fields and extrapulmonary shunt. Echocardiography ruled out a cardiac malformation. The persistence of hypoxemia despite treatment of the extrapulmonary shunt and the absence of parenchymatous pulmonary disease led to suspect misaligned lung vessels with alveolar capillary dysplasia. This diagnosis was confirmed by post mortem microscopic examination of the lung., Conclusion: The diagnosis of misaligned lung vessels with alveolar capillary dysplasia can be suspected on clinical features. The disposition of pulmonary veins must be checked to recognize this disease in case of neonatal death with pulmonary hypertension.