Treosulfan-Based Conditioning Regimen In Pediatric Hematopoietic Stem Cell Transplantation: A Retrospective Analysis on Behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC).

Increasing data on treosulfan-based conditioning regimens before hematopoietic stem cell transplantation (HSCT) demonstrate the consistent benefits of this approach, particularly regarding acute toxicity. This study aimed to describe the results of treosulfan-based conditioning regimens in children, focusing on toxicity and outcomes when used to treat both malignant and nonmalignant diseases. This retrospective observational study of pediatric patients treated in Spain with treosulfan-based conditioning regimens before HSCT was based on data collection from electronic clinical records. We studied a total of 160 treosulfan-based conditioning HSCTs to treat nonmalignant diseases (n = 117) or malignant diseases (n = 43) in 158 children and adolescents. The median patient age at HSCT was 5.1 years (interquartile range, 2 to 10 years). The most frequent diagnoses were primary immunodeficiency (n = 42; 36%) and sickle cell disease (n = 42; 36%) in the nonmalignant disease cohort and acute lymphoblastic leukemia (n = 15; 35%) in the malignant disease cohort. Engraftment occurred in 97% of the patients. The median times to neutrophil engraftment (17 days versus 14 days; P = .008) and platelet engraftment (20 days versus 15 days; P = .002) were linger in the nonmalignant cohort. The 1-year cumulative incidence of veno-occlusive disease was 7.98% (95% confidence interval [CI], 4.6% to 13.6%), with no significant differences between cohorts. The 1-year cumulative incidence of grade III-IV acute graft-versus-host disease (GVHD) was higher in the malignant disease cohort (18% versus 3.2%; P = .011). Overall, the malignant cohort had both a higher total incidence (9% versus 3%; P < .001) and a higher 2-year cumulative incidence (16% versus 1.9%; P < .001) of total chronic GVHD. The 2-year cumulative transplantation-related mortality was 15%, with no difference between the 2 cohorts. The 5-year overall survival was 80% (95% CI, 72% to 86%) and was higher in the nonmalignant cohort (87% versus 61%; P = .01). The 2-year cumulative incidence of relapse was 25% in the malignant cohort. The 5-year cumulative GVHD-free, relapse-free survival rate was 60% (95% CI, 51% to 70%) and was higher in the nonmalignant cohort (72% versus 22%; P < .001). A treosulfan-based radiation-free conditioning regimen is feasible, achieving a high engraftment rate and 5-year overall survival, and is an emerging option for the first HSCT in nonmalignant diseases.

Outcomes for paediatric acute leukaemia patients admitted to the paediatric intensive care unit.

Children with acute leukaemia (AL) are a high-risk population for infections and life-threatening conditions requiring paediatric intensive care unit (PICU) admission, presenting an increased mortality rate. A few literature exists about PICU outcomes in this kind of patients, especially with haematopoietic stem cell transplant (HSCT) background. We investigated the clinical and epidemiological characteristics of these patients as well as their outcomes. A retrospective, single-centre analytical/observational study was conducted from January 2011 to December 2018 in the PICU of a tertiary care hospital. AL patients from 28 days to 18 years old admitted to the PICU were included, excluding those with histories of HSCT or CAR T-cell therapy. We collected epidemiological and clinical characteristics, laboratory and microbiology results and outcomes. Forty-three patients with AL required urgent admission (35 lymphoblastic and 8 myeloblastic) for 63 different episodes. The main reasons were sepsis (21, 33.3%), hyperleukocytosis (12, 19%), respiratory failure (11, 17.5%) and seizures (8, 12.7%). Nineteen (30.2%) required inotropic support, and fifteen (23.8%) required mechanical ventilation. Three patients died at the hospital (3/43, 6.9%). Sixty-day mortality was 9.3%, and 1-year mortality was 13.9%. There was no differences regarding the type of AL and 60-day mortality (log-rank 2.652, p = 0.103).Conclusion: In our study, the main cause of admission for AL patients was infection, which was associated to more severity and longer hospital admission. What is Known: • Acute leukaemia is the most common childhood cancer. Admission to a paediatric intensive care unit is required in 30% of children with acute leukaemia. • Regarding the outcomes of children with acute leukaemia that require admission to the intensive care unit data are scarce. What is New: • Mortality in acute leukaemia patients admitted to the paediatric intensive care unit is lower than that of patients with a history of stem cell therapy but higher than that of patients with solid tumours. • The main reason for admission was sepsis, which is related in literature to more severity and long length of stay.

Long-term outcome of neonates and infants with permanent junctional reciprocating tachycardia. When cardiac ablation changes natural history.

Supraventricular tachycardias (SVT) are the most common arrhythmias in the perinatal period. Permanent junctional reciprocating tachycardia (PJRT) is a rare form of SVT, often incessant and refractory to pharmacological treatments. Our goal was to analyze the clinical features and treatment of PJRT in patients younger than 2 months and to describe their long-term outcomes. METHODS: Retrospective descriptive observational study of patients diagnosed between 2000 and 2015 in the NICU of a referral center for the treatment of pediatric arrhythmias. History of pregnancy, neonatal period, pharmacological treatment, electrophysiological study and long-term follow-up were reviewed. RESULTS: 129 of the 10.198 (1.26%) patients admitted to the NICU had SVT, sixteen of them (12.3%) being diagnosed as PJRT. Ten cases had a prenatal diagnosis. For those six patients postnatally diagnosed, the tachycardia was detected either during a routine check-up or because of acute hemodynamic instability. The majority of patients required combinations of drugs, despite that the tachycardia was poorly controlled. Fifteen patients underwent cardiac ablation, nine patients (60%) in the neonatal period and six during childhood. The procedure was completely effective in all cases. One patient had a transient complete AV block that resolved spontaneously 24 hours after the procedure. No other complications were seen. After a mean follow-up of 10.9 years, no patient has presented recurrence, cardiac dysfunction, signs of ischemia or EKG abnormalities, they all have a normal life. CONCLUSIONS: When PJRT is refractory to multiple drugs, cardiac ablation should be taken into account at early stages even in very young patients.

[Adolescent tuberculosis; a challenge and opportunity to prevent community transmission]. / Tuberculosis en el adolescente; reto y oportunidad de evitar el contagio a la comunidad.

INTRODUCTION: Adolescents may present with adult-type pulmonary tuberculosis (TB), including cavity disease in upper lobes and smear-positive sputum, which involves a significant transmission risk for social and family contacts. PATIENTS AND METHODS: A retrospective (2007-2012) observational study of a case series of TB was conducted in children and adolescents (<18 years) in a paediatric referral centre in Barcelona. Patients aged≤12 and>12 years at diagnosis are compared. RESULTS: The series consisted of 124 patients (56.5% males, median age: 4.0 years). In half of the cases, the patient was of immigrant origina and TB was diagnosed after clinical-radiological suspicion, intra-thoracic disease being the most common (91.9%). Cultures yielded positive results in one third of cases (37.9%) and isolates were sensitive to oral first-line anti-TB agents in 100%. Median (interquartile range) duration of treatment was 6 (6-9) months, directly observed therapy was needed in 10 patients, and there was a satisfactory outcome after treatment in 98.4%. Among adolescents, TB was more prevalent in females (63.2%) and immigrant patients (68.4%), comorbidity at diagnosis and lung cavity forms were more common, and the source case was identified only in 21.1% of the patients. CONCLUSION: Adult-type pulmonary TB is common among adolescents, may be associated with underlying medical conditions, and is often diagnosed late, posing a significant transmission risk to the community.