Allogeneic transplantation in patients with acute lymphoblastic leukaemia in first remission (ALL-CR1) has been studied in several clinical trials. However, no pooled survival analysis has yet been done. We conducted a survival meta-analysis to compare allogeneic transplantation vs chemotherapy or autologous transplantation using an intention-to-treat approach. Our study included the controlled clinical trials, wherein allocation to allogeneic transplant was based on donor availability. The event-free individual survival data were reconstructed on the basis of published information and Kaplan-Meier graphs. We then generated the meta-analytic event-free survival curves for the two treatments. The mean survival gain per patient was estimated and a simplified cost-effectiveness assessment was carried out. In the allogeneic transplantation group, 293 patients were examined and 479 as controls (four trials). The event-free survival difference was statistically significant (P ¼ 0.011). The relative risk for event occurrence was 0.79 for the experimental group vs the controls (95% CI: 0.66-0.96; P ¼ 0.017). The mean survival gain was 1 year per patient. The cost per life-year gained was less than the conventional threshold of h50 000. Allogeneic transplantation in ALL-CR1 improves event-free survival as compared to chemotherapy or autologous transplantation. Its cost-effectiveness profile is acceptable.
Background: MIS-C is a potentially severe inflammatory syndrome associated with SARS-CoV-2 exposure. Intravenous immunoglobulin (IVIG) is considered the first-tier therapy, but it implies infusion of large fluid volumes that may worsen cardiac function.Patients and Methods: Since April 2020, we have developed a treatment protocol that avoids the infusion of IVIG as first-line therapy in the early phase of MIS-C. In this study, we retrospectively analyzed a cohort of consecutive patients treated according to this protocol between 01/04/2020 and 01/04/2021.Results: In the last year, 31 patients have been treated according to the protocol: 25 with high-dose pulse MP (10 mg/kg) and 6 with 2 mg/kg. 67.7% of the patients responded to the initial treatment, while the others needed a step-up, either with Anakinra (25.8%) or with MP dose increase (6.5%). IVIG was administered in four patients. Overall, only one patient (3.2%) needed ICU admission and inotropic support; one patient developed a small coronary artery aneurysm.Conclusions: Timely start of MP therapy and careful fluid management might improve the outcomes of MIS-C patients.
Background The first Covid-19 pandemic affected the epidemiology of several diseases. A general reduction in the emergency department (ED) accesses was observed during this period, both in adult and pediatric contexts. Methods This retrospective study was conducted on the behalf of the Italian Society of Pediatric Nephrology (SINePe) in 17 Italian pediatric EDs in March and April 2020, comparing them with data from the same periods in 2018 and 2019. The total number of pediatric (age 0–18 years) ED visits, the number of febrile urinary tract infection (UTI) diagnoses, and clinical and laboratory parameters were retrospectively collected. Results The total number of febrile UTI diagnoses was 339 (73 in 2020, 140 in 2019, and 126 in 2018). During the first Covid-19 pandemic, the total number of ED visits decreased by 75.1%, the total number of febrile UTI diagnoses by 45.1%, with an increase in the UTI diagnosis rate (+ 121.7%). The data collected revealed an increased rate of patients with two or more days of fever before admission ( p = 0.02), a significant increase in hospitalization rate (+ 17.5%, p = 0.008) and also in values of C reactive protein (CRP) ( p = 0.006). In 2020, intravenous antibiotics use was significantly higher than in 2018 and 2019 (+ 15%, p = 0.025). Urine cultures showed higher Pseudomonas aeruginosa and Enterococcus faecalis percentages and lower rates of Escherichia coli ( p = 0.02). Conclusions The first wave of the Covid-19 pandemic had an essential impact on managing febrile UTIs in the ED, causing an absolute reduction of cases referring to the ED but with higher clinical severity. Children with febrile UTI were more severely ill than the previous two years, probably due to delayed access caused by the fear of potential hospital-acquired Sars-Cov-2 infection. The possible increase in consequent kidney scarring in this population should be considered.
A Meta-analysis W e recently completed a meta-analysis similar to that published by Yanada et al.1,2 For our study, we used a nearly identical method of clinical trial selection (main source of clinical data, MEDLINE in PubMed version; time interval, January 2000 to June 2006) and included 4 controlled studies, 3-6 all of which were cited in the article by Yanada et al.One point of strength of our meta-analysis is that our statistical model 7 accounted for the length of follow-up and, thus, was able to generate a survival metaanalysis (inclusive of the construction of the 2 censored Kaplan-Meier meta-analytic curves for transplantation and chemotherapy, respectively).In their statistical model, Yanada et al considered only crude survival rates (with no adjustment for time); consequently, their results are exposed to a potential bias favoring transplantation, because the same statistical weight was given to early and late events (in a situation in which some early events, eg transplantation-related deaths, are more frequent in the experimental arm).Our survival meta-analysis (relative risk of death or recurrence, 0.79 in favor of transplantation; 95% confidence interval, 0.66-0.96; P ¼ .017) provided results nearly identical to those of Yanada et al. In addition, our statistics allowed us to estimate the mean survival gain produced by transplantation (1.0 year per patient) and to carry out a preliminary cost-effectiveness analysis (cost per life year gained, €37,000, which is less than the conventional threshold of €50,000 or $50,000). In this assessment, information on costs (€82,000 and €45,000 per patient for transplantation and chemotherapy, respectively) was derived from Lee et al. 8 In conclusion, our results are noteworthy, because, on the one hand, they confirm (with a different statistical model) the findings obtained by Yanada et al. 1; and, on the other hand, they suggest that allogeneic transplantation has an acceptable cost-effectiveness profile.
Hemophagocytic lymphohistiocytosis (HLH) is a rare, life-threatening condition characterized by hyperinflammation in an uncontrolled and ineffective immune response. Despite great improvement in diagnosis and treatment, it still represents a challenge in clinical management, with poor prognosis in the absence of an aggressive therapeutic approach. The present literature review focuses on secondary HLH at pediatric age, which represents a heterogeneous group in terms of etiology and therapeutic approach. It summarizes the most recent evidence on epidemiology, pathophysiology, diagnosis, treatment and prognosis, and provides a detailed description and comparison of the major subtypes of secondary HLH. Finally, it addresses the open questions with a focus on diagnosis and new treatment insights.
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