NUEVA TERAPIA CON INMUNOGLOBULINAS EN UN CASO DE HEMOCROMATOSIS NEONATAL

La hemocromatosis neonatal es una enfermedad hepática muy severa del recién nacido y se asocia a una alta mortalidad. Se cree que su etiología es de tipo aloinmune, debido a la presencia de un anticuerpo materno hasta ahora desconocido que interfiere con el metabolismo férrico del feto, llegando a producir gran morb i mortal ¡dad. Basándonos en esta teoría, el tratamiento materno con inmunoglobulinas intravenosas en gestaciones sucesivas podría prevenir el desarrollo de un nuevo cuadro de hemocromatosis neonatal. Se describe el caso de una gestante con un hijo anterior diagnosticado y fallecido neonatalmente por hemocromatosis, a la que en el embarazo actual se le trató con inmunoglobulinas intravenosas consiguiendo un hijo sano y vivo. Es el primer caso descrito en España y demuestra el éxito de esta terapia, tal como describe la literatura.Neonatal hemochromatosis is a severe neonatal liver disease with a high mortality and recurrence rate. It is supposed to be a gestational alloimmune disease because of the existence of maternal antibodies against fetal hepatic metabolism. On the basis of this hypothesis, the administration of intravenous immunoglobulin has been reported as a successful treatment during the following pregnancy. We describe the first case of this treatment in Spain which confirms the data available in the literature

Immunocompromised Children and Young Patients Living with Pets: Gaps in Knowledge to Avoid Zoonosis

Background and Objectives. Although pets are known to be beneficial for children, they could also imply risks for immunocompromised ones. We report the prevalence of children and young patients living with pets in a cohort of immunosuppressed pediatric patients and describe risk behaviors for acquiring zoonosis and compliance with veterinary recommendations. Methods. A cross-sectional, observational study was performed in a large tertiary hospital in Madrid, including immunosuppressed patients from different regions of Spain. The participants were asked to complete an online questionnaire. Results. Two hundred and eighty-four responses were received: 62.3% solid organ transplantation (177/284), 22.8% hematopoietic stem cell transplantation (65/284), and 14.8% inborn errors of immunity (42/284). The median age was 11 years (interquartile range 5.9–15.4), and 55% were boys (156/284). Up to 45% (130/284) of the respondents lived with 201 pets (74% of them dogs and cats). Half of the patients owning dogs or cats did not comply with at least one of the recommendations regarding vaccination, deworming, feeding, and/or veterinarian recommended controls. The poorest findings were related to deworming regimens. Only 42.8% (117/273) of the participants received specific recommendations from their healthcare professionals about companion animals. However, up to 97% of the families considering acquiring a pet did so when the professional did not contraindicate it (31/32), while 72% of the families having pets got rid of their pets when they were advised against animals (8/11). Conclusions. Pet ownership is frequent among immunocompromised children. They presented risky exposures for acquiring zoonoses, and basic veterinary recommendations were not frequently followed. The opinion of professionals significantly influenced the decision to acquire pets, but less than half of the families received recommendations in this regard.Spanish Pediatrics Association (AEP) (2021 Research Grant)MAPFRE FoundationCarlos III Health Institute - Fondos Feder (BAE21/00022)4.521 Q1 JCR 20210.990 Q1 SJR 2022No data IDR 2021UE

Ceftazidime–Avibactam Use in a Case Series of Difficult-to-Treat or Recurrent Infections in Pediatric Patients with Complex Chronic Conditions: Effectiveness and Absence of Resistance Development

The prevalence of multidrug-resistant Gram-negative infections, particularly carbapenem-resistant strains, has become a significant global health concern. Ceftazidime–avibactam (CZA) has emerged as a promising treatment option. However, data on its efficacy and safety in children are scarce, necessitating further investigation. We conducted a descriptive case series at a tertiary hospital in Spain from February 2019 to January 2022. Pediatric patients (n = 8) and biliary atresia (n = 4). The predominant type of infection for which they received CZA was intra-abdominal infection caused or suspected to be caused by OXA-48-producing Klebsiella pneumoniae. CZA was generally well tolerated. Within the first month of starting CZA therapy, two patients died, with one case directly linked to the infection’s fatal outcome. Some patients needed repeated courses of therapy due to recurrent infections, yet no resistance development was noted. In summary, the use of CZA showed effectiveness and safety, while the lack of resistance development highlights CZA’s potential as a primary treatment option against OXA-48-producing infections

Social Resources for Transplanted Children and Families in European Union Hospitals of ERN TransplantChild

Social well-being is an intrinsic part of the current concept of health. In the context of chronic disease, there are many challenges we face in order to provide social well-being to patients and their families, even more if we talk about rare diseases. TransplantChild, a European Reference Network (ERN) in paediatric transplantation, works to improve the quality of life of transplanted children. It is not possible to improve the quality of life if the human and material resources are not available. With this study, we want to identify the economic aids, facilities, services, and financed products that are offered to families in different European centres. We also want to find out who provides these resources and the accessibility to them. We designed an ad hoc survey using the EU Survey software tool. The survey was sent to representatives of the 26 ERN members. In this article we present the results obtained in relation to two of the aspects analysed: long-term financial assistance and drugs, pharmaceuticals and medical devices. Some resources are equally available in all participating centres but there are significant differences in others, such as education aids or parapharmacy product financing. A local analysis of these differences is necessary to find feasible solutions for equal opportunities for all transplanted children in Europe. The experience of centres that already provide certain solutions successfully may facilitate the implementation of these solutions in other hospitals

ABO Incompatible Liver Transplantation in Children: A 20 Year Experience from Centres in the TransplantChild European Reference Network

An increasing number of AB0-incompatible (AB0i) liver transplantations (LT) are being undertaken internationally in recent years due to organ shortages and the need for urgent transplantation. The aim of our study was establish the value of ABOi LT from available retrospective results of AB0i pediatric liver transplantations performed in European reference centers now belonging to the TransplantChild, European Reference Network (ERN). Data from medical records were analyzed, including demographic data, diagnosis, urgency of transplantation, time on the waiting list, PELD/MELD score, desensitization procedures, immunosuppression, selected post-transplant complications, and patient and graft survival. A total of 142 patients (pts) with transplants between 1986 and 2018 in 8 European transplant centers were included in the study. The indications for liver transplantation were: cholestatic diseases in 62 pts, acute liver failure in 42 pts, and other conditions in the remaining 38 pts. Sixty-six patients received grafts from living donors, and seventy-six received grafts from deceased donors. Both patient and graft survival were significantly affected by deceased donor type, urgent transplantation, and the development of vascular complications. In the multivariate analysis, vascular complications had a negative impact on patient and graft survival, while a longer time from the first AB0i LT in the study showed better results, suggesting an international learning experience. In conclusion, we believe that AB0i LT in children is now a safe procedure that may be adopted more readily in children.This study was performed with the participation of centers of the European Reference Network on Pediatric Transplantation (ERN TransplantChild), which is partly cofounded by the European Union within the framework of the Third Health Programme ERN, specific agreement number 847103

Vaccination practices in pediatric transplantation: A survey among member centers of the European reference network <scp>TransplantChild</scp>

Background: There is considerable variation in vaccination practices between pediatric transplant centers. This study aims to evaluate active immunization attitudes and practices among ERN-TransplantChild centers and identify potential areas of improvement that could be addressed by shared evidence-based protocols. Methods: A cross-sectional questionnaire of attitudes and practices toward immunization of pediatric SOT and HSCT candidates and recipients was sent to a representative member of multidisciplinary teams from 27 European centers belonging to the ERN-TransplantChild. Results: A total of 28/62 SOT programs and 6/12 HSCT programs across 21 European centers participated. A quarter of centers did not have an on-site protocol for the immunizations. At the time of transplantation, pediatric candidates were fully immunized (80%-100%) in 57% and 33% of the SOT and HSCT programs. Variations in the time between vaccine administration and admission to the waiting list were reported between the centers, with 2 weeks for inactivated vaccines and variable time (2-4 weeks) for live-attenuated vaccines (LAVs). Almost all sites recommended immunization in the post-transplant period, with a time window of 4-8 months for the inactivated vaccines and 16-24 months for MMR and Varicella vaccines. Only five sites administer LAVs after transplantation, with seroconversion evaluated in 80% of cases. Conclusions: The immunization coverage of European pediatric transplant recipients is still inconsistent and far from adequate. This survey is a starting point for developing shared evidence-based immunization protocols for safe vaccination among pediatric transplant centers and generating new research studies

Current Practices on Diagnosis, Prevention and Treatment of Post-Transplant Lymphoproliferative Disorder in Pediatric Patients after Solid Organ Transplantation: Results of ERN TransplantChild Healthcare Working Group Survey

Background: Post-transplant lymphoproliferative disease (PTLD) is a significant complication of solid organ transplantation (SOT). However, there is lack of consensus in PTLD management. Our aim was to establish a present benchmark for comparison between international centers and between various organ transplant systems and modalities; (2) Methods: A cross-sectional questionnaire of relevant PTLD practices in pediatric transplantation was sent to multidisciplinary teams from 17 European center members of ERN TransplantChild to evaluate the centers' approach strategies for diagnosis and treatment and how current practices impact a cross-sectional series of PTLD cases; (3) Results: A total of 34 SOT programs from 13 European centers participated. The decision to start preemptive treatment and its guidance was based on both EBV viremia monitoring plus additional laboratory methods and clinical assessment (61%). Among treatment modalities the most common initial practice at diagnosis was to reduce the immunosuppression (61%). A total of 126 PTLD cases were reported during the period 2012-2016. According to their histopathological classification, monomorphic lesions were the most frequent (46%). Graft rejection after PTLD remission was 33%. Of the total cases diagnosed with PTLD, 88% survived; (4) Conclusions: There is still no consensus on prevention and treatment of PTLD, which implies the need to generate evidence. This might successively allow the development of clinical guidelines