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Sufficient communication between hematopoietic stem cell transplantation (HSCT) and pediatric intensive care unit (PICU) teams is pivotal for a successful advanced support in the PICU for HSCT-related complications. We evaluated perceived communication and barriers between both teams with the aim of providing recommendations for improvement. In this cross‐sectional survey, a self‐designed online questionnaire was distributed among ESPNIC and EBMT members. Data were analyzed using descriptive statistics. Over half of HSCT respondents employed a transfer indication protocol and patient assessment tool, but less structured checklist prior to patient transfer. Nearly all PICU respondents perceived this checklist as improvement for communication. Most HSCT and PICU physicians have daily rounds upon patient transfer while this is mostly missing between nursing teams. Half of both HSCT and PICU nurses indicated that HSCT training for PICU nurses could improve communication and patient transfer. Most respondents indicated that structured meetings between HSCT and PICU nurses could improve communication. Overall there is good communication between HSCT and PICU units, although barriers were noted between members of both teams. Based on our findings, we recommend use of a structured and specific checklist by HSCT teams, HSCT training for PICU personnel, and structured meetings between HSCT and PICU nurses.

Infections are frequently experienced complications for patients undergoing haematopoietic cell transplant (HCT). To assess current infection prevention strategies, an international survey among HCT nurses was conducted by the Nurses Group and IDWP of the EBMT. Nurse representatives from all EBMT transplant centres were invited to complete an online questionnaire on protective environment in adult and paediatric HCT units. A total of 141 complete questionnaires were returned for the isolation section and 26 for the paediatric section, the majority of respondents (89.4%) being nurses. A small number of centres (7.1%) reported not allowing visitors, the rest have rules for entering patient rooms. Most HCT units (99.3%) indicated that nurses play a critical role in infection prevention and measures differed between bacterial infections and viral infections. Many of the paediatric units (57.7%) had a play area, applying rules of entry. To our knowledge, this is the first survey on protective environment directed at nurses within HCT centres. Despite having different practices, most HCT units tend to decrease isolation procedures and the use of PPE for multi-drug resistant organisms. This must concur with an increase of hand hygiene compliance, for which our data show that there is still room for improvement.

Hematopoietic stem cell transplantation (HSCT) is currently the standard of care for many malignant and nonmalignant blood diseases. As several treatment-emerging acute toxicities are expected, optimal supportive measurements critically affect HSCT outcomes. The paucity of good clinical studies in supportive practices gives rise to the establishment of heterogeneous guidelines across the different centers, which hampers direct clinical comparison in multicentric studies. Aiming to harmonize the supportive care provided during the pediatric HSCT in Europe, the Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT) promoted dedicated workshops during the years 2017 and 2018. The present paper describes the resulting consensus on the management of sinusoidal obstructive syndrome, mucositis, enteral and parenteral nutrition, iron overload, and emesis during HSCT.

Haploidntical hematopoietic stem cell transplantation has been increasingly used in recent years for patients without a matched donor. The αβTCR+/CD19+ depletion technique provide a graft that is enriched with CD34 cells, γδ-T-cells and natural killer. The current experience with αβTCR+/CD19+ depleted grafts in pediatric patients with malignant and non-malignant disorders, demonstrated rapid engraftment, improved immune reconstitution and low risk of GVHD. Future studies will need to define the optimal conditioning regimen in order to improve transplant outcome.

Allogeneic hematopoietic stem cell transplantation (HSCT) is the effective mean of immune restoration in severe combined immunodefiency (SCID). Usually, HSCT without cytoreductive conditioning is attempted. Nevertheless, conditioning procedures are still preferred in a subset of patients. Herein, we describe the immunological outcome in a cohort of conditioned and unconditioned patients, from diagnosis, through transplantation, to follow-up. This retrospective study was conducted on 17 patients with SCID (10 conditioned, 7 unconditioned) who later underwent HSCT. Immune reconstitution was assessed in the post-transplant year by quantification of T cell receptor excision circles (TRECs) and kappa-deleting recombination excision circles (KRECs), among additional laboratory and clinical evaluations. Unconditioned patients were diagnosed and transplanted earlier. TREC and KREC quantification showed a gradual increase in both groups, with higher levels in the conditioned group. Engraftment percentages differed drastically between groups, favoring the conditioned group. Unconditioned patients were significantly more dependent on intravenous immunoglobulins (IVIGs). One patient from each group succumbed to disease complications. Conditioning demonstrated superior laboratorial outcomes. Patients with unique characteristics (i.e., consanguinity, Bacillus Calmette-Guerin vaccination, impaired access to IVIG) may require personalized considerations. The effort to implement secondary prevention of SCID with newborn screening should continue.

Understanding the potential emotional and psychological risks of pediatric sibling HSC donation is an area of research that remains in its infancy. A cross-sectional survey was distributed electronically to directors at all CIBMTR and EBMT centers to describe current transplant center practices for obtaining assent, preparation for the physical/emotional experiences of donation, and monitoring the post-donation well-being of pediatric donors (

An amendment to this paper has been published and can be accessed via a link at the top of the paper.

Autologous CD19 chimeric-antigen receptor (CAR) T cells demonstrated remarkable remission rates in relapsed and refractory acute lymphoblastic leukemia (R/R ALL). Here, we report results from a phase 1b/2 study of in-house produced CD19 CAR with a CD28 costimulatory domain. Twenty-one patients with R/R ALL were enrolled, and 20 infused. The median age was 11 years (range, 5-48). Patients had a median of 4 prior regimens, including blinatumomab in 6 and prior stem-cell transplantation in 10. In total 8 patients had extramedullary (EM) leukemic involvement, and prior to lymphodepletion and CAR 7 had active lesions, a group underrepresented in previous trials. In vivo expansion of CAR T cells was observed in 18 patients. In total 16 patients developed cytokine release syndrome, and 11 patients developed neurotoxicity, with no toxic deaths. All responding patients were referred to an allogeneic hematopoietic stem-cell transplantation. The remission rate was 90%, including resolution of all refractory EM sites. Four responding patients relapsed, 3 who had a PCR-MRD positive remission at 28 days following CAR-T cells and 1 patient 21 months after an MRD-negative response. The estimated 1-year event-free survival and overall survival are 73% and 90%, respectively. Patients with R/R EM ALL may also benefit from CAR-T cell therapy.

The HSCT (haematopoietic stem cell transplant) is a particular treatment for many haematological and non-haematological diseases. Broadly, there are three different categories of transplantation, autologous, allogeneic and syngeneic, which can be applied to most disease scenarios. Haematopoietic stem cells can be derived from the bone marrow, peripheral blood and umbilical cord blood. HSCT treatment can be divided into separate phases that start with the harvest of the stem cells and passing through the conditioning, aplasia and engraftment until the recovery of the haematopoietic functions. HSCT is indicated in many diseases, and these indications depend on numerous factors such as the disease type, stage and response to previous treatment. Among non-malignant diseases, aplastic anaemia, sickle cell disease and, more recently, autoimmune diseases can also be effectively treated with HSCT. One third of the transplants in children are performed for rare indications such as severe combined immunodeficiencies. Allogeneic HSCT can also cure a number of non-malignant diseases in children, such as Wiskott-Aldrich syndrome and chronic granulomatous disease (CGD). This chapter will include transplant in primary immunodeficiency in children as well as inherited bone marrow failure and inborn errors of metabolism.

Allogeneic stem cell transplantation (SCT) is widely used for treatment of various life-threatening pediatric diseases. It is an intensive process that psychologically affects the whole family. Pediatric donors represent a very unique, underreported, group. The aim of this study is to investigate the sibling donors' and their parents' perspective on the donation process. The cohort included 36 sibling donors and 50 parents of pediatric patients who underwent allogeneic SCT between 1995 and 2010 and were alive at the time of the study. Mean age at donation was 14.78±8.350 years in donors' group and 8.22±4.639 years in parents' group. Data were collected by anonymous questionnaires. Three psychological dimensions were analyzed: donors' personal perspective; donor-recipient interpersonal relationship and the influence of the donation on the family unit. Results showed that the donors experienced a wide range of complex emotional responses, positive and negative, whereas the parents' responses were mainly positive and less complex. This study presents both the sibling donor's and parents' perspective, giving a more complete picture of the donation process within the family. The effects of this intense experience of SCT has a long-term impact on the whole family, indicating the need for follow-up and psychosocial support.

Metabolic syndrome (MetS) is a known complication after hematopoietic stem cell transplantations (HSCT) that contributes to long-term morbidity. We assessed the prevalence of components of the MetS in pediatric survivors of allogeneic HSCT and identified associated risk factors. Thirty-eight patients, median age at HSCT, 8.5 years, were evaluated at a median of 3.9 years post-HSCT. Overweight or obesity was seen in 23.7% of the patients, 15.8% had hypertension, 15.8% had hypertriglyceridemia, and 13% had low high-density lipoprotein cholesterol levels according to age and gender. Four (10.5%) met the criteria of MetS; all were transplanted for malignant disease. Twelve patients (31.6%) had at least one component of the MetS. The 5-year probability of developing components of the MetS revealed that patients with BMI-Z score ≥0 at HSCT were significantly at higher risk than those with lower BMI-Z. Patients who developed components of the MetS had higher levels of insulin, homeostasis model assessment, uric acid, leptin, and lower adiponectin levels. Multivariable regression analysis revealed that BMI-Z-score >1.036 at time of evaluation was associated with 4.3-fold increased risk (P=.050) and adiponectin levels ≤6 μg/mL were associated with 6.7-fold increased risk of develop components of the MetS (P=.007). Overweight and obesity and adiponectin levels may be useful as markers in HSCT survivors.

Background Following cessation of intravenous immunoglobulin (IVIg) administration for allogeneic hematopoietic stem cell transplantation (HSCT) recipients at our unit, we observed a sharp decline in the incidence of cytomegalovirus (CMV) infection. Procedure We conducted a retrospective study of the role of IVIg in the prevention of CMV infection in children and young adults who underwent HSCT from matched related donor. Results We included 109 patients (IVIg+/IVIg– ratio 82/27). Median age was 8.5 years. Patients were transplanted for malignant (59.7%) and nonmalignant diseases (40.3%) with myeloablative, reduced-intensity, and nonmyeloablative conditioning in 76, 22, and 2% of the transplants, respectively. Graft sources were peripheral blood stem cells, bone marrow, and cord blood in 58.7, 39.4, and 2%, respectively. The cumulative incidence of CMV infection at 1 year after HSCT was significantly higher in the cohort that did not receive IVIg compared with the one that did (44.4% vs. 13.4%, respectively, P = 0.001). Significant risk factor for CMV infection in the cohort not receiving IVIg was conditioning with total body irradiation (TBI) (87.5% in TBI+ vs. 26.3% in TBI-, P = 0.003). Conclusions We conclude that children and young adults who undergo HSCT with TBI may need a preemptive regimen of anti-CMV treatment, if they do not get IVIg prophylaxis.

Abstract 806 Objective: The analysis of donor safety and early side effects related to hematopoietic stem cells (HSC) collection from bone marrow (BM) or peripheral blood (PB) in pediatric HLA-identical sibling donors. Methods: From 2005 to 2009, data regarding pediatric ( Results: A total number of 410 pediatric sibling donors with median age of 9 (range; 0.3-18) years were enrolled into the study; including 12% aged 4 yrs, OR=9.9, p Conclusions: BM or PB HSC collection in pediatric sibling donors is safe, however there is a risk of mild, short-term and easy to prevent or control early side effects. The risk of SAE in healthy pediatric donors exists, although it is small. Donors and parents must be informed about the risk of possible complication. There is a need of donor outcome and follow-up registry. Disclosures: No relevant conflicts of interest to declare.