Pediatric Bone Marrow Transplant Research Articles

COVID-19 disease in children and adolescents following allogeneic hematopoietic stem cell transplantation: A report from the Turkish pediatric bone marrow transplantation study group.

Data on the risk factors and outcomes for pediatric patients with SARS-CoV-2 infection (COVID-19) following hematopoietic stem cell transplantation (HSCT) are limited. The study aimed to analyze the clinical signs, risk factors, and outcomes for ICU admission and mortality in a large pediatric cohort who underwent allogeneic HSCT prior to COVID-19 infection. In this nationwide study, we retrospectively reviewed the data of 184 pediatric HSCT recipients who had COVID-19 between March 2020 and August 2022. The median time from HSCT to COVID-19 infection was 209.0 days (IQR, 111.7-340.8; range, 0-3845 days). The most common clinical manifestation was fever (58.7%). While most patients (78.8%) had asymptomatic/mild disease, the disease severity was moderate in 9.2% and severe and critical in 4.4% and 7.6%, respectively. The overall mortality was 10.9% (n: 20). Deaths were attributable to COVID-19 in nine (4.9%) patients. Multivariate analysis revealed that lower respiratory tract disease (LRTD) (OR, 23.20, p: .001) and lymphopenia at diagnosis (OR, 5.21, p: .006) were risk factors for ICU admission and that HSCT from a mismatched donor (OR, 54.04, p: .028), multisystem inflammatory syndrome in children (MIS-C) (OR, 31.07, p: .003), and LRTD (OR, 10.11, p: .035) were associated with a higher risk for COVID-19-related mortality. While COVID-19 is mostly asymptomatic or mild in pediatric transplant recipients, it can cause ICU admission in those with LRTD or lymphopenia at diagnosis and may be more fatal in those who are transplanted from a mismatched donor and those who develop MIS-C or LRTD.

Trends in viable microbial bioburden on surfaces within a paediatric bone marrow transplant unit

Despite their role being historically overlooked, environmental surfaces have been shown to play a key role in the transmission of pathogens causative of healthcare-associated infection. To guide infection prevention and control (IPC) interventions and inform clinical risk assessments, more needs to be known about microbial surface bioburdens. To identify the trends in culturable bacterial contamination across communal touch sites over time in a hospital setting. Swab samples were collected over nine weeks from 22 communal touch sites in a paediatric bone marrow transplant unit. Samples were cultured on Columbia blood agar and aerobic colony counts (ACC) per 100cm2 were established for each site. Individual colony morphologies were grouped and identified by matrix-assisted laser desorption/ionization time-of-flight mass spectrometry or 16s rDNA sequencing. Highest mean counts were observed for sites associated with ward management activity and computer devices (3.29 and 2.97 ACC/100cm2 respectively). A nurses' station keyboard had high mean ACC/100cm2 counts (10.67) and diversity, while laundry controls had high mean ACC/100cm2 counts (4.70) and low diversity. Micrococcus luteus was identified in all sampling groups. Clinical staff usage sites were contaminated with similar proportions of skin and environmental flora (52.19-46.59% respectively), but sites associated with parental activities were predominantly contaminated by environmental microflora (86.53%). The trends observed suggest patterns in microbial loading based on site activities, surface types and user groups. Improved understanding of environmental surface contamination could help support results interpretation and IPC interventions, improving patient safety.

Pre-Exposure Prophylaxis and Treatment with Tixagevimab/Cilgavimab for COVID-19 among Immunocompromised Pediatric Patients.

Background: Patients treated with hemato-oncological malignancies (HO) or undergoing cellular therapies such as hematopoietic stem cell transplantation (HSCT) or chimeric antigen receptor T cells (CAR-T) were significantly affected by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). Despite the success of SARS-CoV-2 vaccination, immunocompromised patients remain at increased risk for severe coronavirus disease (COVID-19), rendering this group of population a high priority for additional prevention and treatment options. Tixagevimab and Cilgavimab (TIXA/CILGA, AZD7442, Evusheld®) is a combination of two fully human, long-acting monoclonal antibodies. TIXA/CILGA have been approved as pre-exposure prophylaxis and treatment in patients at risk of severe disease with impaired vaccine response. Our objective was to describe the efficacy and safety among immunocompromised pediatric patients. Methods: This was an observational multicenter cohort study of immunocompromised pediatric patients receiving TIXA/CILGA conducted at nine Polish centers of Pediatric Oncology, Hematology and Bone Marrow Transplantation. We analyzed patients in two groups; those treated with HO and those undergoing cellular therapies: HSCT or CAR-T cells. In addition, two other cohorts were identified: patients given TIXA/CILGA as pre-exposure prophylactic and therapeutic intervention. Results: A total of 78 patients were evaluated during the study period: 69 (88.5%) received TIXA/CILGA as pre-exposure prophylaxis and 9 (11.5%) as a treatment strategy. A total of 52 (66.6%) patients were treated with standard chemotherapy at HO departments; 21 (27%) underwent HSCT, and 5 (6.4%) received CAR-T cell therapy. All children with COVID-19 receiving TIXA/CILGA presented a mild degree of severity. The most common clinical manifestations were fever, cough and coryza. At least one adverse event (AE) was reported in two (3.8%) patients excluding standard injection site reactions. Reported AEs were mild or moderate in intensity. One child reported mild myalgia and one reported moderate bone pain and weakness. Conclusions: In our observational multicenter cohort study, we explored the use of TIXA/CILGA as pre-exposure prophylaxis and treatment for COVID-19 among immunocompromised pediatric patients. While our findings suggest a potential benefit in preventing and managing COVID-19 in this vulnerable population, it is important to note the study's non-comparative design. Our results highlight the need for well-designed clinical trials to confirm these observations and further assess the efficacy and safety of TIXA/CILGA in immunocompromised children.

The Phenotype of a Bone Marrow Transplant Nurse: Survival of the Fittest

There is a need for proficient nursing practice in specialized critical care settings, such as a pediatric Bone Marrow Transplant (BMT) Unit. The staffing crisis continues to be problematic for areas around the country. Nursing tenure has been positively correlated with improving patient outcomes and confidence in giving care, so efforts must be made to retain pediatric BMT nurses. The purpose of this study was to investigate factors of resilience and how it pertains to the retention of pediatric BMT nurses through 1) Understanding current BMT nursing turnover and trend why nurses are leaving; 2) Measure resilience throughout BMT nursing cohort, covering all tenure of nurses; and 3) Interview senior nurses within the cohort to identify themes relating to retention and resilience using grounded theory methodology. This research was carried out in the Bone Marrow Transplant (BMT) Unit at Cincinnati Children's Hospital Medical Center (CCHMC). The study spanned from 2014 to 2019, during which researchers gathered extensive data to evaluate the turnover risk among BMT nurses. A detailed follow-up was conducted in 2019 to further assess this risk. To measure resilience levels, a group of 115 nurses in the unit completed the Connor Davidson Resilience Scale (CD-RISC). Additionally, in-depth interviews were conducted with nine senior nurses in the cohort, continuing until theoretical saturation was reached, ensuring a comprehensive understanding of the factors influencing nurse turnover in the unit. Researchers looked retrospectively at nursing turnover from 2014-2019. Investigators determined the risk of new nurses leaving was between 22 and 24 months (HR 0.025). Further, follow-up was conducted for data points between 2019-present time, which showed a drastic change in the hazard rate curvature. However, the risk remained relatively the same at 22-24 months (HR 0.03). There was no statistical significance found between CD-RISC results and age (p=0.465), gender (p=0.725), working experience (p=0.15), or education (p=0.14). Through a constant comparative process, several themes were identified as positive, negative, and ambiguous contributions to the retention of nurses. The risk of nurses leaving after two years decreases significantly; therefore, we determined that a nurse with a commitment to pediatric BMT occurs with two years' experience. Although our initial hypothesis was that senior nurses had greater resilience than less tenured nurses, there was no statistical significance as the effect of resilience is small. However, we identified several additional factors pertinent to the pediatric BMT field which may be associated with nursing retention.

Outcome and late effects of matched related peripheral-blood stem-cell transplantation in children with severe aplastic anemia

Background Aplastic Anemia (AA) is a life- threatening disease. Cure can be achieved in a large proportion of patients with prompt treatment with matched sibling donor Hematopoietic Stem Cell Transplantation (HSCT) as first line therapy. Aim Study of the outcomes and late effects among children with severe aplastic anemia after receiving matched related peripheral-blood stem-cell (PBSC) transplantation and cyclophosphamide/antithymocyte globulin conditioning regimen at Ain Shams University Pediatric Bone Marrow Transplantation Unit, Cairo, Egypt. Methods Data of 15 patients with severe aplastic anemia who received matched related PBSC transplantation between January 2011 and December 2018 were analysed retrospectively. All long-term survivors were followed up for 2 years. Follow up studies included detailed assessment of growth, graft evaluation, immune reconstitution, long-term effects on the liver, kidney, pulmonary, cardiac, endocrine functions, chronic graft-versus-host disease (GVHD), and infectious complications. Results The median duration of the study was 87 months (43–115 months). The overall survival was 73.3%. Event-free survival was 66.7%. All mortality was within 2 months posttransplant. Blood counts were within normal limits. Chimerism was complete for all except one. Growth assessment revealed Z score of weight, height, and BMI 0 (−1.8–1.6), −0.7 (−1.8–0.95), and 0.34 (−1.29–2). Abnormal thyroid function was found in three (27%) patients. No documented high-grade acute GVHD or extensive chronic GVHD. Cardiac, pulmonary, hepatic, and renal functions showed no abnormality. Immune reconstitution was consistent, and no malignant complications were reported. Conclusion This study confirmed the feasibility and safety of matched related PBSC transplantation in the pediatric population in low-income countries. Overall survival and event-free survival were satisfactory.

Assessment of Risk Factors for Developing Iron Overload in Patients with Pediatric Cancer and Bone Marrow Transplant Survivors: A Single Center Retrospective Chart Review

Background: Pediatric cancer survivors and those with chronic hematologic disorders are at risk for iron overload secondary to frequent transfusion therapy. As this patient population increases in number due to improvement of therapies, it is essential to identify risk factors associated with the development of iron overload. Classifying these risk factors will help improve screening methods and assist in decreasing side effects from iron overload, such as iron deposition resulting in organ damage. Objective: Assessment of risk factors associated with the development of iron overload in pediatric cancer survivors and those who underwent bone marrow transplant. Methods: Retrospective chart review of patients who completed therapy for pediatric malignancy or underwent bone marrow transplant and were screened for iron overload utilizing magnetic resonance imaging (MRI). Iron overload was defined as T2* liver MRI with iron levels of >5mg/g dry weight. Individuals determined to screen positive for iron overload were evaluated for additional demographic and treatment variables, such as race, age at diagnosis, and total transfused blood volume, to assess for risk factors associated with the development of iron overload. Statistical analyses were performed via univariate and multivariate regression models of the variables collected. Results: We report a large cohort of patients with a mean age of 10.6 years who underwent screening T2* liver MRIs. Of 71 patients who underwent liver MRI screening, 89% met criteria for moderately severe iron overload. Blood volume administered (ml) and Non-White Race were found to be statistically significant in association with higher iron content. Mean iron content was 12.9 mg/g in the Non-White group versus 8.8 mg/g in the White group (p=0.0185). For every 100ml/kg increase in the cumulative blood volume transfused, the predicted iron content increased approximately 1 mg/g (p=0.0227); mean transfused blood volume was 340ml/kg among those who met criteria for iron overload versus 224ml/kg among those who did not (p=0.1858). We did not find any correlation between iron content and age at diagnosis, primary diagnosis, BMI at diagnosis, or gender. Conclusions: Iron overload continues to be a risk for pediatric cancer survivors and those who receive chronic transfusions. Our study revealed a statistically significant correlation between increased amount of blood transfusions and development of iron overload; however, no specific quantity of cumulative transfused blood volume was determined to be the threshold for risk. Our study also revealed a statistically significant correlation between Non-White race and the development of iron overload independent of cumulative transfused blood volume. It is possible there are race-based differences in iron metabolism that result in this increased risk. Further studies regarding specific quantities of blood transfusions, assessing physiologic characteristics of the Non-White patient population, and development of iron overload are needed. These findings indicate the importance of continuing to research the development of iron overload in the pediatric cancer and bone marrow transplant population, as well as provide guidance for understanding the risk each unique patient has for developing this complication.

A Novel, Closed-Line Mini Extracorporeal Photopheresis (ECP) Technique Using the Therakos TM CellEx TM Photopheresis System, Developed for Patients with Contraindications Against Conventional ECP Therapy

Introduction: Conventional ECP has proven efficacy for treating several diseases, most significantly steroid-refractory chronic GVHD. Unfortunately, body weight and sufficient venous access limit the availability of this treatment modality. Children, in particular, usually have limited access to ECP. A simplified mini buffy coat ECP technique that allows treating small children and patients with apheresis contraindications has been tested in selected cases during the last 20 years. However, this method relies on an open-line system that requires a longer time and a cell processing laboratory, making this approach challenging. A new -closed-line- method, using the Therakos TM CellEx TM Photopheresis System, has been developed and evaluated in our Center. This method requires no processing laboratory or other special equipment and takes only 60-120 minutes, making the procedure potentially usable in routine practice. Design and methods: A 7-year-old girl had been treated at our Pediatric Bone Marrow Transplantation Unit with grade IV chronic steroid-refractory GVHD (skin, and less severe liver involvement). As all other options were exhausted, ECP became a desirable form of treatment. However, the patient's low body weight and serious venous access problems hindered conventional ECP treatment. We developed a solution that requires 200-250 ml whole blood collection from the patient, takes less than 2 hours, does not need special cell processing equipment, and allows the ECP treatment to commence in the form of a regular autotransfusion. Consent forms were obtained from the patient's caregivers as well as from the local Ethical Committe. The patient's age at the start of treatment was seven years, and her body weight was 28 kg. A total of 17 mini-ECPs were performed weekly up until the submission of this abstract, and the number of treatments per patient was 33. The duration of mini-ECP treatment has been five months. 5 to 8 mL/kg of whole blood was drawn from the patient. Separation of the white blood cell (WBC) rich buffy coat of the patient's blood - with donor blood product supplementation as needed - was performed through an individualized use of the CellEx TM photopheresis kit and blood prime program. The buffy coat was ultraviolet A (UVA)-irradiated after adding 8-methoxypsoralen (8-MOP) within the Therakos TM CellEx TM Photopheresis System and divided into two fractions under a laminar air flow cabinet. The autologous residual blood with the first fraction of the UVA irradiated buffy coat was transfused immediately. The second buffy coat fraction was stored at 4 °C and transfused the following morning through a peripheral vein. Results : The median volume of the collected whole blood was 250 mL (203-250 mL). The median volume was 72 ml (48-88 mL) for the first fraction of the irradiated buffy coat and 71 ml for the second fraction (48- 88 mL). Apoptosis and cell death were analyzed by annexin V staining; apoptosis and lymphocyte proliferation was measured by flow cytometry. UVA irradiation of the 8-MOP infused buffy coat resulted in significant induction of WBC apoptosis at 24 hours. No clinical side effects were observed during mini ECP treatment; all microbiological control tests were negative. Partial response was observed within three months (skin), and the tapering of systemic immunosuppression (methylprednisolone and ruxolitinib) began at five months. Conclusion: Mini whole blood ECP induces apoptosis and lymphocyte proliferation inhibition, both occurring after standard ECP. The closed-line method requires no special equipment and is not demanding for the patient, providing a viable solution to the technical difficulties children with the need for ECP treatment face.

Quality of life in parents of Turkish and Syrian pediatric bone marrow transplant and oncology patients.

The success of the treatment depends on the well-being of the parents in pediatric cancer patients. In addition, migrants need to deal with the consequences of war. Our purpose was to compare the HRQoL of parents of Syrian and Turkish children followed in our bone marrow transplant and oncology units. One hundred SF 36 questionnaires, fifty from each group were collected between October 2019 and March 2020 in this cross-sectional study. Demographic characteristics of the patients (sex, age, cancer type, presence of relapse, duration of follow-up) and education level of parents were recorded. Analyses were performed using IBM SPSS Statistics version 20.0. Turkish parents reported better results in physical functioning, emotional well-being, physical pain, general health, and physical health composite score (P < 0.05). There were more fathers in the Syrian group and education status was significant between the two groups (P = 0.01, P = 0.001, respectively). There was no correlation between QoL and education levels in the Turkish group. In Syrian parents, pain score was high in middle school graduates (P = 0.043). In the presence of relapse, although the physical function score decreased, the physical role function score increased in Turkish parents (P = 0.0035, P = 0.005, respectively). Syrian parents' emotional role function and energy/fatigue score were both increased when children had relapses (P = 0.027for both). Migration is a complex issue. Turkish parents were better in physical functioning, emotional well-being, physical pain, general health, and physical health composite score. Screening for parental burden, assisting them to find appropriate support services is essential in improving the health and QoL of both our patients and their parents.

Assessment of Governmental-Funded International Referral Program to Treat Patients

In Iraq, international referral programs have been adopted, governed by specific inclusion measures. Basra government has adopted an international referral program as a solution to local medical problems, which targeted several countries, mainly Turkey. Since, there has been no party evaluated this program, this study was conducted to assess an international medical referral program. To conduct the study, the assessors used the database at Basra Government offices to obtain the relevant information to be used as a reference to improve the program in the future. The total number of patients was 833, 53% of them were males and 59.42% were children. After re-assessment of medical conditions of the referred patients, 94.8% of the patients were managed successfully, 2.1% of them did not need surgery, in 1.1% the disease stage was inoperable, 0.6% were not fit from health point of view to be operated on, and 0.4% died. Comparative assessments of the program with similar programs implemented in other countries and in Iraq need to be conducted. The assessment showed a high treatment success rate among the referred patients, which can arrow to that there were some gaps in the Iraqi health system in the fields of pediatric CVS diseases, bone marrow and liver transplant and ophthalmology should be fixed. These gaps include shortage in qualified resources in Iraq.

Cardiac iron overload in pediatric oncology and bone marrow transplant survivors.

e22022 Background: Cardiovascular late effects are a leading cause of mortality and morbidity in childhood cancer and Bone Marrow Transplant (BMT) survivors. In patients receiving chronic red blood cell transfusions, excess iron may be deposited in the liver, heart, and pituitary gland. Iron overload cardiomyopathy is characterized as a restrictive physiology with early diastolic dysfunction ultimately progressing to end-stage dilated cardiomyopathy. Iron loading in the heart can also lead to arrhythmias and increase myocardial ischemia reperfusion injury due to increased formation of reactive oxygen species. It is not yet known whether high transfused packed red blood cell (PRBC) volumes in cancer and BMT patients leads to cardiac iron deposition, and whether iron-induced cardiac injury contributes to the known cardiovascular late effects in these patients. Methods: 83 childhood cancer and BMT survivors with elevated serum ferritin levels and subsequent liver Magnetic Resonance (MR) imaging were identified. 18 patients (21.7%) who had liver iron content of &gt; 12mg/g (mean 16.0 ± 7.36 mg/g) also underwent Cardiac MRI (CMR) to determine cardiac iron content. Results: Median transfused blood volume was 393 ml/kg [95% Confidence Interval (CI) 170-780 ml/kg]. All 18 patients had at least one echocardiogram showing normal ventricular ejection fractions [mean 65.1 ± Standard Deviation (SD) 3.53%] and normal shortening fractions (mean 35.4 ± 3.67%). All patients had myocardial iron content within normal ranges (T2* relaxation time mean 35.1 msec ± 7.1 [normal &gt;20 msec], mean ± SD iron content 0.63± 0.2 mg/g dry weight, range 0.5-1.1). There was no association of cardiac iron content with transfused blood volume, anthracycline dose (mg/m2) or liver iron content (Pearson Correlation Coefficients 0.27, 0.29 and 0.89 respectively). Conclusions: Childhood cancer and BMT patients receive multiple PRBC transfusions to treat symptomatic anemia. It is currently unknown whether excess iron deposition in the heart contributes to the cardiac effects of impaired diastolic function and ultimately systolic dysfunction. In this study, 11 of 18 patients (61.1%) with high levels of liver iron had at least 1 other risk factor for the development of future cardiac dysfunction (either anthracycline exposure, radiation to the chest or total body irradiation or both). Larger studies are needed to determine whether oncology patients with high levels of iron deposition in their liver are also at risk for clinically significant cardiac iron loading, and what risk factors may exist for elevated myocardial iron content.

5613274 “A PICTURE IS OFTEN WORTH MORE THAN MANY WORDS: STREET ART AS EMPOWERMENT FOR SICKLE CELL DISEASE PATIENTS” JACKELINE ELIZABETH MARAN1, MARINA PERDIBON1, ILARIA BAIDO1, MICHEL NKONGNE GYSLENE2,FABRIZIO CANONICI2, VANIA MUNARETTO3-4, GIULIA REGGIANI3-4, LAURA SAINATI4, ALESSANDRA BIFFI3-4, RAFFAELLA C

Background: Sickle Cell Disease is a genetic and chronic disease which carries a great burden of recurrent symptoms, acute and chronic complications and stigma therefore greatly impacting quality of life and self-perception. Teenagers and young adults often do not want to talk about their disease or do not know how to communicate their experience to others. “Blood Artists”, was a multistakeholder campaign conducted in Italy (a collaboration between Universities, Hospitals, Pharmaceutical Companies, Patients’ Organizations) to raise awareness on Sickle Cell Disease and Thalassemia. Aims: Its main objective was to make people understand, through the direct language of street art, what it means to live Sickle Cell Disease or Thalassemia, two hereditary blood disorders that afflict thousands of people in Italy. The goal was to raise awareness among the public, especially young people, to make them understand how the disease impacts life and that some simple gestures, like blood donation, can help treatment. The street art initiative involved several italian cities including Padua. Methods: Several meeting with representatives of the Sickle Cell Disease Association (Associazione Malattia Drepanocitica) and the artis were organized to communicate the patients’ experience in living with SCD. Following the testimony of each patient, a draft of the painting was produced and then shared with the patients who commented and added their further contribution. Results: The Artist SteReal donated a wall painting- Street Art painting-, on the wall facing a very busy street of the Pediatric Hematology Oncology and Bone Marrow Transplant Unit of the Azienda-Hospital University of Padua, Veneto Region Reference Center for the Diagnosis, Care and Treatment of SCD in Childhood and ERN-EuroBloodNet Center of Expertiese. In the Street Art (Figure 1) the 3 individuals with SCD expressed what they thought were the most significant and common aspects of SCD: 1)The mural depicts the image of a mother and daughter playing together, and symbolizes’ the genetic transmission of the disease but also the importance of the family bonds in the patients’ lives and in disease coping; 2) the lightning which arrives suddenly and symbolizes the vaso-occlusive crisis pain symptom that disrupt everyday life; 3) the petals of the daisy that, flying in the wind take the Sickle shape of red blood cells, to represent the disease from a clinical and scientific point of view, but also the renaissance of life that can flourish in spite of the disease. Conclusions: In conclusion, the Street Art initiative represents an important means of making the public aware of people’s experience with their disease and the same time encourages patients to talk about it freely without being ashamed or afraid. The wall painting was used in several meeting with patients as a starting point for sharing experiences and discussion.Figure 1:: The Street Art created through the collaboration between the Artis SteReal and the Associazione Malattia Drepanocitica (Sickle Cell Disease Association), in Padova, Italy on the wall of the Pediatric Hematology Oncology and Bone Marrow Transplant Unit in March 2022.

5613386 CELEBRATING THE SICKLE CELL DISEASE WORLD DAY ON JUNE 19TH AFTER TWO YEARS OF PANDEMIC: MY LIFE WITH SCD, PATIENTS’ PERSPECTIVE

Jackeline Elizabeth Maran1, Marina Perdibon1, Ilaria Baido1, Michel Nkongne Gyslene2, Fabrizio Canonici2, Vania Munaretto3-4, Giulia Reggiani3-4, Alessandra Biffi3-4, Laura Sainati4, Raffaella Colombatti3-4 1Fondazione Città della Speranza Onlus 2Associazione Malattia Drepanocitica 3Dipartimento della Salute della Donna e del Bambino, Università degli Studi di Padova Background: After two years of COVID in which activities were reduced due to the pandemic and each one’s life was affected by restrictions and limitations, the Sickle Cell Disease (SCD) Association in Padova teamed up with the Sickle Cell Group at the Pediatric Hematology Oncology and Bone Marrow Transplant Unit to celebrate the Sickle Cell Disease world day by organizing an online meeting with children/youths and their families. Theme of the meeting was: “My Life with SCD: poems, pictures and writings express our view on disease and care”. Aims: One of the goals of this meeting was to create an opportunity for individuals with SCD to meet and have a constructive discussion with each other about the disease and express their feelings after two years of pandemic. Methods: One month before the meeting children, teenager and parents were asked to sharer with the organizing team any drawing, painting, poem, writing, that they felt could express their feelings or experience of the disease itself or how it affected their life, or their experience in the hospital. The materials received were organized in a power point presentation and At the meeting, families were able to see a PowerPoint presentation with the poems, drawings, writings. Each author had the choice to personally share their production or have it read out loud by a member of the team. Free time to comment or share experiences was given. Results: 20 children, teenagers and parents participated. Countries of origin (Nigeria, Ghana, Congo, Albania, Italy), religious background (catholic, muslim, no religion, other) were different as well as disease genotype (HbSS, HbSC, HbSB°), severity or treatment received (Hydroxyurea, transfusion, Hematopoietic stem cell transplantation -HSCT, none). Drawings and writings regarded experience with the disease (mechanism of action, admissions), feelings experienced (fear, hope, light at the end of the tunnel), aspirations (sports) and gratitude (to the social and medical team, to parents) (Figure 1). Surprisingly, families who had a child having undergone HSCT, reported on the need and importance to talk about this experience for years after the event and made a request of a support goup. Finally, all families underlined the need to meet again soon to discuss together issues related to personal experience with SCD, even via web. of discussion with each other and with the drepanocytosis group; and that throug the online telematics platform it is still possible to involve all families, listening and trying to comfort them on doubts and perplexities about the disease, In conclusion, it can be said that after two years of pandemic, in our setting, online meeting can help patients and families reconnect with each other and activities can be planned to aid experiences and feelings. Patients’ associations and Health Care Teams can collaborate in this area.Figure 1:: Poems and drawings on “My Life with Sickle Cell Disease” by children, teenagers and parents from the Associazione Malattia Drepanocitica

Complications and outcomes of gastrostomy versus nasogastric tube feeding in paediatric allogeneic bone marrow transplant: A prospective cohort study

The intensive conditioning regimens administered during bone marrow transplant (BMT) frequently cause mucositis, gastrointestinal toxicity and reduced oral intake. Children are consequently at risk of malnutrition. First-line nutrition support is recommended as enteral nutrition (EN). Nasogastric tube (NGT) is the mainstay for administration. Gastrostomies provide an alternative, but there is limited evidence of their efficacy and safety in paediatric BMT. This study aimed to compare enteral tube complications and nutritional and clinical outcomes between children with a gastrostomy versus NGT throughout BMT. A prospective cohort study was conducted at a single centre in the United Kingdom. During pre-admission consultations families were offered choice of a prophylactic gastrostomy or NGT. Children undergoing allogeneic BMT were recruited from April 2021 to April 2022. Data compared between children with either tube included: tube complications, change in weight, body mass index and mid-upper-arm circumference, calorie, protein and fluid intake, timing and use of EN and parenteral nutrition, survival, graft-versus-host disease and length of admission. Following BMT, data were collected weekly for the first six weeks from electronic records, monthly thereafter from 3-day averaged food diaries and clinic assessments, until six months post-BMT. Nineteen children with NGT were compared to 24 with a gastrostomy. Of gastrostomy complications, 94.2% (129/137) were minor, mechanical issues being most common (80/137). Dislodgement comprised 80.2% (109/136) of NGT complications. No significant differences were seen between tubes on nutritional, anthropometric and clinical outcomes. Gastrostomies were popular with families, relatively safe, associated with mostly minor complications and similarly effective as NGTs in supporting children's nutritional intake and status. Where an NGT may not be tolerated, a prophylactic gastrostomy could be considered. Placement of either tube requires balancing their risks, benefits, the child's nutritional status, conditioning, expected duration of EN and family preferences.

Impact of percutaneous endoscopic gastrostomy on pediatric bone marrow transplantation outcomes: Retrospectice single-center cohort study.

Parenteral nutrition is commonly used to ensure nutrition support and prevent the harmful effects of malnutrition, which frequently occurs after allogeneic hematopoietic stem cell transplantation (aHSCT). Nevertheless, enteral nutrition supports the restoration of the gut barrier and microbiome as well as protects against infectious complications and acute graft-vs-host disease. Percutaneous endoscopic gastrostomy (PEG) may also be beneficial for gastric decompression and drug administration. We performed a retrospective cohort study to evaluate the impact of PEG on treatment outcomes in 75 children who underwent aHSCT with (n = 34) or without (n = 41) PEG from 2005 to 2016. In 34 patients, PEG was used to ensure enteral nutrition support (n = 30), oral drug intake (n = 28), and abdominal decompression (n = 2). During the study period, we observed a beneficial association between PEG placement and transplant-related mortality as well as 5-year overall survival compared with the non-PEG group (12.9% vs 59.0%, P = 0.000; 85.3% vs 35.1%, P = 0.000, respectively). The beneficial impact of PEG was most prominent on 5-year overall survival in older children (12-17 years) with grafts from matched unrelated donors. PEG placement had a positive association with transplant outcomes in pediatric patients undergoing aHSCT. To confirm these results, larger prospective studies are needed.

176 - Outcomes of Pediatric Bone Marrow Transplant Patients Receiving Palifermin with Melphalan-Based Conditioning Regimens

176 - Outcomes of Pediatric Bone Marrow Transplant Patients Receiving Palifermin with Melphalan-Based Conditioning Regimens

486 - Demographics and Treatment-Related Factors Influence PTSD Symptom Development in Pediatric Bone Marrow Transplant Recipients

486 - Demographics and Treatment-Related Factors Influence PTSD Symptom Development in Pediatric Bone Marrow Transplant Recipients

The Determining Factors for Outcome of Pediatric Intensive Care Admitted Children After Stem Cell Transplantation.

Requiring pediatric intensive care unit (PICU) admission relates to high mortality and morbidity in patients who received hematopoietic stem cell transplantation (HSCT). In this study, we aimed to evaluate the indications for PICU admission, treatments, and the determining risk factors for morbidity and mortality in patients who had allogeneic HSCT from various donors. In this retrospective study, we enrolled to patients who required the PICU after receiving allogeneic HSCT at our Pediatric Bone Marrow Transplantation Unit between 2005 and 2020. We evaluated to indication to PICU admission, applications, mortality rate, and the determining factors to outcomes. Thirty-three (7%) patients had 47 PICU admissions and 471 patients underwent bone marrow transplantation during 16-year study period. Also, 14 repeated episodes were registered in 9 different patients. The median age of PICU admitted patients was 4 (0.3 to 18) years and 29 (62%) were male. The main reasons for PICU admission were a respiratory failure, sepsis, and neurological event in 20, 8, and 7 patients, respectively. The average length of PICU stay was 14.5 (1 to 80) days, 14 (43%) of patients survived and the mortality rate was 57%. Multiple organ failure ( P =0.001), need for respiratory support ( P =0.007), inotrope agents ( P =0.001), and renal replacement therapy ( P =0.013) were found as significant risk factors for mortality. Allogeneic HSCT recipients need PICU admission because of its related different life-threatening complications. But there is a good chance of survival with quality PICU care and different advanced organ support methods.

1857. Risk factors for recurrent bacteremia in children undergoing chemotherapy or hematopoietic stem cell transplantation

Abstract Background Sepsis is a complication frequently encountered in children with underlying malignancies, especially due to a majority of patients having indwelling venous catheters. Indications for catheter removal among children with central-line associated blood stream infection (CLABSI) should follow the recommendations for adults, however, difficulties in vascular access often leads to attempting treatment without catheter removal. Therefore, the primary aim of this study was to find risk factors for recurrent sepsis in children undergoing chemotherapy of HSCT and examine whether more aggressive catheter removal after CLABSI in children is necessary. Methods In the Pediatric Bone Marrow Transplant Center of Seoul St. Mary’s Hospital, positive blood cultures were prospectively monitored to control and prevent outbreaks. The date of culture, culture results, symptoms presented, category of blood stream infections (by the CDC/NHSN surveillance definition (2021) of Bloodstream infections), and central-line associated blood stream infection (CLABSI) events were monitored. Results During September 2016 to February 2021, a total 280 cases of laboratory confirmed bloodstream infections (LCBI) or Mucosal Barrier Injury LCBI (MBI-LCBI) were diagnosed in children &amp;lt; 18 years old with underlying malignancies. Of these, 52.9% (n=148) were male, and the mean age was 9.7 (SD±6.1) years old. CLABSI was diagnosed in 51.8% (n=145), and the most common pathogens cultured were S. mitis/oralis (24.0%, n=67), E. coli (15.4%, n=43), and coagulase negative Staphylococci (CNS) (10.4%, n=29). Recurrent sepsis occured in 17.1% (n=48), and 9.6% (n=27) had two indwelling catheters . Multivariable analysis showed that factors associated with recurrent blood stream infections were as follows: duration of indwelling catheter (OR, 1.002; 95% CI, 1.001-1.004; P&amp;lt; 0.001) and no removal of central lines after previous episode (OR, 51.143; 95% CI, 6.6-395.0; P&amp;lt; 0.001). Conclusion Permanent central lines should be removed as soon as possible, and more aggressive approach in permanent catheter removal after LCBSIs in children is necessary to reduce recurrent infections. Disclosures All Authors: No reported disclosures.

1860. Streptococcus mitis/oralis sepsis in patients with neutropenia after chemotherapy

Abstract Background Streptococci are the dominant species in the human oral cavity and upper respiratory tract. Oral mucositis affects 30–40% of the patients receiving chemotherapy and 80% undergoing HSCT. During episode of oral mucositis, patients are at a risk for invasive infections caused by opportunistic pathogens in the oral cavity. The purpose of this study was to investigate the clinical characteristics of S. mitis/oralis invasive infection, and find patients at high risk for with S. mitis/oralis infection in order to prevention of S. mitis/oralis bacteremia. Methods Patients receiving chemotherapy or stem cell transplant for malignancies and primary immunodeficiencies at the pediatric bone marrow transplant center of Seoul St. Mary's hospital admitted during January 2017 to December 2020 for blood stream infections were included in this study. Chart review of the patients were done retrospectively. Results During the four year study period, there were 4,647 admissions, and 2,358 (50.7%) experienced at least 1 episode of fever. Overall, the incidence of bacteremia was 10.9% of all febrile children. The most common pathogen causing bacteremia was S. mitis/oralis (24%), followed by E. coli (14.3%), Klebsiella spp. (10.5%), and S. epidermidis (7.4%). Candida sepsis occurred in 3.5% of the children. By year, the proportion of bacteremia caused by S. mitis/oralis was 19% in 2017, 21% in 2018, 41% in 2019, and 25% in 2020. 51% of S. mitis/oralis bacteremia occurred 8-14 days after day 0 of chemotherapy. A multivariate analyses on risk factors for S. mitis/oralis bacteremia was the underlying disease AML (OR, 4.6; 95% CI, 2.4-8.7; P&amp;lt; 0.001), and the use of cytarabine (OR, 4.5; 95% CI, 2.4-8.5; P&amp;lt; 0.001). Compared to ALL, AML has a HR 3.7 (95% CI 1.9-7.2) of bacteremia with S. mitis/oralis. Prolonged duration of fever ( &amp;gt;7 days) was observed in 13.4% of the patients, and a fever duration &amp;gt;14 days was observed in 2 patients. Complications such as septic shock, n=4 (6.0%), ARDS, n=1 (1.5%), infective endocarditis, n=2 (3.0%) were observed. Conclusion S. mitis/oralis sepsis causes significant morbidity in patients undergoing treatment for malignancies. Patients with AML are at the highest risk, especially after induction or consolidation chemotherapy including cytarabine. Disclosures All Authors: No reported disclosures.

Feasibility & Acceptability of Patient and Family Directed Active Music Making during Pediatric Bone Marrow Transplant Process.

(1) Background: Bone marrow transplant (BMT) is an aggressive and complex medical treatment for children with certain types of cancer and other diseases. The transplant process entails replacing the patient's diseased bone marrow with the healthy marrow of a donor. During the course of treatment, patients are isolated in their room to reduce the risk of infection. Patient's experience a variety of symptoms and side effects during the process including nausea, vomiting, diarrhea, mouth sores, fatigue, pain, discomfort, extreme muscle weakness, and emotional distress. Children often need a parent or caregiver present with them at all times throughout treatment. This process can cause significant stress and anxiety for the patient and their family. (2) Methods: This study explored the feasibility and acceptability of a patient and family directed active music making protocol during the BMT process. Ten patients, their parents, and family members participated in the study during the course of the BMT. (3) Results: Participants reported engaging in active music making 3-4 times per week and completed 121 journal forms reporting their music making experiences. They indicated using active music making to manage pain, discomfort, stress, anxiety, and boredom, foster relaxation and sleep, for enjoyment, and as a way to connect. (4) Conclusions: Patients, parents and family members reported feeling a sense of empowerment when engaged in making music to support their child or loved one.