Children Undergoing Bone Marrow Transplantation Research Articles

One Tube Does Not Fit All: Parent Experiences and Decision-Making for Choosing a Nasogastric Tube or Gastrostomy for Their Child During Allogeneic Bone Marrow Transplant

Background: Children undergoing bone marrow transplant (BMT) are at risk of developing malnutrition. A feeding tube becomes a requirement for most children to meet their nutritional and medication requirements. Two tubes are typically used: nasogastric tube (NGT) or gastrostomy. At the UK center where this study took place, parents are offered a choice between these tubes. Objective: This qualitative data collection in a mixed methods study explored why parents choose either tube and their experiences of using it. Methods: Parents participated in 2 semistructured interviews. First, on admission to explore why they chose either tube. Second, 1–2 months postdischarge to explore their experience of using the tube. Interviews took place over 18 months. Transcripts were thematically analyzed. Results: Sixteen parents whose child had an NGT, 17 a gastrostomy, were interviewed. Choice was experienced across a continuum of difficulty and freedom. Many parents deferred to the expertise of professionals; others felt they were the experts in their child. Influential factors in decision-making included expected duration of need, the child’s age and activity, cosmetic differences, balancing gastrostomy surgery against NGT dislodgement, lay advice, healthcare professionals’ recommendations and prior tube feeding experiences. Conclusions: Parents valued choice appreciating 1 feeding tube might not suit every child. Implications for Practice: Choice of a gastrostomy or NGT should be offered to children prior to BMT. What is Foundational: Parents navigate a complex decision-making process when choosing a feeding tube for their child. Healthcare professionals can facilitate informed decision-making through collaborative discussions, inclusion of peer support, and provision of balanced information.

Kemik İliği Transplantasyonu Yapılan Çocukların Annelerinin Algıladıkları Eş Desteği ve Stres Düzeyleri Arasındaki İlişkinin Belirlenmesi

Bu araştırma Kemik İliği Transplantasyonu (KİT) uygulanan çocukların annelerinin algıladıkları eş desteği ile stres düzeyleri arasındaki ilişkinin belirlenmesi amacıyla gerçekleştirilmiştir. Araştırmanın örneklemini KİT ünitesinde tedavisi devam eden 150 çocuğun annesi oluşturdu. Veriler “Veri Toplama Formu”, “Eş Destek Ölçeği” ve “Algılanan Stres Ölçeği” kullanılarak toplandı. Verilerin değerlendirilmesinde Ki-kare, Kolmogorov-Smirnov testi, Independent t-testi, ve One Way ANOVA, korelasyon ve regresyon analizleri kullanılmıştır. Annelerin öğrenim, çalışma ve sosyoekonomik durumu, çocuk sayısı, yaşanılan yer, aile yapısı, akraba evliliği, sosyal güvence, kronik hastalık varlığı, aile üyesinde kronik hastalık olma değişkenleri eş desteği için; KİT sonrası komplikasyon yaşama ve KİT yapılma sayısı stres düzeyi için yordayıcı bulundu. Araştırmanın sonucunda eş desteği arttıkça annelerin stres düzeylerinin artmaya devam ettiği ve eş desteği ile annelerin baş etmelerinin güçlendiği belirlendi.

Replacement Versus Same-Site Salvage Using Hickman Catheter for Pediatric Stem Cell Transplantation Patients: A Comparative Study

Children undergoing bone marrow transplant need a double-lumen Hickman line. Therefore, changing Port-a-Cath ports to double-lumen Hickman catheter is mandatory. Several methods were described for changing Port-a-Cath ports either through the same-site or a new placement access site. The advantage of one method over the other is still debatable. We conducted this study to compare the safety and effectiveness of replacement versus salvage techniques to change ports to the Hickman lines before bone marrow transplants in pediatric patients. We included 85 pediatric patients who underwent stem cell transplants. Their age ranged from 0.2 to 15 years. According to the Hickman reinsertion technique, we classified the patients into 2 groups; the Replacement group (n = 47) and the Same-site salvage group (n = 38). We compared the data before and after Hickman insertion between both groups. Study outcomes were the catheter duration, its complications, and mortality. The mean age of all patients was 4.7 ± 3.9 years, and 65.9% were males. There was no difference in the baseline data between both groups. During Port-a- Cath first insertion; 16.5% of patients suffered complications, with 10.6% had conversion to cut down, 1.2% had a hematoma, and 4.7% had multiple site insertion. We did not report differences between groups in the complications of the first port insertion. The Hickman duration was longer in the replacement group (4 (Q1-Q2: 2-6) vs. 1 (0.5-3) months, P = 0.005). Increased age (odds ratio [OR]: 1.31, P = 0.001) and male gender (OR: 1.19, P = 0.046) were independent predictors of mortality. Endovascular same-site salvage technique could help preserve vascular access during the tunnel catheter exchange for noninfectious reasons. We recommend the use of the same-site salvage technique in pediatric transplant patients.

Epidemiology and outcome for viremia in children undergoing bone marrow transplant: A retrospective cohort study.

Viral infections pose a serious risk for children undergoing hematopoietic stem cell transplant (HSCT). There are few published case series of prevalence, risk factors, and outcomes examining multiple viruses simultaneously, and no pediatric Australasian data published to date. We describe the real-life experience of viremia in pediatric HSCT in a single tertiary center. All episodes of viremia in children undergoing HSCT between 2000 and 2018 were identified by matching HSCT patients' unique identification numbers with positive blood polymerase chain reaction (PCR) results for human adenovirus (HAdV), cytomegalovirus (CMV), Epstein-Barr virus (EBV), and human herpes virus 6 (HHV-6). Paper or electronic charts and electronic pathology results were used to extract the study variables. Viremia was detected in 177/445 (39.8%) HSCT episodes, of which 46% were allogeneic and 19% autologous transplants. Viremia was disseminated in 96 (21.6%) episodes, with 80 (18%) having more than one virus. HAdV was detected in 108 (24.3% of total episodes) and frequently in autologous transplants, CMV in 71 (16.0%), EBV in 60 (13.5%), and HHV-6 in 38 (8.5%). Of 174 children, 19 (10.9%) died of a viral-associated cause, with viral mortality highest in CMV (18.3%), lowest in HHV-6 (2.6%) with HAdV and EBV similar (6.6% and 6.7%). Adenoviral (but not other virus) dissemination was significantly associated with lower lymphocyte count at time of first detection. CMV dissemination and death were significantly associated with initial and highest CMV viral loads (copies/mL). This study presents the first pediatric-specific Australasian data for viremia in HSCT. Findings may help guide clinicians in prophylaxis and treatment decisions.

Impact of childhood cancer on the family: evidence from Bangladesh

BackgroundThe care of children with cancer creates emotional, financial, and social impacts for their families. Information on the impact of childhood cancer (CC) on the family is scarce in Bangladesh. Thus, the study was set out to assess the impact of CC on the families in the local context. MethodsThis cross-sectional study was conducted from January to June 2018 in three purposively selected tertiary hospitals. All the children diagnosed and treated at those hospitals during the study period were eligible for this study. Children undergoing bone marrow transplantation, or those who were seriously ill, or those transferred to another hospital, or those who died were excluded or whose parents were not willing to participate. A total of 242 children were enrolled in the study and their parents were included in the interview. Measures included socio-demographic attributes, financial burden, personal strain, social impact, mastery, and treatment cost. Informed written consent was obtained from the parents and a face-to-face interview was conducted using a semi-structured questionnaire based on (i) About you and your-family and (ii) the Impact-On-Family (IOF) scale. High scores of the scale correlated to high impact. Medical records were reviewed to collect data on the pattern of CC and treatment costs. ResultsMajor CCs included leukemia (36.0%), blastoma (18.2%), sarcoma (14.9%), and lymphoma (12.4%). The weighted score was highest for mastery (3.63) followed by financial burden (3.33), personal strain (3.27), and social impact (3.21) domains. The difference of IOFS score was significant by family type (p < 0.05), father's occupation (p < 0.05), type (p < 0.01) and duration of cancer (p < 0.01), and treatment cost (p˂0.01). Families adopted diverse coping strategies including changed lifestyle (98.3%), sought social support (86.0%), rely more on religion (98.8%), and reduced family investment (83.9%) to adjust the impact. ConclusionThe impact of CC on the family is evident at many levels. In particular, mean scores of financial burden, personal strain, social impact, and mastery domains of the IOF scale were significantly associated with the employment status of parents, residing place, treatment cost, type, and duration of cancer. The study findings could contribute to devising impact-reducing intervention programs in Bangladesh.

Insertion of central venous catheters in children undergoing bone marrow transplantation: is there a platelet level for a safe procedure?

BackgroundBone marrow transplantation (BMT) is a therapeutic procedure for the management of several hematological diseases and malignancies in pediatric population. Central venous catheters (CVCs) play a pivotal role during the process of BMT. The aim of this study was to compare the complications of CVCs placements in children undergoing BMT with platelet levels above and below 50,000/μL and also to detect if there is a platelet count for a safe insertion. This prospective study included all children who had placements of tunneled CVCs during BMT at our hospital between March 2017 and March 2020. Procedures were divided into two groups accordingly to preoperative platelet counts (above and below 50,000/μL). Data were compared between both groups regarding postoperative complications including bleeding or catheter-related blood stream infections (CRBSIs).ResultsForty-six CVC insertions were performed in 40 patients. There were 20 procedures below 50,000/μL (median 27,500; range 5000–42,000) inserted with perioperative platelet transfusions, and their postoperative levels were median 59,500/μL, range 18,000–88,000. Allogeneic BMT was adopted in 39 patients (97.5%). Beta thalassemia major was the commonest indication (21/40, 52.5%), followed by acute lymphocytic leukemia in six patients (15%). There were nine postoperative complications (bleeding n = 2 and CRBSIs n = 7) encountered in all placements. Four of them occurred in insertions below 50,000/μL (two bleeding complications that managed conservatively, and two CRBSIs). Post-procedural morbidities regarding bleeding or CRBSIs did not differ significantly between both groups (p value = 0.099 and 0.695, respectively).ConclusionsPostponement of CVC insertions in thrombocytopenic children due to the fear of potential complications seems unwarranted, as it has no significant impact on the morbidity. Placements of such catheters can be safe under cover of perioperative platelet transfusions irrespective of the preoperative platelet count.

Longitudinal characterization of olfactomedin-4 expressing neutrophils in pediatric patients undergoing bone marrow transplantation.

Sepsis is an important cause of morbidity and mortality in pediatric patients. Increased expression of olfactomedin-4 (OLFM4), a glycoprotein contained within a subpopulation of neutrophils, has been associated with complicated course in sepsis. The factors that regulate OLFM4 expression are unknown. Here, we followed children undergoing bone marrow transplantation (BMT) to document the percentage of neutrophils that express OLFM4 over time. This population was selected because of the ability to observe nascent neutrophils following engraftment, perform frequent blood sampling, and the children are at high risk for clinical complications that may associate with changes in percentage of OLFM4+ neutrophils. We found a surprising degree of variability of OLFM4 expression between patients. In the weeks following initial neutrophil recovery we also saw great variability in OLFM4 expression within individual patients, indicating that multiple external factors may modify OLFM4 expression. We identified decreased expression of CD64 (a marker associated with response to infection), in OLFM4+ neutrophils. This is the first study to demonstrate fluctuation in OLFM4 expression within patients and provides insight into possible mechanisms for OLFM4 regulation in nascent neutrophils.

Parenteral Nutrition Complications in Children Undergoing Bone Marrow Transplantation.

Parenteral nutrition (PN) seems to be a practical solution to face the negative nutritional effects of bone marrow transplantation. Report and describe all observed PN-related complications in children undergoing allogenic bone marrow transplantation in a tertiary center and determine the possible risk factors. This was a retrospective and observational study including 51 allografted children 2 to 17 years of age. We collected from medical patient records any noticed PN-related complications using an assessment causality method. The independent risk factors for complications were investigated by multivariate analysis. A total of 92% of patients (n=47) developed a total of 136 complications attributable to PN. The incidence rate of complications was 5 for 100 patient days of PN. Infectious complications (32.3%) and electrolytic disorders (27.9%) were the most common conditions identified during our study. Multivariate analysis showed that the duration of PN exposure, age, and hyperglycemia were the risk factors for the onset of these complications. Although we have noticed multiple complications attributable to PN, a certain causal link is difficult to establish in this particular context. Emphasizing enteral nutrition in bone marrow graft protocols would be the best way to avoid these complications.

Changes in the Stool Virome and Fungome of Children Undergoing Bone Marrow Transplant

Background Bacterial dysbiosis is associated with acute graft versus host disease (GVHD) and blood stream infections (BSI) after bone marrow transplant (BMT). Intestinal viral and fungal changes after BMT are unknown in children. Methods Metagenomic shotgun paired reads sequencing was performed for 56 patients who underwent a BMT at Cincinnati Children's Hospital Medical Center. Stool samples were collected pre BMT and at least one post BMT time point (range day+1-day+35). Metagenomics data were analyzed using DIAMOND / MEGAN6 CE and NCBI RefSeq database. Results Demographics are shown in Figure 1. A total of 180 stool samples were analyzed. Twelve of 56 patients had stool samples analyzed at 2 time points (pre BMT and post BMT) while 44 patients had stool analyzed at > 2 time points with one being a pre BMT sample. All patients were on fungal and viral prophylaxis. Fungome Fungi were detected in 19 of 56 patients' stool samples (Fig 2A). The most frequent fungi isolated from stool were Candida sp. Nine of 56 patients had evidence of clinical fungal infection after BMT (Candida N=7, Malassezia N=1, Rhizopus N=1). One patient with clinical Candida infection had Candida detected from stool pre BMT but the remaining patients did not have a fungal infection similar to the fungi isolated from stool. BSI occurred in 7/19 in patients with fungi isolated from stool compared to 11/37 in patients without fungi in stool. GI GVHD occurred in 7/16 (43%) in allogeneic BMT patients with fungi in stool compared to 5/35 (14%) patients without fungi isolated in stool. likely reflecting antibiotic exposure and resultant dysbiosis. Virome Viruses were identified in 31 of 56 patients' stool samples (Fig 2B). Caudalovirales (bacteriophages) were the most frequent organisms seen in stool (n=20). Interestingly Caudalovirales in stool were detected in 18 patients who were on human milk as their nutritional source. Mastadenovirus sp. (adenovirus in clinical practice) was observed in 5 patients. Mastadenoviruses in stool were seen with ATG (n=1) or alemtuzumab (n=4) exposure. Ten of 56 patients had clinical adenoviral reactivation. Four of the 10 patients with clinical adenoviral reactivation had adenovirus isolated in the stool prior to onset of clinical viremia (pre BMT n=1; day+1 n=2; day+13 n=1). Conclusion We observed that fungi were not frequently detected from stool, and presence of fungi did not always imply clinical fungal infection with the same organism but could reflect antibiotic exposure and contribute to intestinal dysbiosis leading to acute GVHD. Presence of bacteriophages in stool is a novel observation, which could be reflective of a diet of human milk. We have previously shown that human milk lowers intestinal inflammation after BMT and these findings will be explored in independent studies. Mastadenovirus was detected in stool before or early after BMT and frequently heralded clinical viral reactivation.

Islam, The Holy Qur'an, and Medical Decision-Making: The Experience of Middle Eastern Muslim Families with Children Undergoing Bone Marrow Transplantation in the United States.

Some Arabic-speaking Muslim family members of children requiring bone marrow transplantation receive medical care for their children in the United States. Muslim family members' use of Islam in the course of their child's bone marrow transplantation was studied using grounded theory, a qualitative research method. Eighteen members of Middle Eastern Muslim families with a total of 13 children receiving bone marrow transplantation were interviewed by an Arabic-speaking healthcare provider. Interviews were coded by an interdisciplinary team. Seven key themes were identified.

PIN30 - The Economic Burden of Varicella in Mexico

To assess the clinical and economic burden of varicella in Mexico, where the varicella vaccine is not publicly available, except in children undergoing bone marrow transplantation. Annual societal costs were estimated for a base scenario by multiplying per-patient costs with varicella incidence from the Mexican epidemiological surveillance system (SUIVE), adjusted for the estimated proportion of public healthcare centers who do not regularly report. In a sensitivity analysis where cost calculations remained stable, epidemiological estimates ranged between raw SUIVE varicella rates (lower bound), and a 25% additive increment to reflect the estimated proportion of public sector patients who utilize private services (upper bound). Outcome Measures Clinical complications; health care resource utilization (HCRU)(frequency and type) including outpatient visits, allied healthcare contacts, doctor’s visits, tests/procedures performed, prescription and over-the-counter (OTC) medications, hospitalizations, and Emergency Room (ER) and Intensive Care Unit(ICU) visits/stays. Charts of 152 varicella patients were included, of which 75 and 77 were outpatients and inpatients [mean (SD) age=3.0 (3.2) and 2.6 (3.5) years, respectively]. Inpatients reported higher complication rates (inpatients vs. outpatients: 84.4% vs. 6.7%), prescription medication use (94.8% vs. 80.0%), over-the-counter medications use (71.4% vs. 80.0%), tests/procedures (87.0% vs. 5.3%), and allied health professional consultations (61.0% vs. 12.0%). Direct/indirect costs per case were $198/$42 (outpatients), and $5,633/$175 (inpatients). Estimated societal costs related to varicella ranged from $77-$218 million. Substantial clinical and associated public health and economic burden were observed among children with primary varicella infection in Mexico. These results highlight the need for implementation of a childhood varicella vaccination program.

Effects of Parenteral Nutrition Formulas on Plasma Lipid Profile in Children with Bone Marrow Transplantation

Background/Aims: Children undergoing bone marrow transplantation (BMT) often require parenteral nutrition (PN). This is a comparative study of plasma lipid profiles in BMT children after fish oil or soybean PN. Methods: A total of 14 children with BMT requiring PN for at least 10 days were recruited during 24 months. They were randomized to receive a lipid emulsion enriched with ω3 polyunsaturated fatty acid, or soybean oil. Clinical monitoring was performed. Blood samples were collected before and after administration of PN to analyze the lipid profile. Results: There were no complications associated with PN. The increase in TG levels was more pronounced after administration of an enriched ω3 emulsion and the decrease in cHDL and apo A was greater after administration of soybean. The ω3 group showed an increase in eicosapentaenoic and a decrease in arachidonic acids compared to the soybean group. Both groups showed similar levels of linolenic acid. Conclusion: PN with soybean or ω3 emulsions for 10 days is safe in children. However, research in children are necessary in order to examine the impact of the duration of PN and the type of lipid formula used, and determine their health benefits in relation to the fatty acid profile.

Next-generation whole genome sequencing identifies the direction of norovirus transmission in linked patients.

Noroviruses are a highly transmissible and major cause of nosocomial gastroenteritis resulting in bed and hospital-ward closures. Where hospital outbreaks are suspected, it is important to determine the routes of spread so that appropriate infection-control procedures can be implemented. To investigate a cluster of norovirus cases occurring in children undergoing bone marrow transplant, we undertook norovirus genome sequencing by next-generation methods. Detailed comparison of sequence data from 2 linked cases enabled us to identify the likely direction of spread. Norovirus complementary DNA was amplified by overlapping polymerase chain reaction (PCR) from 13 stool samples from 5 diagnostic real-time PCR-positive patients. The amplicons were sequenced by Roche 454, the genomes assembled by de novo assembly, and the data analyzed phylogenetically. Phylogenetic analysis indicated that patients were infected by viruses similar to 4 distinct GII.4 subtypes and 2 patients were linked by the same virus. Of the 14 sites at which there were differences between the consensus sequences of the 2 linked viral genomes, 9 had minor variants present within one or the other patient. Further analysis confirmed that minor variants at all 9 sites in patient B w ere present as the consensus sequence in patient A. Phylogenetic analysis excluded a common source of infection in this apparent outbreak. Two of 3 patients on the same ward had closely related viruses, raising the possibility of cross-infection despite protective isolation. Analysis of deep sequencing data enabled us to establish the likely direction of nosocomial transmission.

Unmet care needs of parents of children with cancer in Jordan: implications for bed‐side practice

To evaluate the perceived care needs of parents of children with cancer in Jordan. (1) What are the parents' priority caring needs; (2) to what extent these needs are met by the healthcare team members; (3) the parents' need for further information (4) and whether socio-demographic, disease and treatment variables predict parents' needs for further information. Providing support for families caring for a child with a long-term illness has been recognised all through literature, yet studies focusing on parent needs are lacking. Descriptive survey. A total of 98 parents from a tertiary oncology hospital in metropolitan area in Amman completed the Arabic Family Inventory of Needs-Pediatric II. Most of the 17 needs measured by the Family Inventory of Needs-Pediatric II were considered important. Only 78% of parents needs were met by the healthcare professionals; yet, the results clearly highlighted the importance of informing parents about the child's illness, treatment and outcome. The socio-demographic variables were not significant predictors for the family needs for further information, with two major exceptions; parents with lower level of education and parents of children undergoing bone marrow transplantation or surgery. The needs of parents vary from family to another, and healthcare professionals must become familiar to these needs and acquire the skills to direct their interventions more appropriately. The Family Inventory of Needs-Pediatric II may be a useful adjunct in assessing the parents' needs for care and for information, as well as the quality of care services provided. Priority needs should be central in the daily care of these families and support programmes should be adopted in Jordan for planning and developing the system of care for these families, as well as their children.

Prevalence and Effect of Coagulation Defects in Children Undergoing Bone Marrow Transplantation for Haemoglobinopathies.

Abstract 3037Bone marrow transplantation (BMT) is currently the only cure for thalassaemia major (TM) and sickle cell disease (SCD). Due to the myeloablative preparative regimen used for transplants, patients with TM and SCD become thrombocytopenic prior to platelet engraftment and may occasionally suffer from significant haemorrhage. This may be potentially augmented by co-existing coagulopathies, either inherited, or as a result of abnormal synthesis of clotting factors in the liver due to transfusional hepatic siderosis. The frequency and clinical impact of coagulopathies in patients undergoing BMT for these conditions is not known. The aim of this project was to investigate whether coexisting coagulopathy or hepatic siderosis result in an increase in haemorrhagic complications or increased use of blood products.All patients who underwent BMT for TM or SCD at St Mary’s Hospital from 2002 to 2011 were eligible for this retrospective study. Initially, 98 patients were identified, of whom 4 patients were excluded due to lack of adequate data. Of the 94 patients studied, 69 had TM and 25 had SCD with a mean age of 7.7 years (range 2.2– 17.7). There were 50 females and 44 males. Consanguinity data were obtained in 72 patients, of which 33 had consanguineous parents (46%). Donor and recipient ABO blood group mismatch was present in 62 (66%) patients.Sixteen out of 94 patients had pre-transplant coagulopathies, of which 12 (75%) had TM and 4 (25%) had SCD. See table 1. These patients were generally asymptomatic and their coagulation deficiencies were only identified on pre-transplant screening Mean pre- transplant ferritin levels were 1587μg/l (s.d. 952) in patients with coagulopathies and 1646μg/l (s.d. 896) in patients without (p=0.815). Transfusional hepatic siderosis was assessed by T2* MRI scan of liver in 44 patients, of which 12 had coagulopathies and by dry weight iron estimation by liver biopsy in 53 patients, of which 11 had coagulopathies. There was no significant difference in hepatic siderosis assessed by T2* MRI scan of liver, (12 versus 9 ms in patients with and without coagulopathies, p=0.28) or by dry weight iron in liver biopsy; (2.9 mg/g versus 3.7 mg/g in those with coagulopathies and those without, respectively, p=0.39).Mean number of platelet units used in patients with pre-existing coagulopathies was 15.3 (standard deviation, s.d. 12.2) compared to 21.7 units in patients with no pre-existing coagulopathies, p=0.18. Similarly, there was no significant difference in the number of red cell units used in patients with coagulopathies (12.1 units, s.d. 7.8) and in patients without (12.6 units, s.d. 6.2), p=0.78.Five patients (5.3%) in total had major haemorrhages in the first 30 days post transplant, of which 1 patient had a pre-existing coagulation factor deficiency (FXIII deficiency 36.8 IU/dl, normal range 50–150) and 4 patients had normal coagulation screen prior to transplant.This study concludes that in a cohort of patients being transplanted for haemoglobinopathies, there is a high frequency of coagulopathies (17%) and that at least some of these disorders are likely to have been inherited, as there were no differences in hepatic siderosis in the 2 groups. Furthermore, pre-transplant coagulopathies such as these are not a contraindication to transplant since these patients were not at a higher risk of major haemorrhage during the transplant and their red cell or platelet requirement was not significantly higher than patients with normal pre-transplant coagulation screens. Further studies are needed to clarify the extent to which pre-transplant coagulopathies are familial and increased in frequency due to consanguinity. In such a scenario, family screening for homozygous deficiencies in other family members might then be indicated.Table 1Details of coagulation defects in patients identified on screeningPatient's Coagulopathy1. FXI + FXII deficiency2. Mild Haemophilia A3. Mild FXI deficiency4. Mild FVII deficiency and borderline FXI deficiency5. Type 1 von Willebrand Disease6. Type 1 von Willebrand Disease7. Reduced hepatic synthesis of multiple factors8. FXIII deficiency and hypofibrinogenaemia9. Reduced hepatic synthesis of multiple factors10. Borderline FVII11. Type 1 von Willebrand Disease12. FXI deficiency13. FXII and FXIII deficiency14. Dysfibrinogenaemia15. Dysfibrinogenaemia16. FXII deficiency Disclosures:No relevant conflicts of interest to declare.

Cytomegalovirus gB Genotype Distribution in Mexican Children Undergoing Allogeneic Bone Marrow Transplantation

We used nested multiplex polymerase chain reaction to determine the distribution of cytomegalovirus gB genotype in 30 Mexican children undergoing bone marrow transplantation. The genotypes were: gB1, 9/30 (30%); gB2, 8/30 (27%); gB3, 4/30 (13%), and gB4, 1/30 (3%); mixed genotypes were identified in 8/30 patients (27%). We conclude that the predominant genotypes identified were gB2 and gB1. The gB5 genotype was not detected, and a high proportion of mixed genotype was found.

Olive oil-based intravenous lipid emulsion in pediatric patients undergoing bone marrow transplantation: A short-term prospective controlled trial

Parenteral nutrition (PN) is an important component of the supportive care of children undergoing bone marrow transplantation (BMT). The study aimed to assess short-term safety and metabolic effects of an olive oil-based (OO) lipid emulsion compared with a MCT/LCT (M/L) emulsion in the clinical setting of pediatric BMT. Twenty-eight pediatric BMT patients (age 1-18 years) expected to need PN support for at least 2 weeks, were prospectively enrolled and randomly assigned to receive either OO or M/L lipid emulsions within PN. Clinical and routine laboratory parameters, plasma fatty acids profile, vitamin E and peroxidation status were recorded at baseline and after 14 days of PN. No significant differences were found for hematological parameters, liver enzymes, vitamins, plasma peroxidation status, percentage and time to engraftment. Taking into consideration the baseline fatty acids levels, the OO group showed higher oleic acid (p=0.012), linoleic (p=0.012) and arachidonic acid (p=0.002) enrichment but similar eicosapentanoic and docosahexanoic acids levels compared to the M/L group at day 14. Cholesterol levels decreased significantly in the OO group after 14 days on PN (p=0.017). OO lipid emulsion was well tolerated, maintained essential fatty acids and peroxidation status, and generated a favorable plasma lipid profile. In this study short-term use of OO intravenous lipid emulsions was safe in children who needed PN support during BMT.

Assimilative And Accommodative Behaviors Of Children Undergoing Bone Marrow Transplantation

The purpose of the study was to explore the experience, assimilative and accommodative behaviors of children undergoing bone marrow transplantation, in the framework of a descriptive qualitative study. Based on the diagram of bone marrow transplantation experiences and the semi-structural interview guide, in-depth interviews were conducted on 11 children who received bone marrow transplantation. Each interview lasted 60–90 minutes and was recorded and later composed into descriptive contexts. Content analysis was further applied to identify and categorize the children's personal experiences, assimilative and accommodative behaviors.

In vitro analysis of multipotent mesenchymal stromal cells as potential cellular therapeutics in neurometabolic diseases in pediatric patients

Multipotent mesenchymal stromal cells (MSCs) play an important role in stromal support for hematopoietic stem cells, immune modulation, and tissue regeneration. We investigated their potential as cellular therapeutic tools in neurometabolic diseases as a growing number of affected children undergo to bone marrow transplantation. MSCs were isolated from bone marrow aspirates and expanded ex vivo under various culture conditions. MSCs under optimal good medical practice (GMP)-conform culture conditions showed the typical morphology, immunophenotype, and plasticity. Biochemically, the activities of beta-hexosaminidase A, total beta-hexosaminidase, arylsulfatase A (ASA), and beta-galactosidase measured in MSCs were comparable to those in fibroblasts of healthy donors. These four enzymes were interesting for their expression in MSCs, as each of them is defective, respectively, in well-known neurometabolic diseases. We found that MSCs released significant amounts of ASA into the media. In coculture experiments, fibroblasts from patients with metachromatic leukodystrophy, who are deficient for ASA, took up a substantial amount of ASA that was released into the media from MSCs. Mannose-6-phosphate (M6P) inhibited this uptake, which was in accordance with the M6P receptor-mediated uptake of lysosomal enzymes. Taken together, we show that MSCs produce appreciable amounts of lysosomal enzyme activities, making these cells first-choice candidates for providing metabolic correction when given to enzyme-deficient patients. With the example of ASA, it was also shown that an enzyme secreted from MSCs is taken up by enzyme-deficient patient fibroblasts. Given the plasticity of MSCs, these cells represent an interesting add-on option for cellular therapy in children undergoing bone marrow transplantation for lysosomal storage diseases and other neurometabolic diseases.

Hepatitis B immunoglobulin in combination with lamivudine for prevention of hepatitis B virus reactivation in children undergoing bone marrow transplantation

There is little information in literature about the use of hepatitis B immunoglobulin (HBIg) in recipients of bone marrow transplantation (BMT). Here, we report two children who received IV HBIg (Hepatect-CP) and lamivudine treatment during BMT course for either patient or donor hepatitis B virus (HBV) viremia. A four-year-old girl underwent a fully human leukocyte antigen-matched allogeneic BMT for thalassemia major from her mother positive for hepatitis B surface antigen (HBsAg). A 12-yr-old boy with chronic myeloid leukemia, positive for HBsAg and HBV-DNA received a fully HLA-matched allogeneic BMT from his sister in the first chronic phase of the disease. HBIg was successfully used in both cases to prevent HBV reactivation of the recipients. The results of our observations are encouraging and we suggest that HBIg in combination with lamivudine may be used in such cases especially in post-transplant early period to prevent HBV reactivation.