Pathobiological signatures of dysbiotic lung injury in pediatric patients undergoing stem cell transplantation.

Hematopoietic cell transplantation (HCT) uses cytotoxic chemotherapy and/or radiation followed by intravenous infusion of stem cells to cure malignancies, bone marrow failure and inborn errors of immunity, hemoglobin and metabolism. Lung injury is a known complication of the process, due in part to disruption in the pulmonary microenvironment by insults such as infection, alloreactive inflammation and cellular toxicity. How microorganisms, immunity and the respiratory epithelium interact to contribute to lung injury is uncertain, limiting the development of prevention and treatment strategies.

Mediators of Social Acceptance Among Emerging Adult Survivors of Childhood Cancer.

Examine associations of social developmental factors (e.g., peer/parent social attachment, romantic relationships) and perceptions of social acceptance among emerging adult survivors of childhood cancer.

Effect of Poloxamer 188 vs Placebo on Painful Vaso-Occlusive Episodes in Children and Adults With Sickle Cell Disease: A Randomized Clinical Trial.

Importance: Although effective agents are available to prevent painful vaso-occlusive episodes of sickle cell disease (SCD), there are no disease-modifying therapies for ongoing painful vaso-occlusive episodes; treatment remains supportive. A previous phase 3 trial of poloxamer 188 reported shortened duration of painful vaso-occlusive episodes in SCD, particularly in children and participants treated with hydroxyurea.

Objective: To reassess the efficacy of poloxamer 188 for vaso-occlusive episodes.

A cluster-randomized control trial targeting parents of pediatric cancer survivors with obesity: Rationale and study protocol of NOURISH-T.

Approximately 40-50% of pediatric cancer survivors (PCS) are overweight or obese; increasing their risk for metabolic syndrome and other negative long-term physical health complications. Using our successful pilot trial testing the preliminary feasibility and efficacy of NOURISH for Healthy Transitions (NOURISH-T), we refined our intervention, now NOURISH-T+, and will implement these refinements in this larger, multi-site randomized control trial. Parents of PCS with overweight/obesity (BMI ≥ 85th%ile), age 5-12, ≥6 months off treatment are randomly assigned to the NOURISH-T+ intervention or Enhanced Usual Care (EUC) comparison.

Safety and efficacy of rivaroxaban in pediatric cerebral venous thrombosis (EINSTEIN-Jr CVT).

Anticoagulant treatment of pediatric cerebral venous thrombosis has not been evaluated in randomized trials. We evaluated the safety and efficacy of rivaroxaban and standard anticoagulants in the predefined subgroup of children with cerebral venous thrombosis (CVT) who participated in the EINSTEIN-Jr trial. Children with CVT were randomized (2:1), after initial heparinization, to treatment with rivaroxaban or standard anticoagulants (continued on heparin or switched to vitamin K antagonist).

Pediatric HCT in Florida (2014 -2016): A report from the FPBCC.

FPBCC was formed in 2018 by five pediatric transplant programs in Florida. One of the key objectives of the consortium is to provide outcome analyses by combining HCT data from all the participating centers in order to identify areas for improvement. In this first FPBCC landscape report we describe the patient and transplant characteristics of pediatric patients undergoing first allo and auto HCT between 2014 and 2016 in Florida.

Abatacept is effective as GVHD prophylaxis in unrelated donor stem cell transplantation for children with severe sickle cell disease.

We report results of a phase 1 multicenter stem cell transplantation (SCT) trial from HLA-matched (n = 7) or one-antigen-mismatched (n = 7) unrelated donors (URD) using bone marrow or cord blood as stem cell source, following reduced-intensity conditioning (RIC) in severe sickle cell disease (SCD). Conditioning included distal alemtuzumab, fludarabine, and melphalan (matched donors), with thiotepa (mismatched donors). Abatacept, a selective inhibitor of T cell costimulation, was added to tacrolimus and methotrexate as graft-versus-host disease (GVHD) prophylaxis to offset GVHD risks, and was administered for longer duration in bone marrow recipients than in cord blood recipients because of increased incidence of chronic GVHD with bone marrow.

Integration of Publicly Reported Center Outcomes into Standards and Accreditation: The FACT Model.

The rapid evolution of blood and marrow transplantation (BMT), coupled with diverse outcomes associated with heterogeneous groups of patients, led to the formation of 2 important organizations early in the development of the field: the Center for International Blood and Marrow Transplant Research (CIBMTR) and the Foundation for the Accreditation of Cellular Therapy (FACT). These organizations have addressed 2 of the 9 elements identified by the National Quality Strategy (NQS) for achieving better health care, more affordable care, and healthy people and communities: a registry that promotes improvement of care and accreditation based on quality standards. More recently, a federally mandated database in the United States addresses the third element of the NQS: public reporting of treatment results.

Infusion of Hematopoietic Stem Cell Products Using Pump Mechanism: An Approach Worth Consideration?

Hematopoietic stem cell transplantation using progenitor cells is a potentially curative treatment option for patients with high-risk malignancies and nonmalignant hematologic, immunologic, and genetic conditions. There is a need for evidence regarding safe practices and controlled infusion processes. Syringe and intravenous infusion pumps are not commonly used to deliver hematopoietic stem cell products (HPCs) due to a paradigm of thought that suggests that the pressure from the pump might damage the HPCs.

Unrelated Donor Transplantation in Children with Thalassemia using Reduced-Intensity Conditioning: The URTH Trial.

Allogeneic hematopoietic stem cell transplantation (HSCT) can cure transfusion-dependent thalassemia (TDT). In a multicenter trial we investigated the efficacy of reduced-intensity conditioning (RIC) before unrelated donor (URD) HSCT in children with TDT. Thirty-three children, ages 1 to 17 years, received bone marrow (BM) or umbilical cord blood (UCB) allografts.

"Cancer was a speed bump in my path to enlightenment:" A qualitative analysis of situational coping experiences among young adult survivors of childhood cancer.

Young adult survivors of childhood cancer (N = 47) completed essays exploring situational coping within a mixed methods study. Data were qualitatively analyzed using consensual qualitative research-modified methodology. Five themes emerged: (1) initial reactions to cancer, (2) adjustment/coping with cancer diagnosis and treatment, (3) provisions of social support, (4) perceived effects of cancer experience, and (5) reflections on the cancer experience.

A trial of unrelated donor marrow transplantation for children with severe sickle cell disease.

Children with sickle cell disease experience organ damage, impaired quality of life, and premature mortality. Allogeneic bone marrow transplant from an HLA-matched sibling can halt disease progression but is limited by donor availability. A Blood and Marrow Transplant Clinical Trials Network (BMT CTN) phase 2 trial conducted from 2008 to 2014 enrolled 30 children aged 4 to 19 years; 29 were eligible for evaluation.

Relations among optimism, perceived health vulnerability, and academic, self-regulatory, and social self-efficacy in adolescent survivors of childhood cancer.

This study investigated relations among optimism, perceived health vulnerability, treatment intensity, and academic, self-regulatory, and social self-efficacy in adolescent survivors of childhood cancer. Fifty-six adolescent survivors (Mage = 16.19 years, SD = 2.

HLA-DR expression on myeloid cells is a potential prognostic factor in patients with high-risk neuroblastoma.

The adaptive immune system has been reported to play a dual role in many cancers, on one hand inhibiting tumor growth and, on the other hand, promoting disease progression, escape from cancer immunosurveillance and relapse. We have previously reported that the suppression of the adaptive immune response associated with high levels of myeloid-derived suppressor cells (MDSC) was evident in patients with low-risk neuroblastoma. Here, we report the results of a pilot study demonstrating that the amounts of HLA-DR-positive or negative myeloid cells in the peripheral blood might predict disease outcome among individuals affected by high-risk neuroblastoma.

Effect of race on outcomes after allogeneic hematopoietic cell transplantation for severe aplastic anemia.

We compared outcomes after hematopoietic cell transplantation in patients of African American (n = 84) and Caucasian (n = 215) descent with severe aplastic anemia. African Americans and Caucasians were matched for age, donor-recipient human leukocyte antigen match, graft type, and transplantation year. The median follow-up of surviving patients was 5 years.

Vitamin d deficiency in children with cancer.

A limited number of small studies have examined the vitamin D status of pediatric oncology patients, and the results indicate an increased prevalence of hypovitaminosis. We conducted a cross-sectional study with the primary aim of describing the vitamin D status of our pediatric cancer patients and any associations with demographic characteristics. Our secondary aim was to compare this prevalence to that of a healthy population.

Graft-versus-host disease after solid organ transplantation: a single center experience and review of literature.

Background: Graft-versus-host disease (GVHD) is an uncommon cause of morbidity and mortality after solid organ transplantation that is most likely under-diagnosed. We describe our single center experience with three cases of GVHD diagnosed over a period of 15 years in a total of 2,271 solid organ transplant recipients.

Case Reports: We describe three case reports: (1) a 3-week old neonate who developed GVHD 16 months after living-related liver transplant, (2) a 14-year old adolescent who developed GVHD 4 months following an unrelated cadaveric pancreas transplant and; (3) a 27-year old male who developed GVHD 18 days after simultaneous kidney-pancreas transplant from an unrelated donor.

Outcome of children who experience disease relapse following allogeneic hematopoietic SCT for hematologic malignancies.

Relapse after allogeneic hematopoietic SCT (HSCT) carries a poor prognosis and is a common cause of death. Outcomes of children who relapse post HSCT are not well known. In this retrospective multicenter study we included 532 patients who underwent allogeneic HSCT and examined the outcomes of 160 patients (30%) who relapsed.

Distinct signatures of the immune responses in low risk versus high risk neuroblastoma.

Background: Over 90% of low risk (LR) neuroblastoma patients survive whereas less than 30% of high risk (HR) patients are long term survivors. Age (children younger than 18 months old) is associated with LR disease. Considering that adaptive immune system is well developed in older children, and that T cells were shown to be involved in tumor escape and progression of cancers, we sought to determine whether HR patients may tend to show a signature of adaptive immune responses compared to LR patients who tend to have diminished T-cell responses but an intact innate immune response.

Randomized trial of hydroxychloroquine for newly diagnosed chronic graft-versus-host disease in children: a Children's Oncology Group study.

The Children's Oncology Group conducted a multicenter Phase III trial for chronic graft-versus-host disease (cGVHD). The double-blind, placebo-controlled, randomized study evaluated hydroxychloroquine added to standard therapy for children with newly diagnosed cGVHD. The study also used a novel grading and response scoring system and evaluated clinical laboratory correlates of cGVHD.

Analysis of the role of hematopoietic stem-cell transplantation in infants with acute lymphoblastic leukemia in first remission and MLL gene rearrangements: a report from the Children's Oncology Group.

Purpose: Although the majority of children with acute lymphoblastic leukemia (ALL) are cured with current therapy, the event-free survival (EFS) of infants with ALL, particularly those with mixed lineage leukemia (MLL) gene rearrangements, is only 30% to 40%. Relapse has been the major source of treatment failure for these patients. The parallel Children's Cancer Group (CCG) 1953 and Pediatric Oncology Group (POG) 9407 studies were designed to test the hypothesis that more intensive therapy, including dose intensification of chemotherapy, and hematopoietic stem-cell transplantation (HSCT) would improve the outcome for this group of patients.

Fatigue, sleep-wake disturbances, and quality of life in adolescents receiving chemotherapy.

Background: Adolescents with cancer experience distressing physical and psychosocial symptoms, especially during treatment. Fatigue and sleep disturbances commonly affect adolescents' quality of life, but little is known about how adolescents experience these symptoms during an early month of chemotherapy. This study measured fatigue, sleep disturbances, and quality of life in 20 adolescents over 1 month while they were receiving chemotherapy.

Outcomes of pediatric bone marrow transplantation for leukemia and myelodysplasia using matched sibling, mismatched related, or matched unrelated donors.

Although some trials have allowed matched or single human leukocyte antigen (HLA)-mismatched related donors (mmRDs) along with HLA-matched sibling donors (MSDs) for pediatric bone marrow transplantation in early-stage hematologic malignancies, whether mmRD grafts lead to similar outcomes is not known. We compared patients < 18 years old reported to the Center for International Blood and Marrow Transplant Research with acute myeloid leukemia, acute lymphoblastic leukemia, chronic myeloid leukemia, and myelodysplastic syndrome undergoing allogeneic T-replete, myeloablative bone marrow transplantation between 1993 and 2006. In total, patients receiving bone marrow from 1208 MSDs, 266 8/8 allelic-matched unrelated donors (URDs), and 151 0-1 HLA-antigen mmRDs were studied.