Pediatric Bone marrow transplantation

INTRODUCTION: Sinusoidal obstructive syndrome (SOS), or veno-occlusive disease (VOD), of the liver has been recognized as a complex, life-threatening complication in the post-hematopoietic stem cell transplant (HSCT) setting. The diagnostic criteria for SOS have evolved over the last several decades with a greater understanding of the underlying pathophysiology, with two recent diagnostic criteria introduced in 2018 (EBMT criteria) and 2020 (Cairo criteria). OBJECTIVE: We sought out to evaluate the performance characteristics in diagnosing and grading SOS in pediatric patients of the four different diagnostic criteria (Baltimore, Modified Seattle, EBMT, and Cairo) and severity grading systems (defined by the EBMT and Cairo criteria)...

Objective: To analyze the efficacy and safety of the L-DEP regimen (asparaginase, liposome doxorubicin, etoposide and methylprednisolone) as a salvage therapy for the refractory primary hemophagocytic lymphohistocytosis triggered by Epstein-Barr virus infection (EBV-pHLH) in children. Methods: In this retrospective case study, clinical and laboratory data before and after L-DEP regimen of 4 children diagnosed with EBV-pHLH in Beijing Children's hospital between January 2016 and June 2022 were collected, and the efficacy and safety of L-DEP regimen for the treatment of EBV-pHLH were analyzed...

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...

Traditionally, bone marrow (BM) has been preferred as a source of stem cells (SCs) in pediatric hematopoietic SC transplantation (HSCT); however, the use of peripheral blood SCs (PBSC) has recently increased. With advancing graft-versus-host disease (GVHD) prophylaxis, whether the BM is still a better SC source than PB in sibling donor HSCT remains controversial. Here, we compared the results of BM transplantation (BMT) and PBSC transplantation (PBSCT) in pediatric patients with malignant or non-malignant diseases receiving sibling HSCT using a total of 7...

BACKGROUND: The burden of respiratory syncytial virus (RSV) in high-risk pediatric patients remains unclear. Therefore, this study aims to characterize pediatric RSV cases from January 2019 to December 2022 and assess the impact of the COVID-19 pandemic on RSV burden and RSV-related outcomes. In addition, examining factors influencing RSV-related hospitalization. METHODS: This is a retrospective study that included pediatric patients (aged 14 and below) who presented at King Faisal Specialist Hospital and Research Centre (KFSHRC) in Riyadh, Saudi Arabia with RSV infection identified using real-time reverse-transcriptase polymerase chain reaction assays...

Epidermolysis bullosa (EB) is a genodermatosis that lacks effective treatments and requires supportive care for its severe, life-threatening manifestations. Bone marrow transplantation (BMT) and its derived cells have been suggested to improve clinical symptoms and quality of life. A comprehensive search was conducted for publications evaluating BMT and bone marrow-derived mesenchymal stem cell (BM-MSC) therapy for EB in PubMed/MEDLINE, Google Scholar, and Cochrane databases from inception until June 2023. A total of 55 participants with severe forms of EB had BMT and/or BM-MSCs, with recessive dystrophic EB as the most common EB type; 53 (96...

KEY CLINICAL MESSAGE: Fanconi anemia with Mitomycin C sensitivity is a rare, complex hematological condition. Our case study emphasizes the significance of early diagnosis, appropriate genetic testing, and cautious use of chemotherapeutic agents. ABSTRACT: Fanconi anemia (FA) is a rare genetic disorder characterized by bone marrow failure, congenital anomalies, and predisposition to cancer. Here, we present the case of a 6-year-old boy with a known diagnosis of Fanconi anemia who exhibited sensitivity to Mitomycin C...

Fanconi anemia (FA) is a complex inherited bone marrow failure syndrome characterized by chromosomal instability and defective DNA repair causing sensitivity to DNA interstrand cross-linking agents. Our understanding of the full adult phenotype of the disease continues to evolve, as most patients with Fanconi Anemia died of marrow failure in the first decade of life prior to more recent advances in allogeneic hematopoietic cell transplantation. Herein, we report a previously undescribed, clinically concerning, progressive neurologic syndrome in patients with FA...

Haploidentical stem cell transplantation (haplo-SCT) represents the main alternative for children with inherited bone marrow failure syndrome (I-BMF) lacking a matched donor. This retrospective study, conducted on behalf of the EBMT SAAWP and PDWP, aims to report the current outcomes of haplo-SCT in I-BMFs, comparing the different in vivo and ex vivo T-cell depletion approaches. One hundred and sixty-two I-BMF patients who underwent haplo-SCT (median age 7.4 years) have been registered. Fanconi Anemia was the most represented diagnosis (70...

INTRODUCTION: Temcell is a mesenchymal stem cell (MSC) product approved for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in Japan. However, reports regarding Temcell's efficacy in pediatric patients have been scarce, and the appropriate use of MSC therapy against pediatric SR-aGVHD also remains to be determined. PATIENTS AND METHODS: We retrospectively assessed a cohort of pediatric patients treated with Temcell for SR-aGVHD following allogeneic hematopoietic transplantation...

BACKGROUND: Sarcopenia, characterized by decreased muscle mass, strength, and function, is associated with adverse outcomes. The prevalence of sarcopenia and the effect of the inflammatory response on muscle strength loss in children undergoing hematopoietic stem cell transplantation (HSCT) are unknown. This study aimed to estimate the prevalence of sarcopenia on admission and its associated clinical factors in children and adolescents undergoing HSCT and to determine the extent to which the systemic inflammatory response during hospitalization affects muscle strength...

Severe congenital neutropenia (SCN) is caused by germline mutations, most commonly in ELANE , impacting neutrophil maturation and leading to high risk of life-threatening infections. Most patients with ELANE- mutant SCN can achieve safe neutrophil counts with chronic Granulocyte-Colony Stimulating Factor (G-CSF). However, up to 10% of patients have neutropenia refractory to G-CSF and require allogeneic stem cell transplant. Traditional conditioning for these patients includes busulfan and cyclophosphamide which is associated with significant toxicities...

BACKGROUND: Pneumatosis intestinalis (PI, presence of air in bowel wall) develops in a variety of settings and due to a variety of insults which is then characterized by varying severity and clinical course. Anecdotally, many of these cases are benign with few clinical sequelae; however, we lack evidence-based guidelines to help guide management of such lower-risk cases. We aimed to describe the clinical entity of low-risk PI, characterize the population of children who develop this form of PI, determine if management approach or clinical outcomes differed depending on the managing physician's field of practice, and finally determine if a shortened course of NPO and antibiotics was safe in the population of children with low-risk PI...

Hematopoietic stem cell transplantation can cure various disorders but poses cardiovascular risks, especially for elderly patients and those with cardiovascular diseases. Cardiovascular evaluations are crucial in pretransplantation assessments, but guidelines are lacking. This American Heart Association scientific statement summarizes the data on transplantation-related complications and provides guidance for the cardiovascular management throughout transplantation. Hematopoietic stem cell transplantation consists of 4 phases: pretransplantation workup, conditioning therapy and infusion, immediate posttransplantation period, and long-term survivorship...

Despite aggressive multimodal treatment, the outcome of pediatric patients affected by high-risk (HR) neuroblastoma (NB) remains poor. The rational of use of allogeneic hematopoietic stem cell transplantation (allo-HCT) for NB was based on the possible graft versus tumor effect; however, toxicity represents a limitation to its efficacy. We sought to prospectively assess the feasibility and efficacy of allo-HCT using a reduced intensity conditioning regimen in pediatric patients with HR NB in a multicenter phase II trial...

Hematopoietic Cell Transplantation (HCT) activity was surveyed in the Kingdom of Saudi Arabia (KSA). The overall rate of HCT per 10,000,000 inhabitants doubled every 10 years. 15,031 HCTs were reported by all the functional HCT centers in KSA since inception of HCT program. Out of total HCT 15,031; 10,232(68%) were reported in adults, and 4799(32%) in the pediatric population. Allogeneic HCT constituted 10,489(70%) of total HCT, with majority from Human Leukocyte Antigen matched identical sibling (85.4%)...

BACKGROUND: Inborn errors of immunity (IEI) are often associated with inflammatory bowel disease (IBD). IEI can be corrected by allogeneic hematopoietic stem cell transplantation (HSCT) however peri-transplantation intestinal inflammation may lead to increased risk of gut graft-versus-host disease (GVHD). Vedolizumab inhibits the homing of lymphocytes to the intestine and may attenuate gut GVHD, yet its role in preventing GvHD in pediatric patients with IEI-associated IBD has not been studied...

The primary objective of our multicenter prospective study was to describe the incidence of late-onset non-infectious pulmonary complications (LONIPCs) in children undergoing hematopoietic cell transplantation (HCT) using sensitive criteria for pulmonary function test (PFT) abnormalities including the non-specific pattern of airflow obstruction. Secondary objectives were to assess the factors associated with LONIPC occurrence and the sensitivity of the 2014 NIH-Consensus Criteria of bronchiolitis obliterans syndrome (BOS)...

In leukemogenesis, genotoxic stress in hematopoietic stem and progenitor cells (HSPCs) drives individual context-dependent programs of malignant transformation. In light of the various differentiation stages of HSPCs based on a recently revised definition using CD150/CD48, our analyses showed that a subpopulation of long-term-repopulating HSCs was most susceptible to MLL-ENL-mediated transformation. An analysis of the molecular mechanism identified Bromo-adjacent homology domain and coiled-coil containing 1 (Bahcc1), which encodes a reader molecule of trimethylated histone H3 lysine 27 (H3K27me3), as a candidate gene involved in distinct susceptibility to leukemic transformation...

Radiotherapy of the whole body is called total body irradiation (TBI). It is a well-established component of conditioning regimens before stem cell transplantation in juvenile leukemia. The patient was a three-year-old child with a diagnosis of B-cell acute lymphoblastic leukemia and planned for stem cell transplantation. He was given TBI under anesthesia for three consecutive days prior to the bone marrow transplantation under general anesthesia. The important concerns were related to neutropenia/immune suppression, parental consent for repeated anesthesia, nothing per oral guidelines for the TBI treatment, the possibility of high-grade fever, high chance of respiratory tract infections with repeated anesthesia exposure, etc...