MSC.sensor: Capturing cancer cell interactions with stroma for functional profiling.

Mesenchymal stromal cells (MSCs) contribute to the microenvironment regulating normal and malignant hematopoiesis, and thus may support subpopulations of cancer cells to escape therapeutic pressure. Here, we engineered bone marrow MSCs to express a synthetic CD19-sensor receptor to detect and display interacting primary CD19+ leukemia cells in coculture. This implementation provides a versatile platform facilitating ex vivo drug response profiling of primary CD19+ leukemia cells in coculture with high-sensitivity and scalability.

Voriconazole plasma concentrations and dosing in paediatric patients below 24 months of age.

Voriconazole (VCZ) is an important first-line option for management of invasive fungal diseases and approved in paediatric patients ≥24 months at distinct dosing schedules that consider different developmental stages. Information on dosing and exposures in children <24 months of age is scarce. Here we report our experience in children <24 months who received VCZ due to the lack of alternative treatment options.

Enteric-Coated Cysteamine Bitartrate in Cystinosis Patients.

Cystinosis is a severe inherited metabolic storage disease caused by the lysosomal accumulation of cystine. Lifelong therapy with the drug cysteamine bitartrate is necessary. Cysteamine cleaves intralysosomal cystine, and thereafter, it can exit from the organelle.

Functional analysis of structural variants in single cells using Strand-seq.

Somatic structural variants (SVs) are widespread in cancer, but their impact on disease evolution is understudied due to a lack of methods to directly characterize their functional consequences. We present a computational method, scNOVA, which uses Strand-seq to perform haplotype-aware integration of SV discovery and molecular phenotyping in single cells by using nucleosome occupancy to infer gene expression as a readout. Application to leukemias and cell lines identifies local effects of copy-balanced rearrangements on gene deregulation, and consequences of SVs on aberrant signaling pathways in subclones.

Interaction in vitro of pulmonary surfactant with antifungal agents used for treatment and prevention of invasive aspergillosis.

Background: Optimizing antifungal therapy is important to improve outcomes in severely immunocompromised patients.

Objectives: We analysed the in vitro interaction between pulmonary surfactant and antifungal agents used for management of invasive pulmonary aspergillosis.

Methods: Amphotericin B formulations, mould-active triazoles and echinocandins were tested in vitro against 24 clinical isolates of different Aspergillus spp.

Toxoplasmosis after allogeneic haematopoietic cell transplantation-disease burden and approaches to diagnosis, prevention and management in adults and children.

Background: Toxoplasmosis is a rare but highly lethal opportunistic infection after allogeneic haematopoietic cell transplantation (HCT). Successful management depends on screening, early recognition and effective treatment.

Objectives: To review the current epidemiology and approaches to diagnosis, prevention and treatment of toxoplasmosis in adult and paediatric allogeneic HCT recipients.

Redirecting T cells to treat solid pediatric cancers.

The capacity of single-agent therapy with immune checkpoint inhibitors to control solid cancers by unleashing preexisting local antitumor T cell responses has renewed interest in the broader use of T cells as anticancer therapeutics. At the same time, durable responses of refractory B-lineage malignancies to chimeric-receptor engineered T cells illustrate that T cells can be effectively redirected to cancers that lack preexisting tumor antigen-specific T cells, as most typical childhood cancers. This review summarizes strategies by which T cells can be modified to recognize defined antigens, with a focus on chimeric-receptor engineering.

Moraxella nonliquefaciens bloodstream infection and sepsis in a pediatric cancer patient: case report and literature review.

Background: Moraxella nonliquefaciens is a usually non-pathogenic biofilm-producing Gram-negative coccobacillus which may colonize the upper respiratory tract, rarely causing invasive disease. Although very rare, bloodstream infections caused by this organism have been described, showing often a fatal outcome. Here, we report the case of a pediatric cancer patient with bloodstream infection and sepsis due to M.

Outcome in Relation to Drinking Goals in Alcohol-Dependent Individuals: A Follow-up Study 2.5 and 5 Years After Treatment Entry.

Aims: To evaluate the long-term treatment outcome (up to 5 years) with respect to different drinking goals of patients.

Methods: Alcohol-dependent individuals (n = 349) were recruited from three alcohol treatment units. They were interviewed using the Addiction Severity Index (ASI).

Disseminated Bartonella henselae disease mimicking Langerhans' cell histiocytosis.

Bartonella henselae, the causative agent of cat-scratch disease, has been recognized to be responsible for a broad range of clinical syndromes. We report the case of a patient with disseminated B. henselae infection mimicking Langerhans cell histiocytosis at presentation and its successful management with neurosurgery, prolonged antibacterial therapy, and observation.

The Influence of Smoking and Smokeless Tobacco on the Progression, Severity and Treatment Outcome in Alcohol-Dependent Individuals.

Aims: The influence of tobacco use in alcohol-dependent individuals is not well understood, especially the role of snuffing, which is common in Northern Europe. The aim was therefore to investigate the influence of smoking and snuffing on the progression, severity and treatment outcome in alcohol-dependent individuals. The hypotheses were that concomitant tobacco use (i.