Osteosarcoma, which is most common in people 10 to 30 years of age, is generally treated with resection and adjuvant chemotherapy. Detection of gene rearrangements, copy-number variations, and ...targeted disruption of tumor suppressors by whole-genome sequencing has not yet led to improved treatment.
The article considers the role of the endocrinologist in the treatment of patients with Down syndrome. Particular attention is paid to the diagnosis and therapy, an integrated approach to the ...treatment of complex patients taking into account features of the disease.
Biologic Therapies for Severe Asthma Brusselle, Guy G; Koppelman, Gerard H
The New England journal of medicine,
01/2022, Letnik:
386, Številka:
2
Journal Article
Recenzirano
Odprti dostop
Patients with severe asthma are at increased risk for a decreased quality of life, fixed airway obstruction, hospitalization, and death. Biologics may be required to reduce the disease burden. This ...review discusses the mechanisms, efficacy, and safety of biologics for severe asthma.
Nirsevimab is a monoclonal antibody to the RSV fusion protein that has an extended half-life. In this clinical trial, a single dose of nirsevimab resulted in a significantly lower incidence of ...medically attended RSV-associated lower respiratory tract infection than that with placebo.
Of 23 patients with transfusion-dependent β-thalassemia who received gene-transfected autologous hematopoietic cells, 91% became transfusion independent; the remaining 2 patients had a low level of ...engraftment of vector-expressing cells. With a median of 29.5 months of follow-up, the average hemoglobin level was 11.7 g per deciliter.
Maternal vaccination was 82% effective in preventing medically attended RSV-associated lower respiratory tract illness in infants within 90 days after birth and 69% effective within 180 days.
Patients with neuroblastoma received CAR-positive T cells targeting disialoganglioside. Of 63% with a response, 33% had a complete response (some durable). Cytokine release syndrome was common.
An observational cohort study evaluated immunomodulatory therapy of multisystem inflammatory syndrome in children by comparing IVIG, IVIG plus glucocorticoids, or glucocorticoids alone. The ...investigators found no evidence of the superiority of any of the three therapies, although significant differences may emerge as more data accrue.
An analysis of surveillance data on inpatients younger than 21 years of age who had multisystem inflammatory syndrome in children and were hospitalized between March 15 and October 31, 2020, showed ...that initial treatment with IVIG plus glucocorticoids was associated with a lower risk of cardiovascular dysfunction and a lower incidence of adjunctive therapy use than IVIG alone.
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