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Establishment and validation of a nomogram for predicting intravenous immunoglobulin resistance and coronary artery lesion involvement in Kawasaki disease: a retrospective study.
Clin Rheumatol
January 2025
Department of Pediatrics, Qilu Hospital of Shandong University, Jinan, 250012, China.
Objective: We aimed to develop a useful nomogram for early identification of Kawasaki disease (KD) children at a high risk of intravenous immunoglobulin (IVIG) resistance and coronary artery lesion (CAL) complications to improve KD management.
Methods: Clinical data from 400 patients treated at our hospital between January 1, 2016, and December 31, 2023, were collected. Lasso regression was utilized to screen risk factors for IVIG resistance and CAL involvement.
Dose-intensive therapy (DIT) for infantile Pompe disease: A pilot study.
Mol Genet Metab Rep
March 2025
Department of Pediatrics, University of Minnesota, Minneapolis, MN, USA.
Background: The current standard of care for infantile-onset Pompe disease (IOPD), a severe form of acid α-glucosidase enzyme activity deficiency is: (1) detection by newborn screening, (2) early initiation of intravenous enzyme replacement therapy (ERT) using recombinant human acid alpha-glucosidase (rhGAA), with higher doses of rhGAA increasingly used to improve clinical outcomes, and (3) immune tolerization induction (ITI) using to prevent anti-rhGAA antibody formation, with methotrexate (MTX), rituximab, and IVIG used for patients who are cross-reactive immunologic material negative (CRIM-) and monotherapy with MTX used in patients who are cross-reactive immunologic material positive (CRIM+).
Objectives/methods: A pilot study evaluates a dose-intensive therapy (DIT) using high-dose ERT (40 mg/kg/week) and more frequent exposure to ERT (i.e.
Daratumumab followed by tocilizumab for treatment of late antibody-mediated rejection in renal transplant recipients with high or moderate levels of de novo donor-specific antibodies: a pilot study.
BMC Nephrol
January 2025
Institute of Organ Transplantation, Tongji Hospital, Tongji Medical College, Key Laboratory of Organ Transplantation, NHC Key Laboratory of Organ Transplantation, Key Laboratory of Organ Transplantation, Huazhong University of Science and Technology, Ministry of Education, Chinese Academy of Medical Sciences, Wuhan, China.
Background: Effective treatment of late antibody-mediated rejection (late AMR) is still an unmet medical need. Clearing donor-specific antibody (DSA) and preventing its rebound is the ideal goal of treatment.
Methods: We have summarized the clinical data from seven patients with late or chronic active AMR after renal transplantation who received daratumumab (Dara)-based treatment first (Phase 1) and then tocilizumab (TCZ) therapy (Phase 2).
Post-intrathecal chemotherapy-related paraplegia syndrome in hematological cancer patients: A systematic review.
Neurooncol Adv
December 2024
Center for Translational Immunology, University Medical Center Utrecht, Utrecht, The Netherlands.
Background: Intrathecal (IT) chemotherapy is essential in treating hematological malignancies, but it can lead to ascending paraplegia, a condition that currently lacks clear management guidelines.
Methods: We conducted a systematic review, analyzing 1219 studies and 116 patients, adhering to PRISMA guidelines for individual patient data. The study, registered under PROSPERO (CRD42022362121), focused on the onset, diagnostic approaches, and therapeutic interventions associated with this complication, and management strategies to tackle the ascending paraplegia.
Successful desensitization of donor-specific antibodies in a single cord blood transplant recipient.
Hematology
December 2025
Cellular Therapy & Transplantation Program, Hopital Maisonneuve-Rosemont, Universite de Montreal, Montreal, Quebec, Canada.
Umbilical cord blood (UCB) represents a valuable graft source in the absence of a human leukocyte antigen (HLA)-matched donor for hematopoietic cell transplantation (HCT). Donor-specific anti-HLA antibodies (DSAs), targeting grafts with mismatched HLA antigens, pose a significant obstacle by increasing the risk of primary graft failure, delayed engraftment, and decreased survival. Existing literature on HLA desensitization has primarily focused on haploidentical transplants, and there is a lack of experience regarding the optimal strategy in UCB transplantation.
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