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Hematopoietic stem cell transplantation as rescue therapy for refractory autoimmune retinopathy: a case report.
Front Immunol
January 2025
Department of Ophthalmology, National University Hospital, National University Health System, Singapore, Singapore.
Autoimmune retinopathy (AIR) is a rare, potentially blinding retinal disease that remains a challenging condition to manage when resistant to conventional immune-modulatory approaches. We report clinical and electrophysiological improvement in a 49-year-old patient who underwent an autologous hematopoietic stem cell transplant (aHSCT) for thymoma-associated AIR after experiencing progressive disease despite receiving periocular and systemic steroids, mycophenolate mofetil, baricitinib, tacrolimus, bortezomib, rituximab, plasmapheresis, and intravenous immunoglobulin. The aHSCT had two stages: (i) peripheral blood stem cell harvest following mobilization with cyclophosphamide and granulocyte colony-stimulating factor, and (ii) conditioning regimen with plasmapheresis, rituximab, cyclophosphamide, and anti-thymocyte globulin high-dose therapy, followed by autologous hematopoietic cell infusion of 5.
View Article and Find Full Text PDFThe effects of plerixafor on the hemostatic system in patients undergoing stem cell mobilization.
Hematol Oncol Stem Cell Ther
January 2025
Department of Hematology, Ankara University School of Medicine, Ankara, Turkey.
Despite numerous reports on the procoagulant activities of G-CSF, the effect of plerixafor on the hemostatic system is not clearly understood. This study aims to evaluate the effects of plerixafor on the hemostatic system when used for autologous stem cell mobilization (ASCM) for poor mobilizers (PM) with lymphoma and multiple myeloma. Patients who were performed ASCM with plerixafor in combination with GCSF were prospectively enrolled.
View Article and Find Full Text PDFPEGylated Granulocyte Colony-Stimulating Factor and Plerixafor Enhance Autologous Stem and Progenitor Cell Mobilization and Transplantation in Pediatric Patients.
Stem Cells Dev
January 2025
Princess Máxima Center for Pediatric Oncology, Utrecht, The Netherlands.
Autologous hematopoietic stem cell transplantation is used to restore bone marrow function after high-dose chemotherapy. For apheresis, granulocyte colony-stimulating factor (G-CSF) is standard of care, but obtaining sufficient stem cells can be challenging. Other mobilization agents include plerixafor and PEGylated G-CSF (PEG-G-CSF).
View Article and Find Full Text PDFEnhanced Recovery After Surgery for patients undergoing radical cystectomy: Surgeons' perspectives and recommendations ten years after its implementation.
Eur J Surg Oncol
December 2024
Oncologic Minimally Invasive Urology and Andrology Unit, Department of Experimental and Clinical Medicine, Careggi Hospital, University of Florence, Florence, Italy.
Background And Objectives: Enhanced Recovery After Surgery (ERAS) guidelines for Radical Cystectomy (RC) were published over ten years ago. Aim of this systematic review is to update ERAS recommendations for patients undergoing RC and to give an expert opinion on the relevance of each single ERAS item.
Methods: A systematic review was performed to identify the impact of each single ERAS item on RC outcomes.
Lentivirus-mediated gene therapy for Fabry disease: 5-year End-of-Study results from the Canadian FACTs trial.
Clin Transl Med
January 2025
Department of Pediatrics, Medical College of Wisconsin, Milwaukee, Wisconsin, USA.
Background: Fabry disease is an X-linked lysosomal storage disorder due to a deficiency of α-galactosidase A (α-gal A) activity. Our goal was to correct the enzyme deficiency in Fabry patients by transferring the cDNA for α-gal A into their CD34+ hematopoietic stem/progenitor cells (HSPCs). Overexpression of α-gal A leads to secretion of the hydrolase; which can be taken up and used by uncorrected bystander cells.
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