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Current status and perspectives of hematopoietic cell transplantation in patients with paroxysmal nocturnal hemoglobinuria.
Front Immunol
January 2025
Department of Hematology and Transplantology, Medical University of Gdansk, Gdansk, Poland.
Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare complement-driven acquired hemolytic anemia with specific presentations of hemoglobinuria, abdominal pain, fatigue, and thrombosis.
Objective: To review the current therapeutic strategies for PNH, including anti-complement therapy and allogeneic hematopoietic cell transplantation (alloHCT), focusing on the tailoring of the approach to the disease subtype.
Results: The outcome of alloHCT varies depending on disease severity, thrombotic history, and response to prior therapies.
Targeting the chemokines in acute graft-versus-host disease.
Front Immunol
January 2025
Institute of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) constitutes a critical therapeutic approach for patients with malignant hematological disorders. Nevertheless, acute graft-versus-host disease (GVHD), one of the most prevalent complications associated with HSCT, remains a leading contributor to non-relapse mortality. In recent years, there has been an increasing focus on the interplay between chemokines and their receptors in the context of acute GVHD.
View Article and Find Full Text PDFIntra-mesenteric steroids for steroid-refractory graft-versus-host disease in pediatric patients: A safe option.
Biomedica
December 2024
Servicio de Cardiología Pediátrica, Departamento Materno-Infantil, Fundación Valle del Lili, Cali, Colombia; Facultad de Ciencias de la Salud, Universidad Icesi, Cali, Colombia.
Introduction: Graft-versus-host disease is a serious complication after hematopoietic stem cell transplantation and is a major cause of death post-transplantation. Approximately 50% of acute graft-versus-host disease patients do not respond to systemic steroids and their prognosis is poor regardless of the treatment. This study describes our experience with pediatric patients diagnosed with steroid-refractory graft-versus-host disease who received intra-mesenteric steroid treatment.
View Article and Find Full Text PDFHaploidentical hematopoietic stem cell transplantation using post-transplant cyclophosphamide in patients with inborn errors of immunity: Experience in a reference center in Colombia.
Biomedica
December 2024
acultad de Ciencias de la Salud, Universidad ICESI, Cali, Colombia; Servicio de Alergología e Inmunología Pediátrica, Departamento Materno-Infantil, Fundación Valle del Lili, Cali, Colombia.
Introduction: Inborn errors of immunity is a diverse group of rare diseases caused by over 400 genetic mutations affecting the immune system and increasing infection susceptibility, autoimmunity, and malignancy. Hematopoietic stem cell transplantation offers a curative option for some inborn errors of immunity, with haploidentical donors providing a viable alternative when identical donors are unavailable.
Objective: To determine survival, usefulness of weekly chimerism monitoring, immune reconstitution, and complications in patients with inborn errors of immunity who underwent haploidentical hematopoietic stem cell transplantation at a reference center in Colombia.
Generation of Human Chimeric Antigen Receptor Regulatory T Cells.
J Vis Exp
January 2025
Department of Microbiology and Immunology, Medical University of South Carolina; Department of Regenerative Medicine and Cell Biology, Medical University of South Carolina; Hollings Cancer Center, Medical University of South Carolina;
Chimeric antigen receptor (CAR) T-cell therapy has reshaped the face of cancer treatment, leading to record remission rates in previously incurable hematological cancers. These successes have spurred interest in adapting the CAR platform to a small yet pivotal subset of CD4 T cells primarily responsible for regulating and inhibiting the immune response, regulatory T cells (Tregs). The ability to redirect Tregs' immunosuppressive activity to any extracellular target has enormous implications for creating cell therapies for autoimmune disease, organ transplant rejection, and graft-versus-host disease.
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