A bit of antithymocyte globulin can take you a long way!

Mohty, Mohamad

doi:10.1038/bmt.2012.6

Cited by 3 publications

(1 citation statement)

References 8 publications

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“…Our study is limited by its small size. Furthermore, the addition of rATG in patients receiving grafts from unrelated donor, based on several studies favoring its use [35], makes the interpretation of results more challenging. Nevertheless, overall we demonstrated the feasibility and safety of the combination of PTC and bortezomib for the prevention of GVHD after peripheral blood AHSCT.…”

Section: Discussionmentioning

confidence: 99%

Calcineurin and mTOR Inhibitor–Free Post-Transplantation Cyclophosphamide and Bortezomib Combination for Graft-versus-Host Disease Prevention after Peripheral Blood Allogeneic Hematopoietic Stem Cell Transplantation: A Phase I/II Study

Al-Homsi

Cole

Muilenburg

et al. 2017

Biology of Blood and Marrow Transplantation

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Graft-versus-host disease (GVHD) hampers the utility of allogeneic hematopoietic stem cell transplantation (AHSCT). The purpose of this study was to determine the feasibility, safety, and efficacy of a novel combination of post-transplantation cyclophosphamide (PTC) and bortezomib for the prevention of GVHD. Patients undergoing peripheral blood AHSCT for hematological malignancies after reduced-intensity conditioning with grafts from HLA-matched related or unrelated donors were enrolled in a phase I/II clinical trial. Patients received a fixed dose of PTC and an increasing dose of bortezomib in 3 cohorts, from .7 to 1 and then to 1.3 mg/m, administered 6 hours after graft infusion and 72 hours thereafter, during phase I. The study was then extended at the higher dose in phase II for a total of 28 patients. No graft failure and no unexpected grade ≥3 nonhematologic toxicities were encountered. The median times to neutrophil and platelet engraftment were 16 and 27 days, respectively. Day +100 treatment-related mortality was 3.6% (95% confidence interval [CI], .2% to 15.7%). The cumulative incidences of grades II to IV and grades III and IV acute GVHD were 35.9% (95% CI, 18.6% to 53.6%) and 11.7% (95% CI, 2.8% to 27.5%), respectively. The incidence of chronic GVHD was 27% (95% CI, 11.4% to 45.3%). Progression-free survival, overall survival, and GVHD and relapse-free survival rates were 50% (95% CI, 30.6% to 66.6%), 50.8% (95% CI, 30.1% to 68.2%), and 37.7% (95% CI, 20.1% to 55.3%), respectively. Immune reconstitution, measured by CD3, CD4, and CD8 recovery, was prompt. The combination of PTC and bortezomib for the prevention of GVHD is feasible, safe, and yields promising results. The combination warrants further examination in a multi-institutional trial.

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Section: Discussionmentioning

confidence: 99%

Calcineurin and mTOR Inhibitor–Free Post-Transplantation Cyclophosphamide and Bortezomib Combination for Graft-versus-Host Disease Prevention after Peripheral Blood Allogeneic Hematopoietic Stem Cell Transplantation: A Phase I/II Study

Al-Homsi

Cole

Muilenburg

et al. 2017

Biology of Blood and Marrow Transplantation

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Hemoglobinopathies (Sickle Cell Disease and Thalassemia)

et al. 2018

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Sickle cell disease (SCD) is the most common inherited hemoglobinopathy worldwide. It results from a single-nucleotide substitution that leads to a propensity toward hemoglobin polymerization and sickling of red blood cells. Sickle cell disease is characterized by anemia, ongoing hemolysis, and acute and chronic vaso-occlusive complications affecting multiple organs. SCD affects over 100,000 Americans, and it occurs in about one in 500 African-American births and in one in every 1000-1400 Hispanic-American births (NIH 2014;Piel et al. 2013).The implementation of newborn screening, penicillin prophylaxis, vaccination programs, narcotics, chronic transfusions, hydroxyurea, and the early detection of cerebral vasculopathy with transcranial Doppler (TCD) have improved the perspective for children with SCD (Angelucci

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Hematopoietic stem cell transplantation in patients with transfusion-dependent β-thalassemia. Review article

Тайшихина

Лохматова

Shelikhova

2020

Voprosy gematologii/onkologii i immunopatologii v pediatrii

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Thalassemia is the most common form of hereditary anemia from the hemoglobinopathy group. The genetic disorder underlying thalassemia leads to impaired erythrocyte maturation, hemolysis, and the development of ineffective erythropoiesis with erythroid gland hyperplasia in the bone marrow and extramedullary. Regular blood transfusions and chelator therapy are standard therapy for patients with b-thalassemia. This method increases life expectancy, but does not improve its quality and does not cure the disease. Currently, allogeneic hematopoietic stem cell transplantation remains the only radical treatment for thalassemia. The paper discusses the historical aspects of the development of allogeneic hematopoietic stem cell transplantation in the context of transfusion-dependent form of b-thalassemia treatment.

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A bit of antithymocyte globulin can take you a long way!

Cited by 3 publications

References 8 publications

Calcineurin and mTOR Inhibitor–Free Post-Transplantation Cyclophosphamide and Bortezomib Combination for Graft-versus-Host Disease Prevention after Peripheral Blood Allogeneic Hematopoietic Stem Cell Transplantation: A Phase I/II Study

Calcineurin and mTOR Inhibitor–Free Post-Transplantation Cyclophosphamide and Bortezomib Combination for Graft-versus-Host Disease Prevention after Peripheral Blood Allogeneic Hematopoietic Stem Cell Transplantation: A Phase I/II Study

Hemoglobinopathies (Sickle Cell Disease and Thalassemia)

Hematopoietic stem cell transplantation in patients with transfusion-dependent β-thalassemia. Review article

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