GSE78963 A Multicenter, First-in-Pediatrics, Randomized Study to Evaluate the Safety and Efficacy of Decitabine as Epigenetic Priming with Induction Chemotherapy in Children with Acute Myelogenous Leukemia
Conclusions: This first-in-pediatric trial of epigenetic priming in patients with newly diagnosed AML demonstrates that decitabine pre-treatment followed by standard combination chemotherapy is well tolerated in children with newly diagnosed AML. Morphologic complete responses were similar in both treatment arms. MRD at Day 30 following induction therapy suggests a deeper remission in patients receiving decitabine. No differences were observed between treatment arms in hematologic toxicities although decitabine-treated patients were noted to have more gastrointestinal toxicities, anorexia and hypophosphatemia. Decitabine PK parameters in children were consistent with known adult PK profiles. Molecular changes associated with decitabine pretreatment may be important in the sensitization of clonogenic AML cells. Pre-treatment with decitabine may represent a therapeutic option for use in pediatric AML, when epigenetic modification is a desired focus for treatment.
Discussion: Both fetoscopic and US-guided IUHCT were technically feasible, but fetoscopy caused more intraoperative complications in our pilot series. The discrepancy in chimerism detection predicts the challenges in long-term surveillance of donor-cell chimerism. Further studies of long-term outcomes in the non-human primate are valuable for the development of clinical protocols for IUHCT.Fetal Diagn Ther
Fecal transplant is used to treat gut infections and is now being studied as a treatment for obesity, urinary tract infections, irritable bowel syndrome and more.
Wider use of donation after circulatory death donors may increase transplant numbers substantially. Most countries that have adopted donation after circulatory death donation have focused on controlled donation after circulatory death donors, whereby life-supporting treatment is withdrawn in a coordinated manner. In this issue, del R ío and colleagues report the Spanish experience for kidneys transplanted from uncontrolled donation after circulatory death donors (typically individuals with sudden cardiorespiratory arrest in the community).
Effective therapies for Ig light chain (AL) amyloidosis has led to an increasing proportion of patients with end-stage renal disease requiring renal replacement therapy, yet kidney transplantation is seldom performed in this setting due to concerns of renal and extrarenal disease progression. Angel-Korman et al. report unprecedented positive long-term outcomes in the largest series of kidney transplantation in AL amyloidosis providing the basis for a more proactive approach to this procedure.
Clinical trial compares stem cell transplantation with existing disease-modifying therapies and finds that the former is more effective at slowing down MS.
This investigation highlights the serious potential risks to patients of stem cell therapies administered for unapproved and unproven uses other than hematopoietic or immunologic reconstitution.Morbidity &Mortality Weekly Report
Amanda C. Herrmann, Jin S. Im, Sumedha Pareek, Wilfredo Ruiz-Vasquez, Sijie Lu, Anna Sergeeva, Jennifer Mehrens, Hong He, Gheath Alatrash, Pariya Sukhumalchandra, Lisa St. John, Karen Clise-Dwyer, Dongxing Zha, Jeffrey J. Molldrem
We present a review of TAC therapy in AIH patients. METHODS: A search for studies with keywords 'autoimmune hepatitis' and 'tacrolimus' was performed. Reviews, studies of AIH post-transplant and AIH in children were excluded. Diagnosis of AIH was based on criteria established by the International Autoimmune Hepatitis Group. Complete biochemical response was defined as normalisation of aspartate aminotransferase (AST
AbstractPurpose of ReviewThis review focuses on the recent clinical development of indolamine-2,3-dioxygenase-1 (IDO-1) inhibitors.Recent FindingsIDO-1 alters tryptophan metabolism in a manner enhancing T-regulatory cell activity, but pre-clinical data show that its role in tumorigenesis is context-dependent on host and tumor interaction, highlighting some challenges in understanding the molecular oncology of this enzymatic drug target. Because results from phase I/II trials of IDO-1 inhibitor monotherapy have been disappointing, current clinical trials employ IDO-1 inhibitors in combination strategies with other immunothe...
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