Publications by authors named "Kelly McCaul"

Article Synopsis
  • The ACE-536-MF-001 trial studied the effects of luspatercept on myelofibrosis patients, enrolling 95 participants divided into four groups based on their dependency on transfusions and anemia status.
  • The primary outcome measured was the anemia response rate, where 14%-26% of patients met the criteria based on their cohort, with significant hemoglobin increases noted, particularly in non-transfusion dependent patients.
  • Adverse events were common, with 94% of patients experiencing at least one; the most frequent were hypertension and other manageable issues, while the treatment showed consistent efficacy and safety results similar to previous studies.
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Background: PNT2258 is a liposomal formulation that encapsulates multiple copies of PNT100, a native, chemically unmodified, 24-base DNA oligonucleotide designed to target the regulatory region upstream of the B-cell lymphoma 2 (BCL2) gene.

Methods: This phase II, multicenter, single-arm, open-label, 2-stage design study investigated the single-agent activity of PNT2258 in patients with relapsed/refractory DLBCL. Initially, patients had to have a performance status (PS) of ≤2 and prior exposure to CD20-targeted therapy, an alkylating agent, and a steroid with no upper limit.

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The term hemolytic uremic syndrome (HUS) refers to a heterogenous group of disorders arising from an initial endothelial cell injury with fibrin and platelet thrombi formation in the vasculature, leading to severe organ damage resulting in the well-known triad of microangiopathic hemolytic anemia, thrombocytopenia and kidney disease. The majority of pediatric cases (90 percent) of HUS are caused by Shiga toxin-producing Escherichia coli (STEC-HUS) or Shigella dysenteriae and rarely with Streptococcus pneumoniae (Pneumococcal-HUS). Atypical hemolytic uremic syndrome (aHUS) constitute only 5 percent of all HUS cases and are mediated by dysregulation of complement proteins.

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Therapy of acute myeloid leukemia in older persons is associated with poor outcomes because of intolerance to intensive therapy, resistant disease and co-morbidities. This multi-center, randomized, open-label, phase II trial compared safety and efficacy of three therapeutic strategies in patients 65 years or over with newly-diagnosed acute myeloid leukemia: 1) continuous high-dose lenalidomide (n=15); 2) sequential azacitidine and lenalidomide (n=39); and 3) azacitidine only (n=34). The efficacy end point was 1-year survival.

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A discrepancy between clinical findings and a markedly elevated testosterone (T) level stimulated search to explain this inconsistency. The cause of the falsely elevated T level was determined to be heterophile antibodies from a polyclonal gammopathy in a subject with acute myelogenous leukemia.

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