pediatric patient / parent

A drug that targets a common mutation in myelodysplastic syndromes (MDS) led to historically high response rates as initial treatment for MDS and acute myeloid leukemia (AML), a small clinical trial showed. Overall, 71% of patients with TP53-mutant conditions responded to the…

Amer Zeidan, MBBS, MHS, Yale University School of Medicine and Yale Cancer Center, New Haven, Connecticut, discusses updated results from an ongoing retrospective study examining FLT3 testing trends, treatment patterns, and overall survival in patients with relapsed/refractory…

Administering 2 additional courses of chemotherapy with high-dose cytarabine (Ara-C) after 2 courses of induction therapy improved relapse rates but did not improve overall survival for patients with acute myeloid leukemia (AML), according to research in the Journal of Clinical…

Ivosidenib, an inhibitor of the mutant isocitrate dehydrogenase 1 (IDH1) enzyme, was well tolerated and associated with deep and durable responses in combination with azacitidine in patients with newly diagnosed acute myeloid leukemia (AML), according to findings published in the…

Results from the phase III PEGASUS trial suggest that the targeted C3 inhibitor pegcetacoplan improved hemoglobin levels better than eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were still anemic despite at least three months of eculizumab therapy…

TP53-mutant myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) represent a molecularly distinct, poor-risk patient subgroup frequently associated with complex karyotypes, high propensity for relapse, and inferior overall survival. Across TP53-altered MDS and AML…

Guadecitabine (SGI-110), when given as maintenance therapy following allogeneic stem cell transplantation (allo-SCT), showed promising relapse-free survival rates (RFS) with manageable safety in high-risk patients with myelodysplastic syndromes/acute myeloid leukemia (MDS/AML)…

Patients who underwent allogeneic bone marrow transplantation (BMT) as children were at increased risk for severe or life-threatening chronic health conditions. These findings were presented during the American Society of Hematology (ASH) 62nd Annual Meeting and Exposition. For…

Treatment with the oral agent showed sustained health-related quality of life compared with placebo in patients with acute myeloid leukemia, according to results of the phase 3 QUAZAR AML-001 trial. Treatment with oral azacitidine (CC-486) showed sustained health-related quality…

Delaying treatment for up to 7 days so that genomic data can be utilized to inform a personalized treatment approach is safe, feasible, and can improve overall survival in patients with acute myeloid leukemia. John C. Byrd, MD Delaying treatment for up to 7 days so that genomic…