Bone marrow failure in children and young adults is often suspected to be inherited; however, in many cases, diagnosis remains uncertain. A new study involving 179 patients (from 173 families) found that implementing precision medicine at diagnosis can improve patient management, along with the use of family counseling. This study published in Blood expands the […]
In this series from Blood that tackles four cases studies, the outcomes of AML pediatric patients compared to children with ALL is explored, focusing on the emerging new treatments venetoclax (Venclexta; AbbVie/Genentech), CD33- and CD123-directed CAR T-cell therapy, CD123-directed antibody therapy, and menin inhibitors.
Key Points: In two study cohorts, less-intensive therapies increased mortality in each of three risk groups defined by age, comorbidities, and cytogenetics. The differences became nonsignificant after accounting for physician perception of cure, emphasizing the need for a randomized trial.Design: Multicenter retrospective cohort (1,292), 6 institutions, 2008-2012; prospective cohort (695), 13 institutions, 2013 – 2017Connected […]
Publication: Blood Key Points: The authors identified the following four prognostic indicators that could help control CNS relapse in children with ALL. 1) Prephase dexamethasone treatment, 2) delayed intrathecal therapy, 3) use of total intravenous anesthesia during intrathecal therapy, and 4) flow cytometry examination of diagnostic CSF Design: 7,640 consecutive patients treated on Chinese Children’s […]
Letermovir is a recently approved antiviral shown to decrease clinically significant cytomegalovirus (CMV) infection after hematopoietic cell transplantation (HCT) in a phase 3, randomized, placebo-controlled trial. Appearing in Blood, the authors make two key points: 1) Letermovir prophylaxis may be associated with decreased polyfunctional CMV-specific T-cell immunity after HCT, and 2) COMPASS is an analytical […]
Published in Blood, this article presents initial findings from a proof-of-concept study led by Martin Wermke, MD, from the Technical University of Dresden in Germany. The UniCAR platform uses targeting molecule TM123 and a single-chain variable fragment that is directed against the CD123 antigen, offering a potential new treatment option for patients with relapsed/refractory acute […]
Warm autoimmune hemolytic anemia (wAIHA) is the most prevalent form of autoimmune hemolytic anemia (AIHA), accounting for 60% to 70% of all cases. In this special instructional series from Blood (ASH), five patient case studies and commentaries are examined, backed by visual illustrations and new/existing drug treatment tables for wAIHA, wAIHA secondary to CLL, and […]
In the past decade, several promising immunotherapies have been developed, changing the treatment landscape for children with relapsed acute lymphoblastic leukemia (ALL). These advances in treatment offer real promise for less toxic and more effective therapy for children. In this article from Blood, the authors present several cases highlighting contemporary treatment decision-making.
In this “How I Treat” feature from Blood, the authors conclude with three representative cases to both illustrate the application of the outlined diagnostic schema and discuss common management considerations, specifically the selection of anticoagulation vs. antiplatelet therapy for secondary prevention.