Issue Archive

Edited and introduced by Associate Editor Thomas Ortel, this review series features 3 reviews of von Willebrand factor, factor VIII, and factor IX. These 3 proteins are functionally intertwined, and insights into their structure, molecular interactions, and modifications have led to important advancements in therapies for hemophilia A and B and for treatment for thrombotic thrombocytopenic purpura.

Since European LeukemiaNet (ELN) acute myeloid leukemia (AML) genetic risk classification systems are based on patients treated with intensive chemotherapy, Döhner and colleagues, in this month’s CME article, examine the ability of the systems to predict outcomes in older patients treated with low-intensity therapy. The authors analyzed 279 patients treated with venetoclax-azacitidine and 113 patients treated with placebo-azacitidine, demonstrating that the ELN prognostic criteria discriminate poorly as most patients were classified as adverse risk. They applied a bioinformatic algorithm identifying that a novel prognostic classifier based on TP53, FLT3-ITD, NRAS, and KRAS mutations allows discrimination of 3 risk groups with distinct differences in overall survival. In an accompanying Special Report, Döhner et al codify these observations into a new 2024 ELN genetic risk classification for patients with AML receiving less-intensive therapy.

Edited and introduced by Associate Editor Thomas Ortel, this review series features 3 reviews of von Willebrand factor, factor VIII, and factor IX. These 3 proteins are functionally intertwined, and insights into their structure, molecular interactions, and modifications have led to important advancements in therapies for hemophilia A and B and for treatment for thrombotic thrombocytopenic purpura.

Edited and introduced by Associate Editor Thomas Ortel, this review series features 3 reviews of von Willebrand factor, factor VIII, and factor IX. These 3 proteins are functionally intertwined, and insights into their structure, molecular interactions, and modifications have led to important advancements in therapies for hemophilia A and B and for treatment for thrombotic thrombocytopenic purpura.

Edited and introduced by Associate Editor Thomas Ortel, this review series features 3 reviews of von Willebrand factor, factor VIII, and factor IX. These 3 proteins are functionally intertwined, and insights into their structure, molecular interactions, and modifications have led to important advancements in therapies for hemophilia A and B and for treatment for thrombotic thrombocytopenic purpura.

Since European LeukemiaNet (ELN) acute myeloid leukemia (AML) genetic risk classification systems are based on patients treated with intensive chemotherapy, Döhner and colleagues, in this month’s CME article, examine the ability of the systems to predict outcomes in older patients treated with low-intensity therapy. The authors analyzed 279 patients treated with venetoclax-azacitidine and 113 patients treated with placebo-azacitidine, demonstrating that the ELN prognostic criteria discriminate poorly as most patients were classified as adverse risk. They applied a bioinformatic algorithm identifying that a novel prognostic classifier based on TP53, FLT3-ITD, NRAS, and KRAS mutations allows discrimination of 3 risk groups with distinct differences in overall survival. In an accompanying Special Report, Döhner et al codify these observations into a new 2024 ELN genetic risk classification for patients with AML receiving less-intensive therapy.

Strong and colleagues demonstrate in preclinical studies that lipid nanoparticles can transfer mRNA to platelets suspended in plasma and plasma supplemented with platelet additive solution without affecting the functional attributes of the platelets. The ability to transfect platelets in platelet concentrates for transfusion offers the opportunity to use modified platelets with enhanced properties that improve their survival and functionality.

Bhatla and colleagues present results of the DELPHINUS study evaluating daratumumab with chemotherapy in relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (B-ALL), T-cell ALL (T-ALL), and lymphoblastic lymphoma (LL). Treatment of B-ALL was deemed futile and the cohort closed; however, in T-ALL, daratumumab with chemotherapy yielded excellent results, with overall response rates of over 80% and complete responses of 60% in T-ALL. Though numbers were small, daratumumab is likely to prove effective as a bridge to hematopoietic cell transplant for children and young adults with R/R T-ALL/LL.

It is known that plasma fibrinogen levels vary widely and are influenced by genetic polymorphisms, but studies have been focused largely on European populations. Huffman et al performed a meta-analysis of whole genome sequences in 2 large databases encompassing over 160 000 individuals with broad ethnic diversity and identified 18 novel relevant loci not previously identified. Polygenic risk scores based on these and previously identified loci associate with thrombotic and inflammatory phenotypes, providing avenues for further delineation of the connection between coagulation and inflammation.

In this Letter to Blood, Yousfi and colleagues describe a rapid genomic nanopore sequencing assay for thrombotic microangiopathies that reduces genetic diagnosis from over 6 weeks to less than 3 days, is cheaper than standard sequencing, and offers opportunities for rapid initiation of complement blockade.