- MNT is needed for MYC-driven lymphomagenesis
- Pathogenicity of rare spliceosomal gene mutations
- Circulating heme, hemopexin, and kidney injury in sickle cell disease
- Eculizumab for HSCT-associated thrombotic microangiopathy
- Ruxolitinib, weight gain, and leptin signaling
- How I Treat: pegasparaginase toxicity
This week’s episode will review data on spliceosomal gene mutations, evaluate the impact of Ruxolitinib on weight gain, and assess the effect of compliment blockade with eculizumab on high risk patients with stem cell transplant associated thrombotic microangiopathy.
Avadomide monotherapy in relapsed/refractory DLBCL: safety, efficacy, and a predictive gene classifier
Leveraging gene expression subgroups to classify DLBCL patients and select for clinical benefit from a novel agent Avadomide is a novelAvadomide is a novel cereblon-modulating drug that both kills lymphoma cells directly and stimulates T cells and natural killer cells. Carpio et al describe the safety and preliminary efficacy of avadomide in relapsed diffuse large B-cell lymphoma (DLBCL) and how immune cell composition is associated with differential responses. The companion article by Risueño et al details a new gene expression classifier that discriminates DLBCL based on tumor and immune-cell composition and that may assist in identifying patients most likely to respond to immunomodulatory therapy, such as avadomide.
Complement blockade for TA-TMA: lessons learned from a large pediatric cohort treated with eculizumab
Jodele et al detail their experience with 64 pediatric patients with stem cell transplant–associated thrombotic microangiopathy (TA-TMA) and end organ damage treated with the complement blocker eculizumab. They report improved outcomes with brief, intensive therapy in comparison with historical controls.
Rare and private spliceosomal gene mutations drive partial, complete, and dual phenocopies of hotspot alterations
Mutations in the RNA splicing factors SF3B1, SRSF2, and U2AF1 are common in myeloid neoplasms and usually occur at specific hotspots. Pangallo et al studied rare and private mutations in these genes and demonstrated that most of them are also pathogenic, frequently phenocopying hotspot mutations.
Mollé et al investigated the patterns of weight gain associated with ruxolitinib therapy in patients with myeloproliferative neoplasms, defined high-risk groups for treatment-acquired obesity, and defined a plausible mechanism in mouse models.
Pegasparaginase is a key drug in curative regimens for acute lymphoblastic leukemia (ALL), but has unique toxicities that may be unfamiliar to adult hemato-oncologists. Using 5 cases of adults with ALL who were treated with pediatrically inspired regimens, Aldoss and Douer illustrate the management of several pegasparaginase-associated adverse effects and guide whether and how to continue the drug.