Rare Diseases
From the Journals
Adverse cytogenetics trump molecular risk in NPM1-mutated AML
The findings have implications for postremission treatment decisions, including whether NPM1-mutated patients should receive allogeneic...
News
FDA approves afamelanotide for treatment of rare condition with light-induced pain
This is the first treatment approved to help patients with erythropoietic protoporphyria increase their exposure to light.
Conference Coverage
mIDH1 inhibitor ivosidenib improves progression-free survival in advanced cholangiocarcinoma
BARCELONA - The first-in-class oral inhibitor of mIDH1, significantly improved PFS for advanced cholangiocarcinoma in the global, randomized,...
From the Journals
Anakinra treatment for pediatric ‘cytokine storms’: Does one size fit all?
A retrospective cohort study saw mortality reduced if anakinra was administered early to treat sHLH/MAS. But experts caution that anakinra may not...
From the Journals
Can we eradicate malaria by 2050?
A Lancet Commission report details a series of modeled projections that could point the way.
News
FDA approves Turalio for symptomatic tenosynovial giant cell tumor
Turalio is the first therapy approved for the rare joint tumor and is available only through the Turalio Risk Evaluation and Mitigation (REMS)...
Conference Coverage
Ibrutinib tops chlorambucil against CLL
AMSTERDAM – The Bruton’s tyrosine kinase inhibitor was associated with a halving of risk for death, compared with chlorambucil.
Conference Coverage
Rozanolixizumab may offer new treatment paradigm for ITP
AMSTERDAM – “Data from this study indicate that we can achieve effective increases in platelet levels, we can observe decreasing IgG levels, and...
Conference Coverage
R2 appears active in high-risk FL and MZL
CHICAGO – R2 produced responses in patients who were refractory to rituximab or both lenalidomide and rituximab at baseline.
Conference Coverage
Pazopanib extended PFS in patients with carcinoid tumors
CHICAGO – Significant efficacy results need to be viewed in light of increased toxicity.
News
Pexidartinib gets ODAC nod for tenosynovial giant cell tumor treatment
Missing efficacy data from the pivotal ENLIVEN trial and adverse liver events were points of contention.