Medical Oncology
Physician insights on cancer treatment protocols, immunotherapy, targeted therapies, and clinical trial updates.
Recent Discussions
Do you consider splenic response as adequate to judge efficacy of JAK2 inhibitors in myeloproliferative disorders?
It is the basis for regulatory approval for JAKi, and it is a clear measure of efficacy as it is associated with better clinical outcomes and improved QoL. However, it is not the end-all, be-all for endpoints. As we design studies to test new therapies, we need to move beyond SVR and TSS50 to endpoi...
How would you approach chronic isolated severe non-cyclic neutropenia with negative bone marrow and rheumatologic work-up?
My preference would be to use G-CSF at the lowest effective dose, i.e., the dose that maintains the ANC >1000. The risk of inducing leukemia is low in individuals with idiopathic, chronic neutropenia, but it is positively correlated with the G-CSF dose. There are also other side effects like bone pa...
What further genetic testing would you perform on a pediatric patient with cyclic neutropenia who is negative for the most common associated mutations?
Virtually all autosomal dominant cyclic neutropenia is due to mutation in the ELANE gene. However, all neutropenia that varies over time is not necessarily hereditary cyclic neutropenia, even though the family history suggests so. It would be helpful to know if the family history follows an AD inher...
With new data showing similar outcomes of mismatched unrelated donor and haploidentical related donor allogeneic transplants using post transplant cyclophosphamide, how does one decide which donor is optimal?
It is certainly an unresolved question, and the short answer is that either would be an acceptable option with post-transplant cyclophosphamide (and I have used both). All recipients of mismatched donor transplants should be tested for the presence of donor specific antibodies, and their presence co...
How do you approach evaluation of pulmonary vein thrombosis?
Pulmonary vein thrombosis is a rare condition that is usually associated with injury to the vessel, for example after surgery or lung transplantation, or as a complication of lung cancer. I am not aware of any data on the relative efficacy of different anticoagulants in PVT but from a biologic persp...
Would you consider bone marrow transplant in a pediatric patient with recurrent HLH with no identified exogenous trigger or HLH-associated mutation?
What factors besides disease progression would lead you to de-intensify or change MM therapy for a patient with ongoing response?
After initial therapy (which may or may not include an autologous stem cell transplant), patients are generally on continuous/maintenance therapy with e.g. lenalidomide as the most common regimen. Most patients do well on this as maintenance. However, loose stools are probably the most common compla...