Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What could explain failure to luspatercept in patients with transfusion-dependent beta thalassemia?
Luspatercept works by trapping TGF beta ligands (GDF 11 and 15) and optimizing maturation and differentiation of erythroid precursors, thus facilitating increased output of red cells.In individuals who have beta zero-beta zero thalassemia, this must mean that the cells coming out would contain hemog...
Should special precautions be taken patients with Ehlers-Danlos syndrome receiving radiation therapy?
Not all EDS is the same. Most commonly, an EDS patient these days is a clinically diagnosed patient with hypermobile joints, possibly stretchy skin, and possibly chronic pain syndrome. That is a very different picture from vascular EDS with a COL3A1 mutation, which is rare and would have the extreme...
In patients with lupus nephritis, and MAHA with positive anti-phospholipid autoantibodies, what are the considerations to use or not use anti-coagulation therapy?
I support anticoagulation and C5 inhibition for such patients if they are not adequately responding.
Do you offer fertility preservation to patients prior to CAR-T therapy?
There is no evidence yet about the impact of CAR-T itself on fertility, and this will be difficult to study in isolation in this population. Many CAR-T patients have been heavily pre-treated, so semen cryopreservation and oocyte harvesting may not be successful; but if feasible they should be attemp...
How would you advise a younger patient with residual/recurrent optic nerve meningioma, proceeding with radiotherapy, about the risks of malignant transformation or induction of other brain malignancies because of radiation?
The risk of malignant transformation of an optic nerve sheath meningioma (ONSM) after RT appears to be remarkably low, much lower than the risk of blindness from an untreated, progressive ONSM. In a younger patient, I would lean toward RT for patients with imaging progression or early visual loss, ...
What screening tools or signs do you use to predict if a cancer patient is near end-of-life?
For most of us, long-time practicing oncologists, all we have to do to determine that one of our patients is at the end of their life is to be in the same room with them. No special computer programs or calculators are needed. Just look closely at the patient's current weight, their level of conscio...
What are treatment options for a pediatric patient with PRETEXT IV hepatocellular carcinoma with progressive disease following PLADO and sorafenib and 3 sessions of TACE?
Does this patient definitely have HCC? Or could it be fibrolamellar carcinoma - specifically, did she/he have the DNAJB1-PRKACA fusion? I can help with FLC for sure. I have seen over 250 FLC patients. For HCC, I have only seen about 10, though some do respond to "FLC-like" therapy, such as GEMOX-LEN...
How do you counsel an otherwise healthy patient on how soon they can go back to moderate exercise after a bilateral pulmonary embolism?
Generally, the approach is to have the patient start their exercise regimen at a lower intensity and gradually increase it based on their tolerance.
How should elevated PT of unclear etiology and significance be evaluated?
Mild prolongation of the prothrombin time (PT) may represent a normal ‘outlier’. If there is no obvious explanation for a moderate to marked prolongation of the PT (for example, anticoagulation therapy effect, liver disease, nutritional deficiency like vitamin K deficiency. then the next step is to ...
What are your top takeaways in Hematologic Malignancies from ASH 2025?
The PARADIGM study is a very important one that may result in a paradigm change for the treatment of AML. The study showed that outcomes are equal or better with AZA + VEN among patients with AML suitable for intensive chemotherapy. The efficacy was superior in response rate and EFS (but not overall...