Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
How do you monitor risk of erythrocytosis from testosterone use for female to male transgender patients?
I utilize the Endocrine Society's guidelines for identifying secondary erythrocytosis secondary to gender affirming hormone therapy (GAHT) (PMID 28945902). For initial monitoring, at baseline and then every 3 month hematocrit for the first year and 1-2 times yearly thereafter is typically implemente...
How do you approach and manage anorexia and appetite loss in people with advanced cancer?
Anorexia/cachexia is often distressing to patients and families and it is this distress that is the target of many of the interventions for this syndrome as there are, in general, no effective therapies. Patients and families are routinely battling over the lack of eating as this causes further disc...
How would you approach treatment in a patient with Fanconi anemia and glioblastoma?
This is challenging due to the sensitivity of Fanconi anemia patients to DNA-damaging treatment. I would maximize resection if possible and then treat with radiation, since it is a mainstay of therapy, despite the risk. I would opt for proton radiation if possible to minimize exposure of normal tiss...
Do you consider travel as a risk factor for DVTs in the absence of any other underlying predisposing factors?
Unfortunately, there are no strong guidelines around this topic. In general, I do not count long car rides as a risk factor unless there is something extremely unusual about the trip. Most people are going to get out and stretch their legs every couple of hours. Long flights are different. There are...
What is the treatment of choice for mixed phenotype ALL?
These are difficult to treat. The limited available data suggest that an ALL–like regimen followed by HSCT may be recommended. If no response is achieved, you could try a myeloid-like strategy. For T/Myeloid, combining asparaginase with AML therapy (FA + PEG or Capizzi II) could be an option. The sp...
Which chemo regimen, if any, would you use as adjuvant treatment in a young patient with primary malignant thyroid teratoma?
There is no evidence base to guide you here. The term "malignant teratoma" is not particularly helpful here and does not distinguish between benign teratoma, somatic transformation, or active germ cell malignancies. If classic markers AFP and HCG are significantly elevated, that would favor a malign...
How do you integrate surgery in treatment of recurrent tenosynovial giant cell tumors (TGCT) when using Pexidartinib?
This is an interesting question, but one that has little (if any) data to guide management. With recurrent TGCT, the patient presumably already had surgery. It is possible that a good response to pexidartinib may reduce the morbidity of a repeat surgery but the risk of additional recurrence would re...
What is the choice of treatment for a teenager with low risk neuroblastoma s/p resection?
According to the Revised Neuroblastoma Risk Classification (Irwin et al., PMID 34319759), age is not a factor in determining whether someone is low risk. So if this child has been determined to have low-risk neuroblastoma, they have/had an L1 tumor, either without MYCN amplification, or with MYCN am...
How do you work up a young patient with increasing ferritin and normal TSAT without infectious, inflammatory, or liver disorders?
This is a tough one. If the ferritin is increasing and the TSAT is normal and there is no evidence of hemochromatosis, the ONLY possible explanation is some underlying morbidity, inflammatory, rheumatologic, malignant, or infectious is present. In pediatrics, HLH (hemophagocytic lymphohistiocytosi...
When using daratumumab in the setting of post-transplant red cell aplasia or post-transplant immune-mediated cytopenias, do you hold the drug for certain ANC parameters?
Typically, we do not hold until below ANC 500.