Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
Do you use prophylactic defibrotide in pediatric patients undergoing BMT who are considered to be at high risk for the development of VOD/SOS?
Prophylactic defibrotide can indeed be used in pediatric patients undergoing bone marrow transplant (BMT) who are considered to be at high risk for the development of veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS).Studies, including those leading to defibrotide’s FDA approval for V...
For pediatric patients with iron overload (high ferritin and transferrin saturation), do you perform HFE screening first, or proceed to non-HFE gene sequencing upfront to evaluate for HJV mutation as well?
Because there is no recommended "screening" scenario for iron overload in pediatrics (especially with no family history), the question for me revolves around "why did the patient get tested in the first place?" If it were a routine screen for iron deficiency, which affects millions of children at an...
What are your top takeaways in Classical Hematology from ASH 2025?
My top 3 takeaways in classical hematology:1) ITP - much controversy at the education program session about the draft 2025 guidelines, where TPO mimetic + steroids is going to be recommended in the front line. There does not appear to be compelling new data that supports the revision of this guideli...
What are some alternatives to dexamethasone for brain edema in patients who are allergic, have an intolerance, or refuse the medication?
Dexamethasone is one of the most frequently prescribed medications in neuro-oncology clinics. Dexamethasone is often favored over other corticosteroids owing to its lower mineralocorticoid effects and high potency as well as essentially 1:1 oral to IV ratio meaning that we use similar IV and oral do...
How would you manage HHT with ongoing epistaxis despite IV bevacizumab, with a history of superficial thrombosis?
This is a complex situation with a complex answer, and the patient would likely benefit from establishing care at one of the 30 HHT centers of excellence for a more thorough evaluation. If the patient mentioned has only had cautery, we would consider them seeing an ENT at an HHT center of excellence...
What are your top takeaways from ISTH 2025?
MAYARI trial - this trial was a single-arm trial for "non-severe" iTTP (excluding patients with significant cardiac or neurologic involvement) where PLEX was only used as a rescue therapy. Patients were started on caplacizumab and immunosuppression with steroids and rituximab. A large number of pa...
What is your preferred strategy for young adults with ITP complicated by recurrent autoimmune neutropenia?
This is a great question, and I'll say that AIN can be particularly difficult to treat! I'd first ask how low the ANC is and if the patient is presenting with frequent infections/hospitalizations. If not, there may not be a need to treat the AIN (we may just be treating ourselves); oftentimes, there...
What is the best way to prepare children with von Willebrand disease for tonsillectomy to reduce the risk of post-operative hemorrhage?
I am an adult provider, but the treatment is similar regardless of age. The exact treatment plan will vary depending on the type and severity of VWD (i.e., mild type 1 VWD vs type 2B VWD); however, for tonsillectomies, I provide VWF concentrate 40-80 IU/kg 5-30 min preoperatively, followed by postop...
What is your target Hgb/Hct for women who are pregnant and have sickle cell disease in whom you are doing scheduled transfusions?
Hg 10-11. My main goal is to suppress reticulocytosis and therefore, the production of more sickled cells.
At what point would you recommend initiating psychotherapy or psychotropic medications to manage mood and anxiety symptoms following a new oncology diagnosis in pediatric patients?
Perhaps the most important piece to this question is missing, and that is that more history is required to determine what to do with a recently diagnosed cancer patient. What was the patient’s psychiatric status pre-cancer? If the patient had undiagnosed mental health problems–anxiety or depressio...