Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What is your recommended long term management of extensive dural venous sinus thrombosis in an otherwise healthy young patient?
From a Vascular Neurology perspective, there are two considerations when faced with patients with venous sinus thrombosis. First is the status of the intracranial venous circulation and whether or not there has been adequate reconstitution of venous outflow, either through recanalization or collater...
Do you use direct oral anticoagulants to treat port-a-cath related VTE in patients with an active malignancy?
The initial trials that established DOACs as effective and safe in most patients with cancer-associated thrombosis (Agnelli et al., PMID 32223112, Planquette et al., PMID 34627853) only included patients with lower-extremity DVT or PE but clearly showed equivalence to low-molecular weight heparin (w...
Do you screen for VWD in all women with menorrhagia severe enough to cause iron deficiency anemia?
Yes, testing vWF is needed in this situation of severe menometrorrhagia. Excessive menstrual bleeding, especially when severe enough to cause anemia, is a common presenting symptom of vWD in women. The testing should be done as part of a comprehensive workup for gynecologic etiologies and other coag...
What would your next line of treatment be for a patient with Hb SC with history of recurrent VOC and new bone infarct who is already on hydroxyurea and phlebotomy?
HbSC disease is the stepchild of sickle hemoglobinopathies with almost no studies of its treatment, so that any recommendation is not data based. One might consider adding crizanlizumab that blocks red cell-endothelial cell adherence. Each treatment group of the crizanlizumab study that led to its F...
In patients with VOD post-HSCT, what would be the role of TPO agonists instead of platelet transfusions, given their platelet refractoriness and concerns for fluid overload?
This is an insightful question, as it addresses a key issue in the management of veno-occlusive disease (VOD) - chiefly, how does one minimize the amount of fluid being given, but at the same time, support patients with persistently low platelet counts, the majority of whom will be on a therapy whic...
How would you treat suspected secondary HLH in patients whom etoposide is contraindicated?
Thank you for the interesting question. It would be helpful to understand why etoposide is contraindicated. My general approach for these patients is to first ensure that there is no evidence of malignancy as a trigger as you do not want to mask that with steroids (I strongly recommend obtaining a P...
How do different inflammatory markers like CRP and ferritin contribute differently, if at all, to the monitoring of CART neurotoxicity?
These markers are routinely monitored as they are seen in association with CRS (cytokine release syndrome). Not all patients with CRS will also develop neurotoxicity (ICANS), but most patients with ICANS have antecedent CRS, so in an encephalopathic patient post-CART who does not have significant el...
When would you use proximal complement inhibitors like pegcetacoplan over terminal complement inhibitors for initial treatment of paroxysmal nocturnal hemoglobinuria?
I use either iptacopan or pegcetacoplan as primary therapy for PNH these days. Since its release, I have used mainly iptacopan. Its advantages are 1) it is oral, 2) the side effect profile is very tolerable, 3) it only inhibits the alternative pathway, thus is likely to be associated with less risk ...
What treatment would you give to a pediatric patient with rhabdomyosarcoma in first relapse?
Typically it depends on the previous treatment of the patient’s primary RMS in terms of my approach to relapse RMS. In general, many would go with treatment per ARST0921 with vinorelbine, cyclophosphamide, and Temsirolimus, with the caveat that it may be different depending on previous drug exposure...
Do you screen children with sickle cell disease for silent cerebral infarcts?
We screen children with sickle cell disease for SCIs at our center. This is because data suggest that silent cerebral infarcts (SCIs) are much more common than overt stroke and the long-term negative impacts of SCIs are significant. There are also treatments (e.g., chronic transfusion and stem cell ...