Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
How do you approach deciding which patients with hemophilia B to recommend for gene therapy?
Those of us involved in the care of patients with hemophilia feel like” kids in a candy store” with all these new wonderful treatments, as just in the last year we’ve had 3 FDA approvals of rebalancing agents-fitusiran, concizumab, and marstacimab, and the FIX gene therapy product Hemgenix has been ...
Is your standard of care for newly diagnosed advanced classical Hodgkin now nivolumab-AVD based on SWOG 1826?
I think the new standard of care for newly diagnosed patients with advanced classical Hodgkins Lymphoma is now nivolumab-AVD based on the early data from SWOG 1826. While we often wait for more mature data, the early improvement in PFS and the more favorable toxicity profile of this regimen over BvA...
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...
What is your approach to chronic non-immune mediated thrombocytopenia management in children?
There are whole textbook chapters on this topic, and we use a variant of this question to review the differential diagnosis of thrombocytopenia with our fellows as a didactic exercise. So I take from this question, which is an important one, what might be a framework to consider the differential dx ...
What criteria is needed in relapsed/refractory B-cell ALL to choose CAR-T therapy over conventional stem cell transplant as destination therapy?
Currently, there are no standard criteria used to choose CAR-T over conventional stem cell transplant as destination therapy. However, there are many factors that often push us in one direction or the other. As we learn more about outcomes after CAR-T cell therapy, there are many factors we know are...
How would you manage a child with subtotal resection of an atypical choroid plexus papilloma?
Data from the SickKids Hospital study strongly suggest that the extent of resection is prognostic in atypical CPP, and in the presence of GTR, chemotherapy may be avoided or skipped entirely (Browne-Farmer et al., PMID 34529227). The role of adjuvant treatment in the setting of incompletely resected...
When do you consider including Rituximab into the treatment regimen for a pediatric or AYA patient with intermediate and advanced-stage nodular lymphocyte-predominant Hodgkin lymphoma?
First, I would want to make sure the diagnosis is correct as advanced stage NLPHL is extremely rare. T-cell rich B-cell lymphoma can look identical histologically. Either way, R-CHOP therapy is good therapy for both.
How would you manage a recurrent pineoblastoma following prior CSI?
Recurrent pineoblastoma following prior CSI represents a challenging clinical scenario. As there is very little published data to guide optimal management, our approach is adopted from recurrent medulloblastoma. The prognosis for patients with recurrent medulloblastoma/PNET is poor, with 5-year OS <...
Can aplastic anemia present with multiple infiltrative bone lesions, such as in the spine and pelvis?
There are several studies showing diffuse hypointense pictures with an infiltrative-like pattern on MRI in aplastic anemia and MDS. Some aplastic anemia and refractory cytopenia of childhood show patchy pattern of hematopoiesis with some regions having increased fat fraction and some remnants of hem...
What is your therapy of choice for relapsed metastatic alveolar rhabdomyosarcoma?
I will make just a couple of presumptions. First, I will presume that this alveolar rhabdo is FOXO1-fusion positive. If not, I would treat as I would an embryonal (fusion negative) rhabdo with vinorelbine/PO cyclophosphamide, vinorelbine/cyclo/temsirolimus, vincristine/irinotecan +/- temozolomide, c...