Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
In a patient hospitalized for several days with a refractory pain crisis would you ever consider transfusion, either simple or possibly exchange in an attempt to get the patient over the crisis?
While chronic transfusion therapy has been shown to prevent acute vaso-occlusive pain episodes (see studies that used chronic transfusion to prevent primary and secondary stroke), there are limited data to determine if they are effective for treatment of acute pain caused by vaso-occlusion. There ha...
What is your local control approach to localized relapse of Ewing sarcoma in a site that was previously unresectable and received definitive radiation?
It depends obviously on some other factors including disease-free interval, the site itself, the dose of radiation received, symptoms, etc... Generally, a combined modality approach is attempted. Second-line chemotherapy with either high-dose ifosfamide, irinotecan/temozolomide, or topotecan/cycloph...
Would you use a matched sibling donor with a germline heterozygous BRCA1 mutation for stem cell transplant?
This is a challenging question due to the lack of clarity around the risk of hematologic cancers in patients who have BRCA1 mutations if patients with Fanconi Anemia are excluded. The role of BRCA haploinsufficiency in marrow hematologic function in otherwise healthy donors is unclear. Multiple stud...
How do you approach diagnosis and treatment of HLH/MAS following CAR T-cell therapy?
I maintain that immune effector cell associated hyperinflammatory syndrome is NOT HLH. Most patients post CAR-T cell therapy fulfill the criteria for HLH even if they don’t have hyperinflammatory syndrome, so it makes diagnosis very challenging. Many patients with this “HLH-like” hyperinflammatory s...
In the setting of secondary HLH associated with initial diagnosis of lymphoma, would you adjust steroid dose and type to account for differences in HLH and lymphoma treatment protocols?
I don’t necessarily recommend changing the steroid dose, unless symptoms don’t resolve with chemotherapy. I do recommend using an etoposide-containing lymphoma therapy. Many patients will respond well to lymphoma therapy and have recurrent symptoms before the next cycle is due; in those patients, I ...
What is your approach to treating a young adult with severe aplastic anemia unresponsive to steroids and TPO-mimetics?
My choice would be an HLA haploidentical marrow transplant.
How do you approach dosing of anakinra in MAS?
We generally start with dosing per the rheumatology guidelines of 100 mg/day (1-2 mg/kg in children) subcutaneously. Based on case reports, if this is insufficient to control the hyperinflammation, can be increased (see Ajeganova et al., PMID 33281955).
Can CAR T cell therapy be considered for heart transplant recipients with concurrent lymphoproliferative disorders?
The short version of my answer is: Possibly Yes. There is a very limited evidence from the literature in this topic, however, I will base my explanation on the following case reports: McKenna et l., PMID 37129856, Dang et al., PMID 33002249, Portuguese et al., PMID 36575360.The provided case reports...
Given recent studies investigating ICI myocarditis biomarkers such as Temra CD8+ cells, is there now a growing selection of clinical biomarkers, besides BNP and troponin, being measured routinely in ICI myocarditis cases?
At this time, Temra CD8+ cells have been shown in research experience only and not measured routinely. We hope that in the near future, we will have better, more specific biomarkers than troponin and BNP for the diagnosis of ICI myocarditis given how difficult it is. Diagnosis of ICI myocarditis is ...
Do you check asparaginase levels for all patients receiving receiving E. coli-derived products, or only in certain clinical situations?
Yes, we do. We routinely check levels one week after each dose of long-acting asparaginase. There are several recent reviews which make this recommendation. The main reason is to detect silent inactivation, which is reported to occur after 1-5% of administrations. In some obese patients especially w...