Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
Do you routinely send NGS testing from bone marrow samples in patients with unexplained cytopenia or cytoses?
For patients with unexplained cytopenias in whom I suspect MDS or MDS/MPN, I will often obtain a broad NGS panel for myeloid malignancy gene mutations. The goal is to aid in diagnosis (and prognosis once the diagnosis is made), but I do not rely on the NGS panel alone to make the diagnosis. A bone m...
What recommendations do you have for a transgender female patient with history of prothrombin gene mutation who is interested in starting gender affirming hormone therapy?
A review of the literature suggests that the risk of VTE associated with hormone therapy in this setting is quite low, even in the presence of other risk factors for clotting (see, for example, Mullins et al., PMID 33753543). Furthermore, the presence of an asymptomatic prothrombotic genotype is rar...
Do you hold IV iron in the setting of active infection?
While there is no evidence of harm, there is enough conjecture about the danger to make it prudent to wait until infection is controlled. So yes, I do. Further because of the iron restricted erythropoiesis during infection, the efficacy is likely to be blunted.
When do you refer patients for germline testing when somatic tumor testing is negative for actionable mutations?
Somatic (tumor-only) testing should not be used to conclusively rule in or rule out the presence of a germline pathogenic/likely pathogenic alteration. While most germline sequence alterations (point mutations, small insertions/deletions) will be detected on tumor-only testing, this may miss chromos...
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.
How would you approach an early stage II unfavorable Hodgkins lymphoma following 6 cycles ABVD with persistent Deauville 5 with negative biopsy?
This is an active disease and should be treated accordingly. I would not wait. RT is certainly option number one now, but the patient has a considerable risk for recurrence even after RT, since he/she has Hodgkin‘s that is not responding adequately to treatment. Continuing with ABVD in a patient who...
How would you treat a locally-recurrent extraosseous (pulmonary) Ewing sarcoma?
It is possible to re-challenge the patient with the same regimen. Assuming EF and BNP are normal and can be followed, bolus Doxorubicin with Dexrazoxane would allow safe administration of additional doxorubicin. The alternative is to use high-dose Ifosfamide (14 g/m2/cycle). In the event of a good v...
How do you discern whether elevated liver enzymes are from immunotherapy versus chemotherapy when a patient is on combination chemo/immunotherapy?
There is no consensus on the best method for distinguishing the cause of elevated liver enzymes in patients being treated with ICPi's when combined with various chemotherapies. Important considerations include time of onset, severity, and presence of hepatobiliary metastases. Hepatotoxicity from ICP...
In patients with severe hemophilia A on emicizumab for prophylaxis, in case of noncompliance leading to a lapse of more than 2-3 months off therapy, do you reload with emicizumab?
In general, you need to restart the four-week loading then follow with maintenance. First, you need to find out why they were noncompliant (e.g. did not like/could not do subcutaneous injections, did not work, always noncompliant). If they are planning to continue noncompliance, this is not a good ...
Is there a role for IL2-receptor antagonists and TNF-alpha inhibitors in cytokine release syndrome as a complication of CAR-T or immunotherapy?
I came across a report from China regarding 3 patients who received BCMA CAR-T therapy, developed CRS, and were treated successfully with Etanercept based on elevated TNF-alpha. 2 of these patients did not receive tocilizumab before Etanercept which is not a standard practice. I'm not recommending u...