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...
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...
Do you feel that more frequent genomic sequencing will be cost-effective in the management of high-risk pediatric cancers?
Currently, the increased utilization of tumor genomic sequencing in pediatric cancer (which I assume you mean by “more frequent sequencing”) has resulted in more companies and academic practices implementing NGS platforms and on-site sequencing. This increased clinical use leads to an increased numb...
What is your current practice around genomic sequencing for pediatric cancers?
As mentioned in our article, we currently offer tumor genomic sequencing to patients who have high risk pediatric cancers at diagnosis who do not routinely undergo molecular characterization as a standard of care diagnostic procedure and for pediatric cancer patients whose tumors are refractory to t...
What are the perceived implications of broader genomic sequencing as it pertains to interpreting variants of unknown significance and germline predisposition mutations?
Variants of unknown significance (VUS) are constantly being revised when it comes to identifying “potentially pathogenic mutations.” We work with a molecular pathologist as well as the pathologists at Exact Sciences to understand in real time where VUSs fall in regards to their pathogenicity and act...
How do you envision genomic scoring systems for pediatric cancers being applied in a real-world setting?
I think scoring systems like these are promising, but they must be validated before being ready for application in the clinical setting. A scoring system can be helpful for clinicians, patients, and families, but only if it is based on verified, valid data, which is easier said than done. Many such ...
In a patient with Type I von Willebrand disease with history of VTE and heavy menses, would tranexamic acid be a treatment option?
No, with thrombosis one would not inhibit the thrombolytic pathway. Would anticoagulate with Coumadin with low INR 1.5 to 2.
When do you stop trying to eradicate an acquired factor VIII inhibitor?
First, to be clear, the answer to this question is not well studied in any population, and the clinical setting (i.e. age, titer - historical max or current), and the nature of the complications isn't detailed here. I presume "no causative etiology" includes "not postpartum, and not accompanied by (...
How do you explain TTP to patients?
I tell them there is an enzyme in their blood called ADAMTS13 that helps keep their platelets from sticking together and plugging small blood vessels, and that for unknown reasons, their own immune system is destroying this enzyme. The symptoms of TTP are caused by clumps of stuck-together platelets...
Do you recommend continuous antibiotic prophylaxis for patients on complement inhibitors such as eculizumab?
This is an extremely important and timely question. There simply isn’t enough data or firm guidelines on this leading to different practices. The reality is that there have been a number of meningococcal breakthrough infections in those vaccinated against meningococcal disease. Complement therapies ...