Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
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 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?
We see our impact in genomic scoring system being used to compare the impact of different and evolving tumor genomic sequencing platforms as the NGS assays (and soon cell free tumor (CT) DNA NGS platforms) are constantly being refined and used for clinical care decisions. Some platforms are more com...
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...