Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What are the perceived implications of broader genomic sequencing as it pertains to interpreting variants of unknown significance and germline predisposition mutations?
Engaging families in a discussion of genomic and or germline testing and the possible positive, negative and VUS results is critical to assisting their understanding of tumor sequencing and paired tumor-germline sequencing. Partnering with genetic counselors to ensure language is used that helps fam...
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?
Why not try hormonal IUD or implant as long as the patient is willing to try (as almost no risk of thrombosis)?
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 ...
Does "preoperative rupture" always necessitate whole abdomen RT for Wilms' tumor?
This is controversial. Theoretical arguments aside, our committee (COG) feels strongly that for any preoperative rupture, whole abdomen RT is required.
What is the role of PD-1 inhibitors in relapsed B-cell ALL?
The role of PD-1 inhibitors in pedi is currently being explored in relapsed B-ALL in the study AALL1821. No interim results have been posted.Primary samples from pediatric patients with relapsed B-ALL were shown to demonstrate marked up-regulation of PD-1 on T-cells, and up-regulation of PD-L1 on th...
How would you approach cytopenias 5 weeks after initial dosing in a young patient with MDS treated with Azacitadine as a bridge to transplant?
Bridging with HMA to transplant in MDS patients is a common practice although it did not show improved outcomes. You will definitely have to r/o other potential causes of pancytopenia (i.e., infections, etc). I would repeat a BM A/Bx to make sure that blasts are not increasing (< 20% and preferably ...
What would your next line treatment be for a patient previously treated with repeat resection, craniospinal irradiation, and initial cisplatin/vincristine/CCNU with medulloblastoma?
As you will agree, this is an extremely unfortunate situation. I am assuming this patient has progressed either while on therapy or soon after completion? Depending on his age, a potential option is to enroll him on any available clinical trials. PBTC58 study uses intrathecal omburtumab along with c...