Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
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?
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...
How do you work up a young patient with increasing ferritin and normal TSAT without infectious, inflammatory, or liver disorders?
This is a tough one. If the ferritin is increasing and the TSAT is normal and there is no evidence of hemochromatosis, the ONLY possible explanation is some underlying morbidity, inflammatory, rheumatologic, malignant, or infectious is present. In pediatrics, HLH (hemophagocytic lymphohistiocytosi...