Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
Should post-transplant cyclophosphamide be utilized for all matched unrelated donor peripheral blood stem cell transplants for hematologic malignancy?
In myeloablative transplantation, PTCY did not have an advantage over Tac/MTX as demonstrated in the BMT-CTN 1301 study which compared PTCY, Tac/MTX, and T cell depletion (Luznik et al., PMID 34855460). One caveat is that in this trial, PTCY based prophylaxis did not include Tac or Tac/MMF.BMT-CTN 1...
How do you counsel patients with acute intermittent porphyria when it comes to fasting for religious reasons?
This is a very difficult question and it depends on the individual patient, their disease severity, their particular triggers, and how they have done in the past with caloric restriction. The patient may want to speak with a clergy member for guidance in this situation as well. For inst...
How do you workup splenomegaly related to possible hematologic etiology in the absence of abnormal blood counts, adenopathy or severe constitutional symptoms?
The presence of splenomegaly is an important finding found either on physical exams or by imaging. As noted in the question, the initial work up includes physical exam looking for lymphadenopathy. In addition, laboratory evaluation, including absolute white count, and differential may help explain w...
In pediatric patients with low-grade gliomas showing prolonged response or stable disease on tovorafenib, how would you adjust the duration of therapy?
This is a one-million-dollar question. As far as I know, the design of FIREFLY-2 includes a fixed duration of treatment, and this is also the case for ACSN1833 (selumetinib trial). It will be important to look at the PFS of the patients who discontinued tovorafenib and compare with the results of ch...
Do you recommend using BRAFV600E mutation status to risk stratify treatment for a pediatric patient with langerhans cell histiocytosis?
It’s certainly useful information. Even if you don’t use a BRAF inhibitor as first line therapy, you will have that option in your back pocket.
How would you approach langerhans cell histiocytosis involving a single bone site after resection?
The outcomes for single-site LCH in adults are generally excellent. However, there is still a small risk of relapse. At this time, factors predicting relapse are unknown. It is recommended to ensure that it is truly a single-site disease by FDG PET scan and there is no diabetes insipidus. If yes, th...
How do you manage an adult primary rhabdomyosarcoma of the lung/mediastinum?
COG trials include all patients <50 years old. The 5-year OS of patients treated in the most recent COG trials is over 70%. So, I would recommend you treat the adult patients per COG protocols if you do not enroll your patients in the COG trials. To manage rhabdomyosarcoma, we need to know the risk ...
How is your experience with point-of-care INR systems for home monitoring of vitamin K antagonists?
Point of care (POC) INRs are reliable and can be used to monitor patients once their INRs have been stabilized on warfarin. In fact, there is data on better time in the therapeutic range for self-testing and also self-management of dosing. However, this needs to be done in conjunction with a warfari...
What, if any, cardioprotective measures beyond dexrazoxane may be used to minimize cardiac risk during or after chemo- or radio-therapy?
For cardioprotection, I would recommend: Baseline cardiovascular risk stratification, can use the HFA-ICOS risk calculator (https://www.cancercalc.com/hfa-icos_cardio_oncology_risk_assessment.php), consider alternative chemotherapies if very high risk and alternative therapy equal efficacy. Optimiz...
What infections should we rule out in patients who develop diarrhea on immunotherapy?
Immunotherapy is known to be associated with immune-mediated diarrhea and colitis (IMDC). However, it remains unclear whether cancer patients undergoing immunotherapy are at a heightened risk for Clostridioides difficile colitis infection (CDI). A retrospective study focusing on patients treated wit...