Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What is your preferred treatment agent for type 1 von Willebrand patients needing minor procedures if they have a history of severe hyponatremia with DDAVP?
I would avoid DDAVP. I typically individualize hemostatic management based on the procedure- related risk of bleeding and severity of the VWD. For example, for dental extraction, tranexamic acid alone may suffice; however, communication with the proceduralist to use topical agents such as topical th...
Have you used oral vancomycin as prophylaxis for C difficile infection in patients admitted for allogeneic hematopoietic cell transplant?
We also use oral vancomycin as secondary prophylaxis for anyone who develops C difficile infection, at a dose of vancomycin 125 mg PO BID, for up to 7 days after concurrent antibiotics are discontinued. This recommendation is based on Morrisette et al., PMID 31255741, a retrospective cohort study of...
Would you consider TPO mimetics for chemotherapy induced thrombocytopenia that persists despite dose reductions?
The reported evidence on using a TPO-RA in chemotherapy-induced thrombocytopenia (CIT) is complicated but this seems like a situation where it is worth trying a TPO-RA. Repeated dose reduction and/or delay is not good for treatment of a cancer in general. It is very important to individualize the ca...
How do you approach and manage anorexia and appetite loss in people with advanced cancer?
Anorexia/cachexia is often distressing to patients and families and it is this distress that is the target of many of the interventions for this syndrome as there are, in general, no effective therapies. Patients and families are routinely battling over the lack of eating as this causes further disc...
Do you routinely screen and replace Vitamin D in patients with ITP?
I do not generally screen for vitamin D deficiency in ITP patients, but do recommend that all patients undergo routine screening as recommended by the American Academy of Pediatrics (pediatric provider) and have vitamin D deficiency identified and managed as appropriate for age in general, regardles...
What diagnostic workup is recommended when immune thrombocytopenia is present in multiple family members?
Genetic testing and confirmation that this is indeed immune thrombocytopenia and not a familial thrombocytopenia syndrome.
What is your preferred frontline regimen for metastatic unresectable fibrolamellar carcinoma in an AYA patient?
We have successfully used GEMOX-LEN along with multidisciplinary locoregional tumor debulking to convert more than 50% of unresectable stage IV FLC to definitive R0 resection and surgical remission, nearly 100% achieving 0.00 circulating tumor DNA (we use Signatera): Ginters et al., GI Cancer Trial...
What would be your next line of therapy for a pediatric patient with T-ALL in first relapse who has not received a transplant?
These patients can be challenging to bring into remission, and a multi-agent salvage regimen should be considered. If the patient has not previously received nelarabine, it should be incorporated; re-treatment may also be considered in select cases. During the first cycle, dasatinib may be added in ...
Where do you anticipate positioning Mim8 (denecimig) alongside existing options within your hemophilia A prophylaxis approach, assuming regulatory approval (FRONTIER2)?
It is hard to say at this point, but I suspect it will be similar to emicizumab - i.e., it will be more frequently than the currently available rebalancing agents. What remains to be seen is if it will replace emicizumab by way of better perceived efficacy or only if someone is deemed to have "faile...
What is your approach to screening a cancer survivor for iron overload, and what is your treatment of choice?
We check the ferritin level after completion of chemotherapy. If ferritin is >1,000 ng/ml, we recheck the level as it can be falsely elevated with inflammation/infection. If ferritin is >1,000, we obtain a liver MRI with iron quantification. If liver iron concentration (LIC) is > 5 mg/g dry weight, ...