Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
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, ...
When do you refer patients back to their PCP for the predominant management of their medical care following completion of oncologic or BMT treatment?
Transitions of care are always challenging, especially for patients with complex medical histories, including cancer or stem cell transplantation. There are many different models for how and when to transition patients back to primary care or shared care. The ongoing, often complex needs of survivor...
Do you discuss fertility preservation options with all pediatric oncology patients at the time of diagnosis, and if not, what determines who and when you discuss options?
Yes. We now have a formal fertility preservation program in our institution that was started by and is based in our oncology division. The goal is to discuss fertility preservation options with all patients at the time of diagnosis, regardless of age or prognosis.
What strategies do you find helpful in advanced care planning with patients/families who are very "miracle" centered?
Hope for the miracle yourself! Broaden: “Are there any other things you are hoping for?” Hope for the best, prepare for the worst: “I see how much you want a miracle. I wonder if we can talk about what we should do if this doesn’t happen.” Consider involving a religious leader if relevant.
How do you approach the treatment of HSCT-associated thrombotic microangiopathy?
TMA can be due to multiple insults: If the patient is on tacrolimus or cyclosporine, the dose should be reduced. These drugs cause the renal afferent arterioles to spasm, and RBC fragmentation can occur on that basis. It typically responds to a dose reduction If the patient was conditioning with TB...
What is your treatment approach for a pediatric patient with H3K27M-mutant diffuse midline glioma following progression after radiation therapy?
First, if the patient is at least six months from initial radiation and has had a reasonable initial response, reirradiation is the best proven treatment for recurrence. We would also encourage enrollment on a clinical trial (the DMG National Tumor Board is a helpful resource for determining for whi...
How do you approach deciding which patients with hemophilia B to recommend for gene therapy?
There is no standard protocol for who gene therapy is recommended for, but certainly, potential candidates must meet all criteria according to the manufacturer's recommendations. Gray areas may exist if there is some preexisting liver disease, where hepatology consultation is desired to help determi...