Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
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, ...
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.
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...
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...
What is your approach to managing iron overload in children with transfusion-dependent beta thalassemia who have adherence challenges or toxicity with standard chelation regimens?
I would divide the adherence issues into two populations. The younger children where a caregiver is responsible for administering the chelation, and adolescents where caregivers have passed on the responsibility to the patient. For the former, adherence is reinforced with an explanation of the possi...
What are effective ways to improve compliance/involvement in long-term cancer survivorship care for patients, particularly AYA patients?
As a consequence of exposure to chemotherapy, radiation, and surgery, many survivors of childhood cancer are at high risk for a myriad of chronic conditions compared to age-matched controls. Follow-up in a structured late-effects program can improve survivors' knowledge of their risks, assist with a...
What screening measure on an echocardiogram is most helpful for identifying anthracycline-induced cardiomyopathy?
Cardiotoxicity following cancer treatment can manifest as a range of complications, including left ventricular dysfunction, cardiomyopathy, heart failure, myocarditis, valvular disease, and arrhythmia. Accordingly, careful evaluation of cardiac function in patients receiving chemotherapy is essentia...
How do you screen (imaging, modality) for bone density changes following oncologic treatment in childhood cancer survivors?
Certain survivors of childhood cancer are at risk for reduced bone mineral density, especially those exposed to extensive corticosteroids or radiation, hypogonadism, growth hormone deficiency, and those who have had reduced weight-bearing/mobility for an extended period of time. It is important to s...