Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
Following long-term efficacy and safety data from the beti-cel trials, how do you approach gene therapy for eligible patients with transfusion-dependent β-thalassemia?
The results with beti-cel are excellent. So are the results with exa-cel. These two gene therapies use different approaches to modifying hemoglobin production in erythroid stem cells. Beti-cel adds a modified hemoglobin that resembles fetal hemoglobin using viral-mediated transduction, whereas exa-c...
How do you approach treatment of a young adult with an intracranial anaplastic ependymoma and a single intradural cervical spine metastasis?
A high-grade tumor, such as anaplastic ependymoma, carries a higher risk of CSF seeding down the neuroaxis than lower grades. Secondary spinal drop metastases are considered a serious but relatively rare complication in this disease. Assuming that the metastasis was detected at the time of diagnosi...
Under what circumstances would you consider omitting radiation in patients with early stage, unfavorable (bulky) Hodgkin Lymphoma?
We should first acknowledge that combined modality therapy improves progression-free survival in early-stage HL compared with chemotherapy alone. Stated more succinctly- if you give combined modality therapy, there is a lower risk of relapse; if you give chemotherapy alone, there is a higher risk of...
How do you manage spinal cord compression in a new suspected neuroblastoma diagnosis?
While there is evidence that chemotherapy, surgery, and radiation can all be effective for some patients,1 there are no definitive studies or data that prove the best approach for all patients. A 2017 systematic review concluded that the “currently available literature remains suboptimal as a guide ...
Should extra precautions be taken for pediatric oncology patients in light of the recent COVID-19 outbreak?
I have not personally seen cases. This is not surprising, however, because our patient population is more susceptible to severe outcomes from respiratory infections. This is compounded by weakened immune function and, for some patients, cardio respiratory damage.
How should you manage a pediatric oncology patient who has an ANC > 500 and a normal chest x-ray but is confirmed to be infected with COVID-19 and is immunosuppressed from chemotherapy?
The treatment for pediatric patients with cancer who develop COVID-19 is very poorly defined. The risk of severe disease is unknown because although adults with cancer appear to have worse outcomes than those without, non-immunocompromised children seem to have few severe outcomes from the disease a...
At what cumulative anthracycline dose is it recommended to use dexrazoxane for cardioprotection?
Typically considered in patients with anticipated >300 mg/m2 doxorubicin, as has been mentioned, which is most commonly seen in patients with sarcoma or recurrent lymphomas. Great discussion re: evidence and considerations in this piece in JACC Cardio-Oncology Upshaw et al., PMID 38773999.In practic...
Is dexrazoxane recommended for cardioprotection in AYA patients with Hodgkin lymphoma who are receiving anthracyclines as part of their upfront therapy?
This is a good question that reflects the growing body of evidence in favor of dexrazoxane cardioprotection being safe and beneficial (Chow et al., PMID 26014292; Getz et al., PMID 32343641). For AYAs with Hodgkin lymphoma, doxorubicin remains a key component of therapy even for lower stage disease ...
Can an AYA patient with newly diagnosed AML safely sperm bank after starting cytoreduction with hydroxyurea or cytarabine?
Thanks for the question. Unfortunately, there is not a lot of data available to answer this question with certainty. Because of that, I err on the side of saying that this should not be done. The concern lies with the possible impact on the sperm DNA, and the subsequent effect that might have on a p...
What is your approach to optimizing pre-operative hemoglobin in patients with sickle cell disease?
This is always a question that has to be hyper-individualized. The research, and therefore the associated professional guidance, is thin. The American Society of Hematology (ASH) 2020 Guidelines for Sickle Cell Disease: Transfusion Support and the NHLBI Expert Panel Report (2014) are the only profes...