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...
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 ...
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...
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...
How do you counsel families of prepubertal girls with an oncologic diagnosis about cryopreservation of ovarian tissue?
One of the biggest current issues with ovarian cryopreservation (OTC) in the US is that there is no consensus yet about who should receive OTC, and practice varies across the country. In addition, many patients are diagnosed and treated at centers that do not have oncofertility programs and do not o...
Do you do prophylactic LP/IT chemotherapy in high risk APML prior to starting consolidation?
Extramedullary disease such as CNS involvement is quite uncommon at diagnosis in acute promyelocytic leukemia (APL). However, it can be seen in patients with relapsed disease. Both isolated CNS relapse and CNS relapse associated with morphologic or molecular relapse can occur. Yet one has the impres...
Do you see a role for omidubicel upfront in severe aplastic anemia for patients without a matched donor?
We typically use cord blood products if there is no available family member donor or reasonably matched unrelated donor. The use of haploidentical family donors is very effective, less expensive, and allows supplementation with CD34+ stem cell boosts if counts are suboptimal DeZern et al., PMID 3234...
What is the expected timeframe for the development of radiation myelitis and therapies that have helped with neurologic symptoms?
The incidence and the timeframe of the development of radiation myelopathy are influenced by total radiation dose, radiation dose per fraction, time between courses of radiation, and associated chemotherapy or immunotherapy. Older age, the presence of diabetes, and previous exposure to radiation are...
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...