Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What is your approach to iron supplementation when ferritin levels remain stagnant despite reported good compliance?
This is a really good question. From my reading of the literature, there seems to be an association between a low ferritin level and restless legs syndrome. Not true iron deficiency as defined by a low transferrin saturation, anemia, or a low MCV. I could not find any definitive evidence for a cause...
What is your approach to systemic treatment of a rapidly progressing, symptomatic, unresectable MPNST arising from a plexiform neurofibroma in a patient with NF1?
MPNST is a different ballgame than plexiform neurofibroma. Unfortunately, it is not chemotherapy-sensitive. We currently do not have a good option for MPNST. Perhaps clinical trial enrollment works best. We also do NGS to look for targets, apart from NF1. MEK inhibitors have been tried in the past w...
What are your top takeaways in Sarcoma from ASCO 2026?
I really enjoyed the panel presentation “Unique Challenges in Sarcoma Care for Adolescents and Young Adults", (Jessop et al., ASCO 2026). I also enjoyed “A Multidisciplinary Case-Based Approach to Non-Rhabdomyosarcoma Soft Tissue Sarcoma in Adolescents and Young Adults: Challenges and Emerging St...
Has your practice changed to PLEX-free initial therapy for iTTP?
I am not. The reason is that caplacizumab is not on formulary at my institution, and so implementing PLEX rapidly while obtaining caplacizumab (which typically arrives in 24-48 hours) is my current practice. If I had caplacizumab on formulary, I would utilize it as it was utilized in the MAYARI tria...
Would you use the pneumococcal conjugate-21 vaccine (Capvaxive) instead of the conjugate-20 (Prevnar-20) for routine vaccinations in immunosuppressed patients?
PCV-21 was recently approved by the FDA and supported by ACIP. At this early stage (August 2024), CDC has not finalized guidance on PCV-21, so we do not know how the vaccine schedule will be changed. An important distinction is that PCV-21 covers different serotypes of pneumococcus, as outlined in t...
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...
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 ...