Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
Based on the FIREFLY-1 data, would you consider using tovorafenib monotherapy front-line in pediatric patients who have low grade gliomas that are only amenable to subtotal resection or are unresectable?
Not yet. I think it is important to wait for the results of FIREFLY-2 and also ACNS1831/ACNS1833. We have to keep in mind that the combination of trametinib and dabrafenib is already FDA-approved as frontline treatment for paediatric patients with BRAF V600 mutated LGG. This approval was based on th...
Should testing for genetic causes of HLH be performed in all patients with MAS or secondary HLH regardless of the patient's age?
Familial HLH (fHLH) is a pediatric disease. Therefore, there is no place for genetic testing to establish a diagnosis of fHLH in adults, and treatment for HLH should not be delayed while waiting for genetic testing. However, there are hypomorphic polymorphisms in the fHLH genes that may be a contrib...
How would you counsel patients with personal or family histories of autoimmune disease on immune checkpoint inhibitor therapy for Hodgkin lymphoma?
This is also a tough question. I think patients with autoimmune endocrinopathies (especially Hashimoto’s or Type 1 DM) on stable, longstanding replacement regimens, as well as pre-existing vitiligo, are reasonable candidates for frontline PD-1 based therapies, although they certainly bear very close...
What are your top takeaways from ASH 2023?
Gene therapy for sickle cell disease and beta thalassemia. This was the first approval of CRISPR/Cas-based therapy in humans. Ex-vivo engineering of isologous hematopoietic stem cells followed by their reinfusion after myeloablative conditioning led to induction of 40-50% fetal hemoglobin in patient...
What is the role of radiation therapy in recurrent Rosai-Dorfman disease after surgical resection, which manifests as many cutaneous lesions on the arms and buttocks?
I agree with Dr. @Dr. First Last. When I was in training, we reviewed the cases at MD Anderson. Radiation was only used for 9 patients (infrequently), and local control was achieved in 4 patients. So very small numbers, with uncertain benefits. Median RT dose 36 Gy. In the setting of multifocal dise...
Do you recommend the HPV vaccination for your young patients with HPV-associated cancers or pre-cancers who have not yet been vaccinated?
Yes, worth getting vaccinated to reduce risk for recurrent dysplasia. Lichter et al., PMID 32282601. Di Donato et al., PMID 33919003. Jentschke et al., PMID 32762871.
Are there contraindications to growth hormone therapy in patients who have completed treatment for pediatric cancer?
While there are theoretical and anecdotal concerns about recombinant growth hormone stimulating tumors, multiple reviews have concluded that there is no increase in tumor recurrence or second malignancy in patients treated with GH therapy for GH Deficiency after pediatric cancer treatment. In 2022, ...
What is your standard diagnostic workup to confirm GVHD in a patient post-BMT with skin rash and jaundice?
I assume there is no diarrhea, so sigmoidoscopy would not be helpful. Gut and liver pathology are useful. Skin biopsy is less helpful. It can be fairly nonspecific, but we do it to rule out other diagnoses that have more definitive pathology. Ultimately, it is a clinical diagnosis. Liver biopsy woul...
Does the use of A+AVD versus ABVD affect your decision for consolidation RT for bulky Hodgkin lymphoma?
A+AVD is an acceptable regimen for advanced HL based on results from the ECHELON-1 study (Ansell et al., PMID 35830649) showing an improvement in both PFS (82% vs 75% at 6 years) and OS (94% vs 89%) compared with ABVD. Radiation therapy was not incorporated into this study.In advanced HL, regardless...
How do you approach conversations regarding discontinuation of transfusions in patients with advanced hematologic malignancies who are otherwise appropriate for hospice?
While not an expert in leukemia care or MDS, the answer is nuanced (both in the care of patients and to hospice agencies). Some hospice programs will make exceptions to blood transfusions depending on how frequent - so it is always good to ask. From a clinical perspective, important to consider whet...