Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
When would you use proximal complement inhibitors like pegcetacoplan over terminal complement inhibitors for initial treatment of paroxysmal nocturnal hemoglobinuria?
I use either iptacopan or pegcetacoplan as primary therapy for PNH these days. Since its release, I have used mainly iptacopan. Its advantages are 1) it is oral, 2) the side effect profile is very tolerable, 3) it only inhibits the alternative pathway, thus is likely to be associated with less risk ...
What treatment would you give to a pediatric patient with rhabdomyosarcoma in first relapse?
Typically it depends on the previous treatment of the patient’s primary RMS in terms of my approach to relapse RMS. In general, many would go with treatment per ARST0921 with vinorelbine, cyclophosphamide, and Temsirolimus, with the caveat that it may be different depending on previous drug exposure...
Do you screen children with sickle cell disease for silent cerebral infarcts?
We screen children with sickle cell disease for SCIs at our center. This is because data suggest that silent cerebral infarcts (SCIs) are much more common than overt stroke and the long-term negative impacts of SCIs are significant. There are also treatments (e.g., chronic transfusion and stem cell ...
Given the data from SWOG 1826 suggesting that Nivo-AVD is likely the preferred regimen for advanced Hodgkin lymphoma patients, are there scenarios where alternative regimens may still be preferred?
Unless there was a contradiction to nivolumab (e.g., an active autoimmune disease), I would always favor N-AVD over BV-AVD- particularly in older patients.
Do you omit consolidative RT in pediatric patients with intermediate risk, non-bulky Hodgkin lymphoma who have a rapid early response to chemotherapy?
Yes, if the patients meet the rigid requirements for response, which include a rapid early response (Complete response or very good partial response) after 2 cycles of ABVE-PC chemotherapy AND have a complete response at the end of treatment then the data suggests similar outcomes whether or not the...
In massive transfusion protocol from suspected hemorrhage, is it worth obtaining a TEG to guide transfusion?
There really is no evidence (except expert opinion) on massive transfusion protocols and outcomes. There are a few trials showing that TEG or other viscoelastic tests reduce transfusion and even improve survival or other important outcomes in hemorrhage. So given the choice, if rapid point of care T...
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...