Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What treatment regimen would you recommend for young adults with platinum refractory relapsed germ cell tumors?
We assess the patient risk by IPFSG score, and if it is high score or has extragonadal primary site then we consider tandem high dose chemotherapy and autologous transplantation https://onlinelibrary.wiley.com/doi/full/10.1002/acg2.47 The data sometimes is unclear on the role of HDCT . - the larges...
What cardiac surveillance would you do for an asymptomatic male patient with a remote history of mantle field radiation for Hodgkin's lymphoma?
I would follow the long-term follow-up guidelines from COG based on heart dose - Children's Oncology Group (survivorshipguidelines.org)
How is transferrin saturation a reliable indicator for any parameter if serum iron is not reliable?
The question is a very good question. The Fe/TIBC must be drawn on an overnight fast including any vitamin pills containing iron. Otherwise, the serum iron is speciously elevated which in turn speciously elevates the TSAT. If those conditions are met, the TSAT is as good as the transferrin receptor ...
What subset of sickle cell disease patients are you offering sickle cell disease gene therapy?
All patients with Hgb SS and patients with Hgb SC who have had any significant complications, excluding chronic pain.
When do you refer AYA patients with newly diagnosed severe aplastic anemia for transplant?
In a young person, my first thought is to obtain telomere lengths to be sure he/she does not have a telomeropathy. This has significant implications both with and without transplantation. Certainly, if telomeres are short, allogeneic transplantation is preferred since there is less likelihood of res...
What was the rationale behind using RANO-HGG criteria instead of RAPNO criteria for the primary endpoint in the FIREFLY-1 trial for pLGG?
I think this was a mistake, but I understand that this was requested by the FDA. We all know that MEK and BRAF inhibitors change the pattern of enhancement within days/weeks after the beginning of the treatment. As RANO-HGG is essentially based on the assessment of enhancing measurable and nonmeasur...
How would you treat a stage 1a Sertoli-Leydig cell tumor with heterologous elements?
Assuming this is an ovarian tumor, the patient should be evaluated for a Dicer 1 mutation (Schultz et al., Clin Cancer Res 2018)If the tumor is completely resected and is a stage 1a tumor and the patient is well, then I would consider observing the patient after resection without further therapy (Sc...
How do you manage hyperalgesia in patients with sickle cell disease?
In addition to the obvious, such as PCA narcotics, Lyrica/Neurontin, and Toradol, I have success with IV Tylenol, Zyprexa, po L-Arginine (Morris et al., PMID 23645695) as well as Dexmedetomidine (Precedex), steroids (NEJM: Griffin et al., PMID 8107739) and acupuncture [Johnson et al., Alternative an...
What hemoglobin level do you target for adult beta thalassemia intermedia patients with a pre-transfusion Hb >7 g/dl?
If they have been evaluated by a thalassemia expert and considered to be transfusion dependent then the target minimum pre-transfusion is 9.5 -10, but if that hemoglobin is not adequate to improve quality of life and the patient feels the need for higher hemoglobin to catch with daily work and activ...
How has the recent FDA approval of tovorafenib impacted your treatment decisions for pediatric patients with relapsed/refractory low-grade glioma?
With the FDA approval, and given the tolerability of therapy and the pediatric friendly once weekly dosing and formulations, I will prescribe tovorafenib regularly for my patients with relapsed refractory low grade glioma. I do think it will become a standard choice in this setting. That said, given...