Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
How would you manage metastatic alveolar soft part sarcoma in an young patient failing TKI?
I am going to assume that the patient has either progressive or symptomatic disease, or at least a legitimate concern for any impending complication/s requiring therapy. There is clearly an emerging body of data, presented and published, supporting activity of checkpoint inhibitors in ASPS. Prelimin...
What adjustments, if any, do you make to chemotherapy regimens for patients with chromosomal breakage disorders who develop malignancy?
Patients with known DNA repair disorders (such as Ataxia-Telangiectasia, Nijmegen Breakage Syndrome) are at high risk of developing toxicity from standard chemotherapy approaches for the treatment of their leukemia/lymphoma, which is the most common malignancy they are predisposed to. Agata Pastorcz...
In which platelet function disorders would you consider use of Novoseven as opposed to platelet transfusion?
There are now two recombinant activated factor VII (rFVIIa) available in the US. Trade names are NovoSeven RT and SEVENFACT. NovoSeven RT is US FDA approved for use in Glanzmann thrombasthenia. There are case reports of its efficacy in off label use for other inherited platelet function defects. SEV...
How do you approach treatment of metastatic hepatocellular carcinoma in an adolescent?
Clarification - do you mean fibrolamellar carcinoma? Often mistakenly treated or referred to as a HCC variant or as a subset of HCC? Does it have the characteristic DNAj-PRKACA fusion for FLC? Is there underlying liver disease? Or do you mean conventional HCC? (common in older adults with liver dise...
Under what circumstances would you consider anticoagulation in a young female patient with persistently elevated factor XI activity?
First, get a baseline D-dimer to see how procoagulant she is at that point. If elevated, long travel on plane, pre-op and post-op for 2 months - consider short-term anticoagulation. If past thrombosis - give lifelong anticoagulation. If pregnant - follow D-dimer; if it goes up, anticoagulate.
How do you approach treatment of vasculitis in a sickle cell disease patient?
Will highly depend on the vasculitis type and the acuity of the situation. For ANCA vasculitis, approaches based on rituximab and low glucocorticoid doses might be attractive (granted not severe disease, e.g. RPGN, DAH which should require pulse glucocorticoids). For large vessel vasculitis, a si...
Is there utility in administering adjuvant chemotherapy to a patient with relapsed osteosarcoma for whom CR2 is achieved after surgical resection alone?
Multiple trials have attempted to improve DFS/OS by implementing adjuvant chemotherapy or targeted agents in this scenario. All of these trials have been NEGATIVE. There is no evidence to support the use of adjuvant chemotherapy in patients who achieve complete surgical response after relapse. Nonet...
What is the maximum pRBC volume you can transfuse when performing a manual exchange transfusion in an adult-sized patient with sickle cell disease?
It depends on how much hemodynamic stress the patient can tolerate and the rate of the phlebotomy. The rate is usually 30 minutes for every 500 cc whole blood, but may need to slow down in smaller-sized patients (e.g. 50 kg or less), patients with history of pre-syncope or syncope with phlebotomy, a...
How do you approach management of sickle cell patients who mistrust Western medicine and prefer naturopathic therapies?
With compassion and understanding, I would explain that the lifespan of patients with SCD in regions with access to Western medicine far exceeds that where the disease is most prevalent. Controlled clinical trials have proven the utility of hydroxyurea to alter beneficially the course of disease and...
In which patients with oncologic or hematologic disorders are you recommending a 3rd dose of mRNA COVID vaccine?
In the ideal world, we would tailor the need for booster shots based on whether or not a patient achieves an appropriate immunologic response and maintains that response for long periods of time. This would include both seroconversion and T-cell-mediated immunity. However, we have neither routine no...