Pediatric Hematology/Oncology
Clinical discussions on pediatric blood disorders, childhood cancers, and specialized treatment protocols.
Recent Discussions
What is the role of upfront addition of an ALK inhibitor in an ALK positive mature B Cell lymphoma in an AYA patient?
Crizotinib has gained FDA approval in pediatric patients > 1 as monotherapy for relapsed anaplastic large cell lymphoma (ALK+) based on the COG ADVL0912 study. The agent was subsequently studied in upfront ALCL with aggressive chemotherapy based on the European ALCL99 regimen in ANHL12P1 but there w...
How would you approach immunosuppression for patients with severe aplastic anemia who are not transplant or ATG candidates?
There are relatively few alternatives. One is just supportive care with transfusion, antibiotics, etc. Many patients will adapt to low Hb and do ok with low platelets and do not require therapy. A calcineurin inhibitor alone or with eltrombopag can also be used - the response rate is not as good as ...
How would you manage asymptomatic brain metastases from testicular non-seminomatous germ cell tumor with a choriocarcinomatous component?
Despite the obvious issue of the blood brain barrier, BEP chemo alone (or VIP) is sufficient for treating such patients. We would, however, repeat head MRI just before starting second course of cisplatin combination chemotherapy to ensure appropriate response.
Should assessing for a MTHFR mutation be part of the thrombophilia evaluation in a patient presenting with a thrombus?
MTHFR mutation testing is of very little utility, given that the functional readout of MTHFR dysfunction is homocystinuria. The intake of the treatment of homocystinuria is folate, which is ubiquitous in the US diet. Thus, homocysteine levels would be adequate for testing, if such testing is indicat...
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...